Aydin Huseynov

Aydin Huseynov from Ladenburg asked about Cellectar Biosciences' EMA regulatory strategy for iopofosine I 131, specifically the data considered for conditional marketing approval in Waldenstrom's macroglobulinemia (WM) and the implications for post-BTKi patients. He also inquired about the financial resources required to initiate the U.S. Phase 3 confirmatory trial for FDA accelerated approval and the potential pricing strategy and launch timelines for iopofosine in both European and U.S. markets.

Answer

President and CEO Jim Caruso confirmed the comprehensive data package submitted to the EMA, including recent post-BTKi data, and highlighted the high probability of success for conditional marketing authorization. COO Jarrod Longcor clarified the differences in BTKi usage between the EU and U.S. and detailed the EMA's focus on the post-BTKi patient population due to unmet need. Mr. Caruso and Mr. Longcor outlined the U.S. Phase 3 trial costs, estimating $10 million to initiate and $15 million to reach sufficient enrollment for FDA accelerated approval submission. They also discussed the potential for a premium pricing strategy for iopofosine, noting that a U.S. launch could potentially precede the EU launch depending on trial initiation.

Aydin Huseynov asked about the potential pricing strategy for iopofosine I 131 in both Europe and the U.S., considering the impact of Most Favored Nation (MFN) discussions and the possibility of different branding or labels between EMA and FDA approvals.

Answer

President and CEO Jim Caruso and COO Jarrod Longcor indicated a potential mid-2027 commercial launch ex-U.S., with the possibility of U.S. approval occurring earlier. They discussed a trend towards more flat universal global pricing, the role of the HTA process in Europe, and Cellectar's strong position to negotiate premium pricing due to the significant unmet medical need and lack of direct comparators for iopofosine I 131.

Aydin Huseynov inquired about Cellectar's clinical strategy for iopofosine I 131, including a hypothetical trial against rituximab in earlier lines of therapy for Waldenstrom's Macroglobulinemia, the design of the planned Phase III trial, and the potential commercial opportunity in Europe following a conditional marketing application.

Answer

Executive James Caruso and Chief Operating Officer Jarrod Longcor explained that while earlier-line trials were considered, the current focus is on the relapsed/refractory setting due to the significantly larger and more costly study required for earlier lines. For the Phase III trial, they feel confident in iopofosine's strong efficacy data (~60% major response rate) against any likely comparator, such as rituximab, which shows much lower efficacy in later lines. Regarding Europe, Jarrod Longcor noted their PRIME designation enhances chances for conditional approval and that demonstrating superiority over the widely-used rituximab could drive significant volume, potentially offsetting lower European pricing. James Caruso clarified they expect feedback from the EMA on the conditional pathway in Q3 2025.

Aydin Huseynov from Ladenburg Thalmann & Co. Inc. asked about the duration of response data for Tivesimig and whether responding patients are still on trial. He also inquired about the future regulatory strategy for the PD-1/VEGF bispecific antibody, CTX-10726.

Answer

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.

Asked for an update on the duration of response for tivesimig and whether responding patients are still on trial. Also inquired about the potential future regulatory path for the company's VEGF PD-1 bispecific antibody.

Answer

The company has not yet analyzed duration of response, as it follows PFS and OS in the statistical hierarchy. For the VEGF PD-1 bispecific, the regulatory path will depend on indication selection, with a focus on areas where both pathways are active, such as renal cell or gastric cancer. Some of these indications could offer a single-arm approval pathway.

Aydin Huseynov from Ladenburg Thalmann & Co. Inc. inquired about the duration of response in the Tivesimig trial and the potential future regulatory path for the CTX-10726 (PD-1/VEGF bispecific) asset.

Answer

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

Aydin Huseynov of Ladenburg Thalmann & Co. Inc. asked about the rationale for the specific dosing cohorts in the Phase 2 atopic dermatitis trial design. He also requested a timeline for results from this trial and inquired about how the company's next-generation ITK inhibitor will differ from socolitinib.

Answer

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.

Aydin Huseynov asked about soquelitinib's competitive positioning against DUPIXENT and JAK inhibitors, the potential timeline for its efficacy to plateau, and the reasons for the significant efficacy jump between Cohort 2 and Cohort 3.

Answer

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.

Aydin Huseynov asked about the atopic dermatitis trial, questioning the plans for a potential Cohort 4, the rationale for a 400mg dose versus the 200mg optimal dose in oncology, the prior treatment history of patients in Cohort 3, and the nature of the biomarker data expected in May.

Answer

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.

Aydin Huseynov of Ladenburg Thalmann & Co. Inc. inquired about prior treatments for patients in the atopic dermatitis trial, updates on PTCL trial responses, the mechanism behind the high complete response rate, and the potential clinical focus for systemic sclerosis.

Answer

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.

Aydin Huseynov from Ladenburg Thalmann & Co. Inc. asked for a hypothetical number of patients that could be dosed in 2025 without J-code reimbursement issues and inquired about the timing for Zasturi sales guidance.

Answer

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.

Aydin Huseynov asked about the treatment path for patients who progress on UGN-102 and requested clarification on the gross-to-net (GTN) dynamics for both JELMYTO and UGN-102.

Answer

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.

Aydin Huseynov from Ladenburg inquired about the commercial preparedness for UGN-102, specifically the rationale for increasing the sales force from 52 to 83 reps for a market opportunity projected to be 10 times larger than JELMYTO's.

Answer

Chief Commercial Officer David Lin explained that the sales force size is based on patient epidemiology; unlike the rare disease JELMYTO treats, UGN-102's target patients are seen by most urologists, and 83 territories will cover about 85% of the market. President and CEO Liz Barrett added that this team is supported by a comprehensive field organization including nurse educators, MSLs, and reimbursement managers.

Aydin Huseynov from Ladenburg Thalmann & Co. Inc. posed a hypothetical question about potential M&A interest, asking whether acquirers would be more focused on the company's GI or renal assets given its current valuation.

Answer

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.

Aydin Huseynov of Ladenburg Thalmann asked about the ramp-up to XPHOZAH's $750 million peak sales guidance, whether that figure includes Medicare, and if there has been any payer pushback.

Answer

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.

Aydin Huseynov of Ladenburg Thalmann asked if a full approval would position deramiocel for greater use than other therapies, inquired about the HOPE-3 data disclosure timeline, and questioned the plan for the Priority Review Voucher (PRV).

Answer

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.

Aydin Huseynov inquired about the clinical strategy and potential trial design for deramiocel in Becker Muscular Dystrophy (BMD). He also asked about other heart diseases beyond DMD and BMD where deramiocel could have commercial potential.

Answer

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.

Aydin Huseynov asked about Capricor's commercial plans for unlicensed territories, potential label restrictions alongside other DMD therapies, the likelihood of an AdCom, the financial impact of a future skeletal muscle label, and 2025 development plans, particularly for Becker muscular dystrophy.

Answer

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.

Aydin Huseynov of The Benchmark Company requested details on the new second-generation LSD1 inhibitor, including its origin and development plan. He also asked for an update on competitors like BMS and Imago, and for context on the clinical outcomes for heavily pretreated FET-rearranged sarcoma patients without seclidemstat.

Answer

Director and CEO David Arthur explained the second-generation LSD1 inhibitor is an early-stage, in-house program. Director of Corporate Development Daniela Santiesteban provided competitive updates, highlighting Salarius' differentiated focus. SVP of Clinical Development Nadeem Mirza noted that the stable disease seen with seclidemstat is a strong signal, as untreated, heavily pretreated patients have poor outcomes with survival of less than a couple of months.