Relationships & Accomplishments – Clinical Trials Management

The New England Journal of Medicine Publishes Cologuard Plus™ Test Results from Pivotal BLUE-C Study

Dimitri Z — Wed, 13 Mar 2024 00:22:20 +0000

Cologuard Plus is only noninvasive test to be evaluated head-to-head against an independent fecal immunochemical test, which it significantly outperformed

20,000-participant BLUE-C study included 98 colorectal cancers and reflects racial and ethnic diversity of U.S.

Company will host conference call and webcast at 8:00 a.m. ET on March 14 to discuss study results

MADISON, Wis.–(BUSINESS WIRE)– Exact Sciences Corp. (NASDAQ: EXAS), a leading provider of cancer screening and diagnostic tests, today announced online publication of the BLUE-C study results in The New England Journal of Medicine. The peer-reviewed study, “Next-Generation Multitarget Stool DNA Test for Colorectal Cancer Screening”, will also appear in the journal’s March 14, 2024 print issue.

The 20,000-participant BLUE-C study was designed to determine the performance characteristics of Exact Sciences’ next-generation multitarget stool DNA test, Cologuard Plus, for colorectal cancer (CRC) and to compare that performance to the fecal immunochemical test (FIT), a commonly used noninvasive CRC screening test. Exact Sciences submitted to the U.S. Food and Drug Administration (FDA) its pre-market approval (PMA) application for Cologuard Plus in December 2023, including complete results from BLUE-C, and plans to make the test available in 2025, pending approval.

Cologuard Plus met all BLUE-C study endpoints, demonstrating 94% sensitivity for CRC at 91% specificity including non-advanced findings, and 93% specificity including no findings. Specificity was even better in younger age groups, at 96% in 45-54 year olds. Cologuard Plus will minimize unnecessary follow-up colonoscopies by reducing the likelihood of a false-positive screening test.

Results from BLUE-C also show Cologuard Plus significantly outperformed an independent FIT* for overall CRC sensitivity, treatable-stage CRC (stages I-III) sensitivity, high-grade dysplasia sensitivity, and advanced precancerous lesion sensitivity.

	Cologuard Plus1	Independent FIT1
Cancer sensitivity	94	67
	91	95
	93	96
	93	65
	75	47
	43	23

Note: specificity including non-advanced findings includes colonoscopy results that were negative for cancer or advanced precancer upon histopathological review; specificity including no findings includes no findings on colonoscopy and no histopathological review; high-grade dysplasia is a subtype of advanced precancer that includes carcinoma in situ or stage 0 cancer

To potentially expand the eligible screening population beyond average-risk patients, the BLUE-C study enrolled a small subset of participants with a first-degree relative with a history of CRC. Sensitivities for CRC and advanced precancerous lesions were similar among participants with a first-degree relative with a history of CRC and those without such a relative.

Among the subset of nearly 19,000 participants who were average-risk, without a first-degree relative with a history of CRC, Cologuard Plus exhibited 95% sensitivity for CRC and 43% sensitivity for advanced precancerous lesions at a 91% specificity including non-advanced findings, or 94% specificity including no findings.

“Cologuard Plus is highly sensitive for detecting colorectal cancer,” said Thomas F. Imperiale, MD, professor of medicine at the Indiana University School of Medicine, research scientist at the Regenstrief Institute, and principal investigator for the BLUE-C study. “This noninvasive test also detected a good proportion of the most advanced precancerous lesions, and did so with a low number of false-positive test results.”

The prospective, multi-center BLUE-C study utilized colonoscopy as a reference method, directly comparing Cologuard Plus and an independent FIT. BLUE-C investigators also collected blood samples for evaluation of a blood-based CRC screening test developed by Exact Sciences.

“BLUE-C’s publication in The New England Journal of Medicine reflects a decade of deep scientific and medical research in collaboration with Mayo Clinic,” said Kevin Conroy, chairman and CEO of Exact Sciences. “We’re eager to bring an improved, noninvasive colorectal cancer screening test to patients in Cologuard Plus, as colorectal cancer remains the most preventable, yet least prevented cancer.”

The BLUE-C study cohort was diverse and reflective of the U.S. population. About 40% of all participants identified as Hispanic or Latino, Black, Asian, American Indian or Alaska Native, or Pacific Islander. This enrollment diversity helps ensure that the BLUE-C findings and Cologuard Plus are relevant for all screen-eligible individuals, regardless of race or ethnicity.

“Many of the 53,000 Americans killed by colorectal cancer each year come from communities of color, a disparity we must work together to eliminate,” said Gary A. Puckrein, Ph.D., president and chief executive officer of National Minority Quality Forum. “Access to innovations in colorectal cancer screening like the Cologuard Plus test and navigation that helps people get screened will play an important role in helping reduce the disproportionate and deadly impact colorectal cancer has on communities of color.”

Conference call and webcast details

Exact Sciences management will host a conference call and webcast on March 14 at 8:00 a.m. ET to discuss results of the pivotal BLUE-C study. The webcast will be available at www.exactsciences.com. Domestic callers should dial (800) 715-9871 and international callers should dial +1 646-307-1963. The access code for both domestic and international callers is 5347907. An archive of the webcast will be available at www.exactsciences.com. The webcast, conference call, and replay are open to all interested parties.

About the BLUE-C Study

BLUE-C was a multi-center, prospective study (NCT04144738) of more than 20,000 adults 40 years of age and older.¹ The trial was designed to evaluate the performance of Cologuard Plus (next generation multitarget stool DNA or mt-sDNA). Using colonoscopy as a reference method, the robust study design directly compared Cologuard Plus and an independent FIT. Blood samples were also collected for evaluation of a blood-based screening test developed by Exact Sciences. BLUE-C is one of the largest, noninvasive CRC screening trials ever conducted, and the study population reflects the racial and ethnic makeup of the United States according to the 2020 census.^1,2

About Cologuard Plus

Developed in collaboration with Mayo Clinic, Cologuard Plus features novel biomarkers and improved laboratory processes. It also incorporates enhanced sample stability components to provide patients more time to return their sample to Exact Sciences’ lab and increase the valid result rate. Exact Sciences is preparing for FDA approval and the commercialization of Cologuard Plus.

About Exact Sciences Corp.

A leading provider of cancer screening and diagnostic tests, Exact Sciences gives patients and health care professionals the clarity needed to take life-changing action earlier. Building on the success of the Cologuard^® and Oncotype^® tests, Exact Sciences is investing in its pipeline to develop innovative solutions for use before, during, and after a cancer diagnosis. For more information, visit ExactSciences.com, follow Exact Sciences on X (formerly known as Twitter) @ExactSciences, or find Exact Sciences on LinkedIn and Facebook.

NOTE: Exact Sciences and Cologuard Plus are trademarks or registered trademarks of Exact Sciences Corporation. Oncotype is a registered trademark of Genomic Health, Inc., a wholly-owned subsidiary of Exact Sciences Corporation. All other trademarks and service marks are the property of their respective owners.

Forward-Looking Statements

This news release contains forward-looking statements concerning our expectations, anticipations, intentions, beliefs, or strategies regarding the future. These forward-looking statements are based on assumptions that we have made as of the date hereof and are subject to known and unknown risks and uncertainties that could cause actual results, conditions and events to differ materially from those anticipated. Therefore, you should not place undue reliance on forward-looking statements. Examples of forward-looking statements include, among others, statements we make regarding the development and commercialization of the Cologuard Plus test; the performance characteristics and healthcare benefits of Cologuard Plus in a commercial setting; and the timing and anticipated results of FDA submission. Risks and uncertainties that may affect our forward-looking statements are described in the Risk Factors sections of our most recent Annual Report on Form 10-K and any subsequent Quarterly Reports on Form 10-Q, and in our other reports filed with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

*The commercially-available Polymedco OC-Auto^® Micro 80 iFOB Test

References

Imperiale T, et al. N Engl J Med (2024)
United States Census Bureau. Annual Estimates of the Resident Population by Single Year of Age and Sex for the United States: April 1, 2020 to July 1, 2022 (NC-EST2022-AGESEX-RES). Accessed 16 October 2023. https://www.census.gov/data/tables/time-series/demo/popest/2020s-national-detail.html

Media (U.S.):

Lindsey Dickinson

+1 608-690-0383

lidickinson@exactsciences.com

Investor:

Nathan Harrill

+1 608 535-8659

investorrelations@exactsciences.com

Source: Exact Sciences Corp.

FDA Approves Arcutis’ ZORYVE® (roflumilast) Topical Foam, 0.3% for the Treatment of Seborrheic Dermatitis in Individuals Aged 9 Years and Older

Dimitri Z — Mon, 18 Dec 2023 00:22:20 +0000

FDA Approves Arcutis’ ZORYVE® (roflumilast) Topical Foam, 0.3% for the Treatment of Seborrheic Dermatitis in Individuals Aged 9 Years and Older

ZORYVE foam represents a highly effective, safe, well-tolerated, once-daily steroid-free foam for use on all affected areas of the body, including hair-bearing areas, with no limitations on duration of use
ZORYVE foam provides rapid disease clearance and significant reduction in itch, one of the most burdensome symptoms of seborrheic dermatitis
First drug approved for seborrheic dermatitis with a new mechanism of action in over two decades
Seborrheic dermatitis affects more than 10 million people in the United States
Commercial product expected to be available by end of January
Management will host conference call on Monday, December 18 at 8:30 a.m. EST

WESTLAKE VILLAGE, Calif., Dec. 15, 2023 (GLOBE NEWSWIRE) — Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT), an early commercial-stage company focused on developing meaningful innovations in immuno-dermatology, today announced the U.S. Food and Drug Administration (FDA) has approved the new drug application (NDA) for ZORYVE (roflumilast) topical foam, 0.3% for the treatment of seborrheic dermatitis in individuals 9 years of age and older. ZORYVE foam provides rapid disease clearance and significant reduction in itch, with nearly 80% of individuals achieving the primary efficacy endpoint of IGA Success and just over 50% of individuals reaching complete clearance at Week 8 in the STRATUM trial. ZORYVE is a once-daily steroid-free foam and the first drug approved for seborrheic dermatitis with a new mechanism of action in over two decades.

“We know from dermatology clinicians and those living with seborrheic dermatitis that there has been a real struggle with disease clearance and treatment adherence due to lack of efficacy, difficulty treating certain body areas, inconvenient treatment regimens, and concerns about safety with long-term use,” said Patrick Burnett, MD, PhD, FAAD, chief medical officer at Arcutis. “ZORYVE foam is a once-daily, steroid-free topical treatment that can be used anywhere on the body, including hair-bearing areas, with no limitation on duration of use. We are proud to deliver meaningful innovation through this approval of ZORYVE foam, and to offer a new topical treatment that effectively clears and controls the disease and can simplify its management for the millions of adults and adolescents living with seborrheic dermatitis.”

Seborrheic dermatitis affects more than 10 million people in the United States, and is a common, chronic, and recurrent inflammatory skin disease that causes red patches covered with large, greasy, flaking yellow-gray scales, and persistent itch. In individuals with darker skin tones, inflamed areas may not appear red, but instead can appear pink, slightly purple, or lighter in color than the surrounding skin. It occurs most often in areas of the body with oil-producing (sebaceous) glands, including the scalp, face (especially on the nose, eyebrows, ears, and eyelids), upper chest, and back. Hair-bearing areas make applying topicals like creams, gels, and ointments difficult.

“In the STRATUM trial, ZORYVE foam provided rapid disease clearance as early as Week 2 and significant itch relief in as little as 48 hours. In addition, almost 80% of patients achieved treatment success at Week 8. While multiple factors contribute to seborrheic dermatitis, inflammation and skin barrier dysfunction play key roles. ZORYVE has been shown to effectively reduce the signs of inflammation, redness, and scaling in patients with seborrheic dermatitis, and with its unique formulation, ZORYVE foam effectively delivers the drug without disrupting the skin barrier and has been shown to be safe and tolerable. ZORYVE foam is thus ideally formulated, having the potential to become the new standard of care for seborrheic dermatitis treatment,” said Andrew Blauvelt, MD, MBA, clinical investigator at Oregon Medical Research Center, and investigator on the STRATUM trial.

Beyond the appearance and irritation of physical symptoms, seborrheic dermatitis is associated with a decrease in quality of life and may negatively affect emotional well-being, self-esteem, and day-to-day life, including sleep and work. People with seborrheic dermatitis, and especially adolescents and school-age children, may suffer from social stigma, negative self-image, and low self-esteem associated with very visible skin diseases like seborrheic dermatitis.

“Approximately 10 million people in the United States have seborrheic dermatitis, but until today, there have been limited treatment options. We are thrilled with this FDA approval and are excited to bring to market a new, highly effective steroid-free topical formulation that can be used anywhere on the body,” said Frank Watanabe, president and CEO of Arcutis. “Our commercial team is ready and poised to launch ZORYVE foam very soon, and we are committed to ensuring affordable access to ZORYVE foam to those who may benefit from this novel treatment.”

Arcutis intends to make ZORYVE foam widely available via key wholesaler and dermatology pharmacy channels as a new treatment option by the end of January 2024. The Company is dedicated to responsible pricing and affordable access to therapy. The ZORYVE® Direct Program helps patients access their prescribed Arcutis medication. For patients with seborrheic dermatitis who have been prescribed ZORYVE, this patient support program helps patients navigate the payer process, assists patients with adherence, and includes the ZORYVE Direct Savings Card Program, which can help reduce out-of-pocket costs for eligible commercially insured patients.† Arcutis will also continue to offer the Arcutis CaresTM patient assistance program (PAP) that provides ZORYVE at no cost for financially eligible patients who are uninsured or underinsured.‡

Management will host a conference call on Monday, December 18 at 8:30 a.m. EST. A live webcast of the call and presentation material will be available on the “Events” section of the Company’s Investor website. An archived version of the webcast will be available on the Arcutis website after the call.

ZORYVE Foam Clinical Data

The approval is supported by positive results from Arcutis’ Phase 2 and pivotal Phase 3 trials in seborrheic dermatitis. The STudy of Roflumilast foam Applied Topically for the redUction of seborrheic derMatitis (STRATUM) and the Phase 2 (Trial 203) were parallel group, double-blind, vehicle-controlled studies evaluating the safety and efficacy of ZORYVE foam 0.3% in seborrheic dermatitis. Together the two studies enrolled 683 adults and adolescents ages 9 years and older.

