.Ascendis Pharma has emerged as a prospective risk to BioMarin’s Voxzogo, disclosing stage 3 development disorder data that went beyond expert expectations as well as set up the biotech to declare commendation next year.Copenhagen-based Ascendis tailored the test to compare its once-weekly prodrug of C-type natriuretic peptide (CNP) to sugar pill in 84 kids with the dwarfism shape achondroplasia. Providing little ones along with continual exposure to CNP may increase growth. BioMarin serves that requirement with its own CNP analog Voxzogo– the main feature of its own development technique– yet that medication requires to be administered once a day.Ascendis’ TransCon CNP could challenge Voxzogo.
In the phase 3 research study, the biotech saw an annualized growth velocity (AGV) of 5.89 cm/year in the 57 children who obtained TransCon CNP compared to 4.41 cm/year in the 27-subject inactive drug upper arm. The distinction was actually statistically considerable, inducing the trial to attack its own primary endpoint. The study enlisted little ones grown older 2 to 11 years.
Attendees in BioMarin’s research study ranged in age coming from 5 to 14 years. While that distinction makes complex cross-trial comparisons, Ascendis featured subgroup data on individuals aged 5 to 11 years. Ascendis CEO Jan Mikkelsen claimed he thinks cross-trial comparison is actually “fairly legitimate” on a phone call with real estate investors to cover the data.Placebo-adjusted AGV was 1.78 cm/year in the subgroup.
While BioMarin’s phase 3 test featured older little ones, and also mentioned a placebo-adjusted AGV of 1.13 cm/year, Evercore ISI professional Cory Kasimov pointed out the number for Voxzogo in little ones aged 5 to 11 years was actually 1.74 cm/year. The contrast sustains the end of Gavin Clark-Gartner, Kasimov’s Evercore co-worker, that the drugs’ efficacy remains in the very same ball park.Ascendis’ portion rate went 17% to practically $140 back the data decrease. Meanwhile, BioMarin’s stock dropped just about 18% to below $70.
However, Kasimov and also his staff continue to believe in BioMarin’s vision for its own CNP analog.” Our sight on Voxzogo’s positioning stays [unmodified], and also we still think our style is traditional,” Kasimov stated. “The item will certainly contend minimum a 4+ year head start in the USA (likewise accepted in 44 nations complete), has a robust safety and security account, will certainly possess the 0- to 2-year-old segment (a minimum of initially), as well as is being actually analyzed in 5 other skeletal signs,” they claimed in a note.Ascendis considers to apply for FDA commendation in the first fourth of 2025 and also submit the documentation in Europe in the 3rd quarter of that year. The biotech experienced concerns when obtaining approval of its own hypoparathyroidism drug, acquiring a full response letter and encountering a three-month problem when it refiled, however Mikkelsen said the team has actually gained from the adventure and TransCon CNP is actually “so much more straightforward.”.