Exhibit 99.1
resTORbio Announces Initiation of Phase 3 Clinical Program of RTB101 in Clinically
Symptomatic Respiratory Illness
Top-line data from both PROTECTOR Phase 3 studies expected inmid-2020
BOSTON, Massachusetts, May 9, 2019 – resTORbio, Inc. (Nasdaq: TORC), today announced the initiation of PROTECTOR 1, the first Phase 3 trial of RTB101, an orally administered, small molecule, potent inhibitor of target of rapamycin complex 1 (TORC1). The two PROTECTOR Phase 3 trials are designed to evaluate the safety and efficacy of RTB101 for decreasing the percent of elderly subjects with clinically symptomatic respiratory illness, defined as illness associated with a respiratory tract infection (RTI) based on prespecified diagnostic criteria, with or without laboratory-confirmation of a pathogen.
“The launch of our PROTECTOR Phase 3 program is an important milestone for resTORbio and for the elderly population, as RTB101 has the potential to be the first immunotherapy for reducing the incidence of illness associated with RTIs, regardless of the causative pathogen,” said Chen Schor,Co-Founder, President and CEO of resTORbio. “The majority of RTIs are caused by viruses, and the results of our Phase 2 trials suggest that upregulation of innate antiviral gene expression is a mechanism underlying the reduced incidence of RTIs observed in elderly subjects treated with RTB101 compared to placebo. We plan to enroll approximately 2,600 subjects in our Phase 3 trials, and believe this program will provide us with sufficient clinical data to support the submission of a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for RTB101.”
The PROTECTOR Phase 3 program includes two randomized, double-blinded, placebo-controlled clinical trials that will evaluate the safety and efficacy of RTB101 10mg given once daily for 16 weeks during winter cold and flu season to subjects 65 years of age and older, excluding current smokers and chronic obstructive pulmonary disease patients. The primary endpoint is the reduction in the percentage of subjects with clinically symptomatic respiratory illness with or without laboratory-confirmation of a pathogen.
The PROTECTOR program incorporates feedback from both the FDA and European Medicines Agency on key elements of the Phase 3 trial design. PROTECTOR 1, initiated in the southern hemisphere, is expected to enroll approximately 1,000 subjects. PROTECTOR 2, the second Phase 3 clinical trial, is expected to begin in the northern hemisphere in the fourth quarter of 2019 and to enroll approximately 1,600 subjects. Based on current enrollment expectations for the Phase 3 program, resTORbio expectstop-line data inmid-2020.
About Respiratory Tract Infections in the Elderly
As part of the aging process, the immune system weakens and becomes less effective at detecting and fighting infections such as RTIs. As a result, RTIs are more likely to be of greater severity, prolonged duration, and are more likely to be associated with medical complications in people age 65 years and older as compared to younger adults. In the U.S., RTIs are the fourth leading cause of hospitalization and seventh leading cause of death in people aged 65 and older. Given that RTIs are caused by many different types of viruses, most of which lack effective therapies, there remains a significant unmet medical need for an immunotherapy that enhances the ability of the immune system to fight multiple viruses to reduce illness associated with RTIs in the elderly.
About RTB101
RTB101 is an oral, selective, and potent TORC1 inhibitor product candidate. TORC1 inhibition has been shown to be of therapeutic benefit in multiple aging-related conditions in preclinical species including immunosenescence (aging-related decline in immune function). In two Phase 2 clinical trials enrolling over 900 elderly people, RTB101 was observed to improve immune function by upregulation of antiviral gene expression and to reduce the incidence of RTIs.
