Gary Nachman

Gary Nachman of Raymond James Financial asked about strategies for expanding Ascentive's prescriber base, the timeline for HEOR data, potential gross margin headwinds, and the long-term plan and capital outlay for the newly acquired facility.

Answer

CEO Adam Grossman confirmed new doctors are continuously being added, supported by medical education and a field reimbursement team. CFO Brad Tade stated there are no current headwinds to gross margin expansion. Regarding the new facility, both executives clarified it supports near-term scalability and provides future manufacturing expansion options, ensuring the $1.1B revenue target by 2030 is a 'milestone, not a ceiling.'

Gary Nachman asked how the 20% yield enhancement will affect the management of the ASCENIV patient queue and how quickly new patients can be served. He also requested an update on the status of HEOR data for ASCENIV, the reason for the Q1 voluntary product withdrawal, and the potential strategies for extending ASCENIV's intellectual property beyond 2035.

Answer

President and CEO Adam Grossman stated that while the patient queue for ASCENIV persists, the company is releasing more product than ever and adding new patients weekly. He confirmed that health economic data is on track for publication by year-end to support payer access. Regarding the withdrawal, he explained it was a one-time, voluntary action for a $3.8 million impact due to known, labeled adverse events, taken in the interest of patient safety. On IP, Grossman expressed confidence in extending patents beyond 2035 by leveraging novel internal R&D methodologies, similar to the innovation that led to the yield enhancement approval.

Gary Nachman questioned the size of the ASCENIV patient queue, gross margin trajectory, the potential for an FDA inspection for the yield enhancement process, and the development timeline for the SG-001 pipeline program.

Answer

President and CEO Adam Grossman described the patient queue as 'robust' and noted patients remain on standard IG therapy while waiting, mitigating risk. He projected ASCENIV's 80-85%+ gross margins would continue to expand, especially with the yield enhancement. Grossman also conveyed high confidence in a mid-year approval for the yield process without a pre-approval inspection, based on constructive FDA dialogue. Regarding SG-001, he expects animal data later in the year and noted significant excitement about the program's potential.

Gary Nachman from Raymond James questioned if ADMA needs more of its own plasma centers to reach its $1 billion ASCENIV target, the security of its proprietary screening methods, the conservatism in its full-year guidance, the timeline for SG-001 animal data, and the long-term nature of the new KPMG audit relationship.

Answer

President and CEO Adam Grossman stated that the company does not need more than its 10 highly efficient plasma centers and assured that its patented screening methods are kept in-house, with third parties only receiving donor numbers. He explained the conservative Q4 outlook was due to a non-recurring Q3 spot plasma sale and the discontinuation of a low-margin product, while profit guidance was raised due to a favorable product mix shift. He projected potential SG-001 animal data in the latter half of 2025. Both Grossman and CFO Brad Tade affirmed that KPMG is their long-term auditor for the foreseeable future, representing an important milestone in the company's maturation.

Gary Nachman asked about the sources of additional FCS patients for Tringolsa, the number of prescribing physicians, the split between genetic and clinical diagnoses, and the reason for a projected slowdown in Tringolsa's Q4 acceleration. He also inquired about the possibility of priority review for Olzarsen's SHTG filing.

Answer

Brett Monia, CEO, stated that Olzarsen's SHTG filing is currently assumed for standard review (10 months) but Ionis will pursue all avenues for faster market entry. Kyle Jenne, Chief Global Product Strategy Officer, explained Tringolsa's success is driven by patient identification, HCP education, clinical scoring tools, and streamlined payer processes. He did not provide specific numbers for HCPs or diagnosis splits. For Q4, he cited the shorter duration of the quarter due to holidays and unknown seasonality as reasons for conservative guidance.

Gary Nachman asked about the sources of additional FCS patients for TRYNGOLZA, the number of prescribing physicians, and the split between genetically and clinically diagnosed patients. He also questioned the projected slowdown in TRYNGOLZA's Q4 acceleration and the possibility of priority review for olzarsen in sHTG.

