- Shares in Concert Pharmaceuticals rose higher in value Monday after the Lexington, Massachusetts-based biotech company reported positive results from a late-stage clinical trial testing its most advanced drug, a treatment for the hair loss condition alopecia areata.
- Significantly more trial participants who received Concert's drug, called CTP-543, experienced greater hair regrowth following treatment than did those who were given a placebo in the study. The effect held up across both a lower and a higher dose of CTP-543, which also met the trial's secondary goals.
- Concert's trial, which enrolled about 700 adults with moderate-to-severe alopecia areata, is one of two Phase 3 studies testing the drug. Results from the second are expected in the third quarter, and Concert anticipates the combined data will form the basis of an approval application to the Food and Drug Administration.
Concert follows Pfizer and Eli Lilly in reading out positive late-stage trial results for a type of drug known as a JAK inhibitor in alopecia areata.
Used to treat arthritis and other autoimmune conditions, JAK inhibitors have also shown potential to improve scalp hair regrowth. Lilly, which reported data from two Phase 3 trials last year, is currently awaiting an approval decision from the Food and Drug Administration. (Last week, European regulators recommend authorization in the EU.) Pfizer, meanwhile, plans to submit its JAK drug ritlecitinib for approval in alopecia areata by June.
Concert could follow with its drug, assuming positive results in the second study. Data from the first, which was released via press release from the company, appeared strong, showing CTP-543 outperformed placebo. Participants in the study had, on average, less than 15% scalp hair coverage at the start of the study. After 24 weeks, 42% of people treated with a high dose of Concert's drug achieved 80% or greater hair coverage, compared just under 1% of those given placebo.
The proportion of patients on the lower dose reaching the 80% benchmark was also greater than in the placebo group, with nearly 30% meeting that goal.
Concert said no cases of blood clots in the lungs or deep vein thrombosis were observed, both of which are side effects that have been associated with JAK inhibitors used to treat other types of autoimmune disease. Trial investigators reported serious reactions in nine patients, four of whom were in the placebo group. Only in one patient on Concert's drug were the reactions judged to be possible related to treatment.
Concert will submit the study's full results for publication in a medical journal. If Concert's second study succeeds as well, the company plans to submit for FDA approval in the first half of 2023.