Vikram Purohit • Morgan Stanley

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Guowei Fang, President of R&D, expressed confidence, stating they agree with the new CBER director's focus on survival as a gold standard, a metric where CARVYKTI has demonstrated strong, statistically significant benefits. Alan Bash, President of CARVYKTI, reported that nearly 60% of use is now from the CARTITUDE-4 population and expects this to grow to about 75% by the end of the year.

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Executive Alan Bash outlined a three-stage plan for community adoption. The current stage focuses on educating community oncologists to refer patients to certified centers. The second stage, starting later this year, involves enabling select regional hospitals to administer CARVYKTI. The third stage, beginning next year, aims for wider administration in community practices. He also noted that over half of the business is already in the outpatient setting, which expands capacity at treatment centers.

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CMO Mythili Koneru explained that the frontline cohorts require much longer follow-up to yield meaningful data, so no timeline could be provided. Regarding MRD negativity, she indicated that recent regulatory discussions suggest the FDA is more open to it as an endpoint for frontline studies, and Legend plans to engage with the agency on this topic.

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Chief Operating Officer Karen Massey stated that while specific patient numbers were not being updated, the CIDP launch has strong momentum with continued quarter-over-quarter growth in new patient starts and new prescribers. She emphasized that positive patient experiences and the recently launched DTC campaign are driving switch requests, which neurologists are granting, indicating strong belief in the product's value proposition.

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CEO Tim Van Hauwermeiren explained that the auto-injector's key differentiation is its invisible, non-manipulated needle, which is a significant benefit for a subset of patients. He described it as a logical next step in innovation but noted it was too early to provide a specific update beyond the 2027 target, advising analysts to 'stay tuned.'

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CEO Tim Van Hauwermeiren stated that for MMN, the Phase II trial provided a robust PK/PD model to inform the Phase III dose and that all clinical endpoints moved in sync. For upcoming data, PC-POTS will be evaluated for proof of biology, while the myositis studies will require a signal reasonably stronger than placebo to advance.

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Executive Jan van de Winkel confirmed excitement for immunology, with EPKINLY being the most advanced candidate, but stressed that oncology remains over 80% of the company's focus. For GEN1042, he stated a decision will be made in H2 2025 after reviewing more duration of response data to assess its competitiveness.

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CEO Jan van de Winkel clarified that the company's dominant focus for business development is oncology, with the immediate priority being the successful closing and integration of the ProfoundBio acquisition. He noted that while immunology and inflammation are areas of interest, oncology remains the primary focus. Chief Medical Officer Tahamtan Ahmadi added that for GEN3014, the data delivery and J&J's decision timeline are predefined and confined, with data expected by year-end and a decision to follow shortly after.

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Karen Akinsanya, President of R&D, cautioned that it is an early read from the most advanced program and difficult to compare across indications, but noted the data will reflect the platform's ability to meet its goals. On business development, she confirmed they are constantly in discussions across many disease areas and remain open to collaborations.

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CFO Geoffrey Porges stated that confidence in the revenue guidance is high due to industry under-penetration, new platform capabilities, and a solid base of recurring revenue from hosted contracts. Regarding MALT1, President of R&D, Therapeutics, Karen Akinsanya, clarified that the Q2 data release will be an interim update from an ongoing trial, implying that more comprehensive data would likely be needed for substantive partnership discussions.

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Todd Edwards, Chief Commercial Officer, acknowledged a modest seasonal impact is expected in summer months but emphasized that new launches, like the foam for scalp psoriasis, and the 'portfolio effect' would continue to drive revenue growth. Patrick Burnett, Chief Medical Officer, clarified that the ARQ-255 Phase Ib trial is primarily focused on safety, tolerability, and pharmacodynamics, with the goal of getting an early read on potential clinical response ahead of longer pivotal trials.

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Chief Financial Officer David Topper explained that the revenue delta was primarily due to a nonrecurring adjustment for product return reserves and the company's conservative approach to preannouncements. President and CEO Frank Watanabe affirmed the company's strong confidence in its IP portfolio and its intent to vigorously defend its patents, but declined to comment specifically on the upcoming Markman hearing.

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Chief Commercial Officer Todd Edwards explained that while the Kowa co-promotion is well-received, the primary care selling cycle is longer, and a meaningful contribution is expected in 2025 without specific guidance. Chief Medical Officer Patrick Burnett clarified the ARQ-255 readout is a short-duration safety and biomarker study, where the key goal is to find evidence of safety and efficacy to support advancing to a longer trial.

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Co-Founder and CEO Christopher Gibson clarified that the company views both oncology and rare disease as ideal areas for its platform, often anchored by genetic markers. He stated there are no plans to abandon either field and that the portfolio's balance will ultimately be determined by data.

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Greg Friberg, Executive Vice President and Chief R&D Officer, confirmed that the clinical plan for BMN 333 includes a comparative effectiveness study where they would be looking for a superior outcome versus VOXZOGO. He also noted that while the recent agreement was with the FDA, BioMarin is actively working with regulators around the globe to define the path forward internationally.

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An executive noted the V617F update this year might be smaller than Klar's due to a later start. Executive Steven Stein stated that for Rux-XR, bioequivalence was achieved, and after completing stability studies, a response to the CRL will be filed by year-end, targeting a potential mid-2026 approval.

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Pablo Cagnoni, President, Head of R&D, explained that for both programs, success will be measured not only by impact on traditional endpoints like blood counts and spleen size but also by early evidence of allele reduction. He noted that substantive data from mutant-CALR at various dose levels is expected this year, while the JAK2V617F program is slightly behind but follows the same success criteria.

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An unnamed executive explained that meaningful data for both the mutant CALR antibody and the JAK2V617F program are expected next year, with success being gauged by improvements in disease signs and symptoms, as well as evidence of a decrease in VAF (variant allele frequency). Regarding Opzelura, another executive stated that utilization for AD remains over two tubes per patient per year, while the vitiligo cohort is still being monitored for a reliable number. They also noted that prescription growth is driven by both new patients and refills.

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COO Mark Jacobson explained the filing strategy for AD agitation is a cross-functional decision involving branding, IP, market access, and regulatory factors, with specifics to be shared soon. CEO Dr. Herriot Tabuteau highlighted that AXS-14's potential in fibromyalgia is driven by its ability to address multiple symptoms, including fatigue, which is a major source of disability for patients.

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Jan Mikkelsen, President and CEO, indicated that Novo Nordisk would be the primary source for updates on the collaboration's progress. For oncology, he highlighted the focus on TransCon IL-2 beta/gamma following encouraging data and reiterated that Ascendis remains open to various partnership structures to advance the pipeline and create value.

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President and CEO Jan Mikkelsen advised comparing the data to vosoritide, as the mechanism is most similar. He reiterated the company's goal to demonstrate benefits in both linear growth and disease co-morbidities. He referenced Phase 2 data that showed similar growth velocity to vosoritide but also a unique improvement in quality of life related to physical function, a result he hopes to replicate in the pivotal ApproaCH trial via key secondary endpoints.

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President and CEO Nello Mainolfi explained that the primary challenge for prior attempts was achieving selectivity due to high homology with other IRF proteins, a hurdle Kymera's molecule overcame. He also noted a degrader is better suited to block all IRF5 functions. Regarding development, he stated that while it's early, a broad program is expected, and meaningful Phase 1 clinical data for KT-579 should be available within the company's cash runway, which extends to H1 2028.

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