REG-Arix Bioscience PLC LogicBio Therapeutics Announces Early Clinical Trial Results Demonstrating First-Ever In Vivo Genome Editing in Children

Arix BioscienceAnnouncement

18/10/2021 16:50

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   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

    1 ir@arixbioscience.com  

    

   Optimum Strategic Communications

   Mary Clark, Manel Mateus

   +44 (0)20 3922 1906

    2 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.  3 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 screened for  MMA
   in every state in the United States.

    

   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(TM),  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, sAAVy(TM),  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.

    

    

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   ISIN:           GB00BD045071
   Category Code:  MSCH
   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.:   124624
   EQS News ID:    1241592


    
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