–Company initiates activities to resume
dosing
–Interim clinical data from Phase 1/2 trial
expected to be presented by end of second quarter 2022
LEXINGTON, Mass., May 9, 2022
/PRNewswire/ -- LogicBio® Therapeutics, Inc. (Nasdaq:
LOGC), a clinical-stage company advancing a diversified pipeline of
genetic medicines addressing rare disorders from infancy through
adulthood, today announced that the U.S. Food and Drug
Administration (FDA) has lifted the clinical hold on the company's
LB-001 Investigational New Drug Application (IND), allowing patient
enrollment to resume in the Phase 1/2 SUNRISE trial for pediatric
patients with methylmalonic acidemia. In its letter, the FDA
acknowledged that the company satisfactorily addressed all clinical
hold issues. The company has initiated activities to resume dosing
as soon as possible.
"We are pleased that the FDA has completed its review of the
information we provided and that the hold on our LB-001 IND has
been lifted," said Frederic Chereau,
president and chief executive officer of LogicBio. "We look forward
to dosing the next patient in our SUNRISE trial, which we expect
will occur in the third quarter of 2022."
As previously disclosed, the FDA placed the IND for LB-001 on
clinical hold following the occurrence of two serious adverse
events, categorized as cases of thrombotic microangiopathy (TMA),
in the company's SUNRISE trial. Both cases of TMA resolved within
weeks.
In connection with the lifting of the clinical hold, LogicBio
amended the SUNRISE protocol in a manner that reflected its
dialogue with the FDA. LogicBio expects to proceed with dosing
after it implements the changes to the SUNRISE protocol, which
include enhanced monitoring measures, such as frequent testing for
complement activation, a characteristic of TMA, as well as the use
of a complement inhibitor in the event there are laboratory
findings indicating a potential TMA. LogicBio plans to treat the
next patients, who may be as young as six months old, at the 5e13
vg/kg dose and continually assess safety outcomes.
Following the lifting of the clinical hold, the company
announced that it is reinstating its previous guidance and expects
to present interim clinical data from the SUNRISE trial by the end
of the second quarter of 2022.
About LogicBio Therapeutics
LogicBio® Therapeutics is a clinical-stage genetic medicine
company pioneering genome editing and gene delivery platforms to
address rare and serious diseases from infancy through adulthood.
The company's genome editing platform, GeneRide®, is a new approach
to precise gene insertion harnessing a cell's natural DNA repair
process potentially leading to durable therapeutic protein
expression levels. The company's gene delivery platform, sAAVy™, is
an adeno-associated virus (AAV) capsid engineering platform
designed to optimize gene delivery for treatments in a broad range
of indications and tissues. The company is based in Lexington,
MA. For more information, visit www.logicbio.com, which
does not form a part of this release.
About the SUNRISE Trial
The SUNRISE trial is an open-label, multi-center, Phase 1/2
clinical trial designed to assess the safety, tolerability and
preliminary efficacy of a single intravenous infusion of LB-001 in
pediatric patients with methylmalonic acidemia (MMA) characterized
by methylmalonyl-CoA mutase gene (MMUT) mutations. With the aim of
evaluating LB-001 at an early age, the SUNRISE trial is designed to
enroll patients with ages ranging from six months to twelve years
and evaluate a single administration of LB-001 at two dose levels
(5e13 vg/kg and 1e14 vg/kg) with dose escalation subject
to certain conditions.
About LB-001
LB-001 is an investigational, first-in-class,
single-administration, genome editing therapy for early
intervention in methylmalonic acidemia (MMA) using LogicBio's
proprietary GeneRide® drug development platform. GeneRide
technology utilizes a natural DNA repair process called homologous
recombination that enables precise editing of the genome without
the need for exogenous nucleases and promoters that have been
associated with an increased risk of immune response and cancer.
