First patient dosed in Achilles’ Phase I/II study in melanoma

First cell therapy to enter the clinic that specifically targets all cancer cells

Demonstrates Achilles’ capability to manufacture an entirely personalised T cell therapy

29 May 2020

Syncona Ltd, a leading healthcare company focused on founding, building and funding a portfolio of global leaders in life sciences, notes that its portfolio company, Achilles Therapeutics (‘Achilles’) has dosed the first patient in a Phase I/II clinical study of its clonal neoantigen T cell (cNeT) therapy in patients with recurrent or metastatic malignant melanoma. This tumour-infiltrating lymphocyte (TIL) therapy has been designed to target clonal neoantigens, believed to be present in all tumour cells, and is the first of its kind to enter clinical trials.

Achilles is developing personalised T cell therapies that target clonal neoantigens, the protein markers on the surface of all cancer cells that are unique from patient to patient. With the use of its bioinformatics platform, PELEUS™, and its proprietary TRACERx data set derived from cancer patients over many years, Achilles can identify a patient’s unique tumour profile and manufacture bespoke T cells called cNeTs that look to target and destroy tumours without harming healthy tissue.

The study is an open-label, multi-centre, single-dose Phase I/II trial designed to evaluate the safety, tolerability and clinical efficacy of cNeT therapy in patients with recurrent or metastatic malignant melanoma, for which there are limited treatment options. The trial, known as the THETIS study, expects to recruit approximately 20 patients and to report initial data in the first half of 2021.

Martin Murphy, Chief Executive of Syncona Investment Management Limited and Non-Executive Director of Achilles Therapeutics, said: “We are delighted that Achilles has dosed its first patient with its proprietary cNeT cell therapy. This is the first TIL therapy to enter clinical trials that is specifically designed to target all cancer cells, and that represents an exciting prospect for science and for patients. Importantly, today’s news demonstrates Achilles’ capability to manufacture an entirely personalised T cell therapy, and therefore its potential to deliver a commercially viable therapy that could dramatically improve patient outcomes. This is what motivates the Syncona team, it’s why we founded Achilles in 2016 and it’s why we remain an enthusiastic and engaged supporter of the business.”

About Syncona

Syncona is a leading FTSE250 healthcare company focused on founding, building and funding a portfolio of global leaders in life science. Our vision is to build a sustainable, diverse portfolio of 15 - 20 companies focused on delivering transformational treatments to patients in truly innovative areas of healthcare, through which we are seeking to deliver strong risk-adjusted returns for shareholders.

We seek to partner with the best, brightest and most ambitious minds in science to build globally competitive businesses. We take a long-term view, underpinned by a strategic capital base which provides us with control and flexibility over the management of our portfolio. We focus on delivering dramatic efficacy for patients in areas of high unmet need.

About Achilles Therapeutics

Achilles Therapeutics is a biopharmaceutical company developing personalised T cell therapies targeting clonal neoantigens: protein markers unique to the individual that are expressed on the surface of every cancer cell. Achilles uses DNA sequencing data from each patient, together with the proprietary PELEUS™ bioinformatics platform, to identify clonal neoantigens specific to that patient, and then develop personalised T cell-based therapies specifically targeting those clonal neoantigens.

Achilles was founded in 2016 by lead investor Syncona Ltd and in September 2019 the Company raised £100M in an oversubscribed Series B financing led by RA Capital, cornerstoned by Syncona and joined by new investors including Forbion, Invus, Perceptive Advisors and Redmile Group. For further information please visit the Company’s website at: www.achillestx.com

About Melanoma

Melanoma, the most serious form of skin cancer, is characterised by the uncontrolled growth of pigment-producing cells. It can be more serious than the other forms of skin cancer because of a tendency to metastasize to other parts of the body. The primary cause of melanoma is ultraviolet light (UV) exposure in those with low levels of the skin pigment melanin. Those with many moles, a history of affected family members and poor immune function are at greater risk. The number of people developing melanoma is rising. In 2020, over 196,000 Americans are expected to be diagnosed with melanoma. It is also now the 5th most common cancer in the UK with approximately 16,000 newly diagnosed cases each year.

About TRACERx

The TRACERx (TRAcking Cancer Evolution through therapy (Rx)) is a translational research study, led by Achilles founder, Professor Charles Swanton, aimed at transforming our understanding of cancer evolution and take a practical step towards an era of precision medicine.

Despite major advances in the understanding of cancer biology and the translation of these findings into novel therapeutics, the majority of patients with advanced melanoma fail to derive durable clinical benefit from existing standard-of-care therapies. Through integrative analysis of genomic and immunological landscapes, the Melanoma TRACERx study seeks to address this. Tumour specimens and peripheral blood are studied in highly relevant contexts at multiple stages of a patient’s treatment journey, from potentially curative resections of locally advanced disease, through to biopsies of lesions responding or refractory to systemic therapies in the setting of advanced disease.

Wherever possible, analyses will be performed in a longitudinal manner, allowing serial assessment of anti-tumour immunity, tumour-specific genomics and their interaction. Key objectives of the study include determination of spatial and temporal changes in immunological, genomic and transcriptomic landscapes, identification of novel molecular drivers, immunotherapeutic targets and assessment of the impact of cytotoxic, immune-modulatory and targeted therapies on both the tumour microenvironment and peripheral blood.