Answer

Brett Monia, CEO, Ionis, stated that olzarsen's sNDA filing assumes a standard 10-month review but the company will pursue all avenues for faster market entry. Kyle Jenne, Chief Global Product Strategy Officer, Ionis, attributed TRYNGOLZA's momentum to patient identification, disease state awareness, and improved clinical diagnosis tools. He noted that Q4 guidance reflects the shorter duration of the quarter and potential seasonality, as it's the first full year of launch.

Gary Nachman of Raymond James Financial asked about the momentum of the TRINGOZA launch in FCS, the basis for the strong back-half guidance, expectations for the upcoming SHTG data, and the status of Donidalorsen's FDA review and launch readiness.

Answer

CEO Brett Monia, CGO Kyle Jenne, and CDO Richard Geary responded. Jenne detailed the successful TRINGOZA launch driven by effective patient identification, a strong product profile, and favorable reimbursement. Monia stated that for SHTG, physicians prioritize substantial triglyceride lowering above all else, though a favorable trend in acute pancreatitis (AP) events is expected. Geary confirmed the Donidalorsen PDUFA date is on track for August 21, with the company fully prepared for launch.

Gary Nachman asked about pre-launch preparations for donidalorsen, the expected patient switching dynamics, the market access timeline, the potential impact of new tariffs on the business, and any signs of FDA delays.

Answer

Chief Global Product Strategy Officer Kyle Jenne described the HAE market as having a high rate of patient switching, positioning donidalorsen's profile favorably for uptake with peak sales potential over $500 million. CEO Brett Monia stated that to date, there has been no meaningful impact from tariffs on costs or supply. He also confirmed that interactions with the FDA remain on track, with no delays anticipated for the donidalorsen PDUFA date or other clinical programs.

Gary Nachman of Raymond James asked about the scaling of the commercial organization for upcoming launches in HAE and sHTG, and also inquired about enrollment timing expectations for the competitive Angelman syndrome program.

Answer

Chief Global Product Strategy Officer Kyle Jenne described a purposeful, sequential build of the commercial team, leveraging the core infrastructure already in place and hiring new field teams closer to launch dates. CEO Brett Monia addressed the Angelman program, confirming the Phase III study is on track to start in H1 2025 with a goal of completing enrollment in 2026, citing strong enthusiasm from the patient community as a key factor.

Gary Nachman inquired about the launch preparations for olezarsen in FCS, including the expected timing post-approval, pricing strategy, and the status of labeling discussions. He also asked about the Phase III study design for ION582 in Angelman syndrome, specifically regarding plans for interim data looks and the FDA's alignment on the primary endpoint.

Answer

CEO Brett Monia confirmed that labeling discussions for olezarsen are progressing well and that no interim looks are planned for the Angelman study, noting easy alignment with the FDA on the trial's design. Chief Global Product Strategy Officer Kyle Jenne added that Ionis is prepared to launch olezarsen before the end of the year with an ultra-rare disease pricing model.

Gary Nachman asked about the overall growth and underpenetration of the dry eye market, how Bausch + Lomb plans to invest in MIEBO going forward (tapering spending vs. growing the market), and the company's strategy for accelerating U.S. generics, including its long-term commitment to the business.

Answer

Chairman and CEO Brent Saunders stated that the dry eye market is growing around 10%+ and is sustainable for several years, with combination therapy offering additional expansion. He noted that MIEBO spending would involve surgical tapering for highest ROI, not significant cuts, to maintain the largest field force. For U.S. generics, Mr. Saunders described it as an opportunistic business that creates plant absorption and profitability during market disruptions, emphasizing its role in maintaining profitability rather than sales growth.

Gary Nachman inquired about the overall dry eye market growth, its underpenetration, and how these factors influence future investment strategies for MIEBO, particularly regarding tapering spending while still aiming for meaningful market growth. He also asked about plans to accelerate U.S. generics and the long-term commitment to this business.

Answer

Chairman and CEO Brent Saunders estimated the dry eye market is growing at 10%+ and can sustain this for several years, with future combination therapies offering additional expansion. He noted a 'surgical tapering' of MIEBO spending to maximize ROI while maintaining the largest field force. For U.S. generics, he explained it's an opportunistic business for plant absorption and profitability during market disruptions, rather than a primary growth driver, showing sequential improvement.

