Financial Data and Key Metrics Changes - The company ended the first quarter with a cash position of $204.1 million, which includes approximately $65.5 million in net proceeds from a public offering closed in January 2025 [23] - Total operating expenses for the quarter were $37.8 million, down 82% from the same period in 2024 [25] - Research and development expenses were $22.1 million compared to $36.6 million in Q1 2024, primarily due to decreased spending on AMX-thirty five and reduced payroll costs [25] - Selling, general and administrative expenses were $15.7 million compared to $57.8 million in Q1 2024, mainly due to decreased payroll and consulting costs [25] Business Line Data and Key Metrics Changes - The company is advancing three potential therapies across four clinical trials, including Avexatide for post bariatric hypoglycemia (PBH), AMX-thirty five for Wolfram syndrome and progressive supranuclear palsy (PSP), and AMX-one hundred fourteen for ALS [7][8] - Avexatide has shown promise in treating PBH, with a pivotal Phase III trial (Lucidity) initiated, and the first participant dosed [10][18] - AMX-thirty five is being evaluated in two indications, with positive Phase II data reported for Wolfram syndrome and ongoing trials for PSP [12][14] Market Data and Key Metrics Changes - There are approximately 160,000 people in the U.S. living with PBH, a condition with no approved treatment options [9][19] - The American Society for Metabolic and Bariatric Surgery reported around 270,000 new bariatric surgeries in 2023, indicating a stable market for potential PBH patients [20] - The company estimates that 8% of patients who undergo bariatric surgery will develop PBH within one to three years [48][50] Company Strategy and Development Direction - The company aims to develop novel therapies for diseases with high unmet needs, focusing on execution across its clinical trials [28] - The company is preparing for a potential commercial launch of Avexatide in 2027 if approved [10] - The company is actively engaging with clinical trial sites and expects to complete recruitment for the Lucidity trial by the end of 2025 [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving clinical milestones through the end of 2026, supported by a strong cash position [23][24] - The company is focused on educating the medical community and patients about PBH, given the lack of awareness and treatment options [34][46] - Management highlighted the importance of access to treatments for patients suffering from debilitating conditions like PBH [65] Other Important Information - The company plans to present week 48 data from the HELIOS trial in Wolfram syndrome at an upcoming conference [12][28] - The company is preparing for potential FDA discussions regarding trial designs for its various programs [56] Q&A Session Summary Question: Will a large patient education campaign be required for Avexatide? - Management acknowledged the need for education due to the rarity of PBH and the lack of prior treatments, emphasizing the importance of informing both medical professionals and patients [32][34] Question: What is the expected efficacy for the PSP trial? - Management stated the study has 80% power to detect a 30% effect on the PSP rating scale, with multiple endpoints being considered for decision-making [37][39] Question: How many of the 160,000 PBH patients are seeking treatment? - Management indicated that a larger group, potentially up to 1 million, may experience hypoglycemia post-bariatric surgery, but only about 160,000 have persistent symptoms warranting treatment [46][47] Question: What are the remaining questions with the FDA regarding trial design? - Management noted that the week 48 data will inform the Phase III program, but specific details of FDA discussions were not disclosed [56] Question: Will there be subgroup analysis in the Avexatide study? - Management confirmed that the study will focus on a population experiencing frequent hypoglycemic events, with no anticipated step therapy restrictions [61][62] Question: What gives confidence that Avexatide won't cause safety issues? - Management highlighted the good safety profile observed in both non-clinical and clinical data, noting that Avexatide is a competitive antagonist rather than a GLP-1 agonist [70] Question: How does Avexatide compare to other drugs in the space? - Management emphasized Avexatide's strong profile and breakthrough therapy designation, noting that other programs are earlier in development and face hurdles [82][84]

