Concert gets BTD for alopecia drug, setting up a late-stage showdown with giant rival Pfizer
Concert Pharmaceuticals’ path to developing a drug that treats alopecia areata has been bumpy, but the pharma company scored a win Wednesday.
The FDA granted Concert a Breakthrough Therapy Designation (BTD) for its oral Janus kinase inhibitor, named CTP-543, paving the way for a Phase III study of the drug to begin in the fourth quarter of 2020. The news follows positive Phase II results from last September, which saw the drug meet its primary endpoint in both 8 mg and 12 mg twice-daily doses.
The news spurred a 14% spike in the biotech’s share $CNCE price.
There are currently no drugs on the market to treat alopecia areata and Concert, whose drug blocks JAK1 and JAK2, has been competing with Pfizer to see who can reach shelves first. CTP-543 is going up against PF-06651600, which inhibits JAK3, and PF-06700841, which targets TYK2 and JAK1.
Though the FDA has officially endorsed CTP-543’s potential with the BTD, the drug’s success is far from guaranteed.
JAK inhibitors, which can also be used to treat rheumatoid arthritis and ulcerative colitis, have come under heavy scrutiny in recent years. Both the FDA and EMA have slapped warnings on and restricted larger doses of such inhibitors made by Pfizer (Xeljanz) and AbbVie (Rinvoq) after observing higher rates of blood clots and death. Pfizer ended up dropping its higher-dose treatment of Xeljanz altogether, and Eli Lilly avoided selling high doses of its Olumiant drug as it became available.
Concert itself ran into some trouble a few years ago when the FDA placed a clinical hold on the Phase IIa study for CTP-543, even though the agency did not mention safety concerns in its notice. After quickly fixing the issue, the Phase IIa study was completed in late 2018, but showed Concert’s inhibitors, despite hitting their main goals, did not stimulate hair growth as quickly as Pfizer’s in data released in late 2018. The final data, released the next year, showed the drug to be more competitive and suggested it might even outperform Pfizer.
Pfizer is also researching JAK inhibitors in teens and young adults, testing their efficacy in treatments for atopic dermatitis. Although these studies hit their primary goals, they too reported higher rates of side effects than the placebo.
Alopecia areata is an autoimmune disease that affects around 700,000 people in the United States. The disease tricks the immune system into attacking a body’s own hair follicles, resulting in partial or complete loss of hair. Though the scalp is the most commonly affected area, hair loss can occur on any part of the body.