Compassionate Use Trials of RT001 in 2 Rare Neurodegenerative Disorders Initiated


Two single-patient, Compassionate Use trials for RT001 have been initiated for late onset Tay Sachs (LOTS) disease and familial encephalopathy with neuroserpin inclusion bodies (FEIN or neuroserpinosis).

Two single-patient, Compassionate Use trials for RT001 have been initiated by Retrotope for 2 neurodegenerative disorders, late onset Tay Sachs (LOTS) disease and familial encephalopathy with neuroserpin inclusion bodies (FEIN or neuroserpinosis), at major university medical centers, the clinical-stage pharmaceutical company Retrotope announced today.

RT001 is the first-in-class of a new category of drugs referred to as D-PUFAs (deuterated polyunsaturated fatty acids). D-PUFAs are engineered to protect against free radical damage that causes cell death, which is a primary characteristic of numerous neurodegenerative diseases, such as LOTS and FEIN.

“Early clinical results demonstrating the arrest or reversal of disease progression suggest that RT001 may have application in a number of diseases characterized by neuromuscular deficits,” Peter G. Milner, MD, Retrotope’s chief medical officer said in a recent statement. “Based on compelling safety and tolerability and promising clinical efficacy results to date, we are working closely with patient advocacy groups and physicians to support other single-patient or single-site Expanded Access trials, especially in neurodegeneration that exhibits bulbar, speech, neuromuscular, and cognition deficits.”

At the 2018 American Academy of Neurology meeting in Los Angeles, Retrotope presented positive clinical results of the use of RT001 in Infantile Neuroaxonal Dystrophy (INAD) and Friedreich’s ataxia, both of which share similarities like bulbar function losses, dysarthria, ataxia, and neuromuscular deficits with LOTS and FEIN. In the Friedreich’s ataxia trial with RT001, 18 out of 19 patients completed all safety and efficacy measurements with RT001 while in the case study of 1 INAD patient, improvement was observed after 1 year of RT001 treatment in the INAD patient.

Overall, the RT001 data in INAD and Friedreich’s ataxia demonstrated disease-modifying therapeutic effects (inhibiting progression with possible improvements) in these historically strictly progressive neurodegenerative diseases. Consequently, Retrotope is expanding its Compassionate Use programs and testing RT001 in other rare untreated neurological diseases that have similar symptoms or etiology.

“RT001 now has patient-years of safety in infants and young adults, as well as early signals of efficacy in two different neurodegenerative diseases,” said Robert J. Molinari, PhD, CEO and co-founder of Retrotope. “Expanding our trials based on this success to other related neurodegenerative diseases, initially in Expanded Access trials, allows us to investigate which diseases implicating lipid damage in cell death are most responsive to RT001 therapy. We plan to evaluate drug effect and endpoints in these new studies and characterize what we perceive as the broad therapeutic potential of D-PUFA drugs in neurodegeneration.”

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