Financial Data and Key Metrics Changes - The company achieved $317 million in revenue for Q1 2025, reflecting a 9% increase on a constant currency basis and a 10% increase on an organic basis compared to the prior year [29] - Adjusted operating income rose to $65 million, up from $53 million in Q1 2024, resulting in an adjusted operating income margin of 20% compared to 18% in the prior year [31] - Adjusted diluted earnings per share increased to $0.88 from $0.73 in Q1 2024, driven by operating income growth [32] - Cash balance at March 31 was $738 million, up from $429 million at year-end 2024, primarily due to the reclassification of $295 million of restricted cash [32][33] Business Line Data and Key Metrics Changes - Cardiopulmonary segment revenue was $176 million, a 15% increase year-over-year, with heart-lung machine revenue growing approximately 30% [11] - Epilepsy segment revenue increased by 4%, with U.S. revenue growth at 2% and Europe and rest of world regions combined growth at 13% [14][16] - The company expects cardiopulmonary revenue to grow 9% to 10% for the full year 2025, up from a previous forecast of 7% to 8% [12] Market Data and Key Metrics Changes - The company reported strong demand for oxygenated products, with growth in the high single digits driven by customer demand and pricing [11] - The U.S. epilepsy business faced procedure deferrals due to a voluntary field safety notification, impacting revenue by less than $2 million [15] - The company anticipates low single-digit growth in the U.S. epilepsy market, with Europe and rest of world regions expected to grow in low double digits [16] Company Strategy and Development Direction - The company is focused on expanding its portfolio into areas of high unmet clinical need, particularly in neuromodulation and OSA [52][43] - The company is investing in additional manufacturing capacity to support growth in cardiopulmonary products and anticipates sustaining double-digit growth in heart-lung machines [13][58] - The company is pursuing Medicare coverage for VNS therapy for treatment-resistant depression, which represents a significant growth opportunity [27][43] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to manage tariff impacts, estimating a negligible effect on adjusted operating income for the year [39][40] - The company believes the Italian Supreme Court's decision regarding the SNEA matter effectively removes a long-standing overhang, allowing for greater capital allocation flexibility [37][43] - Management raised the overall organic growth outlook by 100 basis points to between 7% and 8% for 2025, reflecting strong first-quarter performance [19] Other Important Information - The company recorded a liability of $360 million related to the SNEA litigation, which is expected to impact adjusted diluted earnings per share by approximately $0.20 for the remainder of 2025 [36][37] - The company is implementing a tariff mitigation plan that includes a holistic assessment of its supply chain and potential pricing actions [38][39] Q&A Session Summary Question: Can you help us frame where the OSSPREY data puts you on a market competitive basis? - Management highlighted the strength of the clinical data, noting that the trial enrolled more severe patients and achieved a rapid therapeutic response, with 65% of patients responding at twelve months [47][48] Question: What is the outlook for the HLM business and Essence placement rollout? - Management indicated that Essence represented 40% of all HLM placements in 2024 and is expected to reach 60% in 2025, with strong demand and regulatory approvals in new markets like China [58][59] Question: Can you provide more detail on the U.S. Neuromodulation business and updated guidance? - Management noted improved commercial execution in Europe and rest of the world, with a focus on maximizing VNS therapy access in the underpenetrated drug-resistant epilepsy population [64][66] Question: What is the impact of tariffs on guidance? - Management confirmed that the estimated tariff impact on adjusted operating income is less than $5 million, with mitigation strategies potentially offsetting some costs [70][74] Question: How does the company view its leverage ratio and capital allocation flexibility? - Management stated that the leverage ratio is healthy, allowing for continued investment in core businesses and potential inorganic growth opportunities [88][90]

