Arix Bioscience plc LogicBio announces IND clearance for LB-001

 
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 
[phone] or [phone], Conference ID: 1151546. 
 
[S] 
 
Enquiries 
 
For more information on Arix, please contact: 
 
Arix Bioscience plc 
Charlotte Parry, Head of Investor Relations 
[phone] 
[e-mail] 
 
Optimum Strategic Communications 
Mary Clark, Supriya Mathur, Shabnam Bashir 
T: [phone] 
[e-mail] 
 
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 [phone] or [phone], 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 

[e-mail] 
951-206-1200 
 
Media: 
Stephanie Simon 
Ten Bridge Communications 
[e-mail] 
617-581-9333 
 
 
 
END 
 

(END) Dow Jones Newswires

August 10, 2020 07:49 ET (11:49 GMT)