Innovent Biologics advances Phase 2 study of efdamrofusp alfa in diabetic macular edema amid global push for longer-acting therapies

Innovent Biologics, Inc. (HKEX: 01801), a leading Chinese biopharmaceutical company, announced the first patient has been dosed in its pivotal Phase 2 clinical study of efdamrofusp alfa (IBI302), a bispecific fusion protein that simultaneously targets VEGF and complement pathways. The study marks a critical advancement in the treatment landscape for diabetic macular edema (DME), a condition that has become the leading cause of vision loss in diabetic populations, especially in China.

This double-masked, randomized, multi-centre trial directly compares two dual-target drugs—efdamrofusp alfa and Faricimab, currently the most advanced global standard for DME treatment. The trial’s primary endpoint is the change in best corrected visual acuity (BCVA) at 16 weeks, with 150 patients expected to be enrolled across three arms.

Why Is Efdamrofusp Alfa Considered a First-in-Class Drug?

Efdamrofusp alfa is the world’s first recombinant fusion protein that combines a VEGF-binding domain and a complement receptor domain in a single molecule. The dual-action mechanism is designed to inhibit vascular leakage and angiogenesis through VEGF suppression, while also mitigating inflammation and neurovascular damage by blocking the complement cascade. Innovent holds global intellectual property rights for the molecule.

In early-phase trials in neovascular age-related macular degeneration (nAMD) and DME, the high-dose 8 mg form of efdamrofusp alfa demonstrated significant improvements in visual acuity and retinal thickness while offering the possibility of dosing intervals as long as 12 to 16 weeks—addressing a major unmet need in ophthalmology for less frequent intravitreal injections.

What Is the Clinical Trial Design and Its Significance?

The ongoing Phase 2 study (NCT06908876) is structured to evaluate the safety and efficacy of two dose levels of IBI302 (4 mg and 8 mg) against 6 mg of Faricimab, the bispecific anti-VEGF/Ang-2 antibody developed by Roche. By choosing Faricimab as the active comparator, Innovent is positioning its candidate in direct competition with a globally established drug that currently leads the market for extended-dose DME treatment.

According to trial investigators, the primary outcome will assess functional improvement in BCVA from baseline in the study eye, while secondary outcomes will include anatomical improvements and dosing durability. The design underscores Innovent’s strategy to directly challenge global benchmarks using a differentiated mechanism.

How Big Is the DME Challenge in China and Globally?

DME is one of the most serious complications of diabetic retinopathy, leading to significant visual impairment and economic burden. In China alone, over 140 million people are estimated to be living with diabetes, and between 7% to 14% of those with diabetic retinopathy progress to DME—equating to a patient population exceeding 4 million. As diabetes prevalence rises and the population ages, the incidence of DME is projected to climb sharply.

Pathophysiologically, diabetic macular edema results from a breakdown of the blood-retinal barrier (BRB) driven by chronic hyperglycemia, oxidative stress, VEGF overexpression, and complement-mediated inflammation. Current treatments, such as anti-VEGF monotherapies, require frequent injections and show limited long-term durability in a subset of patients—highlighting the pressing need for improved therapeutic options.

What Do Leading Clinicians and Executives Say?

Professor Xiaodong Sun, the study’s Principal Investigator from Shanghai General Hospital, described diabetic macular edema as one of China’s most burdensome public health concerns. He emphasised the unmet clinical need for therapies that extend dosing intervals and improve response rates among refractory patients. He noted that early trial data for efdamrofusp alfa have shown promising improvements in both safety and efficacy, instilling confidence in the investigator community.

Dr. Lei Qian, Senior Vice President of Clinical Development at Innovent, highlighted the global relevance of the trial. He stated that this marks the first head-to-head comparison between two bispecific agents in DME, and that successful results could lay the groundwork for a Phase 3 trial and eventual commercialisation. He further reiterated Innovent’s broader strategic intent to build a robust pipeline across ophthalmology, autoimmune, cardiovascular, and metabolic diseases.

Why Is Innovent Targeting Ophthalmology Now?

Innovent Biologics has historically been best known for its oncology portfolio, but it is now rapidly expanding into chronic disease areas, including ophthalmology, where multi-target biologics are becoming the preferred therapeutic format. Faricimab’s success demonstrated that combination targeting of VEGF and Ang-2 can enhance durability and outcomes, paving the way for next-generation therapies such as efdamrofusp alfa that go further by integrating anti-inflammatory targets like complement.

With diabetic macular edema becoming a global public health concern, Innovent’s decision to develop a bispecific agent tailored for durability and multi-pathway inhibition aligns with global treatment trends and payer demands for value-based care.

How Is the Market Responding to Innovent’s Progress?

Innovent Biologics (HKEX: 01801) has maintained relatively stable stock performance in recent weeks, even as the broader Hong Kong biotech index experienced volatility due to macroeconomic headwinds and investor caution in the sector. The announcement of the Phase 2 trial has not yet led to immediate price action, but analysts interpret this as a “quiet confidence” scenario rather than market apathy.

Several institutional investors—including Hillhouse Capital and Value Partners—have recently reaffirmed or marginally increased their holdings, according to HKEX filings. Foreign institutional investor (FII) participation has remained steady, with no major divestment activity reported. Domestic institutional flows have also been neutral to slightly positive, suggesting that the trial has reinforced long-term investor conviction rather than triggered speculative trading.

Research coverage from regional brokers such as CICC and CITIC Securities continues to rate the stock a “Buy,” with price targets reflecting a 15–20% upside, contingent on successful clinical milestones. JPMorgan and Morgan Stanley analysts have pointed to the company’s expanding pipeline and its potential for strategic partnerships, particularly for out-licensing assets like efdamrofusp alfa in markets outside China.

Technically, the stock remains above its 200-day moving average, a bullish indicator amid sector weakness. Trading volumes suggest accumulation rather than distribution, and options activity has not shown any surge in implied volatility—supporting a consensus view that the market is awaiting further data before rerating the stock.

The risk-reward profile for investors remains favorable, particularly for long-term positions seeking exposure to China’s innovation-driven biopharmaceutical sector. Analysts suggest that the trial could act as a future catalyst, particularly if the 8 mg efdamrofusp alfa arm delivers superior or comparable results to Faricimab with fewer injections.

Forward Outlook for Efdamrofusp Alfa and Innovent

The current Phase 2 study represents a defining moment for Innovent’s ophthalmology ambitions. If efficacy and safety data validate the drug’s dual mechanism and durability claims, the company is expected to initiate a global Phase 3 trial in 2026. Innovent has also hinted at further expansion of IBI302 into additional indications like neovascular AMD and central retinal vein occlusion (CRVO), broadening the molecule’s commercial prospects.

Meanwhile, the company continues to diversify its biologics platform across therapeutic areas, positioning itself as a multi-franchise innovator. As China’s healthcare system evolves to prioritise innovation, affordability, and long-term disease management, Innovent is well-positioned to become a regional leader with global reach.