Brian Abrahams • RBC Capital Markets

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President and CEO Ian Mortimer clarified that the target remains approximately 360 subjects and the study is highly powered, making over-enrollment unnecessary. He emphasized that the primary goal for EXTOL-two is achieving statistical significance to support the NDA filing. Chief Medical Officer Christopher Kenney added that blinded metrics on patient characteristics and rollover rates appear consistent with the successful Phase IIb EXTOL study.

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President and CEO Ian Mortimer characterized the delay as minor, resulting from normal trial variability with no change in competitive dynamics. Chief Medical Officer Dr. Chris Kenney stated that while the investigator-sponsored trial (IST) results are reassuring, they will not alter the existing Phase III MDD program, attributing the strong IST data to the controlled nature of a two-site study.

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CEO Ian Mortimer highlighted that the 36-month data will show continued seizure reduction and an increasing 12-month seizure freedom rate with a consistent safety profile. CMO Dr. Christopher Kenney noted updated retention rates will also be presented. CCO Christopher Von Seggern emphasized AES as a key opportunity to engage with future prescribers and gather feedback on azetukalner's market potential.

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CEO Dr. Matthew Klein confirmed no OpEx changes are expected for the Sefiance launch, as it leverages existing infrastructure. CBO Eric Pauwels added that the European opportunity is significant, with Germany being the second-largest market. He anticipates leveraging early access programs in 5 to 10 other key European markets this year and into next, in addition to upcoming launches in Japan and Brazil, to build global momentum.

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CEO Dr. Matthew Klein expressed satisfaction with the Phase II data, noting it confirmed target engagement, safety, and positive biomarker signals. He stated the results support the hypothesis that Stage 2 patients are the optimal trial population and that the data on HTT and NFL lowering provides a strong basis for discussions with regulators about an accelerated path.

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CBO Eric Pauwels stated that extensive payer research indicates a clear understanding of sepiapterin's value proposition, including diet liberalization and improved Phe control. He noted that payers see no issue with first-line use in poorly controlled or therapy-failed patients and have indicated a willingness to pay a premium price.

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CEO Matthew Klein addressed Translarna, stating the European Commission has approximately 67 days for its review, while the U.S. FDA has no set PDUFA date for the resubmission but a review is expected in the coming months. CBO Eric Pauwels added that for sepiapterin, payers recognize the drug's differentiated efficacy and the high unmet need in PKU, which supports a premium pricing strategy.

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Chief Commercial Officer Thomas Garner noted clear receptivity outside COEs but acknowledged that these physicians require more ongoing education due to seeing Rett patients less frequently. Regarding persistence, he reported that the 12-month rate remains above 50% and the 18-month rate is now above 45%, indicating a stable, long-term patient base.

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President & CCO Charlie Gayer noted that while they monitor policy, they see no immediate impact from MFN and that Medicaid represents a small patient segment (10-15%). CFO Babar Ghias outlined a strategy to become a 'consolidator of rare disease assets,' leveraging the company's strong cash flow to acquire late-stage or commercial products. CEO Jon Stonehouse added that the current capital-constrained environment for many biotechs creates significant opportunities for BioCryst.

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Chief Commercial Officer Charlie Gayer firmly denied any dip in patient numbers, citing extremely consistent demand and patient retention. CEO Jon Stonehouse clarified that the quarterly revenue pattern has shifted due to the rapid conversion to paid drug, but underlying growth remains strong. Regarding patient preference, Gayer explained that after four years on the market, patients and physicians are now confident that ORLADEYO delivers both high efficacy and the convenience of an oral therapy, which is driving the preference shift.

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Chief R&D Officer Dr. Helen Thackray confirmed the pediatric oral granules were well-tolerated, with a profile similar to adults, and that the regulatory path is a straightforward pediatric extrapolation. CEO Jon Stonehouse added that filings are also planned in Japan. CFO Anthony Doyle explained the Q4 ex-U.S. uptick was driven by distributor shipment timing and a revised agreement in Japan, but noted strong U.S. growth may temper the ex-U.S. percentage of total revenue in 2025.