Gary Nachman of Raymond James requested more detail on the Envista recovery, including physician adoption and any ancillary impacts from the recall. He also asked if MIBO's growth stems from new patients or brand switches, and questioned the need for business development to bolster the pharma pipeline.

Answer

CEO & Chairman Brent Saunders explained that the transparent, patient-first handling of the Envista recall helped build customer trust, with loyal surgeons returning immediately. He expects to recapture full momentum by Q1 of next year. On dry eye, he confirmed MIBO is expanding the market by attracting new patients, thanks to its efficacy, tolerability, and strong insurance coverage. Both Saunders and EVP of R&D Yehia Hashad expressed high confidence in the existing innovative pharma pipeline, which is focused on disruptive therapies, thereby reducing the immediate need for major business development.

Gary Nachman from Raymond James asked about Veru's expectations for its end-of-Phase 2 FDA meeting, potential areas of regulatory pushback on the Phase III design, and the plan for communicating the meeting's outcome. He also followed up on the modified-release formulation's safety profile and the requirements to use it in Phase III.

Answer

Chief Scientific Officer, Gary Barnette, stated that the primary goal is obtaining clarity from the FDA. He anticipates the FDA will require a physical function endpoint, which Veru considers a positive outcome, and noted the FDA might encourage expanding the trial to younger patients. Chairman, President & CEO, Mitchell Steiner, indicated that official feedback would likely be communicated in the September timeframe. Dr. Barnette added that the new formulation's lower Cmax could enhance its safety profile while its similar AUC should ensure comparable efficacy, which will be confirmed via a relative bioavailability study for the FDA.

Inquired about the success criteria and data timing for the Phase IIb extension study, the potential design of the Phase III trial (size, dose, patient population), and any manufacturing or sourcing concerns related to tariffs.

Answer

Success for the extension study would be demonstrating that enobosarm can blunt fat regain after stopping a GLP-1. The safety data will be released first this quarter, followed by the extension study data. The Phase III trial is anticipated to enroll approximately 400 patients in an older population, using a single dose (likely 3mg), and may include patients on both semaglutide and tirzepatide. The company does not foresee significant issues with tariffs due to the low cost of goods for enobosarm.

A representative for Gary Nachman of Raymond James & Associates, Inc. asked about the progression of the Phase IIb trial, whether to expect interim updates before the January topline data, the current assessment from the safety committee, and the significance of secondary endpoints like HOMA-IR.

Answer

Dr. Mitchell Steiner, Chairman, CEO, and President, stated that the next update will be the topline results in January 2025, with no interim announcements planned. He confirmed the safety profile is as expected with no surprises. He also clarified that HOMA-IR was mistakenly included on a presentation slide and is planned for the Phase III study, not the current Phase IIb trial.

Gary Nachman inquired about the drivers of Cortrophin's strong Q2 performance, asking if there were any one-time benefits or seasonality. He also questioned the potential for further sales force expansion, the upside in ophthalmology and gouty arthritis, capital allocation priorities, and the reasoning behind the gross margin guidance not increasing more significantly.

Answer

President, Director & CEO Nikhil Lalwani confirmed the growth was driven by underlying demand, not one-time benefits, citing a doubling in new patient starts. He stated there are no immediate plans to expand the sales force further but will continue to evaluate high-ROI initiatives. SVP & CFO Stephen Carey addressed capital allocation, noting the priority is building cash for future business development. He explained that gross margin guidance remains steady because the benefit from Prucalopride's exclusivity will not be present in the second half of the year.

Gary Nachman asked about the growth drivers for Cortrophin, including the impact of its new prefilled syringe, the effectiveness of new sales reps, and differentiation strategies against competitors. He also inquired about the confidence in the full-year guidance for ILUVIEN and YUTIQ given Q1 challenges, the timeline for commercial strategies to take effect, and the impact of sales force turnover.