Financial Data and Key Metrics Changes - Total revenue for Q1 2025 was $149.8 million, compared to $143.6 million in Q1 2024, reflecting an increase driven by core products [14] - Total revenues excluding Trokendi XR and Oxtellar XR increased by 26% year-over-year [6][15] - GAAP net loss for Q1 2025 was $11.8 million, or loss per diluted share of $0.21, compared to a GAAP net earnings of $124,000 in the prior year [16] - Adjusted operating earnings for Q1 2025 were $25.9 million, up from $22.3 million in Q1 2024 [17] - Cash and cash equivalents as of March 31, 2025, were approximately $463.6 million, an increase from $453.6 million at the end of 2024 [17] Business Line Data and Key Metrics Changes - KELBRE saw a 22% increase in prescriptions and a 44% increase in net sales, with March 2025 prescriptions reaching an all-time high of 75,277 [7] - GOCOVRI prescriptions increased by 12% and net sales increased by 16% compared to the same quarter last year [8] - Combined net sales of Trokendi XR and Oxtellar XR were down 46% in Q1 2025 [10] Market Data and Key Metrics Changes - The Medicare Inflation Reduction Act led to a 42% decline in GOCOVRI's Medicare co-pay, resulting in increased prescriptions among Medicare patients [8] - 84% of GOCOVRI's Medicare prescriptions cost patients less than $25 by March 2025 [8] Company Strategy and Development Direction - The company aims to drive growth from core products while investing in its pipeline and launching new products like NAPCO [9][57] - Corporate development remains a top priority to strengthen future growth through strategic opportunities [11][57] - The company plans to initiate a follow-on phase 2b trial for SPN-eight twenty in adults with major depressive disorder [10] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the growth trajectory of KELBRE, emphasizing that growth will be driven more by volume than price [21] - The company is confident in its ability to navigate the current environment, including potential tariff impacts, as most products are shielded from tariffs [12] - Management reiterated guidance for total revenues in 2025 to range from $600 million to $630 million [18] Other Important Information - The company is focused on enhancing its infrastructure for new product launches, ensuring a smooth transition from patient enrollment to product delivery [30] - The company has established a strong support system for patients and physicians, which is seen as a competitive advantage [45] Q&A Session Summary Question: Key growth drivers for KELBRE in 2025 - Growth will be driven by both volume and a small price increase, with a strong prescription growth observed in Q1 [21] Question: Timing for data readout from the study on SPN-eight twenty - The study is expected to start before year-end 2025, with data potentially available in about 18 months [25] Question: Impact of Q1 dynamics on KELBRE net pricing - Q1 typically sees pressure on gross to net pricing, but improvements are expected in subsequent quarters [29] Question: Reimbursement discussions for ONAPCO - The company expects a high percentage of enrollment forms to be fulfilled, with a strong support system in place for reimbursement [34] Question: Competitive dynamics regarding ONAPCO - Initial feedback from physicians has been positive, and the product is seen as a strong alternative to existing therapies [45] Question: Potential indications for SBN-eight twenty - The company is considering multiple indications, including ADHD and sleep-wake disorders, but has not finalized the lead indication yet [48]

