Retrotope drug improves survival in ultra-rare childhood genetic diseases

Retrotope, based in Los Altos, California, cited data of its Phase II / III trial of RT001 in patients with infantile neuroaxonal dystrophy (INAD). They also reported on a concurrent study of the natural history of disease onset and progression in INAD patients.

INAD is an ultra-rare, progressive, fatal childhood genetic neurological disease. It’s on a spectrum of diseases called neurodegeneration associated with PLA2G6. A congenital deficiency of the PLA2G6 enzyme results in an inability to effectively remove lipid peroxidation (LPO) byproducts, which are toxic. This results in a “death signal” which causes the rapid death of neuronal cells in the brains of developing infants.

Alex Fay, Assistant Professor of Neurology, University of California, San Francisco and one of the study’s lead investigators, noted, “INAD is an ultra-rare, relentlessly progressive and fatal disease that currently has no approved treatment. This trial compared a group of children with INAD treated with RT001 with a natural history cohort, and the results indicate that RT001 slows progression and prolongs survival compared to no treatment. The results are striking and suggest that RT001 has the potential to have a significant impact on the neurological function and survival of children with INAD.

RT001 is an isotopically stabilized synthetic linoleic acid (LA) that Retrotope discovered and developed using its technology platform, which fights oxidative stress and cellular degeneration associated with lipid peroxidation (LPO). Polyunsaturated fatty acids (PUFAs) make up cell and mitochondrial membranes. The LPO process targets PUFAs. Retrotope’s technology generates stabilized PUFAs, such as RT001, which integrate into all membranes and down-regulate LOP to protect membranes from degeneration.

The clinical trial included 19 INAD patients and the concomitant natural history of 36 INAD patients as a control group. There were no significant differences between the baseline characteristics in the two cohorts.

Patients in the treatment cohort received RT001 for at least one year with 30-day treatment-free follow-up. Patients receiving RT001 demonstrated a 6.42-point improvement on the modified Ashworth Spasticity Scale compared to the control cohort. Similar therapeutic benefits were observed for the RT001 cohort compared to controls on all five efficacy outcome scales.

Progression-free survival (absence of pneumonia or death) was a predefined secondary endpoint. The RT001 group demonstrated a statistically significant reduction of 82.5% in the risk of morbidity compared to controls. A predefined exploratory endpoint of survival showed a statistically significant reduction in the risk of mortality of 88.8%. Confirmed deaths were “significantly lower” in patients receiving RT001 compared to the control group, 11% vs. 31%, respectively.

“We are very happy with these first results and delighted to meet the FDA to discuss the next steps in our continued efforts to bring RT001 to the INAD patient community, ”said Peter G. Milner, Medical Director of Retrotope. “With clear evidence of a benefit in patient survival, as well as consistent improvements observed in all efficacy endpoints analyzed, including those that measured vital function and activities of daily living, we believe that Results of this study support the potential of RT001 to address a critical unmet need in the patient population with orphan diseases.

Retrotope a In progress trials for RT001 in amyotrophic lateral sclerosis (ALS), progressive supranuclear palsy (PSP) and Friedreich’s ataxia (FA), three of the four most common orphan neurodegenerative diseases. According to Clinical Trials Arena, the company is targeting a funding round in the first quarter of 2022 to raise $ 100 million.

On August 11, the company indicated that he had completed recruitment for his Phase II study of RT001 for progressive supranuclear palsy (PSP). PSP is a progressive neurodegenerative disease causing impaired balance, problems with eye movement, speech and swallowing, stiff muscle tone and ultimately cognitive impairment. It usually affects people between the ages of 60 and 70. It is often misdiagnosed as Parkinson’s disease and Alzheimer’s disease.

April 21, Retrotope completed recruitment of its Phase II trial of RT001 in ALS (Lou Gehrig’s disease). They exceeded the goal of recruiting 40 patients in less than six weeks. They expect data from the trial by the end of the year.

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