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resTORbio shuts down Phase 3 trial in clinically symptomatic respiratory illness

Published on 18/11/19 at 11:58am

US firm resTORbio has revealed its intention to terminate a Phase 3 trial of its oral, selective TORC1 inhibitor RTB101 after the candidate failed to meet its primary endpoint in the treatment of clinically symptomatic respiratory illness (CSRI) in patients over 65.

The study examined the efficacy of RTB101 taken once a day for 16 weeks throughout the winter cold and flu season. In total, 1,024 over-65s were examined, excluding current smokers and people with chronic obstructive pulmonary disease (COPD).

Ultimately, patients receiving the therapy were shown to present a CSRI risk of 0.46 compared to 0.44 with placebo, failing the study’s primary endpoint of reduction in percentage of patients with CRSI, as defined as an illness associated with a respiratory tract infection (RTI) based on specific diagnostic criteria.

Despite this failure, the company confirmed it plans to continue development of the therapy in other therapeutic areas, including Parkinson’s disease.

“While we are disappointed in these results, there are extensive preclinical data supporting the potential therapeutic benefit of TORC1 inhibition in multiple ageing-related diseases, including Parkinson’s disease, for which we have an active Phase 1b/2a trial of RTB101 alone or in combination with sirolimus,” explained Chen Schor, co-Founder, President and Chief Executive Officer of resTORbio.

He continued: “Multiple preclinical models have demonstrated that inhibition of TORC1 decreases protein and lipid synthesis, increases lysosomal biogenesis and stimulates the clearance of misfolded protein aggregates, such as toxic synucleins, that cause neuronal toxicity in Parkinson’s disease. We remain committed to exploring the potential benefits of TORC1 inhibition in patients, and we look forward to the data from our Parkinson’s disease trial, which we expect in mid-2020.”

Matt Fellows

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