Financial Data and Key Metrics Changes - Total product revenues for Q1 2025 were $121.5 million, representing a year-over-year growth of 62% compared to $75 million in Q1 2024 [18][21] - Net loss for Q1 2025 was $59.4 million or $1.22 per share, an improvement from a net loss of $68.4 million or $1.44 per share in Q1 2024 [21] - Cash and cash equivalents at the end of Q1 2025 were $300.9 million, down from $315.4 million at year-end [21] Business Line Data and Key Metrics Changes - Avelity net product sales were $96.2 million for Q1 2025, representing 80% year-over-year growth from $53.4 million in Q1 2024 [18] - Sunosi net product revenues were $25.2 million for Q1 2025, a 17% year-over-year growth from $21.6 million in Q1 2024 [18] Market Data and Key Metrics Changes - Avelity led the market in TRx growth in Q1 with approximately 167,000 prescriptions, reflecting 76% year-over-year growth [23] - Sunosi total prescriptions were over 46,000, representing a 12% growth versus Q1 2024 [25] Company Strategy and Development Direction - The company aims to advance three novel NDA stage product candidates: AXS-fourteen for fibromyalgia, AXS-five for Alzheimer's disease agitation, and AXS-twelve for narcolepsy [8][12] - The company is focused on executing multiple Phase III clinical programs to broaden the potential of current products and candidates [8][14] - The company anticipates a strong position with potentially five marketed products across six indications by 2026 [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to navigate trade policy developments, stating that any potential impact would be immaterial [15] - The company expects 2025 to be a catalyst-rich year with significant updates on product launches and clinical trials [17] Other Important Information - The company is preparing for the launch of Cymbravo, with positive feedback from healthcare providers regarding its clinical profile [26] - The company is actively negotiating to expand payer coverage for Avelity and Sunosi [68] Q&A Session Summary Question: FDA interactions regarding developmental agents - Management indicated that dialogue with the FDA remains status quo and no changes have been noted regarding review teams or agency responsiveness [32] Question: DTC advertising for Avelity - A national campaign is expected to launch later this year [35] Question: Coverage evolution for Avelity - Management is focused on increasing covered lives and reducing utilization management [68] Question: Expected cadence of coverage for Cymbravo - Management stated it is premature to specify the cadence but is focused on securing access quickly [48] Question: Sales opportunity for AXS-fourteen in fibromyalgia - Management is enthusiastic about the potential impact, given the large market size and lack of recent therapeutic innovation [62] Question: Profitability expectations for 2025 - Management remains confident in achieving cash flow positivity but has not provided specific guidance on profitability [94]

Core Viewpoint - Abivax has completed enrollment for the Phase 3 ABTECT trials in patients with moderately to severely active ulcerative colitis, marking a significant milestone for the company and the patient community [2][3]. Group 1: Trial Enrollment and Characteristics - A total of 1,275 participants were successfully enrolled in the Phase 3 ABTECT trials, exceeding the target enrollment of 1,224 by 4% [6]. - The blinded baseline characteristics of participants in the Phase 3 ABTECT trials closely align with those observed in the Phase 2b trial, reinforcing confidence in the drug's potential efficacy and safety [3][9]. - Key baseline characteristics include: - 65% of participants had a baseline MMS score of 7-9 in Phase 3 compared to 71% in Phase 2b - 48% had prior advanced therapy in both trials - 42% of Phase 3 participants had previously failed corticosteroid treatment, compared to 52% in Phase 2b [3][6]. Group 2: Upcoming Milestones - Top-line results from the 8-week induction trials are anticipated in Q3 2025, with 44-week maintenance data expected in Q2 2026 [6][9]. - If the results confirm the safety and efficacy of obefazimod, a New Drug Application (NDA) submission to the FDA is planned for the second half of 2026 [3][9]. Group 3: Company Overview - Abivax is a clinical-stage biotechnology company focused on developing therapeutics that stabilize the immune response in patients with chronic inflammatory diseases [7]. - The lead drug candidate, obefazimod (ABX464), is currently in Phase 3 clinical trials for the treatment of moderately to severely active ulcerative colitis [7].

