BioMarin Pharmaceutical Inc. (NASDAQ: BMRN)
BioMarin Pharmaceutical stock is up today after releasing positive results with regard to the Phase 2 proof-of-concept and dose funding study of BMN 111, also known as vosoritide. Today, we’ll take a look at the results to see what investors are so excited about, talk about what we’ve seen in the market so far as a result, and discus what we can expect from BMRN moving forward. So, let’s get right to it.
BioMarin’s BMN Phase 2 Study Results
BMN 111 is an analog of C-type Natriuretic Peptide for the treatment of children with achondroplasia; the most common form of human dwarfism. In the Phase 2 study, 26 children participated. The study demonstrated a favorable safety profile and efficacy at a dose of 15 micrograms per kilogram per day. 10 children in Cohort 3 were treated with the 15 microgram per kilogram per day dose and had an increase of 50% in anualized growth velocity when compared to their prior 6 month natural history baseline growth velocity. Perhaps more importantly, BMN 111 proved to be safe. There were no serious adverse events; with the majority of adverse events reported being mild headache, hypotension, back pain and cough. Here’s what Wolfgang Dummer, M.D., Ph.D., Vice President of Clinical Development of BMRN had to say…
“We are very encouraged to have observed evidence of activity with vosoritide in children participating in our Phase 2 study…In children receiving the highest dose of 15 micrograms per kilogram daily, we observed a 50% increase in mean annualized growth velocity compared to their own natural history control growth velocity. This increase in growth velocity, if maintained, could allow children with achondroplasia to resume a normalized growth rate…More importantly, vosoritide was well tolerated in all dose cohorts and we have observed no major safety concerns to date. Based on these results, we intend to move into pivotal registration study discussions with health authorities with a does of 15 micrograms per kilogram daily. In addition, to support further exploration of a dose that may enable “catch-up” growth in the event of delayed treatment, we intend to study 30 micrograms per kilogram daily in ancillary studies. The next step in our development plan is to review this Phase 2 data with health authorities and our outside advisors to deelop our path forward with registration enabling studies.”
How BMRN Reacted In The Market
As we’ve come to expect any time positive study results are released, BMRN is having a great day in the market today. Currently (12:01), BioMarin Pharmaceutical stock is trading at $139.32 per share after a gain of 12.72% so far today.
What We Can Expect From BMRN Moving Forward
Moving forward, I’m expecting to see a continuation of the bullish activity we’ve seen today. The reality is that in the short term, investor excitement revolving around the result of the study is likely to continue the upward movement. So, looking for dips to take advantage of the climb may be advantageous. In the long run, I’m also expecting bullish activity from BMRN. The vosoritide treatment is proving to be not only effective, but safe; so, I’d imagine approval will come down the road. Also, with a clear plan, strong management, and a strong team, I’m expecting to see continued growth from the stock.
What Do You Think?
Where do you think BMRN is headed and why? Let us know in the comments below!