Ascendis trial data pressures BioMarin

BioMarin Pharmaceutical, headquartered in San Rafael, California, has emphasized the importance of its current medication for achondroplasia, a common cause of dwarfism, in light of recent layoffs, budget cuts, and research restructuring. However, a new potential competitor could challenge BioMarin’s dominance in this market.

Ascendis Pharma recently unveiled promising results from a clinical trial for their treatment of achondroplasia, which is similar to BioMarin’s drug, Voxzogo. According to Wall Street analysts, Ascendis' drug could be a strong competitor. Voxzogo, approved in the U.S. three years ago, remains the only available treatment for achondroplasia. Ascendis aims to seek FDA approval for its drug early next year, potentially leading to a market entry by late 2025 or early 2026.

This announcement had an immediate impact on the stock market: Ascendis' shares surged by 17%, while BioMarin’s shares fell by 18%. Analysts expressed concerns about BioMarin’s ability to maintain its market share and drive growth in a more competitive environment. Baird analyst Jack Allen highlighted the increased investor apprehension regarding Voxzogo’s future performance.

Stifel analyst Paul Matteis, who maintains a Buy rating on BioMarin, adjusted his price target following Ascendis' data release. He noted that Ascendis’ drug appeared clinically equivalent to Voxzogo. The clinical trial results showed that Ascendis’ treatment, called TransCon CNP, significantly improved annual growth rates compared to a placebo. Over a year, the difference between the treated group and the placebo group was about 1.5 centimeters per year.

Further details from Ascendis indicated that children aged 5 to 11 treated with TransCon CNP had a placebo-adjusted increase in annual growth velocity of 1.78 cm/year. For comparison, Voxzogo’s FDA-approved label cites a placebo-adjusted change of 1.57 cm/year.

One potential advantage for TransCon CNP is its dosing regimen. While Voxzogo requires daily administration, TransCon CNP is administered weekly. This could provide a significant convenience benefit for patients and caregivers. Leerink Partners analyst Joseph Schwartz noted that the efficacy and safety profile of TransCon CNP appears comparable to Voxzogo, but with significantly fewer injections.

The positive data from Ascendis may also impact BridgeBio Pharma, which is developing another achondroplasia treatment called infigratinib. Recently, the FDA granted infigratinib a Breakthrough Therapy designation, intended to expedite its development in the ongoing Phase 3 trial. Following this announcement, BridgeBio Pharma’s shares increased by nearly 5%.

The emergence of potential competitors like Ascendis Pharma and BridgeBio Pharma introduces a new dynamic into the achondroplasia treatment market, which has so far been led by BioMarin’s Voxzogo. As these new treatments advance toward regulatory approval and potential market entry, BioMarin’s strategy and market position will be closely watched by investors and analysts alike.