Syncona launches retinal disease-focussed Beacon Therapeutics

The firm said the newly-formed company was aimed at restoring and enhance the vision of patients with retinal diseases using ophthalmic gene therapy.

Syncona said it had secured £96m in series A financing to support Beacon’s endeavours.

The formation of Beacon Therapeutics involved the consolidation of Applied Genetic Technologies Corporation's (AGTC's) late-stage programme in X-linked retinitis pigmentosa (XLRP) with two other innovative pre-clinical programs.

Syncona holds a value of £60m in Beacon Therapeutics, having committed a total of £75m including the capital invested in the acquisition and restructuring of AGTC.

As Syncona continued to support the company, its stake in Beacon would reach 65.3%.

The financing round was led by Syncona, alongside investors such as Oxford Science Enterprises (OSE).

The funds raised from the series A financing would be used to advance Beacon Therapeutics' gene therapy candidates to critical value inflection points.

The lead programme of Beacon Therapeutics is AGTC-501, currently undergoing phase two clinical trials for the treatment of XLRP.

XLRP is an inherited monogenic disorder that results in progressive vision loss in males, and Syncona said AGTC-501 had shown promising clinical evidence, demonstrating significant efficacy and a favourable safety profile in its recent phase one and two ‘HORIZON’ trial.

The company said it expected to release 12-month data from the phase two ‘SKYLINE’ trial in the second half of this year.

In addition to the XLRP programme, Beacon possessed two pre-clinical programmes with significant potential.

Syncona said it identified the first asset as a gene therapy for dry age-related macular degeneration (AMD) - the leading cause of blindness in individuals over 60.

The second programme, licensed from the University of Oxford, targeted cone-rod dystrophy (CRD) - a group of inherited vision loss disorders affecting the retina.

Syncona said it had combined those assets with AGTC's later-stage asset in XLRP, establishing its third ophthalmic gene therapy company.

The previous ventures, Nightstar and Gyroscope, were successfully sold for $877m and up to $1.5bn, respectively, yielding substantial returns on investment.

Beacon Therapeutics would be led by chief executive officer David Fellows, who the firm said had more than 40 years of experience in ophthalmology, including five years as CEO of Nightstar.

Dr Nadia Waheed, a retinal expert and former chief medical officer of Gyroscope, would serve in the same role at Beacon, while Dr Abraham Scaria, an expert in gene therapy, had joined as chief scientific officer.

Chris Hollowood, CEO of Syncona Investment Management, was appointed chair of Beacon Therapeutics, and Elisa Petris, Syncona’s lead partner, would join the board as a director.

Professor Robert MacLaren, professor of ophthalmology at the University of Oxford, would actively advise on Beacon Therapeutics' CRD programme, and also assume a non-executive director role.

“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,” said Chris Hollowood, chief executive officer of Syncona Investment Management.

“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.”

At 0804 BST, shares in Syncona were up 0.26% at 154.8p.

Reporting by Josh White for Sharecast.com.