Enrollment complete in HEALEY Phase 2/3 trial arm of SLS-005

Study being led by researchers at the Sean M. Healey & AMG Center for ALS

Teresa Carvalho, MS avatar

by Teresa Carvalho, MS |

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Seelos Therapeutics has finished enrolling patients in its Phase 2/3 clinical trial of SLS-005 to treat amyotrophic lateral sclerosis (ALS), the company announced.

The ongoing trial (NCT05136885) recruited 160 patients with familial and sporadic ALS, and top-line results are expected by the second half of 2023.

This is the fifth arm of the HEALEY platform trial (NCT04297683), which is being led by researchers at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH), Boston to investigate several ALS therapies simultaneously against a shared placebo group.

The company also expects to launch an expanded access program (EAP) in the coming months to give people with ALS who aren’t eligible for clinical trials the opportunity to access SLS-005. The EAP will include 70 participants who’ll receive the experimental therapy for 24 weeks. More information is available at Seelos’ website.

“The full enrollment of this study is a significant milestone for Seelos. We look forward to releasing the top-line data later this year and initiating the Expanded Access Program later this quarter,” said Raj Mehra, PhD, Seelos’ chairman and CEO, in a company press release.

EAP will be funded by a grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS).

“We would like to offer our gratitude to the ALS community for its support and to the team at Healey & AMG Center for ALS at Mass General and the clinical trial sites in the Northeast ALS Consortium for their hard work and commitment in running this innovative platform trial. We also thank the NINDS for the financial support of the Expanded Access Program grant under the ACT for ALS,” Mehra said.

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SLS-005 promotes protein clearance

SLS-005 is made of a natural sugar molecule called trehalose that can enter the brain and promote autophagy, the process by which cells break down and recycle old or faulty proteins they no longer need, preventing their toxic buildup.

Administered directly into the bloodstream, SLS-005 has been shown to lower faulty proteins’ aggregation and reduce the accumulation of toxic clumps in animal models of neurodegenerative conditions.

In ALS models, it promoted the clearance of TDP-43 and SOD1 proteins, which are known to accumulate to toxic levels and contribute to nerve cell death. By doing so, the treatment was able to prevent nerve cell loss and delay disease progression in these models.

SLS-005 received orphan drug status — a designation that’s intended to speed the development and review of therapies for life-threatening rare diseases — in both the U.S. and the European Union.

In the SLS-005 arm of the HEALEY ALS platform trial, participants were randomly assigned to weekly intravenous (into-the-vein) injections of SLS-005 or a placebo for 24 weeks (about six months). The patients were randomized in a 3:1 ratio, meaning for each patient assigned a placebo, three were given SLS-005.

The trial’s main goal is to evaluate whether SLS-005 slows disease progression, as measured with the ALS Functional Rating Scale Revised (ALSFRS-R). Secondary goals include changes in lung function, muscle strength, and quality of life, as well as symptoms of disease progression.

Other treatments being investigated in the HEALEY trial include Prilenia Therapeutic’s pridopidine (NCT04615923) and Clene Nanomedicine‘s CNM-Au8 (NCT04414345).

Another regimen, UCB’s zilucoplan (NCT04436497), was discontinued in March 2022 due to a lack of effectiveness in an interim analysis. Biohaven Pharmaceuticals’ verdiperstat (NCT04436510) also failed to meet its primary and secondary goals.