The STRATUM study met its primary endpoint, with nearly 80% of ZORYVE foam treated individuals reaching Investigator Global Assessment (IGA) Success rate at Week 8 (79.5% ZORYVE foam vs 58.0% vehicle; P<0.0001). In Trial 203, 73% of individuals treated with ZORYVE foam achieved IGA Success (73.1% ZORYVE foam vs 40.8% vehicle; P<0.0001.) IGA Success was defined as an IGA score of “Clear” (0) or “Almost Clear” (1), plus a 2-grade IGA score improvement from baseline at Week 8.

Improvement with ZORYVE foam was seen early, with roflumilast demonstrating a statistically significant improvement compared to vehicle on IGA Success at Week 2, the first timepoint assessed in STRATUM. In addition, 50.6% of individuals in the ZORYVE foam treated arm reached complete clearance (IGA=0) at Week 8.

The STRATUM study also demonstrated statistically significant improvement over vehicle on all secondary endpoints, including itch, scaling, and erythema (redness). More than 60% of individuals achieved a ≥4-point reduction in itch at Week 8 as measured by Worst Itch-Numerical Rating Score (62.8% roflumilast foam vs 40.6% vehicle; P=0.0001), and significant improvements in itch were also reported at Week 2 and Week 4. Individuals treated with ZORYVE foam reported a 28% improvement in itch from baseline in 48 hours (compared to 13% on vehicle nominal P=0.0024).

In addition, more than 50% of individuals treated with ZORYVE foam achieved an erythema (redness) score of 0, and more than 50% achieved a scaling score of 0, at Week 8. Treatment with ZORYVE foam demonstrated a significantly larger improvement in patient reported outcomes as early as Week 2 as measured through Dermatology Life Quality Index (DLQI), with improvements maintained through Week 8.

ZORYVE foam was well-tolerated with a favorable safety and tolerability profile during up to 52 weeks of treatment. Incidence of Treatment Emergent Adverse Events (TEAEs) was low and similar between active treatment and vehicle, with most TEAEs assessed as mild to moderate severity. There were no treatment-related Serious Adverse Events (SAEs). Overall, the most common adverse reactions occurring in ≥1% of subjects in the combined Phase 2 and Phase 3 study populations were nasopharyngitis (1.5%), nausea (1.3%), and headache (1.1%).

About ZORYVE®

ZORYVE (roflumilast) topical foam, 0.3%, is indicated for treatment of seborrheic dermatitis in adult and pediatric patients 9 years of age and older. Another formulation of ZORYVE, roflumilast cream 0.3%, is approved by the FDA for the topical treatment of plaque psoriasis in individuals 6 years of age and older. Both ZORYVE foam and cream are topical formulations of roflumilast, a highly potent and selective phosphodiesterase-4 (PDE4) inhibitor. PDE4 is an intracellular enzyme that increases the production of pro-inflammatory mediators and decreases production of anti-inflammatory mediators. It is an established target in dermatology.

INDICATIONS

ZORYVE cream is indicated for topical treatment of plaque psoriasis, including intertriginous areas, in patients 6 years of age and older.

ZORYVE foam, 0.3%, is indicated for treatment of seborrheic dermatitis in adult and pediatric patients 9 years of age and older.

IMPORTANT SAFETY INFORMATION

ZORYVE is contraindicated in patients with moderate to severe liver impairment (Child-Pugh B or C).

Flammability: The propellants in ZORYVE foam are flammable. Avoid fire, flame, and smoking during and immediately following application.

The most common adverse reactions (≥1%) for ZORYVE cream include diarrhea (3.1%), headache (2.4%), insomnia (1.4%), nausea (1.2%), application site pain (1.0%), upper respiratory tract infection (1.0%), and urinary tract infection (1.0%).

The most common adverse reactions (≥1%) for ZORYVE foam include nasopharyngitis (1.5%), nausea (1.3%), and headache (1.1%).

Please see full Prescribing Information for ZORYVE foam and full Prescribing Information for ZORYVE cream.

ZORYVE is for topical use only and not for ophthalmic, oral, or intervaginal use.

About Arcutis

Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT) is an early commercial-stage medical dermatology company that champions meaningful innovation to address the urgent needs of individuals living with immune-mediated dermatological diseases and conditions. With a commitment to solving the most persistent patient challenges in dermatology, Arcutis has a growing portfolio that harnesses our unique dermatology development platform coupled with our dermatology expertise to build differentiated therapies against biologically validated targets. Arcutis’ dermatology development platform includes a robust pipeline with multiple clinical programs for a range of inflammatory dermatological conditions including scalp and body psoriasis, atopic dermatitis, seborrheic dermatitis, and alopecia areata. For more information, visit www.arcutis.com or follow Arcutis on LinkedIn, Facebook, and X.

Forward-Looking Statements

Arcutis cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the Company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding the potential of ZORYVE foam to simplify disease management for care of seborrheic dermatitis, potential of ZORYVE foam to become the standard of care in seborrheic dermatitis treatment, and the Company’s expected timing and plan to commercially launch ZORYVE foam by end of January. These statements are subject to substantial known and unknown risks, uncertainties, and other factors that may cause our actual results, levels of activity, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. Risks and uncertainties that may cause our actual results to differ include risks inherent in our business, reimbursement and access to our products, the impact of competition and other important factors discussed in the “Risk Factors” section of our Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on February 28, 2023, as well as any subsequent filings with the SEC. You should not place undue reliance on any forward-looking statements in this press release. We undertake no obligation to revise or update information herein to reflect events or circumstances in the future, even if new information becomes available. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

† Subject to eligibility criteria and maximum program limitation. This offer is not valid for patients without commercial drug insurance or whose prescription claims are eligible to be reimbursed, in whole or in part, by any government program.

‡ Subject to financial eligibility requirements. Other terms and restrictions apply.

Contacts:

Media

Amanda Sheldon, Head of Corporate Communications

asheldon@arcutis.com

Investors

Derek Cole

Investor Relations Advisory Solutions

derek.cole@iradvisory.com

Arcutis Announces Positive Long-Term Results of Roflumilast Cream 0.15% Showing Durable and Improved Efficacy Over Time and Favorable Safety Profile in Treatment of Mild to Moderate Atopic Dermatitis (AD)

Dimitri Z — Fri, 08 Sep 2023 00:22:20 +0000

Arcutis Announces Positive Long-Term Results of Roflumilast Cream 0.15% Showing Durable and Improved Efficacy Over Time and Favorable Safety Profile in Treatment of Mild to Moderate Atopic Dermatitis (AD)

September 7, 2023 at 8:15 AM EDT

PDF Version

Results for adults and children ages 6 years and older from INTEGUMENT-OLE long-term extension trial highlight that 46.1% and 51.0% of patients who rolled over from the roflumilast cream treatment arm in INTEGUMENT-1 or -2 achieved IGA success at Week 28 and Week 56, respectively
Roflumilast cream maintained disease control even when participants switched to twice weekly maintenance dosing schedule
Long-term safety and tolerability profile consistent with short-term data in AD, with no new safety signals observed during 56 weeks of treatment

WESTLAKE VILLAGE, Calif., Sept. 07, 2023 (GLOBE NEWSWIRE) — Arcutis Biotherapeutics, Inc. (NASDAQ: ARQT), an early commercial-stage biopharmaceutical company focused on developing meaningful innovations in immuno-dermatology, today announced interim results from the INTEGUMENT-OLE long-term open-label study of roflumilast cream 0.15% in adults and children ages 6 years and older with atopic dermatitis. In the study, roflumilast cream was well-tolerated, with no new safety signals observed during treatment up to 56 weeks in duration. Efficacy was not only maintained but improved over time, with 46.1% and 51.0% of participants who rolled over from the roflumilast cream treatment arm in INTEGUMENT-1 or -2 achieving validated Investigator Global Assessment-Atopic Dermatitis (vIGA-AD) success, defined as vIGA-AD value of 0 or 1 plus a 2-grade improvement from baseline, at Weeks 28 and 56, respectively.

Starting at Week 4 of INTEGUMENT-OLE, participants who achieved a vIGA-AD score of clear (0) switched to twice weekly maintenance dosing. Participants were to resume once-daily dosing if vIGA-AD reached mild (2) or if signs or symptoms were not adequately controlled. Over two-thirds of participants who switched to maintenance dosing remained on the twice weekly schedule for more than half of their time in the study (post-Week 4).

“When determining a treatment plan for an adult or child with a chronic, burdensome skin condition such as atopic dermatitis, long-term efficacy and safety are both incredibly important considerations,” said Eric Simpson, MD, MCR, FAAD, Professor of Dermatology at Oregon Health & Science University in Portland, Oregon, and INTEGUMENT trial investigator. “These results build upon the positive findings from the pivotal Phase 3 trials of roflumilast cream 0.15% in atopic dermatitis demonstrating rapid efficacy within the first 4 weeks of treatment, and further validate the long-term durable efficacy and tolerability of roflumilast cream, with continued improvement over the course of the long-term study. Importantly, individuals who reached clear were able to switch to a twice weekly dosing and maintain control of their disease through this schedule.”

“The goal and greatest clinical challenge for treating adults and children with atopic dermatitis is dependable disease control. Due to the instability of this chronic disease, long-term control and safety is key; however, data on prevention and maintenance with topical therapy is lacking. This long-term study was designed to study proactive treatment, for patients with clear skin, to optimize and maintain control with twice weekly dosing,” said Emma Guttman-Yassky, MD, PhD, System Chair of the Department of Dermatology and Waldman Professor of Dermatology and Immunology at the Icahn School of Medicine at Mount Sinai. “These study results provide evidence for a paradigm shift to break the cycle of the current topical paradigm that only reactively chase and manage flares, showing the utility of infrequent preventive treatment regimens to disease control.” Dr. Guttman is a paid consultant with Arcutis Biotherapeutics, Inc.

Additionally, in the study, 61.5% and 66.2% of participants who rolled over from the roflumilast cream arm in INTEGUMENT-1 or -2 demonstrated a 75% improvement from baseline in Eczema Area and Severity Index (EASI-75) after 28 weeks and 56 weeks, respectively.

The long-term study results reinforce the safety profile of roflumilast cream already seen in the short-term INTEGUMENT-1 and INTEGUMENT-2 clinical trials, with no new safety signals observed up to 56 weeks. Overall incidence of adverse events was low, with most being mild to moderate in nature. The most frequently reported adverse events (≥2%) included: COVID-19, upper respiratory tract infection, nasopharyngitis, and headache. Overall, only 3.0% of trial participants discontinued the study due to adverse events.

“Roflumilast cream is uniquely formulated to deliver treatment without sensitizing excipients and irritants, which can often disrupt the skin barrier. We are excited by these results, which demonstrate the long-term efficacy and the safety and tolerability profile of our next generation phosphodiesterase type 4 (PDE4) inhibitor for the treatment of atopic dermatitis,” said Patrick Burnett, MD, PhD, FAAD, chief medical officer at Arcutis. “Based on the positive results we have seen, we are convinced that, if approved, roflumilast cream will provide individuals with atopic dermatitis with an important new long-term treatment option that is designed with their specific needs in mind.”

Arcutis intends to submit a supplemental New Drug Application (sNDA) late in the third quarter of 2023 for roflumilast cream 0.15% for the treatment of mild to moderate atopic dermatitis in individuals ages 6 years and older.

About INTEGUMENT-OLE

The “INterventional Trial EvaluatinG roflUMilast cream for the treatmENt of aTopic dermatitis Open Label Extension” (INTEGUMENT-OLE) was a Phase 3, multicenter, open-label extension study of the long-term safety of roflumilast cream 0.15% in adults and children ages 6 years and older with atopic dermatitis and roflumilast cream 0.05% in children ages 2 to 5 years. Individuals completing the INTEGUMENT-1 or INTEGUMENT-2 Phase 3 trials were eligible to enroll (n=658) for either 24 or 52 weeks.

The study evaluated monotherapy with roflumilast cream with no rescue treatment permitted. Beginning at Week 4 of INTEGUMENT-OLE, any participant who achieved vIGA-AD of ‘0-Clear’ switched to twice weekly maintenance treatment. Participants were able to continue twice weekly maintenance dosing, as long as vIGA-AD remained either ‘0?Clear’ or ‘1-Almost Clear’. Participants resumed once-daily dosing if vIGA-AD reached ≥2-Mild, and could also resume once-daily dosing if signs/symptoms of AD were not adequately controlled with maintenance therapy despite remaining at vIGA-AD of ‘1-Almost Clear’.

The primary objective of the study was to assess the long-term safety of roflumilast cream after either 24 or 52 weeks of treatment. Secondary endpoints include vIGA-AD score of 0 or 1 at each assessment, vIGA-AD success defined as vIGA-AD value of 0 or 1 plus a 2-grade improvement from baseline, Worst Itch Numeric Scale (WI-NRS) score over time, and Eczema Area and Severity Index (EASI) score over time. The assessment of IGA Success and EASI-75 response, as reported here, references baseline of INTEGUMENT-1 and -2.

About Atopic Dermatitis

AD is the most common type of eczema, affecting approximately 9.6 million children and 16.5 million adults in the United States. AD is characterized by a defect in the skin barrier, which allows allergens and other irritants to enter the skin, leading to an immune reaction and inflammation. This reaction produces a red, itchy rash, most frequently occurring on the face, arms, and legs. The rash can cover significant areas of the body, in some cases half of the body or more. AD typically begins in early childhood and is chronic. It persists into adolescence and even adulthood in some individuals. The rash causes significant pruritus (itching), which can lead to skin damage caused by scratching or rubbing. Since a large percentage of AD patients are very young children, safety is a particularly important consideration in treatment selection.

About Roflumilast Cream

Roflumilast cream is a next generation topical PDE4 inhibitor. PDE4 – an established target in dermatology – is an intracellular enzyme that increases the production of pro-inflammatory mediators and decreases production of anti-inflammatory mediators. Roflumilast cream 0.3% (ZORYVE®) is approved by the U.S. Food and Drug Administration (FDA) for the topical treatment of plaque psoriasis, including intertriginous areas, in patients 12 years of age and older. Roflumilast cream for AD was evaluated at lower doses: 0.15% for adults and children 6 years of age and older and is being evaluated at 0.05% for children aged 2 to 5 years.

