Arix Bioscience plc LogicBio announces IND clearance for LB-001
August 10 2020 - 6:49AM
UK Regulatory
TIDMARIX
Arix Bioscience plc
LogicBio announces IND clearance for LB-001
LONDON, 10 August 2020: 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 the clinical trial design
for the planned Phase 1/2 clinical trial of its LB-001 programme in paediatric
patients with methylmalonic acidemia (MMA), following Investigational New Drug
application (IND) clearance from the U.S. Food and Drug Administration (FDA).
LogicBio expects patient enrolment to begin in early 2021.
The announcement can be accessed on LogicBio's investor website at https://
investor.logicbio.com/ and full text of the announcement from LogicBio is
contained below. LogicBio will host a webcast and conference call today, Monday
10 August 2020, at 8:00 a.m. ET/ 1:00 p.m. BST. To access the live webcast and
subsequent replay, please register here. The dial-in details for the call are
+1 833-519-1335 or +1 602-585-9978, Conference ID: 1151546.
[S]
Enquiries
For more information on Arix, please contact:
Arix Bioscience plc
Charlotte Parry, Head of Investor Relations
+44 (0) 20 7290 1072
charlotte@arixbioscience.com
Optimum Strategic Communications
Mary Clark, Supriya Mathur, Shabnam Bashir
T: +44 (0) 20 3950 9144
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 industry to a broader range of investors.
LogicBio Therapeutics Announces SUNRISE Phase 1/2 Clinical Design for LB-001
for the Treatment of Methylmalonic Acidemia in Pediatric Patients
- LogicBio's First IND Clearance Leveraging GeneRide, an In Vivo Homologous
Recombination-based Genome Editing Platform -
- Enrollment to Start with Patients as Young as 3 Years Old, De-escalating Down
to 6 Months old -
- Webcast and Conference Call Today at 8 a.m. ET to Discuss LB-001 Update -
LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc.
(Nasdaq: LOGC) (LogicBio or the Company), a company dedicated to extending the
reach of genetic medicine with pioneering targeted delivery platforms, today
announced the clinical trial design for the planned Phase 1/2 clinical trial
for LB-001 in pediatric patients with methylmalonic acidemia (MMA) .
"We're pleased to announce our plans for developing LB-001 in pediatric
patients with MMA. This is an important milestone for patients, their families,
our company and for the genetic medicines space more broadly as we believe this
is the first Investigational New Drug application (IND) clearance for an in
vivo gene editing program harnessing homologous recombination," said Fred
Chereau, CEO of LogicBio. "We have maintained a steadfast commitment to
execution and the IND clearance is the first of several near-term milestones we
expect to reach in our determined efforts to leverage cutting-edge medicines to
address high unmet medical needs. I look forward to sharing those updates as we
advance our LB-001 program."
Daniel Gruskin, M.D., SVP, head of clinical development, commented, "The
effects of MMA usually appear shortly after birth and can quickly become severe
and life-threatening. Early intervention in this vulnerable population is
critical to combat the manifestation of irreversible clinical disease
pathologies including neurological damage. That's the reason MMA is on the
newborn screening panel in the United States. Our protocol allows for the first
cohort to enroll patients as young as three years old and, once certain safety
parameters are met, we can age de-escalate to as young as six months old. Our
goal is to provide a safe and durable therapeutic with a single administration
at an age when we can make a difference for these patients."
SUNRISE - Phase 1/2 Clinical Trial of LB-001 in Pediatric Methylmalonic
Acidemia (MMA)
The SUNRISE trial is a multi-center, open-label, 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 characterized by methylmalonyl-CoA
mutase gene (MMUT) mutations. Six leading centers in the United States are
expected to participate in the SUNRISE Phase 1/2 trial. The trial is expected
to enroll eight pediatric patients with ages ranging from 6 months to 12 years,
initially starting with 3 to 12 year-old patients and then adding patients aged
6 months to 2 years. The SUNRISE Phase 1/2 trial will evaluate two doses of
LB-001. Patients will participate in a pre-dosing observational period and will
be administered a prophylactic steroid regimen. The primary endpoint of the
SUNRISE trial is to assess the safety and tolerability of LB-001 at 52 weeks
after a single infusion. Additional endpoints include changes in
disease-related biomarkers, including serum methylmalonic acid, clinical
outcomes such as growth and healthcare utilization, and the pharmacodynamic
marker albumin-2A. The Company expects to enroll the first patient in early
2021.
