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CMO Walid Abi-Saab said the pre-BLA meeting will cover top-line data and CMC updates to ensure the package is ready for submission, with an update to follow. CEO Matt Kapusta estimated 50-55 U.S. sites are capable of the procedure and stated that with 35,000 diagnosed patients, the 'overwhelming majority' are in Stage 2 or 3 and would be potentially eligible.

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CMO Dr. Walid Abi-Saab stated that a detailed BLA submission timeline, including CMC updates, will be provided in the next regulatory update. He noted they are leveraging experience from HEMGENIX. Regarding the natural history control, he expressed confidence that the final agreed-upon comparator will not yield significantly different outcomes from what has been previously reported.

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CEO Faheem Hasnain and CMO Dr. Richard Aranda clarified that patients could be on a stable dose of sotatercept for 6 months prior to enrollment but could not start it during the trial (no 'drop-ins'). Dr. Aranda detailed the CIRANATA population, noting it has more stringent hemodynamic criteria than the INCREASE trial and targets specific ILD subtypes, confirming they expect a greater treatment effect in this more severe population.

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CBO Eric Pauwels attributed Emflaza's resilience to strong brand loyalty built over eight years of providing 'white glove' patient services. He stated that since generic pricing has been similar, there has not been a significant impact on gross-to-net. While expecting some erosion, he noted the team continues to add new patients and maintain existing ones.

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CEO Dr. Matthew Klein and CBO Eric Pauwels explained that the prescriber base is highly centralized within approximately 103 centers of excellence. They anticipate rapid adoption across all patient segments, driven by strong interest from physicians and dietitians, and are preparing for a significant bolus of patients at launch.

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CEO Matthew Klein expressed optimism for using Huntington lowering as a surrogate endpoint but stated the company is prepared to initiate an efficacy trial regardless of the outcome, having the capital to do so. He clarified that the FDA's decision to combine the two meeting requests was purely for logistical efficiency due to the significant overlap in topics and personnel.

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Dr. Mark Goldsmith, CEO, stated that the company does not provide timelines for regulatory interactions but will update when plans are definitive. On ex-U.S. strategy, he acknowledged strong interest from potential partners, creating significant optionality. He explained there is no rush to decide, and the company is using time to its advantage as its portfolio and organization mature. The recent hire of a Chief Global Commercialization Officer will help assess all options to best serve patients and the business.

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Executive Timothy Sullivan clarified that revenue is recognized when doses are shipped to distributors, not to physicians, so net price cannot be calculated from the disclosed vial shipments. He confirmed the Q1 gross-to-net was in the low 20% range and reiterated guidance of low-to-mid 20s for 2025. Executive Cedric Francois added that quarter-over-quarter injection volume growth is expected to continue as the market is still in the early stages of adoption.

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CEO Dr. Cedric Francois stated that SYFOVRE adherence levels are similar to anti-VEGF therapies, which is a positive trend. EVP of Commercial David Acheson added that increased sample use is a sign of demand and that while the funding gap issue is temporary, its resolution timing is uncertain. Apellis will continue to supply samples to ensure patient treatment.

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CFO Timothy Sullivan directed the question back to the prepared remarks, reiterating the company's guidance for 'low to mid-single-digit' percentage vial growth for the remainder of 2024. He declined to provide more specific intra-quarter guidance beyond that.

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President and CEO Douglas Ingram stated he did not have specific data on rescheduling but noted the delays were due to patients needing more information after the safety event. Head of R&D Dr. Louise Rodino-Klapac added that a labeling supplement was submitted in April to include the patient death, and the FDA has set a target completion date for the review no later than Q4 2025.

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CEO Douglas Ingram explained that while LGMD Type 2E is an ultra-rare disease, the three upcoming sarcoglycanopathy programs (2E, 2D, 2C) collectively represent a market opportunity about 25% the size of the Duchenne opportunity for ELEVIDYS. Chief Scientific Officer Dr. Louise Rodino-Klapac added that due to improved genetic diagnosis, the patient mix is now roughly an even 50-50 split between ambulatory and non-ambulatory.

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Chief Customer Officer Dallan Murray stated that the vast majority of the 75 certified treatment centers have dosed patients. However, he and CEO Doug Ingram clarified that the company does not provide specific details on dosing concentration per site, preferring to focus guidance on overall net product revenue.

