Leland Gershell • Oppenheimer & Co. Inc.

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CEO Linda Marbán acknowledged the strength of the OLE data but stated the plan is to have all data sets ready for the FDA to determine what is needed for review. Regarding FDA personnel changes, she asserted that the company is focusing on the strength of its clinical data rather than specific reviewers, adopting an "old school" approach.

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CEO Linda Marbán stated that while negotiations with Nippon Shinyaku for Europe are ongoing, Capricor is also working directly with European authorities to evaluate all opportunities. For the U.S., she confirmed NS Pharma has a team of 125 full-time employees, most of whom are now focused on the deramiocel launch, and that Capricor is enhancing its own medical leadership to support the effort.

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CEO Linda Marbán reported very positive feedback from payers, who recognize deramiocel as the only treatment for DMD cardiomyopathy, a critical unmet need. Regarding capital, she stated the company is exploring opportunities for label expansion into skeletal muscle myopathy and Becker muscular dystrophy, advancing its exosome pipeline, and evaluating other strategic options to build shareholder value.

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CEO Linda Marbán confirmed that the San Diego facility and future expansion plans are designed to serve European and Japanese markets. She stated that while a skeletal muscle label could capture outlier patients, the cardiomyopathy label represents the broadest possible market, as 100% of DMD patients develop it. Marbán also affirmed that due to the RMAT designation, Capricor expects a priority review and has built its timelines around that assumption.

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President & CEO Jan Møller Mikkelsen outlined three key factors for the strategic shift: 1) The patient population is growing as genetic testing reclassifies individuals from idiopathic short stature (ISS). 2) The highly positive data from the COACH combination therapy trial suggests it is an ideal treatment for this heterogeneous group. 3) Direct engagement with patients and advocacy groups underscored the significant unmet medical need.

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CEO Jan Mikkelsen and CMO Aimee Shu clarified that renal function is not a factor in reimbursement decisions, with the exception of patients with severe, Stage 4/5 renal impairment who were excluded from studies. Regarding the COACH trial, Jan Mikkelsen stated that while addressing medical complications is the primary goal, linear growth is the established primary endpoint they must use. He expressed high expectations that the combination therapy will 'reset the bar' for both growth and complication outcomes.

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Jan Mikkelsen, President and CEO, clarified that in France, YORVIPATH is available through a special reimbursed, non-promotional program (AP2) that recently started. He confirmed that full commercial launches are planned for the majority of 'Europe direct' countries in Q2 and Q3 of 2025, with international markets also transitioning to full commercialization next year.

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CCO David Lin clarified that the majority of the 2,000 early adopters are in the community setting and that UroGen is actively working with hospital P&T committees to ensure access. CMO Mark Schoenberg stated that for UGN-103, they expect the FDA's requirements to mirror those for ENVISION, focusing on the complete response rate with a reasonable amount of durability data.

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CEO Liz Barrett confirmed continuous, positive interactions with the FDA, stating they are at the end of their review and will not have a late-cycle meeting. Chief Commercial Officer David Lin identified three key initial patient segments for UGN-102: those with multiple recurrences, early recurrers, and patients who are poor surgical candidates. He emphasized the drug's seamless fit into existing clinical workflows.

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President and CEO Liz Barrett stated there were no new updates on the litigation, with a trial date likely in 2026. She affirmed that a favorable outcome would indeed carry over to UGN-102. She also highlighted the strength of their lifecycle strategy, with UGN-103's potential launch in 2027 providing further protection.

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EVP & CFO Michael Kalb acknowledged the flat SG&A in Q2 but stated that due to the accelerated investment in educating HCPs on the new NCCN guidelines, he anticipates 'a little bit of an uptick' in SG&A expenses in the second half of 2025.

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CEO Anish Bhatnagar explained they do not collect severity data but suspect the initial cohort is a mix of severe cases and highly motivated families. CFO Jim Mackaness stated their strong balance sheet provides optionality for a European launch, which could be manageable and efficient due to a concentrated market, though specific OpEx plans are still in development.