Financial Performance - Total Q3 revenues reached $1.1 billion[7] - Net product sales hit a record $267 million, a 60% increase over 3Q19[7, 14] - Collaboration & license agreement revenues surged to $758.3 million, primarily due to $725 million in upfront payments from Merck[22] - ADCETRIS U S and Canada net product sales guidance is $650 to $660 million[27] - Royalty revenues guidance increased to $125 to $130 million[27] Product Updates - PADCEV and TUKYSA contributed to net product sales growth[22] - PADCEV data supports global registrations and expanded U S indication[8] - TUKYSA is expanding capabilities to support launch in Europe, with MAA under review[8] - Tisotumab vedotin BLA submission planned under FDA accelerated approval pathway[8] Clinical Trial Data - EV-301 trial showed a hazard ratio of 0.70 (P=0.001) for overall survival and 0.61 (P<0.00001) for progression-free survival with PADCEV[34] - EV-201 Cohort 2 trial showed a 52% objective response rate with PADCEV[34] - Tisotumab Vedotin showed a confirmed ORR of 24%[42] Strategic Collaborations - Received $1.8 billion under Merck collaborations, including upfront payments and prepaid R&D in Q3, and a $1 billion equity investment closed in Q4[7] - Granted Merck exclusive license to commercialize TUKYSA outside U S, Canada, and Europe[8] - Signed worldwide co-development and commercialization agreement with Merck for ladiratuzumab vedotin[8]

Financial Data and Key Metrics Changes - The company ended 2024 with $251 million in cash and cash equivalents, expected to fund operations into the second half of 2026 [24] - ZYNLONTA net product revenues for Q4 2024 were $16.4 million, slightly down from $16.6 million in Q4 2023, while full-year revenues were $69.3 million compared to $69.1 million in 2023 [25] - The net loss for Q4 2024 was $30.7 million, a significant improvement from a net loss of $85 million in Q4 2023 [26] - For the full year, the net loss was $157.8 million, down from $240.1 million in 2023 [26] - Non-GAAP operating expenses decreased by 15% year-over-year in Q4 2024, contributing to the reduced net loss [25][26] Business Line Data and Key Metrics Changes - ZYNLONTA maintained its position in the third-line-plus DLBCL market, achieving commercial brand profitability [9] - The company reported an overall response rate of 80% and a complete response rate of 50% in the LOTIS-5 trial, indicating strong efficacy [13] - Initial data from LOTIS-7 showed a best overall response rate of 94% and a complete response rate of 72% among relapsed or refractory DLBCL patients [14] Market Data and Key Metrics Changes - The company anticipates a peak revenue opportunity of $600 million to $1 billion in the US for ZYNLONTA, assuming regulatory approval and compendia listing [19][21] - The potential market opportunity for indolent lymphomas is estimated to be between $100 million and $200 million [22] Company Strategy and Development Direction - The company aims to expand the use of ZYNLONTA into earlier lines of DLBCL therapy through ongoing trials [13] - The strategy includes pursuing research collaborations to advance early-stage solid tumor pipelines [29] - The focus remains on hematology, with a disciplined capital allocation strategy to create value [28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's path forward, highlighting the importance of upcoming data readouts in 2025 [30] - The competitive landscape is expected to evolve with the introduction of new therapies, but management believes ZYNLONTA will maintain its market position [76] Other Important Information - The company achieved a double-digit reduction in operating expenses for the second consecutive year [12] - Management emphasized the importance of regulatory strategies and compendia listings for future growth [50] Q&A Session Summary Question: Update on LOTIS-7 - The company has not disclosed the specific forum or timing for sharing LOTIS-7 data but is on track to enroll 40 patients in Q2 [42] Question: Impact of ADCETRIS Approval - Management believes the approval of ADCETRIS will have limited impact on ZYNLONTA's market share, as it is likely to replace older regimens rather than significantly affect current usage [44] Question: Regulatory Meetings for LOTIS-7 - Discussions with regulatory authorities are planned for the second half of the year once sufficient data is available [50] Question: Market Opportunity in Indolent Lymphoma - The peak opportunity for indolent lymphomas is estimated to be between $100 million and $200 million [51] Question: LOTIS-5 and LOTIS-7 Strategy - Both LOTIS-5 and LOTIS-7 are seen as complementary approaches to address different patient needs in the DLBCL market [56] Question: Importance of Upcoming Presentations - The upcoming presentations at AACR are expected to provide differentiating data on the company's ADC platforms [60] Question: Competitive Landscape for DLBCL - The introduction of bispecifics has taken about a third of the third-line-plus market, but ZYNLONTA has maintained consistent sales [75] Question: Compendia Listing and Market Penetration - Peak penetration for ZYNLONTA is typically achieved within the first two years post-approval or listing [86]