Answer

President and CEO Nikhil Lalwani explained that the Cortrophin prefilled syringe has seen positive early feedback and that new reps are gaining traction. He noted the overall ACTH market is growing, allowing room for both competitors, with ANI differentiating through convenience and evidence generation. For ILUVIEN/YUTIQ, he expressed confidence in the guidance due to strong ex-U.S. performance, overcoming seasonality, strengthening the sales force, positive April trends, and exploring new Medicare Part D pathways.

Gary Nachman from Raymond James sought to understand the moving parts within the ILUVIEN and YUTIQ guidance, including potential supply transition issues, and the expected impact from adding the uveitis indication and upcoming clinical data. He also asked for details on the Cortrophin sales force expansion and its ROI.

Answer

Executive Nikhil Lalwani stated the guidance accounts for near-term access issues but is supported by commercial and operational initiatives. He noted that while NEW DAY clinical data is expected in Q2 2025, its potential upside is not factored into the 2025 guidance. Regarding the sales force, he confirmed an addition of 15-20 reps for core Cortrophin indications, citing the company's proven ability to generate high ROI on such commercial investments.

Gary Nachman of Raymond James asked if the implied Q4 revenue for ILUVIEN and YUTIQ is a reasonable run rate for 2025, questioned the strategy to accelerate their growth, and inquired about the potential impact of the NEW DAY study. He also asked about early synergies in ophthalmology and the expected benefit of the Cortrophin prefilled syringe.

Answer

Executive Nikhil Lalwani clarified that the Q4 guidance reflects a transitional period and that ANI expects growth over this run rate in 2025, reiterating the target of $35-$38 million in incremental EBITDA. He noted the deal model does not depend on the NEW DAY study. Christopher Mutz, Head of Rare Disease, added that positive NEW DAY data could encourage earlier use of ILUVIEN. Regarding synergies, Lalwani described early signs from the combined sales force as positive and stated the prefilled syringe is expected to improve patient convenience.

Gary Nachman of Raymond James asked about the 12-month safety data consistency, FDA communications prior to the Day 74 letter, the potential for a priority review, current activities with Omnicom, and updates on the synthetic version of cytisinicline and other partnerships.

Answer

President & CMO Dr. Cindy Jacobs confirmed the long-term safety profile is consistent with previous data and that normal back-and-forth communication with the FDA is ongoing. She noted a priority review has been requested but is not considered a high probability. CCO Jaime Xinos detailed that work with Omnicom is fully operational, focusing on building an AI-enabled launch platform and elevating the medical conversation around nicotine dependence. CEO Rick Stewart added that the focus has been on the NDA, but work on a synthetic version continues and partnership talks can now advance.

Gary Nachman inquired about the release timeline for the full long-term safety data, the average treatment duration in the ORCA-OL study and its real-world implications, key insights from the recent Scientific Advisory Board (SAB) meeting, and the company's pre-commercialization and partnership strategies.

Answer

Dr. Cindy Jacobs, President and Chief Medical Officer, stated the full ORCA-OL data would be compiled after the 120-day safety update in October and presented at a conference late this year or in 2026. She noted the SAB meeting highlighted broad applications for cytisinicline, including in hospital settings. CEO Rick Stewart added that the SAB showed immense enthusiasm for the drug's efficacy and tolerability. On partnerships, he affirmed Achieve can launch alone in the U.S. for nicotine dependence but is defining requirements for potential COPD partners. Jaime Xinos (executive) detailed pre-commercial efforts focused on ensuring drug availability, developing an access and pricing strategy, and raising physician awareness through a digital omnichannel platform.

Gary Nachman inquired about the remaining steps for the Q2 NDA submission for smoking cessation, specifically regarding manufacturing (CMC) requirements and the use of external consultants. He also asked about the commercial access strategy, including the potential for prior authorizations or step-edits, and the use of a specialty pharmacy. Finally, he questioned the progress on securing a partner for comorbid indications or other non-dilutive funding to accelerate the Phase III vaping trial.

