Innovent Biologics completes first patient dosing in its phase 2 clinical trial assessing efdamrofusp alfa in patients with DME

The primary end point of the trial is the change in best correct visual acuity (BCVA) from baseline in the study eye at week 16. Image credit: AdobeStock/xixinxing

Biopharmaceutical company Innovent Biologics has completed the first patient dosing in its phase 2 clinical trial assessing efdamrofusp alfa for the treatment of diabetic macular edema (DME).¹ The recombinant human vascular endothelial growth factor receptor (VEGFR)-antibody human complement receptor 1 (CR1) fusion protein was intravitreally administered in DME patients in the randomized, double-masked, multi-center, active-controlled trial (NCT06908876), which assessed the therapy’s efficacy and safety, according to a news release.

The primary end point of the trial is the change in best correct visual acuity (BCVA) from baseline in the study eye at week 16.¹

"DME is one of the most common ocular fundus diseases in China, affecting up to 4-5 million patients,” Xiaodong Sun, MD, PhD, the leading principal investigator of the study, deputy director of Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, and director of the Ophthalmology Center, stated in the release. “With increasingly the aging of the population and the continuous rise in diabetes prevalence in China, DME has become one of the diseases that seriously endangers public health, imposing heavy burdens to the society and individuals. Anti-VEGF drugs are currently the first-line treatment for DME, but huge unmet medical needs remain, such as frequent injections, long-term efficacy attenuation, and suboptimal response in a subset of patients. As a global first-in-class anti-VEGF-complement bispecific drug, efdamrofusp alfa has shown favorable safety profile, substantial improvement in visual acuity and macular edema, and the potential of extended dosing interval in the completed Phase 1 clinical study in DME patients, which has brought great confidence to our investigator team. We look forward to the success of this phase 2 study, hoping to provide of a new treatment option for a large number of DME patients."

A total of 150 patients are planned to be enrolled in the study and randomized 1:1:1 to the IBI302 4 mg group, IBI302 8 mg, and a farcimad (anti-VEGF/ANG-2 bispecific antibody) 6 mg group.¹

Edfamrofusp alfa has previously been assessed in multiple clinical studies in patients with neovascular age-related macular degeneration and DME. These studies demonstrated significant efficacy in improving visual acuity and retinal edema in high-dose (8 mg) efdamrofusp alfa. The therapy has also shown the potential for extended dosing intervals up to 12-16 weeks with favorable safety and tolerability.¹

Edfamrofusp alfa works through dual mechanism in the N-terminal as the VEGF-binding domain, and the C-terminal as the complement binding domain that binds C3b and C4b. This allows for inflammatory responses to be alleviated by complement activation. Thus, the dual mechanism is demonstrated in its therapeutic effects of inhibiting VEGF-mediated angiogenesis and complement activation pathways.¹

Complement system activation is known to contribute to disease progression by damaging neurovascular units via cytolysis, opsonization, and promotion of proinflammatory microenvironment. Retinal microvascular lesions, neurodegeneration, macular edema, and retinal neovascularization are the result.¹

"Efdamrofusp alfa is an anti-VEGF-complement bispecific fusion protein self-developed by Innovent with global proprietary rights,” said Lei Qian, PhD, senior vice president of Clinical Development of Innovent, in the release. “This clinical study will be globally the first study comparing two dual-target agents, using faricimab, the global standard of care in DME. We look forward to the success of this study, which could provide support for a phase 3 clinical trial and a new treatment option for patients. Innovent's general biomedicine pipeline covers cardiovascular, endocrine and metabolic, ophthalmology, and autoimmune disease areas. We will continue to develop new therapies, providing physicians with more clinical treatment options and benefiting more patients."

Reference:

1. Bispecific vs bispecific: Innovent announces first patient dosed in the phase 2 clinical study of efdamrofusp alfa (IBI302), a first-in-class anti-VEGF and anti-complement bispecific fusion protein for the treatment of diabetic macular edema. News release. PR Newswire. May 6, 2025. Accessed May 7, 2025. https://www.prnewswire.com/news-releases/bispecific-vs-bispecific-innovent-announces-first-patient-dosed-in-the-phase-2-clinical-study-of-efdamrofusp-alfa-ibi302-a-first-in-class-anti-vegf-and-anti-complement-bispecific-fusion-protein-for-the-treatment-of-diabetic-m-302445995.html