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CCO Tsveta Milanova confirmed the market access team is actively educating payers and aims for strong access similar to their success in PK deficiency. CMO Dr. Sarah Gheuens stated the full ENERGIZE-T data will include key secondary endpoints and safety details, and noted that ASH abstracts would be released on November 5, indicating a potential presentation.

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CCO Tsveta Milanova identified the Gulf (GCC) region as the next priority after the U.S., leveraging a partnership with NewBridge. CFO Cecilia Jones described the NewBridge deal as a capital-efficient revenue-split model and indicated a similar structure is planned for Europe. CEO Brian Goff clarified that the new hire reinforces, rather than shifts, their strategy, which prioritizes successful launches, advancing the internal pipeline, and disciplined business development, all supported by a strong balance sheet.

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Chief Commercial Officer Tsveta Milanova reported high awareness among physicians and strong excitement from patient advocacy groups, noting the sales force has been doubled in size for the launch. Chief Medical Officer Dr. Sarah Gheuens confirmed that based on the positive benefit-risk profile across both pediatric studies, Agios intends to seek a broad label covering the full pediatric PKD patient population.

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President & CEO Curran Simpson clarified that the two pivotal studies are sufficient for filing, while ACHIEVE is a post-marketing study. He also stated that over 90% of the prevalent DMD population is expected to be available at launch. CMO Dr. Steve Pakola argued against a class effect label for liver injury, highlighting RGX-202's differentiated product purity and proactive immune regimen, which has resulted in zero liver injury events in the Phase 1/2 study to date.

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CEO Curran Simpson addressed the commercial outlook, stating they expect at least half of the prevalent DMD market to remain available for their potential launch in early 2027 and that the company is already preparing commercial supply. Chief Medical Officer Dr. Steve Pakola confirmed that the 12-week microdystrophin data is the primary endpoint for accelerated approval and would certainly be released. Mr. Simpson added that at the time of filing, they will also have a 'healthy level' of functional data from both the Phase I/II and pivotal cohorts.

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CEO Mark Goldsmith explained that ongoing studies for zoldonrasib and oleronrasib are being monitored for latent safety signals and expanded to ensure robust data, but declined to detail specific data triggers for future decisions. Regarding the Iambic collaboration, he stated that RevMed's massive proprietary dataset of tri-complex inhibitors will be used to train Iambic's AI models, potentially enhancing the efficiency of lead discovery and optimization for both RAS and non-RAS targets.

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Dr. Mark Goldsmith, Chairman and CEO, clarified that the hematology program has been almost entirely outsourced to academic collaborators and is not a top internal priority. The company remains focused on pancreatic, non-small cell lung, and colorectal cancers. While acknowledging the unmet need in AML, particularly as a resistance mechanism, he stated it is not a focus for near-term clinical development but could be considered in the future.

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EVP, Head of Research & Development Ruxandra Draghia-Akli explained the PMC study will provide additional data in a specific population and is expected to start by year-end. President, CEO & Director John Jacobs noted the $70-$90 million cost reflects its scale. Regarding the regulatory environment, Jacobs and EVP, Chief Corporate Affairs & Advocacy Officer Silvia Taylor suggested that reduced focus on mRNA platforms could create opportunities for Novavax's protein-based technology, particularly for its pandemic influenza candidate and seasonal execution with Sanofi.

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CFO James Kelly clarified that the $603 million in APA revenue recognized was non-cash, relating to the retention of cash received in prior years. Head of R&D Dr. Ruxandra Draghia-Akli explained that the CIC study will supplement the platform's already favorable safety database and that the data, available in the second half of the year, will be key for partnership discussions.

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President and CEO John Jacobs confirmed the strategy is to find a partner to fund further CIC development. Head of R&D Ruxandra Draghia-Akli and President and COO John Trizzino detailed that the new RSV program is not a 'redo,' but a new approach using AI/machine learning, a modified antigen construct, and the Matrix-M adjuvant to create a superior, differentiated combination product.

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President and COO John Trizzino provided guidance on net pricing, stating that while the gross price is around $140 per dose, a reasonable net price assumption would be in the 50% to 75% range of gross, or $70 to $100, subject to fluctuations from returns. The question regarding the non-retail channel split was not directly addressed, though Chief Medical Officer Dr. Robert Walker did provide a clarification on total doses administered based on IQVIA data.

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CEO Michael Rossi confirmed the 2A designation was expected for an ultra-rare disease and is the same as the competitor, creating a level playing field. Regarding the FDA, he stated that Y-mAbs maintains close working relationships and continues to move forward effectively, expressing a positive outlook on their interactions regardless of administrative changes.