LB-001 Webcast and Conference Call Information
LogicBio will host a webcast and conference call on Monday, August 10, 2020 at
8:00 a.m. ET. The webcast will feature an overview of recently generated
preclinical data using LB-001 and the planned clinical development plan for
LB-001.
The event will be broadcast live and available under the investor relations
section of LogicBio's website at https://investor.logicbio.com/. The dial-in
details for the call are +1 833-519-1335 or +1 602-585-9978, Conference ID:
1151546. A replay of the webcast will be available on the LogicBio website for
one month following the call.
About LogicBio Therapeutics
LogicBio Therapeutics is dedicated to extending the reach of genetic medicine
with pioneering targeted delivery platforms.
LogicBio's proprietary genome editing technology platform, GeneRide, enables
the site-specific integration of a therapeutic transgene without nucleases or
exogenous promoters by harnessing the native process of homologous
recombination. LogicBio has received FDA clearance for the first-in-human
clinical trial of LB-001, a wholly owned genome editing program leveraging
GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is
expected to begin in early 2021. In addition, LogicBio has a collaboration with
Takeda to research and develop LB-301, an investigational therapy leveraging
GeneRide for the treatment of the rare pediatric disease Crigler-Najjar
syndrome.
LogicBio is also developing a Next Generation Capsid platform for use in gene
editing and gene therapies. Data presented have shown that the capsids deliver
highly efficient functional transduction of human hepatocytes with improved
manufacturability with low levels of pre-existing neutralizing antibodies in
human samples. Top-tier capsid candidates from this effort demonstrated
significant improvements over benchmark AAVs currently in clinical development.
LogicBio is developing these highly potent vectors for internal development
candidates and potentially for business development collaborations.
LogicBio is headquartered in Lexington, Mass. For more information, please
visit www.logicbio.com.
Forward Looking Statements
This press release contains "forward-looking" statements within the meaning of
the federal securities laws, including those related to the Company's plans to
initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of
LB-001 in MMA; the timing, progress and results of the Company's research and
development activities, including those related to the GeneRide technology
platform and Next Generation Capsid Program; and its plans for LB-301 in
Crigler-Najjar. These are not statements of historical facts and are based on
management's beliefs and assumptions and on information currently available.
They are subject to risks and uncertainties that could cause the actual results
and the implementation of the Company's plans to vary materially, including the
risks associated with the initiation, cost, timing, progress and results of the
Company's current and future research and development activities and
preclinical studies and potential future clinical trials. In particular, the
impact of the COVID-19 pandemic on the Company's ability to progress with its
research, development, manufacturing and regulatory efforts, including the
Company's plans to initiate, advance and complete its Phase 1/2 clinical trial
for LB-001 in MMA, and the value of and market for the Company's common stock,
will depend on future developments that are highly uncertain and cannot be
predicted with confidence at this time, such as the ultimate duration of the
pandemic, travel restrictions, quarantines, social distancing and business
closure requirements in the United States and in other countries, and the
effectiveness of actions taken globally to contain and treat the disease. These
risks are discussed in the Company's filings with the U.S. Securities and
Exchange Commission (SEC), including, without limitation, the Company's Annual
Report on Form 10-K filed on March 16, 2020 with the SEC, the Company's
Quarterly Report on Form 10-Q filed on May 11, 2020, and the Company's
subsequent Quarterly Reports on Form 10-Q and other filings with the SEC.
Except as required by law, the Company assumes no obligation to update these
forward-looking statements publicly, even if new information becomes available
in the future.
Contacts:
Investors:
Brian Luque
Associate Director, Investor Relations
bluque@logicbio.com
951-206-1200
Media:
Stephanie Simon
Ten Bridge Communications
Stephanie@tenbridgecommunications.com
617-581-9333
END
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