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CEO Silviu Itescu explained that the adult GVHD trial is set to begin this quarter in collaboration with the NIH-funded BMT CTN group, positioning RYONCIL as an add-on to existing second-line therapies. Regarding the back pain trial, he confirmed that enrollment is accelerating across nearly 40 U.S. sites, with completion anticipated by the end of the year or in Q1 of the next year, to be followed by a 12-month follow-up period.

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The adult trial will be small (60-70 patients per arm) with a mid-single-digit million cost, which will have a very small offset on the 23% cost reduction. The chronic lower back pain trial costs are already covered in the existing budget. Current cash is sufficient through the end of the first calendar quarter of next year, with strategies like partnerships, royalty monetization, and capital markets access to bring in more cash.

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The adult trial is expected to be small (60-70 patients per arm) with a mid-single-digit million cost, which will only slightly offset the overall cost reduction. The back pain trial costs are already budgeted. Current cash is sufficient through at least Q1 of calendar 2024, with plans to raise more capital via strategic partnerships, royalty monetization, and capital markets.

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CEO James Caruso estimated the total confirmatory trial cost at $40-45 million, with $20-25 million needed to complete enrollment. COO Jarrod Longcor added that the trial must be initiated before NDA submission, requiring about $10-12 million upfront. He projected a submission in late 2025 or early 2026, contingent on funding. Both executives expressed confidence in rapid enrollment due to strong existing data and a larger patient pool in the second-line setting.

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President and CEO James Caruso estimated the total cost for the confirmatory study to be approximately $40-45 million, with $20-25 million required to complete enrollment and obtain initial data. Chief Operating Officer Jarrod Longcor added that the FDA requires the study to be initiated at the time of NDA submission and have enrollment ongoing by the time of regulatory action. He noted that about $10-12 million would be needed to get the study operational. Both executives expressed confidence in rapid enrollment due to strong Phase 2 data, high patient awareness, and moving into an earlier line of therapy.

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Chief Medical Officer Dr. Michael Sumner stated that based on interactions with the FDA, an advisory committee is not anticipated and the chance is considered low. President and CEO Dr. Jacqueline Shea addressed the competitive landscape, emphasizing key differentiators in trial design, technology (DNA medicine vs. viral vector), and confirmatory trial approaches (placebo-controlled vs. single-arm).

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CEO Linda Marbán explained that the company will not unblind the HOPE-three trial until it receives clear guidance from the FDA following their upcoming Type A meeting. She emphasized the desire to avoid any perception of bias and stated that a public update would follow once they have official written feedback from the agency.

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CEO Linda Marbán confirmed the pre-licensing inspection is scheduled for the current quarter. Regarding the AdCom, she framed it as a positive development, noting the FDA is actively reviewing the file with over 20 information requests and a mid-cycle review that revealed no substantive issues. She added that Capricor has already conducted two successful mock AdComs in preparation.

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CEO Linda Marbán explained that the partnership involves daily collaboration, with NS Pharma's 125-person U.S. team, already established in the Duchenne space, handling sales and distribution while Capricor shepherds the product to market. She highlighted joint efforts in market forecasting, physician outreach, and payer meetings, emphasizing NS Pharma's deep engagement and excitement for the launch.

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CEO Linda Marbán explained that Nippon Shinyaku's existing Duchenne muscular dystrophy (DMD) infrastructure provides a 'plug-and-play' model with a 125-person team ready for the deramiocel launch. She detailed that Capricor has kept manufacturing in-house, has a commercial-scale facility in San Diego ready for inspection, and is executing plans for a new, larger facility based on a modular, 'plug-and-play' clean room design to ensure scalability.

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The company's primary focus is on the late-line CRC opportunity. Moving to earlier lines is a key long-term goal that will require combination studies, likely starting with bevacizumab. This will happen after the Phase 1 study is complete and the dose for Phase 2 monotherapy is selected. The ultimate vision is to potentially replace irinotecan in second-line therapy.

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Sean A. McCarthy, Chairman & CEO, stated that while the immediate priority is the late-line CRC opportunity, moving into earlier lines is a key long-term goal. He explained this would necessitate combination studies, likely starting with bevacizumab, after the Phase 1 dose is selected and the initial monotherapy study is further along.

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CEO Christopher Anzalone explained that the cash goes to the Viscerna subsidiary, with a large portion to be distributed to its shareholders, including Arrowhead (~56% owner). He also confirmed Sarepta's $50 million annual R&D payment is due in the first quarter of each year, typically February.

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An executive confirmed that Arrowhead has not been advised of an AdCom and does not anticipate one. He also noted that medical affairs teams are educating physicians and that a competitor's launch helps increase disease awareness, which ultimately benefits all players in an orphan market by 'priming the pump'.

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Andy Davis, SVP and Head of Global Cardiometabolic Franchise, confirmed that Arrowhead is planning for commercialization in European markets with a commercial partner. He stated that more details on this partnership would be shared in the future.