About ZORYVE®

ZORYVE (roflumilast) cream 0.3% is indicated for topical treatment of plaque psoriasis, including intertriginous areas, in patients 12 years of age and older.

IMPORTANT SAFETY INFORMATION

The use of ZORYVE is contraindicated in patients with moderate to severe liver impairment (Child-Pugh B or C).

The most common adverse reactions (≥1%) include diarrhea (3%), headache (2%), insomnia (1%), nausea (1%), application site pain (1%), upper respiratory tract infection (1%), and urinary tract infection (1%).

Please see full Prescribing Information.

Forward-Looking Statements

Arcutis cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the Company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding the potential and timing for roflumilast cream to be approved by the FDA for the treatment of adults and children with atopic dermatitis, the potential to use roflumilast cream over a long period of time, or chronically, the potential to use roflumilast cream anywhere on the body, and the potential for roflumilast cream to advance the standard of care in atopic dermatitis and other inflammatory dermatological conditions. These statements are subject to substantial known and unknown risks, uncertainties, and other factors that may cause our actual results, levels of activity, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. Risks and uncertainties that may cause our actual results to differ include risks inherent in our business, reimbursement and access to our products, the impact of competition and other important factors discussed in the “Risk Factors” section of our Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on February 28, 2023, as well as any subsequent filings with the SEC. You should not place undue reliance on any forward-looking statements in this press release. We undertake no obligation to revise or update information herein to reflect events or circumstances in the future, even if new information becomes available. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

Contacts:

Media

Amanda Sheldon, Head of Corporate Communications

asheldon@arcutis.com

Investors

Eric McIntyre, Head of Investor Relations

emcintyre@arcutis.com

Seres Therapeutics and Nestlé Health Science Announce FDA Approval of VOWSTTM (fecal microbiota spores, live-brpk) for Prevention of Recurrence of C. difficile Infection in Adults Following Antibacterial Treatment for Recurrent CDI

Dimitri Z — Thu, 27 Apr 2023 00:22:20 +0000

Seres Therapeutics and Nestlé Health Science Announce FDA Approval of VOWST^TM (fecal microbiota spores, live-brpk) for Prevention of Recurrence of C. difficile Infection in Adults Following Antibacterial Treatment for Recurrent CDI

– First and only FDA-approved orally administered microbiota-based therapeutic, validating Seres’ microbiome platform –

– Phase 3 ECOSPOR III study demonstrated that 88% of treated individuals were recurrence-free at 8 weeks –

– Opportunity to address prevention of recurrence of C. difficile infection in adults with rCDI, including first recurrence, following antibacterial treatment –

– VOWST product availability expected in June –

April 26, 2023 07:46 PM Eastern Daylight Time

CAMBRIDGE, Mass. & HOBOKEN, N.J.–(BUSINESS WIRE)–Seres Therapeutics, Inc. (Nasdaq: MCRB) and Nestlé Health Science today announced the U.S. Food and Drug Administration (FDA) approval of VOWST^TM (fecal microbiota spores, live-brpk), formerly called SER-109, an orally administered microbiota-based therapeutic to prevent recurrence of C. difficile Infection (CDI) in adults following antibacterial treatment for recurrent CDI (rCDI). VOWST is not indicated for the treatment of CDI.

“Recurrent C. difficile infection is a highly debilitating and life-threatening disease, and antibiotics alone do not address the underlying cause of rCDI, dysbiosis of the gut microbiome”

“Since being founded by Flagship Pioneering over a decade ago, Seres has led the development of microbiome therapeutics, and today’s FDA approval of VOWST as the first orally administered microbiota-based therapeutic for the prevention of recurrent C. difficile infection marks a tremendous milestone for the patient community, and for Seres. We are deeply grateful to the patients, caregivers, clinical investigators, and employees who contributed to the discovery, development, and approval of VOWST,” said Eric Shaff, President and Chief Executive Officer at Seres. “With VOWST, we and Nestlé Health Science have the opportunity to prevent recurrence in a broad group of adult rCDI patients, including those who have experienced a first recurrence.”

“Our strategic collaboration with Seres is part of Nestlé Health Science’s ongoing commitment to advancements in the gastrointestinal space to address unmet patient needs,” said Greg Behar, Chief Executive Officer, Nestlé Health Science. “Our teams have vast experience in gastrointestinal disorders and are poised to engage with healthcare professionals to start addressing this critical need for patients. We expect VOWST to be available in June and look forward to helping patients.”

Recurrent CDI represents significant unmet need and is a leading cause of hospital-acquired infection that can result in severe illness and death.¹ Based on data from the U.S. Centers for Disease Control and Prevention (CDC), the companies estimate 156,000 episodes in the U.S. in 2023.

“Recurrent C. difficile infection is a highly debilitating and life-threatening disease, and antibiotics alone do not address the underlying cause of rCDI, dysbiosis of the gut microbiome,” said Carl Crawford M.D., Assistant Professor of Clinical Medicine at Weill Cornell Medical College. “The approval of VOWST provides an important new oral treatment option for this disease, and I am pleased to now be able to offer this medicine to recurrent CDI patients.”

“Recurrent C. difficile infection significantly impacts patients’ quality of life, both physically and emotionally, leaving many living in tremendous fear of future recurrences. Patients have been waiting for new treatment options that address a key concern: prevention of an additional CDI recurrence,” said Christian John Lillis, Executive Director at Peggy Lillis Foundation for C. diff Education and Advocacy.

VOWST Phase 3 Study Data

The FDA approval of VOWST was supported by a robust Phase 3 development program that included the ECOSPOR III and ECOSPOR IV studies. VOWST was previously granted Breakthrough Therapy and Orphan Drug Designations by the FDA.

ECOSPOR III was a multicenter, randomized, placebo-controlled study in individuals with rCDI, the results of which were published in the New England Journal of Medicine.² The study’s primary objective was to demonstrate the reduction of CDI recurrence with VOWST. In ECOSPOR III, VOWST was shown to reduce CDI recurrence at eight weeks, with approximately 88% of individuals recurrence-free at eight weeks post-treatment, compared to 60% in participants who received placebo.² In addition, at six months post-treatment, 79% of the VOWST group were demonstrated to be recurrence-free, compared to 53% in the placebo group.³ No treatment-related serious adverse events were observed in the active arm and the frequency of treatment-related adverse events was similar between the VOWST and placebo arms. The most common adverse reactions through eight weeks in VOWST treated participants versus placebo were solicited events of abdominal distention (31.1% VOWST versus 29.3% placebo), fatigue (22.2% VOWST versus 21.7% placebo), constipation (14.4% VOWST versus 10.9% placebo), chills (11.1% versus 7.6% placebo), and unsolicited event of diarrhea (10.0% versus 4.3% placebo).⁴

ECOSPOR IV was an open-label, single arm study evaluating VOWST in 263 adult participants with rCDI. Study results were published in the JAMA Network Open.⁵ The ECOSPOR IV study results contributed to the VOWST safety database and supported product approval.

Seres and Nestlé Health Science are committed to helping appropriate patients who have been prescribed VOWST obtain access. Additional details about VOWST access programs will be available at launch.

Joint Commercialization Agreement

In July 2021, Seres and Nestlé Health Science entered into an agreement to jointly commercialize VOWST in the U.S. and Canada. Nestlé Health Science is leveraging its global pharmaceutical business and assuming the role of lead commercialization party, including the utilization of its existing infrastructure, gastrointestinal sales force and payer access team.

Seres is due to receive a $125 million milestone payment from Nestlé Health Science associated with the FDA approval of VOWST. Upon VOWST commercialization, each company will be entitled to share equally in commercial profits and losses.

Conference Call Information

Seres’ management will host a conference call tomorrow, April 27, 2023, at 8:30 a.m. ET. Accompanying slides will be posted on the Seres website prior to the call. To access the conference call, please dial 773-305-6867 (domestic) or 866-400-0049 (international) and reference Conference ID 1937506. To join the live webcast, please visit the “Investors and News” section of the Seres website at www.serestherapeutics.com.

A webcast replay will be available on the Seres website beginning approximately two hours after the event and will be archived for at least 21 days.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR VOWST

INDICATION

VOWST is indicated to prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI).

Limitation of Use: VOWST is not indicated for treatment of CDI.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Transmissible infectious agents: Because VOWST is manufactured from human fecal matter, it may carry a risk of transmitting infectious agents. Report any infection that is suspected to have been transmitted by VOWST to Aimmune Therapeutics, Inc. at 1-833-246-2566.

Potential presence of food allergens: VOWST may contain food allergens. The potential to cause adverse reactions due to food allergens is unknown.

ADVERSE REACTIONS

The most common adverse reactions (reported in ≥5% of participants) were abdominal distension (31.1%), fatigue (22.2%), constipation (14.4%), chills (11.1%), and diarrhea (10.0%).

To report SUSPECTED ADVERSE REACTIONS, contact Aimmune Therapeutics at 1-833-AIM-2KNO (1-833-246-2566), or the FDA at 1-800-FDA-1088, or visit www.fda.gov/MedWatch.

DRUG INTERACTIONS

Do not administer antibacterials concurrently with VOWST.

Please see Full Prescribing Information and Patient Information

About Recurrent C. difficile Infection (rCDI)

Recurrent C. difficile infection is a gastrointestinal infection caused by C. difficile bacteria. rCDI is linked to dysbiosis of the gastrointestinal microbiome and is associated with increased mortality. CDI has been characterized as an Urgent Health Threat by the Centers for Disease Control and Prevention (CDC). rCDI results in a substantial burden on the healthcare system¹ with the average rCDI-related annual costs per patient at approximately $43K.⁶

About Seres Therapeutics

Seres Therapeutics, Inc. (Nasdaq: MCRB) is a commercial-stage company developing novel microbiome therapeutics for serious diseases. Seres’ lead program, VOWST^TM, obtained U.S. FDA approval in April 2023 as the first orally administered microbiota-based therapeutic to prevent recurrence of C. difficile infection (CDI) in adults following antibacterial treatment for recurrent CDI and is being commercialized in collaboration with Nestlé Health Science. Seres is also developing a novel class of multifunctional fermented bacterial consortia designed to functionally interact with host cells and tissues to treat disease. For more information, please visit www.serestherapeutics.com.

About Nestlé Health Science

Nestlé Health Science, a leader in the science of nutrition, is a globally managed business unit of Nestlé. We are committed to redefining the management of health, offering an extensive portfolio of science-based consumer health, medical nutrition, pharmaceutical therapies, and vitamin and supplement brands. Our extensive research network provides the foundation for products that empower healthier lives through nutrition. Headquartered in Switzerland, we have more than 12,000 employees around the world, with products available in more than 140 countries. For more information, please visit www.nestlehealthscience.us.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including the timing of commercial launch, the availability of VOWST, the commercial success of VOWST; and other statements which are not historical fact.

These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have incurred significant losses, are not currently profitable and may never become profitable; our need for additional funding; our limited operating history; the impact of the COVID-19 pandemic; our unproven approach to therapeutic intervention; our reliance on third parties and collaborators to conduct our clinical trials, manufacture our product candidates and develop and commercialize our product candidates, if approved; the unknown degree and competing factors of market acceptance for VOWST; the competition we will face; our ability to protect our intellectual property; and our ability to retain key personnel and to manage our growth. These and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), on March 7, 2023, and our other reports filed with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

References:

1. Rodrigues R, Barber GE, Ananthakrishnan AN. A Comprehensive Study of Costs Associated With Recurrent Clostridium difficile Infection. Infect Control Hosp Epidemiol. 2016;38:196-202. DOI: 10.1017/ice.2016.246

2. Feuerstadt P, Louie TJ, Lashner B, et al. SER-109, an Oral Microbiome Therapy for Recurrent Clostridioides difficile Infection. N Engl J Med. 2022;386:220-9. DOI: 10.1056/nejmoa2106516

3. Cohen SH, Louie TJ, Sims M, et al. Extended Follow-up of Microbiome Therapeutic SER-109 Through 24 Weeks for Recurrent Clostridioides difficile Infection in a Randomized Clinical Trial. JAMA. 2022;328:2062. DOI: 10.1001/jama.2022.16476

4. VOWSTTM. Prescribing Information. Seres Therapeutics, Inc.; 2023.

5. Sims MD, Khanna S, Feuerstadt P, et al. Safety and Tolerability of SER-109 as an Investigational Microbiome Therapeutic in Adults With Recurrent Clostridioides difficile Infection: A Phase 3, Open-Label, Single-Arm Trial. JAMA Network Open. 2023;6(2):e2255758. DOI: 10.1001/jamanetworkopen.2022.55758

6. U.S. Bureau of Labor Statistics. CPI Inflation Calculator. U.S. Bureau of Labor Statistics. Published 2022. https://www.bls.gov/data/inflation_calculator.htm. CPI inflation adjusted to March 2023.

Contacts

Seres IR and PR Contact:
Carlo Tanzi, Ph.D.
ctanzi@serestherapeutics.com

Nestlé Health Science PR Contact:
Lindsay Yanek
lindsay.yanek@us.nestle.com

CinCor Pharma Announces Positive Topline Data for Phase 2 BrigHtn Trial Evaluating Baxdrostat, its Selective Aldosterone Synthase Inhibitor, in Treatment-Resistant Hypertension

Dimitri Z — Tue, 09 Aug 2022 00:22:20 +0000

CinCor Pharma Announces Positive Topline Data for Phase 2 BrigHtn Trial Evaluating Baxdrostat, its Selective Aldosterone Synthase Inhibitor, in Treatment-Resistant Hypertension

August 8, 2022 at 7:00 AM EDT

PDF Version

Successfully met the primary endpoint in the BrigHtn trial, delivering a 20.3 mmHg reduction in systolic blood pressure (SBP), or an 11 mmHg (p-value < 0.0001) decline on a placebo-adjusted basis, at 2 mg dose

Independent Data Review Committee determined that the trial met pre-specified statistical criteria of overwhelming efficacy at the highest dose allowing completion of the trial with 275 patients randomized

Dose-dependent reduction in SBP observed

Compelling safety and tolerability profile

Conference call and live webcast today at 8:30 AM Eastern Time

WALTHAM, Mass., Aug. 08, 2022 (GLOBE NEWSWIRE) — CinCor Pharma, Inc. (“CinCor”) announced today the topline results and successful completion of its Phase 2 BrigHtn trial evaluating the efficacy and safety of baxdrostat, a once daily potentially first-in-class, highly selective aldosterone synthase inhibitor in patients with treatment-resistant hypertension, defined as patients taking at least three blood pressure medications at their maximally tolerated doses, one of which must be a diuretic. Results showed baxdrostat met its primary endpoint and achieved statistically significant placebo-adjusted reduction in systolic blood pressure, including 11 mmHg (p-value < 0.0001) at a dose of 2 mg.

