Phase 2a Trial of CTP-543 in Alopecia Areata

Concert Pharmaceuticals Completes Enrollment in Phase 2a Trial of CTP-543 in Alopecia Areata

Concert Pharmaceuticals announce that it has completed patient enrollment of its Phase 2a trial evaluating CTP-543 for the treatment of moderate-to-severe alopecia areata.

The Company expects to report topline data from the 4 mg and 8 mg cohorts of the Phase 2a trial in the fourth quarter of 2018. Additionally, a Phase 2b clinical trial is expected to begin in 2019.

“The speed with which we enrolled the trial speaks to the significant unmet need for alopecia areata and interest in this program. We are grateful to the patients who volunteered to participate in our trial,” stated James Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals.

The Phase 2a trial is a double-blind, randomized, placebo-controlled, sequential dose trial to evaluate the safety and efficacy of CTP-543 in adult patients with moderate-to-severe alopecia areata.

Approximately 90 patients were randomized to receive one of two doses of CTP-543 (4 mg or 8 mg twice daily) or placebo.

The primary outcome measure will utilize the severity of alopecia tool (SALT) after 24 weeks of dosing. If appropriate, the protocol may be amended to explore 12 mg twice daily of CTP-543. Additional information about the trial is available on www.clinicaltrials.gov.

About CTP-543 and Alopecia Areata

CTP-543 was discovered by applying Concert’s deuterium chemistry technology to modify ruxolitinib, a drug which selectively inhibits Janus kinases 1 and 2 (JAK1 and JAK2) and is commercially available under the name Jakafi® in the United States for the treatment of certain blood disorders. Ruxolitinib has been used to treat alopecia areata in academic settings, including an investigator-sponsored clinical trial, and has been reported to promote hair growth in individuals with moderate-to-severe disease.

Deuterium modification of ruxolitinib was found to alter its human pharmacokinetics in ways which may enhance its use as a treatment for alopecia areata. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for CTP-543.

Alopecia areata is an autoimmune disease that results in partial or complete loss of hair on the scalp and body that may affect up to 650,000 Americans at any given time.

Phase 2a trial evaluating CTP-543

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 U.S. Food and Drug Administration (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 U.S. Food and Drug Administration’s (FDA) 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 address unmet patient needs. The Company’s approach starts with starts with previously studied compounds, including approved drugs, in which deuterium substitution has the potential to enhance clinical safety, tolerability or efficacy.

Concert has a broad pipeline of innovative medicines targeting autoimmune and inflammatory 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.

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