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CCO Duncan McKechnie stated that the goal is broad retail availability, and at launch, JOURNAVX was in about 95% of U.S. retail locations. He confirmed the initial script mix is heavily weighted toward the hospital discharge setting, as expected, and this trend should persist for the year. He noted the average prescription duration is about 14 days, which is in line with expectations for moderate to severe acute pain.

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COO Stuart Arbuckle attributed the company's confidence to ALYFTREK's strong clinical profile, including its efficacy and convenient once-daily dosing. He explained that these patients want to be on the best possible therapy and ALYFTREK provides a compelling new option, though he did not provide specific data on the reasons for prior discontinuation.

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President and CEO Jan Mikkelsen responded by highlighting two key metrics that indicate success: patient adherence is extremely high, consistent with clinical trial levels, and the patient dropout rate is exceptionally low at under 1%. He summarized the strong retention by stating, 'If you start on YORVIPATH, you stay on YORVIPATH,' implying the treatment is chronic and highly valued by patients.

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Chief Commercial Officer Tolga Tanguler confirmed a single price exists for both indications. He stated that the use of the QuickStart program is "very limited," consistent with the experience in polyneuropathy. He emphasized that patients are converting to paid therapy effectively across all payer types, with the majority having a zero dollar co-pay, indicating strong access.

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Chief Commercial Officer Tolga Tanguler declined to break down the guidance further but reiterated that growth is expected to accelerate in the second half of the year. Regarding value-based agreements (VBAs), he noted they cover a significant portion of PN lives and are based on clinical outcomes. He anticipates this successful model will carry over to the cardiomyopathy indication.

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Chief Medical Officer Pushkal Garg explained that Alnylam is leveraging human genetics to guide its pursuit of new tissues, noting the platform's favorable tolerability and infrequent dosing profile. He deferred a substantive update to a future R&D Day for competitive reasons. CEO Yvonne Greenstreet added that expanding to extrahepatic tissues is a key innovation driver for the company.

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CEO William Sibold stated that with only 5% penetration in their 315,000-patient target market, there are years of growth ahead. He believes a competitor's entry will expand the market by accelerating diagnosis, and Rezdiffra's strong profile will ensure continued growth. CFO Mardi Dier added that Madrigal expects robust quarter-over-quarter and full-year 2025 sales growth.

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CEO William Sibold indicated that more detailed data would be shared at a future medical meeting. Dr. Michael Charlton, Head of Clinical Development, emphasized the clinical predictivity of the results, noting that a 25% decline in liver stiffness in similar patients has been shown to project a 30-40% decreased likelihood of liver-related clinical events.

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CEO William Sibold explained that patient additions are steady and the company has not yet reached a steady state. Regarding potential competition, he noted that payers have not required a GLP-1 step-through to date and emphasized that real-world practice differs from controlled trials, highlighting Rezdiffra's strong product profile and established payer relationships.

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President Stephen Hoge confirmed a 2026 readout for the Phase III melanoma study remains possible, contingent on event accrual. He highlighted that the Phase II renal cell carcinoma study is now fully enrolled. For the Checkpoint program, he noted they are encouraged by early data and are advancing in first-line non-small cell lung cancer and metastatic melanoma, with plans to expand into more histologies.

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CFO Jamey Mock confirmed significant flexibility exists to reduce R&D spend from the guided $4.1 billion in 2025, noting over 50% of trial costs are for respiratory programs expected to wind down. President Stephen Hoge explained the Norovirus pause was a proactive measure for patient safety and transparency, and since enrollment for the season was complete, no impact on the study's timeline is expected.

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CEO Stéphane Bancel declined to disclose specifics on ex-U.S. contracts but stated that minimum purchase commitments would grow over time, despite a dip in 2025. President Stephen Hoge noted that CMV case accrual is steady and that the time to the final analysis, if needed, would not be a very long period and could happen quite quickly.

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CCO Matt Outten responded that AMVUTTRA appears to be positioned for the mixed phenotype niche, which is about 10% of the market. He noted that BridgeBio is not seeing significant uptake of the competitor outside of that segment, citing AMVUTTRA's higher price and less compelling variant data. He also pointed to the financial challenges of its buy-and-bill model for community physicians as a competitive advantage for Attruby.

