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Interim CEO Ian Smith confirmed that Stoke Therapeutics will present its comprehensive data, including the new 36-month OLE results, to the FDA in the second half of the year. He highlighted that the breakthrough designation was granted based on both seizure reduction and cognitive benefits. While acknowledging the responsibility to seek the fastest path to patients, he noted that the outcome of FDA discussions is uncertain and that the company will provide updates if timelines change.

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Dr. William Rote, Chief Research Officer, clarified that the specific date for the advisory committee is not yet known but will be shared once it is. He added that given the January 13 PDUFA date, it is reasonable to anticipate the meeting will occur sometime in Q4.

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Chief Commercial Officer Peter Heerma and CEO Eric Dube both responded. Heerma highlighted that strong compliance and persistence build upon a robust base from new PSFs. Dube added that a key sustainable growth driver is upgrading the large number of patients currently on RAS inhibitors who are not yet at the new, more aggressive KDIGO guideline targets.

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CEO Eric Dube stated that while the company is pleased with the strong uptake in Q4 following full approval, it represents only a single data point. Management prefers to observe more trends before providing formal guidance but is consistently evaluating the possibility.

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Chief Medical Officer Dr. Jula Inrig stated that an update is a potential outcome. She noted that while FSGS guidelines were recently updated, guideline bodies typically revise them when a new treatment becomes available. She anticipates that a full FDA approval for sparsentan in FSGS would be the appropriate trigger for such an update.

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CEO Mike Raab expressed high confidence in the $750 million peak sales target for Xphozah, citing the large non-Medicare market and modest penetration required. He stated guidance would be provided after seeing more consistency. For Ibsrela, he noted its average duration of therapy is trending at or better than the market average of about five refills.

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CEO Michael Raab and CCO Eric Foster identified key metrics as increased sales activity, growth in new and total prescribers, and a corresponding rise in new and refill prescriptions. They cited Q1 having the second-highest prescription demand quarter to date, despite market headwinds, as a primary reason for their confidence in reaching the greater than $1 billion peak sales target.

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CCO Eric Foster confirmed that both Medicare and non-Medicare patients from 2024 are successfully transitioning to 2025 access pathways, though specific retention metrics were not provided as it is too early. CFO Justin Renz explained the loan strengthens the balance sheet, providing strategic flexibility for growth and pipeline development.

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Chief Financial and Operations Officer, Justin Renz, confirmed that XPHOZAH's GTN deduction improved from 21.4% to 19.3% in Q3, driven by patient payer mix and commercial co-pay utilization. President and CEO, Mike Raab, indicated that 2025 IBSRELA guidance would be provided in the early part of next year, after the impact of the expanded sales force becomes clearer.

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President & CCO Charlie Gayer referenced a recent real-world data analysis showing Orlodeyo's one-year persistence rate is statistically identical to injectable competitors TAXHZYRO and HAEGARDA, at approximately 60%. He noted that Orlodeyo was numerically slightly better, which underscores that the drug's profile is highly competitive and meets patient needs effectively in a real-world setting.

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Chief Commercial Officer Charlie Gayer stated the company would aim for a minimal lag between approval and commercial availability for pediatric ORLADEYO. He noted uptake dynamics will differ as an oral option could shift the treatment paradigm toward earlier prophylaxis in children. For the Netherton trial, Chief R&D Officer Dr. Helen Thackray confirmed the starting patient dose is 6 mg and clarified that the 12-week dosing portion is a parallel extension to gather longer-term data, not a phase triggered by a specific event.

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CFO Matt Abernethy first addressed capacity, stating there are no constraints as the treatment fits into existing care workflows. CEO Kyle Gano then reiterated that the number of transitioning trial patients is minimal—less than 40 in total, split across Q2 and Q3—and does not significantly impact the overall new patient numbers.

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Chief Commercial Officer Eric Benevich stated that INGREZZA's patient persistency has remained very steady since launch and does not differ meaningfully by channel. He noted that while Q1 reauthorizations were more challenging than in prior years, the impact was counterbalanced by the record number of new patient starts.

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Chief Commercial Officer Eric Benevich stated that the primary benefit of the expansion is expected to be in driving new patient starts. He explained that the larger team will increase reach and frequency of contact with providers, particularly in the high-potential psychiatry and long-term care segments. He also noted that the full impact will take time to materialize, with the majority expected in the second half of the year.

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CCO Eric Benevich stated that while there's no standard definition, literature suggests half to two-thirds of patients are moderate-to-severe. He emphasized that even milder movements can be significantly impactful, and the focus remains on the large, untreated population. He noted the 800,000 figure is supportable and likely conservative, reinforcing the significant growth opportunity ahead as less than 20% of patients are treated.

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CEO Faheem Hasnain and CMO Dr. Richard Aranda projected that by seralutinib's launch, a large market of patients who failed, were ineligible for, or had waning effects from sotatercept would exist. They also see potential for combination use and positioning seralutinib as a first-choice therapy for new patients due to its safety profile. For the CIRANATA trial, Dr. Aranda explained they extrapolated from the INCREASE trial, assuming a sicker population and a 30-meter treatment difference, which provides over 94% power.

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CCO Erik Harris and CEO Emil Kakkis attributed the strong growth to positive patient and physician experiences, which are driving broader adoption, including in adult patients. They highlighted that securing national reimbursement in Brazil and Mexico was a critical catalyst for uptake in Latin America. While Turkey remains a named-patient market, strong word-of-mouth and physician conviction are fueling demand. They noted this regional infrastructure bodes well for the future setrusumab launch.

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COO Chris Benecchi stated that Sage has not communicated the specific number of active prescribers. CEO Barry Greene added that the key dynamic driving growth is the continuous addition of new writers each quarter who then become repeat prescribers. While he could not quantify the scripts per provider, he reiterated that prescribing offices see significant growth in their PPD patient treatment and use ZURZUVAE as a frontline therapy over 70% of the time.

