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President and CEO Dan Paterson explained that the NCCN committee will review the totality of the data from recent publications. Chief Commercial & Strategy Officer Michael Crowther stated the launch strategy is focused on the top 100 accounts with a 16-person team, with no current plans to adjust its size. Paterson praised the IQVIA partnership for enabling a rapid launch by providing critical data and infrastructure.

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CMO Martina Flammer explained the HS futility analysis will be based on a 'signal of efficacy' in the first 100 patients, not a specific p-value, and will be assessed by an independent expert panel. CEO William Lewis added that while brensocatib development is limited to its current indications, the next-generation DPP1 inhibitors will target large diseases like COPD, asthma, and IBD, with each new molecule aimed at a specific disease to navigate IRA constraints.

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CEO William Lewis reported positive pre-approval discussions with payers. CFO Sara Bonstein reiterated the price range of $40k-$96k, expecting to be in the upper half. For gross-to-net, she guided to a reasonable range of 25%-35%, factoring in the impact of Medicare catastrophic coverage and analogs from other specialty launches.

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CEO William Lewis described payer discussions as 'very positive' and reiterated confidence in the previously stated price range. CFO Sara Bonstein confirmed the price range of $40k-$96k and expects pricing in the upper half. For gross-to-net, she stated that while not formal guidance, a range of 25% to 35% seems reasonable based on analogs and the impact of IRA catastrophic coverage for Medicare patients.

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CEO Will Lewis explained the DMD program's novelty lies in its intrathecal delivery of an AAV9 capsid to produce microdystrophin. He stated the ALS program targets both SOD1 and sporadic patients, and the Stargardt program uses a novel RNA end-joining technology to construct a larger transgene in vivo. He indicated that clinical data from these programs would likely begin to arrive in 2026.

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CEO Will Lewis confirmed the ENCORE readout is expected in early 2026 and emphasized that the early-stage pipeline is a key priority, aiming to produce 1-2 INDs annually starting within the next year. CFO Sara Bonstein addressed OpEx, stating the quarterly increase was expected due to investments in the expanded sales force and launch readiness for brensocatib, with continued R&D spending across the portfolio.

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CEO Will Lewis confirmed the ENCORE study readout is expected in early 2026 and stressed that the early-stage pipeline (the "fourth pillar") is progressing well, with a goal of 1-2 INDs per year starting within 12 months. CFO Sara Bonstein addressed OpEx, stating the increase was in line with internal plans, driven by SG&A for the sales force expansion and brensocatib launch readiness, and R&D investments across the portfolio, including medical affairs.

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President & CEO Dr. Jeffrey Dayno framed the deal as an early-stage, strategic collaboration for next-generation therapies in refractory epilepsy and narcolepsy. Chief Medical & Scientific Officer Dr. Kumar Budur detailed the platform's technical advantages over traditional stem cells. CFO & CAO Sandip Kapadia emphasized Harmony's strong cash position ($672M) and disciplined approach to deploying capital in an attractive market.

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Dr. Kumar Budur, Chief Medical and Scientific Officer, stated the RECONNECT study is powered at over 90% for its primary endpoint and detailed rigorous patient selection and caregiver training to ensure data quality. Adam Zaeske, Chief Commercial Officer, shared that his initial focus is on learning and listening, with the goal of continuing WAKIX's strong performance and preparing for future launches by evaluating potential strategic tweaks.

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Dr. Jeffrey Dayno, President and CEO, confirmed the Novugen settlement is with one of seven ANDA filers. He stated that legal processes continue, with a Markman hearing scheduled for March, followed by a trial in 2026. While unable to comment on active litigation, he noted the settlement reinforces the strength of their IP portfolio.

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Chief Commercial Officer Jeffrey Dierks explained that WAKIX remains foundational, with pitolisant GR serving as an accretive launch to capture new and previously discontinued patients. He noted pitolisant HD is expected to become the primary product, with most patients likely converting due to its superior profile. Chief Medical and Scientific Officer Dr. Kumar Budur and CEO Dr. Jeffrey Dayno added that the full preclinical data for the orexin program is targeted for presentation at a scientific meeting in the next year.

