Arix Bioscience plc Autolus announces updated results from CARPALL trial

 
TIDMARIX 
 
Arix Bioscience plc 
 
             Autolus announces updated results from CARPALL trial 
 
LONDON, 19 February 2019: 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. 
 
[ENDS] 
 
For more information on Arix, please contact: 
 
Arix Bioscience plc 
Charlotte Parry, Head of Investor Relations 
[phone] 
[e-mail] 
 
Optimum Strategic Communications 
Mary Clark, Supriya Mathur 
[phone] 
[e-mail] 
 
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 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/µg 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  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. 
 
Investor contact: 
Susan A. Noonan 
S.A. Noonan Communications 
[phone] 
[e-mail] 
 
Media contact: 
Silvia Taylor 
Vice President, Corporate Affairs and Communications, Autolus 
[phone] 
[e-mail] 
 
Julia Wilson 
JW Communications 
[phone] 
[e-mail] 
 
 
 
END 
 

(END) Dow Jones Newswires

February 19, 2019 07:12 ET (12:12 GMT)