mRNA-3927 is Moderna’s second rare disease
program with an open IND
mRNA-3927 uses the same proprietary LNP
formulation as mRNA-1944 (antibody against Chikungunya virus)
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 the U.S. Food and Drug Administration
(FDA) has completed their review of the Investigational New Drug
(IND) application for mRNA-3927, its investigational mRNA
therapeutic for propionic acidemia (PA) and allowed it to proceed
to clinic.
“We are excited to have a second open IND for a rare disease
program and are preparing to move mRNA-3927 into a Phase 1/2
clinical trial in patients with propionic acidemia,” said Tal Zaks,
M.D., Ph.D., chief medical officer at Moderna. “There are no
approved therapies to treat the underlying cause of propionic
acidemia, a rare metabolic disorder that can lead to a toxic
buildup of acids in the body. We believe mRNA-3927 has the
potential to restore the missing or dysfunctional proteins and thus
relieve the acidemia that causes this disease in children.”
mRNA-3927 had previously been granted Orphan Drug and Rare
Pediatric Disease designations from the FDA and Orphan Designation
by the European Medicines Agency (EMA).
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 is the result of a deficiency in the mitochondrial enzyme
propionyl-CoA carboxylase (PCC) that is critical for metabolism.
mRNA-3927 contains two mRNAs that encode for the alpha and beta
subunits of PCC, encapsulated within Moderna’s proprietary lipid
nanoparticle (LNP). mRNA-3927 is intended for patients with PA
regardless of whether they have defects in the alpha or beta
subunit.
This is the second rare disease candidate from Moderna’s
pipeline with an open IND. The first program, mRNA-3704, is
designed to treat methylmalonic acidemia (MMA), another rare
metabolic disorder. The Company 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.
PA and MMA 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.
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 lipid nanoparticle (LNP) formulation used in the
Company’s antibody against chikungunya virus (mRNA-1944) and MMA
(mRNA-3704) programs.
About Propionic Acidemia
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 missing or
dysfunctional proteins and thus relieve the acidemia that causes PA
in children; mRNA-3927’s potential for patients with PA regardless
of whether they have defects in the alpha or beta subunit; and
Moderna’s plans to enroll patients with MMA in a Phase 1/2 study of
mRNA-3704. 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-3704 and mRNA-3927; 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.
Moderna Contacts:
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190930005404/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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