Financial Data and Key Metrics Changes - The company ended 2024 with cash totaling $3.7 million, down from $7.5 million as of December 31, 2023 [25] - Research and development expenses for Q4 2024 were $0.8 million, a decrease of $1.1 million from $1.9 million in Q4 2023 [25] - General and administrative expenses for Q4 2024 were $2 million, down from $3.2 million in Q4 2023, primarily due to decreases in professional fees and share-based compensation [27] - The net loss for Q4 2024 was $2.8 million or $0.16 per diluted share, compared to a net loss of $5.1 million or $0.37 per diluted share in Q4 2023 [28] Business Line Data and Key Metrics Changes - The company is advancing its lead antibiotic candidate, ibezapolstat (IBZ), into international Phase 3 clinical trials for the treatment of C. difficile infection [8] - In Phase 2 trials, ibezapolstat demonstrated a clinical cure rate of 96%, with 100% of patients remaining cured one month after treatment [9] Market Data and Key Metrics Changes - The company raised a total of $6.6 million in gross proceeds under its ATM financing program for the year ended December 31, 2024 [25] - The FDA has granted ibezapolstat QIDP and Fast Track designations for the treatment of C. difficile infection, indicating a favorable regulatory environment [22] Company Strategy and Development Direction - The company is well-positioned to commence its international Phase 3 registration program following positive regulatory guidance from both the FDA and EMA [13] - Acurx aims to differentiate ibezapolstat from other antibiotics by demonstrating its microbiome-restorative potential and lower recurrence rates [14][18] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential economic impact of ibezapolstat, which could reduce the annual cost burden of C. difficile infection in the US, estimated at $5 billion [23] - The company is actively pursuing funding opportunities for its Phase 3 clinical trial program, including partnerships and grants [42] Other Important Information - The Japanese Patent Office granted a new patent for the company's DNA polymerase IIIC inhibitors, expiring in December 2039 [19] - The company closed a registered direct offering and concurrent private placement, raising gross proceeds of $1.1 million [20] Q&A Session Summary Question: Differences and similarities between FDA and EMA for Phase 3 program - Management confirmed that the protocols for the Phase 3 program are identical for both FDA and EMA, as they aligned their strategies before approaching the EMA [35] Question: Geographic enrollment for Phase 3 program - The plan is to start with 150 clinical trial sites, with approximately 50% in Europe and the remainder in the US, Canada, and South America [37] Question: Upcoming data releases for ibezapolstat - A full set of Phase 2 data is expected to be published in a prestigious scientific journal within the next 30 days [40] Question: Funding options for development of ACX-375C - Management is exploring various funding opportunities, including partnerships and government grants, to support the Phase 3 trial [42] Question: Partnership environment and timeline for Phase 3 trials - The current tumultuous market conditions have made securing partnerships challenging, but management is optimistic about starting Phase 3 trials in 2025 [56] Question: Timeline for top-line data from Phase 3 trials - Management indicated that it would take approximately two years from the first patient enrolled to obtain top-line data [62] Question: Status of ATM program and NASDAQ listing - The ATM program was suspended in January but can be reactivated when management decides. There is confidence in maintaining the NASDAQ listing despite current share price challenges [70][72]

Financial Data and Key Metrics Changes - The company ended 2024 with cash totaling $3.7 million, down from $7.5 million as of December 31, 2023 [25] - Research and development expenses for Q4 2024 were $0.8 million, a decrease of $1.1 million from $1.9 million in Q4 2023 [25][26] - General and administrative expenses for Q4 2024 were $2 million, down from $3.2 million in Q4 2023, primarily due to decreases in professional fees and share-based compensation [27] - The company reported a net loss of $2.8 million or $0.16 per diluted share for Q4 2024, compared to a net loss of $5.1 million or $0.37 per diluted share for Q4 2023 [28] Business Line Data and Key Metrics Changes - The company is advancing its lead antibiotic candidate, ibezapolstat (IBZ), into international Phase 3 clinical trials for the