Answer

Chief Medical Officer Dr. Cindy Jacobs confirmed the NDA is in its final stages, with summary documents for efficacy and safety being completed and top-tier external consultants assisting in the process. Chief Commercial Officer Jaime Xinos stated the company is considering a 'specialty light' pharmacy to mitigate access barriers and noted that high patient resistance to varenicline could help overcome potential step-edits. CEO Richard A. Stewart added that while partnership discussions for indications like COPD are ongoing, the primary focus is the NDA submission, and the company is prepared to launch independently to maximize shareholder value.

Gary Nachman from Raymond James questioned what a potential adolescent vaping trial might entail, how the company might address the growing nicotine pouch market, and asked for an update on the development of synthetic cytisinicline.

Answer

President and CMO Dr. Cindy Jacobs explained that an adolescent trial would be determined through future discussions with the FDA as part of a pediatric study plan. CEO Rick Stewart stated that while nicotine pouches are on the radar, the focus remains on the smoking cessation NDA and the vaping Phase III trial first. On the synthetic version, he noted it is an ongoing process with progress being made but offered no specific timeline.

Asked about the confidence level for an accelerated approval of dardavapril, the potential commercial opportunity and ramp-up, and whether it can be integrated into the existing oncology sales infrastructure.

Answer

The company is in ongoing conversations with the FDA ahead of the PDUFA date and is excited about the opportunity. Commercially, they expect the product to be used in concentrated academic centers, allowing them to use a small, dedicated group to augment their current footprint. They also highlighted the product's durable patent life into the late 2030s.

Gary Nachman of Raymond James asked about the Ziihera launch in BTC, including the ramp, physician feedback, and market access. He also inquired about potential pricing and promotional changes if the larger GEA indication is approved.

Answer

Renée Galá, President and COO, described the Ziihera launch as having a positive reception but reiterated that revenue would be modest due to the small BTC patient population. She noted no access issues. For a potential GEA launch, she said they would pursue NCCN guideline inclusion post-data and that they are in a good position on pricing, with more details to come upon approval.

Gary Nachman from Raymond James asked for more detail on the 2025 revenue guidance, seeking a breakdown of expected trends for the neuroscience and oncology franchises. He also questioned the 8% decrease in R&D guidance and the company's long-term philosophy on R&D spending levels.

Answer

Philip Johnson, CFO, detailed the revenue outlook, expecting continued growth from Xywav and Epidiolex, normalization of Rylaze sales, and moderating growth from AG royalties. He explained the R&D spending decrease is primarily due to the conclusion of studies for programs like JZP-385 and portfolio prioritization. He clarified that R&D spend is driven by specific investment opportunities, like zanidatamab, rather than being tied to a fixed percentage of revenue.

Gary Nachman of Raymond James requested more detail on the 2025 revenue guidance by franchise and asked for the rationale behind the 8% decrease in R&D spending and the right long-term spending level.

Answer

CFO Phil Johnson provided color on each major product's momentum, noting strong growth expected from Xywav and Epidiolex, normalization of Rylaze, and a moderating drag from Xyrem. For R&D, he explained the decrease is due to the conclusion of certain studies (JZP-385) and portfolio prioritization, and that future spending is driven by investment opportunities like zanidatamab, not a fixed percentage of revenue.

Gary Nachman followed up on oncology, asking about initiatives to expand Rylaze in the AYA market and if peak potential has changed. He also asked if the higher Q3 sales for Defitelio were durable.

Answer

President and COO Renée Galá explained that the Rylaze AYA market build is happening more slowly than anticipated, but they are continuing to educate and pilot new initiatives. For Defitelio, she attributed the quarterly strength to timing and variability, noting it was too early to determine if the trend is durable.

Gary Nachman of Raymond James & Associates, Inc. asked for an update on initiatives to expand Rylaze into the AYA market and questioned whether the strong Q3 performance of Defitelio was sustainable.

Answer

President and COO Renée Galá explained that the Defitelio revenue increase was due to typical quarterly variability and timing, and it is too early to determine if the trend is durable. Regarding Rylaze, she acknowledged that the AYA market build has been slower than anticipated but confirmed they are continuing educational efforts and piloting new programs to drive adoption in that population.

Gary Nachman from Raymond James inquired about the competitive dynamics for SKYRIZI in the IL-23 class against J&J's TREMFYA and asked about the drivers of the strong performance in the neuroscience franchise, including investment levels.

