Navepegritide

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. '

–    TransCon CNP demonstrated AGV superior to placebo with LS mean treatment difference of 1.49 cm/year at Week 52 (p<0.0001) –    For children aged 5-11 years TransCon CNP demonstrated a change from baseline AGV superior to placebo with LS mean treatment difference of 1.78 cm/year at Week 52 (p<0.0001) –    Other endpoints supportive that TransCon CNP may provide benefits beyond linear growth –    TransCon CNP was generally well-tolerated, with low frequency of injection site reactions (0.41 events per patient year), all mild –    Ascendis to host investor conference call Monday, September 16, at 8:00 a.m. E.T. COPENHAGEN, Denmark I September 16, 2024 I Ascendis Pharma A/S (Nasdaq: ASND) today announced topline data from the pivotal double-blind placebo-controlled ApproaCH Trial of TransCon CNP (navepegritide), which included 84 children with achondroplasia (ages 2-11 years) randomized 2:1 (TransCon CNP:placebo). TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to provide sustained release and continuous exposure of active CNP. In the trial, children treated with once-weekly TransCon CNP demonstrated annualized growth velocity (AGV) superior to placebo. TransCon CNP also demonstrated statistically significant improvements in other growth parameters, including height Z-score and change from baseline AGV. “Results of this pivotal trial demonstrate that once-weekly TransCon CNP can, if approved, address the need for an efficacious, safe, tolerable, and convenient treatment for children with achondroplasia,” said Ravi Savarirayan, M.D., at the Murdoch Children’s Research Center (Australia) and an investigator in the ApproaCH Trial. “I am excited about the possibilities TransCon CNP presents for a potential new treatment option for these children that will allow decreased injection frequency, improved compliance, and reduced caregiver burden.” “TransCon CNP-treated children with achondroplasia exceeded the growth rate of the general population, suggesting that once-weekly TransCon CNP provides catch-up growth with safety and tolerability results comparable to placebo with a low incidence of injection-site reactions,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “With SKYTROFA ® and TransCon CNP, Ascendis is uniquely positioned to become the leader in the treatment of skeletal dysplasias and growth disorders.” Highlights of the ApproaCH Trial Topline Data Primary Endpoint AGV Change from Baseline Secondary Endpoints Selected Other Endpoints Safety Summary As of today, all 82 children who completed the double-blind period are continuing in the open-label extension of the ApproaCH Trial. “On behalf of everyone at Ascendis, I want to thank the study participants, families, and clinical investigators contributing to the efforts to improve treatment options for individuals living with achondroplasia,” said Aimee Shu, M.D., Ascendis Pharma’s Senior Vice President of Clinical Development, Endocrine & Rare Disease. “These data support our aspirations for TransCon CNP, and we look forward to working with regulatory authorities as we take the next steps to advance TransCon CNP as a potential new treatment option.” Ascendis plans to submit a New Drug Application (NDA) to the U.S. Food & Drug Administration for TransCon CNP for the treatment of children with achondroplasia during the first quarter of 2025 and a Marketing Authorisation Application (MAA) for the treatment of children with achondroplasia to the European Medicines Agency during the third quarter of 2025. ApproaCH is a pivotal, multicenter, randomized, double-blind, placebo-controlled trial of once-weekly TransCon CNP versus placebo in 84 children with achondroplasia ages 2-11 years old. Patients were randomized 2:1 to receive TransCon CNP or placebo for 52 weeks at the 100μg/kg/week dose in the double-blind period, after which all participants could choose to receive TransCon CNP in an ongoing open-label extension at the 100μg/kg/week dose. A slide presentation with these data can be found on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com . Conference Call & Webcast Information Ascendis Pharma will host a conference call and webcast on Monday, September 16, 2024, at 8:00 am Eastern Time (ET) to discuss the pivotal ApproaCH Trial results. Those who would like to participate may access the live webcast here , or register in advance for the teleconference here . The link to the live webcast will also be available on the Ascendis Pharma Investors & News website at https://investors.ascendispharma.com. A replay of the webcast will be available on this section of the Ascendis Pharma website shortly after conclusion of the event for 30 days. About Ascendis Pharma A/S Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. SOURCE: Ascendis Pharma