Gavin Clark-Gartner • Evercore ISI

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President & CEO Jan Møller Mikkelsen shared a personal success target of a 90% conversion rate at launch maturity, while acknowledging reimbursement challenges will persist for some patients. He confirmed Ascendis will continue to provide necessary KPIs, including enrollment data, until the launch is mature enough for revenue figures to be the primary metric for modeling.

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EVP and President, Ascendis U.S. Jay Wu confirmed that payer conversations are progressing well, with favorable policies in place that are consistent with the label, and gross-to-net arrangements reflect the drug's high clinical value. Chief Business Officer Sherrie Glass stated the vast majority of patients come from conventional therapy, with only 10-15% having prior PTH experience, a subset of whom were on NATPARA. CEO Jan Mikkelsen added that remaining NATPARA patients are expected to transition in Q2 and Q3.

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Jan Mikkelsen, President and CEO, clarified this group largely consists of former NATPARA patients who could not access the special use program. He expressed confidence that these patients will actively seek treatment and that the company's infrastructure, including the ASAP hub program, is prepared to support a rapid uptake upon launch.

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President and CEO Jan Mikkelsen stated the new access policies were implemented gradually through the first half of 2024, with the full effect on volume growth expected in the second half. Regarding IQVIA data, Mikkelsen expressed skepticism, noting that its sampling methodology can be unreliable during shifts in market access and PBM coverage, making it not 'useful' for tracking real-time performance.

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COO & President Peter Radovich reported that for Alagille syndrome, LIVMARLI persistence is approximately 70-75% after one year, with lower attrition thereafter, noting it was too early for PFIC data. Chief Medical Officer Joanne Quan added that the VISTA study's conservative powering and consistent baseline characteristics provide confidence in the trial's progression.

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CEO Christopher Peetz confirmed the pruritus response deepened over time and highlighted the low discontinuation rate as a testament to the drug's benefit. He also noted a newly allowed patent for the tablet formulation extending to 2043. President and Chief Operating Officer Peter Radovich added that the tablet is expected to be an attractive option for patients over 25 kilos.

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CEO Chris Peetz emphasized Mirum's unique position to acquire rare disease assets, leveraging its financial strength. CFO Eric Bjerkholt added that the convertible notes mature in four years and do not meaningfully impact current plans, affirming the company has significant flexibility for attractive business development opportunities.

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Chief Medical Officer Joanne M. Quan explained that Mirum believes the NIH toolbox can serve as a registrational endpoint and plans to engage with the FDA early next year. She highlighted the molecule's selectivity and high brain penetration as key differentiators. CEO Christopher Peetz added that the reliance on patient-conducted tests aligns with Mirum's expertise in patient-reported outcomes.

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Chairman & CEO Krish Krishnan declined to quantify the Q3 slowdown but noted summer pauses are typical. VP of Corporate Development Stéphane Paquette stated the RDEB/DDEB mix was stable at roughly 64/36, similar to the previous quarter.

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Krish Krishnan, Chairman and CEO, acknowledged a quarterly metric would differ but expressed caution in providing one due to the unpredictable nature of patient pausing. Jennifer McDonough, SVP of Patient Access, clarified the calculation methodology, stating that patients are not removed from the denominator; if they pause and restart, their compliance rate is adjusted downward to reflect missed doses over their entire therapy period.

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Chairman and CEO Krish Krishnan confirmed the Q4 increase in dominant patients but stated the mix varies quarterly and is not expected to materially alter compliance. Regarding capital allocation, he acknowledged the company's financial flexibility and confirmed that a share buyback program is a valid point of discussion that management is seriously considering.

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President of R&D Suma Krishnan explained that the year-end update will focus on Cohort 2, with data from a couple of ZZ phenotype patients (some on augmentation therapy). The goal is to see good expression levels of at least one micromolar of A1AT in the ELF and confirm its functionality. Chairman and CEO Krish Krishnan added that as a redosable therapy, even getting close to the target could be sufficient to advance the program into a redosing study.

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CEO Tim Van Hauwermeiren declined to comment on a competitor's specific forecast but agreed that self-administration is a critical factor for patients. He referenced argenx's own increased total addressable market expectations for MG, which were driven by the potential of self-administration to unlock a significant number of new patients, validating the importance of this trend.

