Cosmo Pharmaceuticals says it plans to file for FDA approval of its androgenetic alopecia (AGA) candidate clascoterone 5% topical solution early next year, after the androgen receptor inhibitor generated 12-month Phase III data showing statistically significant continued hair growth as well as positive safety for chronic use.
Data from Cosmo’s Phase III program for clascoterone confirmed the drug’s long-term safety profile was comparable to vehicle, supporting suitability for chronic use in a lifelong condition. Clascoterone also showed a novel mechanism designed to target the underlying biology of hair loss and continued efficacy with ongoing use, Cosmo said.
A total of 1,465 patients were enrolled in the Phase III program, the largest for any topical treatment candidate for male AGA, according to Cosmo. The program consists of the SCALP 1 (NCT05910450) and SCALP 2 (NCT05914805) trials, which evaluated patients across 51 study centers in the United States and Europe.
Patients who remained on continuous clascoterone treatment for the full 12 months achieved a statistically significant 239% improvement in Target Area Hair Count (TAHC) compared with patients who received clascoterone for six months and were then switched to vehicle from month 7 to month 12, according to Cosmo.
That’s down slightly from the 252% improvement in TAHC shown for clascoterone versus “vehicle” or placebo in Cosmo’s six-month results, released in December. Cosmo Pharma CEO Giovanni Di Napoli told GEN that the 6- and 12-month results were not directly comparable due to differences in Part 1 and Part 2 of the Phase III placebo-controlled program and the corresponding patient groups being compared.
Part 1 is a double-blind study assessing if clascoterone was effective and safe compared to placebo when applied twice daily for up to six months. Part 2 is a single blind study that measured clascoterone’s long-term safety and efficacy versus placebo for an additional six months in patients who had responded to the study drug in Part 1. During Part 2, participants were re-randomized to receive either clascoterone 5% solution or vehicle solution.
SCALP 1 enrolled 702 patients in the United States, while SCALP 2 enrolled 763 patients in the Unites States as well as Germany and Poland.
Primary outcome measures
Change in vellus TAHC (hair of up to 30 micrometers in diameter) from baseline was the trials’ primary outcome measure, paired with a patient-reported outcome assessing participants’ perception of hair growth improvement.
Additional assessments of the trials included investigator-reviewed global scalp photography and secondary endpoints that included changes in non-vellus TAHC (thicker, pigmented hair >30-40 micrometers in diameter), and changes in subject’s assessment of satisfaction score.
Patients treated with clascoterone for 12 months reported a statistically significant +24.5% relative improvement in treatment satisfaction versus vehicle groups, according to Cosmo. The clascoterone users also reported positive ease of use and product acceptability at month 12—results that according to the company support positive real-world usability and long-term adherence potential for the drug.
Cosmo said it plans to submit its full Phase III dataset for publication in a leading peer-reviewed medical journal and present its findings at future major dermatology congresses.
‘Defining moment’
“These 12-month Phase III results mark a defining moment for clascoterone and for the treatment of male hair loss,” Di Napoli stated. “We are now seeing the combination that matters most: positive long-term safety, statistically significant continued hair growth through one year, and clear evidence that ongoing treatment drives sustained benefit.”
Investors responded to the positive 12-month data by sending Cosmo shares traded on the SIX Swiss Exchange rising 6% on the day of the announcement, from CHF95.50 ($122.69) to CHF101.40 ($130.27) on April 15. Since then shares have fluctuated in the high CHF 90 range, closing Monday at CHF 98.50 ($126.55).
Di Napoli said clascoterone has the potential to emerge as a major new therapeutic option and a highly valuable growth platform for Cosmo by tackling the most common cause of hair loss in men. Androgenetic alopecia, also called male pattern hair loss, affects approximately 40% of men worldwide—including 39% of males in the United States (65 million men).
“We are moving with urgency toward regulatory submissions and commercialization discussions,” Di Napoli added.
Cosmo’s results “likely now enable more advanced partnership discussions, in our view, with detailed presentation of results the next step to fully de-risk the asset,” Benjamin Jackson, equity analyst with Jefferies, wrote April 15 in a research note.
Jackson predicted clascoterone could generate $4 billion in peak-year worldwide sales.
“Our $4 billion WW potential peak sales require just 4% penetration of treated and 6% penetration of untreated men at peak, assuming a capable commercial partner is successfully found,” Jackson added.
Cosmo said it is preparing to submit not only an NDA for clascoterone in the U.S., but a marketing authorization application to the European Medicines Agency.
Clascoterone’s 1% formulation is already FDA-approved and marketed as Winlevi® for topical treatment of acne vulgaris in patients ages 12 and older.
