One trial down, one to go as Concert Pharmaceuticals tunes up PhIII win for alopecia drug
Just two months ago, Concert Pharmaceuticals offloaded most of its CNS pipeline to tiny Terran Biosciences as a make-or-break data readout was well on its way.
Now, with today’s readout from Concert, it looks like the biotech’s sole candidate pulled it off — and that the biotech could very well be before the FDA next year.
The Lexington, MA biotech announced topline data Monday morning that indicated the primary endpoint, plus all “key secondary endpoints,” were met. The topline data came from THRIVE-AA1, a Phase III trial with more than 700 participants enrolled and one of Concert’s two Phase III trials investigating drug candidate CTP-543.
The drug, a Janus kinase inhibitor that blocks JAK1 and JAKA2, is being investigated in patients with moderate to severe forms of autoimmune disorder alopecia areata, which results in patchy or complete scalp hair loss.
Investors were happy with the news, with $CNCE shooting up over 11% Monday morning.
The primary endpoint, according to Concert, was looking at the percentage of patients achieving an absolute SALT, or Severity of Alopecia Tool score, of 20 or less after 24 weeks of treatment. A SALT score directly correlates to percentage of hair loss across four parts of the scalp — a score of 100 representing total scalp hair loss, and a score of 0 meaning no scalp hair loss. Patients enrolled in the trial were required to have at least 50 percent scalp hair loss due to alopecia areata, as measured by SALT. And according to Concert, the average starting SALT score across all patients was approximately 85.9.
Now onto the data: 41.5% of patients in the 12 mg arm and 29.6% of those in the 8 mg group reached a SALT score of 20 and under by the end of 24 weeks, compared to 0.8% in the placebo group — achieving the primary endpoint and clocking in a p-value of under 0.0001.
Key secondary endpoints that were met included the percentage of respondents on a Hair Satisfaction Patient Reported Outcome (PRO) scale at Week 24 and the percentage of patients achieving the absolute SALT scores of 20 or less at each of Weeks 20, 16, 12 and 8. While Concert did not give specifics as to those endpoints, the biotech said in a statement that “all key secondary endpoints were met with statistical significance in both dose groups.”
As comes with the territory with JAK inhibitors, safety has been a long-standing concern for the drug class. Concert said that the safety profile of CTP-543, for which the biotech got breakthrough therapy designation back in 2020, was consistent with previous studies, including side effects such as increased creatine kinase levels and upper respiratory infection. The biotech added that two patients developed shingles, and SAEs were observed in nine patients, four of which were in the placebo group. One patient, in the 8 mg twice-daily group, had two events “that were assessed as possibly related to treatment.”
As for what’s next, Concert said in a statement it plans to submit the full results for publication and eventual presentation. A spokesperson told Endpoints News that patients were given the opportunity to enroll in an open-label extension trial — and so far, 90% of “eligible patients” opted in. The planned readout for that trial is sometime next year.
In the meantime, Concert is waiting on data results from THRIVE-AA2, a second Phase III trial with 500 patients enrolled — which are expected sometime in Q3. And if those data come back positive, Concert added that it will use the data from those two trials to file an NDA with the FDA by the end of H1 next year.
That said, Concert is now in a better position against Big Pharma rivals Pfizer and Eli Lilly, each with their own JAK inhibitor — the already-approved Olumiant in Eli Lilly’s case and drug candidate ritlecitinib from Pfizer — and all looking to introduce the first approved treatment for alopecia areata. Aside from rivals, Concert has another battle it’s fighting — a patent dispute against Incyte, which Concert appealed back in 2019 after Incyte won a court challenge against Concert. CTP-543 is a deuterated form of Incyte’s Jakafi.