HLB confirms NK treatment's safety, effectiveness in phase 3 study

HLB Therapeutics said on Monday that the first phase 3 clinical trial results (SEER-1) of its Neurotrophic Keratopathy (NK) treatment RGN-259 demonstrated effectiveness and safety in a recently published study in the International Journal of Molecular Sciences.

The research team comprising of HLB Therapeutics researchers and U.S. clinicians confirmed the efficacy and safety of 0.1 percent RGN-259 ophthalmic solution in promoting the healing of persistent epithelial defects in patients with Stages 2 and 3 NK.

NK is a rare ophthalmic disease and is a degenerative corneal disease that causes a decrease or loss of corneal sensation. Initially, corneal epithelial tissue is damaged, but it can develop into serious symptoms such as ulcers, melting, and perforation if not properly treated.

Thymosin beta-4 (T4), a medicinal substance of RGN-259, is most popular as a new drug treatment in ophthalmic diseases because it can treat corneal wounds, promote cell migration, and has anti-inflammatory effects. It has been designated as a rare drug for NK by the U.S. FDA and is  still under development with its subsidiary in the US, ReGenTree.

Complete healing occurred after four weeks in six of the 10 RGN-259-treated subjects and in one of the eight placebo-treated subjects (p = 0.0656), indicating a strong efficacy trend. Additionally, no recurrent defects were observed at day 43, two weeks after cessation of treatment, while the one healed placebo-treated subject at day 28 suffered a recurrence at day 43.

The researchers also reported no significant adverse effects. Overall, the use of 0.1 percent RGN-259 promoted rapid healing of epithelial defects in neurotrophic keratopathy, improves ocular comfort, and is safe for treating this challenging population of patients.

"Although 65,000 patients are currently suffering from this disease in the U.S. alone, the only treatment, Oxervate (ingredient: cenegermin-bkbj) is limited in terms of the high price of $100,000 (127 million won) for eight weeks of treatment, together with inconvenient administration and storage of the product," said HLB Therapeutics CEO Ahn Ki-hong. “We will focus on two Phase 3 clinical trials (SEER-2 and SEER-3) simultaneously underway in the U.S. and Europe, to make RGN-259 a 'best-in-class' global drug."