Retrotope Announces Completion of Enrollment in Phase 2 Study of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS)

Company Exceeds Target Enrollment in Less Than Six Weeks; Data Readout Expected by End of 2021

Los Altos, California, UNITED STATES

LOS ALTOS, Calif., April 21, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today announced that enrollment has been completed for its multicenter Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease). The study’s enrollment target of 40 patients was exceeded in less than six weeks, highlighting the high demand for participation in the trial featuring a novel treatment approach for ALS. Based on this rapid patient enrollment, Retrotope expects data from the trial to be available by the end of 2021.

"Patients living with ALS are in need of new and improved treatment options for this devastating disease," said Caroline Ingre, M.D., Ph.D., head of the ALS Center at Karolinska Institute in Stockholm and a clinical investigator for the ongoing RT001 ALS study. "This trial enrolled very rapidly due to the enthusiasm of ALS patients for a new potential treatment with a unique mechanism for combatting this disease.  We look forward to assessing the results of this investigation later this year."
The Phase 2 trial is a randomized, double-blind, placebo-controlled study evaluating the efficacy, safety and tolerability of RT001 in patients with ALS. Study participants have been randomized to receive either RT001 or placebo daily for 24 weeks. The primary endpoint of the trial is change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) at 24 weeks. Several additional secondary and exploratory endpoints will also be evaluated to further elucidate the efficacy and safety profile of RT001 as compared to placebo.

“The global ALS patient community remains in desperate need of effective treatments for ALS. Clinical trials like the ongoing study of RT001 from Retrotope, are essential if we are to significantly impact ALS progression or symptoms,” said Ammar Al-Chalabi, Ph.D. FRCP, professor of neurology and complex disease genetics at King’s College London and director of the MND Association-funded King’s MND Care and Research Centre.

RT001 is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) discovered and developed with Retrotope’s novel platform technology. This platform is designed to combat the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). While all healthy human tissues undergo this physiological process of cell degeneration and repair, it is well-established that a wide range of serious degenerative diseases are precipitated when the LPO process becomes out of balance. Polyunsaturated fatty acids (PUFAs), which make up cell and mitochondrial membranes throughout the body and are vital to healthy cellular function, are the target of the LPO process due to their inherent instability. Free radicals in the body exploit the instability of PUFAs to trigger chain reactions that drive LPO and the resulting degradation of these vital PUFAs. Retrotope’s novel platform technology creates stabilized PUFAs, such as RT001, that become an integral part of all membranes and are capable of down-regulating LPO in order to protect membranes from degeneration.

RT001, which is currently being evaluated in clinical trials across several neurodegenerative diseases, has been safely administered orally on a daily basis to more than 100 patients, spanning more than 1,000 patient months. This includes the company’s initial clinical research on RT001 in ALS, which comprised treatment of 23 patients for up to 24 months and demonstrated both safety and potential signs of disease slowing. Retrotope seeks to build upon these promising initial data in ALS with its ongoing Phase 2 ALS trial.

“Retrotope is grateful for the confidence shown by investigators and patients for this clinical trial of RT001 to treat ALS. We believe that the interest of the ALS community has been driven by the existing unmet medical need and the unique attributes of RT001,” said Mark G. Midei, M.D., Retrotope’s vice president for medical affairs. “These include RT001’s ability to prevent the oxidative stress and cellular degeneration that arises from lipid peroxidation, all of which appear to play a role in the fundamental degenerative processes involved in the onset and progression of ALS.  Furthermore, as an oral drug, RT001 offers patients significant convenience and administration advantages over other potential treatments that require injections or infusions.”

For more information about the study, including a list of trial sites and contacts, please visit (Identifier: NCT04762589).

About ALS
Amyotrophic lateral sclerosis (ALS) 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 breathe 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.   

About Retrotope

Retrotope is a clinical-stage biopharmaceutical company focused on the development of first-in-class therapies for degenerative diseases ranging from orphan neurodegenerative indications to large market degenerative conditions. The company leverages its proprietary drug discovery platform to create novel, disease-modifying drugs designed to combat the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). It does so through the creation of isotopically stabilized synthetic versions of polyunsaturated fatty acids (PUFAs) that trigger the downregulation of the LPO process. The company’s lead development candidate, RT001, is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) that is in development for a range of orphan neurodegenerative diseases, including infantile neuroaxonal dystrophy (INAD), Friedreich’s ataxia (FA), amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and progressive supranuclear palsy (PSP). In addition, the company is advancing its second development candidate, RT011, an isotopically stabilized, synthetic docosahexaenoic acid (DHA), toward the clinic for the treatment of dry age-related macular degeneration (AMD).

For more information, please visit