SLS-005 Earns Orphan Drug Status in Europe

Vanda Pinto, PhD avatar

by Vanda Pinto, PhD |

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The European Medicines Agency (EMA) has granted orphan drug designation to Seelos Therapeutics‘ investigational therapy SLS-005 for the treatment of amyotrophic lateral sclerosis (ALS), the company announced in a press release.

Orphan drug status in Europe is given to medicines with the potential to be safe and effective treatments for rare, life-threatening, or chronically debilitating conditions affecting no more than one every in 2,000 people. It provides companies with a range of incentives, including assistance with trial protocols, reduced regulatory fees, and a 10-year period of market exclusivity if the treatment eventually is approved.

The designation follows recent a positive opinion from EMA’s Committee for Orphan Medicinal Products, and an orphan drug designation granted by the U.S. Food and Drug Administration late last year.

SLS-005 consists of a natural sugar molecule, called trehalose, found in plants, fungi, and bacteria. Studies have shown that SLS-005 promotes autophagy, a cellular process that breaks down and recycles old and damaged proteins before they accumulate and become toxic.

In ALS, the accumulation of proteins such as TDP-43 and SOD1 in nerve cells can contribute to the development of the disease. Preclinical studies revealed that SLS-005 increases the clearance of these proteins, delaying the progression of ALS and preserving motor neurons and muscle fiber size.

SLS-005 also can cross the blood-brain barrier — the semi-permeable protective layer that normally prevents molecules from traveling to the central nervous system and causing damage — and reach impaired nerve cells.

Late last year, SLS-005 became the fourth potential ALS therapy to be added to the HEALEY clinical trial (NCT04297683), a platform study that is testing multiple therapies at the same time to advance clinical development of promising treatments while reducing costs.

The first medications included are UCB’s zilucoplan, Biohaven Pharmaceuticals‘ verdiperstat, Clene Nanomedicine’s CNM-Au8, and Prilenia Therapeutics’ pridopidine.

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

The main goal is to evaluate how each treatment affects disease progression, measured as changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), lung function, and muscle strength.

The HEALEY ALS Platform trial is currently enrolling adults with sporadic or familial ALS at 54 locations in the U.S. More information about contacts and locations is available here.