“We are thrilled to report that the BrigHtn trial has met its primary endpoint with baxdrostat demonstrating a double-digit placebo-adjusted reduction in blood pressure in patients with treatment-resistant hypertension,” said Marc de Garidel, Chief Executive Officer at CinCor. “We believe these data represent an important scientific breakthrough, supporting the potential of baxdrostat to effectively emerge as a new mechanism of action in the hypertension treatment paradigm, and to potentially be the first meaningful innovation in the treatment of hypertension in decades. Treatment resistant patients represent as many as 15 million adults in the United States alone, and now CinCor looks forward to opening a new era in hypertension innovation.”

“The results of BrigHtn are highly supportive for the continued development of baxdrostat as a novel antihypertensive agent,” added Mason Freeman, M.D, Chief Medical Officer at CinCor. “The goal of BrigHtn was to identify effective doses of the drug and to characterize baxdrostat tolerability in the treatment-resistant hypertension patient population. The fact that these patients with very difficult-to-control hypertension responded so well to baxdrostat at both the 1 mg and 2 mg dose levels, is extremely encouraging, as is the observed tolerability of baxdrostat when used concurrently in patients on multiple background anti-hypertensive agents.”

Key clinical data from BrigHtn suggests impressive efficacy and meaningful dose dependency in the treatment of patients with resistant hypertension:

BrigHtn successfully met its primary endpoint, demonstrating a statistically significant change from baseline in mean seated SBP versus placebo for the 2 mg and 1 mg doses:
- 20.3 mmHg SBP reduction at the 2 mg dose, or 11.0 mmHg placebo-adjusted decline (p < 0.0001)
- 17.5 mmHg SBP reduction at the 1 mg dose, or 8.1 mmHg placebo-adjusted decline (p = 0.0030)
- 12.1 mmHg SBP reduction at the 0.5 mg dose, or 2.7 mmHg placebo-adjusted decline (p = 0.3110)
Secondary endpoint results included baxdrostat significantly lowering diastolic blood pressure (DBP) by 5.2 mmHg in the 2mg dose, and approximately 46% of patients in the 2 mg dose arm achieving blood pressure control (SBP less than 130mmHg)
The BrigHtn trial completed enrollment with 275 patients after an Independent Data Review Committee determined that the trial had met pre-specified statistical criteria of overwhelming efficacy at the highest dose in this dose-ranging trial

Morris Brown, M.D., F. Med.Sci, Professor of Endocrine Hypertension at Barts and the London Medical School, UK, and former president of the British Hypertension Society, added, “The BrigHtn trial results represent the culmination of decades searching for a clinically safe drug which prevents synthesis of aldosterone, but not the similar adrenal gland hormone, cortisol. These impressive results suggest that baxdrostat lowered blood pressure in the BrigHtn trial by more than the reduction observed for spironolactone, as my colleagues and I reported in the PATHWAY-2 study in a similar patient group. For the first time in hypertension, the BrigHtn trial demonstrated that a dose-dependent reduction in blood pressure is accompanied by a dose-dependent reduction in a major cause of hypertension, confirming indeed the importance of aldosterone as a remediable factor in resistance to other commonly used drugs.”

Key clinical safety and tolerability findings of baxdrostat support a safe and well-tolerated profile

No drug related serious adverse events (SAEs) observed or major safety concerns were reported across all three dose cohorts tested after 12 weeks of treatment
Treatment-Emergent SAEs (TESAEs) were reported in 10 patients and deemed by investigators to be unrelated to baxdrostat. These TESAEs included hyponatremia, hyperkalemia, cellulitis, urinary tract infection, dehydration, hyperglycemia, arthralgia, dizziness, syncope, acute kidney injury, nephrolithiasis, acute respiratory failure, and respiratory failure, with one patient in the 2mg dose cohort experiencing six of these SAEs
One subject experienced an isolated instance of elevated potassium above 6mEq/L, which was deemed drug related, although upon retesting, the potassium level for this patient dropped sufficiently to allow resumption of baxdrostat, and the patient completed the trial with normal potassium levels. Overall, observed hyperkalemia rates in the trial were low, and resulted in no clinical safety concerns
Low discontinuation rate of less than 1% (2 patients) due to treatment-related adverse events, which included hyperkalemia and hypotension

Deepak L. Bhatt, M.D., M.P.H., Executive Director of Interventional Cardiovascular Programs at Brigham and Women’s Hospital and Professor of Medicine at Harvard Medical School, further added, “The low rates of hyperkalemia are encouraging since more than 90% of BrigHtn participants were on background antihypertensives that are challenging to combine with aldosterone blocking agents due to concerns over hyperkalemic off-target side effects. Overall, the double-digit placebo corrected reduction in systolic blood pressure, well-tolerated safety profile, and titratable dose response with once-daily baxdrostat has the potential to address multiple unmet needs in the treatment of treatment-resistant hypertension patients.”

The BrigHtn trial was a Phase 2 randomized, double-blind, placebo-controlled dose-ranging study designed to assess the safety and efficacy of baxdrostat in subjects who have not achieved their target blood pressure despite receiving three or more antihypertensive agents at their maximally tolerated doses, one of which must be a diuretic. The trial evaluated three active doses of baxdrostat (0.5 mg, 1.0 mg, and 2.0 mg) compared to placebo control in 275 patients randomized across all four dosing cohorts, with 248 patients completing. The primary endpoint of BrigHtn was the change in SBP from randomization to study end after 12 weeks of treatment.

As recently announced, in July 2022 CinCor completed enrollment in the Phase 2 HALO trial with 249 patients randomized. HALO is studying the efficacy and safety of baxdrostat in patients whose blood pressure is not controlled despite treatment with up to two antihypertensive agents and remains on track to be completed in the second half of 2022. We also announced the initiation of a Phase 2 Open Label Extension trial to evaluate the safety and tolerability of baxdrostat in patients for up to 52 weeks.

Conference Call and Webcast Information

CinCor management will hold a conference call and webcast today at 8:30 AM Eastern Time to provide an update on the BrigHtn Phase 2 trial. The dial-in number for the conference call is 877-407-9039 (U.S./Canada) or 201-689-8470 (international). The conference ID for all callers is 13731977. The live webcast and replay may be accessed by visiting the CinCor website at https://www.cincor.com/events-presentations. The replay will be available for 30 days following the call.

About CinCor

CinCor, founded in 2018, is a clinical-stage biopharmaceutical company with a mission to bring innovation to the pharmaceutical treatment of cardio-renal diseases. Its lead asset, baxdrostat (CIN-107), a highly selective, oral small molecule inhibitor of aldosterone synthase, is in clinical development for the treatment of hypertension and primary aldosteronism.

About Baxdrostat (CIN-107)

Baxdrostat is a highly selective, oral small molecule inhibitor of aldosterone synthase, the enzyme responsible for the synthesis of aldosterone in the adrenal gland, in development for patient populations with significant unmet medical needs, including treatment-resistant hypertension and primary aldosteronism. Hypertension, which is defined by the American College of Cardiology and the American Heart Association as resting blood pressure above 130/80 mm Hg, is generally acknowledged to be one of the most common preventable risk factors for premature death worldwide. Though often asymptomatic, hypertension significantly increases the risk of heart disease, stroke, and kidney disease, amongst other diseases. It is estimated that as much as 20% of the global population suffers from hypertension, including nearly one-half of the adult population in the U.S., or 116 million hypertensive patients.

Forward-Looking Statements

This press release contains certain forward-looking statements, including, but not limited to, statements related to CinCor’s business in general; the results and timing of CinCor’s ongoing and planned clinical trials, including its HALO trial; the anticipated timing of disclosure of results of clinical trials, including for HALO; the progress of CinCor’s research and development programs and clinical trials and studies, including enrollment and retention in clinical trials; plans for initiating future clinical trials and studies; the therapeutic potential of baxdrostat (CIN-107), including its potential to be an effective treatment for patients with treatment-resistant hypertension, uncontrolled hypertension and CKD, and the ability of baxdrostat to address multiple unmet needs in patients; the potential of baxdrostat to emerge as a new mechanism of action in the hypertension treatment paradigm and to potentially be the first meaningful innovation in the treatment of blood pressure in decades; CinCor’s clinical milestones and pipeline; expectations with respect to regulatory matters; expectations with respect to potential market size; and other statements that are not historical facts. Because such statements are subject to risks and uncertainties, actual results may differ from those expressed or implied by such forward-looking statements. Words such as “anticipates,” “believes,” “expected,” “intends,” “plan,” “may”, “will,” “project”, “estimate”, “continue,” “advance” and “future” or similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on CinCor’s current plans, objectives, estimates, expectations and intentions, involve assumptions that may never materialize or may prove to be incorrect and inherently involve significant risks and uncertainties, including factors beyond CinCor’s control, that could cause actual results, performance, or achievement to differ materially and adversely from those anticipated or implied in the statements, including, without limitation, CinCor has incurred significant operating losses since its inception; CinCor has a limited operating history and no history of commercializing products; CinCor will require substantial additional funding to finance its operations; CinCor’s business is entirely dependent at this time on the success of one drug, baxdrostat; initial, interim, “top-line” and preliminary data from clinical trials announced or published from time to time may change; CinCor may not be successful in its efforts to expand its pipeline beyond baxdrostat; success in preclinical studies or earlier clinical trials may not be indicative of results in future clinical trials; enrollment and retention of patients in clinical trials could be delayed; CinCor relies and will rely on third parties to conduct, supervise and monitor existing clinical trials and potential future clinical trials; developments from the company’s competitors and the marketplace for the company’s products; and CinCor’s business, operations and clinical development timelines and plans may be adversely affected by the evolving and ongoing COVID-19 pandemic, geopolitical events, including the ongoing military conflict between Russia and Ukraine and related sanctions against Russia, and macroeconomic conditions, including rising inflation and uncertain credit and financial markets, and matters related thereto; and other risks and uncertainties affecting the company, including those described under the caption “Risk Factors” and elsewhere in CinCor’s Annual Report on Form 10-K for the year ended December 31, 2021 filed with the Securities and Exchange Commission (SEC) on March 22, 2022, quarterly report on Form 10-Q for the three months ended March 31, 2022 filed with the SEC on May 10, 2022, and other filings and reports that CinCor may file from time to time with the SEC including its quarterly report on Form 10-Q for the three months ended June 30, 2022,. Other risks and uncertainties of which CinCor is not currently aware may also affect the company’s forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. CinCor undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts:
Terry Coelho
CinCor Pharma, Inc.
EVP, CFO and CBDO

Investors:
Bob Yedid
LifeSci Advisors
ir@CinCor.com

Myovant Sciences and Pfizer Receive U.S. FDA Approval of MYFEMBREE®, a Once-Daily Treatment for the Management of Moderate to Severe Pain Associated With Endometriosis

Dimitri Z — Tue, 09 Aug 2022 00:22:20 +0000

Myovant Sciences and Pfizer Receive U.S. FDA Approval of MYFEMBREE®, a Once-Daily Treatment for the Management of Moderate to Severe Pain Associated With Endometriosis

August 5, 2022 at 8:50 PM EDT

PDF Version

Data from the Phase 3 SPIRIT program showed MYFEMBREE reduced menstrual pain and non-menstrual pelvic pain in premenopausal women with endometriosis, and a loss of mean bone mineral density of less than 1% from baseline through one year of treatment
Myovant and Pfizer will continue to jointly commercialize MYFEMBREE, with product available immediately
Myovant to host conference call and webcast on Monday, August 8, 2022, at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time

BASEL, Switzerland and NEW YORK, Aug. 05, 2022 (GLOBE NEWSWIRE) — Myovant Sciences (NYSE: MYOV) and Pfizer Inc. (NYSE: PFE) today announced that the U.S. Food and Drug Administration (FDA) has approved MYFEMBREE® (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) as a one-pill, once-a-day therapy for the management of moderate to severe pain associated with endometriosis in pre-menopausal women, with a treatment duration of up to 24 months. The approval is supported by one-year efficacy and safety data, including 24-week data from the Phase 3 SPIRIT 1 and SPIRIT 2 trials, which were published in The Lancet, and the first 28 weeks of an open-label extension study for eligible women who completed either SPIRIT 1 or SPIRIT 2. MYFEMBREE also is approved for heavy menstrual bleeding associated with uterine fibroids in pre-menopausal women. Myovant and Pfizer will continue to jointly commercialize MYFEMBREE in the U.S. and product is available immediately.

“Endometriosis is a painful, chronic disease with limited therapies to manage symptoms,” said Juan Camilo Arjona Ferreira, M.D., Chief Medical Officer of Myovant Sciences, Inc. “The new MYFEMBREE indication helps advance our mission to redefine care for women by helping address a disease with high unmet need, giving women and physicians a new meaningful treatment option to manage moderate to severe pain associated with endometriosis.”

“This approval is an important milestone reflecting Pfizer and Myovant’s commitment to women’s health in areas of significant unmet need,” said James Rusnak, M.D., Ph.D., Senior Vice President, Chief Development Officer, Internal Medicine and Hospital, Global Product Development at Pfizer. “We look forward to making MYFEMBREE available to women with endometriosis and broadening their options in managing this complex disorder.”

MYFEMBREE offers an effective, once-daily treatment option for the management of moderate to severe pain associated with endometriosis, with a treatment duration of up to 24 months. Endometriosis is a serious chronic condition that requires long-term interventions. Optimization of medical therapies is the recommended treatment paradigm.1,2,3 MYFEMBREE introduces an option for up to two years of pharmacological management of moderate to severe pain associated with endometriosis in pre-menopausal women.

