Retrotope Announces Publication of Preclinical Research Showing Cognitive Improvements in a Huntington’s Disease Model Using Deuterated Polyunsaturated Fatty Acid (D-PUFA) Drug Candidate

Los Altos, California, UNITED STATES

LOS ALTOS, Calif. and LOS ANGELES, July 09, 2018 (GLOBE NEWSWIRE) -- Retrotope and collaborators led by Dr. Marie-Francoise Chesselet, the Charles H. Markham Professor of Neurology, and Interim Chair of the Department of Neurology at UCLA, have published a paper demonstrating that the Company’s D-PUFA drug candidate, RT001, reduces cognition deficits in a preclinical in vivo model of Huntington’s disease, while simultaneously downregulating brain lipid peroxidation biomarkers.  The paper, entitled “Deuterium‐reinforced linoleic acid lowers lipid peroxidation and mitigates cognitive impairment in the Q140 knock in mouse model of Huntington's disease,” was published in The FEBS Journal.   A copy of the abstract may be found on The FEBS Journal website:  This work was funded in part by a Fox Family Foundation grant to the Hereditary Disease Foundation (HDF), a non-profit research foundation which funds innovative genetic research towards curing Huntington's disease and other brain disorders.

The study’s results demonstrate that Retrotope’s deuterated polyunsaturated linoleic acid drug candidate, RT001, rescues Q140 knock out mice from cognition deficits, while simultaneously downregulating brain lipid peroxidation markers.  These data, together with prior publications of clinical and preclinical results, support the notion that mitigating lipid peroxidation using Retrotope’s deuterated polyunsaturated fatty acid (D-PUFA) drugs can be a disease-modifying therapy for multiple intractable neurodegenerative diseases. 

Dr. Mikhail Shchepinov, CSO and co-founder of Retrotope, commented: “Lipid peroxidation has been implicated in many neurodegenerative diseases for decades, but has never been successfully addressed therapeutically.  It is now evident from improvements noted in this Huntington’s model, a placebo-controlled Phase 1/2 disease clinical trial in Friedreich’s ataxia, continuing improvement in two Expanded Access subjects with the strictly progressive, fatal disease, Infantile Neuroaxonal Dystrophy, and dozens of specific animal and cell models of diseases, that lipid peroxidation is a validated, druggable target across many diseases of neurodegeneration.” 

Dr. Robert Molinari, CEO of Retrotope adds: “This Huntington’s disease model now adds to multiple models of major and rare neurodegenerative diseases in which disease-modifying effects have been observed using D-PUFA drug candidates, including improvements in cognition and motor skills. Also, due to the essential role of PUFAs in membrane lipid remodeling essential for healthy cells, D-PUFAs are everywhere accessible to tissues that need them—an otherwise key obstacle in neurodegeneration.  It’s time for the neurology community to embrace this new target class and drug strategies that effectively address it.”

About D-PUFAs
D-PUFAs are patented, orally available, modified fatty-acid therapeutics that stabilize (“fireproof”) mitochondrial and cellular membranes against attack and restore cellular health. Retrotope and others have discovered that lipid peroxidation, the free-radical degradation of fatty acids in mitochondrial and cellular membranes, may be the primary source of cell death in several degenerative diseases.

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 Friedreich’s Ataxia (FA) and INAD. The presence of D-PUFAs (RT001) can help protect 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 compassionate use studies for INAD. For more information about Retrotope, please visit .

About the Hereditary Disease Foundation

The Hereditary Disease Foundation’s Mission is to fund innovative research towards curing Huntington’s disease and impacting other brain disorders.  The Hereditary Disease Foundation pioneered many technologies for mapping and finding genes. A decade later the specific Huntington’s disease gene was identified, unlocking critical knowledge needed to find the cure.  The Foundation focuses on curing Huntington’s disease, not only because of its devastating consequences to individuals and families with the disease, but because it is a model for curing other brain disorders like Parkinson's, Alzheimer's and Lou Gehrig's (ALS) diseases.

Retrotope Media Contact:

Justin Jackson, Burns McClellan 212-213-0006, ext. 327,

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