Amylyx Pharmaceuticals achieved what all upstart drugmakers hope to accomplish. In less than 10 years, the Massachusetts-based biotechnology company identified a potential medicine, gathered enough evidence to get it approved and started turning a profit. But success was short-lived. A key trial meant to confirm the medicine was effective for ALS instead found it no better than a placebo. The results sent Amylyx’s share price plummeting, from around $19 to under $4 in the span of a day. The company has since pulled its only product from the market. Now, several months later, Amylyx has bought an experimental drug that propels it into one of the hottest areas of medical research. The drug, named avexitide, acts on a protein tied to “GLP-1” — the hormone at the center of a revolution in obesity and diabetes care. In the body, GLP-1 lowers blood sugar by latching onto certain “receptors” that tell the pancreas to make more insulin. While drugs like Novo Nordisk’s Ozempic and Eli Lilly’s Mounjaro spur insulin production by mimicking this hormone, avexitide effectively does the opposite. It blocks the receptors, and in doing so raises blood sugar. Justin Klee and Joshua Cohen, the co-CEOs of Amylyx, believe that effect can be just as important. They see avexitide becoming a needed treatment option for conditions hallmarked by low blood sugar. Early next year, they plan to push their newly acquired drug into a pivotal study of people with hypoglycemia following bariatric surgery. “We really saw this as, from a business perspective, a pretty unique opportunity to have a therapeutic candidate ready to go into Phase 3 with such strong data behind it,” Cohen said.
According to Klee and Cohen, Amylyx had been looking over the past few years for potential acquisitions that could expand its pipeline. The company evaluated hundreds of drugs. Among them, avexitide stood out for several reasons. Amylyx has also been testing its ALS drug — known as AMX0035 — as a potential treatment for Wolfram syndrome, a rare, inherited condition that causes a medley of health issues, including dangerously high blood sugar levels. Bringing avexitide in house therefore made strategic sense, as it would grow the company’s presence in endocrinology. “There is something very encouraging for drug development, about just how well understood and how clear some of the markers that you can follow are in the endocrine space,” Cohen said.
Additionally, medicines in the GLP-1 field are typically “agonists,” or promoters. As an antagonist, avexitide could become the first drug of its type to reach the market — a title that, in the pharmaceutical industry, often lifts profits. “This was really one of the first [drugs] that checked all the boxes for us,” Klee said.
Eiger filed for bankruptcy this spring, which allowed Amylyx to swoop in and buy the drug at auction for the relatively low price of $35 million. The acquisition, disclosed by Eiger last month, was completed July 9. At the end of March, Amylyx had $373 million worth of cash, cash equivalents and short-term investments. Given all the trials Eiger ran, and that post-bariatric hypoglycemia is uncommon, Cohen and Klee think Amylyx will only need to conduct one late-stage study. They expect results to come in 2026 and, if approval followed, avexitide to reach market by 2027. The executives argue their new purchase doesn’t signal a pivot for Amylyx, but rather an addition. In ALS, the company has a newer, so-called antisense therapy it hopes to move into human testing before the end of the year. It’s also continuing work on a blood-based tool for diagnosing the nerve-destroying disease.