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CEO Dr. Matthew Klein affirmed that they expect Sephience revenue from Europe in 2025, starting with Germany and other early access programs. CFO Pierre Gravier stated that with over $2 billion in cash, PTC is actively evaluating business development opportunities for both commercial and pipeline assets.

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CEO Dr. Matthew Klein stated the Translarna review is active with an outcome expected in H1 2025, but no official PDUFA date is anticipated. He also confirmed longer-term PIVOT-HD data is possible. CFO Pierre Gravier clarified that 2025 guidance includes very limited EU Translarna sales, creating potential for upside.

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CEO Matthew Klein clarified that the two points were unrelated. The endpoint was changed to a Joint RANK test following FDA feedback to better incorporate mortality data into the analysis of the ALSFRS score. The over-enrollment was a separate measure to ensure the target number of patients for the primary analysis was achieved due to a lower-than-expected screening yield.

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CEO John Jacobs reiterated that based on formal FDA communications, the request is for a post-marketing commitment, which occurs after approval. CFO James Kelly confirmed that the BLA approval milestone from Sanofi is not impacted by a post-marketing commitment requirement and remains at $175 million.

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President and CEO John Jacobs stated that Novavax is still working with regulatory authorities on the approval pathway for the CIC program. He noted that the upcoming data from the initial cohort will be informative to that process and that the company will communicate the final path forward once it is determined.

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CEO John Jacobs deferred committing to a specific trial readout timeline until a start date is finalized. Chief Medical Officer Dr. Robert Walker clarified that safety follow-up will be the standard length for adjuvanted vaccines. He confirmed the Phase IIIa study is designed to support licensure for both the CIC and the stand-alone flu vaccine, powered for non-inferiority, with a post-approval efficacy trial likely required.

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Dr. Mark Goldsmith, CEO, affirmed that the response rate-PFS link is a well-known concept. Dr. Wei Lin, CMO, addressed the QT signal, stating it is on par with or lower than monotherapy observations and is not expected to be enhanced in the triplet, remaining within the range of other approved drugs. Dr. Goldsmith also confirmed that dose optimization is ongoing, implying continued enrollment in the cohorts.

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CEO Dr. Mark Goldsmith deferred commenting on the regulatory bar pending discussions with authorities. Chief Medical Officer Dr. Wei Lin stated that the proportion of resectable cases is unlikely to change in the short term due to a lack of standard screening tests. Dr. Goldsmith added that future screening tools, like ctDNA tests, could increase early diagnosis and expand this patient population over the long term.

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Dr. Wei Lin, CMO, confirmed that a quadruplet combination of RMC-6236, pembrolizumab, and platinum-doublet chemotherapy is planned, to be initiated after an appropriate dose for the doublet is established. Jack Anders, CFO, addressed the financials, stating there were no one-time items in the OpEx increase. He noted that expenses are expected to continue increasing in 2025 due to advancing Phase III trials and commercial readiness activities.

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Chief Commercialization Officer Adam Waldman responded that he did not recall the specific 70% figure but emphasized that persistence trends for Briumvi remain 'very positive' and 'strong,' continuing to track above the company's internal expectations.

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CEO Michael Weiss explained that many physicians were already switching patients from other anti-CD20s to BRIUMVI without the introductory dose, and the ENHANCE data provides safety reassurance for this practice. He believes a formal label update would be needed for broad adoption of the 30-minute infusion. Regarding myasthenia gravis, Weiss noted that while the market is not as underserved as it once was, BRIUMVI could offer a highly active, convenient, and cost-effective option compared to existing expensive treatments.

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CEO Michael Weiss explained that randomized, safety-focused trials would be needed for a faster infusion label update. For the subcutaneous program, he stated the primary focus is on bioequivalence to match the PK profile, which then determines the volume and tolerability. He also noted that B-cell decline is not an issue at any dose level being studied.

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Chief Development Officer Anne-Marie Li-Kwai-Cheung explained that 'supportive' means the FDA is open to and engaged on a potential accelerated approval pathway for Huntington's disease using biomarkers like caudate atrophy. CEO Paul Bolno added that the second half of 2025 IND timeline accounts for both detailed study planning and the necessary CMC and manufacturing work required to support a potentially registrational trial. He also noted that external research on caudate atrophy is providing supportive data the FDA has been looking for.

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Chief Medical Officer Dr. Amy Simon clarified that the elevated hemoglobin levels are considered lab abnormalities without clinical symptoms and are not classified as adverse events. Regarding the patient death, she stated that no changes were made to the protocol as the event was within the known risk profile of busulfan, and that therapeutic drug monitoring was already in place.

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Chief Medical Officer Dr. Amy Simon stated the elevated hemoglobin was a mild, asymptomatic lab abnormality not requiring intervention, and the overall improved blood health profile mitigates concern. She confirmed no changes were made to the protocol, as the DMC and FDA agreed the safety profile was unchanged, and noted that therapeutic drug monitoring for busulfan was already in place.

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Dr. Mark Eisner, Chief Medical Officer, reiterated Vir's commitment to advancing the combination of tobevibart and elebsiran, which has received Fast Track Designation from the FDA. He noted that further details on the registrational program design will be disclosed at the company's investor event in November.

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CMO Jared Gollob confirmed the H1 2025 data release will include the full SAD/MAD dataset, with a design comparable to the prior IRAK4 study. CEO Nello Mainolfi added that the trial will enroll up to 120 subjects and that at least one new immunology program will be unveiled next year, though likely closer to clinical entry for competitive reasons.

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Senior Director of Immunology Veronica Campbell and CMO Jared Gollob responded. Veronica Campbell clarified that they don't expect broad immunosuppression because IRF5 is selectively expressed and key antiviral transcription factors like IRF3 and IRF7 are left intact. Jared Gollob noted they have shown superior or comparable activity to other agents like TYK2 inhibitors in lupus models and that IRF5's broader mechanism may be more active than B-cell depleters alone.

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