Translate Bio Announces Timing of Interim Results from mRNA Therapeutic Clinical Trial in Patients with Cystic Fibrosis; Data Expected in Early Q2 2021
-- MRT5005 is the first clinical-stage mRNA therapeutic with nebulized delivery to the lung and first to be administered in multiple doses for the treatment of a chronic genetic disease --
-- Completed enrollment and dosing for upcoming second interim data analysis; includes 8, 12 and 16 mg multiple-ascending dose groups and 20 mg single-ascending dose group --
MRT5005, the first mRNA therapeutic with delivery to the lung, is designed to address the underlying cause of CF, regardless of genetic mutation, by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to cells in the lung through nebulization. Currently approved CFTR modulator treatments are specific to a patient’s genetic mutation; therefore, there remains a significant unmet medical need for patients with CF who have genetic mutations non-amenable to these treatments or for patients who derive little therapeutic benefit from or are unable to tolerate these treatments.
“We are pleased to share the progress we have continued to make in this first-of-its-kind trial of an mRNA therapeutic, MRT5005, in people with cystic fibrosis—especially after having paused enrollment and dosing in response to the pandemic,” said Dr.
MRT5005 is the first clinical-stage mRNA product candidate designed to address the underlying cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization. MRT5005 is being developed to treat all patients with CF, regardless of the underlying genetic mutation, including those with limited or no CFTR protein.
About the MRT5005 Phase 1/2 Clinical Trial
The randomized, double-blind, placebo-controlled Phase 1/2 clinical trial of MRT5005 is designed to enroll at least 40 adult patients with CF who have two Class I and/or Class II mutations. The primary endpoint of the trial will be the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization. Percent predicted forced expiratory volume in one second (ppFEV1), which is a well-defined and accepted endpoint measuring lung function, will also be measured at pre-defined timepoints throughout the trial. The Phase 1/2 clinical trial of MRT5005 for the treatment of CF is being conducted in collaboration with the Cystic Fibrosis Foundation Therapeutics Development Network and the Emily’s Entourage Patient Registry. In
About Cystic Fibrosis
Cystic fibrosis is the most common fatal inherited disease in
Translate Bio Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the potential for MRT5005 to address the underlying cause of CF and benefit CF patients; Translate Bio’s plans to report interim data from its Phase 1/2 clinical trial for MRT5005 in CF in the second quarter of 2021; Translate Bio’s plans to continue to enroll and dose patients in its Phase 12/clinical trial of MRT5005; the potential for mRNA-based therapeutics to apply to the treatment of many diseases caused by protein or gene dysfunction; Translate Bio’s beliefs regarding the broad applicability of its MRT platform; and Translate Bio’s plans, strategies and prospects for its business, including its lead development programs and continued development of mRNA vaccines for the treatment of infectious diseases. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “forward,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to: the current and potential future impacts of the COVID-19 pandemic on Translate Bio’s business, financial condition, operations and liquidity; Translate Bio’s ability to advance the development of its platform and programs, including without limitation, under the timelines it projects, demonstrate the requisite safety and efficacy of its product candidates and replicate in clinical trials any positive findings from preclinical studies; the successful advancement of the collaboration agreement between
Source: Translate Bio, Inc.