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Chief Commercial Officer Charlie Gayer attributed the Q4 guidance to typical seasonality, including a slowdown in achieving paid drug status for new patients late in the year. For 2025, he confirmed no slowdown in demand is being observed and expressed confidence in continued strong performance, while CEO Jon Stonehouse reiterated the company's target of a 20% compound annual growth rate through 2029.

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President & CSO Dr. George Yancopoulos noted that one of the two studies 'turned south' after six months. He speculated that the COVID-19 pandemic created unusual trial conditions, such as lower exacerbation rates, which may have been a factor. He confirmed they are discussing next steps, including a potential additional Phase 3 study.

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President & CSO Dr. George Yancopoulos noted that one of the two replicate studies 'turned south' after six months. He speculated that a major factor was the trial's execution during the COVID-19 pandemic, when exacerbation rates dropped precipitously worldwide. He confirmed they are analyzing the data and discussing a potential additional Phase 3 trial with Sanofi.

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President and CEO Dr. Leonard Schleifer highlighted that the rationale stems from the Regeneron Genetics Center, which links the IL-33 pathway to COPD risk. He noted the Phase III studies passed an interim analysis, boosting confidence. CSO Dr. George Yancopoulos added that strong genetic links support expansion into other areas, such as ASCO, pending the COPD results.

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EVP of Commercial, Marion McCourt, reiterated that Regeneron does not provide guidance but expressed confidence in EYLEA HD's strengthening profile due to an upcoming prefilled syringe, a potential RVO indication, and enhanced dosing flexibility. She acknowledged expected competitive pressure on standard EYLEA but highlighted the franchise's strong 46% market share in Q4.

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President and CEO Dr. Leonard Schleifer confirmed the company anticipates launching the prefilled syringe by mid-2025. He acknowledged a market preference for this format and agreed it could create an inflection point in adoption. He noted that there are additional U.S.-specific requirements the company is working through to ensure the product is highly reliable and avoids issues like inflammation.

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Alisha Alaimo, President & Head of North America, noted that adoption is evolving at an "incredible pace," with testing nearly tripling in the past year. She explained that physicians currently use them for triage but still confirm with PET or CSF. The key to broader adoption is establishing them as the standard for confirmation, which requires education on new guidelines, real-world evidence, and clarity on reimbursement.

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Alisha Alaimo, President & Head of North America, reported that adoption is evolving at an "incredible pace," with testing nearly tripling in the past year. She noted that while awareness is high, the tests are currently used mainly for triage, with physicians often still confirming positive results with PET or CSF. She stated the key to broader adoption is education on new guidelines, real-world evidence, and clarity on reimbursement.

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CEO Christopher Viehbacher explained that the European rollout will be a gradual, market-by-market process. He noted that as a first-in-class therapy, LEQEMBI is an incremental addition to healthcare budgets, which requires time for adoption. However, he believes the rigorous EMA approval process, which assessed efficacy, safety, and economic impact, will aid in reimbursement discussions across the continent.

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President and CEO Christopher Viehbacher described the strategy as 'looking for needles in haystacks,' utilizing AI, increased genetic testing, and omnichannel marketing to find patients. He noted that U.S. reimbursement is manageable with patient support, while ex-U.S. reimbursement is a country-by-country process where strong initial uptake is encouraging for negotiations.

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CFO Michael McDonnell noted the launch is complex and the collaboration is reviewing strategies, highlighting the future subcutaneous version as a key catalyst. Alisha Alaimo, President of North America, added that new prescribers grew 40% in Q3 and the expanded sales force, in the field since September, is already showing accelerated growth in specific territories due to established physician relationships.

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CEO Kyle Gano explained the decision was driven by a strategy to maximize patient access and gain flexibility ahead of the IRA implementation. These contracts were initially targeted for 2026 but were accelerated into 2025. CFO Matt Abernethy added that this sets them up well for 2026, and he does not anticipate further major contracts that would significantly alter the pricing trajectory from the 2025 exit rate.