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CCO Meredith Manning attributed the broad prescriber interest to strong pre-launch awareness campaigns, including webinars and digital marketing. Executive Anish Bhatnagar described the European PWS care model as similar to the U.S. but often more organized, with patients concentrated in centers of excellence, which he views as a favorable dynamic for launch.

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Executive Jennifer Good explained that while the PAciFy study validated the opioid mechanism, Haduvio's dual mechanism demonstrates a much larger effect size (67-74% vs. 40%). She noted physicians prefer a non-Schedule II substance without morphine's risks like respiratory depression. Chief Development Officer Dr. James Cassella added that the PAciFy study's 0% placebo response raises questions about its single-center design.

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Executive Jennifer Good clarified the regulatory strategy: IPF is the planned first NDA, with RCC as a follow-on supplemental NDA (sNDA). She noted that while they need to collect long-term safety data, the required size of the safety database will be negotiated with the FDA for the IPF filing. She added that existing safety data from prior programs could be leveraged, and trial sizes may ultimately be driven more by safety requirements than efficacy.

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CEO Jennifer Good clarified that the increase is not fixed. An external statistician will use actual data from the first 50% of patients to repower the study, resulting in a specific required sample size anywhere between the original 160 and a maximum of 400 patients. Chief Development Officer Dr. James Cassella added that the decision will be based on the conditional probability of the findings.

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Chief Executive Officer Tim Van Hauwermeiren explained the Phase IV switch study is important for providing practical guidance to physicians on how to switch patients, a dynamic not fully studied in the pivotal ADHERE trial. For ARGX-119 in CMS, he noted that patients exhibit strong fatigability similar to autoimmune myasthenia, and the company is testing a menu of endpoints, including classical ADL and QMG measures, against which they have known thresholds for clinical relevance.

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CEO Tim Van Hauwermeiren described CMS as being in the 'bull's eye' of the drug's biological target, closely mimicking spectacular preclinical data. He said they are looking for a strong signal that they can 'move the needle' for these patients, which would be a very important validation of the biology for the entire ARGX-119 platform.

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CFO Hunter Smith explained that some Q4 inventory stocking is a common industry pattern but doesn't expect swings as significant as the last two quarters. Jennifer Chien, Head of North America, clarified that for a rare disease like BBS, finding patients is challenging. Once a physician diagnoses a patient and has a positive experience with IMCIVREE, they become a believer, and the main limitation to repeat prescriptions is simply whether they have other eligible patients.

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EVP Jennifer Chien noted the label expansion occurred too late in Q4 to impact revenue and is not expected to be a major sales driver, but it helps differentiate the disease. CEO David Meeker explained they are still learning about congenital HO, which is challenging to diagnose as it lacks a clear 'before and after' event like an injury.

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Robert I. Blum, President and CEO, pointed to the ongoing FOREST study as supportive of aficamten's profile. Fady Malik, EVP of R&D, added that SEQUOIA-HCM data already showed a 90% reduction in SRT eligibility. He anticipates that long-term data from FOREST will allow for comparisons against real-world registries to demonstrate aficamten's potential to slow disease progression.

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CEO John Shannon explained that the Gvoke sales team is focused on educating clinicians to improve compliance with new guidelines to protect the large, untapped patient population. Regarding XP-8121, he clarified that 2025 investments are for Phase III readiness, with the clinical trial likely to begin sometime in 2026.

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CFO Steven Pieper explained that while there are occasional write-offs, the company expects healthy gross margins to continue, supported by the growing sales of higher-margin Recorlev. CEO John Shannon declined to provide specifics on the XP-8121 trial design, stating that discussions with the FDA are an ongoing, iterative process and an update will be provided in the first half of 2025.

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CEO R. Struthers responded that while other NDC opportunities exist, the company wants to learn from the clinical development of CRN09682 before advancing another. Regarding the TSH antagonist, he stated it was too early to detail the development strategy, as it depends on economic, recruitment, and competitive factors, but affirmed it is the best approach for both indications.

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