Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis
26 February 2020 - 11:30PM
Translate Bio, Inc. (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 the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation for MRT5005
for the treatment of cystic fibrosis (CF). 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. The Phase 1/2 clinical trial of MRT5005
is currently ongoing.
“While there have been significant advances in the
treatment of CF, substantial unmet need remains. Roughly 10 percent
of the CF population is not anticipated to benefit from currently
approved CFTR modulators,” said Dr. Ann Barbier, chief medical
officer, Translate Bio. “MRT5005 has the potential to treat all
people with CF, including those with mutations that result in
limited to no CFTR protein production. The Fast Track designation
will help Translate Bio to expedite the clinical development of
this potentially transformative therapeutic.”
The FDA’s Fast Track program facilitates the
expedited development and review of drugs that are intended to
treat serious or life-threatening conditions and demonstrate the
potential to address unmet medical needs. Clinical development
programs granted Fast Track designation may be eligible for several
benefits, including more frequent meetings and communications with
the FDA and, if relevant criteria are met, the potential for
Accelerated Approval, Priority Review and Rolling Review of a
Biologics License Application (BLA).
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 both Orphan Drug and Fast
Track 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 the single-ascending dose (SAD)
portion of the 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. 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 treat
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 www.translate.bio 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: Translate Bio’s expectations
regarding the potential benefits of Fast Track designation by the
FDA for MRT5005; the potential for MRT5005 to address the
underlying cause of CF and benefit patients; and Translate Bio’s
plans to report data from its Phase 1/2 clinical trial of MRT5005
in the third quarter of 2020. 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; general economic and
market conditions and other important risk factors set forth under
the caption “Risk Factors” in Translate Bio’s Quarterly Report on
Form 10-Q for the quarterly period ended September 30,
2019 filed with the Securities and Exchange
Commission on November 6, 2019 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 |
857-242-7792 |
857-242-7789 |
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