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Dr. Bruce Given, Interim Chief Medical Scientist, confirmed they are actively writing the submission, a pre-NDA meeting is planned, and CMC is progressing, with the year-end filing guidance still in place. Regarding muscle programs, President and CEO Dr. Christopher Anzalone stated no data is expected this year, while Dr. James Hamilton, Chief of Discovery and Translational Medicine, noted readouts would include standard metrics like knockdown and splice correction.

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President, CEO & Director Punit Dhillon explained the Aarecor partnership is a lifecycle management effort to develop a higher concentration formulation for less frequent dosing, separate from the current clinical track. CFO Kaitlyn Arsenault clarified that the R&D expense increase was primarily driven by $9.1 million in contract manufacturing costs for the Phase 2a extension study resupply and the Phase 2b trial supply.

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CEO Punit Dhillon stated the extension maximizes data collection without delaying the overall timeline, slotting in well before the Phase 2b study begins. He emphasized the value of longer-term safety and tolerability data. Dr. Puneet Arora added that the 52-week data will help refine PK/PD models for Phase 2b dose selection and provide invaluable long-term data for the combination arm, as the planned Phase 2b will focus on monotherapy dose-ranging.

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President and CEO Punit Dhillon clarified that enrolling 136 patients versus the planned 120 evaluable patients enhances the robustness of the data without changing the statistical plan. He stated that while the accelerated 26-week data allows for earlier engagement with regulators, the timeline for the Phase IIb study initiation remains planned for the second quarter of 2026, following the receipt of the full 52-week data.

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Punit Dhillon, President and CEO, confirmed that enrollment for the CBeyond study is proceeding very well, with 15 of 18 U.S. sites actively recruiting and the company on track to reach 50% enrollment by year-end. He clarified that the study's primary endpoint is an 8% placebo-adjusted weight loss for nimacimab monotherapy. The combination arm with semaglutide is an exploratory endpoint designed to assess for an additive or synergistic effect and improved tolerability. Dhillon emphasized the study's robust design will provide key data for the non-incretin obesity drug class.

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President & Vice Chairman Dr. Elias Zerhouni highlighted the drug's unique biological mechanism, noting its effect on FGF21, which is known to be anti-fibrotic and promising for NASH patients. He added that the oral version offers a convenient maintenance option. Dr. Zerhouni explained the Phase 1 trial will enroll patients with biomarker evidence of NASH and obesity to assess safety and early efficacy signals, avoiding biopsies initially.

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Executive Elias Zerhouni stated that both the subcutaneous and oral programs are currently in the pre-IND and CMC manufacturing phase. He clarified that the initial Phase 1 trials for both will focus on safety, dose-ranging, and establishing PK/PD profiles to gather foundational data before advancing to Phase 2.

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Executive Adam Logal reiterated the full-year 2025 product sales guidance of $165 million to $175 million. Executive Elias Zerhouni stated that safety and tolerability data for MDX-2001 is expected in late 2025, with early efficacy data more likely in the first half of 2026. He confirmed the hematologic program is in the pre-IND stage and is also being considered for autoimmune diseases.

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Executive Elias Zerhouni outlined a timeline with initial MDX2001 safety data expected in mid-2025 and efficacy data around mid-2026. He also noted that news could come from other molecules nearing INDs or new platform collaborations. Zerhouni confirmed that the natural development path, after establishing monotherapy efficacy, would be to explore combinations with other agents like chemotherapy or IO therapies, particularly when moving into earlier lines of treatment.

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Richard Paulson, President and CEO, highlighted a significant 80% prescriber overlap between multiple myeloma and myelofibrosis, which would enable a rapid launch with minimal additional investment. Sohanya Cheng, EVP and Chief Commercial Officer, detailed that XPOVIO demand is driven by its use as a flexible oral option in community settings and its growing use pre- and post-T-cell therapy in academic centers.

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Executives Paula Ragan, Christophe Arbet-Engels, and Mark Baldry confirmed the enrolled patient demographics are balanced and representative of the target commercial population. They highlighted that the trial is enrolling 'very sick patients' to enrich for success on the infection endpoint, that there is high demand from investigators to enroll more patients, and that market research indicates the CN market is more concentrated than the WHIM syndrome market.

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Mark Baldry (Executive) responded that the company is not disclosing specific patient numbers while it focuses on building demand. He confirmed that a 7% price increase for XOLREMDI was implemented at the beginning of the year, which is a typical practice.

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Chief Medical Officer Christophe Arbet-Engels clarified that the Phase III trial is designed for registration per FDA requirements, and benefits from G-CSF reduction would be assessed in follow-on studies. Chief Commercial Officer Mark Baldry described the new patient-directed campaign for XOLREMDI, which uses digital campaigns, websites, and printed materials to educate patients and encourage physician discussions.

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President and CEO Dr. Paula Ragan stated that the company would not provide specific patient numbers at this time but expressed that they are very pleased with the launch progress. Chief Commercial Officer Mark Baldry added that all U.S. patients from the open-label extension study have been prescribed commercial XOLREMDI and that the company is focused on raising disease awareness to identify new patients.

