A Phase 2b/3 trial investigating the potential of SLS-005 (trehalose) to slow the progression of amyotrophic lateral sclerosis (ALS) has been added to the HEALEY ALS Platform Trial, the first such multi-regimen study created for ALS.
The trial, designed by Seelos Therapeutics, the maker of the candidate therapy, was selected by researchers at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, who are leading the platform trial.
“The inclusion of the registrational phase IIb/III study of SLS-005 in this first ever platform trial is the result of extensive work identifying trehalose as a potential treatment to study in ALS,” Raj Mehra PhD, chairman and CEO of Seelos, said in a press release.
“The Healey Center’s recognition of this potential therapy is a major validation and being part of this platform trial should help to expedite the trial by helping to provide greater access to patients,” Mehra said.
As a platform trial, HEALEY (NCT04297683) is evaluating the potential of multiple ALS treatment candidates simultaneously, with a goal of speeding the development of those that seem to be the most promising.
Based on the same design of platforms successfully used in the field of oncology, HEALEY aims to promote patient access to clinical trials, while lowering the costs and reducing the development time of potential therapies.
The study received authorization from the FDA earlier this year to start evaluating its first three ALS treatment candidates: Ra Pharma’s zilucoplan, Clene Nanomedicine’s CNM-Au8, and Biohaven Pharmaceuticals’ verdiperstat.
Patient dosing began in August across 54 sites in the U.S., all part of the Northeast ALS (NEALS) consortium. After enrollment, participants are randomly assigned to complete a 24-week treatment regimen with either zilucoplan (regimen A; NCT04436497), verdiperstat (regimen B; NCT04436510), or CNM-Au8 (regimen C; NCT04414345).
In addition to SLS-005, two other ALS therapy candidates — Prilenia’s pridopidine and Implicit Bioscience’s IC14 immunotherapy — are in the process of being added to the list of potential treatments evaluated in HEALEY.
The main goal is to assess the safety of each therapy, and evaluate the effectiveness of each at slowing ALS progression, improving patients’ muscle strength, lung function, and survival.
In each regimen, 160 patients will be randomized to either the investigative treatment or a placebo in a 3:1 ratio, meaning that for every three patients on treatment, one will be given a placebo. Treatment will last 24 weeks.
“We are excited to work with Seelos and look forward to studying SLS-005 in an accelerated format through the HEALEY ALS Platform Trial,” said Merit Cudkowicz, MD, director of the Sean M. Healey Center, and principal investigator of HEALEY.
“The design team will work closely with Seelos on their regimen specific protocol as well as completing required steps with the central ethics review board and the FDA,” Cudkowicz added.
Trehalose, SLS-005’s active ingredient, is a natural sugar molecule found in plants, fungi, and bacteria that has been shown to promote autophagy — the process by which cells degrade and recycle old or damaged components they no longer need.
This mechanism of action is also expected to help nerve cells eliminate toxic deposits of certain proteins, such as TDP-43 and SOD1, that are known to contribute to ALS. This has already been demonstrated in preclinical studies of SLS-005. By preventing, or even reversing, the accumulation of these toxic proteins, SLS-005 is also expected to help slow ALS progression.
The investigational therapy was named an orphan drug for the treatment of ALS in the U.S.
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