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President and CEO Joseph Payne addressed safety by highlighting Arcturus's proprietary LUNAR delivery technology and mRNA purification processes, which are designed to mitigate toxicology and tolerability issues seen in prior inhaled RNA therapies. For the OTC program, he confirmed the FDA is aware of their biomarker strategy, including the N15 Ureagenesis Assay, and that achieving regulatory alignment on a pivotal trial design is a key upcoming objective.

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President and CEO Joseph Payne stated that the mid-2025 interim data will draw from two cohorts and include approximately 6 to 9 subjects. He defined the bar for success as achieving reasonable safety and tolerability over the 28-day treatment period and demonstrating a lung function (FEV) improvement of at least 3%. Payne also noted the patient population consists of individuals who do not respond to CFTR modulators, with about half being Class I subjects.

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President and CEO Joseph Payne stated that the interim update, expected by the end of Q2 2025, will include data from multiple patients at multiple dose levels. He reiterated that a 3% absolute improvement in FEV1 lung function would be a key benchmark for advancing the program, based on historical precedents in the CFTR modulator space. He did not provide guidance on the specific method of data disclosure.

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CSO Padmanabh Chivukula stated that while the specific study design is not disclosed, they will be evaluating various biomarkers similar to competitors. CEO Joseph Payne added that the study is open-label, will evaluate multiple doses, does not include bronchoscopy, and will measure FEV throughout. He indicated more details would be shared at a later time.

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CEO Matt Gline expressed hope that the data will highlight a 'paradigm-shifting opportunity' for Graves' patients by demonstrating that remission is achievable for severe, refractory cases. He outlined the data's threefold importance: defining the commercial opportunity, boosting patient and physician enthusiasm for trial enrollment, and strengthening the drug's overall clinical profile.

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CEO & Director Matt Gline stated that the company has not yet disclosed the study's statistical powering but may share more in the coming months. He justified the 600mg dose by referencing data from the batoclimab study showing deeper IgG suppression leads to better outcomes. He argued that in a severe disease like CIDP and a competitive market, maximizing efficacy with the highest dose is paramount for success and will also aid in trial enrollment.

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Chief Research & Development Officer Jonathan Zalevsky explained that with no approved biologics for AA, Respag has an opportunity to be a first-in-class therapy with a sustained effect, a key differentiator from JAK inhibitors. He provided benchmarks for the primary endpoint (percent change in SALT score), aiming for a 30-40% reduction to be competitive with JAKs, and highlighted the importance of registrational endpoints like SALT20 and SALT10. He emphasized the potential for Respag to become the first biologic approved in this indication.

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Chief Research and Development Officer Dr. Jonathan Zalevsky explained the rationale for the three dose cohorts (24mcg/kg twice-monthly, 24mcg/kg once-monthly, and 18mcg/kg twice-monthly), which were designed to model Treg pharmacodynamics and build upon Phase Ib data. He clarified that patients must achieve an EASI-50 or better response at the end of the 16-week induction period to be eligible for re-randomization into the maintenance phase.

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Chief Medical Officer Dr. Mary Tagliaferri responded that while enrollment is on track for a first-half 2025 data readout in atopic dermatitis, the company will not provide further quantification but will update clinicaltrials.gov upon completion. She added that the current Phase IIb study is in biologic-naive patients, and it is too early to extrapolate efficacy to experienced patients without more data from other drugs in that specific population.

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CEO Anish Bhatnagar declined to provide post-quarter data but expressed confidence that VICAT XR is on track to become the standard of care. Chief Commercial Officer Meredith Manning addressed the time-to-fill, noting that as more payer policies are established, the turnaround time could initially slow before stabilizing. She stated that a 30-day turnaround is a standard target in the rare disease space.

