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

First-in-Class, Oral Drug Candidate to be Evaluated in Randomized, Double-Blind, Placebo-Controlled Study in Patients with Debilitating Neurodegenerative Disease

Los Altos, California, UNITED STATES

LOS ALTOS, Calif., June 30, 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 the first patient has been dosed in a multicenter Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with progressive supranuclear palsy (PSP). Clinical investigators have already reported significant patient interest for participation in the trial and expect enrollment to be completed rapidly.

The Phase 2 trial is a randomized, double-blind, placebo-controlled study evaluating the efficacy, long-term safety and tolerability of RT001 in patients with PSP. Study investigators will enroll and randomize approximately 40 patients 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.

“While not as widely known as some other progressive neurodegenerative diseases, such as ALS and Alzheimer’s disease, PSP is devastating to patients and their families and the need for effective treatments is just as critical,” said Stefan Lorenzl, M.D., Ph.D., professor of neurology, chair of the department of neurology at Agatharied Teaching Hospital of the Ludwig Maximilians University of Munich and the principal investigator of the study. “We believe that there is promise in the potential of modifying the course of PSP by mitigating the oxidative stress and cellular degeneration that is caused by pathogenic LPO. This is supported by data from expanded access use of RT001 in this patient population, which, while from a small number of patients, suggest that the treatment may provide meaningful benefit. We are excited to be part of this important clinical study and look forward to evaluating the potential of RT001 in this disease.”

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. 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 newly initiated Phase 2 PSP trial.

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

“With this latest trial initiation, we now currently have four clinical studies of RT001 in Phase 2 or later across a range of orphan neurodegenerative diseases. This breadth of clinical research is a testament to the fundamental role that we believe the targeted down-regulation of LPO can play in staving off the cellular degeneration that is a hallmark of these diseases and, in turn, providing meaningful benefit to patients suffering with these conditions,” said Mark G. Midei, M.D., Retrotope’s vice president for medical affairs. “A further indication of support for this novel therapeutic approach within the scientific and patient communities is the speed with which we have been able to enroll patients in our clinical trials. We recently exceeded the target enrollment for our ongoing Phase 2 study in ALS in less than six weeks and expect enrollment in this PSP trial to follow a similarly rapid timeline.”

For more information about the 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).

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