Amylyx Posts Positive Phase 3 Avexitide Results

The Tip Desk · November 7, 2026 · 3 min read

Cash, cash equivalents and marketable securities stood at $768.1 million after an August offering that raised about $544.6 million in net proceeds.

Amylyx Pharmaceuticals, Inc. (AMLX) reported positive topline results from the Phase 3 LUCIDITY trial of avexitide in post-bariatric hypoglycemia, a readout the company had expected in late August or early September.

The Cambridge, Massachusetts, biopharmaceutical company said LUCIDITY met its prespecified primary endpoint, showing a 55% reduction in the composite rate of Level 2 and Level 3 hypoglycemic events versus placebo (p=0.000003), and met all secondary endpoints. Avexitide was generally well-tolerated through the double-blind period, the company said.

Co-CEOs Joshua Cohen and Justin Klee called the quarter a transformational period, saying the data bring the company one step closer to potentially delivering a first-ever FDA-approved treatment for post-bariatric hypoglycemia. The company said it remains on track to submit a New Drug Application by the end of 2026 and to launch avexitide in 2027, if approved. The FDA has granted avexitide Breakthrough Therapy Designation for the condition.

LUCIDITY enrolled 78 participants at 21 U.S. sites in a 16-week randomized, double-blind, placebo-controlled design, with participants randomized 3:2 to 90 mg of avexitide once daily or placebo. A 32-week open-label extension and an Expanded Access Program remain ongoing.

Results from the trial were accepted as a late-breaking oral presentation at ObesityWeek 2026 on November 15.

Research and development expenses for the three months ended September 30, 2026 were $28.4 million, compared with $19.9 million a year earlier. The company said the increase was primarily due to spending on the pivotal LUCIDITY trial and other avexitide-related costs, partially offset by lower spending on AMX0035 after its discontinuation for progressive supranuclear palsy.

Selling, general, and administrative expenses were $21.3 million, compared with $16.2 million a year earlier, which the company attributed primarily to personnel costs supporting pre-commercialization activities and the vesting of PSU awards.

Net loss for the quarter was $45.5 million, or $0.38 a share, compared with $34.4 million, or $0.37 a share, a year earlier.

Cash, cash equivalents and marketable securities were $768.1 million at September 30, 2026, compared with $250.8 million at June 30, 2026. The company said the position reflects net proceeds of approximately $544.6 million from an underwritten public offering that closed in August 2026. It said the proceeds strengthen its financial position to enter 2027 well-funded to commercialize avexitide, if approved.

During the third quarter the company said it solidified its full commercial leadership team and continued hiring across marketing, market access, patient services, and commercial operations. Its uncoverPBH.com education platform remains active.

In ALS, Amylyx completed enrollment of Cohort 3 (n=12) of the Phase 1 LUMINA trial of AMX0114, an antisense oligonucleotide targeting calpain-2 with FDA Fast Track designation, and began enrollment of Cohort 4. In the prior quarter, Cohorts 1 and 2 were fully enrolled and Cohort 3 was still enrolling. The company presented LUMINA data from the first two dose cohorts at the Annual NEALS Meeting.

IND-enabling studies for AMX0318, a novel GLP-1 receptor antagonist for long-acting administration intended for PBH and other rare diseases, were underway, with an IND filing targeted for 2027.