Concert Pharmaceuticals unveils CTP-543 to treat alopecia areata

Alopecia areata is an autoimmune disease characterized by patchy or widespread hair loss, for which there is significant unmet medical need and currently no FDA-approved treatment.

CTP-543 is a deuterium-modified analog of ruxolitinib, which selectively inhibits Janus kinases 1 and 2, known as JAK1 and JAK2. Ruxolitinib is commercially available under the name Jakafi® in the United States for the treatment of myelofibrosis and polycythemia vera. Concert expects to initiate Phase 1 clinical evaluation of CTP-543 in the second quarter of 2016 with efficacy studies commencing in 2017.

Ruxolitinib has been used to treat alopecia areata in academic settings, including an investigator-sponsored clinical trial, and has been shown to promote hair growth in individuals with moderate to severe disease.

Preclinical studies conducted by the Company demonstrated that CTP-543 retains ruxolitinib’s selective JAK 1/2 inhibition profile and shows improved metabolic stability.

"We are very excited about the potential of CTP-543 to greatly enhance patient care for individuals with alopecia areata. CTP-543 represents an opportunity to address an important disease where new and effective treatment options are needed," said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals.

"Based on demonstrated clinical activity of ruxolitinib in alopecia areata, CTP-543 has the potential to become an important first-in-class oral medicine to treat a devastating condition affecting millions of people in the U.S."

Recently, the U.S. Patent and Trademark Office issued U.S. Patent No. 9,249,149 that claims CTP-543 as a novel composition of matter.

"CTP-543 is a prime example of the application of our deuterium platform to create proprietary new medicines which enhance and extend the utility of known molecules into new therapeutic areas. We believe we have developed a valuable, potentially first-in-class new drug candidate for an important unmet medical need and look forward to advancing CTP-543 into clinical evaluation shortly," said Dr. Tung.

About Alopecia Areata

Alopecia areata is an autoimmune disease that results in partial or complete loss of hair on the scalp and body. 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.

According to MD Anderson, nearly 2% of people in the United States, including both adults and children, suffer from alopecia areata. There are currently no drugs approved by the U.S. Food and Drug Administration (FDA) for the treatment of alopecia areata.

In 2015, the FDA selected alopecia areata as one of eight new disease areas that it will focus on under its Patient-Focused Drug Development Initiative (PFDDI) meeting during fiscal year 2016-2017. The goal of the PFDDI is to bring patient perspectives into an earlier stage of product development.