Ascendis Pharma’s TransCon CNP, a once-weekly treatment for achondroplasia, demonstrated superior annualized growth velocity compared to placebo in a recent trial. This could pose a significant challenge to BioMarin’s Voxzogo, the current leading treatment for achondroplasia. While BioMarin holds a first-mover advantage, the potential for TransCon CNP’s improved dosing schedule and safety profile could impact Voxzogo’s market share in the coming years.
Ascendis Pharma A/S (ASND) announced positive topline data from a Phase 3 trial of TransCon CNP (navepegritide) for children with achondroplasia, a genetic disorder causing dwarfism. The investigational drug demonstrated significant improvement in growth velocity compared to placebo, leading to a potential FDA application in the first quarter of 2025.
The Dow Jones Industrial Average gained over 200 points on Monday, while the broader market saw mixed performance. Alcoa Corporation led the gains after announcing a lucrative sale, while Ascendis Pharma, Bausch + Lomb, and Nuvalent also saw significant price increases.
Ascendis Pharma’s second-quarter sales fell short of expectations, with the company’s Yorvipath pricing coming in higher than anticipated. While the company faces challenges in the growth hormone market, analysts remain optimistic about the potential of Yorvipath in hypoparathyroidism and TransCon CNP in Achondroplasia.
Ascendis Pharma reported disappointing second-quarter results, missing both sales and earnings estimates. Revenue was significantly impacted by a negative adjustment to prior period sales deductions, while Skytrofa sales declined year-over-year despite volume growth. The company lowered its full-year sales guidance for Skytrofa and entered into a royalty funding agreement for its new hypoparathyroidism drug, Yorvipath. Ascendis’ stock price plunged 15% in pre-market trading.
Ascendis Pharma’s Yorvipath (palopegteriparatide) has received FDA approval for the treatment of hypoparathyroidism in adults. This approval fills a crucial gap in treatment options for this rare condition, particularly as Takeda’s Natpar/Natpara will be discontinued in 2024. Yorvipath is expected to be available in the first quarter of 2025.