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CEO Michael Rossi noted that growth plans for DANYELZA will take time to ramp up in 2025. CFO Peter Pfreundschuh elaborated on the guidance, advising analysts to use the midpoint of the wide full-year range and confirming the Q1 guidance is solid. He stated the company intends to provide quarterly guidance updates to account for seasonality and stocking effects. Regarding SADA, Rossi identified the kidney, liver, and bone marrow as key areas for monitoring concentration and potential dose-limiting toxicities, emphasizing that the focus is on actual tissue injury, not just concentration.

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President and CEO Michael Rossi stated that the manufacturing philosophy remains unchanged, utilizing contract manufacturing organizations (CMOs) for both DANYELZA and SADA proteins to invest in drug development rather than infrastructure. Regarding Japan, he explained that a small, 6-patient clinical trial is required, with a potential launch in the second half of 2025 or early 2026. He characterized Japan as an important but smaller market than the U.S., representing an incremental, not massive, expansion of the TAM.

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President and CEO Michael Rossi acknowledged that recruitment was initially slow but has since accelerated for the GD2 trial. He expects a similar pattern for the CD38 trial as physicians gain confidence in the safety data. Chief Medical Officer Dr. Vignesh Rajah highlighted the complexity of Part A, which involves a stepwise process of testing various dose levels and intervals to determine the optimal combination before proceeding to Part B.

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CEO Jeremy Bender reiterated that the company expects a consistent and steady ramp in new patient starts, similar to a rare disease launch rather than a typical oncology product. COO & CFO Charles York added that channel stock remains within the target range of 2-4 weeks of days on hand, with no material impact on the quarter's results beyond the consistent build accompanying sales growth.

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Chief Commercial Officer Lauren Merendino confirmed approximately 800 total prescriptions were added in Q4. Chief Operating and Financial Officer Charles York added that while channel stock volume increased in absolute dollars due to growing demand, the company remains within its target of 2-4 weeks of inventory on hand. He also noted the improved gross-to-net as a key factor in Q4 revenue.

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CEO Jeremy Bender stated that the cadence of new patient starts was very consistent, without seasonal effects. Dr. Samuel Blackman, Head of R&D, explained that the enhanced duration of response data builds prescriber confidence, though clinical decisions on treatment length remain separate from radiographic progression criteria.

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Krish Krishnan, Chairman and CEO, confirmed a minor impact from insurance changes that should be recovered in Q2. He dismissed concerns about the market size, attributing the slower pace of new patient starts to the longer time required for sales reps to educate physicians in community settings. He noted that the company has already begun increasing its commercial team to address this.

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Krish Krishnan, Chairman and CEO, confirmed there were no significant changes in Q4 stocking or inventory levels compared to prior quarters. He explained that the company recalculates the price cap's impact quarterly to smooth out revenue, acknowledging its complexity but stating they have successfully removed volatility from the results.

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Chairman and CEO Krish Krishnan explained that the company does not disclose the specific number of patients hitting the annual cap, emphasizing that the goal is to maintain smooth net revenue growth. Regarding wound closure, he stated that while some patients experience extended durability, the company's thesis remains that the average wound is durable for about 90 days based on collagen half-life. An executive added that as patients become more active, they are eager to restart therapy quickly if new wounds appear.

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CEO John Leonard clarified that all patients have responded and are better off, suggesting the term 'incomplete responders' is inappropriate. He noted that responses mature over time, as seen in Phase I. CMO David Lebwohl added that patients tend to improve with longer follow-up and that more data on these patients will be presented later in the year.

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CFO Ed Dulac stated that with a $945 million cash balance, the company's priority is its three Phase III studies. He also highlighted the importance of the NTLA-3001 gene insertion program and ex vivo opportunities. He noted the current cash runway extends well into the fourth quarter of 2026.

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CEO Dr. Marianne De Backer explained that Vir is defining a set of high-value next-generation targets and is also engaging with potential partners to understand their interests. Dr. Mika Derynck, EVP of Oncology, stated that based on the confidence built from the other programs, the VIR-5525 study will start without any prophylactic steroids, noting the minimal CRS observed to date.

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Dr. Mark Eisner, Chief Medical Officer, stated that Vir will share preliminary monotherapy data in Q1 but did not specify the exact content. He explained the dual-masking hypothesis is to improve the therapeutic index by enabling higher efficacy with good safety, noting this differentiates their assets from competitors like Janux's PSMA program.

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