Press Release Details

Sep 17, 2019

Concert Pharmaceuticals Completes Enrollment in Second Open Label Trial of CTP-543 to Evaluate Once-Daily vs. Twice-Daily Dosing in Patients with Alopecia Areata

LEXINGTON, Mass.--(BUSINESS WIRE)--Sep. 17, 2019-- Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced that it has completed patient enrollment of an open label clinical trial to evaluate once-daily 24 mg compared to twice-daily 12 mg oral dosing of CTP-543 in patients with alopecia areata. Topline data from this dose regimen trial is expected in the first half of 2020.

“We are extremely grateful to the patients who volunteer to participate in our clinical trials and support our mission to develop a new treatment for alopecia areata. These patients, along with the investigators and their teams who work together with us, are essential in the development of new treatment options alopecia areata,” said James V. Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals.

The open label trial (NCT03941548) is a randomized, multi-center study in the United States and Canada to evaluate the efficacy and tolerability of once-daily versus twice-daily dosing of CTP-543 in 66 adult patients with moderate-to-severe alopecia areata. Patients in the trial were randomized to receive either 12 mg twice-daily or 24 mg once-daily of CTP-543 over a 24-week treatment period. The primary outcome measure is the relative change in Severity of Alopecia Tool (SALT) score at Week 24 from baseline. All patients who complete 24 weeks of treatment are eligible to enroll into a long term open label extension study of CTP-543.

Previously, the Company announced it has fully enrolled the first open label dose regimen trial evaluating 8 mg twice-daily compared to 16 mg once-daily of CTP-543 in patients with alopecia areata, with topline data from this trial expected in the fourth quarter of 2019. Available data from the dose regimen trials along with Concert’s recently completed Phase 2 study of CTP-543 are intended to support an end of Phase 2 discussion with the U.S. Food and Drug Administration (FDA) in the first quarter of 2020.

Additional information about CTP-543 clinical trials are available on www.clinicaltrials.gov.

About CTP-543 Phase 2 Results

In September 2019, the Company reported topline results from its CTP-543 dose-ranging Phase 2 trial in patients with moderate-to-severe alopecia areata. At 24 weeks, patients treated with either 8 mg twice-daily or 12 mg twice-daily of CTP-543 met the primary efficacy endpoint with statistically significant differences (p <0.001) relative to placebo in the percentage of patients achieving a ≥ 50% relative change from baseline at 24 weeks using the Severity of Alopecia Tool (SALT). Both of these active dose groups were also significantly different from placebo in the number of patients achieving ≥ 75% and ≥ 90% relative change in SALT at Week 24 from baseline. At Week 24, patients treated with 8 mg twice-daily and 12 mg twice-daily CTP-543 also rated significantly greater improvement in their alopecia areata on the Patient Global Impression of Improvement Scale than did patients who received placebo. Treatment with CTP-543 was generally well tolerated. The most common (≥10%) side effects in the 12 mg CTP-543 dose group were headache, nasopharyngitis, upper respiratory tract infection, and acne. One serious adverse event of facial cellulitis was reported; however, after a brief dose interruption, treatment was continued and this patient completed the trial. Additional information about the CTP-543 Phase 2 results is available in the Scientific Presentations section of Company’s website.

About CTP-543 and Alopecia Areata

CTP-543 is an oral selective inhibitor of Janus kinases JAK1 and JAK2 for the potential treatment of alopecia areata. The FDA has granted Fast Track designation for CTP-543 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 650,000 Americans at any given time1. 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.

About Concert

Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform® (deuterated chemical entity platform) to create novel medicines designed to treat serious diseases and address unmet patient needs. The Company’s approach starts with previously studied compounds, including approved drugs, in which deuterium substitution has the potential to enhance clinical safety, tolerability or efficacy. Concert’s pipeline of innovative medicines targets autoimmune diseases and central nervous systems (CNS) disorders. 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 statements about our expectations on the clinical development of CTP-543, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "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 of future clinical 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 Fricke M. Epidemiology and Burden of alopecia areata: a systemic review. Clinical, Cosmetic and Investigational Dermatology. 2015; Vol 8. 397-403.

Concert Pharmaceuticals Inc., the CoNCERT Pharmaceuticals Inc. logo and DCE Platform are registered trademarks of Concert Pharmaceuticals, Inc.

Source: Concert Pharmaceuticals, Inc.

Justine E. Koenigsberg (Investors)
Concert Pharmaceuticals, Inc.
(781) 674-5284
ir@concertpharma.com

Kathryn Morris (media)
The Yates Network
(914) 204-6412
kathryn@theyatesnetwork.com

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