RegeneRx Biopharmaceuticals, Inc. today announced that it met with the FDA in September and as a result of the meeting may begin Phase 3 to evaluate RGN-259 preservative-free eye drops for the treatment of patients with neurotrophic keratopathy (NK), an orphan disease of the cornea. The Company believes clinical trials could be started by mid-2015.
As is typically required by FDA, the Company is planning to conduct two pivotal phase 3 trials, anticipated to total less than 90 patients to confirm the safety and efficacy of RGN-259 for NK. It is estimated that each trial will take approximately six to nine and months will be designed based on the results of an earlier trial conducted by ophthalmologists at Wayne State University and Beaumont Hospital in Detroit, MI. In moving from Phase 2 to Phase 3 the Company, through its contractors, intends to expand CMC efforts (chemistry, manufacturing, and controls) as required by FDA regulations. RegeneRx has been working with Ora Inc., a global ophthalmic research and product development firm, to develop the regulatory pathway and implement the clinical development plan on this orphan indication. RegeneRx recently retained Ora as CMC consultant and has begun evaluating manufacturers with Phase 3 and commercial production capabilities for this product. The Company plans to submit a CMC plan and final clinical protocol to FDA at the earliest practicable time.
"We are very pleased that we can begin Phase 3 clinical trials with RGN-259, which has shown promise in treating patients with NK, as well as with dry eye syndrome. The FDA was very helpful by providing guidance for Phase 3, which it has done throughout development of this product. We believe the development of RGN-259 for NK, an orphan disease in the U.S. and EU, offers the best and fastest opportunity for commercialization in the U.S. while we await the development of RGN-259 for dry eye syndrome by our two partners in their respective licensed territories. We also look forward to continuing discussions with ophthalmic and pharma companies that have been following our recent progress in this field." stated J.J. Finkelstein, RegeneRx's president and chief executive officer. "As reported in our regulatory filings, we would need to raise additional capital to fully implement this Phase 3 program, or enter a strategic partnership with a company having the resources and capabilities necessary for development of RGN-259," Mr. Finkelstein added.
"NK is a degenerative disease that can cause serious damage to the eye if left untreated, with stromal melting leading to perforation and/or ulceration," said George Ousler, vice president, dry eye at Ora, Inc. "Continuing to show positive results in Phase 3 will be an important breakthrough for treating NK. We are also excited about the initial results in dry eye patients and the opportunity for development of RGN-259 for this underserved patient population."
RGN-259 is a sterile, preservative-free topical eye drop being developed for ophthalmic disorders. Based on a physician-sponsored study, RGN-259 was shown to heal chronic corneal ulcers resulting from neurotrophic keratopathy (NK) in 6 patients out of six patients with Stage 2 and Stage 3 NK. In a recently completed Phase 2 clinical trial in 72 patients with moderate dry eye syndrome, RGN-259 was found to show statistically significant improvements in several signs and symptoms of dry eye, as well as positive trends in other outcome measures. In another clinical trial in 9 patients with severe dry eye, those treated with RGN-259 showed a statistically significant improvement in signs and symptoms compared to those receiving placebo. In each study the drug candidate was safe and well-tolerated. In December 2013, RGN-259 received orphan designation for NK in the U.S. The designation, for novel drugs to treat diseases with a prevalence of less than 200,000 patients in the U.S., provides seven years of extended marketing exclusivity, waiver of FDA user fees, tax credits of up to 50% of qualified clinical trial expenses, and eligibility of FDA research grants for clinical trials.
Neurotrophic Keratopathy (NK) is a serious degenerative disease of the corneal epithelium (the outside layer of the eye). A reduction in corneal sensitivity or complete corneal anesthesia is the hallmark of this disease and is responsible for producing corneal ulceration, perforation, pain and impaired vision. NK typically has three stages: Stage 1 is characterized by mild, nonspecific signs and symptoms. The viscosity of the tear mucus increases. There is decreased tear break-up time, leading to dry spots on the epithelium. Stage 2 involves a non-healing corneal epithelial defect. The surrounding epithelium becomes loose and the stroma swells and becomes edematous. Characteristic of this stage, the defect forms a punched-out oval or circular shape. Stage 3 often ensues if stages 1 and 2 are not treated appropriately and is characterized by stromal melting leading to perforation and/or ulceration.
RegeneRx is focused on the development of a novel therapeutic peptide, Thymosin beta 4, for tissue and organ protection, repair and regeneration. RegeneRx currently has three drug candidates in clinical development for ophthalmic, cardiac, CNS and dermal indications, strategic licensing agreements in China, the EU, and in other Pan Asia countries, including Korea, Japan and Australia, and has an extensive worldwide patent portfolio covering its products.
Ora, Inc. is a global, full service, ophthalmic research and product development company. Over the past 30 years, it has helped clients earn 37 FDA approvals. Ora supports a wide array of organizations, from start-ups to global pharmaceutical and device companies, to efficiently and successfully bring their new products from concept to market. Ora brings together the world's most extensive and experienced network of ophthalmic experts and R&D professionals in order to maximize the value of new product initiatives.
Any statements in this press release that are not historical facts are forward-looking statements made under the provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements concerning clinical development and future commercialization of RGN-259, the design and length of clinical trials, and other regulatory interaction. Any forward-looking statements involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. There can also be no assurance that any clinical trial undertaken by RegeneRx or Ora, Inc. will be commercially successful or that the FDA or other government agency will not have additional comments, restrictions, or guidance revisions regarding future clinical development or drug applications. Please view these and other risks described in the Company's filings with the Securities and Exchange Commission ("SEC"), including those identified in the "Risk Factors" section of the annual report on Form 10-K for the year ended December 31, 2013, and subsequent quarterly reports filed on Form 10-Q, as well as other filings it makes with the SEC. Any forward-looking statements in this press release represent the Company's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. The Company specifically disclaims any obligation to update this information, as a result of future events or otherwise, except as required by applicable law.
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