The first patients to complete a Phase 2 clinical trial have chosen to continue treatment in an open-label extension (OLE) study of Amylyx Pharmaceuticals‘ investigative therapy AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS).
AMX0035 is an oral combination of two small molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). The therapy candidate is designed to reduce nerve cell death by blocking key cellular death pathways that originate in the mitochondria and endoplasmic reticulum — two key players in both nerve cell death and neuro-inflammatory processes.
All patients who complete the Phase 2 CENTAUR (NCT03127514) study will be eligible to enroll in CENTAUR-OLE, the extension study, to receive AMX0035 with no placebo comparator.
The CENTAUR trial began dosing participants in July 2017 but is still recruiting new patients. The randomized, double-blind, placebo-controlled study aims to enroll 132 ALS patients in the U.S. to evaluate the safety and tolerability of AMX0035 and to assess the drug’s impact on disease progression over 24 weeks. CENTAUR is estimated to end in May 2019.
“The CENTAUR-OLE study provides patients with the option of continuing treatment once they have completed the 24-week study period,” Sabrina Paganoni, an MD and PhD, principal investigator for the study, said in a press release.
“The study will provide data on AMX0035’s long-term safety and tolerability as well as additional insights into its efficacy. There is a significant and urgent need for therapies for ALS, as there are very limited treatment options.”
This is the first trial to measure patient muscle strength using a device called Accurate Test of Limb Isometric Strength (ATLIS). The study will also assess promising blood and imaging-based biological markers of ALS to confirm that the therapy is reaching its targets.
Disease progression will be measured by the revised ALS Functional Rating Scale (ALSFRS-R).
The U.S. Food and Drug Administration granted AMX0035 orphan drug status in September 2017.
Amylyx’s announcement was made in collaboration with the ALS Association, ALS Finding a Cure, the Northeast ALS Consortium and the Massachusetts General Hospital Neurological Clinical Research Institute.
Justin Klee, president and co-founder of Amylyx, thanked those organizations and the ALS community for their support in helping Amylyx offer the extension study.