Concert Pharmaceuticals has been waiting to tout Phase III data amidst a
sell-off
of the bulk of its pipeline to Terran Biosciences and several other events for the biotech. And on Monday, the company rolled out the red carpet.
Concert unveiled topline results from its second Phase III clinical trial, dubbed THRIVE-AA2, evaluating its oral candidate currently called CTP-543 in adult patients with moderate to severe alopecia areata. The candidate is an inhibitor of Janus kinases JAK1 and JAK2.
The primary efficacy endpoint, the percentage of patients achieving a certain score on an Alopecia severity scale after 24 weeks, was met with statistical significance in both the 8 mg twice-daily and 12 mg twice-daily dose groups relative to placebo. Treatment of the candidate was also generally well-tolerated.
According to the company, those enrolled were required to have at least 50 percent scalp hair loss as measured by the SALT scale (with a score of 100 representing complete scalp hair loss and zero as no loss). The average baseline SALT score across all patients was approximately 87.9.
Statistically significant proportions of patients treated with either dose levels achieved a SALT score of 20 or less. Concert said 38.3% hit the score in the 12 mg twice-daily dose group and 33% in the 8 mg twice-daily dose group. The study ultimately netted a p-value of p<0.0001 for both doses.
The positive data were welcome news to investors, as the Massachusetts’s biotech’s stock price
$CNCE
rose over 6% since opening.
“Based on the results from our Phase 3 program, we believe CTP-543, if approved, could be a best-in-class treatment option for alopecia areata and have a distinct therapeutic profile with features that are important to patients, including the percentage of patients with clinically-meaningful hair regrowth, patient satisfaction levels, and hair regrowth rates,” said Concert’s chief development officer James Cassella.
On the back of the results, Cassella said the company is now looking to prepare an NDA submission to the FDA in the first half of next year as well as plans to commercialize the drug.
Concert’s path to the FDA comes
as Eli Lilly was the first across the line
in June as baricitinib, aka Olumiant, netted FDA in a once-daily pill in three dose levels, putting pressure on the biotech now to get approval for its candidate.
Secondary endpoints included 47% of patients in the 8 mg twice-daily group and 52% of patients in the 12 mg twice-daily group saying they were “satisfied” or “very satisfied” with the treatment, compared to 2% of patients in the placebo group.
The candidate and the company itself have been on a wild ride.
In 2018
, the drug’s data initially did not hold up against Pfizer’s candidate, but execs decided to forge ahead and got
breakthrough therapy designation
in 2020. CTP-543 is one of the company’s major hopes as the company hit a significant speed bump
last year
when a Phase II trial for its schizophrenia program flopped, causing the company to
ax the program
and its stock to tank by 40%.