Ex-Nightstar, Gyroscope and AGTC teams try again for next retinal gene therapy with $120M for Beacon Therapeutics

Life sciences investor Syncona is taking a second shot at X-linked retinitis pigmentosa, this time with Beacon Therapeutics, a retinal gene therapy startup led by former executives of Syncona-backed companies Nightstar Therapeutics and Gyroscope Therapeutics.

The biotech officially unveiled Sunday night with £96 million, or $120 million, to bankroll late-stage work on a gene therapy that it bought last fall for X-linked retinitis pigmentosa (XLRP), a rare, genetic disease that leads to blindness, predominantly in young men. Some members of the Beacon team had previously worked on a gene therapy for XLRP as Nightstar, which was acquired by Biogen in 2019. But that therapy never moved past a Phase II/III trial.

This time around, the Beacon team thinks it has a better chance. It’s running a program from AGTC, a startup founded in 1999 that struggled for funding during last year’s biotech market squeeze after previously battling safety issues and an axed Biogen pact. Beacon CEO Dave Fellows, former chief executive of Nightstar, told Endpoints News that Beacon has the data, fine-tuned surgical technique, refined patient selection, dose selection understanding and better insight into the endpoints for a second attempt at developing a therapy for XLRP.

Nadia Waheed

Beacon said it expects to present 12-month data on the Phase II program later this year, according to medical chief Nadia Waheed, who joined from Gyroscope, which is now owned by Novartis. The gene therapy aims to correctly express the full-length RPGR protein, where mutations occur in the rare disease, as that’s key to addressing both rods and cones in the disease. This way, both night vision and daily activities like reading, writing and watching TV aren’t taken away, she said.

“It looks very, very encouraging and consistent with what had been presented at the three-month time point,” Waheed said, noting abstracts are being submitted for the one-year data.

Beacon has received initial feedback from European regulators and is in talks with the FDA about a global, registrational clinical trial, she added.

The AGTC program is augmented by a pipeline of preclinical gene therapies, one of which comes from Nightstar founder Robert MacLaren’s lab at the University of Oxford and IP from another undisclosed source. The Oxford asset is for cone-rod dystrophy caused by a mutation in the Cadherin Related Family Member 1, or CDHR1, gene. The other program is an intravitreally delivered AAV-based gene therapy for dry age-related macular degeneration. Waheed had worked on a gene therapy for a form of geographic atrophy at Gyroscope.

“One of the things that’s been really frustrating from a clinician’s perspective is that we have, for retina, multiple validated mechanisms of action that seem to work, and there have been more and more that have been emerging. But, unfortunately, the delivery hasn’t caught up with the mechanisms of action,” said Waheed, a practicing retina specialist for more than 20 years.

Beacon is one of several emerging biotechs working on ophthalmological gene therapies, with recent entrants including Ray Therapeutics, Complement Therapeutics, Replay’s Eudora, and Avista Therapeutics. The proliferation of upstarts makes Syncona CEO Chris Hollowood feel that “we’re in the foothills of something very, very big.”

Chris Hollowood

Beacon came together after Syncona considered forming a third biotech in the retinal gene therapy field, according to Hollowood. In an interview, he noted they had been pulling together a pipeline when the markets took a turn for the worse. That’s when the AGTC opportunity came about. He had known the company for about five years and had “always been impressed” with their XLRP data.

“Dave got a phone call from me that he thought was just a catch-up, but it was actually to beg him to come out of retirement and take over as CEO, and Nadia and I were in regular contact,” Hollowood said.

He noted there was a “very high level of frustration” regarding the direction of the Nightstar program after the Biogen acquisition.

Beacon has 80 employees in Alachua, FL; Cambridge, MA; and London. AGTC had already built a manufacturing process, and Beacon is in the process of making a decision whether to use it for a Phase III study or outsource to a CDMO, Fellows said.

The financing includes money from Oxford Science Enterprises and should take the company “into next year,” Fellows said. Also on the leadership team are chief technology officer Eduardo Jacobo and science chief Abraham Scaria, both of whom were part of the AGTC team. Scaria previously was CSO at Iveric Bio, a biotech being acquired by Astellas and on the way to becoming the second company to secure a geographic atrophy drug approval this year.

“We certainly are committed to going forward and hopefully commercializing ourselves in specific markets, but we’re not closing any doors,” Fellows said about the XLRP asset.

The Nightstar and Gyroscope exits had been “opportunistic,” he said.

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