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Chief Commercial Officer Eric Benevich explained CRENESSITY adoption is broad across all endocrinologist segments, not just centers of excellence. Chief Medical Officer Dr. Eiry Roberts added that the drug's label allows for individualized steroid reduction. On INGREZZA, Benevich noted recent contracts expanded Medicare access and no further immediate agreements are anticipated, providing stability into 2026.

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CCO Eric Benevich stated that Q3 growth was primarily driven by new patient demand and strong compliance. He acknowledged the market is growing and that while competitors are also growing, INGREZZA remains the most prescribed VMAT2 inhibitor. He emphasized that with less than 20% of the market treated, there is a long opportunity ahead, supported by the recent sales force expansion.

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CEO Bill Meury characterized the pediatric indication as an 'incremental growth driver,' with the core AD and vitiligo businesses being the primary engine. He noted strong ex-U.S. growth in key European markets and Canada. EVP & GM of U.S. Dermatology Matteo Trotta added that the pediatric approval would be a 'great tailwind' for the brand's already strong value proposition.

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Pablo Cagnoni, President, Head of R&D, positioned the BET inhibitor as a very good near-term option for patients who progress after Jakafi, which is why the second-line program is being accelerated. For frontline use, he stated that more data is needed on the combination's safety and efficacy, noting that while ASH data was encouraging, more evidence is required before deciding on a first-line study.

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Steven Stein, an executive, detailed the strategy for the CDK2 inhibitor in ovarian cancer. For the platinum-refractory setting, a single-arm study could support a U.S. approval, while a randomized study would be needed for ex-U.S. markets. He also expressed significant interest in the platinum-sensitive maintenance setting in combination with bevacizumab. For the companion diagnostic, he noted they are focused on Cyclin E1 protein expression via IHC, which represents a larger patient population, and are in discussions with regulators.

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President and CEO Douglas Ingram confirmed positive and consistent interactions with the FDA's OTP, with no change in approach. Head of R&D Dr. Louise Rodino-Klapac added that OTP confirmed the accelerated approval pathway for SRP-9003 (LGMD 2E) and accepted a rolling review, keeping the program on track. She did not comment on the specific timing of the public data disclosure.

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CEO Douglas Ingram and CCO Dallan Murray clarified there is no fundamental capacity issue, with over 75 sites available, which is sufficient even for peak years. Murray explained that centers are currently in an adjustment period, managing the unprecedented overnight demand from the entire Duchenne population. He expressed confidence in the centers' ability to ramp up, as they have successfully managed previous complex launches like Zolgensma.

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Chief Commercial Officer Johanna Mercier detailed a highly targeted launch plan, noting that awareness is already high. The initial focus is on the core group of HIV prescribers who also handle PrEP. She confirmed readiness to support both 'buy and bill' and 'white bagging' through specialty pharmacies. A full field team, including nurse educators and reimbursement managers, is mobilized to ensure a smooth customer experience and assist with medical exceptions during the access build-out phase.

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CCO Johanna Mercier reported that the Livdelzi launch is exceeding expectations with strong week-over-week growth and positive feedback on its differentiated profile. She noted that use is primarily in second-line naive patients, per the label, with good payer access. She anticipates the strong momentum will continue through 2025.

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Johanna Mercier, Chief Commercial Officer, estimated that Medicaid represents the mid-to-high 20% range of the total HIV business. She explained that patients have fallback options like the ADAP program, so access is generally not an issue due to various 'parachutes.' Mercier noted that the company is more closely tracking the growth of the 340B channel and will monitor any potential Medicaid changes, though none have been announced.

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President and CEO Gene G. Kinney expressed confidence in productive dialogues with global regulators, including in Europe, due to the universal unmet medical need. COO Brandon Smith outlined a two-phase commercial build-out: first, market education on the mechanism of action leading up to the launch, and second, building the brand identity and ensuring seamless patient access post-approval.

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