Retrotope Announces RT001 Clinical Data Presentation at the 2019 American Academy of Neurology (AAN) Annual Meeting

Positive Findings from Expanded Access Trial in a Patient with Late Onset Tay Sachs (LOTS)

Los Altos, California, UNITED STATES

LOS ALTOS, Calif., May 06, 2019 (GLOBE NEWSWIRE) -- Retrotope announced today positive findings from an expanded access study of the company’s lead candidate, RT001, in a patient with LOTS, as presented at the 71st AAN Annual Meeting, being held in Philadelphia, PA, May 4-10, 2019. RT001 is the first in class of a new category of drugs called D-PUFAs (deuterated polyunsaturated fatty acids), which are designed to protect against free radical damage resulting in cell death, a hallmark of several degenerative diseases, including LOTS. The positive clinical results are being reported at the AAN meeting in a poster titled, “RT001 to Treat Neurodegeneration--Case Study: Improvement in a Single Patient with Late Onset Tay-Sachs Disease (LOTS)” (Poster # 3.8-018). The study showed that administration of RT001 to a 44-year-old LOTS patient appears to have generated improvements in a variety of patient reported outcomes (choking coughs, strength, falls) and in objective measures of performance (timed walks, quantified speech measures, Archimedes spiral). Effects were measured in daily or weekly diaries and test sessions at home. 

Peter G. Milner, M.D., Retrotope’s Chief Medical Officer, commented, “The results presented at AAN provide additional clinical evidence demonstrating that RT001 may be able to both stabilize disease progression in LOTS and help recover capabilities lost to the disease. These findings reflect changes that are of profound importance to the patient’s functioning and provide a critical early look at the promise of this innovative therapy.”

To date, Retrotope has enrolled two controlled clinical trials, a blinded, randomized, Phase 1/2 in Friedreich’s ataxia that showed unexpected post hoc statistical results in the treated group vs a placebo cohort in a measure of mitochondrial capability; and a pivotal trial in the ultra-rare fatal disease of children, Infantile Neuroaxonal Dystrophy, a genetic disease induced by low levels of functioning PLA2G6 lipid repair enzyme. The company also has accumulated over 150 patient-months of drug safety exposure across all clinical trial subjects and expanded access patients on drug.

Robert J. Molinari, Ph.D., CEO and Founder of Retrotope, stated, “Based on the promise of early and robust clinical proofs of concept, we are expanding our clinical pipeline to new areas of unmet medical need such as LOTS, ALS and others. The type of results presented in this poster validates what we perceive as the broad potential of D-PUFAs across neurodegenerative diseases.”

About LOTS
Early symptoms of Late Onset Tay-Sachs include clumsiness and muscle weakness in the legs. About 40% of affected adults experience mental health symptoms such as bi-polar or psychotic episodes. Over time adults with Late Onset Tay-Sachs slowly decline. Adults frequently require more mobility assistance, i.e. cane to walker to wheelchair. Many experience speech and swallowing difficulties but few require a feeding tube. Late-onset Tay-Sachs disease (LOTS) is a lysosomal storage disease caused by deficient beta-hexosaminidase A activity, and is also known as GM2 gangliosidosis.

About RT001
RT001 is a patented, first-in-class, orally available D-PUFA, a deuterated polyunsaturated fatty acid, that incorporates into mitochondrial and cellular membranes and stabilizes them. Retrotope and others have discovered that lipid peroxidation, the free-radical damage of polyunsaturated fats (PUFAs) in mitochondrial and cellular membranes, may be the primary source of cell death in several degenerative diseases, including LOTS. The presence of D-PUFAs (RT001) can help protect (“fireproof”) against this attack and potentially restore cellular health.

About Retrotope
Retrotope, a privately held, clinical-stage pharmaceutical company, is creating a new category of drugs to treat degenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being studied as disease-modifying therapies for many intractable diseases, such as Parkinson’s, Alzheimer’s, mitochondrial myopathies, and retinopathies. RT001, Retrotope’s first lead candidate, is being tested in clinical trials for the treatment of Friedreich's ataxia, a fatal orphan disease, and in a fatal, childhood neurodegenerative disease called Infantile Neuroaxonal Dystrophy. Expanded Access trials calibrating endpoint effects of RT001 in ALS, PSP Parkinsonism, GM1, LOTS, Huntington’s diseases, and others are also underway. For more information about Retrotope, please visit

SOURCE: Retrotope, Inc. 4300 El Camino Real, Suite 201 Los Altos, CA 94022 650-575-7551

For Media: Robert J Molinari, Ph.D.  CEO, 650 575 7551
For Patients: Sarah Endemann, Clinical Coordinator, 619 206 5944
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