“The data from the SPIRIT studies showed the clinical benefit that relugolix combination therapy can have on moderate to severe pain associated with endometriosis and how it can impact patients,” said Linda Giudice, M.D., Ph.D., Distinguished Professor at the University of California, San Francisco (UCSF), and Chair, SPIRIT Program Steering Committee. “This newly approved option for patients with pain from endometriosis offers the convenience of one pill taken once daily with a mean change in bone mineral density of <1% that did not appear to worsen at 12 months of treatment; however, monitoring is recommended.”

This approval is supported by one-year data from the Phase 3 SPIRIT program, which included two 24-week multi-national clinical studies (SPIRIT 1 and SPIRIT 2) in more than 1,200 women with pain associated with endometriosis, as well as the first 28 weeks of an open-label extension study to assess its longer-term use. Overall, these studies showed MYFEMBREE reduced menstrual pain and non-menstrual pelvic pain with a loss of mean bone mineral density of less than 1% from baseline through one year of treatment.4

SPIRIT 1 and 2 each met their co-primary endpoints with 75% of women in the MYFEMBREE group in both studies achieving a clinically meaningful reduction in dysmenorrhea compared with 27% and 30% of women in the placebo groups at Week 24, respectively (both p <0.0001). For non-menstrual pelvic pain, treatment with MYFEMBREE demonstrated a clinically meaningful reduction in pain in 59% and 66% of women, compared with 40% and 43% of women in the placebo groups (p < 0.0001). Adverse reactions occurring in at least 3% of women treated with MYFEMBREE and greater than placebo were: headache, vasomotor symptoms, mood disorders, abnormal uterine bleeding, nausea, toothache, back pain, decreased sexual desire and arousal, arthralgia, fatigue, and dizziness.

The open-label extension study for eligible women who completed either SPIRIT 1 or SPIRIT 2 showed mean bone mineral density loss of less than 1% from baseline through one year of treatment; some patients (19.7%) had losses >3%. Annual bone density measurement is recommended while treating women for endometriosis.

MYFEMBREE is available immediately to patients with moderate to severe pain associated with endometriosis with a prescription from their healthcare provider. Myovant and Pfizer also are committed to supporting women in the U.S. who are prescribed MYFEMBREE throughout their treatment journeys. The MYFEMBREE Support Program provides access support services, including insurance benefits checks, prior authorization support, co-pay support for commercially insured patients, and patient assistance for qualifying uninsured patients. Program terms and conditions apply. For more information and additional resources, please contact 833-MYFEMBREE (833-693-3627), 8 a.m. – 8 p.m. Eastern Time, Monday – Friday.

Myovant Conference Call

Myovant will hold a conference call on Monday, August 8, 2022, at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time to discuss the FDA approval of MYFEMBREE for the management of moderate to severe pain associated with endometriosis. Investors and the general public may access the live webcast here. The live webcast can also be accessed by visiting the company’s investor relations page of Myovant’s website at: https://investors.myovant.com/.

About Endometriosis

Endometriosis is a condition in which tissue similar to the uterine lining is found outside of the uterine cavity, which often causes disruptive symptoms like painful periods, fatigue, pain in the lower back and abdomen, heavy menstrual bleeding, and even painful or difficult sexual intercourse. For endometriosis-associated pain, current treatment options include prescription and over-the-counter pain medications, combined oral contraceptives, progestins, danazol, GnRH agonists and antagonists, and surgical interventions.

Endometriosis can also impact general physical, mental, and social well-being, requiring a multi-disciplinary approach to care. Approximately 190 million women suffer from symptoms of endometriosis globally.5 In the U.S., there are approximately 7.5 million premenopausal women with endometriosis and approximately 75-80 percent of them are symptomatic.6,7,8,9 Many women with pain associated with endometriosis are not able to manage their pain symptoms with current treatment options, underscoring the high unmet need for this disease.10 It can take between four and eleven years to get an endometriosis diagnosis11,12,13 and for some women, current treatment options do not provide relief.14

About MYFEMBREE®

MYFEMBREE (relugolix, estradiol, and norethindrone acetate) is a once-daily oral treatment approved by the U.S. Food and Drug Administration for the management of moderate to severe pain associated with endometriosis, with a treatment duration of up to 24 months. It is also currently available in the U.S. for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months. MYFEMBREE contains relugolix, which reduces the amount of estrogen (and other hormones) produced by ovaries, estradiol (an estrogen) which may reduce the risk of bone loss, and norethindrone acetate (a progestin) which is necessary when women with a uterus (womb) take estrogen.

For full prescribing information including Boxed Warning and patient information, please click here.

Indications and Usage

MYFEMBREE is indicated in premenopausal women for the management of:

Heavy menstrual bleeding associated with uterine leiomyomas (fibroids)
Moderate to severe pain associated with endometriosis

Limitations of Use: Use of MYFEMBREE should be limited to 24 months due to the risk of continued bone loss which may not be reversible.

IMPORTANT SAFETY INFORMATION

BOXED WARNING: THROMBOEMBOLIC DISORDERS AND VASCULAR EVENTS

Estrogen and progestin combination products, including MYFEMBREE, increase the risk of thrombotic or thromboembolic disorders including pulmonary embolism, deep vein thrombosis, stroke and myocardial infarction, especially in women at increased risk for these events.
MYFEMBREE is contraindicated in women with current or a history of thrombotic or thromboembolic disorders and in women at increased risk for these events, including women over 35 years of age who smoke or women with uncontrolled hypertension.

CONTRAINDICATIONS

MYFEMBREE is contraindicated in women with any of the following: high risk of arterial, venous thrombotic, or thromboembolic disorder; pregnancy; known osteoporosis; current or history of breast cancer or other hormone-sensitive malignancies; known hepatic impairment or disease; undiagnosed abnormal uterine bleeding; known hypersensitivity to components of MYFEMBREE.

WARNINGS AND PRECAUTIONS

Thromboembolic Disorders: Discontinue immediately if an arterial or venous thrombotic, cardiovascular, or cerebrovascular event occurs or is suspected. Discontinue at least 4 to 6 weeks before surgery associated with an increased risk of thromboembolism, or during periods of prolonged immobilization, if feasible. Discontinue immediately if there is sudden unexplained partial or complete loss of vision, proptosis, diplopia, papilledema, or retinal vascular lesions and evaluate for retinal vein thrombosis as these have been reported with estrogens and progestins.

Bone Loss: MYFEMBREE may cause a decrease in bone mineral density (BMD) in some patients, which may be greater with increasing duration of use and may not be completely reversible after stopping treatment. Consider the benefits and risks in patients with a history of low trauma fracture or risk factors for osteoporosis or bone loss, including medications that may decrease BMD. Assessment of BMD by dual-energy X-ray absorptiometry (DXA) is recommended at baseline in all women. During treatment, periodic DXA is recommended for women with heavy menstrual bleeding due to uterine fibroids; in those with moderate to severe endometriosis pain, annual DXA is recommended. Consider discontinuing MYFEMBREE if the risk of bone loss exceeds the potential benefit.

Hormone-Sensitive Malignancies: Discontinue MYFEMBREE if a hormone-sensitive malignancy is diagnosed. Surveillance measures in accordance with standard of care, such as breast examinations and mammography are recommended. Use of estrogen alone or estrogen plus progestin has resulted in abnormal mammograms requiring further evaluation.

Suicidal Ideation and Mood Disorders (Including Depression): Evaluate patients with a history of suicidal ideation, depression, and mood disorders prior to initiating treatment. Monitor patients for mood changes and depressive symptoms including shortly after initiating treatment, to determine whether the risks of continuing therapy with MYFEMBREE outweigh the benefits. Patients with new or worsening depression, anxiety, or other mood changes should be referred to a mental health professional, as appropriate. Advise patients to seek immediate medical attention for suicidal ideation and behavior and reevaluate the benefits and risks of continuing MYFEMBREE. Gonadotropin-releasing hormone receptor antagonists, including MYFEMBREE, have been associated with mood disorders (including depression) and suicidal ideation.

Hepatic Impairment and Transaminase Elevations: Steroid hormones may be poorly metabolized in these patients. Instruct women to promptly seek medical attention for symptoms or signs that may reflect liver injury, such as jaundice or right upper abdominal pain. Acute liver test abnormalities may necessitate the discontinuation of MYFEMBREE use until the liver tests return to normal and MYFEMBREE causation has been excluded.

Gallbladder Disease or History of Cholestatic Jaundice: Discontinue MYFEMBREE if signs or symptoms of gallbladder disease or jaundice occur. For women with a history of cholestatic jaundice associated with past estrogen use or with pregnancy, assess the risk-benefit of continuing therapy. Studies among estrogen users suggest a small increased relative risk of developing gallbladder disease.

Elevated Blood Pressure: For women with well-controlled hypertension, monitor blood pressure and stop MYFEMBREE if blood pressure rises significantly.

Change in Menstrual Bleeding Pattern and Reduced Ability to Recognize Pregnancy: Advise women to use non-hormonal contraception during treatment and for one week after discontinuing MYFEMBREE. Avoid concomitant use of hormonal contraceptives. MYFEMBREE may delay the ability to recognize pregnancy because it alters menstrual bleeding. Perform testing if pregnancy is suspected and discontinue MYFEMBREE if pregnancy is confirmed.

Risk of Early Pregnancy Loss: MYFEMBREE can cause early pregnancy loss. Exclude pregnancy before initiating and advise women to use effective non-hormonal contraception.

Uterine Fibroid Prolapse or Expulsion: Advise women with known or suspected submucosal uterine fibroids about the possibility of uterine fibroid prolapse or expulsion and instruct them to contact their physician if severe bleeding and/or cramping occurs.

Alopecia: Alopecia, hair loss, and hair thinning were reported in phase 3 trials with MYFEMBREE. Consider discontinuing MYFEMBREE if hair loss becomes a concern. Whether the hair loss is reversible is unknown.

Effects on Carbohydrate and Lipid Metabolism: More frequent monitoring in MYFEMBREE-treated women with prediabetes and diabetes may be necessary. MYFEMBREE may decrease glucose tolerance and result in increased blood glucose concentrations. Monitor lipid levels and consider discontinuing if hypercholesterolemia or hypertriglyceridemia worsens. In women with pre-existing hypertriglyceridemia, estrogen therapy may be associated with elevations in triglycerides levels leading to pancreatitis. Use of MYFEMBREE is associated with increases in total cholesterol and LDL-C.

Effect on Other Laboratory Results: Patients with hypothyroidism and hypoadrenalism may require higher doses of thyroid hormone or cortisol replacement therapy. Use of estrogen and progestin combinations may raise serum concentrations of binding proteins (e.g., thyroid-binding globulin, corticosteroid-binding globulin), which may reduce free thyroid or corticosteroid hormone levels. Use of estrogen and progestin may also affect the levels of sex hormone-binding globulin, and coagulation factors.

Hypersensitivity Reactions: Immediately discontinue MYFEMBREE if a hypersensitivity reaction occurs.

ADVERSE REACTIONS: Most common adverse reactions for MYFEMBREE (incidence ≥3% and greater than placebo) were:

Heavy menstrual bleeding associated with uterine fibroids: vasomotor symptoms, abnormal uterine bleeding, alopecia, and decreased libido.
Moderate to severe pain associated with endometriosis: headache, vasomotor symptoms, mood disorders, abnormal uterine bleeding, nausea, toothache, back pain, decreased sexual desire and arousal, arthralgia, fatigue, and dizziness.

These are not all the possible side effects of MYFEMBREE.

DRUG INTERACTIONS: P-gp Inhibitors: Avoid use of MYFEMBREE with oral P-gp inhibitors. If use is unavoidable, take MYFEMBREE first, separate dosing by at least 6 hours, and monitor patients for adverse reactions. Combined P-gp and Strong CYP3A Inducers: Avoid use of MYFEMBREE with combined P-gp and strong CYP3A inducers.

LACTATION: Advise women not to breastfeed while taking MYFEMBREE.

About Myovant Sciences

Myovant Sciences aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. Founded in 2016, Myovant has executed five successful Phase 3 clinical trials across oncology and women’s health leading to three regulatory approvals by the U.S. Food and Drug Administration (FDA) for men with advanced prostate cancer, women with heavy menstrual bleeding associated with uterine fibroids, and pre-menopausal women with moderate to severe pain associated with endometriosis, respectively. Myovant also has received regulatory approvals by the European Commission (EC) and the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) for women with symptomatic uterine fibroids and for men with advanced hormone-sensitive prostate cancer. Myovant has a supplemental New Drug Application under review with the FDA for updates to the United States Prescribing Information (USPI) based on safety and efficacy data from the Phase 3 LIBERTY randomized withdrawal study (RWS) of MYFEMBREE in premenopausal women with heavy menstrual bleeding due to uterine fibroids for up to two years. Myovant also is conducting a Phase 3 study to evaluate the prevention of pregnancy in women with uterine fibroids or endometriosis. Myovant also is developing MVT-602, an investigational oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Pharma Co., Ltd., is Myovant’s majority shareholder. For more information, please visit www.myovant.com. Follow @Myovant on Twitter and LinkedIn.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @PfizerNews, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Myovant Sciences Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In this press release, forward-looking statements include, but are not limited to, all statements reflecting Myovant Sciences’ expectations, including: statements regarding Myovant’s aspiration to redefine care for women and for men; the expectations regarding the continued commercialization of MYFEMBREE by Myovant and Pfizer jointly in the U.S. and the timeline of product availability; the expectations that MYFEMBREE’s indication helps advance Myovant’s mission to redefine care for women by helping address a disease with high unmet need, giving women and physicians a new meaningful treatment option to manage moderate to severe pain associated with endometriosis in Dr. Arjona Ferreira’s quote; the expectation of making MYFEMBREE available to women with endometriosis and broadening their options in managing this complex disorder in Dr. Rusnak’s quote; and the expectations of the MYFEMBREE Support Program for patients and the features of such program.

