Round co-led by existing inside investors and new outside family office

LOS ALTOS, Calif., July 19, 2019 (GLOBE NEWSWIRE) -- Retrotope announced today that it closed a Series C financing, enabling expansion of its pipeline of novel first-in-class drugs called D-PUFAs (deuterated polyunsaturated fatty acids). D-PUFAs are designed to protect against and reverse the effects of free radical damage to omega 3 and omega 6 fats in membranes resulting in cell dysfunction and death, hallmarks of multiple neurodegenerative diseases. The $20MM round was oversubscribed. Mehta Family Partners, an outside family fund, co-led the round with Timur Artemev, a UK-based inside investor (along with other insiders who re-invested). Also joining the round was Retrotope’s first institutional investor, Morningside Venture Investments, Ltd.

Robert J. Molinari, Ph.D., CEO and Founder of Retrotope, stated, “Based on the promise of robust clinical proofs of concept in multiple neurodegenerative diseases, we have now secured the funding needed to complete two pivotal trials, one each in Infantile Neuroaxonal Dystrophy and Friedreich’s ataxia. Our new investment round allows us to move beyond the typical mechanisms that have previously failed in degenerative diseases, and places a major bet on lipid peroxidation mitigation which represents a new frontier to restore cellular function and halt cell death in diseases that have previously been intractable.”

Nitin Mehta, the head of Mehta Family partners and a new investor to Retrotope, will join the Retrotope Board of Directors. Dr. Mehta commented, “We loved that the Retrotope team has stayed true to their vision that PUFA peroxidation is crucial to the onset and progression of multiple diseases, and have advanced a way to treat it into pivotal human trials. Retrotope’s novel solution to treat such diseases of degeneration and aging represent a disruptive force in a field that is in dire need of breakthrough. Their results in rare diseases thus far show a possibility to affect disease progression, an impressive feat, particularly in neurodegenerative diseases. These findings reflect changes that are of profound importance and potential value to pharmaceutical treatment also in many large diseases, such as Parkinson’s, Alzheimer’s and retinopathies, in which lipid peroxidation is implicated.”

Timur Artemev, a lead investor in this and prior Retrotope rounds, said “It’s been obvious for me for years that this novel approach to disease should work. Now the many demonstrated improvements from prior animal and human trials can be tested definitively in pivotal trials”.  

To date, Retrotope has enrolled two controlled clinical trials, a completed, blinded, randomized, Phase 1/2 in Friedreich’s ataxia (FA) 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. A third trial, a pivotal trial in FA, is expected to start in Q3 of 2019. The company also has accumulated over 250 patient-months of drug safety exposure across all clinical trial subjects and expanded access patients on drug.

About RT001
RT001 is a broadly patented, first-in-class, orally available D-PUFA that incorporates into mitochondrial and cellular membranes and stabilizes them against disease-induced, excess lipid peroxidation. 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 ALS, Alzheimer’s, Parkinson’s, retinopathies and host of other rare diseases. RT001 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, late onset Tay Sachs (LOTS), Huntington’s disease, and others are also underway. The presence of D-PUFAs (RT001) can help protect (“fireproof”) cells against disease and potentially restore cellular health and function.

About Retrotope
Retrotope, a privately held, clinical-stage pharmaceutical company, is creating a new category of patented 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. For more information about Retrotope, please visit www.retrotope.com.

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