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CEO Anish Bhatnagar declined to provide post-quarter data but affirmed confidence in VICAT XR becoming the standard of care. CCO Meredith Manning explained that the time-to-fill is variable as more payer policies are established. While encouraged by the 33% of lives covered, she noted that as more policies are added, turnaround times could fluctuate before settling toward a target of approximately 30 days.

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Executive James MacKaness advised that Q2 revenues are expected to be modest due to the multi-step conversion process and cautious inventory building by specialty pharmacies. CCO Meredith Manning added that while it is too early to provide specific metrics, the current turnaround time is consistent with other rare disease launches.

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CFO James MacKaness stated that due to the multi-step process and anticipated caution from specialty pharmacies in building inventory, Q2 revenues are expected to be modest. CCO Meredith Manning added that while it's too early for specific metrics, the conversion speed is consistent with typical rare disease launches.

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CEO Bill Sibold stated that Madrigal expects its growth trend to continue, having planned for all scenarios, including the semaglutide launch. He noted that step-edits are complex for patients with comorbidities and it's too early to know specifics. CFO Mardi Dier added that combination use is already common, with about 50% of ResDiffera patients having current or prior exposure to GLP-1s, a dynamic that is already factored into their performance.

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CEO William Sibold noted that while it is still early for definitive data, all early signs point to promising adherence rates consistent with other well-tolerated oral therapies. He attributed this to the drug's favorable profile and the positive biomarker feedback physicians are sharing with patients, which encourages continued use.

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CFO Mardi Dier reiterated that the company is not providing formal guidance at this time as it is too early in the launch. However, she extended her positive commentary to the full year, stating that based on current momentum, they expect the full-year 2025 consensus to also narrow and increase slightly.

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CEO William Sibold stated the launch is tracking well against top-tier specialty medicine benchmarks, which typically see around 50% growth in the third quarter of a launch. CFO Mardi Dier added that while specific 2025 guidance is not yet available, they anticipate that expectations for 2025 will increase to reflect the current robust growth.

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President & CEO Marcio Souza confirmed that vormatrogene showed robust efficacy across various background anti-seizure medications (ASMs), including in patients on cenobamate. He attributed strong trial recruitment to their effective operational engine, which is now being applied to POWER-1. Souza also reaffirmed previous guidance that the POWER-1 study is expected to be completed by the end of the year.

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CEO Marcio Souza stated the interim analysis for ulixacaltamide was moved to Q1 2025 to safeguard the overall program and prevent any potential negative influence on the Study 2 readout. Regarding the Parkinson's program, he explained that growing confidence in the essential tremor outcome prompted planning for a Phase II/III study, leveraging prior FDA feedback, to be ready for initiation post-ET results.

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CEO Marcio Souza confirmed that Essential3 enrollment remains on track and the interim analysis was planned from the outset as an 'insurance policy' to potentially increase sample size if needed, thereby maximizing the probability of success. He explained that the RADIANT study for PRAX-628 will provide crucial intermediate safety, PK, and efficacy data, helping to characterize the drug in an epilepsy population and serving as a first step into generalized epilepsy, all of which will support the overall registration package.

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Executive Marcio Souza explained that the interim analysis for Essential3 is a pre-planned 'insurance policy' to potentially increase sample size if needed, influenced by external factors rather than internal trial issues. He clarified this does not represent a delay. Souza also stated that the RADIANT study will provide crucial safety, PK, and efficacy data for PRAX-628, helping to characterize the molecule and offering an early look at its potential in generalized epilepsy, which will support the overall registration package.

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CEO Faheem Hasnain stated that the current baseline data is not expected to materially change and confirmed enrollment completion by mid-June, with no further updates planned before top-line results. CMO Dr. Richard Aranda clarified that while PVR was an entry criterion, it is not an endpoint. He noted a positive PROSERA result would be a positive signal for the PH-ILD study, as those patients are generally sicker, fitting the thesis that the drug is effective in more severe populations. Mr. Hasnain also highlighted seralutinib's potential antifibrotic properties as an added benefit for PH-ILD.

