Dive Brief:
- An experimental treatment from Veradermics hit the main goal of a Phase 3 trial, helping people with male pattern baldness grow significantly more new hair over six months than those who got a placebo. If approved by regulators, the therapy, an extended-release, oral formulation of the topical treatment minoxidil, could become the first new marketed pill for baldness in 30 years.
- Codenamed VDPHL01, Veradermics’ pill, codenamed VDPHL01, was also “generally well tolerated,” with side effect rates similar to a placebo, the company said Monday. No heart issues of “special interest” were reported by trial investigators, a significant finding because of cases of fluid buildup around the heart that have occurred when minoxidil is used to treat blood pressure.
- Wall Street analysts hailed the results as a “home run” and “best case scenario” for Veradermics, with VDPHL01 surpassing investor expectations on hair regrowth while providing reassurance on safety. Shares surged as much as 48% in morning trading, and have more than quintupled in value since Veradermics’ initial public offering in February.
Dive Insight:
Should it win an approval, VDPHL01 will compete with topical minoxidil, available for decades as Rogaine, as well as a pill called Propecia that the FDA approved in 1997 and is now off-patent. Low-dose minoxidil pills are also prescribed off-label to treat pattern baldness, which is driven by many factors, among them genetics, hormones and aging.
Telemedicine companies like Hims & Hers Health and Ro sell these generic treatments, and over-the-counter solutions are available at retail pharmacies, underscoring the commercial challenge ahead should Veradermics’ drug get to market. But those available therapies have drawbacks; topical minoxidil can cause itchiness and flakiness on the scalp, while finasteride is associated in some cases with erectile dysfunction, anxiety and suicidal thoughts.
Veradermics enrolled 519 men who were randomized to take an 8.5 milligram dose of VDPHL01 once or twice daily, or a placebo. Investigators then studied the therapy’s effects on baldness by measuring patients’ non-vellus hair count per square centimeter in a specific target area.
After six months, those who got the once-daily dose saw their hair count per square centimeter rise by a little more than 30 at six months. Study volunteers who received the twice-daily dose saw that figure climb by 33, significantly more than the just over seven reported among those who got a placebo, Veradermics said.
A second, patient-reported measure also showed a significant benefit for VDPHL01, Veradermics said. Of those in the once-daily arm, 79% reported an improvement on a patient-reported outcome scale called AAIRS, along with 86% of those in the twice-daily arm. By comparison, 36% of placebo recipients reported an improvement.
“Dermatology has been treating hair loss with a drug borrowed from cardiology, in a formulation never intended for our patients, at doses we arrived at informally,” said Michael Gold, a dermatologist who served as a trial investigator, in a statement. “VDPHL01 is the first oral minoxidil formulation developed specifically for pattern hair loss, and now the first to generate positive Phase 3 results of efficacy and safety.”
In separate notes to investors, analysts with Cantor Fitzgerald and Leerink Partners said the results exceeded investor benchmarks that the pill would beat a placebo by 20 or more hairs per square centimeter, and also prove superior to low-dose oral minoxidil formulations.
“Due to better-than-expected efficacy and safety,” the “commercial case for VDPHL01 is now stronger,” wrote Cantor analyst Prakhar Agrawal in a client note. He added that the data suggest the pill could record $1.5 billion to $2 billion in U.S. sales alone.
