Prosper-AA1 Examine Satisfies Major Endpoint for Scalp Hair Regrowth and Essential Secondary Endpoints at The two Doses
Statistically Important Hair Regrowth Noticed as Early as Eight Months
CTP-543 Has Opportunity to be Greatest-in-Course for the Remedy of Alopecia Areata
Topline Facts from Next Section 3 Demo, Prosper-AA2, Anticipated Third Quarter of 2022
LEXINGTON, Mass., May possibly 23, 2022–(Organization WIRE)–Concert Prescription drugs, Inc. (NASDAQ: CNCE) now introduced constructive topline success from its just lately done Section 3 scientific demo, Prosper-AA1, evaluating its oral investigational medication CTP-543 in adult sufferers with average to severe alopecia areata, an autoimmune condition that results in patchy or total scalp hair reduction. The primary efficacy endpoint for Thrive-AA1 was the proportion of individuals achieving an absolute Severity of Alopecia Instrument (SALT) rating of 20 or considerably less at 7 days 24 of treatment, which was fulfilled with statistical importance in each the 8 mg two times-daily and 12 mg two times-each day dose teams relative to placebo. Treatment method with CTP-543 was normally effectively tolerated.
The important secondary endpoints were being the proportion of responders on a Hair Pleasure Client Claimed End result (Professional) scale at 7 days 24 and the proportion of patients acquiring absolute SALT scores of 20 or considerably less at each and every of Months 20, 16, 12 and 8. All crucial secondary endpoints were met with statistical significance in the two dose groups.
“Currently marks an vital milestone in advancing new solutions for alopecia areata, and I’m so joyful to see these types of constructive final results from the first Stage 3 trial with CTP-543,” stated Brett King, M.D., Office of Dermatology, Yale College School of Medication and clinical investigator of Prosper-AA1. “There is a great will need for therapies for this difficult condition, and the final results from the Thrive-AA1 trial propose that CTP-543 may probably supply an vital treatment for treating alopecia areata.”
“With these persuasive Phase 3 facts, we believe that that CTP-543 has the prospective to be a finest-in-course treatment method for sufferers with alopecia areata, a disease that has long been overlooked. We are incredibly grateful to the patients and groups of scientific study professionals who take part in our trials,” explained James V. Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals. “We’re doing the job to transform the treatment landscape and hope that CTP-543 will be a single of the initial Fda-authorised therapy options for this critical ailment.”
Individuals enrolled in Prosper-AA1 ended up required to have at the very least 50 per cent scalp hair loss thanks to alopecia areata, as measured by SALT. A SALT score of 100 signifies full scalp hair reduction whilst a rating of represents no scalp hair reduction. The regular baseline SALT rating across all people was around 85.9 (corresponding to a lot less than 15% regular scalp hair coverage).
A statistically substantial proportion of clients dealt with with either 8 mg twice-everyday or 12 mg 2 times-day by day of CTP-543 knowledgeable larger scalp regrowth in contrast to placebo. The proportion of sufferers obtaining a SALT rating of 20 or much less (which means that 80 p.c or far more scalp hair coverage was obtained) was 41.5 p.c in the 12 mg two times-day by day dose group and 29.6 percent in the 8 mg twice-everyday dose team, as opposed to .8 % of patients in the placebo group, at the 24-7 days endpoint. The treatment method distinction for both dose teams of CTP-543 relative to placebo was statistically major (p<0.0001).
The safety profile seen with CTP-543 in THRIVE-AA1 was consistent with previous studies. The most common (≥5%) side effects in any dose group were headache, acne, upper respiratory infection, increased creatine kinase levels, COVID-19 infection and nasopharyngitis. Upper respiratory infections were greater in the placebo group than in either of the CTP-543 dose groups. No pulmonary embolisms or deep vein thromboses were observed in the trial. One patient treated with the 8 mg twice-daily dose and one patient treated with the 12 mg twice-daily dose developed herpes zoster (shingles). Serious adverse events were reported in nine patients, with only one patient (in the 8 mg twice-daily dose group) having events (2) that were assessed as possibly related to treatment. Four patients who reported serious adverse events were in the placebo group.