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Chief Operating Officer Karen Massey emphasized that the core strategy is to provide broad access and allow patient choice based on lifestyle fit, not price. She expressed confidence in the market access team's ability to secure agreements quickly. Chief Financial Officer Karl Gubitz added that, generally, VBAs are less important in the pharmacy channel compared to the medical channel.

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Chief Financial Officer Karl Gubitz confirmed this understanding. He explained that for a pharmacy benefit product, they expect to provide incremental rebates instead of VBAs, and therefore, the caps associated with the VBAs would not be part of the new payer contracts.

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CEO Tim Van Hauwermeiren stated that no increase in such titers has been observed in the empasiprubart studies. He noted this aligns with the theoretical safety advantages of an anti-C2 antibody and reiterated the clean safety and tolerability profile seen in Phase I data.

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CEO Kabir Nath confirmed the positive COMP005 data was well-received by investigators, leading to strong recruitment for the COMP006 trial. Chief Patient Officer Steve Levine added that SPRAVATO is delivered primarily in individual rooms, which aligns well with the existing infrastructure needed for COMP360.

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CEO Kabir Nath confirmed that the Phase IIb data serves as the benchmark for powering the Phase III studies, making the 5-point delta a reasonable guide. Regarding the advisory committee meeting, he stated Compass will focus on how the committee views the PTSD patient population and interprets changes in the CAPS-5 score, which will inform their own study design.

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Kabir Nath (Executive) and an unnamed executive (Michael) explained that the DSMB is independent and has significant latitude in its assessment. They clarified that an 'imbalance' is not based on a rigid quantitative threshold but is a clinical interpretation considering the severity and nature of the cases. Therefore, they could not provide a specific number that would trigger such a finding.

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President and Head of R&D Pablo Cagnoni provided a clear confirmation, stating 'Yes, Gavin. There will be an update on ET data as well later this year.' He added that the company is moving quickly in ET with the goal of starting pivotal trials in early 2026.

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Executive Steven Stein clarified that the event-driven trial is nearing its required events, so the data readout could occur soon but may track into the second half of the year. He agreed that the Polivy result is 'in the ballpark of a reasonable benchmark' given the similar high-risk patient population.

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An unnamed executive declined to provide details on prior therapy beyond confirming all patients were refractory to antihistamines. He also stated he would not speculate on potential efficacy differences in patient subsets at this time, noting that the drug's mechanism is different from an anti-IgE antibody like omalizumab.

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Dr. R. Struthers, CEO, responded that responder analyses are typically conducted at single time points. He expressed high confidence in the endpoint, noting that A4 is one of the least variable biomarkers in CAH and that requiring both components of the composite endpoint to be met makes the result highly specific and less susceptible to random variability.

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CEO R. Struthers stated that the company is not yet discussing Cohort 4 details. He indicated that more information on the overall Phase III program design will be shared in the near future, at which point it may be relevant to discuss Cohort 4. He anticipated providing more clarity by the next earnings call.

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CEO Douglas Ingram clarified that the annual U.S. incident population is in the low 400s. He noted that the company's terminal value will be driven by more than just incident ELEVIDYS patients, pointing to the enduring PMO franchise, the potential for redosing by the 2030s, and multiple upcoming blockbuster launches from the LGMD and Arrowhead (FSHD, DM1) pipelines.

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President and CEO Doug Ingram confirmed the data readout for the ESSENCE trial is expected in 2026. He clarified that any regulatory decision on the drugs' status would be based on the 'totality of evidence,' which includes study results, data trends, and real-world evidence, not on a single trial outcome.

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CEO Douglas Ingram responded that based on their detailed modeling of capacity and process, peak sales will not be in 2026. He stressed the importance of a responsible, thoughtful rollout for a one-time therapy to ensure patient safety. CCO Dallan Murray added that their high confidence in the forecast comes from granular, patient-level visibility at each site, which allows them to map out the launch trajectory in great detail.

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President and CEO Stephen Mahoney stated that the company feels positive about the meeting and is reiterating guidance to start a pivotal program, but will share more details after receiving the official minutes. He also noted that it was too early to comment on THRIVE's baseline characteristics as enrollment just recently completed.

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Interim CEO Neal Walker stated that based on his experience and market data, black box warnings have not been a significant hindrance to the adoption of effective JAK inhibitors like baricitinib and RINVOQ. He argued that physicians and patients prioritize rapid and substantial disease relief, and the superior efficacy of JAK inhibitors over biologics is a compelling factor that often outweighs the safety discussions.

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