Translate Bio Announces Pipeline Program Update

- Prioritizing and expanding pulmonary disease programs based on early positive cystic fibrosis (CF) data for MRT5005, the first inhaled mRNA therapeutic –

 - Discontinuing development of MRT5201, a liver-targeted treatment for ornithine transcarbamylase (OTC) deficiency –

LEXINGTON, MA, USA I September 09, 2019 I 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 that it is prioritizing the development of pulmonary disease programs including the ongoing development of MRT5005, its clinical candidate for the treatment of CF, as well as the evaluation of targets in additional pulmonary diseases. Additionally, the Company has decided to discontinue the development of MRT5201, a liver-targeted treatment for OTC deficiency.

The Company’s prioritization of pulmonary diseases is supported by the previously reported positive single-ascending dose Phase 1/2 data from the CF program, which utilizes its proprietary lung delivery platform. MRT5005 is a first-in-class mRNA therapeutic 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. The multiple-ascending dose portion of the clinical trial is ongoing with data expected in 2020. Preclinical research efforts are focused on additional pulmonary diseases, including primary ciliary dyskinesia (PCD), pulmonary arterial hypertension (PAH) and idiopathic pulmonary fibrosis (IPF).

The Company’s decision to discontinue the development of MRT5201 for OTC deficiency is based on data from recently completed preclinical studies which do not support the desired pharmacokinetic and safety profile for advancement of the program. These data are related to the first-generation lipid nanoparticle (LNP) designed to be delivered to the liver via intravenous administration for the OTC deficiency program. As such, this LNP is different than that used in the CF and other pulmonary development programs which are designed to deliver the LNP-encapsulated mRNA through nebulization. Additionally, ongoing discovery efforts have generated promising, novel next-generation LNPs supporting the further development of liver disease mRNA therapeutics with potentially favorable product profiles.

“We believe that the success to date in our cystic fibrosis program positions us well to build on our lung delivery platform and maximize the potential of our mRNA technology in additional pulmonary diseases with unmet medical need,” said Ronald Renaud, chief executive officer, Translate Bio. “With respect to our liver disease program, our goal is an optimal product profile with patient safety as our top priority. We look forward to further data from our next-generation delivery program to support that effort.”

Renaud continued, “We are always evaluating various methods for the delivery of mRNA and we continue to believe that LNPs are currently the most promising technology with advantages over other approaches. We design each LNP with distinct chemical structures and formulations specific to the target organ and route of administration in order to optimize safety and potency. We are excited about the robust mRNA delivery effort underway at Translate Bio and we’ll continue to apply our expertise in the development of novel lipids.”

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. The Company’s MRT platform is designed to develop product candidates that deliver mRNA carrying instructions to produce intracellular, transmembrane and secreted proteins for therapeutic benefit. Translate Bio is primarily focused on applying its MRT platform to pulmonary diseases caused by insufficient protein production or where production of proteins can modify disease. The Company also believes its technology is applicable to a broad range of diseases, including diseases that affect the liver, eye and central nervous system. Additionally, the MRT platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology. 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. For more information about the Company, please visit or on Twitter at @TranslateBio.

SOURCE: Translate Bio

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