BioMarin shines as dwarfism drug boosts growth in children

Henry Fuchs

Three years ago, BioMarin top scientist Henry Fuchs pitched BMN-111 as one of the company's most promising early-stage drug prospects, with stellar mouse data to suggest that it was on track to develop a therapy to correct the genetic mutation behind dwarfism. On Wednesday evening, the San Rafael, CA-based biotech ($BMRN) outlined Phase II data from 26 children that provided ample proof-of-concept human data that Fuchs was well justified in his belief.

In particular, the 10 children with achondroplasia taking a 15 micrograms per kilogram per day dose of BMN-111 (or vosoritide) demonstrated a mean increase of 50% in what investigator termed their "annualized growth velocity compared to their annualized prior 6 month natural history baseline growth velocity."

In short, the drug put them back on track to a more normal growth rate. And it did it without triggering any serious safety concerns. The main drawback: There was no improvement in "proportionality." 

Execs for the company went on to tell analysts that they were on a direct path to mounting a pivotal study that could be used for an FDA and EMA approval, allowing Fuchs a clear shot at keeping his promise to handily beat the industry's often lamented record of taking a decade to develop a drug.

"These data offer strong evidence of the drug's efficacy, and importantly incrementally de-risk the vosoritide program," noted Barclay's Geoff Meacham. Leerink analyst Joseph Schwartz has boosted BioMarin's chances of success at hitting the market in three years with a drug that could earn a blockbuster $1 billion return. Only a few thousand people have achondroplasia in the U.S., but like other rare disease drugs, a treatment like this could be marketed for several hundred thousand dollars a year.

BioMarin's stock surged about 8%, an impressive achievement for a company with a market cap near $20 billion.

In addition to the Phase III study now on the drawing board, BioMarin is also setting up a 30 μg/kg/day-dose trial to see if a much higher dose can correct dwarfism.

The trial results will likely trigger a fresh round of takeover rumors. BioMarin has emerged as a leading biotech in the rare disease arena, with the kind of assets that have inspired some hefty bidding on the part of Big Pharma.

- here's the release