Company Overview - Amylyx is presenting at the Citi Global Healthcare Conference, highlighting its lead asset, Avexitide, which is in a pivotal study for post-bariatric hypoglycemia (PBH) [3][4] - The company anticipates completion of enrollment for the study in the first quarter and expects top-line results in the third quarter of the year [4] Market Opportunity - PBH affects approximately 160,000 individuals in the U.S., with no approved treatments currently available, indicating a significant unmet medical need [4] - The company is planning for commercialization of Avexitide in 2027 following the study results [4] Future Developments - Amylyx is also exploring a potential long-acting formulation of Avexitide, which would be a daily subcutaneous treatment [4]

Amylyx Pharmaceuticals (NasdaqGS:AMLX) FY 2025 Conference December 03, 2025 04:00 PM ET Company ParticipantsJustin Klee - Co-CEo and Co-FounderConference Call ParticipantsGeoff Meacham - Senior AnalystGeoff MeachamAll right. Okay, welcome to the Citi Global Healthcare Conference. I'm Jeff Meacham.Global Head of Healthcare.Global Head of Healthcare, thank you. And Senior Analyst, big cap and mid-cap biotech and pharma. Obviously, Jarvis Fang is up here with me from my team. We're thrilled to have Amylyx. So ...

Financial Data and Key Metrics Changes - The company is excited about the upcoming pivotal study for Avexitide, targeting post-bariatric hypoglycemia (PBH), with an estimated 160,000 patients in the U.S. and no approved treatments [2][6][12] - Enrollment for the pivotal study is expected to complete in Q1 2026, with top-line results anticipated in Q3 2026, leading to potential commercialization in 2027 [2][3] Business Line Data and Key Metrics Changes - Avexitide is positioned as a competitive inhibitor of the GLP-1 receptor, with significant unmet needs in PBH and strong prior trial data supporting its efficacy [5][6] - The company is also developing a long-acting formulation of Avexitide, which is expected to enhance market appeal [3][21] Market Data and Key Metrics Changes - There is a growing awareness of PBH among endocrinologists, with initiatives for coverage and coding underway, including a petition for an ICD-10 code [9][36] - The company has identified a clear patient journey for PBH, which aids in diagnosis and treatment planning [34][41] Company Strategy and Development Direction - The company aims to leverage its experience in rare diseases to establish a robust commercialization strategy for Avexitide, focusing on patient connectivity and care pathways [30][32] - Future plans include exploring additional indications for Avexitide beyond Roux-en-Y gastric bypass patients, potentially expanding to sleeve gastrectomy and other surgeries [18][19] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming pivotal trial results and the potential impact of Avexitide on patient quality of life [12][14] - The company is preparing for a chronic treatment model, anticipating ongoing patient management needs [31][32] Other Important Information - The company is also advancing its ALS candidate AMX0114, with ongoing studies to assess its safety and efficacy [53][56] - AMX0035 for Wolfram syndrome is in the pipeline, with plans for a phase three trial pending FDA alignment [65][66] Q&A Session Summary Question: What is the expected timeline for Avexitide's pivotal study results? - The company anticipates completion of enrollment in Q1 2026 and top-line results in Q3 2026, with commercialization targeted for 2027 [2][3] Question: How does the company plan to raise awareness about PBH? - The company is working with the American Society of Metabolic and Bariatric Surgery to develop educational materials and increase awareness among healthcare providers [40][41] Question: What is the significance of the potential ICD-10 code for PBH? - While the ICD-10 code is seen as beneficial for claims databases and awareness, it is not deemed essential for diagnosis, as endocrinologists are already trained to recognize PBH [46][47]

