Patient Dosing Begins in SLS-005 Arm of HEALEY ALS Platform Trial
Dosing has begun in a Phase 2/3 trial of SLS-005, Seelos Therapeutics’ infusion therapy to slow progression in amyotrophic lateral sclerosis (ALS) that is running as the fifth arm of the multi-regimen HEALEY ALS platform trial.
The trial (NCT05136885) plans to enroll 160 people with familial or sporadic ALS, and is part of the larger HEALEY study (NCT04297683) that is already testing four other potential ALS treatments at sites across the U.S. Contact information is available here.
Simultaneously evaluating SLS-005 and other candidates helps to accelerate the development of promising treatments while lowering the costs associated with single-therapy trials.
“Initiating this trial is a major achievement for Seelos and we are honored to be part of the HEALEY ALS Platform Trial. We look forward to offering this investigational therapy to people suffering with this debilitating disease,” Raj Mehra, PhD, chairman and CEO of Seelos, said in a press release.
Seelos called this Phase 2/3 trial registrational in its release, meaning that positive safety and efficacy findings will support requests for regulatory approval of SLS-005.
Participants are being randomly assigned, in a 3:1 ratio, to weekly intravenous (into-the-vein) infusions of SLS-005 (120 patients) or a placebo (40 patients) for 24 weeks, or about six months.
Its primary goal is to evaluate whether SLS-005 slows disease progression, as measured using the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), at 24 weeks. Changes from the study’s start (baseline measures) in patients’ muscle strength, quality of life and lung function will also be assessed, as will treatment safety.
This study’s design — its length, number of enrolled patients, treatment-to-placebo ratio, etc. — is similar to that of all others in the HEALEY trial, with placebo group findings shared across the platform’s five arms to strengthen trial data.
“Partnering with Seelos to determine the efficacy of SLS-005 in this platform trial will give answers sooner because of the sharing of data and infrastructure with other regimens in the platform trial,” said Merit Cudkowicz, MD, the principal investigator of the HEALEY trial, and neurology chief and director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.
SLS-005’s active ingredient is trehalose, a sugar molecule that can enter the brain and promote autophagy — the process by which cells remove old or damaged proteins and cellular components, preventing their toxic accumulation.
Preclinical studies showed that SLS-005 helps to clear ALS-associated proteins, namely TDP-43 and SOD1, which are known to accumulate to toxic levels that damage and kill nerve cells. Data also showed that the treatment could delay disease progression and protect nerve cells from damage.
SLS-005 was designated an orphan drug by the U.S. Food and Drug Administration and the European Medicines Agency, which is intended to speed progress toward the development of therapies for rare diseases.
Candidates for the HEALEY platform trial are selected by a group of ALS experts and members of an evaluation committee. SLS-005 was cleared as the fifth arm in September 2021, joining four other treatments: Clene Nanomedicine’s CNM-Au8 (NCT04414345), Biohaven Pharmaceuticals’ verdiperstat (NCT04436510), UCB’s zilucoplan (NCT04436497), and Prilenia Therapeutic’s pridopidine (NCT04615923). Trials for these first four therapies are fully enrolled.
“This is an important step forward to learning about the safety and efficacy of SLS-005 for people living with ALS,” Cudkowicz said in a separate press release.
“We are thankful to all the patients with ALS who participate in the HEALEY ALS Platform Trial and help develop new treatments in a much faster and more efficient approach,” Cudkowicz added.