Answer

EVP & CCO Jeffrey Stewart stated there is ample market headroom for all IL-23s in IBD, but SKYRIZI's profile positions it for strong performance. He and CEO Robert Michael confirmed the neuroscience growth is primarily volume-driven and that AbbVie is increasing investment, including sales force expansions, to fuel the franchise's momentum and maintain its leadership position.

Gary Nachman inquired about Scholar Rock's launch readiness for apitegromab, including the speed of launch post-approval, commercial team staffing, and available supply. He also asked about the next steps for SRK-439 if the obesity data is positive.

Answer

COO Keith Woods stated the commercial team will be fully staffed by mid-2025, prepared to launch immediately after approval with ample product supply. CEO David Hallal added that the IND for SRK-439 will be filed in Q3 regardless of the EMBRAZE outcome, preserving optionality for its use in either neuromuscular or cardiometabolic diseases.

Gary Nachman asked how SRK-439 is expected to perform relative to apitegromab in preserving lean mass, given the competitive landscape. He also inquired about the progress of initial payer discussions for apitegromab in SMA and the company's latest thinking on pricing strategy.

Answer

Executive Jay Backstrom described SRK-439 as having a strong profile with greater target affinity, potentially allowing for a low-dose subcutaneous formulation. Chief Commercial Officer Tracey Sacco added that payers are receptive to discussing apitegromab, noting that policies already exist for combination treatments in SMA. She stated they expect access to reflect the value apitegromab brings but did not comment on specific pricing.

Gary Nachman asked several questions regarding the upcoming regulatory filing for apitegromab in SMA, including whether the company would seek approval for both the 10mg and 20mg doses, their confidence in securing a broad label for the full 2-21 age range, and if a pre-NDA meeting is planned. He also inquired about the progress of initial payer discussions and any updates on pricing strategy.

Answer

Executive Jay Backstrom confirmed they will file for the 10mg dose, as both doses showed similar efficacy. He expressed high confidence in obtaining a broad label covering the full age range due to consistent data. He also noted that standard pre-BLA meetings with the FDA are planned. CFO and COO Edward Myles added that while it's early for pricing specifics, ongoing payer discussions are positive, supported by market dynamics where payers already cover expensive sequential SMA therapies, indicating a favorable reimbursement environment.

Gary Nachman of Raymond James inquired about Pacira's efforts to expedite NOPAIN adoption in complex hospital systems, the overall progress of physician and hospital education, and the company's capital allocation priorities—including potential M&A—following the EXPAREL patent litigation settlement.

Answer

CEO Frank Lee and CCO Brendan Teehan confirmed that while adoption in large systems takes time, they are seeing significant growth in community hospitals and ASCs, with learnings being applied to larger IDNs. Regarding capital allocation, CFO Shawn Cross and CEO Frank Lee reiterated a disciplined three-pronged strategy: investing in the base business, advancing the pipeline, and returning capital to shareholders, highlighted by a new $300 million share repurchase authorization. Lee also noted the sales force now has the capacity for additional products.

Gary Nachman asked for a breakdown of the 2025 revenue guidance, specifically the balance between volume growth and gross-to-net pressure from GPO contracts. He also inquired about the potential impact of the new direct-to-consumer (DTC) pilot for EXPAREL. As a follow-up, he questioned the significant step-up in R&D spending and whether the company has enough internal innovation, particularly with the GQ Bio acquisition, or if it still needs to pursue external assets.

Answer

CFO Shawn Cross stated that the 2025 revenue growth guidance should be viewed as 'effectively all volume growth,' as price increases and GPO discounting are expected to offset each other. CEO Frank Lee explained that the targeted DTC pilot is timed to activate patient demand now that reimbursement access has improved and will be ROI-driven. On R&D, Lee positioned the increased spending as part of the company's transition to an innovative biopharma, highlighting the GQ Bio acquisition as a key strategic move that adds a platform, preclinical assets, and talent while eliminating future milestone payments.

