Funding Expands Access to SLS-005 Via Compassionate Use

Trial of Seelos' investigational ALS therapy will enroll about 70 people

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by Teresa Carvalho, MS |

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Seelos Therapeutics is planning to launch an expanded access program (EAP) that gives people with amyotrophic lateral sclerosis (ALS) who are not eligible for clinical trials the opportunity to access the company’s investigational therapy SLS-005.

The program will be conducted in collaboration with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH), in Boston. It will be funded fully 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).

The Healey & AMG Center for ALS also is leading the ongoing HEALEY platform trial (NCT04297683), which is investigating SLS-005 and several other therapies as potential treatments for ALS.

“Being chosen to again collaborate with the Healey team on an EAP is a privilege and further supports our commitment to provide a meaningful contribution to the ALS community by continuing to explore investigational treatment options in this devastating disorder,” Raj Mehra, PhD, Seelos’ chairman and CEO, said in a press release.

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EAP programs aim to make specific investigational treatments available outside the clinical trial setting to people whose serious or life-threatening conditions have few or no suitable treatments. They further produce information that can be used in therapy development.

The upcoming EAP is expected to enroll about 70 participants who will receive the experimental medication for 24 weeks. Those interested may find  additional information here.

It will run in parallel with HEALEY’s SLS-005 treatment arm (NCT05136885), which is comparing weekly intravenous (into-the-vein) infusions of SLS-005 against a placebo in 160 participants, also for a period of 24 weeks.

“Through this EAP, we will be able to offer SLS-005 to people who are unable to meet the entry criteria for any clinical trials. Our hope, through this effort, is to collect additional data to help us advance this therapy through the clinic,” added Mehra.

ALS is characterized by the buildup of toxic protein clumps in nerve cells, which result from certain proteins failing to acquire their normal three-dimensional shape and instead sticking to each other, forming aggregates.

SLS-005’s active ingredient, trehalose, is a natural sugar molecule that can promote autophagy in the brain. This is a natural recycling process through which cells break down and recycle old or faulty molecules they no longer need, preventing their toxic accumulation.

The investigational therapy has been shown to decrease clustering of faulty proteins, including TDP-43 and SOD1, and lower their buildup in toxic clumps in animal models of ALS.

‘Basket trial’ underway

In addition to HEALEY, SLS-005 also is being investigated in a basket trial, a trial format that tests one therapy in several conditions with a common molecular feature.

The Phase 2 trial (ACTRN: 12621001755820), ongoing in Australia, is specifically enrolling people with ALS, Huntington’s disease, and spinocerebellar ataxia type 3 (SCA3) – all of which are associated with the accumulation of toxic protein aggregates.

The ACT for ALS Act, which will make available $100 million annually from 2022 to 2026, aims to fund research into fast-progressing neurodegenerative conditions, such as ALS, and provide patients with early access to promising therapies.

The legislation (Public Law 117-79) also is meant to create a U.S. Food and Drug Administration Rare Neurodegenerative Disease Grant Program to support research and development in seriously debilitating neurodegenerative disorders.