Amylyx Deepens Commitment to Blood Sugar Drugs with GLP-1 Acquisition

Amylyx Pharmaceuticals, a biotechnology firm based in Massachusetts, has swiftly advanced from identifying a potential medication to obtaining approval and making a profit within a decade. However, their initial success was fleeting. A pivotal trial intended to verify their ALS drug’s efficacy demonstrated that it was no better than a placebo, causing their stock price to plummet from about $19 to below $4 in a single day. Consequently, Amylyx withdrew its sole product from the market.

Several months later, Amylyx made a strategic move by acquiring avexitide, an experimental drug that positions the company within a prominent area of medical research. Avexitide targets a protein linked to GLP-1, a hormone crucial in obesity and diabetes treatment. GLP-1 reduces blood sugar by activating receptors that prompt the pancreas to produce more insulin. Drugs like Novo Nordisk’s Ozempic and Eli Lilly’s Mounjaro mimic this hormone to stimulate insulin production. In contrast, avexitide blocks these receptors, thereby increasing blood sugar levels.

Amylyx’s co-CEOs, Justin Klee and Joshua Cohen, believe this effect is significant. They envision avexitide as a crucial treatment for conditions characterized by low blood sugar. They plan to initiate a pivotal study early next year on people experiencing hypoglycemia after bariatric surgery. Cohen highlighted the business potential, noting the strong data supporting avexitide and the readiness for Phase 3 trials.

Amylyx had been exploring various acquisition opportunities to diversify its pipeline over the past few years, evaluating hundreds of drugs. Avexitide stood out due to its relatively lower risk compared to other experimental drugs. Its previous owner, Eiger BioPharmaceuticals, had already conducted several mid-stage clinical trials for post-bariatric hypoglycemia and congenital hyperinsulinism, a genetic disorder causing excessive insulin production.

Additionally, Amylyx has been investigating its ALS drug, AMX0035, for treating Wolfram syndrome, a rare genetic condition causing severe health problems, including high blood sugar levels. Acquiring avexitide was strategically sensible, enhancing Amylyx’s presence in endocrinology.

Cohen emphasized that drug development in endocrinology is promising due to the clear and well-understood markers that can be followed. Moreover, while most GLP-1-related medicines are agonists, avexitide is an antagonist. This distinction could potentially make avexitide the first of its kind on the market, which could be highly profitable.

Amylyx purchased avexitide for $35 million during Eiger’s bankruptcy auction. The acquisition was completed on July 9, and at the end of March, Amylyx had $373 million in cash, cash equivalents, and short-term investments. Given the trials already conducted by Eiger and the rarity of post-bariatric hypoglycemia, Amylyx anticipates needing only one late-stage study, with results expected by 2026. If approved, avexitide could enter the market by 2027.

The acquisition of avexitide does not signify a shift for Amylyx but rather an expansion. The company is also developing an antisense therapy for ALS, aiming to begin human trials by the end of the year. Additionally, Amylyx is working on a blood-based diagnostic tool for ALS and exploring the potential of AMX0035 for treating Alzheimer’s disease and progressive supranuclear palsy (PSP), with mid-stage study data for PSP expected next year.