Gary Nachman asked whether competitor launches are creating a headwind or helping to expand the overall treatment category. He also inquired about the reimbursement dynamics for AMVUTTRA as a Part B drug compared to stabilizers, which are Part D drugs.

Answer

CEO Yvonne Greenstreet and CCO Tolga Tanguler both framed it as a "category growth story." Tolga Tanguler cited their success in polyneuropathy, where AMVUTTRA (Part B) achieved 70% of new patient starts despite Part D competition. He stated they see no headwinds from the Part B/D dynamic in the CM launch, supported by value-based agreements and the strength of their comprehensive clinical data.

Gary Nachman asked about the competitive dynamics in polyneuropathy and how the Part B versus Part D reimbursement difference is playing out and might translate to the cardiomyopathy market.

Answer

CEO Yvonne Greenstreet highlighted the strong 2024 TTR performance despite competition. Chief Commercial Officer Tolga Tanguler added that the U.S. business grew 42% year-over-year in the first full year of competition, reinforcing the market growth story. He believes AMVUTTRA is highly differentiated and has not seen significant access headwinds, anticipating this dynamic will continue with the CM launch.

Gary Nachman asked about the expected magnitude of SG&A spending for the ATTR-CM launch next year and the company's commercial infrastructure and opportunity in Europe versus the U.S.

Answer

CFO Jeff Poulton anticipated double-digit growth in SG&A for 2025, reflecting increased investment for the launch, with specific guidance to come in February. CCO Tolga Tanguler highlighted their robust international organization, which has already achieved market leadership in polyneuropathy in Europe and Japan, providing a strong foundation for the cardiomyopathy launch.

Denis Reznik, on behalf of Gary Nachman, asked about patient dropout rates in the XPro Phase II trial, the potential timeline and design for a Phase III study, and the company's commercialization strategy for CORDStrom.

Answer

Dr. C.J. Barnum reported that patient dropouts are lower than expected and typical for an elderly population, not indicating drug-related issues. He stated that the Phase III trial design and timeline are contingent on the Phase II results and subsequent FDA discussions. David Moss, CFO, added that while the company aims to be stand-alone, it would likely seek a commercial partner for both CORDStrom and XPro to leverage distribution expertise.

Gary Nachman asked about the patient selection process for the XPro Phase II Alzheimer's trial, the scalability of its biomarker strategy for Phase III, the timeline for the Q2 2025 data readout, and the company's view on the M&A landscape.

Answer

CEO Dr. RJ Tesi and Head of Neuroscience Dr. CJ Barnum responded. Dr. Tesi explained that targeting patients with inflammation (approx. 50% of the population) de-risks the trial, with scalability being a future regulatory discussion. They confirmed the Q2 2025 data timeline is on track. Regarding M&A, Dr. Tesi emphasized that meaningful partnership discussions are contingent on producing positive data, as their approach is novel.

Gary Nachman asked if the company is now leaning towards having an Ad Comm, requested color on the pediatric study data, and inquired about the required sales force size for a solo launch, the nature of potential partnerships, and the timing of any deal.

Answer

CEO Daniel Barber clarified they have no new FDA insight on an Ad Comm but are preparing diligently. Executive Carl Kraus confirmed pediatric data is on track for the filing. Executive Sherry Korczynski estimated a starting sales force of around 100 reps. Mr. Barber reiterated that any U.S. partner must have significant scale and commitment, and he stressed that the company is not operating on a specific deal timeline.

Gary Nachman of Raymond James asked for details on the Anaphylm pediatric study alignment with the FDA, the NDA preparation process, and the status of CMC work. He also inquired about Anaphylm's expected shelf life versus neffy and the company's confidence in the FDA's alignment on the AQST-108 Phase IIa study design.

Answer

SVP, Chief Technology Officer Stephen Wargacki confirmed that Aquestive received supportive feedback on its CMC package, which is a major win. Chief Medical Officer Carl Kraus expressed confidence in aligning with the FDA on the straightforward pediatric study design and noted the AQST-108 study design is based on published data from an approved drug. CEO Daniel Barber added that NDA preparation is ongoing and that the company is focused on developing a robust shelf-life profile that meets patient needs.