Myovant Sciences’ forward-looking statements are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties, assumptions, and other factors known and unknown that could cause actual results and the timing of certain events to differ materially from future results expressed or implied by the forward-looking statements, including unforeseen circumstances or other disruptions to normal business operations arising from or related to the COVID-19 pandemic and the conflict in Ukraine. Myovant Sciences cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur, and actual results could differ materially from those expressed or implied by these forward-looking statements. Factors that could materially affect Myovant Sciences’ operations and future prospects or which could cause actual results to differ materially from expectations include, but are not limited to, the risks and uncertainties listed in Myovant Sciences’ filings with the United States Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in Myovant Sciences’ Quarterly Report on Form 10-Q filed on July 27, 2022, as such risk factors may be amended, supplemented, or superseded from time to time. These risks are not exhaustive. New risk factors emerge from time to time, and it is not possible for Myovant Sciences’ management to predict all risk factors, nor can Myovant Sciences assess the impact of all factors on its business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. You should not place undue reliance on the forward- looking statements in this press release, which speak only as of the date hereof, and, except as required by law, Myovant Sciences undertakes no obligation to update these forward-looking statements to reflect events or circumstances after the date of such statements.

Pfizer Disclosure Notice

The information contained in this release is as of August 5, 2022. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about MYFEMBREE® (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg), a new indication in the U.S. for the management of moderate to severe pain associated with endometriosis in pre-menopausal women, and a collaboration between Pfizer and Myovant Sciences to develop and commercialize relugolix in advanced prostate cancer and women’s health, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of MYFEMBREE; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical studies; whether and when applications may be filed for any other potential indications for MYFEMBREE; whether and when regulatory authorities may approve any such applications for MYFEMBREE that may be pending or filed, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether MYFEMBREE will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of MYFEMBREE; whether our collaboration with Myovant Sciences will be successful; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2021 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

Myovant Sciences Contacts

Investor Contact:

Uneek Mehra

Chief Financial and Business Officer

Myovant Sciences, Inc.

investors@myovant.com

Media Contact:
Noelle Cloud Dugan

Vice President, Corporate Communications

Myovant Sciences, Inc.

media@myovant.com

Pfizer Contacts:

Media Relations

PfizerMediaRelations@Pfizer.com

+1 (212) 733-1226

Investor Relations

IR@Pfizer.com

+1 (212) 733-4848

Myovant Sciences, Inc.

FDA Approves Arcutis’ ZORYVE™ (Roflumilast) Cream 0.3% For the Treatment of Plaque Psoriasis in Individuals Age 12 and Older

Dimitri Z — Mon, 01 Aug 2022 00:22:20 +0000

FDA Approves Arcutis’ ZORYVE™ (Roflumilast) Cream 0.3% For the Treatment of Plaque Psoriasis in Individuals Age 12 and Older

First and only topical PDE4 inhibitor approved for the treatment of plaque psoriasis, including intertriginous psoriasis
Approved for once-daily treatment in mild, moderate, and severe plaque psoriasis with no limitations on duration of use
Established efficacy – provides rapid clearance of plaques and reduction of itch in all affected areas of the body
Safe and very well-tolerated, steroid-free cream with minimal application site reactions
Commercial product expected to be available by mid-August
Management will host conference call on Monday, August 1 at 8:30 a.m. EDT
Arcutis expects to draw an additional $125 million from the Company’s debt facility with SLR Capital Partners

WESTLAKE VILLAGE, Calif., July 29, 2022 (GLOBE NEWSWIRE) — Arcutis Biotherapeutics, Inc. (NASDAQ: ARQT), an early commercial-stage biopharmaceutical company focused on developing meaningful innovations in immuno-dermatology, announced today that the U.S. Food and Drug Administration (FDA) has approved the New Drug Application (NDA) for ZORYVE (roflumilast) cream 0.3% for the treatment of plaque psoriasis, including intertriginous areas, in patients 12 years of age or older. The first and only topical phosphodiesterase-4 (PDE4) inhibitor approved for the treatment of plaque psoriasis, ZORYVE provides rapid clearance of psoriasis plaques and reduces itch in all affected areas of the body. ZORYVE — a once-daily, steroid-free cream in a safe and well tolerated, patient-friendly formulation — is uniquely formulated to simplify disease management for people living with plaque psoriasis.

“Today Arcutis has reached a major milestone, with our ability to offer this next generation topical PDE4 inhibitor to both adults and adolescents with plaque psoriasis. ZORYVE’s combination of efficacy, safety, and tolerability, coupled with our proprietary HydroARQ Technology formulation, is designed to fit into patients’ everyday lives with no restrictions on duration of use,” said Frank Watanabe, President and CEO of Arcutis. “Additionally, ZORYVE has been shown to rapidly clear plaques and reduce itch across all areas of the body. ZORYVE is the only topical for which data focused on the treatment of intertriginous plaques — a common area affected by plaque psoriasis — have been specifically generated. This FDA approval is the fruition of our efforts, and we are excited to launch ZORYVE, with expected product availability by mid-August.”

Topical therapies remain the primary treatment option for the vast majority of individuals with plaque psoriasis, a common immune-mediated skin disease that affects approximately nine million people in the U.S. and is the most frequent type of psoriasis occurring in both adults and adolescents. Severity can range between mild, moderate, and severe, with itch being the most burdensome and frequently reported symptom.

While the disease may affect any area of the body, plaques in certain areas, like the face, elbows and knees, genitalia, and intertriginous areas (areas of skin-to-skin contact), present unique treatment challenges. As a result, individuals with psoriasis are often prescribed multiple topical medications for different areas, which makes for a complicated treatment regimen.

“In multiple clinical trials, ZORYVE was proven to be safe and effective, with improvements in disease clearance in hard-to-treat areas like knees and elbows, as well as in sensitive areas such as the face, genitalia, and intertriginous areas. ZORYVE is very well tolerated, which is an important consideration for treating a chronic skin disease such as plaque psoriasis,” said Mark Lebwohl M.D., FAAD, principal investigator and Dean for Clinical Therapeutics and Chairman Emeritus of the Kimberly and Eric J. Waldman Department of Dermatology at the Icahn School of Medicine at Mount Sinai. “With this FDA approval, adults and adolescents with psoriasis and their dermatologists have a new steroid-free treatment option for use on all affected areas of the body.”

ZORYVE features HydroARQ Technology™, a proprietary drug delivery formulation that creates a non-greasy moisturizing cream that spreads easily and absorbs quickly.

“Plaque psoriasis is a challenging disease and finding the right treatment option can be complicated, especially if individuals have to use multiple treatments for different parts of their body. We welcome a new treatment option that can make a meaningful difference for adults and adolescents with plaque psoriasis,” says Leah M. Howard, President and CEO of the National Psoriasis Foundation. “Our hope is that new treatments translate into improved outcomes and help alleviate the burdens of chronic disease for people impacted by psoriasis.”

Arcutis intends to make ZORYVE widely available via key wholesaler and national dermatology pharmacy channels as a new treatment option by mid-August, and the Company is dedicated to affordable access to therapy. The ZORYVE Direct patient support program will help commercially insured individuals with plaque psoriasis get access and start ZORYVE treatment as prescribed by their healthcare provider quickly and easily by helping them navigate the payer process, lowering the out-of-pocket cost for eligible patients, and offering programs that support staying on therapy.† Arcutis will also offer the Arcutis Cares patient assistance program (PAP) – the first of its kind for a topical psoriasis treatment – that will provide ZORYVE at no cost for financially eligible patients who are uninsured or underinsured.‡

With this approval, Arcutis has access to, and plans to draw, an additional $125 million tranche as part of the Company’s non-dilutive financing agreement with SLR Capital Partners. Combined with the Company’s cash, cash equivalents, restricted cash, and marketable securities as of June 30, 2022, this additional $125 million will provide for capital resources of over $400 million to support the launch and commercialization efforts for ZORYVE, as well as continue to advance the Company’s pipeline development initiatives.

Management will host a conference call on Monday, August 1 at 8:30 a.m. EDT. Dial-in information for conference participants may be obtained by registering for the event here. A live webcast of the call and presentation material will be available on the “Events” section of the Company’s Investor website. An archived version of the webcast will be available on the Arcutis website after the call.

ZORYVE Clinical Data

The approval is based on comprehensive results from the pivotal DERMIS-1 and DERMIS-2 (trials of PDE4 inhibition with Roflumilast for the Management of plaque psoriasIS One and Two) Phase 3 studies. In these trials, significantly more patients treated with ZORYVE achieved Investigator Global Assessment (IGA) success at Week 8 compared to vehicle (42% in DERMIS-1 and 37% in DERMIS-2 with ZORYVE compared to 6% in DERMIS-1 and 7% in DERMIS-2 with vehicle (P<0.0001 in both studies)). IGA success is defined as an IGA score of clear (0) or almost clear (1), plus a ≥2-grade IGA score improvement from baseline.

ZORYVE improved the severity and impact of itch, as early as Week 2. Two-thirds of patients with a Worst Itch-Numerical Rating Score (WI-NRS) of 4 or higher at baseline achieved a > 4-point reduction in itch at Week 8 with ZORYVE (67% vs. 26% in DERMIS-1 and 69% vs. 33% in DERMIS-2 at Week 8 (P<0.0001)).

ZORYVE is the only topical for which efficacy has been specifically demonstrated in the treatment of intertriginous psoriasis, as measured by Intertriginous IGA (I-IGA) Success (72% vs. 14% in DERMIS-1 and 68% vs. 17% in DERMIS-2 at Week 8 (P<0.0001)).

In both trials, ZORYVE was very well-tolerated with a favorable safety and tolerability profile. The most common adverse reactions reported in DERMIS-1 and -2 (≥1% of subjects treated with ZORYVE for 8 weeks), and for which the rate exceeded the rate for vehicle-treated patients, included diarrhea (3%), headache (2%), insomnia (1%), nausea (1%), application site pain (1%), upper respiratory tract infection (1%), and urinary tract infection (1%).

Of 239 individuals who continued treatment with ZORYVE for at least 52 weeks in an open-label long-term safety trial, 45% were evaluated as an IGA of “Clear” or “Almost Clear” at Week 52.

ZORYVE also demonstrated statistically significant improvements over vehicle on key secondary endpoints, including Psoriasis Area Severity Index-75 (PASI-75), and patient perceptions of signs and symptoms, such as itching, pain, and scaling, as measured by the Psoriasis Symptoms Diary (PSD). In both studies, ZORYVE improved overall signs and symptoms of psoriasis at Weeks 4 and 8 compared to vehicle.

Dr. Lebwohl reports receiving grant support and consulting fees from Arcutis Biotherapeutics.

About Psoriasis

Psoriasis is a common, non-contagious, immune-mediated skin disease that affects approximately nine million people in the United States. The majority of individuals with psoriasis develop “plaques,” or raised, red areas of skin covered with a silver or white layer of dead skin cells. The plaques’ clinical presentation may have more grayish, purplish, or brownish tones in people with darker skin tones. Psoriatic plaques are often itchy and sometimes painful and can appear on any area of the body. Plaques in certain anatomical areas present unique treatment challenges, including the face, elbows and knees, scalp, and intertriginous areas (where two skin areas may touch or rub together).

INDICATION

ZORYVE is indicated for topical treatment of plaque psoriasis, including intertriginous areas, in patients 12 years of age and older.

IMPORTANT SAFETY INFORMATION

The use of ZORYVE is contraindicated in patients with moderate to severe liver impairment (Child-Pugh B or C).

Please see full Prescribing Information.

About Arcutis

Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT) is a medical dermatology company that champions meaningful innovation to address the urgent needs of individuals living with immune-mediated dermatological diseases and conditions. With a commitment to solving the most persistent patient challenges in dermatology, Arcutis harnesses our unique dermatology development platform coupled with our dermatology expertise to build differentiated therapies against biologically validated targets. Arcutis’ dermatology development platform includes a robust pipeline with multiple clinical programs for a range of inflammatory dermatological conditions including plaque psoriasis, atopic dermatitis, and seborrheic dermatitis. For more information, visit www.arcutis.com or follow Arcutis on LinkedIn, Facebook, and Twitter.

Forward-Looking Statements

Arcutis cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the Company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding the potential for ZORYVE to simplify disease management for care of plaque psoriasis; the Company’s expected timing and plan to commercially launch ZORYVE by mid-August; and the Company’s plan to draw down on its loan agreement. These statements are subject to substantial known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. Risks and uncertainties that may cause our actual results to differ include risks inherent in our business, conditions limiting our ability to access additional capital under our debt financing agreement, the impact of competition and other important factors discussed in the “Risk Factors” section of our Form 10-K filed with U.S. Securities and Exchange Commission (SEC) on February 22, 2022, as amended, as well as any subsequent filings with the SEC. You should not place undue reliance on any forward-looking statements in this press release. We undertake no obligation to revise or update information herein to reflect events or circumstances in the future, even if new information becomes available. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

Contacts:

Media

Amanda Sheldon, Head of Corporate Communications

asheldon@arcutis.com

Investors

Eric McIntyre, Head of Investor Relations

emcintyre@arcutis.com

Cara Therapeutics Announces Positive Topline Results from KOMFORT Phase 2 Trial of Oral Difelikefalin for the Treatment of Pruritus in Patients with Notalgia Paresthetica

Dimitri Z — Thu, 30 Jun 2022 00:22:20 +0000

Cara Therapeutics Announces Positive Topline Results from KOMFORT Phase 2 Trial of Oral Difelikefalin for the Treatment of Pruritus in Patients with Notalgia Paresthetica

June 30, 2022

– Study achieved primary endpoint of Worst Itch-Numeric Rating Scale score change from baseline at Week 8 (p=0.001) –

– Onset of action seen at Week 1 and sustained through Week 8 –

– Statistical significance achieved on the WI-NRS ≥4-point responder analysis at Week 8 (p=0.007) –

– Oral difelikefalin was well tolerated with a consistent safety profile –

– Conference call today at 8:30 a.m. ET –

STAMFORD, Conn., June 30, 2022 (GLOBE NEWSWIRE) — Cara Therapeutics, Inc. (Nasdaq: CARA), a commercial-stage biopharmaceutical company leading a new treatment paradigm to improve the lives of patients suffering from pruritus, today announced positive topline results from its Phase 2 proof-of-concept clinical trial (KOMFORT) evaluating oral difelikefalin for the treatment of moderate-to-severe pruritus in patients with notalgia paresthetica (NP), a nerve disorder characterized by chronic pruritus of the upper to middle back.

