Complement Tx set to initiate clinical trials with novel gene therapy in fighting macular degeneration
The company, which has facilities at Stevenage Bioscience Catalyst, reports good progress in terms of R & D with its lead product CTx001 – an AAV gene therapy in late stage preclinical development for the treatment of geographic atrophy, a type of age related macular degeneration and a leading cause of blindness globally.
CEO Dr Rafiq Hasan reveals that Complement Therapeutics is in the final stages of completing preclinical work and plans to submit its data to the regulatory agencies in the US and UK with the intention to initiate its first clinical trial with CTx001 in the coming months.
“This study is designed in such a way that we will have the opportunity to review the data from the initial cohorts of treated patients throughout 2026 and then more formal evaluations of the data thereafter,” says Dr Hasan.
“We are very excited by the potential of CTx001 to help patients with this devastating condition by slowing down the progression of disease and preserving vision.”
The rate of progress for the business has been encouragingly quick since it spun out of the University of Manchester in 2021 with initial funding from BGV. The investor swiftly followed through when it backed a €5 million seed round the following February alongside Dutch powerhouse Forbion.
With that funding, CTx advanced CTx001 through preclinical proof-of-concept, secured an Innovation Passport from the Medicines and Healthcare products Regulatory Agency (MHRA) and initiated a non-interventional natural history study in the UK (i-GAIN). The i-GAIN study provides a strong foundation to support the clinical development planning for CTx001.
April 2023 proved to be a milestone month for the young business when it secured €72 million in Series A financing to advance novel therapies targeting complement-mediated diseases. That sizeable and significant round was also led from Europe – by Gimv, a Belgian based private equity and venture capital firm.
2023 was to prove a game-changing year for Complement Tx. In a heady six months the company continued to make strong progress; in June it was able to move into SBC and unveil its important new laboratory facilities. Then in the November its blockbusting Series A won the Finance Raise of the Year award at LifeStars Award.
Solid foundations are the bedrock of any successful venture and Complement Tx is no exception. It owes its name to our complement system, also known as the complement cascade. This is a part of the immune system that enhances (complements) the ability of antibodies and phagocytic cells to clear microbes and damaged cells from an organism, promote inflammation and attack the pathogen’s cell membrane.
Core to the innate immune system, it consists of some 50 proteins and protein fragments that are predominantly synthesised by the liver and circulate in the blood. When activated by one of several triggers, the complement system works in conjunction with other components of the immune system to clear foreign and damaged material such as invading pathogens like bacteria and viruses.
Dysregulation of the complement system is implicated in the development of a number of diseases including age-related macular degeneration, kidney disease and various haematological conditions. As understanding of the complement cascade progresses, it is apparent that complement plays a pivotal role in disease, with new insights creating the opportunities for identifying and developing new therapeutic strategies.
That's where this thrusting young business comes into the front line of action to arrest progress of the condition.
Based on pioneering research of the founders (Prof Simon Clark, Prof Paul Bishop and Prof Richard Unwin), Complement Therapeutics aims to develop innovative and effective therapeutics to address unmet needs in complement mediated diseases. What a start it has made and, ditto – what super progress towards the acid test of its vital research!
That acid test will come in the form of a first in human clinical trial called Opti-GAIN, which is anticipated to start towards the end of 2025. The study plans to leverage latest technological advances in the analysis of retinal structure and function to assess the safety and potential efficacy of CTx001 in GA.
Leading eye care doctors and centres in the UK and US are collaborating with Complement Tx on this trial, leading to expectations of a high-quality clinical trial that has the potential to bring life-changing treatment closer to patients with geographic atrophy.
