mRNA-3927 is Moderna’s second rare disease
program to receive Fast Track designation
mRNA-3927 uses the same proprietary LNP
formulation as mRNA-1944 (antibody against Chikungunya virus) and
mRNA-3704 (methylmalonic acidemia)
Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology
company pioneering messenger RNA (mRNA) therapeutics and vaccines
to create a new generation of transformative medicines for
patients, today announced that the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation for
mRNA-3927, its investigational mRNA therapeutic for propionic
acidemia (PA). The Company announced the open IND for mRNA-3927 on
September 30, 2019.
“Propioic acidemia is caused by the inability of the body to
breakdown certain proteins and fats which leads to the build-up of
toxic chemicals. The disease is characterized by life-threatening
illnesses in response to minor stressors, neurological dysfunction
and cardiomyopathy,” said George Diaz, M.D., Ph.D., chief, division
of medical genetics, Icahn School of Medicine at Mount Sinai
Hospital. “Currently there are no approved therapies available that
treat the underlying cause of this debilitating disease.”
“Fast Track designation underscores the urgent need for a
therapy that treats the underlying cause of propionic acidemia,”
said Tal Zaks, M.D., Ph.D., chief medical officer at Moderna. “We
are preparing to initiate a Phase 1/2 clinical study of mRNA-3927
to continue learning about the potential for this investigational
therapy to restore enzyme activity in patients with propionic
acidemia.”
Fast Track is designed to facilitate the development and
expedite the review of therapies and vaccines for serious
conditions and fill an unmet medical need. Programs with Fast Track
designation may benefit from early and frequent communication with
the FDA, in addition to a rolling submission of the marketing
application. The Company previously received Fast Track designation
for its investigational Zika vaccine (mRNA-1893) and methylmalonic
acidemia (MMA) (mRNA-3704) programs.
Moderna plans to initiate an open-label, multi-center, dose
escalation Phase 1/2 study of multiple ascending doses of mRNA-3927
in primarily pediatric patients with PA in the United States and
Europe. The objectives of this study are to evaluate the safety and
tolerability of mRNA-3927 administered via IV infusion,
characterize the pharmacokinetic profile of mRNA-3927 and assess
the pharmacodynamic response as assessed by changes in plasma
biomarkers.
PA and MMA are rare diseases that share similar disease
pathology and are both typically treated by metabolic specialists.
In order to characterize and describe the natural history of these
disorders and identify potential clinical and biomarker endpoints,
Moderna is conducting a global, multi-center, non-interventional
observational study for patients with confirmed diagnosis of PA or
MMA. More information about this study can be found at
ClinicalTrials.gov. Moderna is currently recruiting patients with
MMA for a Phase 1/2 study of mRNA-3704. More information about this
study can be found at ClinicalTrials.gov.
About mRNA-3927
mRNA-3927 is designed to instruct the body to restore the
missing or dysfunctional proteins that cause PA. mRNA-3927 contains
two mRNAs that encode for the alpha and beta subunits of the
mitochondrial enzyme propionyl-CoA carboxylase (PCC), encapsulated
within Moderna’s proprietary lipid nanoparticle (LNP). mRNA-3927 is
intended to treat patients with PA regardless of whether they are
missing the alpha or beta subunits. mRNA-3927 uses the same
proprietary LNP formulation used in the Company’s antibody against
chikungunya virus (mRNA-1944) and MMA (mRNA-3704) programs.
In addition to Fast Track designation, mRNA-3927 has also been
granted Orphan Drug and Rare Pediatric Disease designations from
the FDA and Orphan Designation by the European Medicines Agency
(EMA).
About Propionic Acidemia (PA)
PA is a rare, life-threatening, inherited metabolic disorder
that is the result of a deficiency in PCC that is an enzyme
critical for metabolism. This deficiency can lead to a toxic
buildup of acids in the body. Symptoms of PA typically become
apparent during infancy and may include weak muscle tone, poor
feeding, vomiting and lack of energy. More severe health problems
can also occur, including heart abnormalities, seizures and
coma.
