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      Arix Bioscience plc: Autolus Announces Updated Results From CARPALL Trial


      News provided by

      United Business Media

      Feb 19, 2019, 09:08 ET


      Arix Bioscience plc (LSE: ARIX) ("Arix"), a global healthcare and life science company
      supporting medical innovation, notes that its portfolio company, Autolus Therapeutics plc
      (NASDAQ: AUTL), has presented updated data from the ongoing Phase 1 CARPALL trial of AUTO1
      in a poster presentation at the European Hematology Association 1st European CAR T Cell
      Meeting held in Paris, France, February 14-16, 2019.  
      
          The announcement can be accessed on Autolus' investor website at
      https://www.autolus.com/investor-relations and the full text of the announcement from
      Autolus is contained below.
      
          About Arix Bioscience plc 
      Arix Bioscience plc is a global healthcare and life science company supporting medical
      innovation. Headquartered in London and with an office in New York, Arix Bioscience
      sources, finances and builds world class healthcare and life science businesses addressing
      medical innovation at all stages of development. Operations are supported by privileged
      access to breakthrough academic science and strategic relationships with leading research
      accelerators and global pharmaceutical companies.
      
          Arix Bioscience plc is listed on the Main Market of the London Stock Exchange. For
      further information, please visit http://www.arixbioscience.com
      
          Autolus Therapeutics Announces Updated Results From Ongoing CARPALL Trial of Pediatric
      Acute Lymphoblastic Leukemia Presented at the EHA 1st European CAR T Cell Meeting in Paris
      
                            Updated results confirm AUTO1 data from ASH 2017 
      
                            No severe cytokine release syndrome (Grade 3-5) 
      
                   86% molecular complete response rate after a single dose of AUTO1  
      
          LONDON, UK, February 19, 2019 -- Autolus Therapeutics plc (NASDAQ: AUTL), a
      clinical-stage biopharmaceutical company developing next-generation programmed T cell
      therapies for the treatment of cancer, today announced that Professor Persis Amrolia,
      Consultant in Bone Marrow Transplant at Great Ormond Street Hospital (GOSH) and NIHR
      Research Professor of Transplantation Immunology at University College London (UCL) Great
      Ormond Street Institute of Child Health, presented updated data from the ongoing Phase 1
      CARPALL trial of AUTO1 in a poster presentation at the European Hematology Association 1st
      European CAR T Cell Meeting held in Paris, France, February 14-16, 2019.  
      
          Enrolled patients had a median age of 9 years with a median of 4 lines of prior
      treatment. Seventeen patients were enrolled, and 14 patients received an infusion of CAR T
      cells. Ten of 14 patients had relapsed post allogeneic stem cell transplant. Eight
      patients were treated in second relapse, 5 in >second relapse and 3 had relapsed after
      prior blinatumomab or inotuzumab therapy. Two patients had ongoing CNS disease at
      enrollment.
      
          Updated safety results 
      
          This data set confirms that AUTO 1 induces no severe cytokine release syndrome (CRS)
      (Grade 3-5). Nine patients experienced Grade 1 CRS, and 4 patients experienced Grade 2
      CRS.  No patients required tociluzumab or steroids. As previously reported, one patient
      experienced Grade 4 neurotoxicity; there were no other reports of severe neurotoxicity
      (Grade 3-5). The mean cumulative exposure to AUTO1 CAR T cells in the first 28 days as
      assessed by AUC was 1,721,355 copies/microg DNA. Eleven patients experienced cytopenia
      that was not resolved by day 28 or recurring after day 28: 3 patients Grades 1-3 and 8
      patients Grade 4. Two patients developed significant infections, and 1 patient died from
      sepsis while in molecular complete response (CR).  
      
          Updated efficacy results 
      
          With a single dose of CAR T cells at 1 million cells/kg dose, 12/14 (86%) achieved
      molecular CR.  
      
          Five patients relapsed with CD19 negative disease. Event free survival (EFS) based on
      morphological relapse was 67% (CI 34-86%) and 46% (CI 16-72%) and overall survival (OS)
      was 84% (CI 50-96%) and 63% (CI 27-85%) at 6 and 12 months, respectively.
      
