Translate Bio Provides MRT5005 Program Updates

— FDA grants Rare Pediatric Disease designation for MRT5005 for treatment of cystic fibrosis –
 — Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date —LEXINGTON, Mass., March 26, 2020 (GLOBE NEWSWIRE) — Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced two updates related to its MRT5005 clinical development program in cystic fibrosis (CF). First, the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for MRT5005 for the treatment of CF. MRT5005, the first inhaled mRNA therapeutic, is designed to deliver mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to cells in the lung through nebulization. Also, as the worldwide healthcare system remains focused on the response to the COVID-19 pandemic, the Company anticipates related interruptions in enrollment, dosing and follow-up in the ongoing Phase 1/2 clinical trial in patients with CF. The Company plans to provide updated timing on the expected interim data readout of the additional single-ascending dose (SAD) group and the multiple-ascending dose (MAD) portion of the clinical trial at a later date.“Our priority is always patient safety and well-being, and specifically during this unprecedented public health crisis, we join our health and local authorities, healthcare providers, and patient advocacy groups in ensuring that patients with CF exercise an abundance of caution to prevent exposure to SARS-CoV-2. As more patients with CF and their physicians choose to avoid unnecessary risk of exposure to SARS-CoV-2, we, like many companies across the healthcare and biopharmaceutical industry, anticipate that patient dosing and follow-up visits in our ongoing trial will be affected,” said Dr. Ann Barbier, chief medical officer, Translate Bio. “We are in regular communication with our clinical trial sites to monitor the impact of COVID-19 and to continue to provide the best possible care and support to the patients in the trial. Given the uncertain and dynamic nature of this situation, we will provide an update when we have additional insight into enrollment and dosing metrics.”Dr. Barbier continued, “More than 75 percent of people with CF are diagnosed by age 2 — as such, we are pleased to receive the FDA’s Rare Pediatric Disease designation for MRT5005 for the treatment of CF and we are planning future trials of MRT5005 in pediatric CF patients. This important designation underscores the significant unmet medical needs for people living with cystic fibrosis who are still waiting for life-saving treatments, and the potential role that MRT5005 could play in treating patients who are not amenable to current treatment options.”About Rare Pediatric Disease Designation
The FDA grants Rare Pediatric Disease designation for a disease that is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals ages 18 years or younger and fewer than 200,000 people in the United States. If a new Biologics License Application (BLA) for MRT5005 is approved, Translate Bio may be eligible to receive a Priority Review Voucher, which can be redeemed to obtain Priority Review for any subsequent marketing application for a different product, and which may be sold or transferred. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
About MRT5005
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. The FDA has granted MRT5005 Orphan Drug, Fast Track and Rare Pediatric Disease designation.
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. In July 2019, the Company announced interim results from single-ascending dose (SAD) portion of Phase 1/2 clinical trial of MRT5005 in patients with CF. For more information about the Phase 1/2 clinical trial, visit
About Cystic Fibrosis
Cystic fibrosis is the most common fatal inherited disease in the United States, affecting more than 30,000 patients in the U.S. and more than 70,000 patients worldwide. CF is caused by genetic mutations that result in dysfunctional or absent CFTR protein. This defect causes mucus buildup in the lungs, pancreas and other organs. Mortality is primarily driven by a progressive decline in lung function. According to the Cystic Fibrosis Foundation, the median age at death for patients with CF was 30.8 years in 2018. There is no cure for CF. Currently marketed CFTR modulators are effective only in patients with specific mutations, and patients still experience pulmonary exacerbations and a progressive decline in lung function, which represents a significant unmet need.
About Translate Bio
Translate Bio is a clinical-stage mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. Translate Bio is primarily focused on applying its technology to treat pulmonary diseases caused by insufficient protein production or where the reduction of proteins can modify disease. Translate Bio’s lead program is being developed as a treatment for cystic fibrosis (CF) and is in an ongoing Phase 1/2 clinical trial. The Company also believes its technology is applicable to a broad range of diseases, including diseases that affect the liver. Additionally, the platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology. For more information about the Company, please visit or on Twitter at @TranslateBio.
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 patients; Translate Bio’s beliefs regarding the broad applicability of its MRT platform; Translate Bio’s expectations regarding interruptions in patient dosing and follow-up in the ongoing Phase 1/2 clinical trial of MRT5005; Translate Bio’s expectations on when it will provide an interim data readout from the trial; Translate Bio’s plans for future trials of MRT5005 in pediatric CF patients and Translate Bio’s plans, strategies and prospects for its business, including its lead development programs. 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: Translate Bio’s ability to advance the development of its platform and programs 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 content and timing of decisions made by the FDA, other regulatory authorities and investigational review boards at clinical trial sites, including decisions as it relates to ongoing and planned clinical trials; Translate Bio’s ability to obtain, maintain and enforce necessary patent and other intellectual property protection; the availability of significant cash required to fund operations; competitive factors; the COVID-19 pandemic; general economic and market conditions and other important risk factors set forth under the caption “Risk Factors” in Translate Bio’s Annual Report on Form 10-K for the fiscal year ended December 31, 2019 filed with the Securities and Exchange Commission on March 12, 2020 and in any other subsequent filings made by Translate Bio. Any forward-looking statements contained in this press release speak only as of the date hereof, and Translate Bio specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
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