Tessa Romero • JPMorgan Chase & Co.

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CCO Tsveta Milanova stated the initial launch will target approximately 65% of the 6,000 diagnosed U.S. adults. She identified three segments: transfusion-dependent patients (seen frequently, e.g., monthly), non-transfusion-dependent patients with comorbidities, and NTD patients with debilitating fatigue. Specific breakdowns were not provided, but she noted these groups are actively engaged with the healthcare system.

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CMO Dr. Sarah Gheuens stated that the regulatory review is ongoing and the final label details will be available at the PDUFA date. She also mentioned that while the open-label extension data will be published, the team's current priority is the ongoing regulatory reviews.

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CMO Dr. Sarah Gheuens explained that the FDA review is a continuous process of interaction rather than one with formally defined meetings like the mid-cycle review. She stated that engagement is collaborative and ongoing, and it is currently too early for labeling negotiations, which occur later in the review cycle. While ultimate certainty on a REMS comes only with approval, she conveyed that the process is progressing normally and they are pleased with where they stand.

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CFO Cecilia Jones indicated that both SG&A and R&D expenses are expected to grow year-over-year to support upcoming launches and pipeline advancement, though she did not quantify the growth. For ex-U.S. approvals, CMO Dr. Sarah Gheuens noted that timelines are less certain than the U.S. PDUFA date, but progress is on track. CCO Tsveta Milanova added that a time lag between approval and commercialization is expected in these regions due to pricing and reimbursement processes.

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EVP of R&D Fady Malik stated that physicians in their trials recognize aficamten's different DDI profile, which doesn't involve CYP2C19, and that any potential interactions are rare and manageable without a formal monitoring program. EVP & CCO Andrew Callos clarified that the company does not anticipate pharmacy-level monitoring being part of a potential REMS program for aficamten.

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President and CEO Robert Blum stated that while he could not share specifics on the ongoing FDA review, an ideal label would reflect aficamtan's differentiated properties. Andrew Callos, EVP & Chief Commercial Officer, added that market research indicates a differentiated label and REMS would support strong uptake and expansion into community cardiology.

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Robert I. Blum, President and CEO, stated that the decision not to submit a REMS was based on their interpretation of FDA feedback from three meetings. He explained the lack of disclosure was a strategic decision to maintain competitive advantage and was not unconventional. The FDA's request for the REMS was recent, which then prompted the company's public communications.

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Fady Malik, EVP of R&D, stated that the trial is highly anticipated by investigators and has enrolled the target patient population. He noted the trial is conservatively powered to detect a delta-Peak VO2 of 1.5 to 2.0 mL/kg/min, a range consistent with previous positive data, expressing confidence in the study's design and hypothesis.

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President and CEO Ian Mortimer explained that the last patients would be randomized following an eight-week baseline period. Chief Medical Officer Christopher Kenney added that while final numbers are pending, the geographic split and site strategy are expected to be similar to the successful EXTOL study, though with more sites to accommodate a larger trial. Kenney also confirmed constant interest in compassionate use, but stated no program is currently active.

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President and CEO Ian Mortimer noted that recruitment is nearing completion and will be finished in the next few months, attributing some variability to normal trial ebbs and flows. He clarified that the few late-additions of trial sites are not significant. Chief Medical Officer Dr. Chris Kenney added that the screen failure rate is very similar to the Phase II study due to nearly identical inclusion/exclusion criteria.

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EVP, Head of Research & Development, Elizabeth Thompson, stated that the top-line release will focus on the primary endpoint and a high-level overview of safety and tolerability. She noted that secondary endpoints include clinician assessments and a responder analysis for the HQCT. Dr. Thompson also mentioned that a key focus during the trial has been robustly monitoring assessment performance and variability to ensure data consistency across sites.

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An executive, likely CCO Thomas Garner, explained that DAYBUE's growth will be driven by an expanded field force and improved patient persistency, while NUPLAZID's momentum will be sustained by direct-to-consumer campaigns. CFO Mark Schneyer added that for both brands, volume will be the primary growth driver for the remainder of the year, with DAYBUE also benefiting from gradual price increases.

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CFO Mark Schneyer clarified that the patient base is expected to remain stable, with the anticipated Q1 revenue decline driven by seasonal pull-forward and net price changes, not fewer patients. CCO Tom Garner added that with 70% of the Rett population yet to try DAYBUE, a key growth strategy is penetrating community settings, which treat 65% of patients but currently have only 25% penetration.

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CEO David Hallal outlined the plan to work with CDMOs on robust responses to the FDA. President of R&D Akshay Vaishnaw stated the ideal label would cover patients aged two and older, based on consistent SAFIRE trial data across age groups. Hallal added that patient advocacy groups like Cure SMA have advocated for such broad labels.

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CEO David Hallal and President of R&D Akshay Vaishnaw both reiterated that the regulatory process is on track. Dr. Vaishnaw described interactions with the FDA as 'extremely constructive' but declined to provide specific details on meetings or labeling discussions, while reaffirming confidence in the September 22 PDUFA date.

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Executive Jay Backstrom explained that the company feels obligated to explore adjacent indications like DMD and FSHD following apitegromab's success in SMA. He stated the clinical team has the capacity to take on at least one additional study and emphasized the goal is to build a multi-billion dollar neuromuscular franchise, starting with the potential $2 billion-plus opportunity in SMA alone.

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CMO Ramiro Ribeiro stated that the focus is now on study conduct, leveraging experience from prior trials. He highlighted close collaboration with clinical sites to prevent protocol deviations and diligent safety tracking. CEO Jay Duker added that the non-inferiority trial design is familiar to physicians and the patient dropout rate is very low at less than 2%, which helps de-risk the studies.

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Chief Medical Officer Dr. Ramiro Ribeiro, as directed by CEO Dr. Jay Duker, attributed the strong enrollment to the robust Phase I and II data giving investigators confidence, and a patient-centric, easy-to-follow trial design. Dr. Ribeiro stated the team is focused on ensuring data integrity across all areas to prepare for an efficient NDA submission.

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Chief Medical Officer Ramiro Ribeiro stated that most U.S. sites are activated, with approximately 60 active sites per study. He added that the company plans to activate between 60 and 80 ex-U.S. sites per study later in the year, noting that enrollment numbers are progressing very well.

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