Maurice Raycroft • Jefferies

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CEO Sean Nolan confirmed the primary endpoint will be objective, clinically meaningful, and easy to measure, consistent with their focus on functional gains. He indicated a single-arm study is likely, which would necessitate bias mitigation measures such as video assessments and blinded raters, on which they have secured FDA alignment.

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CEO Sean Nolan stated that the company's goal is to align the announcement of the final FDA-agreed trial design with the next data update in the first half of 2025. He also confirmed that the plan for Part B already includes the 3-to-5-year-old cohort, as part of a broader strategy to treat patients of all ages.

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CEO Sean Nolan stated that the company wants to complete Cohort 2 dosing and its natural history analysis before having a comprehensive discussion with the FDA. While the intent is to hold this meeting in the first half of 2025, he noted it's too early to specify the exact amount of follow-up data required. The RMAT process will be leveraged to facilitate ongoing dialogue with the agency and provide more clarity on requirements in the coming quarter.

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Chief Medical Officer and Head of R&D, Dr. James Hamilton, stated that while the company is not providing specific guidance on first-in-human data, the key potential benefits being evaluated are the quality of weight loss, including visceral fat reduction and lean muscle mass preservation. He noted that initial data for ARO-INHBE is expected around the end of 2025, with ARO-ALK7 data to follow.

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An executive, likely Dr. Bruce Given, explained that the SHASTA-5 timeline is uncertain as it's an event-driven outcomes study focused on pancreatitis, primarily for payers and ex-U.S. health authorities. He clarified that it would not replace a CVOT, as atherosclerotic risk from triglycerides is a separate clinical question from pancreatitis risk.

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Dr. Bruce Given, Interim Chief Medical Scientist, stated that the ESC presentation will offer more detail on primary and secondary endpoints. He highlighted the statistical significance on pancreatitis as an exciting and important part of their FDA filing. Dr. Christopher Anzalone, President and CEO, clarified that the company has not disclosed the number of patients in the expanded access program.

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President and CEO Peter Greenleaf corrected that historically, the second or third quarter has been softer due to summer effects, and suggested using past performance as the best predictor for the year's cadence. On the ANDA filings, Greenleaf confirmed that patent infringement lawsuits have been filed against all challengers, triggering a 30-month stay. He emphasized that the company will vigorously defend its patents and is focused on ensuring the long-term value of LUPKYNIS.

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President and CEO Peter Greenleaf explained that maintaining the guidance range is a conservative approach that accounts for potential Q4 variables like holidays and year-end insurance changes. On the ENLIGHT-LN study, Dr. Greg Keenan, Chief Medical Officer, noted it reveals high LUPKYNIS use in African-American populations and emerging use in combination with other therapies. Peter Greenleaf added that the 'restart' phenomenon reflects current physician practice, but he sees a significant growth opportunity as treatment guidelines evolve toward longer, continuous therapy.

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CEO John Leonard explained the age change for HAELO reflects the goal of achieving the broadest possible label. Regarding ATTR-CM enrollment, he confirmed it is well ahead of schedule and that the company's guidance to exceed 550 patients by year-end is a conservative estimate they may over-deliver on, guaranteeing it will be more than 100.

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CMO David Lebwohl responded that event rates are expected to be similar to other recent TTR trials. He highlighted that the favorable Phase 1 data, showing a low event rate and disease stabilization, suggests a good possibility of positive findings at an interim analysis that could potentially stop the study early, with more details to be provided in the future.

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CEO John Leonard and CMO David Lebwohl expressed enthusiasm for the AHA presentation. They confirmed it will include biomarker, functional, and clinical data for all 36 patients at the 12-month mark, linking deep TTR reduction to clinical outcomes. Lebwohl noted their study includes a sicker patient population than competitor trials, providing a robust test of the therapy.

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CEO Thomas Lingelbach confirmed the FDA review for the adolescent label extension is ongoing. Regarding safety, he stated that investigations into adverse events in frail older individuals are continuing and Valneva is supporting regulators to determine if additional precautions are warranted. For the Lyme study, he declined to disclose the specific p-value but affirmed the study is sufficiently powered, noting the historical difficulty in establishing an immunological correlate for Lyme disease.

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Chief Commercial Officer Charlie Gayer attributed the paid-rate success to team expertise, the positive impact of the Inflation Reduction Act on Medicare affordability, and improved execution in the commercial segment. He stated the current rate should be a sustainable 'floor' for the future. For gross-to-net, Gayer explained that the improvement was driven by a favorable mix change from more paid Medicare patients and more disciplined management of co-payment assistance for commercial patients.

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Chief Commercial Officer Charlie Gayer detailed a strategy that includes using claims data for a key feature ('bamboo hair'), expanding clinical trial sites, and educating dermatologists. CEO Jon Stonehouse added they will follow established rare disease playbooks, leveraging genetic testing and patient advocacy groups. Regarding pricing, he stated that while no final decision has been made, the pediatric formulation would likely be priced comparably to the adult version.

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Chief Commercial Officer Charlie Gayer noted that the 67 new prescribers represented one of the best quarters in two years but was consistent with the strong average of 62, with an even split between Tier 1 and Tier 2 physicians. Chief R&D Officer Dr. Helen Thackray explained the Phase IV study is observational, designed to capture how physicians manage the transition and to describe the patient experience post-switch to inform other prescribers.

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Chief Commercial Officer Peter Heerma indicated the prescriber mix is 'slightly more skewing to experienced prescribers' but with a healthy rate of new prescribers. On compliance, he did not provide a specific duration but stated that rates are at the 'very high end' of benchmarks for chronic, non-symptomatic diseases, reflecting high patient satisfaction.

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Adam Logal, an executive, projected that BioReference is on a path to reach breakeven in Q1 2025 and achieve profitability thereafter, noting that significant new investment in the test menu is not required. Regarding the EBV vaccine, executive Phillip Frost indicated a decision from Merck on advancing to Phase 2 could come in Q3 2025, but Adam Logal clarified that the specific milestone value is confidential until it is earned.

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Elias Zerhouni, an executive at OPKO, explained that the oral form, developed with Entera Bio, shows competitive potential and that preclinical work on the once-weekly injectable is advancing. He noted the company is actively pursuing partnerships for this differentiated GLP-1/glucagon co-agonist. For MDX2001, Zerhouni projected that initial safety and tolerability data would be available towards the end of the first half of 2025, with efficacy data from larger cohorts expected around mid-2026.

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Chief Medical Officer Pushkal Garg expressed enthusiasm for the ongoing FDA review, stating it remains on track. He expects the label to reflect the study's demonstrated benefits on mortality and hospitalizations, as well as ancillary benefits like preservation of functional ability and quality of life, which were statistically significant, predefined endpoints.

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CCO Madhav Vasanthavada clarified that while the MS-DRG 018 designation is for Medicare, it positively signals the technology's value to commercial payers, and the new ICD-10-PCS code will streamline billing for all. He stated pricing will be a flat rate per treatment kit, with subsequent treatments billed as new, separate events. CEO Vishwas Seshadri added that demand will likely exceed initial supply, which can ramp to ~10 patient runs per month by the end of 2025, while the new facility's timeline is still in the design phase.

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CEO Emil Kakkis explained that the 14-month setrusumab data showed a profound and rapid separation between treatment groups, giving them confidence to implement the interim analyses. For Angelman, he clarified the upcoming update will focus on existing patients through day 338 to demonstrate the potential outcome at the end of the Phase 3 trial, rather than introducing new patient data.

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