Dosing of SLS-005 to Soon Begin in Phase 2b/3 HEALEY ALS Trial Arm

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by Marta Figueiredo PhD |

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Seelos Therapeutics has announced plans to start patient dosing in a pivotal Phase 2b/3 trial testing its experimental therapy SLS-005 (trehalose), designed to prevent protein clumping in cells, in adults with amyotrophic lateral sclerosis (ALS) by the close of September.

The study is part of the HEALEY ALS Platform Trial (NCT04297683), the first multi-regimen trial in ALS, and will mark the fifth treatment to be simultaneously tested in the study. The simultaneous assessment of several potential therapies aims to speed the development of those showing the most promise, while lowering costs.

HEALEY is being led by Harvard Medical School at Massachusetts General Hospital, and is recruiting adults with sporadic ALS or familial ALS at more than 50 U.S. trial sites. More information on contacts and locations can be found here.

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SLS-005, administered directly into the bloodstream, consists of a natural autophagy-promoting sugar molecule found in plants, fungi, and bacteria, called trehalose. Autophagy is the process by which cells breakdown and recycle old or damaged proteins and components they no longer need, preventing their potentially toxic accumulation.

ALS is characterized by the toxic buildup of several proteins, such as TDP-43 and SOD1, in nerve cells, contributing to their damage and death. Preclinical studies showed that SLS-005 promotes the clearance of these proteins, delaying ALS progression and preserving motor neurons and muscle fiber size.

In addition, SLS-005 can cross the blood-brain barrier — the highly selective membrane that prevents circulating microbes and potentially harmful molecules from reaching the central nervous system (CNS; brain and spinal cord) — overcoming a common obstacle of CNS-targeted therapies.

The therapy received orphan drug designation in the U.S. and in the European Union. The designation is meant to accelerate SLS-005’s development by providing financial incentives, regulatory support, and market exclusivity for seven years in the U.S. and 10 years in Europe upon approval.

Late last year, Seelos announced the inclusion of its Phase 2b/3 trial of SLS-005 in the HEALEY platform study, which began dosing patients in its first three experimental therapies in August 2020.

These potential treatments are UCB’s zilucoplan (NCT04436497), Biohaven Pharmaceuticals’ verdiperstat (NCT04436510), and Clene Nanomedicine’s CNM-Au8 (NCT04414345).

Prilenia Therapeutics’ pridopidine was also added to list of experimental regimens being tested in the platform trial (NCT04615923), with the first patient enrolled for this group in January.

In each arm of HEALEY, 160 patients are randomly assigned to the investigative treatment (120 patients) or a placebo (40 patients) for 24 weeks (about six months). The placebo group will be shared among all regimens to strengthen trial data.

The main goal is to assess the effectiveness of each therapy at slowing disease progression, measured as changes in the ALS Functional Rating Scale-Revised. Secondary measures include safety, changes in lung function and muscle strength, and survival.

In the mid-year business and clinical update that included the announcement of SLS-005 patient dosing plans, Raj Mehra PhD, Seelos’s chairman and CEO, noted that the company had been issued a patent by the Australian Patent Office covering the into-the-vein administration of SLS-005 in several neurodegenerative conditions.

Seelos is continuing to evaluate SLS-005 as a potential treatment for additional disorders, Mehra added.