Concert Pharmaceuticals Reports Positive Topline Results for Second CTP‑543 Phase 3 Clinical Trial in Alopecia Areata

LEXINGTON, Mass.–(BUSINESS WIRE)–Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) immediately introduced optimistic topline outcomes from its second Section 3 medical trial, THRIVE-AA2, evaluating its oral investigational drugs CTP-543 in grownup sufferers with average to extreme alopecia areata, an autoimmune dysfunction that leads to patchy or full scalp hair loss. The first efficacy endpoint for THRIVE-AA2 was the share of sufferers reaching an absolute Severity of Alopecia Software (SALT) rating of 20 or much less at Week 24 of therapy, which was met with statistical significance in each the 8 mg twice-daily and 12 mg twice-daily dose teams relative to placebo. Remedy with CTP-543 was usually effectively tolerated.

“It’s a new period of innovation for creating therapy choices for sufferers with alopecia areata, lots of whom usually undergo bodily and mentally with this difficult autoimmune illness,” mentioned Maryanne Senna, MD, Director of the Lahey Hair Loss Clinic of Excellence and medical investigator of THRIVE-AA2. “These outcomes are very promising and counsel that CTP-543 has the potential to deal with vital wants for folks dwelling with alopecia areata.”

“With the profitable completion of our two THRIVE-AA Section 3 trials, Live performance is now transferring quickly to arrange our NDA for submission to the U.S. FDA within the first half of 2023. We’re additionally persevering with to advance our business planning as we place CTP-543 to probably enter {the marketplace} for the massive underserved inhabitants of sufferers with alopecia areata,” mentioned James V. Cassella, Ph.D., Chief Growth Officer of Live performance Prescribed drugs. “Primarily based on the outcomes from our Section 3 program, we imagine CTP-543, if authorized, may very well be a best-in-class therapy choice for alopecia areata and have a definite therapeutic profile with options which are vital to sufferers, together with the share of sufferers with clinically-meaningful hair regrowth, affected person satisfaction ranges, and hair regrowth charges.”

Dr. Cassella added, “The Live performance staff continues to be enthusiastic and dedicated to working to convey CTP-543 to market as quickly as attainable as a possible new therapy choice for sufferers dwelling with alopecia areata.”

Sufferers enrolled in THRIVE-AA2 had been required to have no less than 50 % scalp hair loss on account of alopecia areata, as measured by SALT. A SALT rating of 100 represents whole scalp hair loss, whereas a rating of 0 represents no scalp hair loss. The typical baseline SALT rating throughout all sufferers was roughly 87.9 (comparable to roughly 12% common scalp hair protection).

A statistically vital proportion of sufferers handled with both 8 mg twice-daily or 12 mg twice-daily of CTP-543 skilled better scalp regrowth in comparison with placebo. The proportion of sufferers reaching a SALT rating of 20 or much less (which means 20 % or much less scalp hair loss) was 38.3 % within the 12 mg twice-daily dose group and 33.0 % within the 8 mg twice-daily dose group, in comparison with 0.8 % of sufferers within the placebo group, on the 24-week endpoint. The therapy distinction for each dose teams of CTP-543 relative to placebo was statistically vital (p<0.0001).

The important thing secondary endpoints had been the share of responders on a Satisfaction of Hair Affected person Reported End result (SPRO) scale at Week 24 and the share of sufferers reaching absolute SALT scores of 20 or much less at every of Weeks 20, 16, 12 and eight. 47% of sufferers within the 8 mg twice-daily group and 52% of sufferers within the 12 mg twice-daily group reported being “glad” or “very glad,” as in comparison with 2% of sufferers within the placebo group. The therapy distinction for each teams relative to placebo was statistically vital. SALT scores of 20 or much less at Weeks 20, 16 and 12 had been statistically vital in each dose teams.

The security profile seen with CTP-543 in THRIVE-AA2 was in line with earlier research. The commonest (≥5%) unwanted side effects in any dose group had been COVID-19 an infection, nasopharyngitis, elevated creatine kinase ranges, zits and headache. No pulmonary embolisms or deep vein thromboses had been noticed within the trial. Two sufferers handled with the 8 mg twice-daily dose and two sufferers handled with the 12 mg twice-daily dose developed herpes zoster (shingles). 5 severe antagonistic occasions had been reported in 5 sufferers, with just one within the 8 mg twice-daily dose group that was assessed as probably associated to therapy.

Live performance expects to submit the complete outcomes from this research for future scientific publication and presentation. These information, together with information from the primary Section 3 medical trial, THRIVE-AA1, are supposed to type the idea of a New Drug Software (NDA) deliberate to be submitted to the U.S. Meals and Drug Administration (FDA) within the first half of 2023.

