resTORbio Announces Delay of its Ongoing Phase 1b/2a trial of RTB101 in Patients with Parkinson’s Disease Due to COVID-19 Level 4 Alert in New Zealand
“We want to ensure the safety of our clinical trial participants, many of whom are older adults at high risk of COVID-19 morbidity and mortality. Therefore, we have postponed enrollment in the fifth cohort of our ongoing Phase 1b/2a clinical trial of RTB101 in patients with PD. Enrollment in the first four cohorts has been completed, and we are actively monitoring the situation in
Phase 1b/2a Trial of RTB101 alone and In combination with sirolimus in Parkinson’s disease study results
The multicenter, 2:1 randomized, double-blind, placebo-controlled Phase 1b/2a trial is evaluating the safety and tolerability of RTB101 alone or in combination with escalating doses of sirolimus (2 mg, 4 mg and 6 mg) once weekly for 4 weeks in patients with Parkinson’s disease. To date, patients have been enrolled in four cohorts and dosed once weekly with 300 mg of RTB101 alone, 2 mg of sirolimus alone, a combination of 300 mg RTB101 and 2 mg of sirolimus, or a combination of 300 mg RTB101 and 4 mg of sirolimus. Results of the interim study analysis after the first 3 cohorts indicated that all 3 dosing regimens were well tolerated and RTB101 300 mg once weekly was observed to cross the blood brain barrier. The concentrations of RTB101 in cerebrospinal fluid (CSF) in subjects dosed with RTB101 300 mg once weekly monotherapy were higher than expected and based on preclinical models, have the potential to induce autophagy in the brain. Sirolimus at the dose of 2 mg, alone or in combination with RTB101, was not detected in the CSF. Data from the first three cohorts in the study suggest that the concentrations of RTB101 observed in the CSF four hours after dosing were highest when RTB101 was given as a monotherapy. Enrollment and dosing of the RTB101 300 mg in combination with sirolimus 4 mg once weekly cohort has been completed.
About Parkinson’s Disease
Parkinson’s disease (PD) is a progressive neurodegenerative disease that affects approximately 7.5 million people worldwide. The incidence of PD increases rapidly in people 60 years of age and older, with a mean age at diagnosis of 70.5 years. Inhibition of TORC1 has been shown in multiple preclinical models to ameliorate neurodegenerative diseases including Parkinson’s disease. TORC1 inhibition with RTB101 alone or in combination with sirolimus, may provide a therapeutic benefit to PD patients by ameliorating levodopa-induced dyskinesia and/or by inducing autophagy which leads to the breakdown of protein aggregates and improved neuronal survival.
RTB101 is an oral, selective, and potent TORC1 inhibitor product candidate that inhibits the phosphorylation of multiple targets downstream of TORC1. Inhibition of TORC1 has been observed to extend lifespan and healthspan in aging preclinical species and to improve the function of aging organ systems, including neurologic function, suggesting potential benefits in several aging-related diseases.
resTORbio, Inc. is a clinical-stage biopharmaceutical company developing innovative medicines that target the biology of aging to treat aging-related diseases. resTORbio’s lead program selectively inhibits TORC1, an evolutionarily conserved pathway that contributes to the decline in function of aging organ systems, including neurologic function. Learn more about resTORbio, Inc. at www.resTORbio.com.
Forward Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding: our expectations of the potential impact of COVID-19 on strategy, future operations, and the timing of its clinical trials and specifically the enrollment of the fifth cohort in our ongoing Phase 1b/2a trial; the safety, efficacy and regulatory and clinical progress of our product candidates, including RTB101 alone or in combination with sirolimus. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, express or implied statements or guidance regarding our Phase 1b/2a clinical trial of RTB101 in combination with sirolimus in Parkinson’s disease, our plans to develop RTB101 alone or in combination with rapalogs, including the therapeutic potential and clinical benefits thereof and the potential patient populations that may be addressed by our product candidates, our ongoing and future clinical trials for RTB101 , including the timing of the initiation and anticipated results of these trials, the continued expansion of our pipeline into Parkinson’s disease, and our ability to replicate results achieved in our clinical trials in any future trials, constitute forward-looking statements. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” and other similar words or expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
Such forward-looking statements are subject to a number of material risks and uncertainties that are more fully discussed in the section entitled “Risk Factors” in the Annual Report on Form 10-K filed by resTORbio, Inc. with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.
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