Translate Bio Presents MRT5005 Data at the 33rd Annual North American Cystic Fibrosis Conference
October 31 2019 - 12:15PM
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 presented data for MRT5005, an inhaled mRNA
therapeutic, at the 33rd Annual North American Cystic Fibrosis
Conference (NACFC) taking place in Nashville, Tennessee from
October 31 to November 2, 2019. MRT5005 is designed to address the
underlying cause of cystic fibrosis (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 poster presentation today and the oral presentation tomorrow
describe the previously announced interim results from the
single-ascending dose (SAD) portion of a first-in-human Phase 1/2
clinical trial of MRT5005 in 12 patients with CF. The
multiple-ascending dose (MAD) portion of the clinical trial is
ongoing with data expected in 2020.
“It’s an exciting time in the field of CF treatment where
significant advancements have been made for the majority of people
with CF; however, there are still those with different mutations
who do not benefit from these new medicines,” said Jonathan
Zuckerman, M.D., Director, Adult Cystic Fibrosis Program, Maine
Medical Center and RESTORE-CF investigator. “These initial data are
encouraging and highlight the potential for MRT5005 as a treatment
for people with CF who currently have no treatment options.”
The interim results describe data from 12 adult patients with CF
who received a single dose of either MRT5005 or placebo (3:1
randomization). Patients who received MRT5005 were assigned to one
of three dose groups (8, 16 or 24 mg). An overview of the results
from the SAD portion of the study include:
- Marked increases in ppFEV1 after a single MRT5005 dose were
observed in 4 of 9 patients, primarily at the mid-dose level
- ppFEV1 increases were observed in a patient considered
non-amenable to current CFTR modulator regimens as well as in
patients on CFTR modulator regimens
- Early improvement in ppFEV1 after a single dose suggests
MRT5005 is crossing the mucus layer and enabling production of
functional CFTR protein
- No serious adverse events were reported at any dose level
- MRT5005 was generally well tolerated at low and mid-dose
levels; primarily at high dose, patients receiving a single dose of
MRT5005 experienced transient, mild to moderate febrile
reactions
- Evaluation of immunogenicity markers showed no detection of
anti-CFTR anti-drug antibodies or T-cell sensitization
A copy of the poster and oral presentation will be available on
Translate Bio’s investor website at the time of the data
presentation.
About the Phase 1/2 Clinical Trial The
randomized, double-blind, placebo-controlled Phase 1/2 clinical
trial of MRT5005 is designed to enroll up to 40 adult patients with
CF who have two Class I and/or Class II mutations. The primary
endpoint of the trial is to assess 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, is also being 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.
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 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
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; Translate Bio’s
expectation regarding the potential benefits and advantages of its
technology; Translate Bio’s plans to enroll patients in the Phase
1/2 clinical trial of MRT5005; and Translate Bio’s plans to report
data from the MAD portion of the Phase 1/2 clinical trial of
MRT5005 in 2020. The words “anticipate,” “believe,” “continue,”
“could,” “estimate,” “expect,” “intend,” “may,” “plan,”
“potential,” “predict,” “project,” “should,” “suggest,” “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 later-stage 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 June 30, 2019
filed with the Securities and Exchange Commission on July 31, 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 |
617-817-8655 |
617-233-1154 |
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