12 Best Biotech Penny Stocks To Invest In Now

5. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX)

Number of Hedge Fund Investors: 26

Stock Price as of December 26: $4.00

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is a biotechnology company dedicated to developing innovative treatments for patients with neurodegenerative diseases and endocrine conditions characterized by high unmet medical needs. The company has built a robust pipeline of promising therapies, including avexitide, AMX0035, and AMX0114.

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is focusing on the advancement of avexitide, a GLP-1 receptor antagonist with both FDA therapy and orphan drug designations. Avexitide is being developed for the treatment of post-bariatric hypoglycemia (PBH), a debilitating condition that affects a significant portion of bariatric surgery patients. The company is on track to initiate a pivotal Phase III trial in PBH in the first quarter of 2025. This trial is designed to evaluate the efficacy and safety of avexitide in reducing hypoglycemia events, with topline data expected in 2026. The positive results from earlier clinical trials, including a 66% reduction in Level 3 hypoglycemia events and a 53% reduction in Level 2 hypoglycemia events, provide a strong foundation for the Phase III program.

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is also focusing on the continued development of AMX0035, a novel therapy with potential applications in multiple rare neurodegenerative diseases. The company recently reported positive topline data from the Phase II HELIOS trial of AMX0035 in Wolfram syndrome, a rare genetic disorder characterized by progressive insulin-dependent diabetes, optic atrophy, and deafness. The trial demonstrated significant improvements in pancreatic function and other disease measures, providing a strong rationale for advancing AMX0035 into a Phase III program. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) plans to meet with the FDA and other stakeholders to inform the design of this Phase III trial, with updates expected in 2025. Additionally, the ORION trial of AMX0035 in progressive supranuclear palsy (PSP) is recruiting well, and the company expects to share interim data in mid-2025.