Lin Tsai of Jefferies questioned the company's confidence in its Q2 guidance for 100-110 patient infusions, asking for confirmation of a current spike in patient uptake to support the forecast.
Chief Commercial Officer Daniel Kirby affirmed the company's confidence in the Q2 guidance, stating that demand in the second quarter is strong and supports the projected number of patient infusions.
Lin Tsai of Jefferies Financial Group Inc. asked about the competitive efficacy targets for Bysanti in Major Depressive Disorder (MDD), the study design for VQW-765 in social anxiety, and the regulatory strategy for tradipitant in gastroparesis following its Complete Response Letter (CRL).
President and CEO Dr. Mihael Polymeropoulos explained that for Bysanti, a specific placebo-adjusted margin on MADRS/HAM-D is not prespecified due to high variability in depression studies. For VQW-765, he noted the Phase III study will begin in Q3 2025, with a design similar to a previous study using the Trier test, which is soon to be published. Regarding tradipitant, he described the ongoing and complex FDA hearing process, confirming that a new filing is not required as it is part of the original review cycle.
Lin Tsai inquired about the sales trends for Ohtuvayre moving from Q1 into Q2 2025, asking for color on metrics in April and how the sales curve might evolve through the rest of the year, considering seasonality and refill rates.
CEO David Zaccardelli, while declining to provide Q2 projections, confirmed the launch is accelerating across all metrics, including prescriptions, new prescribers, and refills. He noted that while the launch is still early, the company is confident in its growth trajectory, which will build as new patients are added and the refill business grows. Zaccardelli acknowledged the vast untapped market of symptomatic COPD patients, suggesting significant room for continued growth.
Lin Tsai of Jefferies inquired about Ohtuvayre's sales trends moving from Q1 into Q2, the potential impact of Q1 seasonality, and the anticipated shape of the sales growth curve for the remainder of the year.
David Zaccardelli, Chief Executive Officer, declined to provide forward-looking guidance for Q2 but affirmed the launch is performing exceptionally well across all metrics, including prescriptions, new patients, and refills. He acknowledged the typical Q1 seasonality but expressed strong confidence in continued growth, emphasizing the large, untapped patient population and the early stage of the launch.
Lin Tsai inquired about the potential Q1 impact from insurance resets on sales and gross-to-net margins, and also asked for commentary on the 2025 revenue consensus and the timeline for achieving breakeven.
Chief Financial Officer Mark Hahn explained that while commercial co-pay assistance could be affected, the overall impact on gross-to-net in Q1 should be minimal due to the small percentage of commercial patients. Chief Commercial Officer Chris Martin added that he believes the strong launch momentum will outweigh any negative effects from deductible resets. Regarding breakeven, Mark Hahn stated that a cash flow breakeven run rate is achievable at an annual sales rate of $250 million to $300 million, which could be reached by the end of the year. Executive David Zaccardelli expressed confidence in the growth trajectory without directly addressing the consensus figure.
Lin Tsai asked for clarification on the statement that October net sales exceeded the entire third quarter's sales, questioning if this implies a Q4 run-rate of at least $17 million. He also inquired about potential headwinds like holiday slowdowns or patient drop-offs, and asked for the number of unique patients as of October.
David Zaccardelli, an executive at Verona Pharma, confirmed the underlying math but did not provide formal Q4 guidance, stating the company sees no reason for a slowdown and expects continued acceleration. He noted that the vast majority of the 5,000+ prescriptions filled are for unique patients and that early refills are just beginning, characterizing the patient growth as fluid and accelerating week-over-week.
Lin Tsai inquired about the expected payer rejection and patient abandonment rates for Ohtuvayre, the availability of a free drug program, and whether the company was comfortable with Q3 consensus revenue estimates, which imply around 1,400 patients.
CEO David Zaccardelli confirmed the existence of patient support and bridging programs instead of a traditional sampling program. Chief Commercial Officer Chris Martin elaborated that while it's early, they anticipate lower abandonment rates as Ohtuvayre is reimbursed under a medical benefit, not pharmacy. He also noted the submission of a product-specific J-code application. CFO Mark Hahn cautioned against simple patient number calculations for revenue, highlighting that channel inventory fill will also be a factor.
