Yuanda Pharmaceutical (00512): The international multi-center phase III clinical trial of the innovative global ophthalmology drug CBT-001 for pterygium reached the main clinical end

Zhitong Finance App News, Yuanda Pharmaceutical (00512) announced that the Group's international multi-center phase III clinical trial of CBT-001 (GPN00153), a global innovative ophthalmology drug for the treatment of pterygium, recently reached a major clinical end. Comparing the length of conjunctival hyperemia at month 3 and pterygium lesion length at month 12 in the placebo control group, the two doses of CBT-001 showed a highly statistically significant improvement. This clinical result marks the first time that a drug has shown a therapeutic effect on pterygium disease by statistically significantly improving congestion and lesion size. Other analyses are ongoing.

Key data summary:

Conjunctival hyperemia: It directly targets the most prominent ocular surface congestion symptoms in patients with pterygium. The aim is to objectively verify the rapid relief effect of the drug on local inflammation and redness. Clinical data obtained this time showed a high level of statistically significant improvement (p<0.001) in both CBT-001 dosage groups (0.1% and 0.2%, twice daily administration) compared with the control group at 3 months. A more positive sign is that this clinical improvement can be observed as early as 2 weeks after administration, and continues throughout the 12-month follow-up cycle. This result is consistent with the results of the phase II clinical trial.

Pterygium lesion length: This indicator is aimed at evaluating the ability of drugs to stop the lesion from continuing to expand into the cornea. According to the preliminary results of phase III published this time, at 12 months, compared with the control group, both dose groups showed a significant statistically significant decrease in pterygium lesion length at 12 months (p<0.001). In terms of lesion size, the benefit of the treatment group compared to the placebo control group was about 10% of the baseline. It is worth noting that at 12 months, the observed therapeutic effects have not yet reached the plateau period.

Safety: Superficial punctate keratitis was the most commonly reported adverse event in the CBT-001 0.1% group (20%, compared to 8% in the placebo control group), and most events were relieved after treatment with artificial tears. The incidence of other individual adverse events in patients in the 0.1% dosage group was less than 10%. Similar adverse events were also reported in the 0.2% dosage concentration group, but the incidence was slightly higher.

Cloudbreak Pharma Inc. is conducting additional analyses of the Phase III study results, including secondary endpoints and group analysis, and plans to meet with the US Drug and Food Administration to review the findings and discuss potential follow-up actions to the CBT-001 plan, including the clinical and regulatory implications of the findings.

This study [CS-301 (NCT05456425)] is an international multicenter, randomized, double-blind, placebo-controlled phase III clinical trial. It was administered twice daily for 24 months to compare the efficacy of two doses (0.1% and 0.2%) of CBT-001 ophthalmic lotion and placebo control groups in patients with pterygium. Key end points include reduction of conjunctival congestion associated with vascularized pterygium lesion at 3 months and suppression of pterygium growth at 12 months. The clinical protocol also includes a 12-month extension period to obtain additional 12 months of safety data. Patient recruitment began in June 2022 in the US and expanded to China, New Zealand, Australia and India. The Topline Findings Summary Report covered 659 patients.

Pterygium is a common chronic inflammatory proliferative ocular surface disease. It usually occurs in the conjunctiva at the corner of the eye and gradually invades the cornea, causing astigmatism or blocking the pupil, leading to decreased vision or even blindness. According to the World Health Organization's “World Vision Report”, the global prevalence of pterygium is 10.2%, up to 33% in rural China. According to relevant literature, the prevalence rate among people aged 40 and above in China is 13.4%, and the trend continues to rise with age. Currently, there is no specific treatment for pterygium in clinical practice. When there is obvious inflammation, artificial tears without preservatives can be used topically to lubricate the eye surface, or low-concentration glucocorticoid eye drops can be used for a short period of time to relieve congestion and edema. Existing treatments have not shown accurate efficacy and there are long-term safety risks. Serious patients can be treated with surgery, but after sclerectomy, the recurrence rate of pterygium is as high as 31-83% after surgery. Multiple recurrences can cause serious complications such as eyelid adhesions, conjunctival sac narrowing, eye movement restrictions, strabismus, and diplopia. As can be seen, there is still a huge unmet clinical need for treatment of pterygium.