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CEO Curran Simpson outlined that the next functional data update will include longer-term follow-up for patients at both dose levels and initial data for new patients in the dose level 2 cohort. Chief Medical Officer Dr. Steve Pakola added that they will report on time function tests and NSAA. He confirmed they compare results to matched external natural history controls to provide context and that this methodology was part of their successful end-of-Phase II discussions with the FDA.

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Chief Medical Officer Noah Berkowitz stated that while the frontline trial will likely exclude certain rapid progressors, there are no plans for molecular enrichment, as vepdegestrant targets both wild-type and mutant ESR1. CEO John Houston and CSO Angela Cacace explained that the company's focus remains on oncology and neuroscience, though some assets are being explored preclinically for immune indications. The speed to a candidate now largely depends on the availability of a quality ligand for a given target.

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CEO John Houston acknowledged the biological challenge but believes a portion of the wild-type population will respond. CMO Noah Berkowitz stated that the base case for approval is the ESR1 mutant subpopulation, following regulatory precedent. He clarified the study is not powered for the non-mutated group alone but is powered for the overall ITT population.

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CEO Nello Mainolfi clarified that preclinical data shows STAT6 expression levels do not impact the drug's degradation kinetics. CMO Jared Gollob added that since preclinical models for both indications show optimal activity at similar degradation levels (90%+), they expect to use similar doses in both the AD and asthma Phase IIb studies.

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President and CEO Nello Mainolfi and Senior Director of Immunology Veronica Campbell addressed the concerns. Nello Mainolfi highlighted that IRF5's safety is supported by its cell-specific expression and activation only in disease contexts. Veronica Campbell added that broad genomic analysis shows little evidence for a tumor suppressor role and that the initial report has not been replicated. She noted that other IRF family members critical for immune surveillance remain intact.

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Chief Commercial Officer Cristin Hubbard stated that the U.S. revenue contribution for VOXZOGO, currently around 25%, is expected to increase over time. She attributed non-treatment to factors like awareness and access to prescribers, which are being addressed via increased commercial efforts. Chief R&D Officer Gregory Friberg added that they are continuously building the clinical data set on proportionality and quality of life to increase physician and family comfort with the therapy.

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EVP, Chief Commercial Officer Cristin Hubbard attributed the growth primarily to strong Palynziq uptake in the U.S., driven by new patient starts and re-engagement of discontinued patients. She outlined future initiatives including high-yield diagnostic efforts, adherence programs, and geographic expansion, noting that the new business unit structure will also drive urgency and accountability.

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CMO Dietmar Berger confirmed that enrollment is on track. CCO Johanna Mercier added that this partial responder population represents about one-third of patients, or 20,000-25,000 people in the U.S. and a similar number in Europe, highlighting a significant opportunity to expand Livdelzi's reach.

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President and CEO Bradley Campbell outlined a strategy focused on acquiring de-risked, late-stage, or commercial assets within the next 12-24 months to leverage their existing infrastructure. Chief Development Officer Dr. Jeff Castelli and Chief Business Officer Sebastien Martel added that internal efforts are focused on next-generation therapies for Fabry and Pompe, while external BD targets adjacent rare disease areas like renal, cardio, and metabolic disorders.

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President and CEO Bradley Campbell stated that, as anticipated, most patients switching from Nexviazyme have been on that therapy for one to two years. He noted a large group of patients is now reaching this timeframe, creating a significant opportunity. He also observed that some patients switch earlier, often influenced by positive word-of-mouth, a trend the company hopes will grow.

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Chief Strategy Officer Ryan Richardson stated that BioNTech is working with Pfizer on a next-generation flu-COVID combo vaccine. While it's too early for a precise roadmap, he expressed confidence that issues from the initial trial can be addressed through construct optimization, supported by early evidence. He indicated that updates and a more definitive roadmap are planned for 2025.

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Dr. Christopher Anzalone, President and CEO, explained that plans for the ARO-RAGE Phase II study are currently in development as they evaluate patient types and treatment duration. He provided no new guidance on data timing for other programs, stating data will be presented once studies are complete and results are interpretable.

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The company stated that the German ruling on one patent won't affect others, as each is judged on its merits. The potential value for damages is tied to the patent's grant date, with earlier patents being more valuable. They highlighted the strength of their broad IP portfolio.

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