REG-Arix Bioscience PLC Clinical Development Update from Portfolio Company LogicBio

Arix BioscienceAnnouncement

09/05/2022 15:30

============

   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.

   ══════════════════════════════════════════════════════════════════════════

                                        

   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  1 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

    2 ir@arixbioscience.com  

    

   Powerscourt Group

   Sarah MacLeod, Ibrahim Khalil

   +44 (0)20 7250 1446

    3 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.  4 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, sAAVy™, 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  5 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.

   ══════════════════════════════════════════════════════════════════════════

   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

   ══════════════════════════════════════════════════════════════════════════

    6 fncls.ssp?fn=show_t_gif&application_id=1347399&application_name=news&site_id=refinitiv

References

   Visible links
   1. https://eqs-cockpit.com/cgi-bin/fncls.ssp?fn=redirect&url=7050c2abcf0f6ab3d5ed7180670d22d7&application_id=1347399&site_id=refinitiv&application_name=news
   2. mailto:ir@arixbioscience.com
   3. mailto:arix@powerscourt-group.com
   4. https://eqs-cockpit.com/cgi-bin/fncls.ssp?fn=redirect&url=0e82cce39f5bc66fe0ead5d02956d352&application_id=1347399&site_id=refinitiv&application_name=news
   5. https://eqs-cockpit.com/cgi-bin/fncls.ssp?fn=redirect&url=a10d3223e3d8aaf370f590d9a69baa52&application_id=1347399&site_id=refinitiv&application_name=news


============