GRAND PHARMA (00512): Global Innovative Ophthalmic Drug CBT-001 for Pterygium Meets Primary Clinical Endpoint in International Multicenter Phase III Clinical Trial

GRAND PHARMA (00512) announced that the international multicenter Phase III clinical trial of CBT-001 (GPN00153), the Group's globally innovative ophthalmic drug for the treatment of pterygium, has recently met its primary clinical endpoints. Compared with the placebo control group in terms of conjunctival hyperemia at Month 3 and pterygium lesion length at Month 12, both doses of CBT-001 demonstrated highly statistically significant improvements. These clinical results mark the first time a drug has demonstrated a disease-modifying treatment effect on pterygium through statistically significant improvement in hyperemia and lesion size. Additional analyses are ongoing. Summary of key data: Conjunctival hyperemia: This directly targets the most prominent ocular surface hyperemia symptom in pterygium patients, aiming to objectively verify the drug's rapid relief effect on local inflammation and redness. The clinical data obtained show that at Month 3, both dose groups of CBT-001 (0.1% and 0.2%, administered twice daily) demonstrated highly statistically significant improvements in conjunctival hyperemia compared with the control group (p<0.001). An even more positive signal is that this clinical improvement could be observed as early as Week 2 after dosing and persisted throughout the entire follow-up period up to Month 12. This result is consistent with the Phase II clinical trial results. Pterygium lesion length: This indicator aims to evaluate the drug's ability to curb the continuous expansion of the lesion toward the cornea. As shown in the preliminary Phase III results announced this time, at Month 12, compared with the control group, both dose groups showed statistically significant reductions in pterygium lesion length (p<0.001). In terms of lesion size, the benefit of the treatment groups compared with the placebo control group was approximately 10% of baseline. Notably, at Month 12, the observed treatment effect had not yet reached a plateau. Safety: Superficial punctate keratitis was the most commonly reported adverse event in the CBT-001 0.1% group (20%, versus 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% concentration group was below 10%. Similar adverse events were also reported in the 0.2% concentration group, but with a relatively slightly higher incidence. Cloudbreak Pharma Inc. is conducting additional analyses of the Phase III study results, including secondary endpoints and subgroup analyses, and plans to meet with the U.S. Food and Drug Administration to review the study results and discuss potential next steps for the CBT-001 development program, including the clinical and regulatory implications of the study results. This study [CS-301 (NCT05456425)] is an international multicenter, randomized, double-blind, placebo-controlled, parallel-group Phase III clinical trial with continuous twice-daily dosing for 24 months, comparing the efficacy of two doses (0.1% and 0.2%) of CBT-001 ophthalmic emulsion versus placebo in patients with pterygium. The primary efficacy endpoints include reduction of conjunctival hyperemia associated with vascularized pterygium lesions at Month 3 and inhibition of pterygium growth at Month 12. The clinical protocol also includes a 12-month extension period to obtain an additional 12 months of safety data. Patient enrollment began in the United States in June 2022 and expanded to China, New Zealand, Australia, and India. The top-line study results summary report covers 659 patients. Pterygium is a common chronic inflammatory proliferative ocular surface disease that usually occurs in the conjunctiva at the canthus and gradually invades the cornea, causing astigmatism or obscuring the pupil, thereby leading to decreased vision and even blindness. According to the World Health Organization's World Report on Vision, the global prevalence of pterygium is 10.2%, and in rural areas of China it is as high as 33%. According to relevant literature, the prevalence among people aged 40 and above in China is 13.4%, and it continues to rise with age. Currently, there is no clinical drug specifically for the treatment of pterygium. When accompanied by obvious inflammation, preservative-free artificial tears may be used topically to lubricate the ocular surface, or low-concentration glucocorticoid eye drops may be used short-term to reduce hyperemia and edema. Existing therapeutic drugs have not shown definitive efficacy and are associated with long-term safety risks. Severe patients can be treated with surgery, but after scleral excision for pterygium, the postoperative recurrence rate is as high as 31-83%. Multiple recurrences can cause serious complications such as symblepharon, conjunctival sac shortening, restricted eye movement, strabismus, and diplopia. It is evident that there remains a huge unmet clinical need for the treatment of pterygium. CBT-001 is a novel multi-kinase inhibitor (MKI) eye drop independently developed by Cloudbreak Pharma Inc. (HKEX: 2592), and is an improved innovative formulation of nintedanib, the core active ingredient of the marketed pulmonary fibrosis treatment OFEV. The drug can block multiple kinase receptor pathways, inhibit