LB-001 is designed to non-disruptively insert a corrective copy of
the methylmalonyl-CoA mutase (MMUT) gene into the albumin locus to
drive lifelong therapeutic levels of MMUT expression in the liver,
the main site of MMUT expression and activity. LB-001 is delivered
to hepatocytes intravenously via liver-targeted, engineered
recombinant adeno-associated virus vector (rAAV-LK03). Preclinical
studies found that LB-001 was safe and demonstrated transduction of
hepatocytes, site-specific genomic integration, and transgene
expression. LB-001–corrected hepatocytes in a mouse model of MMA
demonstrated preferential survival and expansion (selective
advantage), thus contributing to a progressive increase in hepatic
MMUT expression over time. LB-001 resulted in improved growth,
metabolic stability, and survival in MMA mice. The U.S. Food
and Drug Administration (FDA) granted fast track designation,
rare pediatric disease designation and orphan drug designation for
LB-001 for the treatment of MMA. In addition, the European
Medicines Agency (EMA) granted orphan drug designation for
LB-001 for the treatment of MMA.
About Methylmalonic Acidemia (MMA)
Methylmalonic acidemia (MMA) is a rare and life-threatening
genetic disorder affecting approximately 1 in 50,000 newborns
in the United States. In the most common form of MMA, a
mutation in a gene called methylmalonyl-CoA mutase (MMUT) prevents
the body from properly processing certain fats and proteins. As a
result, toxic metabolites accumulate in the liver, in muscle tissue
and in the brain. Symptoms include vomiting, lethargy, seizures,
developmental delays and organ damage. There is no approved medical
therapy addressing the underlying cause of the disease. To manage
the symptoms, patients go on a severely restrictive, low-protein,
high-calorie diet, often through a feeding tube. Even with
aggressive management, these patients often experience
life-threatening metabolic crises that can require recurrent
hospitalizations and cause permanent neurocognitive damage. Because
of this risk for irreversible damage, early intervention is
critical, and newborns are screened for MMA in every state
in the United States.
Forward-Looking Statements
Statements in this press release regarding LogicBio®'s strategy,
plans, prospects, expectations, beliefs, intentions and goals are
forward-looking statements within the meaning of
the U.S. Private Securities Litigation Reform Act of
1995, as amended, including but not limited to statements regarding
resumption of the SUNRISE clinical trial and timing thereof; our
expectations to continue enrollment and dosing of clinical trial
subjects and timing thereof; the potential of the GeneRide®
platform; and the anticipated timing of announcing interim clinical
data. The terms "aim," "anticipate," "designed," "enables,"
"evaluate," "expects to," "look forward to," "will," and similar
references are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Each forward-looking statement is subject to
risks and uncertainties that could cause actual results to differ
materially from those expressed or implied in such statement,
including the risk that we will need to obtain the approval of each
clinical trial site's institutional review board prior to resuming
dosing in our SUNRISE trial; we may encounter difficulties
enrolling patients; the potential direct or indirect impact of the
COVID-19 pandemic on our business, operations, and the markets and
communities in which we and our partners, collaborators and vendors
operate; manufacturing risks; risks associated with management and
key personnel changes and transitional periods; the actual funding
required to develop and commercialize product candidates, including
for safety, tolerability, enrollment, manufacturing or economic
reasons; the timing and content of decisions made by regulatory
authorities; the actual time it takes to initiate and complete
clinical studies; the competitive landscape; changes in the
economic and financial conditions of LogicBio; and LogicBio's
ability to obtain, maintain and enforce patent and other
intellectual property protection for LB-001 and any other product
candidates. Other risks and uncertainties include those identified
under the heading "Risk Factors" in LogicBio's Annual Report on
Form 10-K for the year ended December 31, 2021 and other
filings that LogicBio may make with the U.S. Securities and
Exchange Commission in the future. These forward-looking
statements (except as otherwise noted) speak only as of the date of
this press release, and LogicBio does not undertake, and
specifically disclaims, any obligation to update any
forward-looking statements contained in this press release.
Investor Contact:
Stephen Jasper
Gilmartin Group
(858) 525-2047
stephen@gilmartin.com
Media Contact:
Adam Daley
Berry & Company Public Relations
W:212-253-8881
C: 614-580-2048
adaley@berrypr.com
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SOURCE LogicBio Therapeutics, Inc.
