Aydin Huseynov • Ladenburg Thalmann & Co. Inc.

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CEO Thomas Schuetz explained that duration of response has not been analyzed yet, as it follows PFS and OS in the hierarchical testing plan. For CTX-10726, he outlined a strategy of pursuing indications where both VEGF and PD-1 targeting are proven effective, such as renal cell, gastric, and hepatocellular cancer, suggesting single-arm pathways could be possible in post-PD-1 settings.

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CEO Richard Miller explained the Phase 2 trial design (200mg QD, 200mg BID, 400mg QD, and placebo) is standard for determining an optimal dose-response profile. He projected a 12-15 month enrollment period with results expected in approximately 18 months. Regarding the next-generation ITK inhibitor, Miller declined to share specific details for competitive reasons but suggested it may target different aspects of ITK's function.

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Richard Miller, an executive, acknowledged the small sample size but noted the 4-week data is competitive, suggesting soquelitinib's safety and oral format could position it for early-line therapy. He explained that the efficacy curves are still decreasing at day 28, prompting the protocol amendment to test longer treatment durations. Miller attributed the efficacy jump in Cohort 3 to the doubled dose, which achieves nearly 24-hour target occupancy, a finding consistent with their lymphoma studies.

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Executive Richard Miller explained that the decision to proceed with Cohort 4 (400mg) is pending a full review of data from Cohort 3, though it is currently planned. He noted that Cohort 3 deliberately included 'sicker' patients with more DUPIXENT failures and higher baseline EASI scores. Regarding biomarkers, Miller mentioned that earlier findings are being confirmed and that studies into T regulatory cells are yielding 'extremely interesting' and 'provocative' results, suggesting a durable effect.

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Executive Richard Miller clarified that atopic dermatitis trial patients have so far failed topical therapies. In the PTCL trial, he noted one partial response is showing continued improvement. He attributed the high complete response rate to the drug's ability to induce a broad immune response against heterogeneous tumors. For systemic sclerosis, the focus would be on lung disease, and he speculated that dosing for autoimmune conditions might differ from oncology.

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CEO Liz Barrett declined to speculate on a specific number but stated it would be 'significantly more,' reiterating that reimbursement is the main barrier, not clinical need. CFO Chris Degnan stated that the company will get through the initial launch phase this year and will look to potentially provide guidance for 2026, while reiterating the drug's peak potential of over $1 billion.

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Chief Medical Officer Dr. Mark Schoenberg noted that true disease progression was rare, and standard TURBT was safe for patients who recurred. CEO Liz Barrett added that they expect physicians to retreat with UGN-102 upon recurrence and plan to generate data to support this. CFO Chris Degnan stated JELMYTO's GTN is in the mid-70% range and expects a more favorable profile for UGN-102 over time due to a shift to the community setting.

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President and CEO Mike Raab responded by stating his belief that the company is undervalued across both its GI and renal franchises. He deflected direct M&A speculation, emphasizing that the primary objective is to build a great, sustainable enterprise focused on serving patients and maximizing shareholder value.

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CEO Michael Raab clarified that the $750 million peak sales guidance for XPHOZAH explicitly *excludes* Medicare and is based on the non-Medicare population (40% of dialysis patients). He stated it is too early for 2025 guidance. CCO Eric Foster added that they are not currently seeing pushback from commercial payers.

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CEO Linda Marbán stated that if the current BLA is approved, the HOPE-3 trial would likely be expanded internationally and remain blinded for a global label expansion. If needed for a U.S. filing, it would be unblinded in Q3/Q4 2025. She confirmed the company's plan is to sell the PRV upon receipt to strengthen the balance sheet, as they believe future indications would likely receive Priority Review regardless.

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CEO Linda Marbán stated that Capricor is actively working with the FDA on a program for Becker, aiming to leverage the Duchenne data for a potentially accelerated pathway, with more details expected in the next quarter or two. For other indications, she mentioned the company is evaluating various orphan cardiomyopathies where deramiocel's immunomodulatory and antifibrotic mechanism of action could be beneficial, as part of its pipeline expansion strategy.

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CEO Linda Marbán stated that Capricor currently retains rights for all territories outside the U.S., Europe, and Japan and plans to market directly in those regions. She anticipates an open label without restrictions for use with other therapies, based on current FDA guidance. While not expecting an AdCom due to strong data and a collaborative FDA relationship, the company is preparing for one. Marbán noted a skeletal muscle label would add value but emphasized the cardiomyopathy indication is the primary opportunity. For 2025, a key goal is indication expansion, with plans for a Becker muscular dystrophy trial being developed with KOLs and the FDA.

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