Translate Bio Provides MRT5005 Program Updates
March 26 2020 - 3:30PM
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 DesignationThe 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 TrialThe
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 https://clinicaltrials.gov/ct2/show/NCT03375047.
About Cystic FibrosisCystic 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 BioTranslate 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 www.translate.bio or on
Twitter at @TranslateBio.
Cautionary Note Regarding Forward-Looking
StatementsThis 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.
Contacts for Translate Bio
Investors |
Media |
Teri Dahlman |
Maura Gavaghan |
tdahlman@translate.bio |
mgavaghan@translate.bio |
617-817-8655 |
617-233-1154 |
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