A slimmed-down BioMarin started the new year with a bang, hitting the annual J.P. Morgan Healthcare Conference with the goal of tapping its skillset to strike deals that bolster its pipeline of medicines for genetic diseases.
“The challenging financing environment for biotech combined with BioMarin’s resources and global capabilities in research and development, manufacturing and commercialization puts us in a strong position,” Gregory Friberg, M.D., BioMarin’s chief R&D officer, told Fierce Biotech in an interview.
BioMarin's resources did not go unnoticed by the deal-hungry biotech community.
“We had 155 meetings at J.P. Morgan,” CEO Alexander Hardy said on the company’s Feb. 19 earnings call. “We're now in the process of looking at these various assets,” he said, which range from preclinical to clinical.
While he wouldn't detail specific modalities BioMarin was seeking, Friberg said that, in general, the company's products should be engineered to address genetic disease in a measurable way and should carry a large therapeutic effect and high societal value.
The R&D head is looking for "clever science," which Friberg said is readily apparent in BioMarin’s recent research pact with CAMP4 Therapeutics. The partners are using CAMP4’s regulatory RNA platform to go after two undisclosed genetic disease targets.
The deal is a good example of the kind of preclinical collaborations BioMarin is after, Friberg said—an off-the-shelf, genetically targeting therapy with “a clever use of science to accomplish something that perhaps prior generations weren't.” In this case, the goal is to up-regulate certain proteins.
The search for partnerships and external assets is a part of BioMarin’s bold plan to increase annual revenue to $4 billion in 2027. The California company’s 2024 figure was $2.85 billion, an 18% increase from 2023.
Pursuit of that revenue boost is also what led BioMarin to shake up its corporate structure last year, separating into three business units—skeletal conditions, enzyme replacement therapies and the gene therapy Roctavian—while also laying off 7% of its global workforce.
Friberg joined the firm amid this major shift, alongside Chief Business Officer James Sabry, M.D., Ph.D.
Within its new business units, BioMarin is doubling down on areas in which it has already seen success, like achondroplasia, the most common form of dwarfism, and phenylketonuria (PKU), a rare disorder that stops the body from being able to break down the amino acid phenylalanine. Two of the biotech’s early-stage assets are in these indications, even though the company already has approved drugs for each—Voxzogo (vosoritide) for achondroplasia and Palynziq (pegvaliase) for PKU.
“We feel that the products we have, while they're safe and effective, helping a lot of patients, we feel that we can improve on them,” Friberg said.
In achondroplasia, for example, BMN 333 recently began dosing for a first-in-human study, according to BioMarin's earnings report. Like Voxzogo, BMN 333 is a C-type natriuretic peptide, a type of hormone that promotes bone growth, Friberg said. But this next-generation molecule has a half-life extension technology added onto it, he said, with the goal of creating a drug that can be taken less frequently and has greater exposure at the growth plates of children with achondroplasia.
With PKU prospect BMN 390, BioMarin is swapping out the polyethylene glycol of Palynziq for a long polymer with the fittingly lengthy name of poly(oligo(ethylene glycol) methyl ether methacrylate), or POEGMA. The hope is that using POEGMA will reduce immune reactions compared to Palynziq, Friberg said, and the compound’s lower viscosity should also improve the experience of patients injecting it into themselves.
Redoubling efforts in these indications is in part due to a recent rise in competition. For example, Ascendis’ dwarfism drug recently reported phase 3 data and poses a threat to Voxzogo because it is injected once a week, compared to Voxzogo's once-a-day doses. Meanwhile, Japanese pharma Otsuka recently picked up a clinical-stage PKU asset from Jnana Therapeutics that's taken orally rather than through subcutaneous injection like Palynziq.
Palynziq was first approved in 2018 and Voxzogo in 2021. At the time, BioMarin “really built itself on molecules in areas where there were no other therapies available,” Friberg said. “We were really the only show in town.”
But the landscape has changed since then. “I like to say that good ideas don't stay lonely for very long,” he said.
Good ideas have to start somewhere
Friberg recognizes that many of biotech’s good ideas start at research agencies like the National Institutes of Health (NIH)—and that weakening the NIH could seriously halt the progress of innovative new medicines.
“I'm a card-carrying medical oncologist by background,” Friberg said. “I got to really be on the front lines to see the value of what NIH funded research could accomplish. The clinical trial network in the United States is heavily dependent on that funding.”
As President Donald Trump and the Department of Government Efficiency headed up by Elon Musk take aim at federal health agencies like the NIH, the FDA and the Centers for Disease Control and Prevention, including mass layoffs and attempts to pause grant funding, Friberg pointed to a statement made by his professional society, the American Association for Cancer Research (AACR).
“The AACR is deeply concerned that the administration’s recent actions are threatening the NIH and its lifesaving mission to accelerate progress for patients with cancer and the hundreds of other diseases that afflict millions of Americans,” the statement reads. Mass firings, funding cuts and work disruptions are “jeopardizing progress and delaying innovations and treatments that are pivotal to improving patient outcomes and saving lives.”
Though BioMarin is a global organization, Friberg said, the U.S. remains a vital source of innovative research that pharma companies ultimately turn into medicines.
“We're hoping that pipeline of good ideas, which often comes from NIH-funded research, doesn't dry out,” he added. “It would be a shame for the United States to take a step back from being the leader in that space.”
Agencies like the NIH and the FDA also support research and drug development for rare diseases, which includes genetic conditions like achondroplasia and PKU that primarily first appear in childhood.
"These are children with life-threatening, life-changing conditions,” Friberg said. “We're hopeful again that there are no steps back taken.”
Asked whether the administration’s changes to federal health agencies have had any effect on BioMarin’s business strategy or whether the company is planning to take any action in response, Friberg declined to answer.
“I really can't comment at this point,” he said.