neovascularization and tissue fibrosis processes, and intervene at the root of the pathological basis of pterygiumfibrovascular proliferationrather than merely relieving clinical symptoms. The previously completed U.S. Phase II clinical trial of CBT-001 showed that it can significantly and sustainably inhibit vascular proliferation and ocular hyperemia symptoms of pterygium, with good safety. The product's scope of application covers mild, moderate, and severe stages of pterygium, and it 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, the Hong Kong Special Administrative Region, the Macao Special Administrative Region, and the Taiwan region of China. If successfully approved for marketing, CBT-001 will become the world's first drug for the treatment of pterygium, and is expected to completely change the current clinical landscape dominated by surgery and palliative treatment, building the Group's differentiated competitive advantage in the field of ophthalmic anti-inflammatory and proliferative disease treatment. The Group has always regarded the ophthalmic field as one of its important strategic development directions, continuously focusing on ophthalmic drug innovation, adhering to a professional development path, and continuously enhancing its industry position and market competitiveness. At present, through a strategy combining cooperative introduction and independent research and development, the Group has built a reserve of multiple globally innovative products for the treatment of "dry eye disease," "demodectic blepharitis," "postoperative ophthalmic anti-inflammatory and analgesic treatment," "pterygium," and "myopia," building differentiated competitive advantages and expected to provide patients with more treatment options and improve quality of life. At the same time, by continuously strengthening the construction of the clinical evidence-based medicine system and professional academic promotion system for products, the Group provides clinical experts with comprehensive disease diagnosis and treatment plans and detailed product service plans. In the future, this segment will continue to deepen its presence in frontier innovation tracks, continuously strengthen its industry influence, and achieve new breakthroughs in business areas. In the ophthalmic segment, Xinterun (varenicline tartrate nasal spray, OC-01) for the treatment of dry eye disease is currently the world's first and only approved preservative-free, multi-dose, sterile-packaged nasal spray for the treatment of mild, moderate, and severe dry eye. The product was approved for marketing in the United States in October 2021, obtained approval for marketing from the National Medical Products Administration ("NMPA") of the People's Republic of China in November 2024, and achieved the first commercial prescriptions in mainland China after formal approval in July 2025; the globally innovative ophthalmic drug Manmu'an (lotilaner ophthalmic solution 0.25%, TP-03) for the treatment of demodectic blepharitis was approved for marketing by the Pharmaceutical Administration Bureau of the Macao Special Administrative Region Government in May 2025, was approved for marketing by the NMPA in March 2026, and achieved the first commercial prescriptions in mainland China in October of the same year; GPN00833, a hormonal nano-suspension eye drop for anti-inflammatory and analgesic treatment, completed domestic Phase III clinical research and reached clinical endpoints in November 2024; and GPN00884, a globally innovative ophthalmic drug for slowing the progression of myopia in children, completed first-patient enrollment in its Phase IIa clinical study conducted in China in October 2025. In the future, the Group will accelerate the comprehensive and differentiated layout of its R&D pipeline and continuously enrich its reserve of innovative products in ophthalmic sub-sectors. In addition, the Group has attracted and cultivated a group of professionals with both clinical and marketing experience in the ophthalmic field, and has established a customer-centered, academically led professional marketing team, forming a marketing network covering the whole country. With the continuous approval of innovative ophthalmic products, the Group will fully leverage its core advantages in this field, continue to deepen its presence in frontier innovation tracks in ophthalmology, further strengthen the professional promotion and brand building of core products, and provide new momentum for the Group's sustained and healthy development. The Group has always attached great importance to the research and development of innovative products and advanced technologies, taking patient needs as the core and technological innovation as the driver, increasing investment in globally innovative products and advanced technologies in response to unmet clinical needs, enriching and improving the product pipeline and industrial layout, adopting the strategy of "global operational layout, dual-circulation business development," forming a new pattern of domestic and international dual-circulation linkage and mutual promotion, fully leveraging the Group's industrial advantages and R&D strength, and rapidly bringing technological innovation products to market, providing patients worldwide with more advanced and diverse treatment options.