Release Date: April 24, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Q & A Highlights
Q: Can you give us some sense of what you need to see to support continued investment in the Roctavian program? Or maybe asked another way, what would you have to see over the next several months in order to decide to out-license Roctavian or curtail future investments there?
A: Alexander Hardy - BioMarin Pharmaceutical Inc. - President, CEO & Director: Thanks for the question, Phil. We have a high level of current Roctavian investments and continued challenges with commercial uptake. At Investor Day, we will communicate our evaluation criteria and the timing for that evaluation. Possible outcomes include seeing meaningful uptake, rightsizing the investment across the organization, or potentially removing it from our portfolio and divesting the asset.
Q: I know you've talked about label expansion for Voxzogo being of the highest importance strategically. What are the next steps in optimizing the current formulation for Voxzogo?
A: Henry J. Fuchs - BioMarin Pharmaceutical Inc. - President of Worldwide Research & Development: We are focused on expanding our CNP franchise into areas where there's potentially transformative medical benefit for children with statural impairments. This includes entering into a Phase III registration-enabling trial in hypochondroplasia and progressing well in terms of idiopathic short stature. We have a lot of confidence in the future of the CNP franchise.
Q: On the strategic review across the pipeline, what are the levers for lowering OpEx from this line? And how are you thinking separately about the mid- to late-stage pipeline in this context?
A: Brian R. Mueller - BioMarin Pharmaceutical Inc. - CFO & Executive VP: The strategic review is looking at both strategy and operations and efficiency. We're leveraging what we've built over the last many years, streamlining the business, and prioritizing the work on the right assets. We'll share more details on what the specific levers are and the tools around cost transformation and business efficiency at Investor Day.
Q: What's driving the difference between the reported Voxzogo demand increase and the revenue increase from the numbers?
A: Brian R. Mueller - BioMarin Pharmaceutical Inc. - CFO & Executive VP: The difference is due to order timing and the timing of patient additions. We manage orders at the specific market and SKU level, especially with the current supply constraint. This dynamic can cause a disconnect between patient additions and revenue recognition.
Q: Is there an increased appetite to divest the gene therapy franchise? And can you outline how much spend this makes up currently on OpEx?
A: Alexander Hardy - BioMarin Pharmaceutical Inc. - President, CEO & Director: Our focus right now is establishing what the Roctavian opportunity is. One possible outcome could be removing it from our portfolio and divesting, but we are not engaging with people around the divestment of Roctavian at this moment. Our focus is really on establishing the opportunity right now.
Q: Can you help us understand how you're thinking about the relative size of the opportunities between ISS and genetic short stature pathways?
A: Alexander Hardy - BioMarin Pharmaceutical Inc. - President, CEO & Director: The indications beyond achondroplasia, such as hypochondroplasia, ISS, Noonan's, Turner's, SHOX, represent about 600,000 patients compared to the total global addressable population for achondroplasia of 21,000. We're going for a more severe patient population in idiopathic short stature editions. We'll share more at Investor Day about the TAMs for all the different indications.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.