Jonathan Wolleben • Citizens JMP

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David Meeker, Chairman, CEO, and President, explained that discontinuations were varied, including patients unable to manage the trial burden and some due to adverse events. He noted that the data imputation method for dropouts is conservative. For real-world persistence, he stated that while the global discontinuation rate is around 30%, he expects the rate for the HO population to be better, citing the low discontinuation rate in the trial and high patient motivation.

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CEO David Meeker confirmed the Phase III protocol allows for dose titration to manage tolerance. Regarding receptor activity, he stated that RM-718 and setmelanotide have highly similar potency, while a head-to-head assay for bivamelagon is underway. However, preclinical models suggest bivamelagon achieves similar results at the doses being studied.

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Chief R&D Officer Dr. Helen Thackray stated that the program has a standard safety profile for a protein therapeutic, which is supported by the open INDs in the U.S. and Australia. The key clinical efficacy metric is skin normalization, assessed by patient-reported itch and physician assessment of redness and scaling. She explained that Part 3 of the trial will have fewer than 10 patients, with Part 4 being slightly larger, and that clinical signs of improvement could appear within 4 to 8 weeks.

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Chief Commercial Officer Charlie Gayer noted that about half of all patients undergo reauthorization in Q1 and early Q2, and the process is proceeding as expected. He clarified that the pediatric opportunity, estimated at around 500 U.S. patients, is not yet included in the $1 billion peak global revenue forecast and may represent upside, especially given new data on early disease onset. CEO Jon Stonehouse reiterated that Q1 revenue would likely be flat to slightly down due to seasonal dynamics.

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President and CEO Dr. Pravin Dugel responded that the statistical powering for SOL-R remains robust and unchanged, as the original patient number was dictated by safety requirements, not statistics. He stressed the high value of the label, stating that a superiority label with 6-to-12-month flexibility would be a first-of-its-kind, providing critical scientific data for physicians and a significant commercial advantage with payers.

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CMO Dr. Scott Harris addressed the questions by explaining that the higher 2.4mg dose of pemvidutide could be used in Phase III to achieve significant weight loss. He expressed confidence in achieving strong adherence, citing the very low discontinuation rates due to adverse events observed in prior pemvidutide trials, which he believes will carry over to the IMPACT trial.

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CEO Chris Peetz described the potential label as a 'definition of exclusion' for rare cholestasis causes. He clarified the 1,000-patient estimate for the U.S. and Europe is a tangible figure, comparable to the PFIC opportunity, derived from analyzing compassionate use requests and physician interest rather than a formal epidemiology study.

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CEO Daniel Tassé and Chief Medical Officer Dr. Pharis Mohideen explained that 'patch wear-time experience' is a holistic measure including hours worn, day-to-day variability, and tolerability. While declining to provide specific percentages for the 'label-in' group pending FDA alignment, Mr. Tassé noted the overall EPITOPE response rate was 67%, implying the 'label-in' group's response would be higher. He also confirmed that resolving the COMFORT Toddlers protocol with the FDA is the priority before addressing the related COMFORT Children study.

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