CTP-543 Alopecia Areata Treatment Phase 2 Trial Success Announced

As part of its 2019 Annual Meeting, the American Academy of Dermatology (ADD) heard the latest clinical trial interim results update on CTP-543, a potential treatment for even the most extreme forms of Alopecia Areata.

The ADD hosted a presentation from its developers, Concert Pharmaceuticals, as part of the Late-Breaking Research Program on 2nd March 2019, in Washington DC, USA.

CTP-543 is a novel drug created from a deuterium chemistry-modified version of ruxolitinib - the JAK inhibitor and blood disorder medication, often known by its brand name, Jakafi.

CTP-543 Phase 2 trial achieves key aim

In January of this year it was announced that new cohorts were being introduced to the on-going CTP-543 Phase 2 trial in March 2019. These involved investigating higher doses of the drug, with findings due for release in December 2019.

The ADD presentation explored the interim results of the 104-patient, randomised CTP-543 Phase 2a trials regarding the 4mg and 8mg doses, compared to placebo.

These showed that treatment with an 8mg dose of
CTP-543 administered twice per day resulted in "statistically significant greater hair regrowth" for those patients, when compared to the control group receiving a placebo treatment. Changes in hair loss and hair regrowth were measured by scoring each participant's amount of shedding before and at the end of the trial, using the Severity of Alopecia Tool (SALT).

It was stated that 47% of participants treated with 8 mg of CTP-543 twice-daily reduced their SALT score by more than 50% overall, compared to only 8.6% of those using the placebo twice-per-day over the same 24 week period.

A roughly even spread of Alopecia Areata, Alopecia Totalis and Alopecia Universalis was noted among the responders in the 47% group.

The fact that, for these patients, hair regrowth did not show signs of plateauing by the end of the trial period, was also recorded.

Non-serious side effects noted

One of the concerns surrounding JAK inhibitors - the janus kinase inhibiting suite of drugs which includes ruxolitinib, on which CTP-543 is based, as previously mentioned - is safety and the potential side effects.

The presentation, given by Concert’s Chief Development Officer,
James V. Cassella, Ph.D., advised that no serious adverse events were reported during the trial.

However, other side effects from this interim analysis were disclosed. The most commonly found complaints were headache, upper respiratory tract infection, cough, acne and nausea.

In a Concert Pharmaceuticals press release regarding this presentation, Dr. Brett King, a prominent hairloss researcher and Associate Professor of Dermatology at Yale School of Medicine, advised “The data for CTP-543 shows that JAK inhibitors have the potential to be an effective treatment option for patients with this challenging disease.”

Alopecia Areata treatments in development

At present, whilst there are many studies going on into potential hair loss treatments for the various forms of autoimmune alopecia, there are three which are considered of particular significance.

These are Concert's CTP-543, Aclaris Therapeutics' ATI-502, and Pfizer's PF-06651600.

The reason these have established themselves as front-runners in the race to develop the first treatments to be authorised by both the MHRA and FDA, is due to their early recognition by the FDA.

Whilst the first two have been received Fast Track awards by the American food and drug regulatory board, the latter Pfizer trial has been given Breakthrough status. This means these three projects will receive special, expedited assistance from the FDA, in order to ensure trial findings relating to these medications are reviewed as quickly as possible.

This suggests that the research involved in the development of each novel therapy - all involving or based on JAK inhibitors - has been deemed of sufficient integrity and interest to be worth the regulator's attention.

Although Alopecia Areata which affects the scalp only will generally clear up naturally within 12 months, there are various treatment options available, which can often prove successful. A consultation with a hair loss specialist can help to determine the relevant options and the individual's suitability for them.

The more severe phenotypes - Alopecia Totalis and Alopecia Universalis - currently have fewer effective treatments and are generally less likely to see hair regrowth, however. With the possibility of effective future treatments looking to be closer than ever - with an estimated release date of 2022 for some, assuming everything goes to plan - this situation may change within the next few years.