The only effective treatment for severely affected individuals
is liver transplant, which replaces the deficient PCC enzyme.
Currently there are no approved therapies to treat the underlying
cause of PA, including no enzyme replacement therapy, due to the
complexity of the PCC enzyme that requires mitochondrial
localization.
About Moderna
Moderna is advancing messenger RNA (mRNA) science to create a
new class of transformative medicines for patients. mRNA medicines
are designed to direct the body’s cells to produce intracellular,
membrane or secreted proteins that have a therapeutic or preventive
benefit with the potential to address a broad spectrum of diseases.
Moderna’s platform builds on continuous advances in basic and
applied mRNA science, delivery technology and manufacturing,
providing the Company the capability to pursue in parallel a robust
pipeline of new development candidates. Moderna is developing
therapeutics and vaccines for infectious diseases, immuno-oncology,
rare diseases and cardiovascular diseases, independently and with
strategic collaborators.
Headquartered in Cambridge, Mass.,
Moderna currently has strategic alliances for development programs
with AstraZeneca, Plc. and Merck, Inc., as well as the Defense
Advanced Research Projects Agency (DARPA), an agency of the U.S.
Department of Defense and the Biomedical Advanced Research and
Development Authority (BARDA), a division of the Office of the
Assistant Secretary for Preparedness and Response (ASPR) within the
U.S. Department of Health and Human Services (HHS). Moderna has
been ranked in the top ten of Science’s list of top biopharma
industry employers for the past four years. To learn more, visit
www.modernatx.com.
Special Note Regarding
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended including, but not limited to, statements
concerning: Moderna’s plans to initiate an open-label,
multi-center, dose escalation Phase 1/2 study of multiple ascending
doses of mRNA-3927; mRNA-3927’s potential to restore enzyme
activity in children with PA; and mRNA-3927’s potential for
patients with PA regardless of whether they have defects in the
alpha or beta subunits of PCC. In some cases, forward-looking
statements can be identified by terminology such as “will,” “may,”
“should,” “could,” “expects,” “intends,” “plans,” “aims,”
“anticipates,” “believes,” “estimates,” “predicts,” “potential,”
“continue,” or the negative of these terms or other comparable
terminology, although not all forward-looking statements contain
these words. The forward-looking statements in this press release
are neither promises nor guarantees, and you should not place undue
reliance on these forward-looking statements because they involve
known and unknown risks, uncertainties and other factors, many of
which are beyond Moderna’s control and which could cause actual
results to differ materially from those expressed or implied by
these forward-looking statements. These risks, uncertainties and
other factors include, among others: whether the Phase 1 results
for mRNA-1944 will be predictive of any future clinical studies for
other development candidates with the same lipid nanoparticle (LNP)
formulation, including mRNA-3927 and mRNA-3704; the fact that
clinical development is lengthy and uncertain, especially for a new
class of medicines such as mRNA, and therefore Moderna’s clinical
programs or development candidates may be delayed, terminated, or
may never advance; no mRNA drug has been approved in this new
potential class of medicines, and may never be approved; mRNA drug
development has substantial clinical development and regulatory
risks due to the novel and unprecedented nature of this new class
of medicines; and those risks and uncertainties described under the
heading “Risk Factors” in Moderna’s most recent Annual Report on
Form 10-K filed with the U.S. Securities and Exchange Commission
(SEC) and in subsequent filings made by Moderna with the SEC, which
are available on the SEC’s website www.sec.gov. Except as required
by law, Moderna disclaims any intention or responsibility for
updating or revising any forward-looking statements in this press
release in the event of new information, future developments or
otherwise. These forward-looking statements are based on Moderna’s
current expectations and speak only as of the date hereof.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191022006101/en/
Media: Colleen Hussey Senior Manager, Corporate
Communications 203-470-5620 Colleen.Hussey@modernatx.com Dan
Budwick 1AB 973-271-6085 dan@1abmedia.com Investors: Lavina
Talukdar Head of Investor Relations 617-209-5834
Lavina.Talukdar@modernatx.com
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