Christopher Raymond • Piper Sandler

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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