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Chief Commercial Officer Ari Maizel confirmed summer seasonality across all three markets, particularly depression. Chief Financial Officer Nick Pizzie addressed the IRA, stating that Auvelity would likely face negotiations around 2029-2030, with potential price implementation in the 2031-2032 timeframe.

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Nick Pizzie, CFO, reiterated confidence in reaching cash flow positivity with the current cash on hand but did not provide specific guidance on the timing of profitability. He explained that R&D spending is expected to increase from Q1 levels due to new trial initiations and an NDA filing fee in Q2. SG&A will also rise with the hiring for the SYMBRAVO launch and a DTC campaign later in the year.

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CEO Dr. Herriot Tabuteau suggested that if solriamfetol is effective in MDD, a natural positioning would be for patients with comorbid depression and excessive daytime sleepiness. CCO Ari Maizel noted that for Symbravo's uptake, recent oral CGRP launches are the best analogs, but Axsome's launch will be more targeted to headache specialists and neurologists with a smaller sales force.

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CEO Herriot Tabuteau stated that with two prior positive pivotal studies, a positive result from either of the two ongoing trials would result in at least three positive trials, strongly supporting an NDA filing. CFO Nick Pizzie guided for Auvelity's GTN to remain around 50% or worsen slightly in Q4, with a negative seasonality impact in Q1 2025.

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CEO Dr. Cedric Francois reiterated Apellis's clear market leadership across all key metrics. He expressed confidence that Apellis has a better grasp on market dynamics due to its superior, long-term four-year data set compared to the competitor's older one-year data, and its more comprehensive market tracking.

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Co-Founder and CEO Cedric Francois reiterated Apellis's clear market leadership across every metric and attributed their perspective to having a more thorough understanding of market dynamics based on robust, long-term data. He contrasted Apellis's four-year data showing increasing benefits with the competitor's reliance on older, one-year data, suggesting Apellis has a better grasp on the space.

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CEO Dr. Cedric Francois reiterated Apellis's clear market leadership across all metrics and its use of more thorough market data. He contrasted Apellis's robust, four-year dataset showing increasing benefits with the competitor's reliance on older, one-year data, suggesting Apellis has a better grasp on the market's reality.

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CEO Dr. Cedric Francois reiterated Apellis's clear market leadership across all key metrics. He stressed that Apellis has a better grasp on the market dynamics, supported by superior long-term (4-year) data and a more comprehensive market tracking system, compared to the competitor's reliance on older, 1-year data.

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Executive Cedric Francois characterized the current environment as one of steady growth that takes time for adoption. He identified potential acceleration points, particularly the development of AI tools that will help physicians and patients visualize the drug's protective effect on retinal tissue and its impact on visual function. Executive Caroline Baumal added that data-driven tools from their large dataset will help physicians understand the disease and patient response, which will help grow the market.

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CMO Dr. Caroline Baumal responded that they are 'just at the start' and that 'no one should be comprised of this treatment.' She expressed optimism that with an aging population and continued education across the entire eye care provider market, not just retina specialists, the penetration rate for this chronic disease will continue to increase significantly over time.

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Gavin Wood explained the debt paydown was a strategic decision to manage the balance sheet and confirmed the 'substantial doubt' about going concern remains, declining to give specific runway guidance due to multiple variables. CEO Adrian Rawcliffe attributed the confidence in revenue guidance to strong visibility into the patient funnel, the 21 patients already apheresed, and the 100% manufacturing success rate to date.

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CEO Adrian Rawcliffe emphasized that the primary focus is a successful U.S. launch, with ex-U.S. opportunities being explored via partnerships. Executive Gavin Hilary Wood addressed the financials, noting the 10-K filing would be on Monday and reiterated the expected $50 million OpEx reduction in 2025, supplemented by new savings from pausing preclinical programs, with more detailed guidance to come.

