US-based RegeneRx Biopharmaceuticals has received approval from the US Food and Drug Administration (FDA) to start Phase III trials to evaluate RGN-259 preservative-free eye drops to treat patients with neurotrophic keratopathy (NK), an orphan disease of the cornea.
RGN-259 is a sterile, preservative-free topical eye drop under development for ophthalmic disorders.
By mid-2015, the company intends to conduct two pivotal Phase III trials, which are expected to enrol about 90 patients, to confirm the safety and efficacy of RGN-259 for NK.
According to the company, each trial is estimated to take about six to nine and months and will be designed based on the results of an earlier trial conducted by ophthalmologists at Wayne State University and Beaumont Hospital in Detroit.
In moving from Phase II to Phase III, the company, through its contractors, expects to expand chemistry, manufacturing, and controls (CMC) efforts as required by FDA regulations.
The company has been working with a global ophthalmic research and product development firm Ora, to develop the regulatory pathway and implement the clinical development plan on this orphan indication.
Recently, RegeneRx retained Ora as CMC consultant and has started evaluating manufacturers with Phase III and commercial production capabilities for this product.
The company intends to submit a CMC plan and final clinical protocol to FDA soon.
RegeneRx president and chief executive officer JJ Finkelstein said: "We believe the development of RGN-259 for NK, an orphan disease in the US and EU, offers the best and fastest opportunity for commercialisation in the US while we await the development of RGN-259 for dry eye syndrome by our two partners in their respective licensed territories.
"As reported in our regulatory filings, we would need to raise additional capital to fully implement this Phase III programme, or enter a strategic partnership with a company having the resources and capabilities necessary for development of RGN-259."
The company said that based on a physician-sponsored study, RGN-259 was shown to heal chronic corneal ulcers resulting from NK in six patients out of six patients with Stage II and Stage III NK.