VCs ‘Complement’ European biotech with $79M for retinal AAV gene therapy trial next year 

The disease, which leads to blindness, gots its first approved treatment this year via Apellis’ Syfovre. Iveric Bio is close behind.

 By Kyle LaHucik, Associate Editor

Like those drug developers, the UK and German startup is going after the complement cascade, thought to contribute to multiple diseases. Instead of frequent injections, Complement wants a one-and-done gene therapy to thwart the onset of blindness, and the biotech will test that approach starting in September or October 2024, CEO and managing director Rafiq Hasan told Endpoints News.

“Up until now, there was always some question marks. Is complement the right approach for the treatment of GA?” Hasan said.

The funding will take Complement through the end of 2026. By the middle of that year, the biotech expects to be “sitting on some nice data,” Hasan said. Complement hopes to gather data that could maybe spur partnership talks, he added.

While others are pulling back on viral-based gene therapies like AAV, Hasan said Complement sees strong potential for potency in its lead asset CTx001 and there’s a precedent in Luxturna, Novartis’ AAV for the eye. He spent more than a decade at the Swiss Big Pharma in the 2000s, mostly in the ophthalmology space. The biotech will disclose preclinical data in the coming week, the CEO added.

“Formed in 2021 out of the University of Manchester, the biotech is focused on complement receptor 1, which Hasan described as “one of the most potent co-factors in actually modulating the activity of C3, and C3 is really this pro-inflammatory part of the cascade. That’s the central part that really drives a lot of the pathology in geographic atrophy.” Apellis’ Syfovre targets C3 and Iveric’s avacincaptad pegol, up for an Aug. 19 FDA decision, is a C5 inhibitor.”

Complement has created a smaller version of CR1 that it can package into AAV and deliver via sub-retinal injection so it will “convert the eye into a factory for this truncated CR1 that then modulates complement activity in the eye.”

The five-employee biotech is expecting to hire a chief technology officer and medical chief in the coming quarter or two, Hasan said, after bringing on chief scientific officer Dennis Keefe last fall.

A natural history study is underway for genetic profiling, imaging and understanding of the systemic complement protein levels of an expected 250 people, most of whom the biotech hopes will then enroll in the Phase Ib, the CEO said. With about a dozen sites already in the UK, a move into the US is on the horizon.

“The rationale is that we really enrich the population,” Hasan said, noting GA is a heterogenous disease, with complement not contributing to about 20% to 30% of cases.

Beyond the eye
Complement also sees potential in treating diseases above and below the eyes, including a rare kidney disease known as IgA nephropathy, Hasan said.

Travere Therapeutics notched an accelerated approval for its drug Filspari in that indication in February.

Hasan said emerging evidence might also send Complement into the brain, where it could expand the pipeline to include Alzheimer’s disease and line up partnerships before entering the clinic.

A lab is coming together in the next couple of weeks in Stevenage, UK, a subsidiary has been created in the US and the biotech is working with CDMO Pharmaron, Hasan said.

Belgian PE and VC fund Gimv led the Series A. Other backers include Forbion, BioGeneration Ventures, Panakès Partners, Cambridge Innovation Capital (CIC), Hadean Ventures and Seroba Life Sciences.