BioMarin quarter net loss reduced. Plans to expand gene therapy for hemophilia A

BioMarin Pharmaceutical said Wednesday it plans to expand its development plan for BMN 270, its investigational gene therapy for hemophilia A, to include an additional Phase 3 study of the 4e13 vg/kg dose from its ongoing open-label Phase 1/2 study of BMN 270.  

BioMarin said it plans to initiate two separate Phase 3 studies as soon as possible, one with the 4e13 vg/kg dose and one with the 6e13 vg/kg dose.  It has also started authorized its commercial gene therapy manufacturing facility and expects to start the Phase 3 program in the fourth quarter of 2017.

BioMarin said that a total of six patients received a single dose of BMN 270 at the 4e13 vg/kg dose. Based on the most recent data, for the three patients who were given the 4e13 vg/kg dose in November/December 2016, at week 32, all are in or near to the normal range of Factor VIII activity levels, with both median and mean Factor VIII levels of 51%.

All six patients who received a single dose of BMN 270 at 4e13 vg/kg dose had severe hemophilia A and had been treated with prophylactic Factor VIII pre-study.  After receiving BMN 270 and then reaching a Factor VIII activity level above 5%, through the July 28th data cut, the mean Annualized Bleed Rate (ABR) was reduced by 92% from 12.2 to 1.0.  The median ABR for those same patients was reduced from 8.0 to zero.  The mean annualized Factor VIII infusions were reduced by 97% from 144.2 to 4.8.  The median annualized Factor VIII infusions were reduced from 155.5 to zero.

“With the additional eight weeks of data for BMN 270 at the 4e13 vg/kg dose, we now plan to move forward as rapidly as possible with two separate Phase 3 studies with the 4e13 vg/kg and the 6e13 vg/kg doses.  By concurrently moving both of these doses into Phase 3 development, we have the opportunity to determine if patients may be better served by having one or both of these doses commercially available. BioMarin is well positioned with the resources and manufacturing capacity to undertake the studies necessary to explore both doses to determine the better potential therapeutic option,” said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin.

Fuchs said that he expects that around 90% of patients would be treatment candidates for BMN 270 based on that criteria.

The company also reported record total revenues of $317.4 million in the Second Quarter of 2017
with net loss reduced to $36.8 million. Non-GAAP income was $26.6 million in the second quarter of 2017