Retrotope’s Experimental RT001 Now Available for ALS Under Expanded Access Program

Retrotope is providing its investigational fatty acid being developed for Friedreich’s ataxia, RT001, to people with amyotrophic lateral sclerosis (ALS) under an expanded access program, also called “compassionate use.”

Individual patients with life-threatening or severely debilitating diseases can petition the U.S. Food and Drug Administration, through their physicians, for expanded access to experimental therapies currently in clinical trials, but have not yet been FDA-approved.

The petition for access was filed by investigators at major medical centers. For the expanded access program, they expect to enroll a few ALS patients from their centers to gather initial clinical data on the therapy’s performance in people with ALS.

Retrotope announced that the first patient has been dosed.

The sponsor, in this case Retrotope, carries the ultimate decision whether to release their investigational therapy for expanded access programs.

ALS is associated with impaired mitochondrial functions — the small energy powerhouses inside every cell — whose function is vital, particularly for nerve cells due to their high need for energy.

Impaired function of mitochondria is increasingly recognized as an initial event in ALS, but also as an underlying cause for other neurodegenerative diseases, including Alzheimer’s, Parkinson’s, Huntington’s disease, and inherited disorders, such as Friedreich’s ataxia and infantile neuroaxonal dystrophy (INAD).

Poor mitochondria function results in the generation of damaging molecules, called free radicals and reactive oxygen species (ROS), that attack and degrade polyunsaturated fats (PUFAs), a type of fat (or lipid) that is essential for the maintenance of cellular membranes.

RT001 is a highly stabilized version of linoleic acid, also known as Omega-6 fatty acid. An essential PUFA, linoleic acid can only be obtained from food.

RT001 blocks the chemical reactions that damage these lipids. It is incorporated into the membranes and restores membrane functions and cellular health.

“Early results in clinical trials of RT001 and expanded access programs for a variety of neurodegenerative diseases, including Friedreich’s ataxia, infantile neuroaxonal dystrophy (INAD), and late-onset Tay Sachs, which involve oxidative mitochondrial dysfunction, have generated positive results along with a favorable safety profile,” Peter G. Milner, MD, the chief medical officer of Retrotope, said in a press release.

“We look forward to providing updates on this and our other studies in the coming months,” he added.

Initially developed for inherited disorders such as Friedreich’s ataxia, the newly expanded access program will provide the first clinical data in ALS.

“Patients are excited to try a potentially efficacious and disease-modifying treatment for largely untreated, devastating and fatal diseases,” said Robert J. Molinari, PhD, the CEO and co-founder of Retrotope.

“Therefore, while we prepare and initiate our pivotal studies of RT001 in INAD and Friedreich’s ataxia, our ability to supply drug to expanded access programs requested by patients and their physicians allows us to provide access to this important experimental therapy and generate valuable clinical information about our drug’s performance in different diseases,” he said.