New JAK Inhibitor CTP-543 Starts Phase 2 Alopecia Areata Trial

More information has emerged this week of a potentially important Phase 2a clinical trial which is investigating the use of a modified drug as a treatment option for people with Alopecia Areata, which leads to sudden, patchy hair loss.

The trial is being conducted by US firm Concert Pharmaceuticals, and will investigate the safety and efficacy of their version of a drug named ruxolitinib. This belongs to a range of drugs known as JAK inhibitors, and is one of several that have been in the headlines over the past few years because they have shown promise at treating people with the autoimmune disorder  Alopecia Areata though they have not yet been approved or made generally available for this specific use.

Modified analog

Concert’s version is named CTP-543, and it is a “deuterium-modified analog”, often referred to as deuterated ruxolitinib. It has an increased half-life in the bloodstream which may mean that the drug can stay active in the body longer than it otherwise would and thus reduce the number of doses needed for it to be effective.

The Phase 2a trial will last for 12 months, with the primary efficacy analysis at week 24. Concert Pharmaceuticals expects to report topline data in the first quarter of 2018. The first phase clinical trial took place in 2016 and this new phase is designed to be a full scale investigation into the efficacy, safety and tolerability of the drug in relation to autoimmune hairloss.

"Alopecia Areata is a devastating autoimmune disease that impacts half a million or more patients in the US at any given time,” says Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. “There is a significant unmet medical need with no FDA-approved therapy. We intend to be at the forefront of advancing a new oral treatment for Alopecia Areata patients and look forward to assessing CTP-543's therapeutic efficacy in the Phase 2 trial."

While his assertion that there are no FDA-approved treatments for the condition is true, Belgravia's approach to Alopecia Areata treatment involves the use of topical preparations of high strength minoxidil, one of two medically-proven treatments licensed by the MHRA and approved by the FDA for treatment of genetic hair loss. Very encouraging results have been seen on many occasions.

Mr Tung is certainly correct in his statement that, “the treatment community, and patients suffering from this disease, have been eager for new treatments for Alopecia Areata.” He says that we may be looking at the dawn of a new era “in which we are advancing treatments into clinical evaluation that target the underlying biology of the disease."

Click to View Belgravia's Alopecia Areata Treatment Success Stories (Results may vary and are not guaranteed)

Moderate to severe Alopecia Areata

The Phase 2a trial is a double-blind, randomised, placebo-controlled, parallel dose trial that has been designed to evaluate the safety and efficacy of CTP-543 in adult patients with “moderate-to-severe” Alopecia Areata though it appears that Concert Pharmaceuticals is more inclined towards people with rather severe cases of Alopecia Areata as one of the inclusion criteria is that participants must have at least 50 per cent of hair loss on their scalp.

Around a dozen clinics and research centres across the US will be involved in the trial, which is currently actively recruiting (as at May 2017).

The study is seeking 100 patients who will randomly be given one of four doses of CTP-543 (4, 8, 12 and 16 mg twice daily) or a placebo. The primary outcome measure will be the severity of alopecia tool (SALT) after 24 weeks, and the trial will include an additional 28 weeks of dosing where all patients enrolled in the study will receive CTP-543.

In Phase 1 clinical studies with CTP-543, Concert states that the pharmacokinetic profile showed increased exposure with increasing doses of CTP-543. “CTP-543 was well-tolerated across all dose groups and there were no serious adverse events reported,” it states. “The most commonly reported adverse event was headache.”

Unlike the front-running JAK inhibitor treatments for Alopecia Areata which are also being designed to treat severe autoimmune hair loss from AA's more extreme sister conditions which both cause total baldness of the head: alopecia totalis and alopecia universalis, CTP-543 is currently only treating patchy hair loss from AA. There are currently no cures or treatments for these conditions, though for people experiencing alopecia areata in its moderate form - sudden hair loss resulting in bald spots or rounded patches to the scalp - there are alopecia areata treatment courses available now.