News and Analysis

Cosmo Pharmaceuticals (SIX Swiss: COSM) found itself this past week in the enviable position of attracting welcome attention from investors and other market watchers after coming out with topline Phase III data for its androgenetic alopecia (AGA) candidate clascoterone that was anything but hair-raising.
Clascoterone 5% topical solution is an androgen receptor inhibitor whose 1% formulation is already FDA-approved and marketed as Winlevi® for topical treatment of acne vulgaris in patients ages 12 and older. Since winning the agency’s approval in 2021, Winlevi has generated more than 1.6 million prescriptions—enough, Cosmo says, to make it the nation’s leading branded prescription drug for topical acne.

This past week, Cosmo made an arguably strong case for clascoterone in the potentially much more lucrative market of AGA, also called male pattern baldness. The company released data from its two identically designed Phase III trials which it understandably called “compelling.”
A total 1,465 patients were enrolled in SCALP 1 (NCT05910450) and SCALP 2 (NCT05914805), making it the single largest Phase III program for any topical AGA candidate, according to Cosmo. The two trials reported a combined 252% relative improvement in Target-Area Hair Count (TAHC) for clascoterone vs. “vehicle” or placebo.
Investors appeared especially impressed with results from SCALP 2 since it showed an eye-popping 539% (5.39x) relative improvement in TAHC vs. placebo, compared with SCALP 1’s 168% (1.68x) TAHC improvement. 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.

“It’s not uncommon for a Phase III program with two identical studies to have a bit of variability in results, but these both were statistically significant,” Diana Harbort, president of Cosmo’s Dermatology Division, told GEN.
Cosmo plans to present detailed data from SCALP 1 and SCALP 2 next year at a medical conference and in a peer-reviewed journal, she added.
Buying surge

Investors reacted to Cosmo’s anything-but-bad hair day with a buying surge that sent its shares on the SIX Swiss Exchange soaring nearly 40%. Shares jumped 24% Wednesday from CHF 65.50 ($81.44) to CHF 81.20 ($100.96) as of 9:31 a.m. ET before finishing the day at CHF 78.30 ($97.35) and a 19.5% gain.
Cosmo’s momentum continued Thursday, with the stock rising another 14% to CHF 89 ($110.65), and increasing another 3% Friday, finishing at CHF 91.40 ($113.64).
Cosmo’s next move will be completing 12-month safety data, something the company expects to have in spring 2026. Should that data also prove positive, Cosmo will be filing for approvals of clascoterone for AGA with the FDA and European Medicines Agency (EMA).

During that process, Harbort said, Cosmo would entertain discussions with potential partners to handle commercial activity—including distribution, sales, and marketing—of clascoterone for AGA.
“We’re not in any particular rush, because we are well-funded,” Harbort said. Cosmo finished the first half of 2025 with €50.749 million ($59.114 million) in cash and cash equivalents, up nearly 15% from €44.296 million ($51.597 million) at the end of 2024.
“We would expect a deal that might be commensurate with the massive market opportunity and innovation we’re bringing,” Harbort added.
Numerous treatments in development

If approved by regulators, clascoterone would be the first topical androgen receptor inhibitor to win approval with an AGA indication. Cosmo’s clascoterone is furthest along among numerous treatments in development for AGA, including:

ET-02 (Eirion Therapeutics)—In January 2025, Eirion announced positive results for a first-in-man Phase I trial assessing the topical small molecule in androgenic alopecia (age-related hair loss). Topical ET-02 is also in preclinical phases for hair greying and an undisclosed indication, while an oral form of ET-02 is in preclinical phase for androgenic alopecia.
KX-826 (Kintor Pharmaceuticals)—The 1% topical androgen receptor agonist completed patient enrollment in the Phase III stage of its Phase II/III pivotal trial (NCT06622824), the Suzhou, China-based drug developer said in July. The Phase III stage is expected to be completed by the beginning of 2026.
PP405 (Pelage Pharmaceuticals)—The stem cell-targeting small molecule is expected to advance into Phase III trials in 2026, after reporting positive data in a Phase IIa trial (NCT06393452) in June. Pelage is funding advancement of PP405 through clinical studies using proceeds from a $120 million Series B financing completed in October.

