BioMarin Pharmaceutical (BMRN) investor relations material

Financial performance and Amicus

• Q3 revenue grew 32% and reached $1B for the first time; Metabolic Conditions revenue grew 44%.

• Amicus added GALAFOLD for Fabry and POMBILITI/OPFOLDA for Pompe; GALAFOLD patients in one market grew 20% YoY and POMBILITI/OPFOLDA revenue grew nearly 60%.

• Identified ~$220M in non-GAAP operating expense synergies, expected to be fully realized for a full year in 2028; integration expected to be mostly complete by mid-2027.

• Amicus business targeted to reach ~60% operating margin by decade-end; two Q3 acquisitions also drove some in-process R&D expense.

Pipeline development

• Four assets added this year: BMN 450, BMN 440, preclinical BMN 430 for ReNU syndrome, and partnered BMN 820 for FSGS, with U.S. rights.

• BMN 440 is an oral ENPP1 inhibitor designed to reduce inorganic pyrophosphate production; development will address distinct HPP populations and endpoints in Phase II.

• BMN 440 Phase I/II study is ongoing; data expected next year will include healthy volunteers and patients, single and multiple dosing, PK, and pyrophosphate markers.

• A 30%-50% reduction in inorganic pyrophosphate is viewed as a target range; U.S. estimates cited 9,000 diagnosed HPP patients, with ~10%-30% on treatment.

• BMN 450 is a subcutaneous Fabry enzyme replacement therapy designed to be mutation-agnostic; early patient studies are in dose escalation, with a low double-digit number of patients evaluated.

Clinical programs and commercial outlook

• BMN 333 Phase II tests three doses in 30 patients, including 10 on VOXZOGO as an internal control; Phase II data expected in 2027, with global recruitment underway.

• BMN 333 showed up to eightfold or greater free CNP AUC versus YUVIWEL in Phase II; healthy-volunteer cyclic GMP rose two- to fivefold at higher exposure levels.

• BMN 820 is an oral CCR2 inhibitor studied on top of angiotensin receptor blockade; its fully enrolled Phase III passed futility analysis, with results expected in 2028 after two full years of follow-up.

• Hypochondroplasia has 14,000 estimated addressable patients in markets, assuming diagnosis; the PDUFA date is May 30, 2027, and any launch would be mid-year, with 2027 guidance still under review.