Laura Chico • Wedbush Securities

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

CEO Faheem Hasnain and CCO Bob Smith outlined a future market where seralutinib could target patients who have failed or are ineligible for sotatercept, as well as new patients, positioning it as a first-line option due to its safety profile and potential for long-term improvement. CMO Dr. Richard Aranda explained the PH-ILD trial's powering was extrapolated from the INCREASE trial, assuming a sicker population and targeting a ~30-meter treatment effect, resulting in over 94% power.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

CEO Marcio Souza clarified that the interim's information fraction range (50-75%) remains unchanged. He explained the separation of results is a precaution to protect the integrity of Study 2 from potential patient influence. The Parkinson's program restart is a strategic preparation to act quickly on positive ET data, leveraging a stronger safety profile and market synergies.

Ask Fintool Equity Research AI

Answer

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.

Ask Fintool Equity Research AI

Answer

President and CEO Stephen Mahoney stated that a profile similar to TEPEZZA would be considered successful for both efficacy and safety. He noted that lower drug exposure and Cmax could potentially improve the hearing profile but that a similar profile would be a good outcome. He explained the active control arm in STRIVE is a standard requirement for a well-controlled study needed for the BLA safety database.

Ask Fintool Equity Research AI