The ALS Association and I AM ALS have launched a petition calling on the FDA and Amylyx Pharmaceuticals to bring AMX0035, a promising new drug developed by Amylyx, to market as soon as possible. Phase 2 testing of AMX0035 showed the drug slowed the progression of ALS. The petition asks that FDA agree to forego a phase 3 trial and instead, require rigorous follow-on testing as the drug is made broadly available to people living with the disease at the earliest possible opportunity. The results of its AMX0035 phase 2 trial were published on Wednesday in the New England Journal of Medicine.

AMX0035 is an oral therapy dissolved in water that combines two existing drugs, sodium phenylbutyrate and taurursodiol. Trial results indicate the drug cocktail slowed ALS progression in a statistically meaningful and clinically relevant way and that the drug combination is generally safe and well-tolerated. Moreover, the safety of the underlying drugs that make up AMX0035 has been well established clinically.  

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The phase 2 trial included 137 people with ALS across 25 medical centers that are part of the Northeast ALS (NEALS) continuum. People with ALS who received AMX0035 performed 2 points better on the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), which could mean the difference between a person with ALS being able to feed themselves versus being fed, or the difference between needing a wheelchair versus not needing one.  

Given the large phase 2 sample size, clinically relevant slowing of disease progression, and significant evidence that the drug is safe, the Association and I AM ALS launched the petition asking that Amylyx and the FDA move with urgency make this drug available for all people living with ALS. People with ALS cannot wait for a phase 3 trial to conclude given the promising results shows in phase 2. Since the FDA has emphasized the appropriateness of exercising flexibility when dealing with serious diseases and unmet medical needs, there is no reason to withhold this promising drug from the ALS community. Read and sign the petition:  www.als.org/petition. 

The ALS Association supported the development of AMX0035 with a $2.2 million grant in 2016 – one of the first research grants funded with ALS Ice Bucket Challenge donations. As a standard provision in philanthropic support for drug development, the grant included a repayment provision allowing the Association to recover up to 150 percent of its support, or up to $3.3 million.  The funds will be reinvested into ongoing global research into treatments and, ultimately, a cure.