Ascendis Pharma A/S (ASND) announced promising topline data on Monday from a clinical trial involving 84 children with achondroplasia, a genetic disorder that causes dwarfism. The trial focused on the efficacy of TransCon CNP, a once-weekly treatment, in promoting growth. The results showed that children treated with TransCon CNP demonstrated superior annualized growth velocity (AGV) compared to those receiving a placebo.
This news has sent ripples through the pharmaceutical industry, particularly impacting BioMarin Pharmaceutical Inc. (BMRN), whose Voxzogo (vosoritide) currently holds the market lead in treating achondroplasia. William Blair, a financial services firm, has noted that TransCon CNP’s effectiveness appears comparable to Voxzogo, which is approved for stimulating growth in children with achondroplasia and open growth plates. Early data suggests that TransCon CNP could potentially erode Voxzogo’s market share by the end of 2025.
TransCon CNP stands out due to its convenient once-weekly dosing schedule and potentially improved safety profile, making it a more attractive option for patients. However, William Blair’s analysts acknowledge that Ascendis’ freedom to operate may be uncertain. There’s a possibility that TransCon CNP could infringe upon BioMarin’s patent portfolio for long-acting CNP, potentially hindering Ascendis’ launch or leading to royalty payments to BioMarin.
Despite the competition, BioMarin maintains its first-mover advantage and is actively pursuing additional trials to expand the applications for Voxzogo. The company also plans to submit an investigational new drug application for its own long-acting CNP in the fourth quarter of 2024. However, the emergence of TransCon CNP as a viable alternative is expected to exert ongoing pressure on Voxzogo’s market share, potentially affecting the stock’s performance in the near future.
BioMarin recently unveiled its ambition to achieve $4 billion in revenue by 2027 and target an adjusted operating margin of 40%. However, the competitive landscape with TransCon CNP entering the scene could complicate these ambitious plans.
The news of Ascendis’ successful trial has led to a significant drop in BioMarin’s stock price. As of Monday’s close, BMRN stock was down by 17.70% at $69.86. This highlights the potential impact of TransCon CNP’s success on the future trajectory of BioMarin’s business.
