LogicBio Therapeutics Announces Early Clinical Trial Results Demonstrating First-Ever In Vivo Genome Editing in Children
19 October 2021 - 02:47AM
UK Regulatory
Arix Bioscience PLC (ARIX) LogicBio Therapeutics Announces Early
Clinical Trial Results Demonstrating First-Ever In Vivo Genome
Editing in Children 18-Oct-2021 / 16:47 GMT/BST Dissemination of a
Regulatory Announcement, transmitted by EQS Group. The issuer is
solely responsible for the content of this announcement.
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Arix Bioscience plc
LogicBio Therapeutics Announces Early Clinical Trial Results
Demonstrating First-Ever In Vivo Genome Editing in Children
LONDON, 18 October 2021: Arix Bioscience plc ("Arix", LSE:ARIX),
a global venture capital company focused on investing in and
building breakthrough biotech companies, notes that its portfolio
company, LogicBio Therapeutics Inc. ("LogicBio", Nasdaq:LOGC),
today announced clinical trial results demonstrating the first-ever
in vivo genome editing in children. Early data from the Company's
Phase 1/2 SUNRISE clinical trial showed measurable levels of
albumin-2A, a technology-related biomarker indicating site-specific
gene insertion and protein expression. The SUNRISE trial is
evaluating the safety, tolerability and preliminary efficacy of
LB-001, the Company's investigational, single-administration genome
editing therapy, in pediatric patients with methylmalonic acidemia
(MMA). The Company remains on track to present additional interim
data by the end of 2021.
The announcement can be accessed on LogicBio's website at:
www.investor.logicbio.com and full text of the announcement from
LogicBio is contained below.
[ENDS]
For more information on Arix, please contact:
Arix Bioscience plc
+44 (0)20 7290 1050
ir@arixbioscience.com
Optimum Strategic Communications
Mary Clark, Manel Mateus
+44 (0)20 3922 1906
optimum.arix@optimumcomms.com
About Arix Bioscience plc
Arix Bioscience plc is a global venture capital company focused
on investing in and building breakthrough biotech companies around
cutting-edge advances in life sciences.
We collaborate with exceptional entrepreneurs and provide the
capital, expertise and global networks to help accelerate their
ideas into important new treatments for patients. As a listed
company, we are able to bring this exciting growth phase of our
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www.arixbioscience.com
LOGICBIO THERAPEUTICS PRESS RELEASE LogicBio Therapeutics
Announces Early Clinical Trial Results Demonstrating First-Ever In
Vivo Genome Editing in Children
- Early data from Phase 1/2 clinical trial in pediatric patients
with methylmalonic acidemia showed measurable levels of a biomarker
indicating site-specific gene insertion and protein expression
- Based on safety data from first two patients, independent Data
Safety Monitoring Board recommended continuation of trial, enabling
enrollment of children as young as six months and dose
escalation
- Company remains on track to report additional interim clinical
data by end of 2021 LEXINGTON, Mass., Oct. 18, 2021 -- LogicBio
Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine
company, today announced clinical trial results demonstrating the
first-ever in vivo genome editing in children. Early data from the
company's Phase 1/2 SUNRISE clinical trial showed measurable levels
of albumin-2A, a technology-related biomarker indicating
site-specific gene insertion and protein expression. The SUNRISE
trial is evaluating the safety, tolerability and preliminary
efficacy of LB-001, the company's investigational,
single-administration genome editing therapy, in pediatric patients
with methylmalonic acidemia (MMA). These results follow a
recommendation from the independent Data Safety Monitoring Board
(DSMB) overseeing the SUNRISE trial to continue the study without
modification. The DSMB's recommendation was based on an evaluation
of the safety data from the first two patients enrolled in the
trial. Per the FDA-cleared protocol, albumin-2A detection together
with the DSMB continuation recommendation enables LogicBio to begin
enrolling two patients in the higher dose (1 x 1014 vg/kg) cohort
(with ages ranging three to twelve years old) and two patients in
the lower age (six months to two years old) cohort at the lower
dose (5 x 1013 vg/kg) of LB-001. "We are very excited to have
achieved this significant milestone in the field of genetic
medicine," said Fred Chereau, president and chief executive officer
of LogicBio. "These early data indicate that we can precisely edit
hepatocytes in vivo to treat a genetic liver disease with a single
intravenous infusion using our proprietary GeneRide(TM) technology.
Today's announcement is a demonstration that homologous
recombination genome editing without the use of nucleases is a
potential alternative to genome editing technologies in development
that use nucleases, such as CRISPR. The ability to insert the
correct version of a gene in a cell's genome without nucleases is
an important step to unlocking the potential of GeneRide(TM) to
treat a larger number of genetic diseases." SUNRISE is a
first-in-human, open-label, multi-center, Phase 1/2 clinical trial
designed to assess the safety and tolerability of a single
intravenous infusion of LB-001 in pediatric patients with MMA.
LB-001 is designed to non-disruptively insert a corrective copy of
the MMUT gene into the albumin locus to drive lifelong therapeutic
levels of MMUT expression in the liver. LB-001 is based on the
company's proprietary GeneRide technology, which uses homologous
recombination, a natural DNA repair process, to enable 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. "MMA is a rare, life-threatening
genetic disorder for which there are no treatments addressing the
underlying cause of the disease. By demonstrating for the first
time ever that in vivo, nuclease-free genome editing in pediatric
patients is achievable, we are one step closer to bringing a safe
and effective genetic medicine to children suffering from MMA and,
potentially, other early onset genetic diseases where early
intervention is critical to achieve optimal health outcomes," said
Daniel Gruskin, MD, chief medical officer of LogicBio. "I would
like to thank the patients, their families and the investigators
who are participating in this landmark trial. We look forward to
continuing to progress the clinical study to better understand the
biochemical and clinical effect of this genome editing therapy."
The Company remains on track to present additional interim data by
the end of 2021. About the SUNRISE Trial The SUNRISE trial is an
open-label, multi-center, Phase 1/2 clinical trial designed to
assess the safety and tolerability of a single intravenous infusion
of LB-001 in pediatric patients with methylmalonic acidemia (MMA)
characterized by methylmalonyl-CoA mutase gene (MMUT) mutations.
Seven leading centers in the United States and one in Saudi Arabia
are expected to participate in the trial. With the aim of
evaluating LB-001 at an early age, the SUNRISE trial initially
enrolled 3-12 year old patients and, following a recommendation
from the trial's independent Data Safety Monitoring Board and
detection of a biomarker indicating site-specific gene insertion,
is permitted to enroll infants as young as 6 months old. The
SUNRISE trial is designed to enroll up to 8 patients and evaluate a
single administration of LB-001 at two dose levels. 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(TM) 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
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