Retrotope Presents Data Demonstrating Arrest of Disease Progression with Treatment of RT001 in Patients with Infantile Neuroaxonal Dystrophy (INAD)
- Category: Small Molecules
- Published on Tuesday, 16 October 2018 08:03
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Results presented at the 2018 National Organization for Rare Disorders (NORD) Rare Diseases & Orphan Products Breakthrough Summit®
LOS ALTOS, CA, USA I October 15, 2018 I Retrotope today reported new data that demonstrate arrest of disease progression with treatment of RT001 in patients with infantile neuroaxonal dystrophy (INAD). The data were presented at the National Organization for Rare Disorders (NORD), Rare Diseases & Orphan Products Breakthrough Summit® on October 15-16, 2018, in Washington, D.C.
“INAD is a rare and devastating disease that strikes the very young and there are no current available treatments,” said Peter G. Milner, M.D., Chief Medical Officer of Retrotope. “The two patients enrolled in an expanded access program and presented here have been treated with RT001 for more than one year and six months, respectively. Both show disease arrest and some improvement in several components contained in the INAD rating scale. In the meantime, we are preparing to initiate a pivotal study of RT001 in INAD patients with the objective of confirming its efficacy and safety in this patient population.”
To date, two INAD patients have been enrolled in expanded access programs, the first patient having initiated treatment in March 2017 and the second patient in November 2017. Promising results from the first patient were presented at the annual meeting of the American Academy of Neurology (AAN). Results from both patients were presented at NORD. RT001 has been granted U.S. FDA orphan drug designation for the treatment of PLA2G6-associated neurodegeneration (PLAN), which includes 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,” said Robert Molinari, Ph.D., Chief Executive Officer and Co-Founder of Retrotope. “For this reason, we are investigating RT001 in several neurodegenerative diseases. Aside from INAD, we have already presented clinical data in Friedreich’s ataxia (FA) and there are several ongoing investigator-sponsored expanded access programs in amyotrophic lateral sclerosis (ALS), progressive supranuclear palsy (PSP), late-onset Tay-Sachs disease (LOTS) and several others.”
INAD is an ultra-rare, devastating life-shortening neurodegenerative disorder that affects only a few hundred patients worldwide. It is caused by a genetic defect in the PLA2G6 housekeeping gene that removes damaged lipids from cells. 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.
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.
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 Infantile Neuroaxonal Dystrophy (INAD), ALS, Late Onset Tay Sachs (LOTS), Progressive SupraNuclear Palsy (PSP), Huntington’s disease, 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 fatal, neurodegenerative diseases such as INAD, in which a formal trial is about to start. For more information about Retrotope, please visit www.retrotope.com.