Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a rare, progressive neurodegenerative disease that primarily involves the nerve cells, or neurons, that are responsible for controlling voluntary muscle movements such as walking, talking and chewing. ALS and its related disorders are caused by the gradual degeneration of motor neurons, which are responsible for controlling communication between the brain and muscles responsible for voluntary movements. As these motor neurons deteriorate, communication between the brain and these muscles is impaired, leading to difficulty for patients in performing voluntary movements. As the disease progresses, voluntary muscle control becomes more and more difficult for patients, leading to an inability to breath on their own and ultimately death. There is currently no cure for ALS and no effective treatment to halt, or reverse, the progression of the disease.

Retrotope is currently developing RT001, its lead clinical candidate, for the treatment of ALS. The company initiated a multicenter, randomized, double-blind, placebo-controlled Phase 2 study evaluating the efficacy, safety and tolerability of RT001 in patients with ALS in March 2021. Due to demand for new ALS treatment options and interest in RT001 within the ALS research community, Retrotope exceeded the study’s enrollment target of 40 patients in less than six weeks. Based on this rapid enrollment, the company expects data from the study to be available by the end of 2021.

Additional patient and healthcare provider resources on Amyotrophic Lateral Sclerosis are available in the Patient Resources section.

“Despite the growing global awareness and advocacy for ALS, the therapeutic needs of people living with this devastating disease remain unmet.  Our team is excited to be involved in this clinical study as we believe that RT001, by virtue of its ability to down-regulate lipid peroxidation and protect cellular and mitochondrial membranes, represents a promising approach to mitigating the debilitating course of ALS.”

— Leonard H. van den Berg, M.D., Ph.D., professor of neurology at UMC Utrecht in the Netherlands, director of the Netherlands ALS Center, chairman of TRICALS, an ALS clinical trial-focused European research initiative spanning 42 top research centers in 15 countries, and the principal investigator of the ongoing Phase 2 trial of RT001 in ALS.