BioMarin Pharmaceutical, after months of layoffs, spending cuts and research reorganizations, has staked its future on the continued success of its approved drug for a common cause of dwarfism. But the San Rafael, California-based biotechnology company could have a new competitor that might complicate those well-laid plans.
On Monday, Ascendis Pharma released summary results from a clinical trial testing a treatment that, like BioMarin’s drug, is designed to treat children with achondroplasia, a skeletal disorder that leads to disproportionate short stature.
To analysts on Wall Street, the benefit provided by Ascendis’ medicine appeared competitive to BioMarin’s, which was approved in the U.S. three years ago and is still the only available treatment. Ascendis plans to ask the Food and Drug Administration early next year for clearance of its drug, which could set up a regulatory decision and market entry by late 2025 or early 2026.
The news sent Ascendis shares up by 17% and pushed BioMarin’s down by 18% Monday, reflecting Wall Street’s reset expectations for how much of the achondroplasia market the latter company may claim with its drug, called Voxzogo.
“We believe the entry of this competitor into the market could increase investor concerns surrounding the ability for Voxzogo to drive continued top-line growth in a competitive market,” Baird analyst Jack Allen wrote in an investor note.
Paul Matteis, an analyst at Stifel who has a “Buy” rating on BioMarin, reduced his team’s price target for the company’s stock as a result of Ascendis’ data. “We view the drug as clinically equivalent for Voxzogo,” he added in a note to clients.
The trial results from Ascendis show that its treatment, dubbed TransCon CNP, increased annual growth velocity by significantly more than placebo. The statistical difference between the two groups, measured at one year, was about 1.5 centimeters per year.
Ascendis also reported a change in participants’ annual growth velocity compared to the study’s start, allowing for rough comparisons to the data supporting Voxzogo’s clearance. Among children aged 5 to 11 years, treatment with TransCon CNP led to a placebo-adjusted change in annual growth velocity of 1.78 cm/year. For Voxzogo, the drug’s FDA-approved label describes a placebo-adjusted change of 1.57 cm/year.
One advantage TransCon CNP could hold, if approved, is in how it’s dosed. Ascendis’ drug requires weekly injections, while Voxzogo is taken once daily.
“[Ascendis] data] demonstrates a profile which suggests safety and efficacy that is comparable to BioMarin’s Voxzogo with 1/7th the number of injections,” wrote Leerink Partners’ Joseph Schwartz in a client note.
Another company that could be affected by Ascendis’ positive data is BridgeBio Pharma, which is developing a drug called infigratinib. On Tuesday, the company disclosed that the FDA had granted infigratinib a Breakthrough Therapy designation, which it said will help speed its development through an ongoing Phase 3 trial. Shares in the company rose by nearly 5% Tuesday.
