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Chief Medical Officer David Lebwohl confirmed that enrollment is progressing well and that the company expects over 50% of patients to be on baseline tafamidis, which is valuable for demonstrating added benefit. He noted that while no patients have crossed over to a silencer therapy yet, the trial's statistical plan accounts for this possibility.

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CEO John Leonard expressed high confidence, citing the company's track record of under-promising and over-delivering. He stated that enrolling 550 patients by year-end is a 'very highly likely outcome' based on robust current enrollment rates and that the study is expected to complete enrollment well within the 2027 timeframe.

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Robert I. Blum, President and CEO, stated that the MAPLE data will not be submitted during the current review cycle to avoid disruption but will be part of a subsequent submission post-approval. Fady Malik, EVP of R&D, confirmed the submitted REMS aligns with aficamten's previously discussed differentiated properties, such as less frequent monitoring and minimal drug-drug interactions.

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CEO Reshma Kewalramani declined to provide more detail on the VX-522 pause to maintain study integrity, reiterating that the team is assessing a tolerability issue. COO Stuart Arbuckle explained that comparing ALYFTREK's launch to ORKAMBI or TRIKAFTA is difficult because those drugs opened up treatment to tens of thousands of new patients, whereas ALYFTREK's new patient population is smaller. He affirmed the company is pleased with the launch to date.

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CEO Jon Stonehouse described the paid rate improvement as a 'snapback.' Chief Commercial Officer Charlie Gayer elaborated that it was driven by the IRA resolving Medicare affordability issues and the team's use of long-term real-world data to convince commercial payers. For the DME program, Chief R&D Officer Dr. Helen Thackray stated they are looking for a 'clear and uncontroversial reduction in edema' rather than a specific micron number, which would signal that the dose is effective. If not, the dose will be increased.

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Chief Commercial Officer Charlie Gayer stated that Medicare patients constitute about 20% of the total patient base and their paid rate was around 55-56% at year-end, a figure that is now improving. Chief R&D Officer Dr. Helen Thackray explained the Phase 1 trial design involves dose escalation in healthy volunteers to establish safety before moving to patients to assess KLK5 activity, with the goal of achieving nearly complete suppression of the target.

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CEO Jon Stonehouse stated the Medicare trend is positive, with the paid rate already improving to 55%, and the only uncertainty is the speed of further improvement. Chief R&D Officer Dr. Helen Thackray confirmed that skin biopsies are a critical first step for BCX-17725 to demonstrate drug penetration and target binding. Key biomarkers will assess KLK5 inhibition and potentially downstream cytokines, with the drug's potency possibly allowing for extended dosing intervals.

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Greg Friberg (Chief R&D Officer) said the BMN 333 PK goal is to show multifold increases in sustained free CNP exposure. He dismissed competitor growth hormone combos, citing historical data showing transient benefits and added risks. Brian Mueller (CFO) confirmed that VOXZOGO drug substance is sourced from the United States.

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CEO Alexander Hardy confirmed that IP action was initiated in Munich against a competitor with a decision expected in 12-15 months, and that the company will defend its IP in the U.S. as needed. Chief R&D Officer Gregory Friberg explained that for BMN 349, the healthy volunteer study may provide early pharmacodynamic insights into circulating Z protein levels from a small number of AATD patients enrolled.

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CEO Yvonne Greenstreet and CCO Tolga Tanguler clarified that none of the Q1 revenue was from the ATTR-CM indication, as approval occurred in the final days of the quarter. Tolga Tanguler avoided direct competitor comparisons, instead pointing to Alnylam's full-year guidance of approximately 36% TTR franchise growth and emphasizing that the overall treatment category is expanding.

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CEO Yvonne Greenstreet reiterated that pricing considers the product's value and ensuring optimal access. Chief Commercial Officer Tolga Tanguler confirmed that a J-code for AMVUTTRA is already in place, so no disruption is expected. He also highlighted patient affordability, noting that 70% of current patients have a $0 co-pay, a dynamic he expects to continue.

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CFO Jamey Mock attributed the low Q1 revenue to customer inventory management rather than market share loss, pointing to script data showing a stable 38% share. President Stephen Hoge declined to provide a specific number of events for the flu analysis but confirmed it was a large number due to an intense flu season.

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President Stephen Hoge stated that for the Norovirus hold, the FDA needs time to review the submitted materials and that the outcome is up to the agency. For the CMV vaccine, he confirmed cases continue to accrue and results are still expected in 2025, but did not commit to a specific half of the year.

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CFO Jamey Mock confirmed the analyst's pricing calculation was not far off and stated that a $140 million provision was released from the prior season's return reserve. President Stephen Hoge explained that a priority review voucher for the combo vaccine was not used because it would not accelerate approval in time for the 2025 contracting season.

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Chief Medical Officer Dr. Adrian Kilcoyne confirmed that the full Phase I dose escalation data set for UCART22 will be released in Q3, with additional data to be shared at the ASH conference. Chief Business Officer Arthur Stril added that the primary data release is planned for a company-hosted ad hoc event, with more details to be provided soon.

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CEO Curran Simpson responded that they expect to present microdystrophin data for one patient from the 1-to-4-year-old cohort at the MDA conference, noting a general trend of higher expression in younger patients. Chief Medical Officer Dr. Steve Pakola addressed the second question, stating that while no specific timing has been guided for the ALTITUDE DME cohort data, any update would include standard endpoints like visual acuity, retinal thickness, treatment burden reduction, and safety.

