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Chairman, President & CEO, Mitchell Steiner, and Chief Scientific Officer, Gary Barnette, emphasized the strength of the new formulation's intellectual property, describing it as a complex manufacturing process that creates a unique pharmacokinetic profile, making it difficult for generics to replicate. Dr. Steiner noted positive preliminary feedback from international patent offices. Dr. Barnette stated that an open-label extension is not currently planned, as they believe the safety database from the planned trials will be sufficient to meet FDA requirements.

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Chairman, CEO, and President Dr. Mitchell Steiner acknowledged the FDA has indicated a potential benefit in younger patients. Chief Scientific Officer Dr. K. Barnette outlined a possible Phase III design that would be powered for efficacy in the older population while including a younger cohort for observational data to support a broader label. Dr. Steiner explicitly confirmed that the full safety data set from both parts of the study will be reported upon unblinding.

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Dr. Mitchell Steiner, Chairman, CEO, and President, noted that safety data exists for doses up to 18mg from breast cancer trials with no drug-induced liver injury observed. He also highlighted that enobosarm has previously shown a reduction in triglycerides. Dr. K. Barnette, Chief Scientific Officer, added that while patients with known alcohol-related liver disease are excluded, moderate alcohol intake is not prohibited but is monitored.

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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 they will not amend the UGN-103 trial to ensure it remains comparable to the UGN-102 study for a smoother approval process. Chief Medical Officer Mark Schoenberg added that for ICVB-1042, IND-enabling studies are underway this year with substantive data likely next year. For UGN-301, he anticipates reporting durability data from the combination arms later this year.

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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 responded that while the company doesn't collect severity data, he suspects the initial uptake is a mix of severe cases and highly motivated families. CFO Jim Mackaness added that their strong balance sheet provides optionality for a European launch, which could be manageable and efficient due to concentrated call points, whether done solo or with a partner.

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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 and omnichannel marketing efforts. CEO Anish Bhatnagar described PWS care in Europe as similar to the U.S. but often more centralized, particularly in countries like France and Germany, where patients are tracked through centers of excellence, creating a favorable environment for a new therapy.

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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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CEO Dr. Anat Cohen-Dayag stated that Compugen could not provide guidance on AstraZeneca's data presentation. Chief Medical Officer Dr. Michelle Mahler detailed the market opportunity, estimating an initial addressable population of 8,000 to 12,000 patients, with potential for expansion into broader combinations and a larger market upon success.

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Chief Scientific Officer Dr. Eran Ophir and President and CEO Dr. Anat Cohen-Dayag confirmed the current focus for GS-0321 is on solid tumors where they believe they have an edge. Chief Medical Officer Dr. Michelle Mahler explained the COM701 trial is 'adaptive' as it allows for adding arms or increasing sample size post-interim analysis, with the final size dependent on the observed effect.

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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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President and CSO Dr. Anish Suri confirmed that exciting data is being generated for both programs. For CUE-401, data from several disease models developed with ONO will be released in due course. For CUE-501, in vivo experiments are underway to further characterize the relationship between dosing and B-cell depletion. These emerging datasets will inform future development and indication strategy.

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The company believes endocrinologists will welcome an oral option, as the mechanism is understood to be different from growth hormone and the lack of treatment for 6 months in the placebo arm is not considered detrimental for this patient population. The Phase III trial will focus strictly on the primary growth endpoints for approval; however, ancillary metabolic benefits are being explored in a separate investigator-initiated study.

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Executive VP and Chief Medical Officer Alex Zukiwski explained that the NDA for CNCT19 is expected to be filed in H2 2022, with a potential 2023 approval. He noted that while two imported CAR-T therapies exist, their high price point (approx. $200,000) limits access. He believes CNCT19, as the first domestically produced CAR-T, will have a significant cost advantage. He also highlighted that CASI's existing commercial infrastructure for EVOMELA positions them well for the launch, as it targets the same key opinion leaders.

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Chairman and CEO Wei-Wu He stated that competitor CAR-T therapies are priced around RMB 1.2 million, and CASI intends to price its locally manufactured CNCT19 significantly lower. Chief Medical Officer Alex Zukiwski clarified that their partner, BioInvent, will be responsible for announcing the timing for the BI-1206 data release and advised monitoring BioInvent's public statements.

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Wei-Wu He, Chairman & CEO, explained that the commercial team is expanding aggressively to prepare for the CAR-T 19 launch. He noted that EVOMELA revenue exceeding $40 million is expected to cover the commercial team's costs, creating a unique strategic position. For business development, He stated that CASI is very selective, focusing on synergistic assets like CAR-T 19 and Thiotepa that fit the existing commercial engine, rather than adhering to a predefined pace.

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Chief Medical Officer and Head of Research, Barbara White, responded that the company will first conduct additional analyses of the SSc data to confirm the FVC signal, after which it would design a new study and seek agreement from regulators. CEO Yuval Cohen added that they will also engage with experts in the field and that a decision is likely a "next year event." Regarding the CB1 inverse agonist program, Dr. White stated that the current plan is to select the single most promising compound to advance into clinical development to conserve resources, rather than pursuing multiple candidates simultaneously.

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Barbara White, Chief Medical Officer and Head of Research, stated that the company will conduct further post-hoc data analysis to confirm the robustness of the forced vital capacity (FVC) signal before designing a new study and engaging with regulators. CEO Yuval Cohen added that a decision is likely a "next year event" (2021) following engagement with experts. Regarding the preclinical pipeline, Dr. White explained that the current plan is to advance one of the more promising CB1 inverse agonist compounds into clinical development to conserve resources, while keeping others as backup options.

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Dr. Barbara White, Chief Medical Officer, confirmed that should both studies yield positive data, the company's current thinking is to submit a single, combined NDA. She noted that there is significant potential for overlap in several modules of the application (non-clinical, CMC, safety), which would create efficiencies. She also stated that waiting for the CF data would not substantially delay the overall submission timeline.

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