Arix Bioscience PLC (ARIX) Clinical Development Update from
Portfolio Company LogicBio 09-May-2022 / 15:28 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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PRESS RELEASE
Arix Bioscience plc
Clinical Development Update from Portfolio Company LogicBio
LONDON, 09 May 2022: Arix Bioscience plc ("Arix", LSE:ARIX), a
global venture capital company focused on investing in breakthrough
biotechnology companies, notes that its portfolio company, LogicBio
Therapeutics, Inc. (Nasdaq: LOGC), today announced that the US Food
and Drug Administration (FDA) has lifted the clinical hold on the
company's LB-001 Investigational New Drug Application (IND),
allowing patient enrolment to resume in the Phase 1/2 SUNRISE trial
for paediatric 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.
The announcement can be accessed on LogicBio's website here 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
Powerscourt Group
Sarah MacLeod, Ibrahim Khalil
+44 (0)20 7250 1446
arix@powerscourt-group.com
About Arix Bioscience plc
Arix Bioscience plc is a global venture capital company focused
on investing in breakthrough biotechnology 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
industry to a broader range of investors.
www.arixbioscience.com
LogicBio Press Release
LogicBio Therapeutics Announces FDA Lifts Clinical Hold on
SUNRISE Trial in Pediatric Patients with Methylmalonic Acidemia
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, sAAVyT, 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.
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ISIN: GB00BD045071
Category Code: PFU
TIDM: ARIX
LEI Code: 213800OVT3AHQCXNIX43
OAM Categories: 3.1. Additional regulated information required to be disclosed under the laws of a Member State
Sequence No.: 160504
EQS News ID: 1347399
End of Announcement EQS News Service
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