Learn about Amylyx’s investigational therapy for ALS
Clara Health

Learn about Amylyx’s first in class investigational therapy for ALS

AMX0035 is an oral therapy that demonstrated a significant treatment benefit in the phase 2 study

Amylyx’s drug, a powder that has been going by the designation AMX0035, combines two separate medicines. Each strikes a different line of attack that ALS takes to destroy the motor neurons in patients.

The CENTAUR study is not accepting new participants. To be introduced with study teams for ALS trials that are currently active, please fill out the interest form below.


More than 16,000 Americans have ALS, which kills off the nerve cells that control muscle movements. As these motor neurons die, patients lose the ability to eat, speak, move and breathe. Within three to five years of symptoms, most patients die.

Only two drugs have been approved to treat the underlying cause of the disease, and neither provides a cure.

About the CENTAUR study

Dr. Sabrina Paganoni from Massachusetts General Hospital discusses CENTAUR,
a phase 2 trial for people with ALS.

Study results

Who is this study for?

In the CENTAUR trial, AMX0035 was tested in patients who were diagnosed within 18 months of symptoms appearing and had developed problems like trouble speaking, difficulty walking and arm weakness.

The 137 subjects in the trial took either AMX0035 or a placebo for six months in addition to any other ALS drugs they were taking.

What should I expect?

Participants in CENTAUR were given the option after the trial to enroll in an open-label extension study to receive treatment with AMX0035. Nearly 90 percent of participants who completed CENTAUR elected to enroll in the extension study.

Interim data from the ongoing extension study will be presented in 2020.

Frequently asked questions

Will the treatment be available after the study ends?

The CENTAUR-OLE study provides patients with the option of continuing treatment once they have completed the 24-week study period.

What will this study evaluate?

The trial will evaluate AMX0035’s ability to have an impact on daily functioning, muscle strength, and respiratory function as well as its ability to alter biological markers of ALS.

Additional details

"To accelerate developing new treatments for ALS, the two foundations issued a global, competitive call for grant proposals for therapies that had a strong scientific rationale and where a biomarker of drug effect could be measured. AMX0035 was chosen as one of the top new therapeutic candidates to bring forward for ALS."

- Merit Cudkowicz, MD, Chief Medical Officer for ALS Finding A Cure and Chief of Neurology at Massachusetts General HospitalMerit Cudkowicz

Have a question?

To connect with ongoing ALS studies, fill out the interest form, contact +1 (415) 326-8831, or email the Clara Health team at [email protected]