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Press Release

Retrotope Announces RT001 Clinical Data Presentation at the 2018 American Academy of Neurology (AAN) Annual Meeting

Posted on: 24 Apr 18

Positive Findings from Compassionate Use Study in Patient with Infantile Neuroaxonal Dystrophy (INAD) Presented

LOS ALTOS, Calif., April 24, 2018 (GLOBE NEWSWIRE) -- Retrotope announced today positive findings from a compassionate use study of the company’s lead candidate, RT001, in a patient with INAD, as presented at the 70th AAN Annual Meeting, being held in Los Angeles, CA, April 21-27, 2018. 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 several degenerative diseases, including INAD. The positive clinical results are being reported at the AAN meeting in a poster titled, “Case Report: Expanded Access Treatment of an Infantile Neuroaxonal Dystrophy (INAD) Patient with a Novel, Stabilized Polyunsaturated Fatty Acid Drug” (Poster No. P3.327). The study showed that administration of RT001 to a ~2.5-year-old patient appears to have arrested disease progression and led to re-acquisition of lost developmental milestones. Specifically, after 2 months on drug, the patient regained sufficient bulbar function to terminate syringe feeding of liquids and returned to drinking from a child’s cup.  Periodic videotaped therapy sessions or exams over 12 months showed the patient improved in qualitative measures, including eye tracking, responsiveness to verbal commands, head control, lifting, and reaching and grasping (previously lost skills).

“The INADcure Foundation is encouraged by the results seen in previous preclinical research and the benefits more recently observed in children under an Expanded Access program,” stated Leena Panwala, INADcure’s Co-founder and President ( “Many INAD families have feared that they would never see an opportunity for a clinical trial in their child’s lifetime. We are very excited with the potential of this program progressing into formal clinical testing.”

Peter G. Milner, M.D., Retrotope’s Chief Medical Officer, commented, “The results presented at AAN provide the first clinical evidence demonstrating that RT001 may be able to both stabilize disease progression in INAD and help recover lost developmental milestones. These findings reflect profoundly important changes in functioning and provide a critical early look at the promise of this innovative therapy in meeting compelling patient needs in INAD.”

To date, Retrotope has enrolled two patients in compassionate use studies, the first patient having completed a full year of treatment and the second patient having enrolled in November 2017. Under the compassionate use protocols, the patients have received daily oral dosing of RT001, with videotaped exams scheduled at baseline, 1 month, 3, 6, 9, and 12 months.

Robert J. Molinari, Ph.D., CEO and Founder of Retrotope, stated, “Based on the promise of the INAD compassionate use program, we have expanded our clinical pipeline to an area of compelling unmet patient needs beyond our current clinical program in Friedreich’s ataxia (FA).  This speaks to what we perceive as the broad potential of D-PUFAs, and we look forward to the progress of these programs.”

About INAD
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.

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 FA and 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 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 a fatal, childhood neurodegenerative disease called Infantile Neuroaxonal Dystrophy.  For more information about Retrotope, please visit

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 650-575-7551


Last updated on: 24/04/2018

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