An oral treatment for all forms of the autoimmune disorder Alopecia Areata which causes varying amounts of hair loss, from bald spots on the scalp to complete baldness from head to toe, has reported great progress.
CTP-543, an investigational selective JAK inhibitor which specifically targets Janus kinases JAK 1 and JAK 2, is being developed by Concert Pharmaceuticals and has been granted Fast Track designation by the FDA.
On Tuesday 3rd September 2019 the topline results were released from the latest CTP-543 Phase 2 trial, during which higher doses were explored; the results have been welcomed as “highly encouraging” by hair loss specialists, including Dr. Brett King, Associate Professor of Dermatology at the Yale School of Medicine in America, who is known for his work in the area of JAK inhibition.
Robust results for both safety and efficacy
This time 149 patients with a confirmed diagnosis of any form of Alopecia Areata, from the scalp-only phenotype to the more extensive Alopecia Totalis and Alopecia Universalis, which also involve the loss of facial and body hair, were given either 8mg of CTP-543 twice a day, 12mg of CTP-543 twice per day, or a placebo twice each day.
Results show “statistically significant differences” for both the 8mg and 12mg doses when compared to the placebo results, according to a Concert press release and data – as well as hair regrowth progress photos – which can be seen by clicking on the image below.
The average amount of hair loss in each patient at the start – their baseline measurement – was 88 per cent (where 100 per cent is total baldness).
The groups where 8 mg or 12 mg were administered twice each day saw far more patients achieve hair regrowth of over 75% and over 90% than the placebo group, at the 24 week mark. This was measured by improvements seen in their Severity of Alopecia Tool (SALT) score but was also reflected in patients own reporting via the Patient Global Impression of Improvement Scale data.
This information comes from the trial’s topline results, with complete study results expected to be presented in the near future.
In terms of safety and tolerability, the press release advises that both doses were generally well-tolerated. Of the five participants who discontinued the trial, three were taking the placebo and two were taking 8mg doses – none were taking the 12mg twice-per-day dosage.
Concert reports on adverse events during the trial, as follows:
“The most common (≥10%) side effects in the 12 mg CTP-543 dose group were headache, nasopharyngitis, upper respiratory tract infection, and acne. One serious adverse event of facial cellulitis was reported as possibly related to treatment; however, after a brief interruption, treatment was continued and this patient completed the trial. No thromboembolic events were reported during the trial.”
Preparing for Phase 3 trials with FDA guidance
James V. Cassella, Ph.D., Concert’s Chief Development Officer said of the news, “We are very pleased with these clinical results and continue to believe CTP-543 has potential to be a best-in-class treatment for alopecia areata… We are highly focused on the need for an effective and safe treatment for alopecia areata, and we plan to advance CTP-543 into Phase 3 testing next year.”
Indeed the next steps include preparing to talk to the FDA about CTP-543 Phase 2 end-point data and what the regulatory body wants to see from Phase 3 trials so these can be properly structured.
Concert stated they may consider looking at once a day dosing versus twice a day doses as having a strong efficacy and safety profile is the company’s main concern, which would also be likely to lead to a regulatory advantage.
In a follow-up phone call to discuss these findings, Concert’s representatives advised they are not planning on testing higher doses, though did note that many of the trial participants, especially from the 12mg CTP-543 cohort, have opted to continue treatment, and monitoring.
Therefore, they believe it is possible that this longer duration of treatment may increase the response rate of the current dosages – 8 and 12 mg.
In terms of Pfizer cross-trial comparisons, given Pfizer is the other front-runner when it comes to using JAK inhibitors to treat Alopecia Areata since Aclaris dropped out of the race – Concert was confident their work stacked up well, stating:
“We believe trial data is as strong as Pfizer’s. The 75 per cent change and 90 per cent change from baseline – we offer real benefit and have robust results compared to the data Pfizer has released.”
Whilst Alopecia Areata treatment can currently be beneficial for adults with the scalp-only form, there are no MHRA-licensed nor FDA-approved hairloss solutions for any of its iterations, and the more extensive they are, the lower the success rates tend to be. This is why the FDA in particular is lending its weight to helping bring about a safe and effective treatment to address this unmet need.
The Belgravia Centre is an organisation specialising in hair growth and hair loss prevention with two clinics and in-house pharmacies in Central London, UK. If you are worried about hair loss you can arrange a free consultation with a hair loss expert or complete our Online Consultation Form from anywhere in the world. View our Hair Loss Success Stories, which includes the world’s largest gallery of hair growth comparison photos and demonstrates the levels of success that so many of Belgravia’s patients achieve. You can also phone 020 7730 6666 any time to arrange a free consultation.