TDM-105795 (TechnoDerma Medicines)—The topical small molecule is designed to stimulate hair growth by activating the Wnt/β-catenin pathway. In February 2024, Chengdu, China-based TechnoDerma reported positive data from its Phase IIa trial (NCT05802173) showing mean changes from baseline of  24.3 hairs with the high label strength (0.02%) and 20.3 hairs with the low label strength (0.0025%) topical solution vs. 14.0 hairs with placebo in 1 cm2 test areas.

$20B+ U.S. market opportunity

Cosmo estimates clascoterone’s total addressable market opportunity in AGA as exceeding $20 billion in sales in the United States alone, where 65 million American men have the condition, of which 43 million are already being treated or actively seeking treatment, according to the company. The United States is a sliver of a global AGA market that Cosmo has estimated to be between 1.2 billion and 2 billion men.
“These other drugs have early data, or data developed in China. We have very compelling data that spans Phase I, II, and III, and we show very, very compelling clinical results that are robust,” Harbort said. “This is a lifestyle drug that patients elect to use. This drug will require a prescription, but it’s a lifestyle drug, which means that it needs to be really safe. And patients are going to make a decision not only on the clinical results, but the safety. And we have very compelling safety.”
Treatment emergent adverse effects (TEAEs) in the two Phase III trials were 5.1% for clascoterone and 6.1% for placebo. According to Cosmo, most TEAEs were unrelated to clascoterone and were mild to moderate in severity, making them similar to those of placebo
Male AGA, the most common form of hair loss, is characterized by a receding frontal hairline and diffuse hair thinning, as hair follicles are progressively miniaturized, a process driven by dihydrotestosterone (DHT). Clascoterone works by competing with testosterone and DHT to bind to androgen receptors on scalp hair follicles. By blocking DHT directly at the hair-follicle receptor without systemic absorption, clascoterone is the first treatment designed to target the biological root cause of male AGA without the risks associated with oral therapies.
First new MOA in decades

Clascoterone would apply the first new mechanism of action (MOA) in AGA treatment since the arrival to market of the two treatments most often used for male pattern baldness. One is finasteride, launched in 1997 as Propecia® for male pattern hair loss after initially entering the market five years earlier as Proscar® to treat enlarged prostate or benign prostatic hyperplasia (BPH). The other is minoxidil, re-launched as Rogaine® in 1988 after initially winning FDA approval in 1979 as Loniten® to treat high blood pressure.
According to a paper published by researchers at Weill Cornell Medicine and University of California’s Irvine School of Medicine, common adverse effects of oral minoxidil include dose-dependent hypertrichosis (24% incidence), transient shedding (16-22%), and mild peripheral edema (2%), while serious complications, including pericardial effusion, are rare at doses used for alopecia.

As for finasteride, a 2020 paper cited previous studies to link finasteride and another BPH treatment, dutasteride, to “greater risk of erectile dysfunction, loss of libido, and ejaculatory dysfunction.”
Male AGA is generally managed with finasteride, though oral minoxidil is prescribed in younger males, a research team led by Aditya K. Gupta, MD, PhD, of the University of Toronto School of Medicine reported in a paper published in PLoS, based on data from the NIH’s “All of Us” research program.
Linked to social determinants