Concert expects to submit the full results from this study for future scientific publication and presentation. These data, along with data from THRIVE-AA2, a second Phase 3 clinical trial, are intended to form the basis of a New Drug Application (NDA) planned to be submitted to the U.S. Food and Drug Administration (FDA) in the first half of 2023, assuming positive results from THRIVE-AA2. Topline data from THRIVE-AA2 are expected in the third quarter of 2022.
THRIVE-AA1 (NCT04518995) is a randomized, double-blind, placebo-controlled clinical trial in 706 adult patients age 18-65 with moderate to severe alopecia areata at sites in the U.S., Canada and Europe evaluating the regrowth of scalp hair after 24 weeks of dosing using the SALT score. Patients were randomized to receive either 8 mg twice-daily or 12 mg twice-daily of CTP-543 or placebo for 24 weeks. The primary endpoint is the percentage of patients achieving a SALT score ≤ 20 at 24 weeks. All patients who completed 24 weeks of treatment in THRIVE-AA1 had the opportunity to continue in a separate extension study to evaluate long-term safety and efficacy of CTP-543.
About CTP-543 and Alopecia Areata
CTP-543 is an investigational oral selective inhibitor of Janus kinases JAK1 and JAK2. The FDA has granted CTP-543 Breakthrough Therapy designation for the treatment of adult patients with moderate to severe alopecia areata and Fast Track designation for the treatment of alopecia areata.
Alopecia areata is an autoimmune disease in which the immune system attacks hair follicles, resulting in partial or complete loss of hair on the scalp and body. Alopecia areata may affect up to approximately 1.5 million Americans at any given time.1 The scalp is the most commonly affected area, but any hair-bearing site can be affected alone or together with the scalp. Onset of the disease can occur throughout life and affects both women and men. Alopecia areata can be associated with serious psychological consequences, including anxiety and depression. There are currently no drugs approved by the FDA for the treatment of alopecia areata.
The FDA selected alopecia areata as one of eight new disease areas that it focused on under its Patient-Focused Drug Development Initiative (PFDDI) in 2016-2017. The goal of the PFDDI is to bring patient perspectives into an earlier stage of product development. Following the FDA’s Patient-Focused Drug Development meeting held in September 2017 on alopecia areata, the FDA summarized the input shared by patients and patient representatives in a Voice of the Patient report. Additional information on the PFDDI is available online.
Concert Pharmaceuticals is a clinical stage biopharmaceutical company that is developing small molecule drugs that it discovered through the application of its DCE Platform® (deuterated chemical entity platform). Selective incorporation of deuterium into known molecules has the potential, on a case-by-case basis, to provide better pharmacokinetic or metabolic properties, thereby enhancing their clinical safety, tolerability or efficacy. Concert’s lead product candidate is in late-stage development for the treatment of alopecia areata, a serious autoimmune dermatological condition. Concert is also assessing a number of earlier-stage pipeline candidates. For more information please visit www.concertpharma.com or follow us on Twitter at @ConcertPharma or on LinkedIn.
Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including, among others, statements about our expectations regarding the development of CTP-543, the potential for CTP-543 to be a best-in-class treatment for the treatment of alopecia areata, the timing of availability of clinical trial data and the timing of regulatory filings, including an NDA for CTP-543, and any other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation, timing and design of future clinical trials, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals and other factors discussed in the “Risk Factors” section of our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission and in other filings that we make with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release.
1 Benigno M. Clinical, Cosmetic and Investigational Dermatology 2020
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Justine E. Koenigsberg (Investors)
Concert Pharmaceuticals, Inc.
Kathryn Morris (media)
The Yates Network