AVA6000 receives Orphan Drug Designation from FDA

5 September 2022

Avacta Group plc

("Avacta" or "the Group" or "the Company")

AVA6000 receives Orphan Drug Designation from the US Food and Drug Administration

Avacta Group plc (AIM: AVCT), a clinical stage oncology drug company and developer of powerful diagnostics based on its innovative Affimer^® and pre|CISION™ platforms, announces that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the company's lead pre|CISION drug candidate, AVA6000, for treatment of soft tissue sarcoma.

AVA6000 is a form of the generic chemotherapy doxorubicin that has been modified using the pre|CISION technology so that it is activated predominantly in the tumour with the aim of sparing healthy tissue from exposure and improving the safety, tolerability and efficacy of the drug. Doxorubicin has a market size that is expected to grow to $1.38bn by 2024, and is widely used as part of the standard of care in several cancer types. Avacta has an ongoing Phase 1 clinical trial to assess the safety and pharmacokinetics of AVA6000 which has potential as a treatment for patients with a range of cancer types, including soft tissue sarcoma.

The FDA can grant ODD based on a review of preclinical data from investigational treatments for rare diseases, such as soft tissue sarcoma, which are defined as conditions affecting fewer than 200,000 people in the US. This designation qualifies the developer of the drug for certain incentives, including, seven years of market exclusivity upon drug approval from the FDA.

Soft-tissue sarcoma is a rare mesenchymal malignancy which accounts for less than 1% of all adult tumours. Despite the successful advancement of localised therapies, such as surgery and radiotherapy, these tumours can recur, often with metastatic disease. The American Cancer Society estimates that, in 2022 approximately 13,190 new soft tissue sarcomas will be diagnosed, and about 5,130 people are expected to die of the disease in the US.

Dr Alastair Smith, Chief Executive Officer of Avacta Group, commented:

"We are delighted to receive Orphan Drug Designation from the FDA for AVA6000, which is a reflection of the high quality of the preclinical data and the potential benefit the pre|CISION platform can bring to cancer patients.

"This designation provides tax credits and other incentives for drug developers addressing rare diseases. Most notably the Orphan Drug Designation will give Avacta, if AVA6000 is approved for treatment of soft tissue sarcoma, seven years of market exclusivity in the US, which is a significant commercial advantage."

-Ends-

The information contained within this announcement is deemed by the Company to constitute inside information as stipulated under the Market Abuse Regulations (EU) No. 596/2014 as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018 ('MAR'). Upon the publication of this announcement via Regulatory Information Service ('RIS'), this inside information is now considered to be in the public domain.

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About Avacta Group plc - https://www.avacta.com

Avacta Group is developing novel cancer immunotherapies and powerful diagnostics based on its two proprietary platforms - Affimer^® biologics and pre|CISION™ tumour targeted chemotherapies.

The Affimer^® platform is an alternative to antibodies and is derived from a small human protein. Affimer technology has been designed to address many of the negative issues of antibodies, principally: the time taken to generate new antibodies, the reliance on an animal's immune response; poor specificity in many cases; in addition to, the complexity and high cost of manufacture. Despite these shortcomings, antibodies currently dominate markets, such as diagnostics and therapeutics, which are worth in excess of $100bn.

Avacta's pre|CISION™ targeted chemotherapy platform is designed to selectively release active chemotherapy in FAP rich tumour tissue to limit the systemic exposure that causes damage to healthy tissues, and thereby aims to improve the overall safety and therapeutic potential of these powerful anti-cancer treatments.

The Avacta Group comprises two divisions: The therapeutics development activities are based in London, UK and a separate diagnostics business unit is based in Wetherby, UK. The Group is generating near-term revenues from Affimer^® reagents for diagnostics, bioprocessing and research.

Avacta's Diagnostics Division is developing an in-house pipeline of Affimer-based diagnostic assays, including the AffiDX^® SARS-CoV-2 Lateral Flow Rapid Antigen Test, and works with partners world-wide to develop bespoke Affimer^® reagents for third party products.

Avacta's Therapeutics Division is working to generate more tolerable and durable treatments for oncology patients who do not respond to existing therapies. By combining its two proprietary platforms the Group is building a wholly owned pipeline of clinically differentiated cancer therapies. In 2021 Avacta transitioned to become a clinical stage biopharmaceutical company, when it commenced a phase I trial in patients with locally advanced or metastatic selected solid tumours. The study was a first-in-human, open label, dose-escalation and expansion study of the Group's lead pre|CISION™ prodrug, AVA6000 (a pro-doxorubicin).

Avacta has established drug development partnerships with pharma and biotech, including a multi-target deal with LG Chem worth up to $400m, a joint venture in South Korea with Daewoong Pharmaceutical focused on cell and gene therapies incorporating Affimer immune-modulators and a recent license agreement with Point Biopharma for them to develop radiopharmaceuticals based on the pre|CISION™ platform.

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