REG-Arix Bioscience PLC Arix Bioscience PLC: Clinical development update from portfolio company LogicBio

Arix BioscienceAnnouncement

02/02/2022 13:10

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   Arix Bioscience PLC (ARIX)
   Arix Bioscience PLC: Clinical development update from portfolio company
   LogicBio

   02-Feb-2022 / 13:05 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

                                        

          Clinical development update from portfolio company LogicBio

    

   LONDON, 2 February 2021: Arix Bioscience plc ("Arix", LSE:ARIX), a  global
   venture capital company focused on investing in and building  breakthrough
   biotech companies,  notes the  update today  from its  portfolio  company,
   LogicBio  Therapeutics,  Inc.  (Nasdaq:  LOGC)  on  its  LB-001   clinical
   development programme.

    

   LogicBio announced that the U.S. Food and Drug Administration (FDA) has
   notified the company that its Phase 1/2 SUNRISE clinical trial of LB-001
   in paediatric patients with methylmalonic acidemia (MMA) has been placed
   on clinical hold. The company will host a conference call and webcast at
   08:30 ET (13:30 GMT) to discuss the update.

    

   As of close of business on 1 February 2022 Arix held 2,716,211 shares in
   LogicBio.

    

   The full announcement from LogicBio is set out below and can be
    1 accessed here.

    

    

                                      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 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.  4 www.arixbioscience.com

    

                                        

                             LogicBio Press Release

                                        

      LogicBio Therapeutics Provides Update on LB-001 clinical Development
                                    Program

      - Company to host conference call and webcast today at 8:30 a.m. ET

    

   LEXINGTON, Mass., Feb. 2, 2022 /PRNewswire/ -- LogicBio Therapeutics,
   Inc. (Nasdaq: LOGC), a clinical-stage genetic medicine company, today
   provided an update on the LB-001 clinical development program. The U.S.
   Food and Drug Administration (FDA) has notified the company that its Phase
   1/2 SUNRISE clinical trial of LB-001 in pediatric patients with
   methylmalonic acidemia (MMA) has been placed on clinical hold. The company
   will host a conference call and webcast this morning to discuss this
   update.

    

   To date, four patients have been dosed in the SUNRISE trial with LB-001,
   LogicBio's investigational, single-administration, adeno-associated virus
   (AAV) genome editing therapy. In accordance with the FDA-cleared protocol,
   the first two patients dosed were in the older age group (3 to 12 years
   old) and received 5 x 1013 vg/kg of LB-001. These first two patients are
   doing well, have not experienced drug-related serious adverse events
   (SAEs), and are being monitored in accordance with the protocol.

    

   As previously disclosed, the third patient dosed in the SUNRISE trial, who
   received 5 x 1013 vg/kg of LB-001 and is in the younger age group (6
   months to 2 years old), experienced a drug-related SAE, which was
   categorized as a case of thrombotic microangiopathy (TMA). TMA has been
   previously reported in association with other AAV genetic therapies. The
   patient was hospitalized and responded well to intravenous fluids and
   parenteral nutrition. Following this SAE, the company implemented
   additional safety measures in the SUNRISE trial, and reported the SAE to
   the FDA and the Data Safety Monitoring Board for the trial (DSMB).
   In December 2021, the company announced that the SAE experienced by the
   third patient had resolved.

    

   In January 2022, the fourth patient dosed in the SUNRISE trial, who
   received 5 x 1013 vg/kg of LB-001 and is in the younger age group,
   experienced a drug-related SAE, which was categorized as a case of TMA.
   The patient is being closely followed by the patient's care team and has
   been steadily improving. The company reported the SAE to the FDA and the
   DSMB. The FDA subsequently notified the company that the SUNRISE trial has
   been placed on clinical hold. LogicBio will be working closely with the
   FDA and the DSMB to determine the next steps for the SUNRISE trial and the
   LB-001 program.

    

   "Patient safety is our first priority. I would like to thank the patient
   and the patient's family for participating in our trial as well as the
   on-site team for the excellent care they are providing," said Fred
   Chereau, president and chief executive officer of LogicBio. "We look
   forward to working closely with the FDA and the DSMB to determine the next
   steps for the trial and the program." 

    

   Until the company has more clarity regarding the impact of the clinical
   hold, LogicBio is suspending guidance on the timing of announcing interim
   data for the SUNRISE trial. 

    

   Conference Call and Webcast Details

   LogicBio will host a conference call and webcast today, Wednesday,
   February 2, 2022, at 8:30 a.m. ET to discuss the program update. To listen
   to the conference call, please dial +1 (833) 519-1335 (domestic) or +1
   (602) 585-9978 (international) using conference ID number 7545016. A live
   webcast of the call can be accessed via the Investors section of the
   company's website at  5 https://investor.logicbio.com. A webcast replay
   will be available following the call and archived for approximately 30
   days.

    

   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 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  6 www.logicbio.com,
   which does not form a part of this release.

    

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   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.:   140506
   EQS News ID:    1275905


    
   End of Announcement EQS News Service

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