Autolus Therapeutics, a UCL spinout company, 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 and means the drug is licensed to be sold in the UK. 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 $1B, with most of this invested in the UK, including the development of a state-of-the-art manufacturing facility, The Nucleus in Stevenage, employing 450 people.
Dr Anne Lane, CEO of UCL Business commented: "This is a major milestone for Autolus as a groundbreaking UK-developed cancer treatment becomes licensed for UK patients for the first time.
“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.
Dr Christian Itin, Chief Executive Officer of Autolus said: “Continuing our momentum, this MHRA licence 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 94 patients, 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.”
Professor Karl Peggs, Director of the UCLH BRC, said: “It has been fantastic to see the progression of a potentially life-saving therapy from our academic laboratories through to industry-led regulatory approvals. I’m particularly glad for Martin Pule, who has pushed CAR-T therapies forward within the UK over the past decade. These kinds of successes are rare and should be celebrated. The key next step is to gain NICE approval for re-imbursement so that we can hopefully move swiftly to providing this therapy as a standard of care within the NHS.”
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 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 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.
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