BioMarin on Wednesday said it’s wrapped up a pipeline review and will stop investing in four experimental therapies because they don’t meet its threshold for patient impact and commercial opportunity.
Two of the discontinued programs, BMN 355 for long-QT syndrome and BMN 365 for PKP2 arrhythmogenic cardiomyopathy, hadn’t yet been tested in humans. The other two, BMN 331 for hereditary angioedema and BMN 255 for a certain kind of liver disease, were in early stages of clinical testing, according to a BioMarin presentation for investors in September.
BioMarin said its full pipeline will be subject to “ongoing assessment.” But three programs have met its “highest bar for advancement” and will now be accelerated: BMN 333 for multiple growth disorders, BMN 349 for AATD-associated liver disease and BMN 351 for Duchenne muscular dystrophy.
BioMarin is revamping its research and development priorities as it struggles to win reimbursement for Roctavian, the first marketed gene therapy for the most common form of hemophilia. Once billed as a potential blockbuster, net revenue for Roctavian reached just $800,000 in the first quarter. Sales for all of last year came to $3.5 million, down from an earlier company estimate of as much as $150 million.
“The handwriting is on the wall regarding Roctavian,” Piper Sandler analyst Christopher Raymond said in a note to clients Wednesday. “We model this drug as a commercial flop with peak revenue of $50 million to $60 million, and increasingly view it as a distraction at best.”
The company expects to share more about the path forward for Roctavian at an investor day on Sept. 4, new CEO Alexander Hardy told analysts Wednesday. Sales could ramp up, allowing the company to stay the course. Other options include “right-sizing” BioMarin’s investment or possibly divesting the gene therapy, he said.
“Our focus from the R&D side is to allocate assets to the highest value and clearly, we have a high level of current Roctavian investment and continued challenges with commercial uptake,’’ Hardy said.
BioMarin’s shares dropped 11% in morning trading Thursday after the company released first quarter earnings and outlined its new plans for research. But analysts including Raymond said they are still buyers of the stock, pointing to the potential for further restructuring and the commercial success of another BioMarin therapy called Voxzogo for the bone growth condition achondroplasia.
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