Important next step for Revolutionary T-cell treatment for Acute Lymphoblastic Leukemia, discovered at UCL

Autolus Therapeutics submits FDA application in the U.S and confirms it is on track with a marketing licence application in EU.

A ground-breaking new therapy for adults with relapsed B-Cell Acute Lymphoblastic Leukemia could be a step closer to market, following a ‘Biologics Licence Application’ being submitted to the FDA by licencee Autolus Therapeutics.

Obe-cel (abecabtagene autoleucel) is a T-cell therapy that involves engineering precisely targeted, controlled and highly active T-cells designed to better recognise target cancer cells, break down their defence mechanisms and eliminate them.

The Biologics Licence Application is based on data from the pivotal Phase 2 FELIX study of obe-cel, involving over 100 patients across 30 leading academic and non-academic centres in the United States, U.K and Europe. The data, which were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in June 2023, will be updated at the upcoming Annual Meeting of the American Society for Hematology Meeting in December in San Diego.

The T-cell technology on which obe-cel evolved was from research conducted by Dr Martin Pule and his team at the UCL Cancer Institute and NIHR University College London Hospitals Biomedical Research Centre.

To realise the potential of the research, UCL Business (UCLB), UCL’s commercialisation company, created spinout company Autolus in September 2014. Dr Richard Fagan, UCLB’s Director of BioPharm worked with Dr Pule to protect the technology, securing a suite of patents, supporting the setup of Autolus and helping attract additional funding.

Dr Fagan said: “This is another big milestone for one of most eagerly-anticipated treatments for Leukaemia, and one which UCLB has worked on for almost a decade. It can be a lengthy business supporting such pioneering new therapies through to market to benefit patients, so it is tremendously exciting to see obe-cel take its next steps towards that goal.”

Programming T cells to fight cancer

T-cells are a naturally occurring part of our immune system that normally kill infected cells.  But in cancers, it can be difficult for these cells to recognise the difference between a cancer cell and a normal one. So the cancer can hide away and go unnoticed.

However, thanks to new research, scientists have found a way to reprogram T-cells so they can recognise and fight cancer cells in the body, just as they would naturally attack an infection.

This programming works by taking T-cells from a person’s blood sample, and introducing a gene for an artificial protein called a chimeric antigen receptor, or CAR.

The cells can then be programmed to develop precisely targeted T-cell therapies that are capable of recognising cancer cells, breaking down their defences, and killing them.

From bench to bedside
Autolus are now using their expertise to develop advanced T-cell therapies that have the potential to deliver life-changing benefits in a range of cancers.

The breadth of the Autolus technology platform which was licenced from UCLB at company formation allows the company to select from a range of T-cell programming options. This modular approach will facilitate the development of a suite of therapies to address a range of target cancers, and to improve already-established therapies.

Four products are currently in development to treat cancers that start in blood-forming tissue (such as leukaemia and lymphoma), as well as one solid tumour programme.

About Autolus Therapeutics plc
Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer and autoimmune disease. 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 target 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, solid tumors and autoimmune diseases. For more information, please visit www.autolus.com.

About obe-cel (AUTO1)
Obe-cel is a CD19 CAR T cell investigational therapy designed to overcome the limitations in clinical activity and safety compared to current CD19 CAR T cell therapies. Obe-cel is designed with a fast target binding off-rate to minimize excessive activation of the programmed T cells. Clinical trials of obe-cel have demonstrated that this “fast off-rate” profile reduces toxicity and T cell exhaustion, resulting in improved persistence and leading to high levels of durable remissions in r/r Adult ALL patients. The results of the FELIX trial, a pivotal trial for adult ALL, are being prepared for regulatory submissions with the FDA and EMA. In collaboration with Autolus’ academic partner, UCL, obe-cel is currently being evaluated in a Phase 1 clinical trials for B-NHL.

About obe-cel FELIX clinical trial
Autolus’ Phase Ib/II clinical trial of obe-cel enrolled adult patients with relapsed / refractory B-precursor ALL. The trial had a Phase Ib component prior to proceeding to the single arm, Phase II clinical trial. The primary endpoint is overall response rate, and the secondary endpoints include duration of response, MRD negative CR rate and safety. The trial enrolled over 100 patients across 30 of the leading academic and non-academic centers in the United States, United Kingdom and Europe. [NCT04404660]

Further information: 

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