“We are pleased to have demonstrated clinical proof of concept for oral difelikefalin in the treatment of pruritus associated with notalgia paresthetica,” said Joana Goncalves, M.D., Chief Medical Officer at Cara Therapeutics. “These topline results coupled with the results from our other programs support the broad development of oral difelikefalin across disease areas regardless of the underlying cause of pruritus. We look forward to completing our data analyses and discussing next steps with the U.S. Food and Drug Administration.”

“With no approved treatments available for notalgia paresthetica, the condition is challenging to manage and burdensome for patients,” said Mark Lebwohl, M.D., the lead investigator and Professor and Dean for Clinical Therapeutics and Chairman Emeritus of the Department of Dermatology at Icahn School of Medicine at Mount Sinai. “These are encouraging results that underscore the potential for oral difelikefalin to be the first treatment option to address pruritus associated with notalgia paresthetica.”

Phase 2 Proof-of-Concept Trial Design & Topline Results

The Phase 2 multicenter, randomized, double-blind, placebo-controlled, 8-week study was designed to evaluate the efficacy and safety of oral difelikefalin for moderate-to-severe pruritus in approximately 120 patients with NP. Patients were randomized to oral difelikefalin 2 mg taken twice daily versus placebo for 8 weeks, followed by a 4-week active extension period.

The primary efficacy endpoint was the change from baseline in the weekly mean of the daily 24-hour Worst Itch-Numeric Rating Scale (WI-NRS) score at Week 8. Other endpoints included the ≥4-point responder analysis, itch-related quality of life scores, and safety assessments.

Patients treated with oral difelikefalin achieved the primary endpoint (-4.0 difelikefalin vs. -2.4 placebo, p=0.001) with significant improvement observed as early as Week 1 and sustained through Week 8.

In addition, a statistically significantly greater proportion of patients treated with oral difelikefalin achieved a ≥4-point improvement in WI-NRS score at Week 8 vs. placebo (41% difelikefalin vs. 18% placebo, p=0.007).

Oral difelikefalin was generally well tolerated with a safety profile consistent with that seen in earlier clinical trials. The most common treatment-emergent adverse events reported in ≥5% of patients treated with oral difelikefalin and greater than placebo were: nausea, headache, dizziness, constipation and urine output increased.

Conference Call & Webcast

Cara management will host a conference call and live webcast today at 8:30 a.m. ET to discuss the positive topline results.

To participate in the conference call, please dial (855) 445-2816 (domestic) or (484) 756-4300 (international) and refer to conference ID 6999079. A live webcast of the call can be accessed under "Events & Presentations" in the News & Investors section of the Company's website at www.CaraTherapeutics.com.

An archived webcast recording will be available on the Cara website beginning approximately two hours after the call.

About Pruritus Associated with Notalgia Paresthetica

Notalgia paresthetica (NP) is a common, although under-recognized, chronic, sensory neuropathy affecting the upper back.1 It is estimated that chronic pruritus affects up to 13% of the population in the United States, and about 8% of these patients suffer from neuropathic itch, including NP.2,3 One of the hallmark features of NP is chronic pruritus, which can be significantly burdensome and undermines the affected patients’ quality of life and overall well-being. 3 The exact etiology of NP still has not been fully elucidated; however, it is widely accepted that NP is a sensory neuropathy caused by alteration and damage to thoracic spinal nerves.3

The management of NP is challenging and is often resistant to multiple therapies. There is currently no approved treatment for NP and conventional treatments for pruritus, such as antihistamines and topical steroids, are largely ineffective.4

References:

Matthew Howard, Lukas Sahhar, Frank Andrews, Ralph Bergman and Douglas Gin. Notalgia paresthetica: a review for dermatologists. International J of Dermatology 2018,57, 388-392.
Manuel P. Pereira, Hannah Lüling, Annette Dieckhöfer, Sabine Steinke, Claudia Zeidler and Sonja Ständer. Brachioradial Pruritus and Notalgia Paraesthetica: A Comparative Observational Study of Clinical Presentation and Morphological Pathologies. Acta DV 2018; 98:82-88.
Mollanazar, N.K., Koch, S.D. & Yosipovitch, G. Epidemiology of Chronic Pruritus: Where Have We Been and Where Are We Going?. Curr Derm 3. Rep 4, 20–29 (2015)
Mirna Šitum, Maja Koli?, Nika Franceschi and Marko Pe?ina. Notalgia Paresthetica. Acta Clin Croat 2018; 57:721-725.
Ahmed Ansari, David Weinstein & Naveed Sami. Notalgia paresthetica: treatment review and algorithmic approach. Journal of Dermatological Treatment 2019.

About Cara Therapeutics

Cara Therapeutics is a commercial-stage biopharmaceutical company leading a new treatment paradigm to improve the lives of patients suffering from pruritus. The Company’s novel KORSUVA™ (difelikefalin) injection is the first and only FDA-approved treatment for moderate-to-severe pruritus associated with chronic kidney disease in adults undergoing hemodialysis. The Company is developing an oral formulation of difelikefalin and has initiated Phase 3 programs for the treatment of pruritus in patients with non-dialysis dependent advanced chronic kidney disease and atopic dermatitis. The Company has completed the placebocontrolled phase of a Phase 2 proof-of-concept trial of oral difelikefalin for the treatment of moderate-to-severe pruritus in patients with notalgia paresthetica. A Phase 2 proof-of-concept trial in primary biliary cholangitis patients with moderate-to-severe pruritus is ongoing. For more information, visit www.CaraTherapeutics.com and follow the company on Twitter, LinkedIn and Instagram.

Forward-looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Examples of these forward-looking statements include statements concerning the Company’s planned future regulatory submissions and potential future regulatory approvals, expected timing of the initiation, enrollment and data readouts from the Company’s planned and ongoing clinical trials, the potential results of ongoing clinical trials, timing of future regulatory and development milestones for the Company’s product candidates, the potential for the Company’s product candidates to be alternatives in the therapeutic areas investigated, including NP, and the potential for oral difelikefalin to address additional pruritic indications, the size and growth of the potential markets for pruritus management, the Company’s expected cash reach, and the potential impact of COVID-19 on the Company’s clinical development and regulatory timelines and plans. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Risks are described more fully in Cara Therapeutics’ filings with the Securities and Exchange Commission, including the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the year ending December 31, 2021 and its other documents subsequently filed with or furnished to the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Cara Therapeutics undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

MEDIA CONTACT:

Annie Spinetta

6 Degrees

973-768-2170

aspinetta@6degreespr.com

INVESTOR CONTACT:

Iris Francesconi, Ph.D.

Cara Therapeutics

203-406-3700

investor@caratherapeutics.com

Source: Cara Therapeutics, Inc.

FDA Approves Mycovia Pharmaceuticals’ VIVJOA™ (oteseconazole), the First and Only FDA-Approved Medication for Recurrent Vulvovaginal Candidiasis (Chronic Yeast Infection)

Dimitri Z — Mon, 02 May 2022 00:22:20 +0000

FDA Approves Mycovia Pharmaceuticals’ VIVJOA™ (oteseconazole), the First and Only FDA-Approved Medication for Recurrent Vulvovaginal Candidiasis (Chronic Yeast Infection)

– Approval of VIVJOA™ marks a significant therapeutic advancement for reducing the incidence of RVVC,

a condition with substantial unmet need, in permanently infertile and postmenopausal women

– VIVJOA™ is the first FDA approval in Mycovia’s pipeline of novel treatments for fungal infections

– U.S. commercial launch of VIVJOA™ expected in Q2

Durham, N.C. – April 28, 2022 – The U.S. Food and Drug Administration (FDA) approved VIVJOA™ (oteseconazole capsules), an azole antifungal indicated to reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who are NOT of reproductive potential. VIVJOA is the first and only FDA-approved medication for this condition and provides sustained efficacy demonstrated by significant long-term reduction of RVVC recurrence through 50 weeks versus comparators. VIVJOA is the first FDA-approved product for Mycovia Pharmaceuticals, Inc. (Mycovia), an emerging biopharmaceutical company dedicated to recognizing and empowering those living with unmet medical needs by developing novel therapies.

RVVC, also known as chronic yeast infection, is defined by the Centers for Disease Control and Prevention (CDC) as three or more symptomatic acute episodes of yeast infection in 12 months. RVVC is a distinct condition from vulvovaginal candidiasis (VVC), and until now, there have been no FDA-approved medications specifically indicated for it. Nearly 75% of all adult women will have at least one yeast infection in their lifetime, with approximately half experiencing a recurrence. Of those women, up to 9% develop RVVC.

“After nearly two decades of living with chronic yeast infection and feeling like there was no hope from the itchiness, irritation and constant dread of when the next yeast infection would return, I was overjoyed to even be a part of this clinical trial,” said Leslie Ivey, RVVC patient and clinical trial participant. “It is gratifying to see RVVC finally get the attention it deserves.”

Symptoms of RVVC include vaginal itching, burning, irritation and inflammation. Some women may experience abnormal vaginal discharge and painful sexual intercourse or urination, causing variable but often severe discomfort and pain.

VIVJOA’s FDA approval is based upon the positive results from three Phase 3 trials of oteseconazole – two global, pivotal VIOLET studies and one U.S.-focused ultraVIOLET study, including 875 patients at 232 sites across 11 countries. In the two global VIOLET studies, 93.3% and 96.1% of women with RVVC who received VIVJOA did not have a recurrence for the 48-week maintenance period compared to 57.2% and 60.6% of patients who received placebo (p <0.001). In the ultraVIOLET study, 89.7% of women with RVVC who received VIVJOA cleared their initial yeast infection and did not have a recurrence for the 50-week maintenance period compared to 57.1% of those who received fluconazole followed by placebo (p <0.001). The most common side effects reported in Phase 3 clinical studies were headache (7.4%) and nausea (3.6%). VIVJOA is contraindicated in those with a hypersensitivity to oteseconazole, and based on data from rat studies, also in females who are of reproductive potential, pregnant, or lactating. Please see additional Important Safety Information below.

Patrick Jordan, CEO of Mycovia Pharmaceuticals and Partner at NovaQuest Capital Management, stated, “We celebrate this important milestone for Mycovia, as VIVJOA is the first antifungal in our pipeline to obtain FDA approval and achieves our goal to fulfill a previously unmet medical need among women suffering from RVVC. We are honored to lead this advancement in women’s health.”

“We believe the market need for VIVJOA is strong, and we are eager to execute our commercial plans,” Jordan continued. “As we enter a new chapter of our history as a commercial biopharmaceutical company, we will continue driving our mission forward to develop novel therapies for overlooked conditions.”

Oteseconazole is designed to inhibit fungal CYP51, which is required for fungal cell wall integrity, and this selective interaction is also toxic to fungi, resulting in the inhibition of fungal growth. Due to its chemical structure, oteseconazole has a lower affinity for human CYP enzymes as compared to fungal CYP enzymes. The FDA granted oteseconazole Qualified Infectious Disease Product and Fast Track designations.

“A medicine with VIVJOA’s sustained efficacy combined with the clinical safety profile has been long needed, as until now, physicians and their patients have had no FDA-approved medications for RVVC,” stated Stephen Brand, Ph.D., Chief Development Officer of Mycovia. “We are excited to be the first to offer a medication designed specifically for RVVC, a challenging and chronic condition that is expected to increase in prevalence over the next decade.”

Mycovia is planning its commercial launch of VIVJOA™ in the second quarter of 2022.

About Recurrent Vulvovaginal Candidiasis

RVVC is a debilitating, chronic infectious condition that affects 138 million women worldwide each year. RVVC, also known as chronic yeast infection, is a distinct condition from vulvovaginal candidiasis (VVC) and defined as three or more symptomatic acute episodes of yeast infection in 12 months. Primary symptoms include vaginal itching, burning, irritation and inflammation. Some women may experience abnormal vaginal discharge and painful sexual intercourse or urination, causing variable but often severe discomfort and pain.

About VIVJOA™

VIVJOA™ (oteseconazole) is an azole antifungal indicated to reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who are NOT of reproductive potential. VIVJOA is the first and only FDA-approved medication that provides sustained efficacy demonstrated by significant long-term reduction of RVVC recurrence through 50 weeks versus comparators. Oteseconazole is designed to inhibit fungal CYP51, which is required for fungal cell wall integrity, and this selective interaction is also toxic to fungi, resulting in the inhibition of fungal growth. Due to its chemical structure, oteseconazole has a lower affinity for human CYP enzymes as compared to fungal CYP enzymes. The FDA approved VIVJOA based upon the positive results from three Phase 3 clinical trials of oteseconazole – two global, pivotal VIOLET studies and one U.S.-focused ultraVIOLET study, including 875 patients at 232 sites across 11 countries.

Please click here for full Prescribing Information.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

VIVJOA is contraindicated in females of reproductive potential. Females who are NOT of reproductive potential are defined as: persons who are biological females who are postmenopausal or have another reason for permanent infertility (e.g., tubal ligation, hysterectomy, salpingo-oophorectomy).

VIVJOA is contraindicated in pregnant and lactating women.

VIVJOA is contraindicated in patients with known hypersensitivity to oteseconazole.

WARNINGS AND PRECAUTIONS

Based on animal studies, VIVJOA may cause fetal harm. The drug exposure window of approximately 690 days (based on 5 times the half-life of oteseconazole) precludes adequate mitigation of the embryo-fetal toxicity risks. Advise patients that VIVJOA is contraindicated in females of reproductive potential, and in pregnant and lactating women because of potential risks to a fetus or breastfed infant.

ADVERSE REACTIONS

The most frequently reported adverse reactions among VIVJOA-treated patients in clinical studies included headache (7.4%) and nausea (3.6%).

DRUG INTERACTIONS

VIVJOA is a Breast Cancer Resistance Protein (BCRP) inhibitor. Concomitant use of VIVJOA with BCRP substrates (e.g., rosuvastatin) may increase the exposure of BCRP substrates, which may increase the risk of adverse reactions associated with these drugs.

Please see full Prescribing Information and Patient Information.