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CEO Adrian Rawcliffe projected a faster uptake for lete-cel because the full network of ~30 treatment centers will already be established from the Tecelra launch. He expressed high confidence in the PRAME program, citing it as a broadly expressed target and highlighting Adaptimmune's expertise in TCR design. CFO Gavin Wood added that the cost savings in 2025 would be split roughly 60% R&D and 40% SG&A, with the R&D portion increasing in outer years.

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CEO Adrian Rawcliffe stated the revenue split would roughly follow the patient opportunity, which is about 40% for Tecelra and 60% for lete-cel. He also emphasized a 2-3 month frame shift for initial sales. Executive Dennis Williams added that the lete-cel regulatory path mirrors Tecelra's and that primary data from its pivotal trial will be presented later in the year.

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Daljit Aurora, Chief Operating and Development Officer, clarified the NMRA-511 study is a signal-seeking Phase Ib trial, not powered for statistical significance, with the primary endpoint being change on the CMAI scale. President Joshua Pinto explained the M4 PAM differentiation strategy focuses on high blood-brain barrier penetration to improve the therapeutic window, with more details to come in mid-2025.

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Chief Operating and Development Officer Daljit Aurora stated directly that the company has not modified the powering assumptions with respect to the overall design of the KOASTAL-2 and 3 studies.

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Chief Financial Officer Joshua Pinto stated that with two successful studies, the company would be positioned to file for approval. He affirmed that Neumora's current balance sheet and team are set up to support commercializing the program independently within the U.S.

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Richard Miller, an executive, clarified that the extension cohort will exclusively test the 200-milligram BID dose. He explained that the slight drop in Cohort 2's EASI 75 results was due to small numbers and some patients being very close to the threshold. He expressed confidence in the Cohort 3 data, noting the tight standard error bars and consistent results at day 15. Miller stated that the complete Cohort 3 data could be shared in a press release within a month or two.

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Executive Richard Miller provided a detailed background on ALPS, a genetic disease affecting about 2,500 patients in the U.S. He explained that soquelitinib was dramatically effective in a mouse model with the identical genetic mutation. Miller stated that there is no known patient subset that would be more or less amenable to the therapy. He highlighted the trial as a key opportunity to study the drug's effect on various autoimmune manifestations, which could have relevance for other diseases.

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Executive Richard Miller confirmed the company will report a full range of efficacy metrics, including mean EASI reduction and EASI-50/75/90 scores. He expressed confidence in the drug's mechanism, stating the goal is to be superior to, not just comparable with, injectable therapies like Dupixent. He declined to comment in detail on other oral competitors.

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President and CEO Dr. Jay Duker confirmed that the lack of co-pay assistance programs is likely boosting enrollment for all wet AMD trials. Chief Medical Officer Dr. Ramiro Ribeiro added that investigators choose their trial due to the robust Phase II safety and efficacy data, patient-centric design, and strong support from EyePoint's teams.

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CEO Dr. Jay Duker confirmed that the lack of funding for the 'Good Days' co-pay program is likely helping enrollment for all wet AMD trials, as study participation offers treatment at no cost. Chief Medical Officer Dr. Ramiro Ribeiro reiterated that the key differentiator driving enrollment is the robust Phase II data, which gives physicians confidence in DURAVYU's efficacy and safety, along with a patient-centric design and strong site support.

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CFO George Elston highlighted that the state-of-the-art facility is online and the team has begun activities for registration batches to support an NDA filing. President and CEO Dr. Jay Duker added that the facility was built with foresight to meet FDA and EMA specifications and is expected to supply the entire global demand for DURAVYU.

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The executive, Jay Duker, responded that formal market research is ongoing, so he could not share quantitative results. However, he noted that qualitative feedback from both payers and practitioners has been very positive. He mentioned that the retina community is increasingly enthusiastic about EYP-1901's potential, citing a recent conference poll where it was named the most exciting new agent in development.

