Clascoterone

The data suggest clascoterone could become the first “potential innovation in over 30 years in male hair loss,” Cosmo said. Cosmo Pharmaceuticals has revealed topline results from two phase 3 trials which indicate that its topical cream clascoterone could become a game-changing treatment for male-pattern hair loss.The parallel studies, which included 1,465 participants with male androgenetic alopecia (AGA) in 50 sites across Europe and the U.S., demonstrated statistically significant and clinically meaningful improvements in hair growth compared to placebo, the company said. One trial showed a 539% relative improvement in target-area hair count (TAHC) through six months, while the other trial showed a 168% gain versus placebo.The difference in the trial figures is driven “entirely by baseline hair counts, not by a difference in drug performance,” Cosmo CEO Giovanni Di Napoli said in an email. “When the two groups experience a similar absolute increase in hair count—but start from different baselines—the study with lower baseline values will show a larger relative percentage.”The treatment could become the first “potential innovation in over 30 years in male hair loss,” the Dublin-based company added.With the news, Cosmo shares were up 20%.“These data have the potential to redefine how dermatologists treat androgenetic alopecia worldwide,” Maria Hordinsky, M.D., of the University of Minnesota’s Department of Dermatology, said in a release.Cosmo's subsidiary Cassiopea scored FDA approved for clascoterone in 2020 as a treatment for acne. It is known commercially as Winlevi and is the top branded topical acne treatment in the U.S., Cosmo said (PDF) in its mid-year report, having drawn 1.4 million prescriptions since its launch in November of 2021.The first-in-class topical androgen receptor inhibitor blocks dihydrotestosterone (DHT) directly at the hair-follicle receptor without systemic absorption, targeting the root cause of male-pattern hair loss without the risks of oral therapies, Cosmo said.  “For decades, patients have had to choose between available treatment options with limited efficacy or safety issues due to systemic hormonal exposure, often resulting in patients not treating their hair loss at all,” Hordinsky added. “These findings show the potential for clascoterone 5% topical solution to change that equation by delivering real, measurable regrowth with negligible systemic exposure.”Cosmo added that patient-reported outcomes further “reinforced the strength” of the topline results, with one study reaching statistical significance and the other showing a positive trend.Treatment-emergent adverse events (TEAEs) were similar across both studies, and similar to placebo, Cosmo added, with most TEAEs not related to clascoterone. After completion of a 12-month safety and durability study in the spring of next year, the company plans parallel submissions with the FDA and European Medicines Agency. Of the 166 million men in the U.S., 65 million have AGA, including 16 million who have been treated and 27 million who are seeking treatment, Cosmo said (PDF) in its most recent annual report. The company’s market research indicates it is a $20 billion opportunity in the U.S., Cosmo added on Wednesday.“At our investor day in July, Cosmo presented in-depth U.S. market research results showing an enormous, underserved demand,” Di Napoli said in a release. “These compelling data give us full confidence in respect to the upcoming regulatory submissions.”Cosmo describes itself as focused on MedTech AI, dermatology, gastrointestinal diseases and contract development and manufacturing (CDMO). The company reported 2024 revenue at 266 million euros ($310 million) for a 186% year-over-year increase.

Cosmo Pharmaceuticals’ shares increased 18% on the Swiss exchange Wednesday morning after it released positive late-stage results from its drug candidate in men with androgenetic alopecia. The Dublin-based biotech has plans to file with US and European regulators next year. Cosmo’s treatment, a 5% solution of clascoterone, delivered “statistically significant and clinically meaningful” improvements in target-area hair count (TAHC) versus placebo in two identically-designed Phase 3 studies. The drug produced a roughly five-fold relative improvement in hair count in one study, and an almost two-fold improvement in the other, the company said. The trials together enrolled more than 1,400 patients with male androgenetic alopecia (AGA) in the US and Europe, according to a company release . Clascoterone targets the sex hormone dihydrotestosterone directly at the hair follicle receptor, meaning there’s no systemic absorption or related safety risks, Cosmo said. The candidate was well tolerated in the Phase 3 studies, with a side-effect profile similar to placebo. This is important because current oral treatments for AGA involve systemic hormonal exposure, Cosmo said. This can lead to issues like decreased libido, erectile dysfunction and ejaculation problems. The biotech said it expects to complete a 12-month safety follow-up in spring 2026. CEO Giovanni Di Napoli described Wednesday’s results as “a pivotal moment for Cosmo and for billions of men worldwide who struggle every day with the emotional and social impact of hair loss.”