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Interim CEO Frederick Vogt and CCO Dan Kirby confirmed that patient infusions grew from 85 in Q1 to 102 in Q2 2025. They stated the price increase to $562,000 on April 1st did not negatively impact demand. While declining to provide quarterly patient forecasts, they reiterated confidence in the full-year revenue guidance of $250M to $300M, which implies continued strong demand in the second half of the year.

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Interim CEO Frederick Vogt acknowledged the guidance is conservative and could be exceeded. Chief Financial Officer Jean-Marc Bellemin confirmed the increase in COGS in Q1 was due to a spike in out-of-spec manufacturing relative to lower revenue, but stated this is expected to improve in Q2 and that standard gross margin remained positive.

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Interim CEO and President Frederick Vogt reported approximately 95 revenue infusions for Q4. He described the quarter-over-quarter growth as strong and sufficient to meet their targets, noting that growth will fluctuate, similar to early CAR-T launches, but the company is focused on driving continued growth.

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Interim CEO Frederick Vogt stated that there was month-over-month growth in Q3 infusions and this trend is expected to continue. He clarified that growth is primarily driven by manufacturing capacity and slot availability, which accommodates rising demand from both new and existing ATCs, with improving out-of-spec rates also contributing.

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CEO John Leonard stated they see no significant clinical difference between stabilizer generations. EVP & CMO David Lebwohl added that the trial design anticipated the availability of silencers and prohibits their use as a first-line agent for trial entry. He noted that potential crossovers from tafamidis to vutrisiran during the trial are factored into the statistical analysis.

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Chief Medical Officer David Lebwohl confirmed that enrollment remains brisk globally, including in the U.S., and has even accelerated recently despite Amvuttra's approval. He noted that physicians intending to use Amvuttra are advised not to enroll patients in the study, and the company does not expect significant crossover, as the trial offers a chance to add a potentially superior therapy on top of tafamidis.

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CEO John Leonard explained that the update would extend the follow-up on previously reported measures. Key learnings would be to confirm a continued low rate of disease progression, observe if more patients show improvement from baseline, and validate the low clinical event rate, which provides further confidence for the ongoing Phase III trial.

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CEO John Leonard explained the small size is possible due to the large expected effect size of TTR reduction. He and CMO David Lebwohl clarified that the placebo-controlled design was agreed upon with the FDA and is feasible because the trial is conducted ex-U.S. where silencers are not available, meaning patients will not receive them.

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CEO Brett Monia confirmed the combined CORE studies have accumulated more total AP events than the FCS study, though the rate is lower. CGO Kyle Jenne stated the FCS market size aligns with their estimate of up to 3,000 patients and noted that the pricing strategy for the SHTG indication is still under review, with the price to be announced upon approval.

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Chief Global Product Strategy Officer Kyle Jenne attributed WAINUA's growth primarily to new patient starts in an expanding market, though he noted patient switches are occurring, driven by the convenience of self-administration. CEO Brett Monia expressed enthusiasm for regaining full rights to the alpha-synuclein and LRRK2 programs, framing Biogen's decision as one of R&D prioritization and affirming Ionis's commitment to advancing both assets.

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Chief Global Product Strategy Officer Kyle Jenne expressed confidence in payer acceptance for olezarsen, citing education on the ultra-rare disease and strong hospitalization data. CEO Brett Monia confirmed commitment to the 140-week CARDIO-TTRansform readout but left the door open for what's best for the drug. He highlighted the strengths of their Angelman trial design, including its placebo-control as the 'gold standard,' without directly comparing it to a competitor's.

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CMO Walid Abi-Saab clarified that the SAP submission contained methodologies, not results. However, he noted that in their internal evaluations, both methods yielded very similar estimates of disease decline. He confirmed that weighting uses a larger control group (~3,000) while matching uses a smaller subset, but the final estimate is robust and similar between methods.

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CMO Dr. Walid Abi-Saab confirmed the primary analysis will be on 3-year data and that the choice of natural history study (e.g., TRACK-HD vs. Enroll-HD) does not significantly alter outcomes with proper matching. CEO Matthew Kapusta declined to comment on competitor data directly, instead highlighting uniQure's own strong results, bioavailability, and differentiated therapeutic target.

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CEO Sean Nolan clarified that the FDA endorsed the design Taysha proposed. He attributed the streamlined process not to FDA resource issues but to the productive, data-driven discussions enabled by the RMAT designation, which allowed them to achieve alignment on key trial elements without needing a formal meeting to resolve disagreements.

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CEO Sean Nolan positioned the competitor's data as a positive for the Rett community, validating MECP2 as a therapeutic target, but refrained from direct comparisons. He confirmed that Taysha could dose higher, noting their non-human primate toxicology studies were clean at 2x10^15 vg, but stated that the current high dose is considered optimal based on preclinical evidence.

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CEO Sean Nolan confirmed the first-half guidance provides flexibility and expressed confidence in meeting it. He acknowledged the current protocol specifies a 42-day stagger but hinted that this is a topic under consideration and could potentially change in the future, contingent on continued positive safety data. He affirmed the company is comfortable with its current guidance.

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President and CEO Douglas Ingram (speaking in place of Dallan Murray) explained the guidance was lowered due to a multifactorial drop-off, including the safety event's impact, but the company is now seeing an uptick in start forms that supports a projected summer recovery. Regarding peak sales, he stated the ultimate opportunity (area under the curve) remains unchanged, but the timing to reach peak sales may be delayed, and the company will update its view later.

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President Dr. Giuseppe Ciaramella explained that achieving a >60% HbF to <40% HbS ratio effectively mimics the asymptomatic profile of sickle cell trait. He emphasized that HbF is anti-sickling and that biomarker data shows a near-complete elimination of the HbS-only cells responsible for sickling, suggesting a potential for deeper and more durable disease resolution than current treatments.

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President Dr. Giuseppe Ciaramella explained that achieving an HbF/HbS ratio of over 60/40 mimics the profile of asymptomatic sickle cell trait individuals. He added that HbF is inherently anti-sickling and that biomarker data shows a near-complete elimination of the HbS-only cells most likely to cause sickling, suggesting the potential for a deeper and more durable clinical resolution beyond just VOCs.

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