June 17, 2016
By Mark Terry, BioSpace.com Breaking News Staff
San Rafael, California - BioMarin Pharmaceutical recently indicated on its website that it has temporarily suspended recruitment for its hemophilia A trial. Company took a little hit, but seems to have recovered, probably for several reasons, including a rumor that Paris-based Sanofi might buy the company if its pursuit of Medivation falls through. Shares are currently trading for $82.68.
The suspension is not really new and probably isn’t a major concern. The company had already indicated before that it planned to discuss the trial with EU regulators prior to dosing the last three patients in the gene therapy trial.
“The discussion with regulators prior to dosing the last patients was meant to be an early safety precaution and was self-imposed as part of BioMarin’s protocol for a first-in-human hemophilia A gene therapy,” wrote Evercore in a note to investors. It expects to dose those remaining patients by August and have the full 16-week data on all 12 patients by the end of the year.
In April, BioMarin reported preliminary data from the Phase I/II trial of BMN 270 that were quite positive. “If BMN 270 allows hemophilia A patients to maintain around 5 percent of normal levels of Factor VIII, it could have a real and meaningful clinical benefit by reducing the need for Factor VIII infusions and spontaneous bleeds,” said John Pasi, professor of Haemostasis and Thrombosis at Barts and the London School of Medicine and Dentistry, and primary investigator for the trial, in a statement. “I am looking forward to further assessing the data over the 16 weeks and beyond in this ongoing study.”
BMN 270 is a gene therapy treatment, in which a functional copy of the factor VIII gene is delivered to patients’ cells, using an adeno-associated virus (AAV) as a vector.
There were also rumors today that Sanofi, which has been in the process of a hostile takeover bid of Medivation, may turn toward BioMarin if its Medivation bid fails.
Although that may have made the stock go up, the company has had other problems. On June 1, it announced it was abandoning its Kyndrisa (drisapersen) program for Duchenne Muscular Dystrophy (DMD). It was rejected in January. It is also abandoning its European application for the drug, and killing its programs for three other first-generation follow-on drugs, BMN 044, BMN 045, and BMN 053, all of which were in Phase II trials for DMD.
The company has a number of other promising prospects in its pipeline, though, including BMN 190 (cerliponase alfa) for a rare neurodegenerative disease, CLN2, its phenylketonuria candidate pegvaliase, and, of course, BMN 270, which Leerink analysts have said “could be transformational.”
TheStreet Ratings team gave the company stock a “sell” rating with a score of D+. It wrote, “BioMarin’s weaknesses include its feeble growth in its earnings per share, deteriorating net income, disappointing return on equity, weak operating cash flow and generally disappointing historical performance in the stock itself.”