Autolus gains UK licence for cancer therapy

UCLB spinout Autolus Therapeutics has received authorisation from the UK Medicines and Healthcare products Regulatory Agency (MHRA), for a next-generation CAR T-cell therapy developed to treat adults with an aggressive blood cancer.

The personalised therapy, named Obecabtagene autoleucel (obe-cel) and marketed as AUCATZYL®, is for adult patients (≥ 18 years) with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (r/r B-ALL).

It is the biopharmaceutical company’s first MHRA authorisation. The licence follows an equivalent decision made by the U.S. Food and Drug Administration (FDA) in November last year.

Autolus Therapeutics was founded by Dr Martin Pule, based at UCL Cancer Institute, and, with the support of UCL Business (UCLB), UCL’s commercialisation company, was spun out in 2014.
It has since raised over $1bn, with most of this invested in the UK, that includes a state-of-the-art manufacturing facility, The Nucleus in Stevenage, employing 450 people.

Anne Lane, CEO of UCL Business commented: “This is a major milestone for Autolus. It shows that supporting academics to take their great research from the lab to the market benefits us all, through life-changing impacts and by creating jobs, growth and inward investment. It’s also a clear demonstration of how university innovation fuels our world-leading life sciences ecosystem – delivering for patients and driving the UK’s future economic growth.”

Working with Autolus, UCLB helped protect the IP for the CAR-T obe-cel technology and attract investment to enable the business to take AUCATZYL® through clinical trials.

Richard Fagan, Director of BioPharm, UCLB, added: “The close relationship between Autolus and UCLB has spanned more than a decade since UCLB helped establish the company in 2014. I’m delighted about the approval of AUCATZYL in the UK which represents a huge milestone in Autolus’ success story. It also underscores the important role of long-term capital investment and other support in helping university spinouts bring advanced therapeutics from the research lab to the market where they can benefit patients.”

Dr. Christian Itin, Chief Executive Officer of Autolus said: “Continuing our momentum, this MHRA license is a significant milestone for Autolus as a company. With our scientific expertise, operations and manufacturing based in the UK, this is an important achievement for our company.”

Around 8,400 new cases of ALL are diagnosed every year in the US and EU. Of these, over 3,000 will either relapse or not respond to standard treatment. For adult patients with r/r ALL, prognosis is poor with a median overall survival of eight months.

Obe-cel was authorised by the MHRA based on results from the multi-centre Autolus FELIX clinical trial in adult patients with r/r B-ALL, which was published in the New England Journal of Medicine in November 2024. In the pivotal cohort of patients (n=94), 77% achieved overall complete remission, representing the elimination of all signs of cancer in response to treatment.

Dr Claire Roddie, lead investigator of the FELIX study and Associate Professor of Haematology at UCL Cancer Institute, said: “Having treated a number of patients with AUCATZYL as part of the FELIX clinical trial, I am delighted that we have moved closer to eligible relapsed/refractory B-ALL patients being able to access AUCATZYL.”

Dr Roddie added: “We now look forward to NICE completing its assessment of the medicine to potentially make it an option for eligible patients on the NHS.”
Dr Martin Pule, UCL Cancer Institute and Chief Scientific Officer and Founder of Autolus, said: “AUCATZYL was designed to address an unmet need for eligible adult r/r B-ALL patients and it is satisfying that is has been licensed in the country where it was created.”

AUCATZYL will be manufactured at Autolus’ commercial manufacturing site in Stevenage, which will supply the therapy globally.

Autolus submitted obe-cel/ AUCATZYL® for appraisal by the National Institute for Health and Care Excellence (NICE) in Q4 2024 and is working with NICE and the NHS to potentially achieve access for patients in England. NICE provides guidance to the NHS in England on the clinical and cost-effectiveness of medicines, treatments, and technologies based on a rigorous process of evidence review and consultation with professionals and patients.

A marketing authorisation application (MAA) for obe-cel/ AUCATZYL® in adult R/R B-ALL is also under review with regulators in the EU, with a submission to the European Medicines Agency (EMA) accepted in March 2024.

What is CAR T-cell therapy?
CAR T-cell therapy involves collecting a patient’s own white blood cells (T-cells, responsible for fighting infection), ‘reprogramming’ them in the laboratory to seek and ‘fight’ the cancer cells and giving them back to the patient via infusion.

For the FELIX trial, patients had their T cells genetically modified with a new type of CAR called obecabtagene autoleucel or obe-cel.
This treatment programmes immune T cells to make an artificial protein called a CD19 chimeric antigen receptor (CAR) on their surface, directing them to specifically recognise cancerous cells.
Obe-cel has been designed to overcome two common constraints associated with ‘first generation’ CAR T-cell therapies.

One problem was that the immune system became over-activated, causing a toxic reaction called ‘cytokine release syndrome.’ The other problem was that T-cells were not able to persist in a patient’s body.

Development of obe-cel/AUCATZYL®
Obe-cel was designed by scientists from UCL Cancer Institute, along with collaborators at the UCL Great Ormond Institute of Child Health and UCLH. Dr Pule leads the UCL CAR-T cell programme, which is supported by the National Institute for Health and Care Research UCLH Biomedical Research Centre (BRC).

The manufacture of obe-cel was developed at the Centre for Cell, Gene & Tissue Therapeutics, Royal Free Hospital. Clinical development in adult B-ALL was built on the ALLCAR19 clinical study, led by UCL and UCLH, and supported by grant funding from the NIHR.