Phase III LUCIDITY Initiation and Enrollment Complete
Completed recruitment for the pivotal Phase III LUCIDITY trial of Avexatide (90 mg daily) in post-bariatric hypoglycemia (PBH); expect to randomize and dose remaining eligible participants this month and deliver top-line data in Q3 2026.
Strong Clinical Efficacy Signal from Prior Trials
Five prior Avexatide trials informed LUCIDITY; Phase 2 data showed a 64% least squares mean reduction versus baseline in composite level 2 and 3 hypoglycemic events (p=0.0031). Phase 2b 90 mg showed ~64–66% effect with median patient event rate of zero.
Regulatory and Commercial Preparations Underway
NDA drafting and regulatory preparations underway to enable rapid submission following positive top-line data; commercial buildout includes medical affairs hires, market research, disease education, and market access planning targeting a potential 2027 launch if approved.
Pipeline Expansion and New Development Candidate
Announced nomination of AMX318 (long-acting GLP-1 receptor antagonist) as a development candidate in January; IND-enabling studies underway with IND targeted for 2027 — supports lifecycle and broader GLP-1 antagonist strategy.
AMX114 Progress in ALS
AMX114 received Fast Track designation for ALS; cohort 1 of Phase 1 LUMINA showed favorable safety/tolerability with no treatment-related serious adverse events, enabling progression to cohort 2 and expected completion of cohort 2 enrollment this month. Biomarker data from cohort 1 planned in H1 2026.
Improved Financial Position and Cash Runway Extended
Ended Q4 with $317 million in cash and marketable securities (down from $344 million at end of Q3), with management stating an anticipated cash runway into 2028 to fund key milestones including LUCIDITY top-line readout and potential NDA/commercial activities.
Quarterly Expense Reductions
Total operating expenses for Q4 were $36.6 million, down 8% year-over-year. R&D was $21.2 million (down from $22.9 million in Q4 2024, ≈7.4% decrease) and SG&A was $15.4 million (down from $17.1 million in Q4 2024, ≈10% decrease). Non-cash stock-based compensation was $6.4 million vs $6.8 million prior quarter (≈5.9% decrease).
Market Opportunity Quantified
Internal analysis and literature triangulation estimate ≈160,000 people living with PBH in the U.S., reinforcing a sizable orphan-market opportunity and informing go-to-market planning.