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CEO Barry Greene stated that the current ACOG guidelines are already 'phenomenal,' as they were updated around the time of launch to specifically mention ZURZUVAE, which has been a significant advantage. COO Chris Benecchi added that these favorable guidelines were one of several key tailwinds supporting the launch, alongside strong media attention and broad payer access.

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CEO Barry Greene described this as an area of continuous improvement and a key part of ZURZUVAE's value proposition. He stated that while many patients receive the drug within 2-3 days, the company is not satisfied and is working to shorten the median delivery time for all patients. He characterized current performance as 'doing well' but with room to 'get better.'

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CEO Marcio Souza clarified that the information fraction for the interim analysis (50-75% of patients) has not changed. He explained the separation of study results is a cautious measure to protect the integrity of Study 2 from potential patient bias. The Parkinson's program restart is driven by a strong scientific rationale and a desire to be prepared to launch immediately following positive essential tremor results, leveraging go-to-market synergies.

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CFO Timothy Sullivan confirmed the cash runway guidance 'absolutely does' include the C3G launch, noting the incremental costs are not massive. COO Adam Townsend addressed the second point, stating that the use of every-other-month dosing has been very stable, accounting for 80% to 90% of all SYFOVRE prescriptions, and is expected to remain at that level.

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CMO Dr. Toby Ferguson deferred specific questions on the partnered programs to Neurocrine, which controls their development, but noted that other approved drugs have paved a path by targeting broad patient populations. He declined to comment on future milestones. CEO Dr. Al Sandrock added that Voyager's frataxin-replacement therapy for Friedreich's ataxia has a distinct mechanism from the approved drug, suggesting potential for additive benefit.

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Executive Robin Swartz stated that while a milestone was achieved, the company does not provide guidance on future potential milestone payments, noting they are not included in the cash runway guidance into 2027. Executive Alfred Sandrock addressed the strategic question by explaining that a positive result for VY-TAU01 would likely lead to seeking a partner for late-stage development, given the scale of Alzheimer's trials. He also noted the option to vectorize the antibody for a 'once and done' treatment remains a high-interest possibility.

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President and CEO Stephen Mahoney stated that a successful outcome for the THRIVE trial would be an efficacy and safety profile similar to TEPEZZA. He noted that lower drug exposure and a lower Cmax could potentially improve the hearing safety profile, but this remains to be seen. Mahoney explained that the STRIVE study is a standard requirement to build the safety database for a BLA submission, and the active control arm is necessary for it to be a well-controlled study.

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On exon skipping, the executive noted that NHP data shows the largest increase between the first and second doses, with levels plateauing by the fourth dose, which is expected to be reflective of long-term results. For DM1, greater than 25% splicing correction is considered important, as preclinical models show this leads to robust myotonia correction. The FREEDOM1 update will include functional assessments like vHoT (myotonia), strength tests, and walk tests, though meaningful changes in some functional measures may require multiple doses.

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CEO Will Chou explained that protocol amendments are subject to regulatory review, but the goal is to treat patients at an earlier stage to maximize the risk-benefit profile. Chief Medical Officer Mark Forman stated that NHP availability has not impacted their preclinical pipeline to date, though it is being carefully monitored.

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CEO Will Chou explained that any protocol amendments are subject to regulatory review and that the goal is to treat patients at an earlier stage to maximize the risk-benefit profile. CMO Mark Forman addressed the second question, stating that NHP availability has not impacted the advancement of their preclinical pipeline to date, though it is being carefully monitored.

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Chief Medical Officer Mark Forman explained that he expects a low screening failure rate for FTD due to broad inclusion criteria. He confirmed the 60-day waiting period between patients is in effect for the entire trial to ensure safety. For GM1, he stated the company seeks regulatory alignment on the use of external cohorts and the overall registrational study design.

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Interim CEO Edgar Cale addressed the CEO search, confirming the Board has engaged Russell Reynolds, the search is active, and they are making good progress in evaluating candidates. Chief Medical Officer Mark Forman explained that FTD recruitment challenges are multifactorial, involving patient identification, competition from other trials, and patients meeting specific eligibility criteria. However, he reiterated that their outreach and genetic testing initiatives are proving successful.

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President of Biohaven Labs Michael Bozik explained that by avoiding GABA activation, BHV-7000 is expected to have a superior side effect profile (less somnolence, vertigo) and better compliance, which will be a key point of differentiation measured in clinical trials. CEO Vlad Coric clarified that for the HEALEY ALS platform trial, Biohaven will receive and announce topline data for its asset, verdiperstat, as soon as it reads out around mid-year, without waiting for other assets in the study.

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CEO Dr. Vlad Coric clarified that the company's strategic priority was to secure an ex-U.S. partner, as the U.S. launch is already outperforming expectations, and Biohaven retains full U.S. optionality. For Europe, he explained that while pricing processes are ongoing, the core value proposition of reducing migraine-related disability will be a central argument for payers, similar to the successful strategy employed in the U.S.

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CCO BJ Jones did not provide specific data but suggested competitor data could be skewed by multi-dose regimens. He stressed that Biohaven sees a high degree of patient satisfaction with NURTEC ODT. CEO Vlad Coric added that NURTEC's single-dose efficacy is a more efficient treatment approach and cautioned against misinterpreting script data that reflects the need for second doses with other therapies.

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CEO Vladimir Coric attributed NURTEC's favorable views to its differentiated label, which he believes will drive adoption in primary care. CCO BJ Jones added that as markets reopen, they will focus on activating patients and broadening physician communication to increase productivity. The capital allocation question was not directly addressed.

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