BioMarin actively looking for gene therapy and orphan disease assets, CEO says
11 MAY 2018
BioMarin Pharmaceutical [NASDAQ:BMRN], a major rare disease drugmaker, is actively scouring the landscape for promising gene therapy companies to acquire, according to Jean-Jacques Bienaimé, chairman and CEO.
The San Rafael, California-based drugmaker is already in late stage clinical trials for gene therapy treatment for a genetic condition called PKU, but the company is looking at the broader gene therapy and orphan drug market for additional treatments.
“Although we don’t want to turn into a pure gene therapy company, we’re looking at different opportunities in the gene therapy field,” he said this week on the sidelines of the Financial Times US Healthcare and Life Sciences Summit in New York.
BioMarin does not have a “real need” to do a large transaction, Bienaimé cautioned, but said it could afford up to USD 2bn. The company reported cash and short term investments of nearly USD 14bn as of end-2017.
It’s possible that Biomarin could make a licensing or acquisition deal in the next year but it would be need to be the “right asset at the right price,” he said.
The company is also looking for assets in other areas of orphan disorders, as well as potential treatments for central nervous system disorders, Bienaimé noted.
In terms of a potential rare disease expansion, the company is looking for “somewhat larger indications,” he said, explaining that it’s difficult to get a return on investment for products with very small patient populations.
“There are a lot of little gene therapy companies but a lot of them are going after very small indications and we don’t believe there is an economic argument to acquire them,” said Bienaimé.
BioMarin is one of the largest rare-disease drugmakers with a recent market cap of USD 15.49bn. The company is known for its phenylketonuria (PKU) treatment Kuvan and Morquio syndrome drug Vimizim, its two best-selling products in 1Q18.
In recent years, BioMarin has been reported as a possible takeover target for a number of large pharma players including Gilead Sciences [NASDAQ: GILD], Amgen [NASDAQ: AMGN] and Merck [NYSE: MRK].
BioMarin’s most recent large acquisition was that of Netherlands-based Prosensa Holding, which it agreed to buy in 2014 for about USD 680m, with an additional USD 160m in milestone payments. The acquisition gave BioMarin new capabilities in treating a rare disease called Duchenne muscular distrophy.
Gene therapy is emerging as one hottest areas in drug development. Novartis [NYSE: NVS] acquired gene therapy biotech, AveXis, in April for USD 8.7 bn. In 2016, Pfizer [NYSE:PFE] grew its business in that field with a takeover of Bamboo Therapeutics for up to USD 645m, netting its experimental mini-dystrophin drug.
FDA commissioner Scott Gottlieb said earlier this year that he believes gene therapy will eventually become a “mainstay therapeutic approach” in treating inherited genetic diseases.
BioMarin is currently developing a gene therapy for hemophilia A, known as valoctocogene roxaparvovec. Both Spark Therapeutics [NASDAQ:ONCE] and Sangamo Therapeutics [NASDAQ:SGMO] are also developing gene therapies or hemophilia A.
In the company’s 4Q17 earnings call, Bienaimé said that the company is interested in earlier stage opportunities and said it may announce one to two new pipeline projects before the end of the year.