HAIXI PHARMA (02637) announced that its innovative drug candidate HXP056, a potential first-in-class oral medication for hemorrhagic retinal diseases, has completed patient enrollment for the Phase I clinical trial segments in China. The trial, which began recruiting patients with wet age-related macular degeneration (wAMD) in early July 2025, has finalized enrollment for both the Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) phases. Following four weeks of continuous dosing, the study has completed Dose-Limiting Toxicity (DLT) assessments and collected Pharmacokinetic (PK) data. A preliminary efficacy evaluation is also currently underway. In the fourth quarter of 2025, the company initiated a Phase II dose-expansion clinical study.

The human immune system is a complex structure involving the coordinated action of multiple pathways and mechanisms. The company's small-molecule innovation platform, MultiSel-Opt, is designed to modulate inflammatory and immune responses by selectively targeting multiple protein targets. It leverages the properties of small-molecule compounds to achieve synergistic multi-mechanistic effects with a single drug molecule. The MultiSel-Opt platform enables the exploration of novel mechanisms for disease treatment, fostering significant innovation in drug development and resulting in candidate compounds with unique profiles compared to other industry offerings. Another key feature of the platform is its focus on optimizing drug distribution to disease sites, such as designing optimal compounds for effective penetration of the blood-retinal barrier (BRB) or blood-brain barrier (BBB).

HXP056 was developed using the MultiSel-Opt platform with a single-drug, multi-selective mechanism design. It aims to provide an oral treatment for hemorrhagic retinal diseases, including wAMD, diabetic macular edema (DME), and retinal vein occlusion (RVO). HXP056 is expected to overcome several technical challenges, achieving both BRB penetration to reach retinal lesions and optimized systemic exposure to ensure patient safety. It has the potential to become the world's first oral therapy for these retinal conditions, representing a major technological breakthrough with significant global market potential.

Preliminary data from the Phase I wAMD study for HXP056 indicate a favorable safety and tolerability profile, along with a clear dose-exposure relationship. Furthermore, preliminary improvements in fundus morphology and retinal function were observed in both treatment-naïve and previously treated wAMD patients enrolled in the Phase I trial. Encouraged by these initial results, the company commenced patient recruitment for a Phase II expansion study in China during the fourth quarter of 2025, while the Phase I dose-escalation study was still ongoing. The company's objective is to rapidly evaluate and identify the optimal therapeutic dose, laying a solid foundation for upcoming Phase III clinical studies in the near future.