Christopher Raymond

Christopher Raymond of Piper Sandler asked if the current Avastin supply disruption is different from past events and whether a reduced role for compounded Avastin could create a larger opening for biosimilars.

Answer

EVP of Commercial Marion McCourt acknowledged that retina practices are becoming more accustomed to handling Avastin shortages due to prior disruptions, which may be diminishing confidence in its supply. She emphasized that physicians value choice and that both EYLEA and EYLEA HD are well-positioned to compete in the market, as demonstrated by the franchise's growth.

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 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.

Christopher Raymond asked for an update on the progress of driving ERT business demand through diagnostic harmonization across geographies. He also requested clarification on the timing of the BMN 351 data, noting a discrepancy between 'second half' and 'mid-2025'.

Answer

Chief R&D Officer Gregory Friberg clarified the BMN 351 data will be presented at a scientific congress in the 'early second half of the year.' Chief Commercial Officer Cristin Hubbard detailed diagnostic initiatives for MPS and CLN2, such as gene panels and cascade screening, which are showing success in markets like Brazil. She also highlighted strong double-digit growth from PALYNZIQ as a key driver for the ERT business.

Christopher Raymond questioned the clinical premise for BMN 333, asking why it would mechanistically achieve a better growth benefit than Ascendis's TransCon CNP, which has not shown a discernible improvement over Voxzogo.

Answer

EVP, Worldwide R&D Greg Freiberg explained that BMN 333 is designed to achieve a higher level of free CNP exposure than other agents, which preclinical models suggest could lead to better growth. He emphasized that the goal is to build upon Voxzogo's best-in-class evidence package by improving not only growth velocity but also other health and wellness measures.

Christopher Raymond of Piper Sandler inquired whether the regulatory perspective on sickle cell therapies might have changed following the Oxbryta situation, asking if the bar for approval has been raised or lowered.

Answer

CMO Dr. Sarah Gheuens stated that Agios's regulatory strategy has not changed because its approach was fundamentally different from the start. She referenced a 2021 poster outlining the Phase III design, which, unlike Oxbryta's accelerated approval path, was built to demonstrate clinical benefit on both hemoglobin and sickle cell pain crises from the outset.

Christopher Raymond of Piper Sandler inquired how the pediatric PKD studies offer insights for future pediatric programs in thalassemia and sickle cell, and whether an FDA advisory panel for the thalassemia PDUFA might be beneficial to address liver safety concerns.

Answer

Chief Medical Officer Dr. Sarah Gheuens explained that the pediatric PKD trials provided invaluable hands-on experience in trial logistics and execution that will benefit future pediatric programs. Regarding a potential panel, she stated that Agios has not been notified of one and believes the existing PKD label update, which includes a warning agreed upon with the FDA, already provides sufficient clarity on the liver safety profile.

Christopher Raymond of Piper Sandler questioned the potential for future pricing pressure on XPHOZAH from private payers, given the significant volume of free drug being provided to Medicare patients.

Answer

President and CEO Michael Raab reiterated that Ardelyx does not currently offer payer discounts, relying instead on the prior authorization process. CCO Eric Foster added that their primary goal is patient access, and while they have the flexibility to negotiate if needed, current access for non-Medicare patients remains strong without discounts.

Christopher Raymond questioned how physician use of XPHOZAH would not pull back if 60% of the patient base loses coverage, and how the financial impact of a free drug program would manifest on the P&L.

Answer

President and CEO, Mike Raab, clarified the strategy is about ensuring access, not reimbursement. He explained that physicians should continue prescribing as usual, and the ArdelyxAssist program will manage access on the back end, providing the drug to patients who qualify for the patient assistance program. He confirmed this would appear as a free drug expense in marketing, similar to current programs.

Christopher Raymond noted that Rinvoq's growth appears stronger in rheumatology and gastro than in atopic dermatitis, asking about the growth dynamics in atopic derm and whether other assets like lutikizumab will be more significant contributors.

Answer

EVP & CCO Jeffrey Stewart acknowledged past flatness but highlighted a recent inflection in atopic dermatitis, with in-play share now ramping above 20% driven by data on stringent endpoints versus Dupixent. He noted international shares are often higher. EVP & CSO Roopal Thakkar added that with only 5% market penetration, there is significant room for growth, positioning lutikizumab as the next asset and confirming continued investment in the space.

Christopher Raymond asked about the safety profile of the high dose of TSHA-102, particularly the time window for AAV-related adverse events, and also sought to understand the reaction from Key Opinion Leaders (KOLs) to the decision to drop the RSBQ as an endpoint.

Answer

President and Head of R&D Sukumar Nagendran detailed potential mechanisms for AAV-related immune responses without speculating on competitor data, highlighting the safety profile of intrathecal delivery. CEO Sean Nolan and Dr. Nagendran both confirmed that KOLs are supportive of dropping the RSBQ endpoint, citing its high variability, subjectivity, and its focus on behavior rather than clinically meaningful function.

Christopher Raymond asked about the expected time on therapy for the high-dose data presentation in H1 2025 and whether the follow-up duration would be standardized.

Answer

CEO Sean Nolan explained that the company aims to provide a more comprehensive update. He stated that for the high-dose cohorts, which will have three patients each, the plan is to present data when the majority of those patients have a minimum of six months of follow-up. This approach is intended to provide more consistency and a better line of sight into the drug's effect.

Christopher Raymond inquired about the ideal patient profile for the Wilson disease gene therapy and asked for an explanation of the negative binomial regression model being used for the setrusumab trial.

Answer

CEO Emil Kakkis explained that the most addressable Wilson disease patients are the 20% who are not well-controlled on current therapies, but others could also benefit. Regarding setrusumab, he noted the negative binomial model is a standard, FDA-agreed method for event-driven analysis that controls for variables like baseline fracture rates, and affirmed the study is well-powered for success.

Christopher Raymond asked about the PABlu launch, suggesting a recent supply disruption for Avastin could create a larger market opening, and questioned if this dynamic makes the launch more attractive for Amgen.

Answer

EVP of Global Commercial Operations Murdo Gordon highlighted Amgen's reliable, world-class manufacturing as a key advantage, noting the company has benefited from competitor shortages before with its own Avastin biosimilar, MVASI. He stated that retina specialists value a reliable supplier and Amgen is well-positioned to meet demand for PABlu.

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.

Christopher Raymond of Piper Jaffray sought clarification on the delay of the Crigler-Najjar program update to Q2 and inquired about any plans to publish the preclinical data for the Pompe program.

Answer

President and CEO Matthew Patterson attributed the Crigler program's delay to complexities in patient identification and screening. For the Pompe program, he indicated a cautious approach to data disclosure to maintain a strategic advantage, but suggested that AT845-specific data might be shared in the fall, post-IND filing.