Therapy Areas: Autoimmune
Concert Pharmaceuticals Presents Interim CTP-543 Phase 2 Data in Alopecia Areata
4 March 2019 - - US-based Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) presented interim results from its Phase 2 clinical trial evaluating its product candidate CTP-543 in patients with moderate-to-severe alopecia areata in an oral presentation during the late-breaking clinical trials session at the American Academy of Dermatology over the weekend, the company said.

The interim results from this dose-ranging trial showed that treatment with CTP-543 administered at an 8 mg twice-daily dose for 24 weeks met the primary endpoint with a statistically significant greater hair regrowth responder rate compared to placebo.

The primary endpoint measures the proportion of patients who are responders to treatment, defined as a ≥ 50% relative reduction in their overall Severity of Alopecia Tool score from baseline.

Patients with moderate-to-severe alopecia areata enrolled in the first two cohorts received 4 mg or 8 mg of CTP-543 twice-daily or placebo twice-daily for 24 weeks. The final cohort evaluating 12 mg of CTP-543 or placebo twice-daily is ongoing.

The trial randomised 104 patients in the 4 mg and 8 mg cohorts.

The oral presentation will highlight the interim results for the 8 mg twice-daily dosing cohort of CTP-543, which achieved the Phase 2 trial's primary endpoint, in patients with moderate-to-severe alopecia areata after 24 weeks of dosing.

47% of patients treated with 8 mg of CTP-543 twice-daily achieved a ≥ 50% relative reduction in their overall SALT, score from baseline, which was a significant improvement compared to 8.6% for placebo (p < 0.001).

The responders in the 8 mg twice-daily dose group were evenly distributed among patients with patchy alopecia areata and the more severe forms, alopecia totalis and alopecia universalis.

The percentage of patients achieving the primary endpoint continued to increase up to Week 24, and regrowth of hair did not appear to plateau at Week 24.

For the 4 mg cohort, 21% of patients achieved a ≥ 50% relative reduction in their overall SALT score from baseline, however these differences were not significantly different from placebo.

The most common side effects in the interim analysis were headache, upper respiratory tract infection, cough, acne and nausea. No serious adverse events were reported.

Details from the oral presentation, entitled "JAK Inhibitor CTP-543 Achieves Primary Endpoint in Phase 2a Trial in Alopecia Areata," will be posted in the Scientific Presentations section of Concert's website at the start of the oral presentation at the AAD on Saturday, March 2, 2019 at 1:20 p.m. ET.

The Phase 2 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. The primary outcome measure will utilise the Severity of Alopecia Tool after 24 weeks of dosing.

Patients were sequentially randomized to receive one of three doses of CTP-543 (4 mg, 8 mg, or 12 mg) or placebo twice-daily.

The final cohort evaluating a 12 mg twice-daily dose of CTP-543 compared to placebo is fully enrolled and topline data for the overall study, including the 12mg cohort, is expected in the third quarter of 2019.

CTP-543 was discovered by applying Concert's deuterium chemistry technology to modify ruxolitinib, a drug which 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.

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 US Food and Drug Administration 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.

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 with the majority of patients initially having symptoms by age 40.

It is believed to equally affect 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.

Following the FDA's Patient-Focused Drug Development meeting held in September 2017 on alopecia areata, the FDA summarised the input shared by patients and patient representatives in a Voice of the Patient report.

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 disorders.
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