18 September 2017

Autolus commences clinical trial programmes


Syncona Ltd (“Syncona”), a leading healthcare company focused on investing in and building global leaders in life science, today notes that its portfolio company, Autolus, which is developing and commercialising next-generation engineered T-cell therapies to combat cancer, has announced the commencement of three clinical trials in its two lead programmes, AUTO2 and AUTO3.

In its AUTO2 APRIL study in multiple myeloma, Autolus has announced the initiation and completion of the first dose cohort of its Phase I/II study of its dual-targeted Chimeric Antigen Receptor (CAR) T cell therapy in patients with relapsed/refractory multiple myeloma. AUTO2 is the first dual targeting CAR-T cell therapy in clinical development for the treatment of multiple myeloma.


Autolus has also initiated its AUTO3 programme with two Phase I/II studies, AMELIA in pediatric Acute Lymphoblastic Leukaemia and ALEXANDER in adult Diffuse Large B Cell Lymphoma. AUTO3 is the first dual targeting CAR T cell therapy to enter clinical studies targeting CD19 and CD22 with independently-acting CARs.




Syncona Ltd

Siobhan Weaver

Tel: +44 (0) 20 7611 2031


Tulchan Communications

Martin Robinson

Lisa Jarrett-Kerr

Tel: +44 (0) 207 353 4200

Copies of this press release and other corporate information can be found on the company website at: www.synconaltd.com  

About Syncona:

Syncona is a leading FTSE250 healthcare company focused on investing in and building global leaders in life science. Our vision is to deliver transformational treatments to patients in truly innovative areas of healthcare while generating superior returns for shareholders. Our current investment portfolio consists of seven high quality companies in life science and a leading range of fund investments.  We seek to partner with the best, brightest and most ambitious minds in science to build globally competitive businesses. We are established leaders in gene therapy, cell therapy and advanced diagnostics, and focus on delivering dramatic efficacy for patients in areas of high unmet need.

Our market leading funds portfolio seeks to generate superior returns by investing in long only and alternative investment funds. This represents a productively deployed evergreen funding base which enables us to take a long term approach to investing in life sciences as we target the best new opportunities and support our existing portfolio companies to grow and succeed.

Syncona is aligned with two of the premium charitable funders in UK science, the Wellcome Trust, original founder of Syncona, and Cancer Research UK, both of which are significant shareholders in our business.  We make a donation of 0.3% of Net Asset Value to a range of charities each year.

About Autolus:

Autolus is a clinical-stage, biopharmaceutical company, focused on the development and commercialisation of engineered T-cell immunotherapy products to combat cancer. Building on its advanced cell programming and manufacturing technologies, Autolus has developed a pipeline of product candidates for the treatment of both haematological malignancies and solid tumours. For further information please visit the Company’s website at: www.autolus.com

About AUTO2 and the APRIL study:

AUTO2 is a chimeric antigen receptor T cell (CAR-T cell) therapy that targets both B cell maturation antigen (BCMA) and transmembrane activator and calcium modulator and cyclophilin ligand interactor (TACI). By targeting TACI in addition to BCMA on the same cancer cell, more patients may be eligible for CAR-T treatment and less patients may be at risk of cancer relapse due to loss of BCMA expression on their cancer. In addition, AUTO2 also carries an RQR8 safety switch which allows the T cells to be removed with a single high dose of rituximab.

The APRIL Study is a single-arm, open-label, multi-centre, Phase I/II Study evaluating the safety and clinical activity of AUTO2, a CAR-T Cell Treatment Targeting BCMA and TACI, in patients with relapsed or refractory multiple myeloma. In the dose-escalation phase I/II study cohorts of patients will receive ascending doses of AUTO2 to determine the maximum tolerated dose and establish a recommended dose. The second part of the study is an expansion phase where patients will receive AUTO2 to further evaluate the safety, tolerability and clinical activity at this recommended dose.

Multiple myeloma is a type of blood cancer that affects the plasma cells and is the second most commonly diagnosed blood cancer, after non-Hodgkin lymphoma. There are a number of approved therapies to treat the disease but there is currently no cure.

About AUTO3 and the AMELIA and ALEXANDER studies:

AUTO3 is an autologous T cell product, genetically modified to express two separate chimeric antigen receptors (CARs) which recognise CD19 and CD22, two antigens expressed by cancer cells in B cell leukaemia and lymphoma. AUTO3 is designed to minimize the risk of relapse due to antigen loss, a key mechanism of resistance shown in single antigen targeting CAR-T therapies.

The AMELIA study is is a single‑arm, open-label, multi-centre, phase I/II study evaluating the safety and clinical activity of AUTO3 in paediatric and young adult patients with relapsed or refractory B Cell Acute Lymphoblastic Leukaemia (ALL). The ALEXANDER Study is a Phase I/II, open-label, multi-centre study to evaluate the safety and efficacy of AUTO3 administered by intravenous infusion in adult Diffuse Large B Cell Lymphoma (DLBCL) patients. The studies are dose-escalation phase I/II trials in which cohorts of patients will receive ascending doses of AUTO3 to determine the maximum tolerated dose and establish a recommended dose. The second part of the study is an expansion phase where patients will receive AUTO3 to further evaluate the safety, tolerability and clinical activity at this recommended dose. In addition to the effects of AUTO3 alone, combination with short-duration use of a checkpoint inhibitor is also being evaluated in the ALEXANDER Study.

ALL is a cancer of the bone marrow and blood, in which the body makes abnormal white blood cells (lymphocytes). The disease progresses quickly and is the most common childhood cancer in the U.S and EU. DLBCL is an aggressive type of non-Hodgkin lymphoma (NHL) that develops from the B cells in the lymphatic system. It is a rapidly growing blood cancer, which can occur in lymph nodes or outside of the lymphatic system, in the brain, bone, breast, skin, thyroid, gastrointestinal tract, and testes.

About CAR-T therapy:

CAR-T therapy involves re-programming a patient’s immune system to kill tumour cells. T-cells, a type of white blood cell, are extracted from a patient’s blood, manipulated outside the body to incorporate the CAR gene, and then returned to the patient by infusion. The CAR gene introduces a targeting mechanism to the T-cells, enabling them to recognise, engage and destroy tumour cells in a highly specific manner. Clinical trials of CAR T-cells in multiple myeloma and B-cell malignancies, including non-Hodgkin lymphoma, suggest that this approach may transform treatment of cancer patients, many of whom have no other therapeutic options.

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