Amylyx Pharmaceuticals

Amylyx Pharmaceuticals

The U.S. Food and Drug Administration (FDA) approved Relyvrio from Amylyx Pharmaceuticals (NASDAQ: AMLX) in 2022 as a treatment for the underserved population of patients with amyotrophic lateral sclerosis (ALS).

Relyvrio is the company's first and only drug, and its initial launch has been a success with sales that reached $103 million during the third quarter of 2023. Continued success, though, depends on phase 3 trial results expected in March.

ALS is a progressive neurodegenerative disorder that is typically fatal within several years of receiving a diagnosis. Due to a lack of disease-modifying therapies, the FDA was somewhat lenient and approved Relyvrio based on results from a 137-patient phase 2 trial called Centaur.

In Centaur, patients randomized to receive Relyvrio scored significantly lower on an ALS severity test 24 weeks after beginning treatment. The benefit was statistically significant, but there's been criticism regarding the way data was collected.

The European Medicines Agency hasn't approved Amylyx's drug yet, so plenty of eyes will be watching for results from an ongoing phase 3 study, named Phoenix, that could wrap up in March. Amylyx needs success in the Phoenix study to gain marketing authorization for its drug in the EU. In America, though, Relyvrio already has full approval from the FDA, so there's little that the agency can do if Phoenix flops.

Amylyx Pharmaceuticals' $1.1 billion market cap seems too low even if we assume Phoenix will produce less-than-satisfying results. A dearth of treatment options for the ALS community drove Relyvrio sales up to an annualized $411 million in Q3.

With just one drug, any investment in Amylyx is a risky one even if the stock appears deeply undervalued. If you have a high tolerance for risk, though, smashing the buy button on this stock looks like the right move.


https://finance.yahoo.com/m/8ff4c26f-7ae...-huge.html