PharmaVoice 17 July 2024. Interesting article and keen to hear...

PharmaVoice 17 July 2024. Interesting article and keen to hear thoughts especially from those with a medical bent

Among rare diseases, ALS doesn’t suffer from a lack of research.

Just this week, a group of scientists released findings from a study in Spain that uncovered a new genetic link to ALS. Conducted in a region with a higher-than-normal incidence of ALS, the small study revealed mutations in ARPP21 in 10 patients from seven unrelated families. ALS researchers have already identified 40 genes related to the disease, and the scientists who conducted this study suggested ARPP21 should be reclassified as a known cause of ALS to guide diagnosis and drug R&D.

Josh Cohen, co-CEO of ALS drug developer Amylyx Pharmaceuticals, noted the company has long benefited from the robust level of research in the space.

“In ALS, there is more going on every year, and through that, people are uncovering more about the underlying science that will lead to better therapeutics,” Cohen said.

Despite these frequent discoveries, drug development success in ALS has remained elusive, as Amylyx well knows. Earlier this year, the company’s ALS treatment Relyvrio was pulled from the market after a confirmatory trial showed it didn’t work.

Today, we’re featuring a conversation with the company’s co-CEOs about what they learned from that experience and how they’re staging a comeback with a new late-stage candidate in the GLP-1 space.