The protocol for a second phase 3 clinical trial for a treatment of neurotrophic keratopathy (NK) – a degenerative disease of the corneal epithelium resulting from impaired corneal innervation, has been submitted.
RegeneRx Biopharmaceuticals announced that its U.S. joint venture partner and licensee, HLB Therapeutics, together called ReGenTree, submitted the submission to the U.S. Food and Drug Administration (FDA) on September 26 evaluating RGN-259 as a treatment for NK.
In July, HLB announced that it had signed a contract with a global contract research organization (CRO) to simultaneously conduct SEER-2 as the second Phase 3 clinical trial in the United States and SEER-3 as the third clinical trial. phase 3 in the United States and Europe to obtain approval for NK treatment as soon as possible.
Recruitment of clinical studies
The clinical study design of SEER-2 and SEER-3 is based on the results of the first phase 3 clinical trial (SEER-1), where the number of subjects was relatively small, but good results were obtained. In the SEER-2 and SEER-3 studies, approximately 70 patients will be enrolled in each study to assess efficacy, in particular complete recovery after four weeks of treatment.
ReGenTree is seeking clinical trial sites by contacting over 70 hospitals and eye clinics and aims to start at its first site in November this year (2022).
For the SEER-3 clinical trial, which will be conducted simultaneously in the United States and Europe, the study site selection process is underway by ReGenTree from among 80 candidate sites in six European countries, and the preparation of applications for deposit via CTI (Clinical Trials Information System) is also underway.
NK is a degenerative disease that reduces corneal sensitivity and, in severe cases, worsens to corneal perforation, and is a rare disease that occurs in approximately five out of every 10,000 people in the United States. The only approved treatment so far is Italian pharmaceutical company Dompé’s Oxervate, a recombinant form of human nerve growth factor that is extremely expensive for eight weeks of treatment.
RGN-259 previously received orphan drug designation for the treatment of NK from the FDA, and the company says a notable difference to many rare diseases is that the market is very large. It states that due to the prevalence of NK patients who need to be treated despite high drug cost, the market is expected to reach US$324 million in 2027. One of the goals of SEER-2 and SEER-3 is to successfully complete the treatment. within four weeks.
ReGenTree plans to significantly shorten the development period for RGN-259 by conducting the two Phase 3 clinical trials in the United States and Europe simultaneously rather than sequentially. Therefore, HLB Therapeutics is focusing all its capabilities to achieve the possibility of applying for an FDA Biologics license application in 2025 as the ultimate goal of its NK project.
Dry eye syndrome
RGN-259 is a sterile, preservative-free eye drop developed to reduce damage and improve healing for various ophthalmic indications, including dry eye syndrome and neurotrophic keratopathy.
To date, over 1,700 subjects have received RGN-259 indicating varying degrees of efficacy in both dry eye and NK. RGN-259 has been shown to be fast acting, well tolerated and does not have the documented side effects of other pharmaceuticals approved for these indications.
Thymosine beta 4, the active pharmaceutical ingredient of RGN-259, which has demonstrated various mechanisms of action including promotion of cell migration, cytoprotection, wound healing and anti-inflammatory properties.