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    ARIX   GB00BD045071

ARIX BIOSCIENCE PLC

(ARIX)
  Report
Delayed London Stock Exchange  -  11:38 2022-11-30 am EST
103.00 GBX   -0.48%
11/15Alexion Pharmaceuticals, Inc. completed the acquisition of LogicBio Therapeutics, Inc. (NasdaqGM:LOGC) from a group of shareholders.
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11/02IN BRIEF: Arix Bioscience Chair Moncreiffe buys 78,000 more shares
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10/14Arix Bioscience Portfolio Company Imara To Merge With Enliven Therapeutics
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LogicBio announces clinical and corporate updates

04/27/2021 | 09:01am EST
Arix Bioscience PLC (ARIX) 
LogicBio announces clinical and corporate updates 
27-Apr-2021 / 14:00 GMT/BST 
Dissemination of a Regulatory Announcement, transmitted by EQS Group. 
The issuer is solely responsible for the content of this announcement. 
=---------------------------------------------------------------------------------------------------------------------- 
Arix Bioscience plc 
LogicBio announces clinical and corporate updates 
- First patient in Phase I/II SUNRISE trial expected to be enrolled by mid-year 2021 
- Company has entered into a strategic collaboration with CANbridge Pharmaceuticals and a research partnership with 
Daiichi Sankyo 
LONDON, 27 April 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. 
(Nasdaq:LOGC) today announced an update on screening and enrolment activities for the SUNRISE Phase I/II clinical trial 
 of LB-001, the company's investigational treatment for methylmalonic acidemia (MMA), a rare and life-threatening 
genetic disorder. 
LogicBio also announced that it has entered into a strategic collaboration with CANbridge Pharmaceuticals and a 
research partnership with Daiichi Sankyo. Under the terms of the agreement with CANbridge Pharmaceuticals, LogicBio is 
eligible to receive an upfront payment of USD10 million in addition to up to USD581 million in option payments and 
milestones payments, as well as up to double-digit royalties. 
The announcement can be accessed on LogicBio's website at: https://investor.logicbio.com/press-releases and full text 
of the announcement from LogicBio is contained below. 
[ENDS] 
 
For more information, 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 
+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 industry to a broader range of investors. www.arixbioscience.com 
 
 
 
 
LogicBio Therapeutics Announces Clinical and Corporate Updates 
 
- First patient for Phase I/II SUNRISE trial expected to be enrolled by mid-year 2021 
- Company also entered into strategic collaboration with CANbridge Pharmaceuticals and research partnership with 
Daiichi Sankyo 
LEXINGTON, Mass., April 27, 2021 -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine 
company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through 
adulthood, today announced an update on screening and enrollment activities for the SUNRISE Phase I/II clinical trial 
of LB-001, the company's investigational treatment for methylmalonic acidemia (MMA) based on LogicBio's proprietary 
gene insertion platform, GeneRide(TM). 
"The first two patients who entered pre-dosing screening activities did not meet all of the enrollment criteria for the 
SUNRISE trial, each for different reasons, and we will continue to screen additional patients in the coming weeks," 
said Daniel Gruskin, M.D., senior vice president and head of clinical development at LogicBio. "With COVID-19 
restrictions being lifted in some U.S.states, additional clinical trial sites have recently been activated and have 
begun recruiting and screening patients." 
"We expect to enroll our first patient in the SUNRISE trial by mid-year 2021, which represents a slight delay from our 
earlier communications," said Frederic Chereau, president and chief executive officer of LogicBio. "We are also pleased 
to report that we have pre-identified enough patients to fully enroll SUNRISE, assuming a certain number of screening 
failures." 
Today, LogicBio also announced a strategic collaboration with CANbridge Pharmaceuticals and research partnership with 
Daiichi Sankyo. 
LogicBio entered into a strategic collaboration with CANbridge Pharmaceuticals for an exclusive option to license 
LB-001 in Greater China (China, Taiwan, Hong Kong and Macau). The deal also grants CANbridge a worldwide license with 
respect to AAV sL65, the first capsid based on LogicBio's proprietary sAAVyTM platform, to support development and 
commercialization of CANbridge's gene therapy programs for Fabry disease and Pompe disease, with options for two 
additional indications. Under the terms of the agreement, LogicBio is eligible to receive an upfront payment of USD10 
million in addition to up to USD581 million in option payments and milestones payments, as well as up to double-digit 
royalties. A separate press release with more details can be found on LogicBio's investor relations website at 
investor.logicbio.com 
 
