Retrotope Announces Completion of Enrollment in Phase 2 Study of RT001 in Patients with Progressive Supranuclear Palsy (PSP)

Target Enrollment for Study Surpassed in Less Than Six Weeks

Los Altos, California, UNITED STATES

LOS ALTOS, Calif., Aug. 11, 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 progressive supranuclear palsy (PSP). The study’s enrollment target of 40 patients was exceeded in less than six weeks, highlighting the PSP patient community’s significant interest in participating in a clinical trial of a novel, oral treatment approach to this devastating neurodegenerative disease.

“PSP is a progressive, neurodegenerative disorder that dramatically impacts the health and lives of not only patients, but also their families and other caregivers. With no effective treatment options available for PSP, a tremendous need exists for the development of novel therapeutic options such as RT001,” said Günter U. Höglinger, Ph.D., director of the department of neurology, Hannover Medical School, Germany. “In my experience, the rate of enrollment in this study is unprecedented in the area of PSP, which I believe underscores the commitment of patients with PSP and researchers to advance new treatments with disease modifying potential.”

The Phase 2 trial was conceived after cell culture research and clinical experience demonstrated compelling evidence of a beneficial effect of RT001 in PSP.1 The current trial is a randomized, double-blind, placebo-controlled study evaluating the efficacy, long-term safety and tolerability of RT001 in patients with PSP. Study participants have been randomized to receive either RT001 or placebo daily for 48 weeks. The primary endpoint of the trial is change from baseline in the PSP Rating Scale (PSPRS) at 48 weeks. The PSPRS, which is a clinician-rated quantitative measure of disease severity for patients with PSP, is comprised of 28 items spanning the following six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline. Additionally, study investigators will also evaluate the efficacy of RT001 using a second PSP rating scale suggested by the United States Food and Drug Administration (FDA). The study also includes several secondary and exploratory endpoints intended to further elucidate the efficacy and safety profile of RT001 as compared to placebo.

"The rapid enrollment of patients into this clinical trial demonstrates how motivated PSP patients and physicians are to find a treatment for this incurable disease,” said Anne-Marie Wills, M.D. M.P.H., director of the CurePSP Center of Care, and assistant professor of neurology, Harvard Medical School Movement Disorders Clinic at the Massachusetts General Hospital. “We in the field will be very interested to learn the results of this clinical trial."

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 now well-established that a number 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. To date, RT001 has been administered to four PSP patients through expanded access, with three of these having received treatment for at least 27 months. For these patients, disease progression significantly slowed or reversed at 12 months and 24 months according to standard severity scales used in the measurement of the disease, including the PSPRS. Retrotope seeks to build upon these promising initial data with its ongoing Phase 2 PSP trial.

RT001 has been granted orphan drug designation by the FDA for the treatment of PSP.

“Our ongoing PSP study is the latest example of Retrotope’s ability to rapidly reach and exceed enrollment targets for clinical trials of RT001 in devastating neurodegenerative diseases. Similar to our ongoing Phase 2 trial of RT001 in amyotrophic lateral sclerosis or ALS, this PSP study surpassed the original enrollment goal in less than six weeks,” said Mark G. Midei, M.D., Retrotope’s vice president for medical affairs. “We believe that the high level of interest for participation in these studies is a direct reflection of the dramatic need for new treatments in the areas of PSP and ALS. This need, combined with the unique mechanism of action for RT001 and its oral route of administration, has generated excitement among our clinical trial investigators and their patients.”   

For more information about the PSP study, including a list of trial sites and contacts, please visit (Identifier: NCT04937530).

About PSP

Progressive Supranuclear Palsy (PSP) is a progressive neurodegenerative disease that results in impaired balance, difficulty with eye movement, speech, and swallowing, rigid muscle tone and ultimately, cognitive impairment. The condition, which typically strikes individuals between the ages of 60 and 70, is associated with deterioration and death of specific cells within brain responsible for balance/coordination, walking, speech, swallowing, eye movement and cognition. PSP affects approximately five-to-six people out of every 100,000 and is commonly misdiagnosed as other neurodegenerative diseases such as Parkinson’s disease and Alzheimer’s disease. There are currently no effective treatments for PSP, though some symptoms of the disease can be managed therapeutically.

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


1Angelova 2021 DOI:


Contact Data