• Third Company Launch underpinned by promising Opthalmic Programmes

News & Views

Third Company Launch underpinned by promising Opthalmic Programmes

Jun 21 2023

With £96 million Series A financing, Lifescience company developer Syncona has launched Beacon Therapeutics Holdings, a leading ophthalmic gene therapy company focused on restoration and improvement of vision of patients with retinal diseases. The new portfolio company has been created 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. When all current commitments are invested, Syncona will have a 65.3 per cent stake in the business. The financing round was led alongside additional investors including Oxford Science Enterprises (OSE).

The Series A financing will advance Beacon Therapeutics’ gene therapy candidates through to key value inflection points, including its lead programme AGTC-501, currently in Phase II clinical trials for the treatment of XLRP, an inherited monogenic disorder leading to progressive vision loss in males; 12-month data from its Phase II SKYLINE trial is expected in H2 CY2023.

Two other promising pre-clinical programmes include an intravitreally delivered gene therapy for dry age-related macular degeneration (AMD), the leading cause of blindness in people over 60; and in-licensed from the University of Oxford, a project targeting cone-rod dystrophy (CRD), a group of inherited vision loss disorders affecting the retina. Beacon Therapeutics is Syncona’s 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. “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,” he said.

“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.”

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.

Professor Robert MacLaren, Professor of Ophthalmology, University of Oxford and Co-Founder of the new company commented: “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.”

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