HLB submits clinical protocol for neurotrophic keratitis rare drug candidate to FDA

HLB Therapeutics said that it submitted a clinical protocol to the U.S. FDA on Monday to conduct a second phase 3 clinical trial for a neurotrophic keratitis (NK) treatment through its U.S. subsidiary, ReGenTree.

NK is a degenerative disease in which the sensation of the cornea gradually decreases, and in severe cases, corneal perforation occurs.

According to the company, the second phase 3 clinical trial (SEER-2) and the third phase 3 clinical trials (SEER-3) will be simultaneously conducted in the U.S. and Europe, respectively, to shorten the treatment lead-time for NK. The clinical design was designed based on the results of the first phase 3 clinical trial (SEER-1), where positive results were derived. Clinical drugs and placebo will be administered to about 70 patients for four weeks to evaluate the cure rate of corneal wounds.

Currently, the only approved treatment is Oxervate from Dompe, Italy, a recombinant protein formulation of the human nerve growth factor, but the FDA-approved eight-week treatment costs a minimum of $110,000 (156 million won). Also, it needs to be refrigerated before administration, which has a high discomfort and complicated dosing preparation process.

However, 'RGN-259', an eye disease treatment substance of HLB, is a disposable vial eye drops and can be used and stored the same as ordinary eye drops. The treatment period will also be reduced by four weeks than the existing product and has been designated by the FDA as a rare drug for the treatment of NK.

“We anticipate that by having two trials in the U.S. and Europe at the same time, we will be able to significantly reduce the clinical lead-time to the level of one Phase 3 clinical trial," said an HLB Therapeutics official, "We plan to proceed with the application for authorization after completing the trial by 2024 (SEER-2) in the United States and 2025 (SEER-3) in Europe."

Although neurotrophic keratitis is a rare disease, the expected market size for 2027 is projected to be $324 million.