FDA Gives Green Light to Healey ALS Platform Trial


The first trial of its kind in amyotrophic lateral sclerosis will first examine 3 drugs and add additional treatments as more become available.

Sabrina Paganoni, MD, PhD

Sabrina Paganoni, MD, PhD

The FDA has approved the investigational new drug (IND) application for the HEALEY ALS (amyotrophic lateral sclerosis) platform trial, which will first test zilucoplan, verdiperstat, and nanocrystalline gold in patients with ALS.1 The trial, which is the first of its kind in the disease, will take place across 54 sites in the Northeast ALS Consortium (NEALS) in conjunction with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in Boston.

“Through dedication, enthusiasm, and collaboration, the Healey Center, and its many collaborators are one step closer to accelerating ALS therapy development. We are very grateful for the support of the Healey Center, Tackle ALS, the ALS Association, sALSa for a cure, ALS One, and the recent ice bucket challenge 2 to help launch this initiative,” Merit Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS at Mass General, said in a statement.1

Patients will be randomly assigned 3:1 to receive daily subcutaneous doses of 0.3 mg/kg of zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 developed by Ra Pharmaceuticals, or placebo.2 Using the ALS Functional Rating Scale-Revised (ALSFRS-R) as a primary end point, investigators will assess the disease progression compared to placebo. The company plans to enroll 160 patients for a 24-week treatment period and will offer an open-label extension study for patients following their completion.2

READ MORE: The Road to Success in ALS: Details of the AMX0035 CENTAUR Trial

The trial will also assess verdiperstat, an oral myeloperoxidase inhibitor developed by Biohaven Pharmaceutical. The third drug observed, CNM-Au8 nanocrystalline gold, is an intracellular nanocatalyst that supports cellular bioenergetics and is developed by Clene Nanomedicine. Plans for the use of verdiperstat and nanoncrystalline gold were not disclosed.

Investigators will add new treatments to the perpetual trial as they become available.

“Obtaining a ‘May Proceed’ notice from the FDA means there are no major issues with the study and the FDA agrees that we can move forward with the first three experimental treatments to be tested,” Sabrina Paganoni, MD, PhD, assistant professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospitaland co-lead investigator for the HEALEY ALS Platform Trial, said in a statement. “This is a crucial step in providing more access and more options for people with ALS.”

Paganoni recently hedged another success in ALS with the announcement of positive top-line data from the phase 2 CENTAUR trial, which is evaluating Amylyx Pharmaceuticals’ AMX0035. Treatment with the drug resulted in a statistically significant (P <.05) slowing of disease progression in patients with ALS compared with placebo.

Notably, nearly 90% of the trial participants who completed the 24-week study went on to enroll in the ongoing open-label extension, which will readout data in 2020.


1. Sean M. Healey & AMG Center for ALS at Mass General receives “May Proceed” notice for three drugs in first ALS platform trial [news release]. Boston, MA: Massachusetts General Hospital. January 22, 2020. massgeneral.org/neurology/als/news/2020-01-22-Healey-ALS-platform-trial-may-proceed. Accessed January 22, 2020.

2. Ra Pharmaceuticals announces clearance of IND applications for the HEALEY ALS platform trial [news release]. Ra Pharmaceuticals. January 22, 2020. finance.yahoo.com/news/ra-pharmaceuticals-announces-clearance-ind-140000447.html. Accessed January 22, 2020.

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