Pivotal Phase III LUCIDITY Enrollment Complete and On Track
Randomized and dosed the last participant in late March; clear line of sight to complete the 16-week trial with top-line readout expected in Q3 2026. Study is powered at 90% and was designed to replicate prior positive trials (prior trials showed ~55% and 64% reductions in hypoglycemic events).
Regulatory Momentum and NDA Preparation
Avexitide has FDA Breakthrough Therapy designation; the company is actively drafting NDA sections now to accelerate a potential submission following top-line data, aiming to shorten typical post-Phase III timelines.
Commercial Launch Readiness and Market Opportunity
Active commercial preparations including hires across marketing, market access and commercial operations, and a disease-state education campaign planned for this summer. Independent claims analysis and field engagements support an estimated U.S. PBH population of ~160,000 people who underwent common bariatric surgeries. Company is preparing for potential commercialization in 2027 if approved.
Expanded Access Program (EAP) Launched
U.S. expanded access program launched to provide avexitide for up to 250 adults with PBH (initially eligible: participants who completed LUCIDITY or prior avexitide trials), addressing urgent unmet need.
Progress Across Broader Pipeline
AMX0318: IND-enabling studies underway with a target 2027 IND filing and a $4.0M milestone recognized related to its selection as a development candidate. AMX0035: week-96 data from Phase II HELIOS in Wolfram syndrome expected to be presented at an upcoming meeting. AMX0114 (ALS): Cohort 2 of Phase I LUMINA fully enrolled; early biomarker data from Cohort 1 to be presented at ENCALS in June.
Balance Sheet and Runway Supporting Key Milestones
Cash and marketable securities of $279.8M at quarter end and management states this funds anticipated cash runway into 2028, covering key expected milestones including LUCIDITY top-line readout (Q3 2026) and potential FDA approval and commercial launch of avexitide in 2027.