Sabry Lays Out BioMarin’s New Business Development Plan

BioMarin chief business officer James Sabry is known as one of the industry’s most prolific dealmakers, having joined the rare disease specialist last year from Roche, where he led pharma partnering. His jump to BioMarin, under a new leadership team helmed by former Genentech CEO Alexander Hardy, signaled the company is moving into a more active period of business development.

Sabry talked to Scrip at the J.P. Morgan Healthcare Conference about BioMarin’s plans to kickstart business development as the company evolves under new management.

“It’s this wonderful sense of all coming together at this new company to work together to build BioMarin,” Sabry said. In addition to Hardy and Sabry, BioMarin’s new leadership includes chief commercial officer Cristin Hubbard, formerly head of global pharma product strategy at Roche, and new chief R&D officer Greg Friberg, who led rare disease medical affairs at Amgen.

Sabry had been looking for an opportunity to return to a smaller pharma after spending 14 years at Roche.

“I always knew I wanted to come back into smaller companies, but I didn’t want to go back into raw start-ups the way I had before, so I looked carefully at a large number of medium-sized companies and BioMarin, by far, was the one that was most intriguing to me, not only because of the people, but also because of the science and business of what it was,” Sabry said.

Focused on rare genetic diseases, BioMarin has built, over 20 years, a portfolio of enzyme replacement therapies for rare diseases like Aldurazyme (laronidase) for mucopolysaccharidosis I (MPS I) and Naglazyme (galsulfase) for MPS VI. More recently, the company launched Voxzogo (vosoritide) for skeletal disorders including achondroplasia, which has been a big new growth driver, and the hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec), which has been less commercially successful.

For 18 years, BioMarin was led by CEO Jean-Jacques Bienaimé, who stepped down last year after the company finally achieved profitability for the first time. On the strength of Voxzogo, the company’s on solid financial footing with revenues in the first nine months of 2024 up 19% to $2.11bn and diluted earnings per share of $1.56 up 103% year-over-year. However, some investors are worried about BioMarin’s longer-term growth and late-stage pipeline, which is largely focused on expansion of Voxzogo to new indications, but competition is looming.

Business development could allay some investor concerns about the mid- to late-stage pipeline.

Deals Will Get Signed This Year

BioMarin is poised to finalize deals this year, Sabry said. They will range across the spectrum from early-stage research to late-stage clinical projects.

“There’s a huge number of possible deals we could do,” Sabry said. “The real challenge for us is not going to be whether to do a deal, but which deals to do and how are they structured.”

The focus will be on genetically defined medicines, which will include rare diseases, BioMarin’s traditional area of focus, but it won’t exclude diseases outside of rare.

“Rare disease is a prevalence base. What defines the kinds of things we’re interested in is the fact that there’s a genetic cause of the disease that we are going to mitigate in order to treat the disease,” Sabry said.

“This is a huge, huge opportunity, I think, in the industry, and one for which BioMarin is really well poised to continue to lead in,” he added.

The main priority is to bring in new products that could launch later in the decade or early in the 2030s. Hardy has told investors the company is interested in deals with a value of around $1.5bn.

“That’s a good sort of guide as to the kinds of things that we’re looking at,” Sabry said, confirming the company could complete multiple deals in that range.

One area BioMarin is not immediately looking to expand further in is gene therapy, where the company’s launch of Roctavian has struggled to get off the ground.

“We’re definitely focused away from AAV gene therapy,” Sabry said. “We’re excited about Roctavian. It’s a fantastic medicine, but there are some real challenges, in my opinion, with AAV-based gene therapy.”

BioMarin is well positioned to be a partner to smaller biopharmas, Sabry said, because of its infrastructure that reaches all the way from research through commercial in 80 countries and an experienced internal development organization.

“We are a fully integrated company all the way from invention to market in this area of genetically defined diseases,” he said.

As for how the business development environment is shaping up for biopharma in the year ahead, Sabry said the environment remains challenging. Some of that, he said, is driven by insecurity around a new US presidential administration and policy, as well as uncertainty about interest rates and the financing environment.

He wrapped up the J.P. Morgan meeting feeling “relatively cautious” on dealmaking, he said, though pharma companies, including BioMarin, are eager to do deals.