Ellie Merle • UBS Group

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CEO Nello Mainolfi stated he could not comment on IRAK4 clinical data per Sanofi's guidance but noted asthma and COPD are high-priority indications. He explained that molecular glues and heterobifunctional degraders are complementary technologies. For CDK2, a molecular glue was used to achieve high selectivity against CDK1, which is a challenge for traditional binders.

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CBO Eric Pauwels explained that it's too early for coverage approvals, as the company is currently in the multi-day process of insurance verification. Regarding patient distribution, he stated that, similar to the U.S., patients in Europe are highly concentrated in centers of excellence in major cities. He used Germany as an example, where their compassionate use program targeted centers overseeing nearly 8,000 patients, indicating a centralized care model.

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President & CEO Jan Møller Mikkelsen responded that it was too early to discuss specific label language but highlighted that their placebo-controlled data demonstrates benefits beyond linear growth, such as improved leg bowing and muscle function, which he sees as a key differentiator. Regarding IP, he expressed strong confidence in their position, noting extensive filings on optimal and sustained-release profiles dating back to 2015.

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CFO Jamey Mock stated that the U.S. product sales guidance of $1 to $1.5 billion already incorporates variability from competitive pressures, including pricing and contracting. He confirmed that contracting is now 'basically complete' but declined to provide specific pricing details, expressing confidence in the current financial framework.

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President & CEO Curran Simpson stated that the company is taking a cautious approach to non-ambulatory DMD and will first establish a robust safety database in the ambulatory population before engaging in detailed FDA discussions. CMO Dr. Steve Pakola explained that for wet AMD, a key secondary endpoint is reducing injection burden, with at least a 50% reduction being a clinically meaningful target that would create a compelling value proposition.

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President of R&D Steve Kelsey acknowledged that amplification of the mutant RAS allele is a major escape mechanism, particularly for multi-selective inhibitors, but noted it can be overcome therapeutically, for instance with a RAS-ON doublet. He stated there is currently no evidence that a degrader is superior to an inhibitor for any oncology target, pending clinical data from degrader companies. CEO Mark Goldsmith added that this resistance mechanism validates the effectiveness of diraxonrasib.

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Dr. Mark Goldsmith, Chairman and CEO, stated they will not wait for second-line Phase III results to initiate a first-line study. Dr. Wei Lin, CMO, added that the current second-line data provides sufficient proof-of-concept. The plan for the first-line Phase III trial includes a monotherapy arm and potentially arms combining RMC-6236 with standard-of-care chemotherapy. He expressed confidence that efficacy will translate to the first-line setting as RAS is a fundamental driver across lines of therapy.

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CFO Mardi Dier responded that the company has no near-term plans to issue revenue guidance. She focused on the remainder of 2025, stating they expect to steadily add patients and that growth will track with other successful specialty launches. Regarding gross-to-net, she confirmed predictability for 2025 and said the company would address 2026 at a later time.

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President and CEO Dr. Reshma Kewalramani highlighted ongoing Phase 4 studies showing positive data on pain control and opioid reduction. EVP and CCO Duncan McKechnie added that the patient support program is a temporary bridge that will be retired as national payer coverage is achieved, which is expected by year-end.

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CCO Tolga Tanguler stated that while they expect both patient segments to continue growing from the strong 1,400-patient start in Q2, the company will not report specific patient numbers quarterly. He cited the difficulty of precisely tracking the cardiomyopathy indication with Amvuttra's single SKU.

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Greg Friberg, Chief R&D Officer, stated that current studies for other short stature indications are on track to enable the start of pivotal trials in 2027. He explained that the development timeline for BMN 333 in achondroplasia will provide the necessary data to make informed decisions about its potential use in other indications later, suggesting the timelines align well for strategic planning.

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President and CEO Bradley Campbell attributed the guidance change to two main factors: a timing delay in patient starts in newly reimbursed countries shifting from Q1 to later in the year, and the unexpected impact of the U.K.'s VPAG rebate. He noted the Netherlands is a significant opportunity with over 150 patients, but uptake is slowed by a single-center treatment bottleneck, with growth expected in the second half of the year.

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CEO John Houston outlined a significant market opportunity, estimating a pool of up to 25,000 new patients annually in the second-line plus ESR1 mutant setting. CFO Andrew Saik explained that the financial impact of the restructuring would begin to be seen prior to Q4, with the full run-rate savings realized in Q4 2025, and a related charge would be recorded in Q2.

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Dr. James Hamilton, Chief of Discovery and Translational Medicine, confirmed that Part 1 of the study will assess body composition using full-body MRI and weight loss. He added that they will also evaluate standard Phase I metrics like safety, pharmacokinetics, and the direct biomarker for gene target knockdown.

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CMO Dr. Scott Harris stated the ESSENCE results were modest and expected, requiring a longer 72-week duration. He expressed high confidence that pemvidutide's superior liver fat reduction will enable it to show statistically significant MASH resolution and fibrosis improvement at a much earlier 24-week time point, combined with meaningful weight loss, setting a new standard of care. CEO Dr. Vipin Garg added that this offers a "complete solution" for MASH patients.

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