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CEO Michael Exton clarified that the end-of-Phase II meeting with the FDA is not considered a gating factor for ongoing strategic partnership discussions for pilavapadin. Dr. Craig Granowitz added that future data presentations will detail secondary endpoints like pain quality and functionality, plus pharmacokinetic data. For the SONATA trial, he explained that the key inclusion criterion is symptomatic disease, reflecting diastolic dysfunction, and that they have high confidence in the nonobstructive cohort based on prior HFpEF and LVH data.

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For the SONATA (HCM) study, enrollment is progressing well with sites open in the U.S. and internationally, targeting final results by late 2026 and an FDA filing in Q1 2027. For pilavapadin, they plan for an end-of-Phase II meeting with the FDA in the second half of the year, with a potential Phase III start before the end of 2025. For LX9851, they are on track to complete IND-enabling studies and file the IND in 2025.

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Dr. Craig Granowitz, SVP & Chief Medical Officer, explained that the blinded data they receive is extremely limited and primarily focused on safety monitoring. He reiterated that the tolerability profile in the current study appears different from the pilot trial but could not comment further on the overall drug profile or potential for a dose response.

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An executive, likely CSO Dr. Scot Roberts, confirmed that the IMPACT trial's baseline demographics are very similar to other MASH studies. CBO Ray Jordt explained the target population is MASH with obesity, leveraging the drug's dual action on the liver and weight loss. Jordt also stated that while Altimmune is open to compelling partnerships, moving to Phase 3 is not contingent on securing one.

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CMO Dr. Scott Harris explained that while Altimmune is at the forefront of using NITs like MRI-PDFF, the FDA's future acceptance of them as endpoints is uncertain. He noted strong internal interest in a cirrhosis study but stated that any disclosure would likely occur around the end-of-Phase II meeting, expressing high confidence in pemvidutide's potential success in the F4 population.

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CEO Dr. Vipin Garg described the FDA alignment as a "very important milestone" that provides clarity for potential partners. He explained the new indications leverage glucagon's benefits and will be detailed after regulatory clearance. CMO Dr. Scott Harris added that while the VELOCITY trials would ideally run in parallel, the exact sequencing is still under review.

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Dr. Alan Krasner, Chief Endocrinologist, explained that the study is very highly powered to detect significant differences. He clarified the endpoint is a composite responder analysis, not a co-primary endpoint. Regarding Cohort 4, he stated that it is currently enrolling and he could not yet comment on the timing of data availability.

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CEO R. Struthers stated that the communication cadence with the FDA is good. Chief Medical and Development Officer Dana Pizzuti confirmed that the mid-cycle review has not yet occurred but is scheduled, and the overall NDA review is proceeding normally with a productive relationship with the agency.

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Chief Endocrinologist Dr. Alan Krasner outlined an oncology-style Phase I dose-escalation study in patients with progressing SST2-expressing tumors, followed by dose expansion cohorts. CEO R. Struthers added that the market size is significantly larger than the carcinoid syndrome population (which is ~20% of NETs) and could potentially expand to other SST2-expressing cancers.

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CFO Mark Hahn stated that the gross-to-net discount was 'well below 20%' in Q1 and is beginning to stabilize with little room for further dramatic improvement. CEO David Zaccardelli added that while Q1 growth was blunted by industry-wide seasonality, the company expects substantial continued growth driven by an expanding prescriber base and a 'stacking phenomenon' of new and refilling patients. He emphasized that the launch is still in its early stages with plenty of room to grow.

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Mark Hahn, Chief Financial Officer, stated that the gross-to-net discount was 'well below 20%' in Q1 and is beginning to stabilize. CEO David Zaccardelli added that while Q1 growth was blunted by industry-wide seasonality, he expects substantial continued growth driven by a 'stacking phenomenon' of new patient starts and strong refills, noting the launch is still in its early stages with significant room to grow.

