Shares of Amylyx Pharmaceuticals Inc. (AMLX) touched a new 52-week high of $17.20 yesterday, and that represents a gain of over 50% in less than a month.
This clinical-stage company is currently focused on developing four investigational therapies across several neurodegenerative and endocrine diseases - Avexitide as a treatment for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism; AMX0035 for Wolfram syndrome; AMX0114 for Amyotrophic Lateral Sclerosis (ALS); and AMX0318 for PBH and other rare diseases.
-- Avexitide, the most advanced drug candidate in the pipeline, is under a double-blind, placebo-controlled phase 3 clinical trial in participants with PBH following Roux-en-Y gastric bypass (RYGB) surgery. The study, dubbed LUCIDITY, designed to enrol 75 participants, is expected to complete enrolment this quarter, with topline data anticipated in Q3 2026.
Next up is AMX0035, which, in a phase 2 trial in Wolfram syndrome, dubbed HELIOS, demonstrated improvement or stabilization across measures of glycemic control, visual acuity, and overall symptom burden.
Wolfram syndrome is a rare, progressive neurodegenerative disorder that progressively impacts multiple organs and systems. It is characterized by childhood-onset diabetes mellitus, optic nerve atrophy, and neurodegeneration. About 3,000 people are living in the U.S. with Wolfram syndrome, and there are currently no approved treatment options.
Pending alignment with the FDA, the company plans to initiate a focused, pivotal phase 3 clinical trial of AMX0035 in Wolfram syndrome in the second half of 2026.
AMX0114, being developed for the treatment of Amyotrophic Lateral Sclerosis, is under a phase 1 trial, dubbed LUMINA. The safety and tolerability data from participants enrolled in cohort 1, reported last month, demonstrated that AMX0114 was generally well-tolerated, with no treatment-related serious adverse events. The biomarker data from the first cohort of LUMINA participants are expected in the first half of 2026.
AMX0318, identified through a research collaboration with Gubra A/S, is expected to advance into investigational new drug (IND) enabling studies later this year, with an IND targeted for 2027, pending successful completion of IND-enabling studies.
Cash Position
The company ended September 30, 2025, with cash, cash equivalents, and marketable securities of $344.0 million.
We alerted readers to AMLX on June 25, 2025, when it was at $6.41. Had you invested in AMLX then, you would have made a gain of 168% now in just 7 months.
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