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CEO Dr. Matthew Klein stated the FDA has not yet decided on an AdCom for vatiquinone. For PIVOT-HD, he explained the FDA was not prescriptive on a specific correlation metric but wants to see associations between HTT lowering and clinical measures. He added that the entire biomarker package, including both blood and CSF, is important for the assessment.

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Chief Scientific Officer Dr. Louise Rodino-Klapac confirmed that the data readouts for both FSHD1 and DM1 in the second half of the year will be from the SAD studies. The data will focus on safety, muscle concentration, and early biomarker signals like knockdown and splicing restoration. CEO Douglas Ingram added they are particularly excited about the potential to directly measure DUX4 protein knockdown in FSHD, a significant proof-of-biology milestone.

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Dr. Louise Rodino-Klapac, Head of R&D and Chief Scientific Officer, confirmed the entire PPMO franchise is being discontinued. She explained the decision was multifactorial, based on FDA feedback that the accelerated approval pathway was not open, an internal benefit-risk assessment citing prolonged hypomagnesemia, and the evolving Duchenne treatment landscape with ELEVIDYS. CEO Doug Ingram added that the high safety bar for PMOs was a key consideration.

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CEO Douglas Ingram clarified that 'modest' Q3 growth for ELEVIDYS translates to a robust 30% increase over Q2, followed by a doubling of revenue in Q4. He confirmed the 2025 guidance is largely in line with external consensus, attributing the company's confidence to deep experience with Duchenne launches, granular data on patient start forms, and strong knowledge of the epidemiology.

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The company confirmed the vast majority of the 30 scheduled 2025 starts are for Q1. They expect commercial LYFGENIA cell collection cycles to be similar to clinical trials (1-2 cycles for 85% of patients). To manage the cash gap, they are renegotiating key contracts and working closely with their partner, Hercules.

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CEO Sandy Macrae and Chief Development Officer Nathalie Dubois-Stringfellow responded. Dubois-Stringfellow confirmed the 52-week eGFR slope data from all patients in the STAR study will serve as the primary basis for accelerated approval, with 104-week data used to verify clinical benefit for full approval. Macrae stressed that no additional confirmatory study is required, which pulls the BLA timeline forward by three years. Regarding partnerships, Macrae stated that the positive regulatory update has generated significant interest and refreshed ongoing discussions, with the goal of securing a partner to support a BLA filing in H2 2025.

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CEO Ying Huang emphasized that the competitor's patient population appears less sick and that CARVYKTI's durability and progression-free survival (PFS) are the key differentiators. For safety, he noted proactive management is effective. SVP of Commercial Development, Steven Gavel, anticipated an NCCN guideline update by the end of Q4. Ying Huang added that submissions to the FDA and EMA are planned, with label updates expected by the end of the following year.

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CEO Emil Kakkis confirmed the Angelman data would be the same at both conferences. He clarified that the setrusumab interim timing updates were for specificity, not due to incoming fracture data. He declined to share baseline characteristics until the Phase 3 data release but noted the study has a higher proportion of more severe Type III and IV patients compared to Phase 2, and is primarily a pediatric study.

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CEO John Evans acknowledged the sad outcome as a reflection of the known risks of chemotherapy and transplant, which underscores the severity of the underlying disease. Chief Medical Officer Dr. Amy Simon elaborated that the event was consistent with busulfan's known pulmonary toxicity, which can manifest up to 100 days post-transplant and can be fatal. She confirmed the event was deemed unrelated to BEAM-101 by the investigator and that the patient's blood profile was correcting as expected.

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CEO John Evans stated the event highlights the known risks of chemotherapy and transplant, which are weighed against the severity of sickle cell disease. Chief Medical Officer Dr. Amy Simon added that busulfan has known dose-dependent pulmonary toxicity that can manifest months post-transplant, and the event was consistent with this known risk profile, leading to no changes in the trial protocol.

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CEO John Evans acknowledged the known risks of chemotherapy and transplant, emphasizing the severity of the underlying sickle cell disease. Chief Medical Officer Dr. Amy Simon elaborated that busulfan has known dose-dependent pulmonary toxicity which can manifest up to 100 days post-transplant, and an extensive workup ruled out other causes, making the event consistent with known complications of the conditioning regimen.

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Dr. Mark Eisner, Chief Medical Officer, confirmed that 'target not detectable' (TND) is a critical measure and that the FDA guidance includes a composite endpoint of TND plus ALT normalization, which will be presented. CEO Dr. Marianne De Backer added that since the Sanofi deal closed recently, they are still in a transition phase but will share initial monotherapy data for two T-cell engagers in Q1 2025.

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Chief Medical Officer Baisong Mei noted that vein-to-vein time varies from 3 to 10+ months and that the process has been faster for adolescents. Chief Scientific Officer Linda Burkly clarified that the indel strategy is differentiated and is *not* a knockdown approach; instead, it disrupts a repressor's binding site to upregulate gene expression.

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The time from enrollment to dosing varies significantly (3 to 10+ months) and appears to be faster for adolescent patients. The indel upregulation strategy is differentiated from a knockdown strategy; it works by creating an indel to disrupt a repressor's binding site, thereby upregulating gene expression, rather than knocking down the repressor itself.

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