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, indicating a significant unmet need [4][9] - 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 [4][12] Business Line Data and Key Metrics Changes - Avexitide is the lead asset, with a focus on treating PBH, and the company is also developing a long-acting formulation and two other candidates, AMX0035 for Wolfram syndrome and AMX0114 for ALS [5][20] - The phase 2B trial of Avexitide showed a 64% reduction in severe hypoglycemic events, which supported the FDA Breakthrough Therapy Designation [16] Market Data and Key Metrics Changes - There is a growing awareness of PBH among endocrinologists, with initiatives for coverage and coding, including a petition to CMS for continuous glucose monitoring (CGM) coverage and a presentation to the CDC for an ICD-10 code [12][38] - The company estimates that the number of patients with PBH will increase, highlighting the urgency for treatment options [9] Company Strategy and Development Direction - The company aims to leverage its experience in rare diseases to establish a robust go-to-market strategy for Avexitide, focusing on understanding the patient journey and ensuring continuity of care [32][50] - There is a strategic emphasis on expanding the indications for Avexitide beyond Roux-en-Y gastric bypass to include other bariatric surgeries and conditions associated with hyperinsulinemic hypoglycemia [21][23] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential impact of Avexitide on patients' lives, emphasizing the low bar for clinical meaningfulness due to the high unmet need in PBH [15][14] - The company is preparing for commercialization while ensuring that the pivotal trial is executed effectively, indicating strong potential for Avexitide in the market [50] Other Important Information - The company is also advancing AMX0114 for ALS, focusing on targeting Calpain-2, a key player in axon degeneration, with ongoing studies to assess safety and tolerability [56][58] - The company is working on AMX0035 for Wolfram syndrome, with plans to initiate a phase three trial in the second half of next year [66] Q&A Session Summary Question: Can you provide context on Avexitide as an opportunity? - The company has been actively seeking to in-license assets in rare diseases and found Avexitide to be a unique opportunity due to its strong pharmacology and significant unmet need in PBH [6][9] Question: What does success look like for the pivotal study? - Any reduction in hypoglycemic events would be considered meaningful, as these events are debilitating for patients [14][15] Question: How does the company plan to address the continuity of care? - The company is mapping out the patient journey and focusing on adult endocrinologists as primary call points for treatment [32][34] Question: What is the significance of the ICD-10 code for PBH? - While an ICD-10 code would be beneficial for claims databases and awareness, it is not essential for diagnosis, as endocrinologists are already trained to recognize PBH [47][48] Question: How does the company view the treatment landscape for ALS? - The company believes that combination therapy will be key in treating ALS, with AMX0114 targeting axon degeneration as a critical component [64]