BioMarin Pharmaceutical Inc. Q2 FY2025 earnings call

Strong Growth: Achieved double-digit year-over-year revenue growth and significant profitability expansion in Q2. ### Pipeline Progress: BMN 333, a long-acting therapy for children with achondroplasia, achieved its target profile and plans to advance to a registrational Phase II/III study in H1 2026. The acquisition of Inozyme on July 1 broadened the enzyme therapies portfolio, with BMN 401 (formerly INZ-701) expected to have pivotal data readout in H1 2026. ### Commercial Activities: VOXZOGO had 20% YOY revenue growth in Q2, with strong global expansion. Work ongoing on hypochondroplasia indication for VOXZOGO, aiming for Phase III data in H1 2026 and potential launch in 2027. Enzyme Therapies business unit saw 15% YOY growth in Q2, with PALYNZIQ and VIMIZIM performing strongly. ### R&D Updates: BMN 333 Phase I study ongoing with safety and pharmacokinetics monitoring. Plan to submit applications for age extension of PALYNZIQ to include adolescents in H2 2025. BMN 401 in Phase III for ENPP1 Deficiency, with pivotal data in H1 2026.

In the second quarter, VOXZOGO revenue increased 20% year-over-year to $221 million. Enzyme Therapies revenue rose 15% year-over-year to $555 million, with PALYNZIQ marking 2 consecutive quarters of 20% growth and VIMIZIM increasing 21% year-over-year. ROCTAVIAN revenue was $9 million in the second quarter. Total revenues grew 16% in the quarter and 15% in the first half of 2025 compared to the same period in 2024.

Full year 2025 total revenue guidance raised to a lower end of $3.125 billion. Non-GAAP operating margin guidance raised to between 33% and 34%. Non-GAAP earnings per share guidance raised to between $4.40 and $4.55. The Inozyme acquisition's IPR&D expense will impact Q3 2025 financial results, and full year guidance will be updated when Q3 is reported.

Q: Congratulations on the quarter. I had a couple of questions on BMN 333. I just wanted to clarify that the competing program you're referring to is TransCon CNP relates to the comparison to AUC data. And then as it relates to the data itself, can you just confirm the safety profile that you're targeting and whether or not increased exposure is running into any increased risk of hypotension or other side effects?

A: So Paul, this is Greg Friberg. I can confirm that the agent you're referring to is in that reference that we commented there. With regard to the profile we're seeing with 333 from a safety standpoint, absolutely nothing unexpected, though we have to remember this is a healthy volunteer study. It's in adults, not in children. That being said, nothing unexpected. And again, we also look at the genetic data in this disease, where -- from the standpoint of patients who either have inborn mutations that cause them to have high CNP levels or activated receptors, nature has shown us that those patients, the only problems that they tend to have in their lifetime is that, number one, they're quite tall and there are skeletal complications of being over 7 feet tall. But reassuringly, those patients don't have challenges in other organ systems. So again, we're hopeful that, that read-through will again be recapitulated when we pharmacologically go to higher levels of CNP.

Q: Great to see the continued execution in the preliminary 333 data. On the achondroplasia front, I'd be interested in your thoughts on the evolving competitive landscape here with the recent data showing a long-acting CNP in combination with growth hormone and how you think that could impact the market down the road?

A: Thanks, Cory. This is Greg Friberg, again. I'll take a stab at that. When the early data for the growth hormone combinations card turned over, it was not surprising to see that there was added growth at a very early time point when growth hormone was added to CNP. Being able to achieve small or at least short-term increases has never been the problem with growth hormone. On the contrary, it's been whether or not those accelerations of growth can persist over time. Of course, achondroplasia is a condition which is not growth hormone deficient. And long-term follow-up of these patients particularly even the Japanese patients where it's approved in Japan have only shown a couple of centimeters an increase in final adult height. Similarly, the kind of evidence beyond growth that we're seeing with CNP affecting facial morphology, looking at tibial bowing, all of these evidence of improving the patient's well-being and health. Those haven't been as profoundly documented with growth hormone. So the question with the growth hormone combination, there are a few of them. One would be, is this something that's achievable over time. Secondarily, do we see mechanisms that accelerate bone age, do we see those? You won't see those in the first 6 months. So we'll want to -- as I think as field, we'll want to see additional data before the story is written as to whether that combination will add anything significant for patients with achondroplasia.