Chris Raymond

Chris Raymond asked about the importance of EYLEA HD label enhancements for dosing flexibility and clinic inventory policies, and how private equity in the retina space might be influencing these policies.

Answer

EVP of Commercial Marion McCourt explained that retina specialists value EYLEA HD's clinical efficacy, safety, and durability. She noted that as a newer product, availability and payer coverage are important, and emphasized that label enhancements are crucial for providing dosing flexibility, which is highly valued by the retina community for selecting the right product for patients.

Chris Raymond of Raymond James inquired about the significance of EYLEA HD label enhancements, how clinic inventory policies have evolved, and the influence of private equity in the retina space on these policies.

Answer

Executive Vice President of Commercial, Marion McCourt, explained that retina specialists prioritize selecting the right product based on clinical efficacy, safety, and EYLEA HD's durability. While not an expert on inventory, she noted that EYLEA HD's strong characteristics are driving its uptake. President and CEO, Leonard Schleifer, added that significant upswing is dependent on these enhancements.

Chris Raymond asked if BioMarin is more concerned about trans-con-CNP than infigratinib regarding 2027 guidance, and if the lower end of the FactSet number assumes Ascendis gets first-cycle approval. He also asked Alexander Hardy about how BioMarin has leveraged its infrastructure to improve upon Enzyme's efforts for the BMN-401 asset in terms of patient identification and outreach.

Answer

Brian Mueller (CFO, BioMarin Pharmaceutical) declined to comment on specific competitors but stated that lower-end 2027 estimates assume middle-of-the-road timelines for competitor approvals and successful launches. Alexander Hardy (President and CEO, BioMarin Pharmaceutical) highlighted BioMarin's capability in rare diseases at scale, operating in 80 countries, to magnify BMN-401's impact. Greg Friberg (Chief R&D Officer, BioMarin Pharmaceutical) noted it's early days since the Enzyme Pharma acquisition (July 1), but BioMarin is leveraging capabilities for regulatory interactions and exploring additional indications, with an adult indication for ENPP1 deficiency being a future focus.

Chris Raymond asked if BioMarin is more concerned about trans-con-CNP than infigratinib regarding competition, and if the lower end of the 2027 guidance assumes Ascendis receives first-cycle approval with one year's data. He also inquired how BioMarin has leveraged and improved upon Enzyme Pharma's efforts in patient identification and outreach for BMN-401 since the acquisition.

Answer

Brian Mueller, Chief Financial Officer, stated BioMarin would not comment on specific competitors but confirmed that lower-end 2027 estimates assume middle-of-the-road timelines for competitor approvals and successful launches. Alexander Hardy, President and CEO, highlighted BioMarin's scale in rare disease execution across 80 countries, expressing confidence in leveraging this infrastructure for BMN-401. Greg Friberg, Chief R&D Officer, noted it's early days post-acquisition (July 1), but BioMarin is leveraging capabilities for regulatory interactions and exploring additional indications, with preparation for an adult indication for ENPP1 deficiency underway.

Chris Raymond of Piper Sandler asked for the rationale behind pursuing the anti-myostatin asset in SMA given its prior failure in DMD and inquired about the Phase 3 design. He also asked if the Kv7 asset is still being targeted for epileptic encephalopathy and questioned the company's philosophical reason for not providing financial guidance.

Answer

CEO Vlad Coric and President and GM of Biohaven Ireland Cliff Bechtold clarified that while the myostatin asset failed as a monotherapy in DMD, it established a strong pediatric safety profile, and recent competitor data provides clinical proof-of-concept in SMA as a combination therapy. President of Biohaven Labs Michael Bozik confirmed they will pursue adult focal epilepsy and are committed to exploring pediatric epilepsy, including KCNQ2. CFO Matthew Buten stated the company will be more comfortable guiding next year due to current variables like the Pfizer EU launch and competitive dynamics.

Chris Raymond from Piper Sandler questioned the prescriber mix for NURTEC ODT between primary care physicians (PCPs) and headache specialists, and asked about any differing access considerations for these groups.

Answer

CCO BJ Jones explained that while specialists are typically faster to adopt, Biohaven is seeing a strong and growing uptake among PCPs. He anticipates that primary care will eventually constitute the bulk of the category. He also stated that there are no significant or unique access issues for PCPs at this time.

Chris Raymond from Piper Sandler & Co. inquired about the strategic assumptions behind the company's five-year cash runway guidance, particularly regarding business development, and asked for an update on the A2A biomarker for inupadenant.

Answer

President and CEO Michel Detheux explained that the cash runway accounts for the GSK partnership costs, advancing programs into Phase 3 studies, internal pipeline development, and opportunistic external innovation. Chief Medical Officer Joe Lager added that biomarker data for inupadenant is being further investigated to inform indication selection, with a more detailed data release anticipated next year.

Chris Raymond of Piper Sandler inquired about the expected data for EOS-850 (inupadenant) at the upcoming ASCO conference, potential new indications for the drug beyond the current ones, and the decision timeline for pursuing gastric and pancreatic cancers for EOS-448.

Answer

President and CEO Michel Detheux clarified that the ASCO update for inupadenant will feature monotherapy expansion data and biopsy results linking a biomarker to clinical benefit. Chief Medical Officer Dr. Joe Lager identified non-small cell lung cancer and endometrial cancer as additional indications under consideration for inupadenant. For EOS-448, Dr. Lager stated that an update on the development plan for gastric and pancreatic cancer could be provided later in the year.

Chris Raymond of Piper Jaffray asked about the company's comfort level with the AT132 pivotal trial endpoint given observed patient variability, the status of the Pompe IND review which appeared beyond the 30-day window, and the timeline for NCH's construct dosing.

Answer

CEO Matt Patterson explained that it was too early to draw conclusions from a single patient's data and expressed high confidence in the AT132 pivotal plan, especially after expanding enrollment. He clarified the Pompe IND delay was simply due to the FDA being busy, a situation they've encountered before. COO Natalie Holles added that timelines for the NCH and Audentes' AT702 constructs are converging, with the focus now on AT702 as the registration-directed candidate.