Retrotope Announces Full Enrollment of Phase 2/3 trial in patients with Infantile Neuroaxonal Dystrophy (INAD)

Study expected to read out by Q3, 2020

Los Altos, California, UNITED STATES

LOS ALTOS, Calif., Aug. 07, 2019 (GLOBE NEWSWIRE) -- Retrotope announced today that it has fully enrolled all patients in the first clinical trial of a possible treatment for infants with the ultra-rare neurodegenerative disease, Infantile Neuroaxonal Dystrophy (INAD).  The U.S. Food and Drug Administration (FDA) has granted the company approval to conduct this trial as an open-label Phase 2/3 clinical trial in 15-20 patients.  With FDA guidance Retrotope is currently also enrolling a prospective natural history study using the same evaluation metrics in a matched group of INAD patients who are not participating in the trial.  RT001, the test drug, 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 to omega 3 and 6 fats that results in cell death and is a hallmark of numerous neurodegenerative diseases including INAD.

Retrotope previously enrolled two INAD patients in individual Expanded Access trials, the first patient of which has been on drug over 2 years.  RT001 has been granted orphan drug designation in the US and Europe for the treatment of PLA2G6 associated neurodegeneration (PLAN) and INAD; respectively.

“The Phase 2/3 clinical trial is an important milestone in the development pathway of RT001” commented Harry J, Saal, Executive Chairman of Retrotope. “INAD is a pure form of neurodegeneration with many symptoms in common with many other intractable neurological diseases. Preclinical models suggested that RT001 could reduce high levels of lipid peroxidation induced cell death in PLA2G6-deficient Drosophila and human fibroblast models.  We are hopeful for patients that this effect can provide a first approved treatment for this disease of inexorable decline”.

Leena Panwala, President and Co-founder of INADcure (, noted, “INAD is a devastating disease for which there is currently no treatment. We are excited that RT001 is in formal clinical testing so that its effects can be more thoroughly investigated.” The INADcure Foundation is a 501(c)(3) nonprofit charity whose mission is to support the development of treatments, including a cure, for Infantile Neuroaxonal Dystrophy (INAD) and other forms of PLA2G6-related neurodegeneration (PLAN). 

Peter G. Milner, MD, Retrotope’s Chief Medical Officer, commented, “This disease broadly affects all aspects of neurological control systems, including fine motor, gross motor, bulbar, ocular, and fronto-temporal systems.  Our customized evaluation scale for this trial has adapted measures from well-known infantile development capability testing to assess this disease.   If we halt progression in a meaningful way or have positive effects in several of these areas, we believe we can help these patients.”

About INAD

INAD is an ultra-rare, devastating life-shortening neurodegenerative disorder that affects at most only a few hundred patients worldwide. It is caused by a genetic defect in the PLA2G6 housekeeping gene that removes damaged lipids from cell membranes, resulting in damaged lipids accumulating in membranes and causing further damage. Infants with INAD appear to develop normally until approximately 6 to 18 months of age, when they begin to experience progressive mental and psychomotor development declines as they lose developmental milestones. Later-stage disease typically involves a feeding tube and breathing via ventilator. Life-threatening complications typically develop by the end of the first decade.

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, complemented by a vast body of independent research of others, has  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 INAD. 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 in several other diseases in which lipid peroxidation damage has been reported, such as Parkinson’s, INAD, ALS, Friedreich's ataxia (FA), Late Onset Tay Sachs (LOTS), mitochondrial myopathies, and retinopathies.  RT001, Retrotope’s first lead candidate, is being tested in a phase 2/3 study in INAD with plans for a pivotal trial in FA. For more information about Retrotope, please visit

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