BioMarin Pharmaceutical Inc. (NASDAQ: BMRN)
Shares of BioMarin Pharmaceutical Inc are climbing today after the company announced the results of its Phase 2 study into proof-of-concept and dosing of the experimental drug vosoritide (BMN 111). The results of the study were overwhelmingly positive, sending the stock into skyrocket like activity. So today, we’ll talk about the results fo the study, how the market reacted to the results, and what we can expect to see from BMRN in the market ahead.
Vosoritide Study Results Were Overwhelmingly Positive
Vosoritide, also known as BMN 111, is designed for the treatment of children that suffer from achondroplasia. The treatment is an analog of C-type Natriuretic Peptide and has proven to be effective in the treatment of the most common form of human dwarfism. The Phase 2 study aimed at proving the concept behind the drug, and demonstrating high tolerability in those who use it; and the results proved just that. In the study, 26 children participated with 10 of them being treated with a 15 microgram per kilogram dose. Throughout the treatment, the children showed an average increase of about 50% in anualized mean growth velocity in comparison to their natural growth history over the 6 months prior. Another key factor to this data release is that there were no series side effects noted in the study; although, some of the children did report mild adverse effects; including hypotension, back pain, cough and headache. In the announcement, BioMarin Pharmaceutical’s Vice President, Dr. Wolfgang Dummer had the following to say…
“We are very encouraged to have observed evidence of activity with vosoritide in children participating in our Phase 2 study,” He went on to say “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 velocity, if maintained, could allow children with achondroplasia to resume a normalized growth rate,” Dr. Wolfgang Dummer then moved to a discussion with regard to adverse events; having the following to say… “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 develop our path forward with registration and enabling studies.”
What We’ve Seen From BMRN In The Market Today
Any time a biotech company releases positive data about a recent study, we tend to see positivity in the market follow. That’s exactly what we’ve seen from BMRN today as the news caused massive growth. Currently (3:08), the stock is trading at $137.72 per share after a gain of 11.42%.
What We Can Expect To See Moving Forward
The news we received today is likely to lead to more gains in both the short and long term. In the short term investors are likely to maintain a level of excitement; driving the stock up. There may be pull backs here and there, but I’d imagine we’ll see uptrends overall. In the long run, I think BMRN is on to something that’s going to really generate growth. With vosoritide possibly having the ability to sustain normalized growth, this is a very big deal that’s likely to become incredibly profitable in the long run. So, look for pull backs for buying opportunities as this is likely to turn into long run gains!
What Do You Think?
Where do you think BMRN is headed and why? Let us know in the comments below!