First Patients Dosed in Phase 2 Trial of RT001

Retrotope has dosed the first patients in its recently-initiated Phase 2 clinical trial evaluating RT001 for the treatment of amyotrophic lateral sclerosis (ALS).

Enrollment is open across four European sites for patients ages 20–75 whose disease symptoms began less than three years ago and do not need assistance for everyday activities. More information about trial contacts and locations is available here.

The trial (NCT04762589) is comparing the safety, tolerability, and efficacy of RT001 versus a placebo in approximately 40 ALS participants. As more than 40 patients have so far demonstrated interest in participating, Retrotope expects the study to complete enrollment quickly and to announce new data by the end of the year.

Participants will receive three RT001, or placebo, capsules three times daily for one month,  followed by three capsules twice a day for the next five months.

The main goal is to assess changes in disability, using the revised ALS Functional Rating Scale,  after the six months of treatment. Additional goals are to measure changes in self-reported health status, changes in respiratory function, a composite measure of death in disease progression, and side effects.

“Despite the growing global awareness and advocacy for ALS, the therapeutic needs of people living with this devastating disease remain unmet,” Leonard van den Berg, MD, PhD, professor of neurology at UMC Utrecht, in the Netherlands, and the study’s lead investigator, said in a press release.

“The initiation of this trial represents a critical milestone for Retrotope and our efforts to develop a novel treatment for ALS that has the potential to provide a meaningful therapeutic impact for patients battling this terrible disease,” said Peter Milner, MD, Retrotope’s chief medical officer.

RT001 is a stabilized version of an essential dietary fat called linoleic acid. It substitutes the naturally-occurring fatty acids found in cell and mitochondrial membranes and fortifies them against damage from unstable oxygen molecules known as free radicals.

Mitochondria are organelles found within the cell that produce energy for nearly all cellular processes. Damage to mitochondria is thought to be an early, if not initial, event in ALS.

An initial study demonstrated the safety and tolerability of RT001 among 23 participants who received the treatment for up to two years.

Following a petition from researchers at several major medical centers, Retrotope also made RT001 available to eligible ALS patients through an expanded access program, or compassionate use.

“Data that have been collected through our limited ALS expanded access program suggest that RT001 may play a role in slowing the progression of the disease and we are eager to test this hypothesis in a randomized, double-blind, placebo-controlled study,” Milner added.

“We look forward to enrolling patients in this trial and helping advance the evaluation of this novel treatment,” van den Berg said.