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Executive David Zaccardelli responded that the company does not foresee specific market or competitive headwinds due to the significant unmet need in COPD, identifying standard operational execution (manufacturing, regulatory) as the primary risk-management focus. Regarding Europe, he clarified that the current strategy is to advance the regulatory process in the EU and UK to strengthen their position for partnering discussions, not to establish their own commercial operations.

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Executive David Zaccardelli called it premature to give 2025 guidance but expects an 'enormous year of growth.' He confirmed that nearly 50% of prescriptions are for patients already on triple therapy, but use is broad across all patient types. Chief Commercial Officer Chris Martin added that the commercial focus remains on increasing the frequency of interactions with their 14,500 Tier 1 and Tier 2 physicians to accelerate adoption.

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CEO David Zaccardelli stated that while the company aims for transparency, the specific metrics shared will evolve as the launch matures. He highlighted the strong early momentum, with over 100 healthcare providers (HCPs) prescribing Ohtuvayre within the first few days of launch, indicating significant initial uptake.

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Robert I. Blum, President and CEO, firmly rejected the term "mishap," characterizing the events as a normal part of the iterative FDA review process. He stressed that the FDA's request does not reflect any new safety concerns but rather a shift in how the risk will be managed—formally through a REMS rather than just labeling, without changing the underlying differentiated profile of aficamten.

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EVP of R&D Fady Malik commented that the ODYSSEY baseline characteristics were not surprising, showing patients with significant symptoms, elevated biomarkers, and reduced exercise capacity. He noted the striking similarity to oHCM patients, which he believes bodes well for the potential effectiveness of CMIs in this population. He also highlighted that the rapid enrollment in both trials indicates nHCM is a significant unmet need.

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Andrew Callos, EVP and CCO, cited market research indicating ~25% of patients have contraindications and 80-90% experience side effects with beta-blockers. He explained the value argument for aficamten would be elevated beyond cost to focus on superior outcomes (e.g., KCCQ, NYHA class, avoiding surgery), with the MAPLE trial serving to expand the prescriber market.

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Executive Peter Salzmann confirmed that a key benefit of the trial design is the ability to disclose data from the open-label period, creating a catalyst. He stated the trial is fully powered for its primary endpoint. Dr. Peter Taylor added that achieving DAS28 remission is 'enormously meaningful' for these patients and that other metrics could be even more impressive.

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Executive Peter Salzmann confirmed that a key benefit of the trial design is the ability to disclose data from the open-label period, creating a catalyst before the final readout. He affirmed the trial is fully powered as a pivotal study. Dr. Peter Taylor added that DAS28 remission is an enormously meaningful outcome, and metrics that don't include CRP, like the CDAI, could show even more impressive results.

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CEO Barry Greene pointed to the strong Zurzuvae launch momentum. Chief Medical Officer Laura Gault highlighted the strong scientific rationale for dalzanemdor in Huntington's, noting the role of the NMDA receptor and that the genetically defined, younger, and more homogeneous patient population makes it easier to detect a clinical signal.

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CEO Barry Greene affirmed that OB/GYNs are the critical audience to unlock the PPD market. He noted that due to early success, Sage plans to expand its sales force in Q4 to deepen its reach. CBO Chris Benecchi added that early data shows physicians who prescribe ZURZUVAE begin to diagnose and treat more PPD patients overall, signaling a significant paradigm shift in their practices.

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The company plans to present data from the entire Phase II program after its FDA meeting to provide a complete picture. They are targeting the chronic TED population due to high unmet need and are confident in their ability to enroll the Phase III trials based on their current Phase II experience.

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The futility analysis in the ENSURE trials was planned from the start for the event-driven study to ensure its success and proper investment. For the CALLIPER trial, the primary endpoint is brain volume change, with key secondary endpoints like disability worsening (EDSS) also being crucial. While specific thresholds weren't given, any improvement in non-relapsing secondary progressive MS would be considered a success due to the high unmet need.

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