Syncona Limited

Syncona launches new gene therapy company Beacon Therapeutics with £96 million Series A financing

12 June 2023

·      Beacon Therapeutics launches as a leading ophthalmic gene therapy company, created by combining Applied Genetic Technologies Corporation's (AGTC's) late-stage X-linked retinitis pigmentosa (XLRP) programme with two proprietary pre-clinical programmes

·      Syncona is leveraging its deep domain expertise and track record to create its third gene therapy company addressing retinal diseases, following Gyroscope and Nightstar

·      Syncona has committed £75 million to Beacon and will have a 65.3 per cent stake[1] in the business, with a current holding value of £60 million[2]

Syncona Ltd, a leading healthcare company focused on creating, building and scaling a portfolio of global leaders in life science, today announces that it has launched Beacon Therapeutics Holdings Limited (Beacon Therapeutics), a leading ophthalmic gene therapy company with a purpose to restore and improve the vision of patients with retinal diseases, with a £96 million Series A financing. The new portfolio company has been created by Syncona through the combination of Applied Genetic Technologies Corporation's (AGTC's) late-stage programme in X-linked retinitis pigmentosa (XLRP), with two other innovative pre-clinical programmes.

Syncona's holding value in Beacon Therapeutics is £60 million, having committed £75 million to the company, including the capital already invested to acquire and restructure AGTC. Syncona has been supporting the company to re-define its clinical plan, optimise its manufacturing plan for commercial roll-out, restructure costs in the business, broaden the pipeline and expand the team. At the point all current commitments are invested, Syncona will have a 65.3 per cent stake in the business. Syncona led the financing round alongside additional investors including Oxford Science Enterprises (OSE).

The Series A financing will be used to advance Beacon Therapeutics' gene therapy candidates through to key value inflection points, including Beacon Therapeutics' lead programme AGTC-501, which is currently in Phase II clinical trials for the treatment of XLRP, an inherited monogenic disorder that leads to progressive vision loss in males. AGTC-501 has a strong body of clinical evidence having demonstrated meaningful efficacy and a good safety profile in its recent Phase I/II HORIZON trial. 12-month data from its Phase II SKYLINE trial is expected in H2 CY2023.

Beacon Therapeutics also has two promising pre-clinical programmes that Syncona identified. The first asset is an intravitreally delivered gene therapy for dry age-related macular degeneration (AMD), the leading cause of blindness in people over 60, and the second has been in-licensed from the University of Oxford and is targeting cone-rod dystrophy (CRD), a group of inherited vision loss disorders affecting the retina. Syncona has combined these assets with AGTC's later-stage asset in XLRP to create its third ophthalmic gene therapy company, following Nightstar, which sold for $877 million, at a 4.5 multiple of cost, and Gyroscope, which was sold for up to $1.5 billion, at a potential 5.1x multiple of cost.

David Fellows will lead Beacon Therapeutics as Chief Executive Officer (CEO), bringing 40 years' experience in ophthalmology to the management team, including five years as Nightstar's CEO. He will be joined by Chief Medical Officer (CMO) Dr Nadia Waheed, a retinal expert and former Chief Medical Officer of Gyroscope, and gene therapy expert Dr Abraham Scaria, who has joined as Chief Scientific Officer. Syncona Investment Management Limited CEO, Chris Hollowood, has been appointed as Chair and Syncona Lead Partner Elisa Petris will be joining the Board as a Director. Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford, will be an active advisor on Beacon Therapeutics' CRD program and has also become a non-executive director.

Chris Hollowood, Chief Executive Officer of Syncona Investment Management Limited, said: "By acquiring AGTC and combining its late-stage programme with highly complementary and innovative science from the University of Oxford and elsewhere, we have created an exciting new gene therapy company in an area where we have a great breadth and depth of knowledge and expertise. The creation of this unique company demonstrates Syncona's creativity and the proactive and proprietary nature of our approach to sourcing new opportunities.

We have the opportunity to leverage our expertise, track record and network to support Beacon Therapeutics as it develops gene therapies for people suffering from debilitating inherited retinal diseases. We look forward to working closely with Beacon Therapeutics' exceptional management team to advance these assets through the clinic and towards patients."

David Fellows, Chief Executive Officer of Beacon Therapeutics, commented: "Beacon Therapeutics combines a broad development pipeline, a deep scientific foundation, a strong clinical network, and a highly experienced management team to drive forward a unique late-stage clinical and pre-clinical pipeline. With the 12-month data from our Phase II SKYLINE trial for AGTC-501 expected shortly and two highly innovative and differentiated pipeline assets for prevalent and rare blinding diseases, we are excited to be building a new leader in the ophthalmic gene therapy space."

Professor Robert MacLaren, Professor of Ophthalmology, University of Oxford, and Co-Founder of Beacon Therapeutics, commented: "I have dedicated many years to translating pioneering gene therapies from the lab through to clinical trial phase with the support of my team in the Nuffield Department of Clinical Neurosciences and NIHR Oxford Biomedical Research Centre (BRC). Beacon Therapeutics is quite unique in being a company that at launch is already underpinned by excellent clinical trial data. The company has both highly innovative retinal gene therapy programmes and the means to target them to the main causes of blindness in both the young and old."

[ENDS]

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

Forward-looking statements - this announcement contains certain forward-looking statements with respect to the portfolio of investments of Syncona Limited. These statements and forecasts involve risk and uncertainty because they relate to events and depend upon circumstances that may or may not occur in the future. There are a number of factors that could cause actual results or developments to differ materially from those expressed or implied by these forward-looking statements. In particular, many companies in the Syncona Limited portfolio are conducting scientific research and clinical trials where the outcome is inherently uncertain and there is significant risk of negative results or adverse events arising. In addition, many companies in the Syncona Limited portfolio have yet to commercialise a product and their ability to do so may be affected by operational, commercial and other risks.

Enquiries

Syncona Ltd

Annabel Clark / Fergus Witt

Tel: +44 (0) 20 3981 7940

FTI Consulting

Ben Atwell / Natalie Garland-Collins / Tim Stamper

Tel: +44 (0) 20 3727 1000

About Syncona

Syncona's purpose is to invest to extend and enhance human life. We do this by creating and building companies to deliver transformational treatments to patients in areas of high unmet need.

Our strategy is to create, build and scale companies around exceptional science to create a diversified portfolio of 20-25 globally leading healthcare businesses for the benefit of all our stakeholders. We focus on developing treatments for patients by working in close partnership with world-class academic founders and management teams. Our balance sheet underpins our strategy enabling us to take a long-term view as we look to improve the lives of patients with no or poor treatment options, build sustainable life science companies and deliver strong risk-adjusted returns to shareholders.

About Beacon Therapeutics

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease.

Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon Therapeutics also has access to a target generation technology platform that will identify, screen and search secreted proteins in the ophthalmology space.

The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises.

Find out more about us online at: www.beacontx.com.