Alopecia Areata Treatment Trial On Hold Due to FDA Concerns

A much-anticipated clinical trial into the use of a potent new drug on people whose hair loss is caused by Alopecia Areata has been temporarily put on hold by America’s Food and Drug Administration.

Massachusetts-based Concert Pharmaceuticals was in the process of recruiting 100 people with the bald-patch-causing autoimmune disorder for a Phase 2a double-blind trial designed to see if its modified version of an existing drug named ruxolitinib was both safe and effective.

Concert's version, which it has called CTP-543 and which is designed to stay in the bloodstream for longer than the drug on which it is modelled, was to be tested over a 12-month period. But US Government intervention on May 17 has now put a dent in the company's schedule.

Shares in the company fell by around 10 per cent on the day of the announcement.

Review of studies is needed

According to Concert’s website, the FDA had previously informed the company that it could initiate the Phase 2a clinical trial. However, before volunteers were given their first doses of the drug the FDA told the Company that a review of certain recently completed non-clinical toxicology studies is required before proceeding.

Apparently the FDA did not cite a safety concern, but they intend to review these additional non-clinical data as support for the one-year dosing duration as planned in the trial.

Concert states that it is “working diligently with the FDA to resume enrolment in the Phase 2a trial as quickly as possible.”

The US government body’s decision is sure to have been a blow to Concert Pharmaceuticals, the company clearly having hoped to see exploitable results from its much-anticipated study, the roots of which can be found in a 2016 phase 1 clinical trial. Around a dozen clinics had been lined up to administer the drugs and monitor patients and these are presumably now on stand-by.

The drug that the Concert treatment is based on ruxolitinib is part of a group of drugs known as JAK inhibitors, which were originally developed to treat everything from rheumatoid arthritis to certain cancers. Their ability to regrow hair on people who had lost it to Alopecia Areata (and also more severe forms of the disorder, Alopecia Totalis and Alopecia Universalis) were discovered almost by chance. Today, several different companies and research bodies are involved in trials to ascertain how effective these may be in treating all kinds of Alopecia Areata and, in at least one case, their scope extends to genetic hair loss, too.

Concert’s unique take on proceedings is to use a “deuterium-modified analog” of the drug which may result in less frequent doses.

New era for treatment?

The company’s President and CEO, Roger Tung, recently said that the world could be about to enter a new era in which novel treatments for Alopecia Areata that target the underlying biology of what is widely-accepted to be a particularly complex condition enter the market.

Indeed, there are currently no FDA-approved treatments for the condition although Alopecia Areata treatment at Belgravia involves the use of topical preparations of high strength minoxidil, applied directly to the bald patches. Minoxidil is one of two medically-proven treatments licensed by the MHRA and approved by the FDA for treatment of pattern hair loss in men and women. On many occasions, however, encouraging results - and even full regrowth - have been seen when using recommended formulations as part of a custom alopecia areata treatment course.