BioMarin Pharmaceutical (NASDAQ:BMRN) outlined its growth strategy, integration plans for Amicus Therapeutics and pipeline priorities during a Canaccord discussion with Chief Financial Officer Brian Mueller.
Mueller described BioMarin as one of the largest rare-disease-focused biopharmaceutical companies, with more than 25 years of operating history, six first-in-disease medicines and nine approved therapies. The company operates in more than 80 countries and expects revenue approaching $4 billion this year, he said.
Quarterly Performance and Amicus Integration
BioMarin reported nearly $1 billion in second-quarter revenue, representing 20% year-over-year growth, Mueller said. The company raised total-revenue guidance to a range of $3.875 billion to $3.925 billion and raised non-GAAP earnings-per-share guidance to $4.90 to $5.10.
During the quarter, BioMarin completed its acquisition of Amicus Therapeutics, adding two commercial rare-disease products: Galafold for Fabry disease and Pombiliti and Opfolda for Pompe disease. Mueller characterized the products as high-growth assets that can benefit from BioMarin’s larger global infrastructure.
BioMarin estimates peak revenue of $1.4 billion for Galafold and $1.2 billion for Pombiliti and Opfolda, with those opportunities expected in the mid- to late 2030s, respectively. The company also expects to realize synergies equal to roughly 50% of Amicus’ legacy operating-cost base, primarily through general and administrative expenses and other support functions.
Mueller said BioMarin is preserving and investing in the acquired sales and marketing organization rather than pursuing aggressive commercial cost reductions. “These products are in their high-growth phase,” he said, adding that maintaining customer-facing capabilities is important to sustaining growth.
BioMarin originally targeted a leverage ratio below 2.5 times within two years of the acquisition’s closing. Following its assessment of revenue opportunities, operating synergies and the combined company’s EBITDA profile, management now expects to reach that target by the middle of next year, roughly a year earlier than originally planned.
The company raised approximately $3.6 billion in debt for the transaction and also has a $600 million convertible note maturing next May, Mueller said. By 2030, BioMarin expects the Amicus business to operate at an approximately 60% operating margin.
Commercial Opportunities for Galafold and Pompe Franchise
Mueller cited geographic expansion as a major growth driver for the acquired portfolio. Amicus had commercialized Galafold in roughly 40 markets and Pombiliti in approximately 15 markets, compared with BioMarin’s presence in more than 80 countries.
BioMarin does not expect to bring both medicines to every market where it operates, but Mueller said its on-the-ground commercial, market-access, medical and regulatory teams could accelerate international expansion in complex markets.
For Galafold, BioMarin sees an opportunity to improve diagnosis of Fabry disease, which Mueller said remains significantly underdiagnosed. The company plans to use efforts including AI-enabled patient identification, newborn screening and familial genetic cascade screening.
For Pombiliti and Opfolda, BioMarin’s strategy includes encouraging treatment switches from existing enzyme therapies. Mueller said there is an emerging body of real-world evidence suggesting improved outcomes with Pombiliti and Opfolda, while the efficacy of some existing therapies can wane over time.
Pipeline, Business Development and VOXZOGO
BioMarin’s business-development focus remains centered on rare, genetically defined diseases. The company plans to prioritize opportunities that can use its internal manufacturing, development, regulatory, clinical and commercial infrastructure, Mueller said.
Management will continue to pursue early-stage collaborations and in-licensing opportunities, including a recently announced collaboration with the n-Lorem Foundation for a rare neurologic disease. As BioMarin reduces leverage, it may consider larger mid- to late-stage opportunities, though Mueller did not provide specifics on deal size or timing.
The company also raised revenue guidance for VOXZOGO to at least $1 billion after what Mueller described as strong first-half performance. VOXZOGO revenue grew 14% in the first quarter, and BioMarin added 20% new patients globally, he said.
VOXZOGO is approved for achondroplasia, and BioMarin has submitted a supplemental new drug application to the FDA for hypochondroplasia following positive Phase III results. If the filing is accepted, a potential approval and launch could occur next year, according to Mueller.
BioMarin estimates there are about 24,000 achondroplasia patients worldwide and approximately 14,000 hypochondroplasia patients. Mueller said diagnosis will be a key challenge in hypochondroplasia, but the company believes its established VOXZOGO presence in achondroplasia provides a foundation for the potential label expansion.
Separately, Mueller highlighted BMN 820, formerly DMX-200 at Amicus, as a potentially meaningful development asset. The small-molecule treatment is being studied for focal segmental glomerulosclerosis, or FSGS, a kidney disorder. BioMarin holds U.S. rights, estimates a U.S. population of roughly 30,000 patients and noted that the FDA agreed to proteinuria as the primary endpoint in the fully enrolled two-year study.
About BioMarin Pharmaceutical (NASDAQ:BMRN)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