About THRIVE-AA2

THRIVE-AA2 (NCT04797650) is a randomized, double-blind, placebo-controlled medical trial in 517 grownup sufferers age 18-65 with average to extreme alopecia areata at websites within the U.S., Canada and Europe evaluating the regrowth of scalp hair after 24 weeks of dosing utilizing the SALT rating. Sufferers had been randomized to obtain both 8 mg twice-daily or 12 mg twice-daily of CTP-543 or placebo for twenty-four weeks. The first endpoint is the share of sufferers reaching a SALT rating ≤ 20 at 24 weeks. All sufferers who accomplished 24 weeks of therapy in THRIVE-AA2 had the chance to proceed in a separate extension research to judge long-term security and efficacy of CTP-543. The THRIVE-AA pivotal program, consisting of two Section 3 medical trials that enrolled over 1,200 sufferers, is likely one of the most complete improvement applications in alopecia areata.

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 Remedy designation for the therapy of grownup sufferers with average to extreme alopecia areata and Quick Observe designation for the therapy of alopecia areata.

Alopecia areata is an autoimmune illness during which the immune system assaults hair follicles, leading to partial or full lack of hair on the scalp and physique. Alopecia areata could have an effect on as much as roughly 1.5 million Individuals at any given time.¹ The scalp is essentially the most generally affected space, however any hair-bearing web site may be affected alone or along with the scalp. Onset of the illness can happen all through life and impacts each men and women. Alopecia areata may be related to severe psychological penalties, together with anxiousness and despair. There are at the moment restricted therapy choices accessible for alopecia areata.

The FDA chosen alopecia areata as one in all eight new illness areas that it targeted on below its Affected person-Centered Drug Growth Initiative (PFDDI) in 2016-2017. The aim of the PFDDI is to convey affected person views into an earlier stage of product improvement. Following the FDA’s Affected person-Centered Drug Growth assembly held in September 2017 on alopecia areata, the FDA summarized the enter shared by sufferers and affected person representatives in a Voice of the Patient report. Extra info on the PFDDI is out there online.

About Live performance

Concert Pharmaceuticals is a late-stage medical biopharmaceutical firm that’s creating small molecule medicine that it found by way of the applying of its DCE Platform^® (deuterated chemical entity platform). Selective incorporation of deuterium into recognized molecules has the potential, on a case-by-case foundation, to supply higher pharmacokinetic or metabolic properties, thereby enhancing their medical security, tolerability or efficacy. Live performance’s lead product candidate CTP-543 is being developed for the therapy of alopecia areata, a severe autoimmune dermatological situation. Live performance can be assessing various earlier-stage pipeline candidates. For extra info please go to www.concertpharma.com or observe us on Twitter at @ConcertPharma or on LinkedIn.

Cautionary Be aware on Ahead Trying Statements

Any statements on this press launch about our future expectations, plans and prospects, together with, amongst others, statements about our expectations relating to the event of CTP-543, the potential for CTP-543 to be a best-in-class therapy for the therapy of alopecia areata and the deliberate timing for submitting an NDA for CTP-543, and every other statements containing the phrases “anticipate,” “imagine,” “proceed,” “might,” “estimate,” “anticipate,” “intend,” “could,” “plan,” “potential,” “predict,” “challenge,” “ought to,” “goal,” “will,” “would” and related expressions, represent forward-looking statements throughout the which means of The Non-public Securities Litigation Reform Act of 1995. Precise outcomes could differ materially from these indicated by such forward-looking statements because of numerous vital components, together with: the uncertainties inherent within the initiation, timing and design of future medical trials, the provision and timing of information from ongoing and future medical trials and the outcomes of such trials, whether or not preliminary outcomes, together with security profiles, from a medical trial will probably be predictive of the ultimate outcomes of that trial or whether or not outcomes of early medical trials will probably be indicative of the outcomes of later medical trials, expectations for the timing of the submission of an NDA, the provision of regulatory approvals and different components mentioned within the “Threat Elements” part of our most up-to-date Quarterly Report on Type 10-Q filed with the Securities and Alternate Fee and in different filings that we make with the Securities and Alternate Fee. As well as, any forward-looking statements included on this press launch symbolize our views solely as of the date of this launch and shouldn’t be relied upon as representing our views as of any subsequent date. We particularly disclaim any obligation to replace any forward-looking statements included on this press launch.

¹ Benigno M. Clinical, Cosmetic and Investigational Dermatology 2020