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David Berman, Head of R&D, detailed the strategy, which includes expanding brenetafusp into chemo combinations for platinum-resistant ovarian cancer and exploring combinations with osimertinib and docetaxel in lung cancer. He indicated that data would be shared within the next 12 to 18 months, once a comprehensive data story is available.

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Ralph Torbay explained that ex-U.S. sales are relatively flat because they have already achieved high penetration in key European markets. Future growth will be primarily driven by the U.S. market through deeper penetration into community oncology practices. Bahija Jallal added that ongoing country-by-country price negotiations in Europe also play a role.

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The company received direct and actionable feedback from the FDA on the TRANQUILITY protocol. They are actively pursuing all financing options to strengthen the balance sheet and fund operations through the upcoming data readout, but did not provide specific guidance on cash runway sufficiency.

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Executive Vincent O'Neill described the FDA feedback as 'direct, actionable, and very concise' but did not provide specific details. Executive Vimal Mehta addressed financing, stating the company is exploring all previously outlined options with its strategic partners to fund operations through the data readout. He confirmed no specific cash runway guidance has been provided but the trial was initiated with the goal of reaching completion.

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Richard Steinhart, an executive, stated that guidance on the cash runway remains unchanged. Vimal Mehta, an executive, explained that the strategy is to fund both trials through various alternatives, including royalty monetization, OnkosXcel monetization, and strategic partnerships, which are being explored in parallel.

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The pivot to an efficacy study was due to the risk and challenge of using nonvalidated caregiver instruments. The company will provide more details on trial design and costs after receiving FDA minutes, but states the trials are capital-efficient and all financing options are being explored.

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Dr. Camille Bedrosian, Chief Medical Officer, clarified that the composite endpoint is well-established in diabetes trials and captures all clinically meaningful events. Co-CEO Justin Klee added that strong effects were seen on both Level 2 and 3 events in prior studies. For the PSP trial, Dr. Bedrosian stated the decision will be based on clear clinical activity, primarily the change in PSPRS score versus placebo, along with biomarker data.

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Dr. Gary Romano, Chief Medical Officer, explained that the proportional MMRM method is particularly advantageous for the trial's common close design, as it allows for the use of all data from patients with varying treatment durations. He noted that while novel, the method has been used in other Alzheimer's studies and was selected in agreement with partner AbbVie to best analyze the biomarker-rich dataset and increase statistical power.

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CMO Richard Bryce reiterated that the study is 94% powered to show a hazard ratio of 0.50. CEO Avanish Vellanki expressed high confidence in the Q2 timeline. SVP of Finance Nelson Cabatuan and CSO Robert Doebele clarified that cash runway guidance was withdrawn due to the proximity of the pivotal MANTRA results, which will dictate future spending, not due to a change in the burn rate.

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CEO Laurent Fischer explained that the company started with a high dose and is now straddling the effective 2E11 dose for first-line therapy. Dr. Arshad Khanani (Managing Director, Sierra Eye Associates) added that he expects potent efficacy from the new doses, especially in the less difficult-to-treat naïve patient population. CMO Aaron Osborne noted the FDA was aligned with this dose-ranging approach. Regarding DME, Laurent Fischer stated that topline data from the fully enrolled INFINITY study will be reported in the second half of the year.

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Chief Medical Officer Aaron Osborne clarified that the patient, along with another in Cohort 3, had stable vision and only minimally met the retreatment criteria. He suggested this indicates the current criteria may be too strict for a continuous delivery therapy and that the pivotal trial may adopt more relaxed criteria, similar to other long-acting therapy programs. He affirmed that both high and low doses continue to show a strong benefit.

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Chief Medical Officer Aaron Osborne justified using the same doses as in OPTIC by noting that standard anti-VEGF therapies use similar dosing for both indications, but mentioned the trial design allows for adaptation. President & CEO Leone Patterson stated that guidance on INFINITY data timelines would be provided after the first patient is treated.

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