According to the paper, intended as a snapshot of the epidemiology and management of AGA in the United States, most men with the condition were between ages 20 and 39, compared with ages 60 to 69 for females with AGA.
“AGA is linked to the social determinants of health; addressing the AGA may help better manage the underlying mental and physical state,” Gupta and colleagues concluded.
If Cosmo’s sales meet expectations, clascoterone would surpass the sales of the current top-selling prescription drug, Merck & Co.’s multi-indication cancer immunotherapy Keytruda® (pembrolizumab), which racked up $23.303 billion in the first nine months of this year, on top of $29.482 billion in 2024 sales. However, Keytruda is set to lose patent exclusivity in 2028, while clascoterone 5% for AGA is protected by patents stretching until 2036.
Blockbuster-level sales would be a leap forward for Cosmo, which finished the first half of 2025 with €4.9 million ($5.704 million) in earnings before interest, depreciation, taxes, and amortization or EBITDA, down 94% from €84.9 million ($98.843 million) in H1 2024, on revenue that fell 62% year-over-year, to €51.72 million ($60.214 million) from €136.237 million ($158.61). The H1 2024 number was skewed by a €92.5 million ($107.69 million) milestone payment from Medtronic due to the expansion of an AI-driven partnership with the medtech giant.
Following H1, Cosmo more than doubled its investor guidance, saying that it now expects to finish with EBITDA including R&D investments of €5.5 million to €7.5 million ($6.403 million to $8.732 million), compared to its previous forecast of €1 million to €3 million ($1.164 million to $3.493 million), citing higher-than-expected “other” income, defined as revenue sources outside its primary operations, and improved operational efficiencies.
However, Cosmo kept its projected total revenue range between €102 million and €107 million ($118.8 million and about $125 million), with most of that revenue expected to be recurring (€85 million to €90 million, or $99 million to nearly $105 million).
uniQure tumbles on negative FDA update

uniQure (NASDAQ: QURE) shares slipped nearly 11% from $25.48 to $22.74 on Thursday after announcing that it received minutes of its October 29 meeting with the FDA, confirming that data from the Phase I/II studies of its Huntington’s disease candidate AMT-130 are unlikely to sway the agency toward supporting the company’s Biologics License Application (BLA) submission. Shares fell another 6% on Friday, to $21.33.
The minutes confirmed what uniQure announced last month—that what it called “game-changing” data in September, showing significant slowing of Huntington’s disease (HD) progression in patients treated with its gene therapy candidate AMT-130, may not be enough to secure FDA approval. The announcement sparked a 62% plunge in uniQure stock, from $67.69 to $25.69.
uniQure said it was “carefully evaluating the feedback and plans to urgently request a follow-up meeting with the FDA set for the first quarter of 2026.
uniQure maintains that the FDA’s position represents a reversal of earlier agency guidance supporting the company’s collection of data from the Phase I/II studies compared with an external control.
“We still view the agency’s change in guidance as very surprising given the numerous interactions uniQure has had with the FDA since its November 2024 meeting,” Sami Corwin, PhD, a biotechnology-focused healthcare analyst with William Blair, wrote in a research note.
Corwin also cited the FDA’s articulation of a new “plausible mechanism pathway” toward approval for personalized therapies where randomized trials are deemed to be unfeasible, as well as Commissioner Martin A. Makary, MD’s announcement this past week that the FDA plans to require only one clinical trial for new drug approvals instead of two.
“Given the path to and timing of BLA submission for AMT-130 are still unclear, we believe the stock will remain under pressure until there is a line of sight to a reasonable and expeditious path to regulatory submission,” Corwin added.
Capricor nearly quintuples

Capricor Therapeutics (NASDAQ: CAPR) shares nearly quintupled, soaring 371% on Wednesday from $6.36 to $29.96 after the company announced positive topline results from its pivotal Phase III HOPE-3 trial (NCT05126758) assessing its lead pipeline candidate Deramiocel, the company’s allogeneic cardiosphere-derived cell therapy candidate, as a treatment of Duchenne muscular dystrophy (DMD).
Capricor said Deramiocel met the trial’s primary endpoint by showing a nearly 54% slowing of skeletal muscle disease progression—specifically a change as measured by the Upper Limb test, version 2 (PUL 2.0). Deramiocel also aced the trial’s key secondary cardiac endpoint with a 91% slowing of left ventricular ejection fraction (LVEF). Both outcomes were compared to placebo.
The positive data comes five months after the FDA stunned Capricor by rejecting its BLA for Deramiocel, instead issuing a Complete Response Letter (CRL).
“These HOPE-3 topline results are encouraging for the Duchenne community, particularly non-ambulatory individuals for whom there are limited therapeutic options,” Parent Project Muscular Dystrophy observed.
The post StockWatch: Cosmo Surges 40% on Baldness Candidate’s Phase III Data appeared first on GEN – Genetic Engineering and Biotechnology News.