LogicBio also entered into a research collaboration with Daiichi Sankyo for the development of treatments for two 
indications based on GeneRide(TM). The agreement also grants Daiichi Sankyo an option to negotiate and acquire 
worldwide licenses to LogicBio's development programs in these indications. A separate press release with more details 
can be found on LogicBio's investor relations website at investor.logicbio.com. 
 
About LogicBio Therapeutics, Inc. 
LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms 
to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRide(TM) platform is 
a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable 
therapeutic protein expression levels. LogicBio's cutting-edge sAAVy(TM) capsid development platform is designed to 
support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. 
For more information, visit https://www.logicbio.com/. 
 
About the SUNRISE Trial 
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 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. With the aim of evaluating LB-001 at an early age, before irreversible tissue 
damage has occurred, the SUNRISE trial is initially enrolling pediatric patients from 3 -12 years old, with the 
possibility to include patients as young as 6 months old after meeting certain safety and biomarker parameters. The 
SUNRISE trial will evaluate two doses of LB-001. Patients will participate in a pre-dosing observational period, will 
be administered a prophylactic steroid regimen, and will be followed for 52 weeks. Secondary and exploratory 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. 
About LB-001 
GeneRide(TM) 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 are associated with an increased risk 
of immune response and cancer. LB-001 is a first-in-class, single-dose gene-editing therapy for early intervention in 
methylmalonic acidemia (MMA) designed using the GeneRide(TM) platform to insert a corrective copy of the MMUT gene into 
the ALB 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 via an engineered rAAV vector utilizing the highly efficient and 
selective liver-targeted LK03 capsid (rAAV-LK03). Preclinical assessment in human hepatocytes demonstrated that LB-001 
is capable of site-specific insertion. Preclinical in vivo studies found LB-001 to be safe and demonstrated 
transduction of hepatocytes, genomic integration, and liver 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. 
About Methylmalonic Acidemia (MMA) 
Methylmalonic acidemia, or MMA, is a rare and life-threatening genetic disorder, affecting 1 in 25,000 to 50,000 
newborns. In the most common form of MMA, a mutation in a gene called 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 cure for MMA. 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 cause 
permanent neurocognitive damage. 
Forward-Looking Statements 
This press release contains "forward-looking" statements within the meaning of the federal securities laws, including 
with respect to the Company's expectation regarding patient enrollment in the SUNRISE trial, its strategic 
collaboration with CANbridge Pharmaceuticals, its research partnership with Daiichi Sankyo and the potential of the 
GeneRide(TM) platform. 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 

(MORE TO FOLLOW) Dow Jones Newswires

April 27, 2021 09:00 ET (13:00 GMT)

Stocks mentioned in the article
ChangeLast1st jan.
ARIX BIOSCIENCE PLC -0.48% 103 Delayed Quote.-15.57%
DAIICHI SANKYO CO., LTD. 1.81% 4594 Delayed Quote.55.08%
DOW JONES INDUSTRIAL 2.18% 34589.77 Real-time Quote.-6.84%
MSCI TAIWAN (STRD) 1.24% 533.406 Real-time Quote.-29.96%
TRANSGENE 0.00% 1.82 Real-time Quote.-28.35%
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Yield 2022 -
Capitalization 129 M 154 M 154 M
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Capi. / Sales 2023 -
Nbr of Employees 7
Free-Float 69,0%
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Robert William Henry Lyne CEO, COO, Director & General Counsel
Nikki Edgar Head-Finance
Peregrine D. E. M. Moncreiffe Non-Executive Chairman
Debra Stephanie Barker Senior Independent Non-Executive Director
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