Translate Bio Announces FDA Clearance to Proceed with a Single-ascending Dose (SAD) Phase 1/2 Clinical Trial for Ornithine Tr...
June 26 2019 - 7:00AM
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 has received clearance
from the U.S. Food and Drug Administration (FDA) to proceed with a
single-ascending dose (SAD) Phase 1/2 clinical trial of MRT5201 in
adult patients with ornithine transcarbamylase (OTC) deficiency.
MRT5201 is a first-in-class treatment designed to directly address
the underlying cause of OTC deficiency by providing mRNA encoding
the fully functional OTC enzyme in patients with the disease.
“We are very pleased to be able to move forward with this
first-in-human clinical trial of MRT5201 in patients with OTC
deficiency,” said Ronald Renaud, chief executive officer, Translate
Bio. “This represents a significant milestone for our team as we
advance our second mRNA therapeutic for the treatment of a genetic
disease into clinical development. Progressing this program
underscores the potential application of our MRT platform in
multiple disease areas.”
As previously announced, in December 2018, the Company submitted
an investigational new drug application (IND) for a SAD and
multiple-ascending dose (MAD) Phase 1/2 study of MRT5201, which the
FDA placed on clinical hold, pending additional preclinical
toxicology data. After discussions with the FDA and after the
Company amended the protocol, the FDA has removed the clinical hold
and will allow the Company to move forward with a SAD clinical
trial. Additional preclinical studies will be required to
support future clinical development of MRT5201, including a MAD
clinical trial. These additional preclinical studies are currently
underway, and the Company plans to submit data from these studies
to the FDA in the fourth quarter of 2019.
About MRT5201 and OTC DeficiencyMRT5201 is
designed to treat patients with OTC deficiency by intravenous
delivery of mRNA encoding fully functional OTC enzyme to the liver
to enable the hepatocytes to produce the normal OTC enzyme. MRT5201
has been granted orphan drug designation for the treatment of OTC
deficiency in the U.S. and EU. OTC deficiency is a metabolic liver
enzyme disorder that results from a mutation in the OTC gene, and
is the most common urea cycle disorder. Based on published
research, the incidence of OTC deficiency is estimated to be 1 in
56,500 live births in the United States.
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 believes that its
MRT platform is applicable to a broad range of diseases caused by
insufficient protein production or where production of proteins can
modify disease, including diseases that affect the lung, liver, eye
and central nervous system. The Company also believes its 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 two lead programs are being
developed as treatments for cystic fibrosis (CF) and ornithine
transcarbamylase (OTC) deficiency. 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: Translate Bio’s plans to
conduct additional preclinical studies for MRT5201 and its plan to
submit data from these studies to FDA in the fourth quarter of
2019; Translate Bio’s beliefs regarding the broad applicability of
its MRT platform; and Translate Bio’s plans, strategies and
prospects for its business, including its lead development
programs. The words “anticipate,” “believe,” “continue,” “could,”
“estimate,” “expect,” “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; Translate Bio’s
ability to obtain additional preclinical data to support further
clinical development of MRT5201; the content and timing of
decisions made by the FDA, other regulatory authorities and
investigational review boards at clinical trial sites, including 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 March 31, 2019
filed with the Securities and Exchange Commission on May 9, 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.
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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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