- In phase II trials, 26 children with achondroplasia taking BMN 111 (vosoritide) had a mean increase of 50% in annualized growth velocity.
- BioMarin researcher Henry Fuchs presented preclinical data on this drug only three years ago.
- BMN 111 is designed to correct the genetic mutation that drives dwarfism.
It's been a very productive three years for BioMarin, as the company's researchers have successfully demonstrated proof-of-concept for BMN 111 in children with dwarfism. However, despite the fact that the drug improves growth velocity, it does not improve "proportionality."
Nonetheless, phase II studies are currently being designed, with different dosing protocols. And although achondroplasia affects only a few thousand people in the United States, Leerink analyst Joseph Schwartz has predicted a potential $1 billion return for BioMarin.
Not only is BioMarin's stock up on the news, with a market cap of close to $20 billion, but the company is showing the industry what it's like to develop a drug under an efficient timeline.