Amylyx Pharmaceuticals, Inc. (AMLX)

Amylyx Pharmaceuticals emerged in 2013 as a startup built on an unconventional bet. Cofounders Jonah Shacknai and Joshua Cohen pursued a therapeutic strategy that most of the pharma world had avoided: rather than chasing a single blockbuster molecule, they looked for synergistic effects from combining two compounds. The pair identified amyloid-related disorders as their primary focus, particularly amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease where standard care had barely changed in decades. For years, the company remained largely below the public radar, working through preclinical research and early-stage clinical trials with limited resources and long odds.

The clinical momentum shifted in the late 2010s and early 2020s. Amylyx’s lead candidate—a combination of sodium phenylbutyrate and taurursodiol, later branded as Relyvrio—demonstrated something rare in ALS research: statistically significant slowing of disease progression in a pivotal Phase 2b/3 trial. In an arena where most drug candidates fail at late-stage testing, this result caught the attention of neurologists, patient advocates, and the FDA. The regulator approved Relyvrio in July 2023 under accelerated approval, recognizing both the strength of the data and the acute unmet medical need in a disease with no disease-modifying treatments available for decades.

The company has since shifted gears, pursuing public market status and scaling operations to bring its approved therapy to patients while continuing development of additional candidates within its amyloid-focused pipeline. Like many biotech firms, Amylyx faces the perpetual challenge of biopharmaceutical economics: Relyvrio’s commercial success must fund pipeline advancement, regulatory compliance, and competition from larger players exploring the same territory. The combination-therapy thesis that seemed contrarian in 2013 is now Amylyx’s operational foundation, wagered on the belief that shared disease mechanisms justify multiple compound approaches in this therapeutic space.