Navepegritide

Pictured: BioMarin headquarters in California/iSto iStock , Sundry Photography The biotech beat Wall Street’s third-quarter revenue forecast by 6%, driven by increased uptake of its achondroplasia drug Voxzogo. However, William Blair downgraded BioMarin’s shares to market perform due to a “lack of near-term catalysts” and uncertainty around Voxzogo’s potential revenue growth. BioMarin Pharmaceuticals on Tuesday announced $746 million in total revenue in the third quarter , 28% year-over-year growth driven by the continued strong uptake of its therapies for rare genetic diseases. The biotech beat consensus estimates, surpassing Wall Street’s expected Q3 revenue of $703 million by 6%. BMO Capital Markets analyst Kostas Biliouris in an investor note attributed BioMarin’s strong performance to the continued growth of its achondroplasia medicine Voxzogo, with sales of $190 million jumping 54%. There are now over 3,800 patients on Voxzogo, according to BioMarin, up from around 3,500 in the second quarter . Still, Wall Street anticipated Voxzogo generating $195 million for BioMarin in Q3, a bar the company missed by 3%. Roctavian, a gene therapy approved in June 2023 for hemophilia A , brought in $7 million in the quarter, in line with the analyst consensus. And while its relative contribution to BioMarin’s total Q3 revenue is low, Roctavian presents a high-growth opportunity for the biotech with 600% sales growth versus the same period the year prior. Biliouris said that this “continued uptake” of Roctavian reflects the company’s efforts to focus on the U.S., Italy and Germany markets, potentially paving the way for hitting around $60 million in revenue for the gene therapy by 2025. Truist Securities analyst Joon Lee also credited BioMarin’s Sanofi-partnered enzyme replacement therapy Aldurazyme for its Q3 beat. Sales of Aldurazyme, indicated for mucopolysaccharidosis I, jumped 407% in the quarter bringing in $71 million and beating the consensus expectation of $30 million by $137%. Encouraged by its strong Q3 showing—and by the continued growth of its products—BioMarin on Tuesday raised its full-year 2024 revenue guidance to between $2.79 billion and $2.825 billion. The biotech previously expected a range of $2.75 billion to $2.825 billion this year. Despite BioMarin’s promising outlook for 2024, Lee warned about impending competitive pressure on Voxzogo. Last month, Ascendis Pharma revealed strong pivotal data for its investigational drug TransCon CNP, also known as navepegritide, which showed significant improvements in annual growth velocity in children with achondroplasia. Ascendis is gearing up for a regulatory filing for TransCon CNP in the first quarter of 2025. “In light of better-than-expected data, we’re left to wonder what further impact competition from TransCon-CNP may have on BioMarin’s mid- to long-term guidance,” Lee said. Likewise, William Blair analyst Sami Corwin in a note to investors said that despite the Q3 beat, her firm downgraded shares of BioMarin to market perform. “We do not think its clinical pipeline will be a source of significant value creation in the near term,” according to Corwin. “Voxzogo remains the key top-line growth driver for the company, but the threat of competition could erode its revenue growth and market share.”

BioMarin Pharmaceutical, after months of layoffs, spending cuts and research reorganizations, has staked its futureon 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 BioMarins 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 BioMarins, 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 BioMarins down by 18% Monday, reflecting Wall Streets 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 teams price target for the companys 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 studys start, allowing for rough comparisons to the data supporting Voxzogos 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 drugs FDA-approved label describes a placebo-adjusted change of 1.57 cm/year.One advantage TransCon CNP could hold, if approved, is in how its 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 BioMarins 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. '