Retrotope announced today that it has begun enrollment of the first patients and has started dosing of infants with the ultra-rare neurodegenerative disease, Infantile Neuroaxonal Dystrophy (INAD).

Parkinson’s Institute and Clinical Center, which provides comprehensive patient care, while discovering new treatment options to improve the quality of life for all Parkinson’s disease (PD) patients, and Retrotope announced today a collaboration on a new drug therapy to treat patients with Progressive SupraNuclear Palsy (PSP), a type of treatment-resistant PD.

Retrotope today announced that it has supplied its drug RT001 for the initiation of an expanded access (EA) trial to treat patients with amyotrophic lateral sclerosis (ALS). This EA program in ALS, requested by renowned investigators at major medical centers, is expected to enroll a few patients at each site and is intended to provide information to guide the design of future randomized placebo-controlled trials.

Retrotope and collaborators led by Dr. Marie-Francoise Chesselet, the Charles H. Markham Professor of Neurology, and Interim Chair of the Department of Neurology at UCLA, have published a paper demonstrating that the Company’s D-PUFA drug candidate, RT001, reduces cognition deficits in a preclinical in vivo model of Huntington’s disease, while simultaneously downregulating brain lipid peroxidation biomarkers. The paper, entitled “Deuterium‐ reinforced linoleic acid lowers lipid peroxidation and mitigates cognitive impairment in the Q140 knock in mouse model of Huntington's disease,” was published in The FEBS Journal.

Retrotope announced today that the U.S. Food and Drug Administration (FDA) has granted the company 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.

Retrotope announced today the start of two single-patient, Compassionate Use trials of RT001 at major university medical centers in Late Onset Tay Sachs (LOTS) disease and Familial Encephalopathy with Neuroserpin Inclusion Bodies (FEIN or neuroserpinosis). 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 LOTS and FEIN.

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.

Retrotope announced today peer-reviewed publication of positive Phase 1b/2a trial results for the company’s lead candidate, RT001, in patients with Friedreich’s ataxia (FA). 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 FA. In an article titled, “Randomized, Clinical Trial of RT001: Early Signals of Efficacy in Friedreich’s Ataxia”