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Retrotope Receives FDA Clearance to Commence RT001 Phase 2/3 Clinical Trial
in Patients with Infantile Neuroaxonal Dystrophy (INAD)
The U.S. Food and Drug Administration (FDA) has granted Retrotope approval to conduct an open-label Phase 2/3 clinical trial of its investigational drug RT001 to evaluate efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD). 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 that is a hallmark of numerous neurodegenerative diseases including INAD. To date, Retrotope has enrolled two INAD patients in two separate Expanded Access trials, the first patient having begun treatment in March 2017 and the second patient having enrolled in November 2017. Promising results from the first study were presented at the recent annual meeting of the American Academy of Neurology, and treatment of both children remains ongoing. RT001 has been granted U.S. FDA orphan drug designation for the treatment of PLA2G6 associated neurodegeneration (PLAN), which includes INAD.
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, INAD, ALS, Friedreich’s ataxia (FA), Late Onset Tay Sachs (LOTS), Familial Encephalopathy with Neuroserpin Inclusion Bodies (FEIN or neuroserpinosis), mitochondrial myopathies, and retinopathies. RT001, Retrotope’s first lead candidate, is being tested in clinical trials for the treatment of FA, a fatal orphan disease, and in compassionate use studies for the fatal, neurodegenerative diseases such as INAD, LOTS, FEIN, and a genetic form of Alzheimer’s disease. For more information about Retrotope, please visit www.retrotope.com.
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 (“fireproof”) against this attack and potentially restore cellular health.