Concert Pharmaceuticals Report – GuruFocus.com

Concert+Pharmaceuticals%2C+Inc. (NASDAQ: CNCE) right now introduced optimistic topline outcomes from its second Part 3 scientific trial, THRIVE-AA2, evaluating its oral investigational drugs CTP-543 in grownup sufferers with reasonable 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 attaining an absolute Severity of Alopecia Instrument (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. Therapy with CTP-543 was typically effectively tolerated.

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

“With the profitable completion of our two THRIVE-AA Part 3 trials, Live performance is now shifting quickly to organize 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 doubtlessly enter {the marketplace} for the massive underserved inhabitants of sufferers with alopecia areata,” mentioned James V. Cassella, Ph.D., Chief Improvement Officer of Live performance Prescription drugs. “Based mostly on the outcomes from our Part 3 program, we imagine CTP-543, if authorized, might be a best-in-class therapy choice for alopecia areata and have a definite therapeutic profile with options which can be 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 deliver CTP-543 to market as quickly as doable as a possible new therapy choice for sufferers dwelling with alopecia areata.”

Sufferers enrolled in THRIVE-AA2 have been required to have at the very least 50 p.c scalp hair loss on account of alopecia areata, as measured by SALT. A SALT rating of 100 represents complete scalp hair loss, whereas a rating of 0 represents no scalp hair loss. The common baseline SALT rating throughout all sufferers was roughly 87.9 (equivalent 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 larger scalp regrowth in comparison with placebo. The proportion of sufferers attaining a SALT rating of 20 or much less (that means 20 p.c or much less scalp hair loss) was 38.3 p.c within the 12 mg twice-daily dose group and 33.0 p.c within the 8 mg twice-daily dose group, in comparison with 0.8 p.c 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 have been the share of responders on a Satisfaction of Hair Affected person Reported Consequence (SPRO) scale at Week 24 and the share of sufferers attaining 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 have been statistically vital in each dose teams.

The protection profile seen with CTP-543 in THRIVE-AA2 was according to earlier research. The commonest (≥5%) unwanted side effects in any dose group have been COVID-19 an infection, nasopharyngitis, elevated creatine kinase ranges, pimples and headache. No pulmonary embolisms or deep vein thromboses have 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 critical opposed occasions have 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 total outcomes from this examine for future scientific publication and presentation. These knowledge, together with knowledge from the primary Part 3 scientific trial, THRIVE-AA1, are meant to kind the premise of a New Drug Utility (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 scientific trial in 517 grownup sufferers age 18-65 with reasonable 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 have 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 attaining 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 examine to judge long-term security and efficacy of CTP-543. The THRIVE-AA pivotal program, consisting of two Part 3 scientific trials that enrolled over 1,200 sufferers, is likely one of the most complete growth 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 reasonable to extreme alopecia areata and Quick Observe designation for the therapy of alopecia areata.

Alopecia areata is an autoimmune illness wherein 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 website may be affected alone or along with the scalp. Onset of the illness can happen all through life and impacts each ladies and men. Alopecia areata may be related to critical psychological penalties, together with nervousness and melancholy. There are presently restricted therapy choices obtainable for alopecia areata.

The FDA chosen alopecia areata as considered one of eight new illness areas that it centered on underneath its Affected person-Centered Drug Improvement Initiative (PFDDI) in 2016-2017. The objective of the PFDDI is to deliver affected person views into an earlier stage of product growth. Following the FDA’s Affected person-Centered Drug Improvement 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 obtainable online.

About Live performance

Concert+Pharmaceuticals is a late-stage scientific biopharmaceutical firm that’s growing small molecule medicine that it found by means of the applying of its DCE+Platform%3Csup%3E%26reg%3B%3C%2Fsup%3E (deuterated chemical entity platform). Selective incorporation of deuterium into recognized molecules has the potential, on a case-by-case foundation, to offer higher pharmacokinetic or metabolic properties, thereby enhancing their scientific security, tolerability or efficacy. Live performance’s lead product+candidate CTP-543 is being developed for the therapy of alopecia areata, a critical autoimmune dermatological situation. Live performance can also be assessing various earlier-stage pipeline candidates. For extra info please go to www.concertpharma.com or observe us on Twitter at %40ConcertPharma or on LinkedIn.

Cautionary Be aware on Ahead Wanting Statements

Any statements on this press launch about our future expectations, plans and prospects, together with, amongst others, statements about our expectations concerning 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 some other statements containing the phrases “anticipate,” “imagine,” “proceed,” “may,” “estimate,” “anticipate,” “intend,” “could,” “plan,” “potential,” “predict,” “challenge,” “ought to,” “goal,” “will,” “would” and comparable expressions, represent forward-looking statements throughout the that means of The Personal Securities Litigation Reform Act of 1995. Precise outcomes could differ materially from these indicated by such forward-looking statements because of varied vital components, together with: the uncertainties inherent within the initiation, timing and design of future scientific trials, the provision and timing of knowledge from ongoing and future scientific trials and the outcomes of such trials, whether or not preliminary outcomes, together with security profiles, from a scientific trial will likely be predictive of the ultimate outcomes of that trial or whether or not outcomes of early scientific trials will likely be indicative of the outcomes of later scientific trials, expectations for the timing of the submission of an NDA, the provision of regulatory approvals and different components mentioned within the “Threat Components” part of our most up-to-date Quarterly Report on Type 10-Q filed with the Securities and Change Fee and in different filings that we make with the Securities and Change Fee. As well as, any forward-looking statements included on this press launch signify 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%2C+Cosmetic+and+Investigational+Dermatology 2020

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