Financial Data and Key Metrics Changes - The company ended Q3 2025 with a strong cash position of $344 million, compared to $181 million at the end of Q2 2025, reflecting a public offering that provided approximately $191 million in net proceeds [12] - Total operating expenses for the quarter were $36 million, down 53% from the same period in 2024, primarily due to one-time expenses related to the acquisition of Avexitide incurred in Q3 2024 [12][13] - Research and development expenses decreased to $19.9 million from $21.2 million in Q3 2024, mainly due to reduced spending on AMX0035 for PSP and ALS [13] - Selling, general, and administrative expenses were $16.2 million, down from $17.8 million in Q3 2024, attributed to decreased consulting and professional services expenses [13] Business Line Data and Key Metrics Changes - The lead program, Avexitide, is focused on post-bariatric hypoglycemia (PBH) and has shown statistically significant reductions in hypoglycemic events across five clinical trials [4][8] - The pivotal phase 3 Lucidity Trial is designed to replicate previous trial results, with a focus on enrolling a similar patient population and collecting data consistently [4][9] - The company anticipates completing recruitment for the Lucidity Trial in Q1 2026, with top-line data expected in Q3 2026 [5][12] Market Data and Key Metrics Changes - The company estimates approximately 160,000 individuals in the U.S. suffer from PBH, highlighting a significant unmet need for treatment options [6][17] - The ongoing market research and claims analysis support the understanding of the burden of PBH and the urgent need for an FDA-approved treatment [6][17] Company Strategy and Development Direction - The company is preparing for a potential launch of Avexitide in 2027, pending FDA approval, and is building its medical affairs and commercial organizations [5][6] - The company is also advancing the clinical development of AMX0035 for Wolfram syndrome, with plans to initiate a pivotal phase 3 trial in the second half of 2026 [6][55] - The research collaboration with Gubra is progressing well, with new molecules demonstrating strong potency and extended half-lives [16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution of the Lucidity Trial and the potential of Avexitide to be the first approved therapy for PBH [10][18] - The company remains focused on addressing the significant unmet need in the PBH market, with ongoing engagement from clinical trial sites and high participant interest [9][51] - Management acknowledged the steady enrollment rate in the Lucidity Trial and emphasized the importance of quality in participant selection and data collection [22][51] Other Important Information - The company is preparing for early NDA submission efforts for Avexitide, with a focus on market research and disease education [5][6] - The anticipated cash runway extends into 2028, supporting the company's strategic initiatives and potential product launch [12] Q&A Session Summary Question: Enrollment update and operational dynamics affecting the study - Management acknowledged the importance of site startup and participant commitment to dietary restrictions, noting a steady enrollment rate and the focus on quality [20][22] Question: Phase 3 study patient history and screen failure rates - Management indicated that most patients would have been on dietary therapy for several years prior to entering the study, but did not disclose specific screen failure rates [27][28] Question: Addressable market for PBH and patient eligibility - Management reiterated the estimate of 160,000 PBH patients, emphasizing that this figure includes those not controlled on diet and experiencing significant hypoglycemic events [31][36] Question: Differences in site enrollment rates and engagement - Management confirmed that differences in enrollment rates across sites are common, but overall engagement from sites remains high [51] Question: Manufacturing and CMC processes for Avexitide - Management stated that registration batches have been manufactured and are undergoing stability testing, with a focus on quality and inspection readiness [49] Question: Measuring diet adherence in the Lucidity trial - Management confirmed that diet adherence is monitored through blinded continuous glucose monitoring (CGM) data, with interventions possible if significant deviations are observed [44] Question: Future regulatory path for Avexitide in other GI surgeries - Management indicated that while the current phase 3 study focuses on Roux-en-Y gastric bypass, there is interest in exploring other surgical types in the future [46]

Summary of Amylyx Pharmaceuticals (AMLX) Conference Call - September 02, 2025 Company Overview - **Company**: Amylyx Pharmaceuticals (AMLX) - **Focus**: Development of therapies for post-bariatric hypoglycemia (PBH) and Wolfram syndrome Key Points on PBH and Avexitide - **Avexitide**: A GLP-1 receptor antagonist currently in Phase 3 trials, aimed at reducing hypoglycemic events in PBH patients [1][2] - **Market Opportunity**: Approximately 160,000 individuals in the U.S. suffer from PBH, a rare complication following bariatric surgery, with an estimated 8% of bariatric surgery patients developing PBH [5][6] - **Unmet Need**: Current treatments for PBH are limited, primarily involving medical nutrition therapy, which is often ineffective and burdensome [9][10] - **Patient Impact**: PBH significantly affects daily life, causing anxiety and limiting activities due to the risk of severe hypoglycemic events [3][8] - **Clinical Trials**: Recruitment for the Phase 3 LUCIDITY study is on track to complete by year-end 2025, with data expected in the first half of 2026 [11][12] Competitive Landscape - **Current Treatments**: Limited options exist, with off-label therapies showing minimal efficacy and significant side effects [10] - **Differentiation**: Avexitide is positioned as a unique treatment with a strong competitive profile, addressing a significant unmet need in the PBH market [10][21] Insights on Patient Population - **Demographics**: Predominantly affects women in their 40s, often misdiagnosed due to overlapping symptoms with menopause [24][26] - **Awareness and Diagnosis**: Increased awareness among endocrinologists and advocacy groups is leading to better identification of PBH cases [36][37] Wolfram Syndrome Insights - **AMX0035**: A combination therapy for Wolfram syndrome, showing promise in initial studies with stabilization or improvement in diabetic outcomes [27][30] - **Patient Community**: Strong advocacy from families, particularly mothers, is driving awareness and support for research in Wolfram syndrome [35] Future Directions - **Long-Acting Formulations**: Collaboration with Gubra aims to develop a long-acting GLP-1 receptor inhibitor, enhancing treatment options for PBH and potentially other conditions [39][41] - **Regulatory Engagement**: Ongoing discussions with regulatory bodies to streamline the path for pivotal studies in rare diseases like Wolfram syndrome [31][32] Conclusion - **Overall Outlook**: Amylyx Pharmaceuticals is positioned to address significant unmet needs in both PBH and Wolfram syndrome, with promising clinical data and a strong focus on patient impact and education [23][27]