Edward Tenthoff • Piper Sandler & Co.

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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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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 & 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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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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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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