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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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CEO Brian Sullivan confirmed the Q3 2025 timeline for top-line data is certain, following a primary completion in June. He noted the process from database lock to data reporting typically takes less than three months. Sullivan clarified that the initial press release will be limited to median Progression-Free Survival (PFS) and hazard ratios, with full data, including any survival trends, to be presented at a medical conference later in the year.

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CEO Brian Sullivan stated that Celcuity is not commenting on specifics regarding event rates but reiterated the guidance for a Q2 2025 top-line data release. He explained the company hopes to request a Real-Time Oncology Review (RTOR) or priority review, leveraging its breakthrough status, to shorten the review time and would begin submitting the package within months of approval.

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Executive Brian Sullivan clarified that the timeline adjustment is solely due to the rate of progression events, not enrollment, which is actually ahead of schedule. Regarding competition, Sullivan stated that the inavolisib approval supports Celcuity's hypothesis that inhibiting all three key pathways is beneficial. He asserted that it does not reduce the addressable second-line market for gedatolisib, as the VIKTORIA-2 trial is designed to more comprehensively address the first-line, endocrine-resistant population.

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Chief Development Officer Nathalie Dubois-Stringfellow confirmed the top-line data will feature the mean eGFR slope and that the statistical plan has been agreed upon with the FDA. CEO Alexander Macrae added that the dataset is robust, with 19 patients reaching two years of follow-up. Regarding partnerships, Macrae stated that Sangamo is in discussions with multiple potential partners, noting that the recent positive FDA Type B meeting on CMC has been beneficial for these negotiations.

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CEO Sandy Macrae confirmed the company aims to finalize the third STAC-BBB deal by the end of the quarter, suggesting the upfront payment would be comparable to previous agreements. CFO Prathyusha Duraibabu stated that operating expenses have been significantly reduced and are expected to remain at current levels in 2025 to support the neurology pipeline.

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CEO Sandy Macrae stated the FDA will consider the 'totality of the data,' which includes the eGFR slope, sustained alpha-Gal levels, lyso-Gb3 control, and patient-reported outcomes, noting all 18 patients on ERT have successfully withdrawn. Chief Financial Officer Prathyusha Duraibabu confirmed that the full $50 million from Genentech has been received, although only $40 million was reflected in the quarter-end cash balance.

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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 stated that the FDA review for the adolescent label extension is ongoing. Regarding safety, he confirmed investigations are active, particularly concerning frail older individuals, and the company is supporting regulators to determine if additional precautions are warranted. For the Lyme study, Lingelbach declined to disclose the specific p-value but noted the study is sufficiently powered. He mentioned that while there is no established immunological correlate for Lyme, a joint publication with Pfizer hypothesizes a potential protective threshold.

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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 Reshma Rangwala confirmed that baseline patient characteristics in the blinded SENTRY trial are tracking consistently with their Phase I study and other contemporary Phase III trials. While unable to share unblinded data on discontinuations, she stated the rates are within expectations. Dr. Rangwala also clarified that excluding fatigue from the primary TSS analysis is aligned with the FDA and consistent with historical precedent from the COMFORT trials that led to ruxolitinib's approval.

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EVP, Chief Medical Officer Reshma Rangwala explained that positive evolving data allowed for a smaller trial size (120 patients) in the SPd study without compromising statistical power. An amendment is with the FDA, and a timeline update will follow regulatory feedback. For the myelofibrosis study, she confirmed patients continue treatment beyond 24 weeks for long-term endpoints, but the 24-week data on SVR35 and absolute TSS are the key drivers for regulatory decisions.

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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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Executive Daniel Passeri stated that the company is in late-stage discussions with several potential pharma partners for CUE-501, which is viewed as a highly differentiated and attractive asset. Regarding the ONO deal, he explained that it would not make sense to renegotiate at this time, as the partnership is highly productive and both parties are aligned on the current path forward.

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The company does not need to go back to the FDA to start the randomized phase. A full data update on the first two cohorts is expected in Q3, after which they can provide better guidance on timelines for cohorts 3 and 4. The data presented was a mix from both cohorts, and so far, no differences in efficacy or safety have been observed between the two dosing strategies.

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Executive Lan Huang stated that discussions with China's NMPA for CIN are positive and ongoing. Chief Medical Officer Dr. Ramon Mohanlal confirmed that an additional Phase III study for CIN will be required in the U.S. and design discussions are active with the FDA. For NSCLC in the U.S., Dr. Mohanlal noted that while the data is positive, its applicability is under discussion with the FDA due to the high percentage of Chinese patients in the DUBLIN-3 study.

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CEO Lan Huang confirmed that extensive population PK data shows comparability between Asian and Western patients and has been submitted to regulators, though it is not typically presented at medical conferences like ESMO. Regarding the CIN NDA, she reported frequent and supportive communication with the FDA, a completed mid-cycle review, and noted that label discussions typically begin one to two months prior to the PDUFA date and had not yet started.

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Dr. Lan Huang, Co-Founder, Chairwoman and CEO, confirmed that the 439 death events for the DUBLIN-3 final analysis have been reached and data cleaning is in process, maintaining the mid-year guidance for top-line data release. She stated that baseline characteristics are balanced, the study focuses on EGFR wild-type patients, and more detailed information on the study design would be provided at the upcoming R&D Day.

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Barbara White, Chief Medical Officer and Head of Research, reported a low discontinuation rate of around 8% for the dermatomyositis study, with 166 patients completing the week 28 visit and 90% of eligible subjects enrolling in the open-label extension. Regarding SSC, she noted the Actemra approval was of great interest and that Corbus is re-examining its own forced vital capacity data. She confirmed that supportive data from the dermatomyositis study will be crucial, and any decision to approach the FDA would follow the analysis of that data, likely in the second half of the year.

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