Concert Pharmaceuticals, a small biotechnology company based in Lexington, Massachusetts, plans to seek Food and Drug Administration approval for its experimental hair loss drug after reporting success in the second of two late-stage clinical trials.
As in the first study, results from the second showed Concert’s treatment helped regrow hair to a significantly greater degree than a placebo, the company said in a Monday statement. The study also met its secondary goal assessing participant satisfaction with their hair growth.
Concert expects to seek U.S. approval of the drug, dubbed CTP-543, in the first half of the year, potentially positioning the biotech to compete for market share with Eli Lilly and Incyte. In June, the two companies won FDA approval for the first systemic therapy for alopecia areata, an autoimmune condition that causes hair loss.
Both Concert’s drug as well as Lilly and Incyte’s treatment work similarly, blocking proteins known as Janus kinases that are involved in a range of immune disorders. Pfizer is also developing a so-called JAK inhibitor for alopecia areata and recently submitted for regulatory approval in the U.S., U.K. and Europe.
Other JAK inhibitors are approved to treat arthritis, eczema and ulcerative colitis. But studies have linked the drugs to a range of serious safety risks, including infection, heart attacks, strokes and blood clots. In response, the FDA has limited who can take these drugs for those conditions, a warning the regulator included when it approved Lilly and Incyte’s treatment for alopecia areata as well.
Concert noted that, in its second study, there were no reports of pulmonary blood blockages or deep vein thromboses. Yet the FDA’s caution around JAK inhibitors could still apply in the event CTP-543 is approved.
“At the end of the day, this is an FDA decision, but they are applying box warnings to the class in inflammatory conditions and we might expect similar labeling as others,” Concert CEO Roger Tung said in an emailed answer to questions from BioPharma Dive.
Four patients on Concert’s drug in the second study developed shingles, which has also been associated with JAK inhibitors. Tung said many patients are vaccinated for shingles prior to starting treatment.
While treatment comes with risks, Concert was able to show its drug could effectively regrow hair in both of its studies, which collectively enrolled about 1200 adult patients with moderate-to-severe alopecia areata.
In the second trial, participants had, on average, 12% scalp hair coverage at the start of the trial. After six months of treatment, 38% of people given a high dose of CTP-543 had 80% or greater scalp hair coverage, as did 33% of those on the low dose. Only 1% of placebo-treated participants achieved the same mark.
About half of participants on either dose of Concert’s drug reported being “satisfied” or “very satisfied” with their hair growth versus only 2% of those on placebo, the company said.
Concert plans to submit full results for publication in a scientific journal and will present them in the future. The company’s application to the FDA will include results from both Phase 3 studies.