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Executive Alexander Sapir explained that any increase in fetal hemoglobin (HbF) is beneficial, referencing data showing a 1% HbF increase can lead to a 4-8% reduction in vaso-occlusive crises (VOCs). He noted that even single-digit absolute increases in HbF can be clinically meaningful. Iain Fraser, Head of Development, added that they aim to reaffirm the magnitude of induction seen previously and that mid-single-digit percentage increases are expected to be clinically significant.

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Executive Iain Fraser clarified that the withdrawal of Oxbryta has simplified recruitment, and CEO Alexander Sapir added that a protocol amendment now allows concomitant use of crizanlizumab and L-glutamine. Fraser noted the DSMB has no specific safety signal to monitor but will conduct a broad review. Regarding positioning, Sapir stated it's early but believes pociredir could be broadly used and transformative, and they will engage the FDA on relaxing the hydroxyurea exclusion for future studies.

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Executive Alexander Sapir confirmed the 12mg and 20mg cohort data will be released at different times in 2025, with more detailed guidance on timing expected in early 2025. An executive from the clinical team added that post-PIONEER plans, which could range from a combined Phase II/III to separate studies, will be determined after an end-of-Phase I meeting with the FDA. Sapir also noted internal discussions about using fetal hemoglobin as a surrogate endpoint.

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The layoffs primarily affect luvelta-related roles and manufacturing, which is being externalized, in order to pivot resources to the early-stage pipeline. While actively seeking a partner for luvelta, the company remains open to partnering its other pipeline assets to secure non-dilutive capital.

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Executive Jane Chung clarified that the layoffs primarily affect luvelta-related workstreams and that manufacturing operations are being externalized. She confirmed that while actively seeking a partner for luvelta, Sutro will continue to pursue partnerships for its pipeline candidates as a method to secure non-dilutive capital.

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Eric Shaff, President and CEO, stated that while he cannot speak for the FDA, the precedent with VOWST suggested a safety database of at least 300 patients was a key consideration. Lisa von Moltke, CMO, added that the required database size is not fixed; it evolves based on the emerging safety profile and the specific patient population. She confirmed these conversations with the agency would continue as more data is generated.

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President and CEO Eric Shaff explained that while Seres' recent value creation has been in innovation and early development, the long-term aspiration is to become a vertically integrated company with commercialization capabilities, similar to legacy biotechs like Genentech. He noted that certain indications could lend themselves to a small, focused commercial team. However, in the short term, the strategy is focused on R&D to drive shareholder value following the VOWST sale.

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Chief Medical Officer Noah Berkowitz acknowledged the changing landscape, including the rise of ribociclib and PI3K pathway drugs. He noted that while many new agents are being combined with fulvestrant, Arvinas aims to conduct studies demonstrating that vepdegestrant can be a preferred oral backbone therapy for various combinations in this setting, offering a potential alternative to injectable fulvestrant.

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CEO John Houston confirmed that the handover of data and materials to Novartis is complete. He reiterated Novartis's commitment to developing ARV-766 in both early and late-stage prostate cancer. He stated that while future significant updates will come from Novartis, Arvinas is pleased with the transition and Novartis's strategic plan.

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President Stephen Hoge confirmed the trial is run by partner Vertex and has completed its single ascending dose portion. The study is now in the multiple ascending dose phase, with a data readout expected sometime in 2025. He noted that Vertex would provide any specific timing updates.

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President Stephen Hoge explained that the pivotal study for MMA, expected to start in H1 2025, will likely use a biomarker for approval, allowing for a quicker path. The PA program will rely on event rates, which may take more time. He reiterated the goal of launching these products in the 2026+ timeframe, with timing dependent on patient enrollment.

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Dr. David Roth, Chief Medical Officer, explained that the aza label shows a 17% complete response (CR) rate, with recent trials showing rates in the mid-to-high teens. He clarified that the AML trial tested a different combination (a triplet with venetoclax) and is not predictive of the MDS trial's outcome. However, he noted the AML study did reinforce tamibarotene's favorable safety profile, showing no added myelosuppression.

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Chief Medical Officer Dr. David Roth clarified that the SELECT-MDS-1 trial is powered at over 90% to detect a difference in the primary endpoint of CR rate between the two arms, based on an enrollment of 190 patients. While not disclosing the specific numerical assumptions for each arm, he noted the assumptions were based on standard performance for azacitidine in the control arm and that the final readout will also consider durability and safety.

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President and CSO Dr. Anish Suri highlighted three key differentiators: 1) The platform's clinical derisking in over 120 patients with no clinically relevant immunogenicity; 2) Superior safety from selective T-cell engagement, avoiding the high cytokine release seen with pan T-cell engagers; and 3) The ability to harness the body's natural, long-lasting antiviral T-cells. He also noted the significant manufacturing advantages of an antibody-based biologic over autologous cell therapies.

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CFO Tom Burns clarified that declining biodefense and Servier revenues were expense reimbursements, so the net impact is positive, and confirmed that cash projections include all scheduled debt payments. CEO John Varian added that future revenue would be sought from new licensing deals and that the Servier alliance is being terminated, giving XOMA 100% ownership of gevokizumab.

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