Lin Tsai of Jefferies inquired about Avadel's revised 2025 guidance, asking if the implied second-half growth was achievable and seeking a rank-ordered list of the key growth drivers. He also asked if the recent favorable court ruling was a base-case or best-case scenario and what the next litigation steps are.
CFO Thomas McHugh confirmed the guidance implies continued sequential quarterly growth in the second half of the year. CEO Gregory Divis attributed the strong performance to broad improvements across all key metrics, including increased patient demand from sales force expansion and better patient pull-through from the reimbursement team. Divis characterized the court ruling as their 'base case assumption' and a critical win that provides a clear path to pursue an FDA submission for LUMRYZ in Idiopathic Hypersomnia (IH).
Lin Tsai asked about patient trends observed in October and early November, the company's confidence in a strong Q4 growth trajectory despite seasonality, and whether LUMRYZ will continue to gain share from all patient segments, including existing oxybate users.
Richard Kim, an executive, confirmed that strong demand for LUMRYZ has continued into the fourth quarter. He acknowledged potential seasonal impacts from fewer office visits and selling days, as mentioned by CFO Tom McHugh. Kim reiterated that switch patients remain a core and important part of the business, even as the new-to-oxybate segment shows accelerated growth.
Lin Tsai from Jefferies sought confirmation that the recent settlement with Teva establishes the earliest possible generic entry for Auvelity. He also asked if this extended patent protection emboldens Axsome to pursue additional indications for AXS-05 beyond the currently planned ones.
Hunter Murdock, General Counsel, confirmed that Teva has a 180-day regulatory exclusivity, making it the first generic to launch. Herriot Tabuteau, CEO, added that the company has always planned to pursue other indications for AXS-05 and reiterated that the near-term focus remains on the sNDA filing for Alzheimer's disease agitation and initiating a Phase III trial for smoking cessation.
Lin Tsai from Jefferies questioned whether the initial single ascending dose (SAD) data for the DM1 and FSHD programs would be conclusive enough for investors to assess their potential superiority, or if MAD data would be required.
CEO Douglas Ingram characterized the upcoming SAD data as a very important proof-of-biology moment, highlighting safety, muscle concentration, knockdown, and gene splicing. While cautioning against overselling early data, he noted it should provide signals of the potential product profile. Dr. Louise Rodino-Klapac added that they will be looking for the strong preclinical-to-clinical translation seen in other Arrowhead programs, which would be a good indicator of future success.
Lin 'Andrew' Tsai of Jefferies asked for reconciliation of a comment from partner Roche about 450 U.S. patients being treated with ELEVIDYS, suggesting this could imply a Q4 sales figure above Sarepta's current guidance.
President and CEO Doug Ingram declined to confirm or comment on the number provided by Roche, stating that Sarepta will use revenue as its primary performance metric and is standing by its existing guidance. He qualitatively noted that an extraordinary number of patients have been dosed, including over 80 late-ambulatory or non-ambulatory patients, with a consistently positive safety profile.
Lin Tsai asked about the assumed placebo rate for the ongoing X-TOLE2 and X-TOLE3 studies and what measures are being taken to control for placebo risk.
CEO Ian Mortimer stated that their statistical models are based on the actual high-teens placebo rate from the Phase II X-TOLE study, providing over 99% power for the high dose. CMO Dr. Christopher Kenney added that placebo risk is being mitigated through careful selection of geographic regions, experienced clinical sites, and the use of electronic diaries to ensure high-quality data.
Lin Tsai from Jefferies asked for clarification on the latest SPN-817 seizure reduction data, the potential positive read-through from AbbVie's recent pump approval for SPN-830, and whether the strong MADRS reduction seen in the SPN-820 MDD study could be replicated in the upcoming TRD study.
Executive Jack Khattar confirmed the robust SPN-817 data, noting seizure reduction improved to 66% in the post-maintenance extension period, which informed the Phase IIb dose selection. Regarding the SPN-830 pump, he stated that a competitor's launch is welcome as it helps build the market category. For SPN-820, Khattar acknowledged the challenge of predicting placebo-controlled results but emphasized that the open-label data, showing rapid and high remission rates, is exceptionally strong.