          CAR T cell expansion was observed in all responding patients (N=12), with CAR T cells
      comprising up to 84% of circulating T cells at the point of maximal expansion. The median
      persistence of CAR T was 215 days.
      
          The median duration of remission in responding patients was 7.3 months with a median
      follow-up of 14 months. Five of 14 patients (37%) remain in CR with ongoing persistence of
      CAR T cells and associated B cell aplasia.  
      
          "AUTO1 combines a high molecular CR rate with excellent persistence and a good safety
      profile in pediatric acute B cell leukemia patients," said Professor Amrolia.
      
          About AUTO1 
      
          AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the
      limitations in safety - while maintaining similar levels of efficacy - compared to current
      CD19 CAR T cell therapies.  Designed to have a fast target binding off-rate to minimize
      excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less
      prone to T cell exhaustion, which could enhance persistence and improve the T cells'
      abilities to engage in serial killing of target cancer cells. In 2018, Autolus signed a
      license agreement under which Autolus acquired global rights from UCL Business plc (UCLB),
      the technology-transfer company of UCL, to develop and commercialize AUTO1 for the
      treatment of B cell malignancies. AUTO1 is currently being evaluated in two Phase 1
      studies, one in pediatric ALL and one in adult ALL.
      
          For information about the CARPALL trial, visit
      https://clinicaltrials.gov/ct2/show/NCT02443831
      
          About Pediatric Acute Lymphoblastic Leukemia (ALL) 
      
          According to the American Cancer Society, ALL is the most common cancer diagnosed in
      children, with approximately 3,400 new cases diagnosed in the United States each year.
      Pediatric ALL occurs when the bone marrow makes too many immature lymphocytes, which are a
      type of white blood cell. The current standard of care for pediatric ALL patients is
      combination chemotherapy. Although pediatric patients typically respond well to first-line
      treatment, 10 to 20% of total patients relapse with chemotherapy-resistant disease,
      leading to a significant unmet need in pediatric patients with high-risk relapsed or
      refractory ALL.
      
          About Autolus Therapeutics plc 
      
          Autolus is a clinical-stage biopharmaceutical company developing next-generation,
      programmed T cell therapies for the treatment of cancer. Using a broad suite of
      proprietary and modular T cell programming technologies, the company is engineering
      precisely targeted, controlled and highly active T cell therapies that are designed to
      better recognize cancer cells, break down their defense mechanisms and eliminate these
      cells. Autolus has a pipeline of product candidates in development for the treatment of
      hematological malignancies and solid tumors. For more information please
      visit http://www.autolus.com
      [https://c212.net/c/link/?t=0&l=en&o=2345145-1&h=2243192194&u=http%3A%2F%2Fwww.autolus.com%2F&a=www.autolus.com ]
      .
      
          Forward-Looking Statements 
      
          This press release contains forward-looking statements within the meaning of the "safe
      harbor" provisions of the Private Securities Litigation Reform Act of 1995.
      Forward-looking statements are statements that are not historical facts, and in some cases
      can be identified by terms such as "may," "will," "could," "expects," "plans,"
      "anticipates," and "believes." These statements include, but are not limited to,
      statements regarding the company's product candidates and research programs. Any
      forward-looking statements are based on management's current views and assumptions and
      involve risks and uncertainties that could cause actual results, performance or events to
      differ materially from those expressed or implied in such statements. For a discussion of
      other risks and uncertainties, and other important factors, any of which could cause our
      actual results to differ from those contained in the forward-looking statements, see the
      section titled "Risk Factors" in the company's Annual Report on Form 20-F filed
      on November 23, 2018 as well as discussions of potential risks, uncertainties, and other
      important factors in the company's future filings with the Securities and Exchange
      Commission from time to time. All information in this press release is as of the date of
      the release, and the company undertakes no obligation to publicly update any
      forward-looking statement, whether as a result of new information, future events, or
      otherwise, except as required by law.
      
      
      
      The content and accuracy of news releases published on this site and/or
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      accuracy of our services, such releases are not actively monitored or reviewed
      by PR Newswire or its partners and under no circumstances shall PR Newswire or
      its partners be liable for any loss or damage resulting from the use of such
      information. All information should be checked prior to publication.

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