Sean McCutcheon • Raymond James Financial

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CEO Thomas Schuetz stated the PFS analysis is powered for a hazard ratio of approximately 0.6, which recent market research suggests would be highly meaningful. He noted the high progression rate in the paclitaxel arm suggests its efficacy is low. Regarding the OS analysis methods, he said the company has not received specific feedback from the FDA on the difference between the ITT and the crossover-adjusted (RPSFT) analyses.

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President & CEO Curran Simpson confirmed this is the strategy. He stated that once the pivotal cohort is enrolled by October, the company will continue enrolling patients directly into the confirmatory study cohort. This approach aims to expand the safety database, which will be important for regulatory discussions and addresses potential risk around sample size requirements.

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Chief Medical Officer Dr. Steve Pakola highlighted the large market opportunity and the increased optionality provided by the in-office suprachoroidal delivery. He emphasized that for diabetic retinopathy (DR) in particular, a one-time in-office treatment is highly compelling for patients to avoid indefinite injections, addressing a significant unmet need.

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Chief Medical Officer Dr. Steve Pakola argued that the primary barrier to current NPDR treatments is the burden of repeated injections, a problem that even long-acting TKIs do not fully solve. He positioned REGENXBIO's ABBV-RGX-314 as a 'paradigm shift,' where a onetime, in-office injection could address this massive unmet need. He cited the program's 89% reduction in vision-threatening complications at one year as the key data point driving the program into pivotal studies.

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EVP of Oncology Dr. Mika Derynck explained that the current Phase 1 study enrolls patients who have exhausted standard of care, and since it's open in Australia and Europe, there are fewer patients with prior radioligand therapy. However, they plan to open U.S. sites to generate this data. She highlighted the importance of less frequent (Q3 week) dosing for convenience in earlier lines of therapy, noting that data from the HER2 program showed no resensitization or loss of efficacy with a less frequent schedule, which bodes well for VIR-5500.

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Pravin Dugel, Executive Chairman, President & CEO, acknowledged the importance of demonstrating a clear link between the SOUL-one results and the SOLAR trial. He assured that the company intends to provide data cuts that will directly address this translation, although he did not specify the exact nature of the data to be released.

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President and CEO Dr. Pravin Dugel stated that while specific rescue criteria are not yet public for competitive reasons, they will align with traditional standards and FDA guidance. He highlighted a key strategic advantage: regulatory agencies will review SOL-R's rescue data in the context of a positive SOL-1 study, which will have already established AXPAXLI's 9-to-12-month durability. This, he believes, will provide a favorable context for the evaluation of the SOL-R rescue data.

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President and CEO Dr. Pravin Dugel highlighted the advantage of leveraging the successful commercial team from DEXTENZA. While stating it is too early to discuss specific pricing strategies, he affirmed that the company firmly believes it will be able to obtain premium pricing for AXPAXLI.

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Dr. Pravin Dugel, President and CEO, responded that while the primary goal was regulatory alignment, the data will have significant commercial value. It will allow for a comparison against what may be considered the 'next generation' of Eylea. He emphasized that the totality of data from SOL-1 (a superiority study showing durability) and SOL-R (a non-inferiority study showing dosing flexibility) will collectively answer nearly all physician questions about AXPAXLI's performance.

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Chief Medical Officer Mythili Koneru stated that dose escalation for the Claudin 18.2 study should complete by late summer, with expansion cohorts to follow. She highlighted the opportunity for CAR-T in this space due to its sensitivity in lower-expressing cells, which could be an advantage over approved antibodies. The company did not comment on the partnership strategy for the program.

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Executive Jessie Yeung explained that while the Tandem data was from single centers, it opened a conversation, and many centers are interested. The company is actively investigating the strategy in internal and external studies to provide more data. An Unknown Executive clarified the Tech Lane expansion plan: Phase 1 will be completed this year, and Phase 2 is an incremental $150 million investment starting in the second half of 2025 and completing in 2028.

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CEO Ying Huang and CMO Mythili Koneru explained that with very high long-term PFS rates in frontline myeloma, short-term follow-up is not meaningful. Regarding manufacturing, Ying Huang stated the out-of-spec rate is improving quarterly, is in the low teens, and that preliminary data suggests a lower rate for the earlier-line patient population.

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Robert I. Blum, President and CEO, expressed strong optimism for ACACIA-HCM, highlighting its design based on encouraging Phase II results and enthusiastic global enrollment. Fady Malik, EVP of R&D, added that confidence is high due to using the same successful dosing regimen from the REDWOOD-HCM Phase II study, the availability of a higher dose proven effective in the FOREST study, and a rigorous patient screening process by experienced investigators.

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SVP & CMO Stuart Kupfer explained that observations from SEQUOIA-HCM showed that the specific threshold for exercise incapacity (e.g., <90% or <80%) did not make a significant difference, as patients were already significantly symptomatic with exercise deficits due to their obstruction. Therefore, the company felt comfortable relaxing the criteria for MAPLE-HCM while still enrolling patients who could benefit from treatment.

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Stuart Kupfer, SVP and CMO, explained that data from nHCM patients and competitor trials are encouraging. The AMBER trial will enrich for patients with EF of at least 60% and is a rigorous, placebo-controlled study. He highlighted CK-586's favorable Phase I data, which showed a shallow and predictable PK/PD relationship for LVEF, as a key valuable property.

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CMO Samit Hirawat explained that an MRD-based approval would require supportive evidence from other endpoints. Both Hirawat and CCO Adam Lenkowsky noted that the opportunity for oral CELMoDs remains large, as CAR-T therapy is concentrated in academic centers while the majority of myeloma patients are treated in the community setting, where oral options are needed.

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CMO Samit Hirawat detailed the radiotherapeutics pipeline, highlighting a Phase III readout in 2026 for the lead asset and several earlier-stage programs. CEO Christopher Boerner added that the company believes in the platform and would consider further business development to enhance the RayzeBio acquisition if the science and financials are compelling.

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