- Small Italian biotech Cassiopea is riding high, reporting Monday a second slate of positive clinical results for its lead compound in as many weeks. Shares, which trade on the Swiss stock exchange, have nearly doubled in value compared to their closing price July 9.
- In the latest readout, interim data from a dose-finding Phase 2 study showed Cassiopea's topical antiandrogen therapy clascoterone is on track to meet its primary goal versus vehicle in treating androgenetic alopecia, or hair loss, in men.
- On a second measure, results at the six-month data check were not yet statistically significant but showed a "directional improvement" favoring treatment, Cassiopea said. The study will continue through to 12 months, but the biotech believes it has already seen enough evidence to merit initiation of a proof-of-concept trial in women.
The results for clascoterone in alopecia follow the success last week of the drug — in a different formulation and strength — as a treatment of acne. Together, the clinical results underpin Cassiopea's near-term potential and have boosted share prices by more than 90% since July 9, before the acne data were released.
While the results in acne provided much of the boost behind the biotech's stock, Monday's readout should offer investors some confidence in the ongoing Phase 2 trial of clascoterone in alopecia.
In the 404-subject study, Cassiopea tested four dose regimens of the drug against a vehicle comparator. Six-month results from 375 participants showed treatment with all four doses led to a statistically significant change from baseline in target area headcount (TAHC). The highest dose, 7.5% solution applied once a day, led to the greatest mean change from baseline.
The study's primary endpoint will measure the change from baseline in TAHC at 12 months.
On the study's other primary goal, a measure known as hair growth assessment (HGA), results fell short of statistical significance at the interim check. A greater proportion of participants in the treatment arms did report increased hair growth by HGA than did those given vehicle.
Cassiopea believes these results, while preliminary, show competitive efficacy to on-market alopecia treatment Propecia (finasteride).
Clascoterone also hasn't been associated with the kind of systemic hormonal effects observed with the orally administered Propecia.
"Breezula which is applied locally has to date not been associated with any hormonal imbalances which could represent an important competitive differentiator if positive long-term efficacy can be demonstrated," wrote Jefferies analyst Peter Welford in a July 16 note, using Cassiopea's proposed brand name for clascoterone in alopecia.
Results from the Phase 2 study showed no treatment-related adverse events. Local skin reactions were "predominantly classified as mild," the company said.
If the 12 month data holds up, Cassiopea plans to advance clascoterone into Phase 3 testing for alopecia, with a potential approval forecast for 2021. Jefferies' Welford estimates peak annual U.S. sales to be relatively modest, at $215 million.
Clascoterone in acne, however, could represent a greater opportunity, and the recent Phase 3 successes set up a potential filing for approval in early 2019.