CBT-001 is a novel multi-kinase inhibitor (MKI) eye drops independently developed by CloudBreak Pharma Inc. (HKEX: 2592). It is an improved innovative formulation of nidanib, the core active ingredient of OFEV, a marketed pulmonary fibrosis treatment drug. The drug can inhibit various kinase receptor pathways, block the process of neovascularization and tissue fibrosis, and fundamentally interfere with fibrous angiogenesis, the pathological basis of pterygium, rather than simply relieving clinical symptoms. Previously completed US phase II clinical trials of CBT-001 showed that it can significantly and continuously inhibit pterygium angiogenesis and ocular congestion symptoms, and is safe. The scope of application of this product covers light, medium and severe stages of pterygium, and can also be used to prevent recurrence after pterygium surgery. The Group has obtained exclusive production (including technology transfer) and commercialization rights for CBT-001 in mainland China, Hong Kong Special Administrative Region of China, Macau Special Administrative Region of China and Taiwan. If successfully approved for marketing, CBT-001 will become the world's first drug for the treatment of pterygium. It is expected to completely change the current clinical pattern of surgery and palliative care, and build the Group's differentiated competitive advantage in the field of anti-inflammatory and proliferative diseases in ophthalmology.

The Group has always taken the field of ophthalmology as one of the important strategic development directions, continuously focusing on ophthalmic drug innovation, adhering to the path of specialized development, and continuously improving its position in the industry and market competitiveness. Currently, through a strategy combining cooperative introduction and independent research and development, the Group has stocked up a variety of innovative global products for the treatment of “dry eye,” “demodex blepharitis,” “post-operative anti-inflammatory pain relief,” “pterygium,” and “myopia,” which is expected to provide patients with more treatment options and improve quality of life. At the same time, through continuous strengthening of the product's clinical evidence-based medical system construction and professional academic promotion system construction, the group provides clinical experts with complete disease diagnosis and treatment plans and detailed product service plans. In the future, the sector will continue to cultivate cutting-edge innovation tracks, continuously strengthen the industry's influence, and achieve new breakthroughs in the business field.

In the ophthalmology sector, Xingterun® (vanillin tartrate nasal spray, OC-01) for treating dry eye is currently the world's first and only preservative-free, multi-dose, aseptically packaged nasal spray approved to treat mild, moderate, and severe dry eyes. The product was approved for marketing in the US in October 2021, and was approved by the China Drug Administration (“FDA”) of the People's Republic of China in November 2024. The first batch of commercial prescriptions for treating blepharitis was launched in mainland China in July 2025. Innovative ophthalmic drugs are full Mu'an® (lotirana eye drops 0.25%, TP-03) was approved for marketing by the Drug Administration of the Macau Special Administrative Region Government in May 2025, approved by the China Drug Administration in March 2026, and implemented the first batch of commercial prescriptions in mainland China in October of the same year; GPN00833, a hormone nano-suspension eye drop for anti-inflammatory pain, completed domestic phase III clinical research in November 2024 and reached the clinical end point; GPN00884, a global innovative ophthalmology drug used to slow the progression of myopia in children in October 2025 The first patient was enrolled in a phase IIa clinical study conducted in China.

In the future, the Group will accelerate the comprehensive and differentiated layout of R&D pipelines and continuously enrich innovative product reserves in the field of ophthalmology. In addition, the Group has attracted and trained a group of professionals with both clinical experience and marketing experience in the field of ophthalmology, established a customer-centered and academic-led professional marketing team, and formed a nationwide marketing network. As innovative ophthalmology products continue to be approved, the Group will give full play to its core advantages in this field, continue to deepen the cutting-edge ophthalmology innovation circuit, further strengthen the specialized promotion and brand building of core products, and provide new momentum for the Group's sustainable and healthy development.

The Group has always attached great importance to research and development of innovative products and advanced technology, with patients' needs as the core, scientific and technological innovation as the core, increasing investment in innovative global products and advanced technology, enriching and improving product pipelines and industrial layout in response to unmet clinical needs, adopting a “global operation layout, dual cycle business development” strategy to form a new pattern of domestic and international dual cycle linkage development and mutual promotion, give full play to the Group's industrial advantages and R&D strength, and quickly launch innovative scientific and technological products on the market to provide patients around the world with more advanced and diverse treatment plans.