treatment of C. difficile infection [8] - In Phase 2 trials, ibezapolstat demonstrated a clinical cure rate of 96%, with 100% of patients remaining cured one month after treatment [9][10] Market Data and Key Metrics Changes - The company raised a total of $6.6 million in gross proceeds under its ATM financing program for the year ended December 31, 2024 [25] - The FDA has granted ibezapolstat QIDP and Fast Track designations for the treatment of C. difficile infection, indicating a favorable regulatory environment [22] Company Strategy and Development Direction - The company is well-positioned to commence its international Phase 3 registration program following positive regulatory guidance from both the EMA and FDA [13] - The company aims to differentiate ibezapolstat from other antibiotics by demonstrating its microbiome-restorative potential and lower recurrence rates [14][18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential for partnerships and funding opportunities to support the Phase 3 trial, emphasizing the importance of government and quasi-government partnerships [42][43] - The company believes that ibezapolstat could significantly reduce the annual cost burden of C. difficile infections in the US, which is estimated at $5 billion [23] Other Important Information - The Japanese Patent Office granted a new patent for the company's DNA polymerase IIIC inhibitors, expiring in December 2039 [19] - The company closed a registered direct offering and concurrent private placement, raising gross proceeds of $1.1 million [20] Q&A Session Summary Question: Differences and similarities between FDA and EMA for Phase 3 program - Management confirmed that the protocols are identical and there is complete agreement from both agencies [35] Question: Geographic enrollment for Phase 3 program - The plan is to have 150 clinical trial sites, with half in Europe and the other half in the US, Canada, and South America [37] Question: Upcoming data releases for ibezapolstat - A full set of Phase 2 data is expected to be published in a prestigious scientific journal within the next 30 days [40] Question: Funding options for development of ACX-375C - Management is exploring partnerships and grants from government bodies as primary funding sources [42] Question: Partnership environment and timeline for Phase 3 trials - Management noted challenges in securing partnerships but remains optimistic about potential collaborations in 2025 [56] Question: Timeline for top-line data from Phase 3 trials - It is expected to take two years from the first patient enrolled to obtain top-line data [62] Question: Status of the ATM program and NASDAQ listing - The ATM program was suspended but can be reactivated, and management is confident in maintaining the NASDAQ listing [70][72]

Core Insights - Lexicon Pharmaceuticals reported positive topline results from the Phase 2b PROGRESS study of pilavapadin, a novel non-opioid therapy for diabetic peripheral neuropathic pain (DPNP), indicating a meaningful pain reduction at the 10 mg dose, which is set to advance into Phase 3 development in 2025 [3][7] - The company is focusing on its R&D pipeline, with significant progress on three key programs: pilavapadin for DPNP, LX9851 for obesity, and sotagliflozin for hypertrophic cardiomyopathy (HCM) [3][4] - Financial results for Q4 2024 showed a substantial increase in revenues, driven by sales of INPEFA and a $25 million upfront payment from a licensing agreement [11][12] Group 1: Clinical Development - Pilavapadin (LX9211) demonstrated meaningful pain reduction and was well-tolerated in the PROGRESS Phase 2b study, supporting its advancement to Phase 3 trials [3][7] - LX9851 is progressing towards an IND filing in 2025, targeting obesity and related metabolic disorders [5] - Enrollment is ongoing for the SONATA Phase 3 study of sotagliflozin in HCM, with a target of 500 patients [8] Group 2: Financial Performance - Q4 2024 revenues increased to $26.6 million from $0.7 million in Q4 2023, and full-year revenues rose to $31.1 million from $1.2 million in 2023, reflecting increased sales of INPEFA and the licensing agreement [11] - R&D expenses for Q4 2024 rose to $26.7 million from $14.8 million in Q4 2023, primarily due to investments in clinical trials [12] - The net loss for Q4 2024 was $33.8 million, an improvement from a net loss of $49.8 million in Q4 2023 [14] Group 3: Strategic Focus - The company is strategically repositioning to prioritize R&D efforts, aiming to address significant unmet medical needs in its pipeline [3][4] - Lexicon's focus on generating clinical data for sotagliflozin aims to differentiate it in the market, particularly in reducing cardiovascular risks [18] - The company has a strong cash position of $238 million as of December 31, 2024, up from $170 million a year earlier, providing a solid foundation for ongoing development [15]