Amylyx Pharmaceuticals Begins Enrolling Phase 2 Study on AMX0035 Recently, Amylyx Pharmaceuticals announced the opening of enrollment of their Phase 2 clinical trial of AMX0035 in people with ALS. AMX0035 is a combination of two compounds, sodium phenylbutyrate (NaPB) and tauroursodeoxycholic acid (TUDCA).

Amylyx has reported that their proprietary combination of these compounds works synergistically to address pathways activated in the mitochondria and endoplasmic reticulum of cells that have been linked to advancing their death. Independent research on the two compounds in preclinical models, both cell and animal based, have produced both positive and neutral/negative results.

The clinical trial, dubbed “The CENTAUR trial” by Amylyx, includes two dozen enrollment sites spanning the United States. Several sites are open for enrollment, with the remaining sites expected to open sporadically throughout the rest of 2017. Updates on site enrollment status and other changes to the study are monitored by ALS TDI and sent monthly via email to those subscribed to the clinical trial mailing list.

Amylyx aims to enroll 132 people with ALS in this placebo controlled study. Two out of every 3 people enrolled will be given the active compound, AMX0035, and the remainder will receive the placebo. The six-month study is being enrolled by the Northeast ALS Consortium. Study investigators, including Patrick Teramian, MD (Amylyx) and Sabrina Paganoni, MD (MGH), are seeking individuals with ALS that have a slow vital capacity value of greater than 60% and have experienced their onset of ALS symptoms within 18 months of their enrollment in the trial. Several key exclusion criteria have been stated, such as allergy to bile salts, abnormal liver function, renal insufficiency, poorly controlled arterial hypertension, among others.

Individuals that complete the 24 week study will be offered access to AMX0035 through an open-label continuation study.

A full description of this clinical trial including contact information can be found online here.

Helpful Links:

AMX0035 Clinical Trial Design and Contact Information 

Results from TUDCA Clinical Trial in Italy

Press Release from Amylyx on Starting Enrollment