BioMarin leaps on positive mid-stage vosoritide results

Shares of US biotech firm BioMarin Pharmaceutical (Nasdaq: BMRN) jumped 12.8% to $138.76 in morning trading, after the company released positive mid-stage trial results for its dwarfism drug candidate.

The company announced Phase II proof-of-concept and dose finding study of BMN 111 (vosoritide), an analog of C-type natriuretic peptide (CNP), in children with achondroplasia. Achondroplasia is the most common form of human dwarfism. Vosoritide has Orphan designation in both the USA and Europe.

Data from the 26 children participating in the Phase II study demonstrated a favorable safety profile and efficacy at the 15 micrograms/kilogram/daily dose. The 10 children in Cohort 3 treated with 15 micrograms per kilogram per day had a mean increase of 50% (p-value = 0.01) in their annualized growth velocity compared to their annualized prior six month natural history baseline growth velocity. Changes from baseline in proportionality as measured by upper to lower body ratio were not observed. No Serious Adverse Events (SAEs) were observed for the duration of the study. The complete data from the study will be presented at a medical meeting later in the year.