At the recent JPMorgan Conference, biotech BioMarin Pharmaceutical (BMRN) offered a presentation that included important and new information regarding its R&D pipeline for 2019, observes biotech specialist John McCamant, editor of The Medical Technology Stock Letter.
ValRox, for severe hemophilia A gene therapy, continues to hold a leading position in the race for this potential blockbuster market. In line with the FDA guidance for gene therapy hemophilia drugs, the company has completed the enrollment in the Phase I/II ValRox trial/
The company expects to be able to file for accelerated approval by H2:19, should FVIII levels remain in the normal range, including (1) a comprehensive CMC package; (2) data from a Phase 3 cohort (~20- 30 patients) with normalized FVIII levels; (3) 3.5 years of data at time of filing from Ph1/2 (6e13 dose).
The total market size estimated at an enormous $30-90 billion global market opportunity, based on ~117,000 patients worldwide. The company will decide by H2 to file, and it has become one of the leading manufacturers of gene therapy and that will add to its competitive advantages.
If all goes well, ValRox could be on the market by next year. The company is working out pricing and payment systems, with a minimum price of $1 million, and working closely with payors to possibly devise a pay over time strategy.
The next key pipeline compound remains vosorotide. Vosoritide in achondroplasia data is expected by yearend 2019. The Phase II was very positive given evidence of sustained growth demonstrated (~5.7cm of additional height gain over 42 months of treatment).
The submission package will also include safety data from an ongoing Phase II trial in infants and toddlers with achondroplasia (0-5 years old; as vosoritide was generally well-tolerated in early dosing), and further data from the Natural History to Assess Final Adult Height study.
Additionally, vosoritide, an analogue of C-natriuretic peptide (CNP, a central player in regulation of skeletal growth in humans), could lead to broad label expansion down the road (e.g., in idiopathic short stature (~100,000 patients in the US).
Hence a lot of steady positive progress at Biomarin, with novel leadership compounds in rare diseases and a widening gene therapy presence. In our view, the company is and has been an ideal takeover target and feels appropriately sized for the current M&A environment.