To report SUSPECTED ADVERSE REACTIONS, contact Mycovia Pharmaceuticals, Inc. at 1-855-299-0637 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

About Mycovia Pharmaceuticals Mycovia Pharmaceuticals is an emerging biopharmaceutical company dedicated to recognizing and empowering those living with unmet medical needs by developing novel therapies. VIVJOA™ (oteseconazole), the first FDA-approved product for Mycovia, is an azole antifungal indicated to reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who are NOT of reproductive potential. Oteseconazole received FDA Qualified Infectious Disease Product and Fast-Track designations to become the first FDA-approved therapy for RVVC. In 2019, Mycovia licensed oteseconazole to Jiangsu Hengrui Pharmaceuticals Co., Ltd., to develop and commercialize oteseconazole in China, including mainland China, Hong Kong, Macau and Taiwan, and Gedeon Richter Plc., a Hungary-based pharmaceutical company, to commercialize and manufacture oteseconazole in Europe, Russia, the Commonwealth of Independent States, Latin America and Australia. Mycovia also recognizes a tremendous potential for its oral fungal inhibitors and a growing need to treat a range of multi-drug resistant fungal pathogens. For more information, please visit www.mycovia.com.

About NovaQuest Capital Management

NovaQuest Capital Management, located in North Carolina’s Research Triangle, is a life science investment firm with a specialization in biopharmaceuticals. Founded in 2010, and with more than $2.5 billion raised across four funds, NovaQuest provides tailored capital solutions that fund innovation in biopharmaceutical development and invests in compelling healthcare companies with products and technologies aimed at helping humans and animals live healthier, longer, more productive lives. Learn more at www.novaquest.com.

Contacts:

Mycovia Pharmaceuticals, Inc.

mediarelations@mycovia.com

Media Relations

Elizabeth Comtois

FleishmanHillard

Tel: (919) 334-3786

elizabeth.comtois@fleishman.com

SCYNEXIS Announces Positive Results from Its Pivotal Phase 3 CANDLE Study of Oral Ibrexafungerp for Prevention of Recurrent Vaginal Yeast Infections, Clearing the Way for Regulatory Submission and Potential Approval of Additional Indication by End of 2022

Dimitri Z — Mon, 14 Feb 2022 00:22:15 +0000

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Ibrexafungerp, the only fungicidal oral treatment for vaginal yeast infections, successfully achieved statistically significant superiority over placebo for the primary and key secondary study endpoints.

SCYNEXIS will submit a supplemental New Drug Application (sNDA) for BREXAFEMME^® (ibrexafungerp tablets) for the prevention of recurrent vaginal yeast infections (rVVC) in the first half of 2022 with anticipated approval by the end of the year.

Additionally, a one day treatment of ibrexafungerp achieved a substantial reduction or complete elimination of symptoms in a subset of study patients who failed to respond to a three-day regimen of fluconazole.

In the study, ibrexafungerp was generally safe and well-tolerated with findings consistent with the existing BREXAFEMME label

JERSEY CITY, N.J., Feb. 10, 2022 (GLOBE NEWSWIRE) — SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company pioneering innovative medicines to overcome and prevent difficult-to-treat and drug-resistant infections, today announced positive results from its global Phase 3 CANDLE study investigating the safety and efficacy of oral ibrexafungerp for prevention of recurrent vulvovaginal candidiasis (rVVC), also known as vaginal yeast infection. The Company plans to submit the results to the U.S. Food and Drug Administration (FDA) in the first half of 2022 and anticipates receiving approval by year-end.

"Ibrexafungerp is the only oral fungicidal agent to successfully complete pivotal trials in both the acute treatment and prevention of yeast infections. As the only non-azole, we believe ibrexafungerp is changing how yeast infections are treated," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. "These important results support an additional indication for BREXAFEMME for the prevention of rVVC. This exciting achievement is another step in our efforts to bring to market meaningful and innovative solutions in the fight against a broad range of fungal infections and enhance shareholder value.”

This trial was conducted under a Special Protocol Assessment (SPA), a process in which sponsors work with FDA to reach agreement on the design and size of a clinical trial to adequately address scientific and regulatory requirements for a study that could support marketing approval.

The Phase 3 CANDLE study evaluated the efficacy and safety of oral ibrexafungerp compared to placebo in 260 female patients with rVVC, defined as three or more episodes of VVC in the previous 12 months. All patients initially received a three day regimen of fluconazole, and responders were randomized to receive either 300 mg ibrexafungerp BID or matching placebo one day a month, for six months. The primary endpoint was efficacy as measured by the percentage of subjects with clinical success at test-of-cure (24 weeks).

The study showed that 65.4% of patients receiving ibrexafungerp achieved clinical success by having no recurrence at all, either culture-proven, presumed or suspected, through Week 24 compared to 53.1% of placebo-treated patients (p=0.02). The advantage of ibrexafungerp over placebo was sustained over the three-month follow-up period and remained statistically significant (p=0.034). Secondary endpoints were consistent with the results of the primary analysis, and will be presented at a future medical meeting.

The study also evaluated an additional group of 24 patients who failed to respond to the initial three-day regimen of fluconazole and received a one-day open-label treatment course of ibrexafungerp (300 mg BID). In those patients treated with ibrexafungerp 71% successfully achieved a significant reduction or elimination of signs and symptoms.

In the study, ibrexafungerp was generally safe and well-tolerated. There were no serious drug-related adverse events, and no patients treated with ibrexafungerp discontinued therapy due to adverse events. The most commonly reported events were headaches and gastrointestinal events, which were mostly mild and generally consistent with the current BREXAFEMME label.

“After developing ibrexafungerp as the first approved oral non-azole agent to treat VVC, this study shows that it also prevents recurrences of the disease, as well as helps patients who failed to respond to multiple doses of fluconazole,” said David Angulo, M.D., Chief Medical Officer of SCYNEXIS. “We are excited to see these important results that further demonstrate the unmatched abilities of ibrexafungerp as a powerful oral antifungal therapy with potential broad utility against fungal infections. This is excellent news for women seeking relief from challenging and recurring yeast infections. We would like to thank all the patients and investigators who participated in this study and contributed to progressing the development of this innovative antifungal agent.”

About the CANDLE Study

CANDLE was a Phase 3, multi-center, randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of oral ibrexafungerp compared to placebo in 260 female patients with rVVC, defined as three or more episodes of VVC in the previous 12 months. The primary endpoint was clinical efficacy as measured by the percentage of subjects with documented Clinical Success (defined as subjects having no culture-proven, presumed or suspected recurrences of VVC through the test-of-cure (TOC) evaluation at Week 24).

All patients in the CANDLE study initially received a three-day regimen of oral fluconazole to treat their acute episode present at screening. Patients who responded to oral fluconazole for their acute episode were enrolled in the prevention of recurrence phase of the study and randomized to oral ibrexafungerp (300mg BID for one day) or placebo, given once per month for six months (a total of six treatment days). Patients who failed to sufficiently respond to fluconazole treatment for their acute episode were included in an open-label sub-study, in which they were offered one day of oral ibrexafungerp treatment (300mg BID) for the unresolved acute episode.

About Ibrexafungerp

Ibrexafungerp [pronounced eye-BREX-ah-FUN-jerp] is an antifungal agent and the first representative of a novel class of structurally-distinct glucan synthase inhibitors, triterpenoids. This agent combines the well-established activity of glucan synthase inhibitors with the potential flexibility of having oral and intravenous (IV) formulations. Ibrexafungerp is in late-stage development for multiple indications, including life-threatening fungal infections caused primarily by Candida (including C. auris) and Aspergillus species in hospitalized patients. It has demonstrated broad-spectrum antifungal activity, in vitro and in vivo, against multidrug-resistant pathogens, including azole- and echinocandin-resistant strains. The U.S. Food and Drug Administration (FDA) approved BREXAFEMME^®(ibrexafungerp tablets) on June 1, 2021. The FDA also granted Qualified Infectious Disease Product (QIDP) and Fast Track designations for the IV and oral formulations of ibrexafungerp for the indications of invasive candidiasis (IC) (including candidemia) and invasive aspergillosis (IA) and has granted Orphan Drug Designation for the IC and IA indications. Ibrexafungerp is formerly known as SCY-078.

IMPORTANT SAFETY INFORMATION

BREXAFEMME is contraindicated during pregnancy and in patients with a history of hypersensitivity to ibrexafungerp

BREXAFEMME administration during pregnancy may cause fetal harm based on animal studies. Prior to initiating treatment, verify pregnancy status in females of reproductive potential and advise them to use effective contraception during treatment

When administering BREXAFEMME with strong CYP3A inhibitors, the dose of BREXAFEMME should be reduced to 150 mg twice a day for one day. Administration of BREXAFEMME with strong CYP3A inducers should be avoided

Most common adverse reactions observed in clinical trials (incidence ≥2%) were diarrhea, nausea, abdominal pain, dizziness, and vomiting

To report SUSPECTED ADVERSE REACTIONS, contact SCYNEXIS, Inc. at 1-888-982-SCYX (1-888-982-7299) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For more information, visit www.brexafemme.com. Please click here for full Prescribing Information.

About SCYNEXIS

SCYNEXIS, Inc. (NASDAQ: SCYX) is a biotechnology company pioneering innovative medicines to help millions of patients worldwide overcome and prevent difficult-to-treat infections that are becoming increasingly drug-resistant. SCYNEXIS scientists are developing the company’s lead asset, ibrexafungerp (formerly known as SCY-078), as a broad-spectrum, systemic antifungal for multiple fungal indications in both the community and hospital settings. SCYNEXIS has initiated the launch of its first commercial product in the U.S., BREXAFEMME^® (ibrexafungerp tablets). The U.S. Food and Drug Administration (FDA) approved BREXAFEMME on June 1, 2021. In addition, late-stage clinical investigation of oral ibrexafungerp for the prevention of recurrent vulvovaginal candidiasis (VVC) and the treatment of life-threatening invasive fungal infections in hospitalized patients is ongoing. For more information, visit www.scynexis.com.

Forward-Looking Statements

Statements contained in this press release regarding expected future events or results are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including but not limited to statements regarding: progressing filing of an sNDA for rVVC, of ibrexafungerp, its potential use by physicians and patients in multiple healthcare settings. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, but are not limited, to: risks inherent in SCYNEXIS' ability to successfully develop and obtain FDA approval for ibrexafungerp for additional indications, including the IV formulation of ibrexafungerp; unexpected delays may occur in the timing of acceptance by the FDA of an NDA submission; the expected costs of studies and when they might begin or be concluded; SCYNEXIS’ need for additional capital resources; and SCYNEXIS' reliance on third parties to conduct SCYNEXIS' clinical studies and commercialize its products. These and other risks are described more fully in SCYNEXIS' filings with the Securities and Exchange Commission, including without limitation, its most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, including in each case under the caption "Risk Factors," and in other documents subsequently filed with or furnished to the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. SCYNEXIS undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

CONTACT:
Investor Relations
Irina Koffler
Managing Director
LifeSci Advisors, LLC
646-970-4681
ikoffler@lifesciadvisors.com

Media Relations
Gloria Gasaatura
LifeSci Communications
646-970-4688
ggasaatura@lifescicomms.com

Source: Scynexis

Released February 10, 2022

Relationships & Accomplishments – Clinical Trials Management

The New England Journal of Medicine Publishes Cologuard Plus™ Test Results from Pivotal BLUE-C Study

FDA Approves Arcutis’ ZORYVE® (roflumilast) Topical Foam, 0.3% for the Treatment of Seborrheic Dermatitis in Individuals Aged 9 Years and Older

FDA Approves Arcutis’ ZORYVE® (roflumilast) Topical Foam, 0.3% for the Treatment of Seborrheic Dermatitis in Individuals Aged 9 Years and Older

ZORYVE foam represents a highly effective, safe, well-tolerated, once-daily steroid-free foam for use on all affected areas of the body, including hair-bearing areas, with no limitations on duration of use

ZORYVE foam provides rapid disease clearance and significant reduction in itch, one of the most burdensome symptoms of seborrheic dermatitis

First drug approved for seborrheic dermatitis with a new mechanism of action in over two decades

Seborrheic dermatitis affects more than 10 million people in the United States

Commercial product expected to be available by end of January

Management will host conference call on Monday, December 18 at 8:30 a.m. EST

Arcutis Announces Positive Long-Term Results of Roflumilast Cream 0.15% Showing Durable and Improved Efficacy Over Time and Favorable Safety Profile in Treatment of Mild to Moderate Atopic Dermatitis (AD)

Seres Therapeutics and Nestlé Health Science Announce FDA Approval of VOWSTTM (fecal microbiota spores, live-brpk) for Prevention of Recurrence of C. difficile Infection in Adults Following Antibacterial Treatment for Recurrent CDI

CinCor Pharma Announces Positive Topline Data for Phase 2 BrigHtn Trial Evaluating Baxdrostat, its Selective Aldosterone Synthase Inhibitor, in Treatment-Resistant Hypertension

CinCor Pharma Announces Positive Topline Data for Phase 2 BrigHtn Trial Evaluating Baxdrostat, its Selective Aldosterone Synthase Inhibitor, in Treatment-Resistant Hypertension

Myovant Sciences and Pfizer Receive U.S. FDA Approval of MYFEMBREE®, a Once-Daily Treatment for the Management of Moderate to Severe Pain Associated With Endometriosis

Myovant Sciences and Pfizer Receive U.S. FDA Approval of MYFEMBREE®, a Once-Daily Treatment for the Management of Moderate to Severe Pain Associated With Endometriosis

FDA Approves Arcutis’ ZORYVE™ (Roflumilast) Cream 0.3% For the Treatment of Plaque Psoriasis in Individuals Age 12 and Older

FDA Approves Arcutis’ ZORYVE™ (Roflumilast) Cream 0.3% For the Treatment of Plaque Psoriasis in Individuals Age 12 and Older

Cara Therapeutics Announces Positive Topline Results from KOMFORT Phase 2 Trial of Oral Difelikefalin for the Treatment of Pruritus in Patients with Notalgia Paresthetica

FDA Approves Mycovia Pharmaceuticals’ VIVJOA™ (oteseconazole), the First and Only FDA-Approved Medication for Recurrent Vulvovaginal Candidiasis (Chronic Yeast Infection)

SCYNEXIS Announces Positive Results from Its Pivotal Phase 3 CANDLE Study of Oral Ibrexafungerp for Prevention of Recurrent Vaginal Yeast Infections, Clearing the Way for Regulatory Submission and Potential Approval of Additional Indication by End of 2022

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