FDA Tells RegeneRx It Needs 3rd Phase III Trial On RGN-259 For Dry Eye Syndrome
Executive Summary
The FDA is requiring an additional RGN-259 Phase III trial to further show efficacy in both signs and symptoms of dry eye in a larger patient population.
Although the FDA has accepted all safety data from two Phase III trials of dry eye treatment candidate RGN-259, the agency wants the drug's backers to conduct an additional Phase III trial to further demonstrate its efficacy, a requirement that will push back its prospects for approval in treating a condition with high unmet medical need that affects more than 344 million patients globally.
The RGN-259 setback was announced on April 9 by RegeneRx Biopharmaceuticals Inc. and South Korea's GtreeBNT Co., Ltd, who are jointly developing the candidate therapy in the US and Canada for ophthalmic indications through their ReGenTree LLC joint venture.
News that an additional Phase III trial is needed follows the release in November of mixed results from ReGenTree's second pivotal Phase III clinical trial (ARISE-2) in the US of RGN-259 for the treatment of dry eye. ARISE-2 was not successful in duplicating the results of ARISE-1, where the study population was limited and less diversified and the mixed results fanned doubts over whether RGN-259 would be approved for the general dry-eye population.
ARISE-3 To Start This Year
Those doubts will grow after news that the FDA wants an additional Phase III evaluation done for RGN-259 before it can progress further down its planned regulatory pathway.
"ReGenTree is now planning to initiate the ARISE-3 trial this year." - ReGenTree CEO Won Yang
"Although the FDA is requiring an additional Phase III trial (ARISE-3) to further demonstrate efficacy in both signs and symptoms of dry eye in a larger patient population, all safety data from ARISE-1 and ARISE-2 were accepted by the FDA; no additional nonclinical efficacy and safety studies are required by the FDA, and the company's chemistry and manufacturing control plans for the drug substance and drug product were considered complete and acceptable for NDA submission," ReGenTree's CEO Won Yang said in a statement, adding: "ReGenTree is now planning to initiate the ARISE-3 trial this year."
RGN-259 is a sterile, preservative-free eye drop formulation manufactured using blow-fill-seal technology that has as its active pharmaceutical ingredient the novel compound thymosin beta 4 (TB4), a 43-amino acid peptide occurring naturally in all tissues.
There are two main types of dry eye disease: aqueous tear-deficient dry eye where the lacrimal glands fail to produce enough of the watery component of tears to maintain a healthy eye surface; and evaporative dry eye which may result from inflammation of the glands that produce the lipid or oily part of tears needed to slow evaporation and keep the tears stable.
Dry eye disease is currently treated mainly with artificial tears, corticosteroid drops, cyclosporine drops, or tear-stimulating drugs, like cholinergics.
In the US, there are only two main prescription drugs currently approved for dry eye disease: Allergan PLC's Restasis (cyclosporine) and Xiidra from Shire PLC. The number of available therapies does not increase drastically outside the US, analysts say.
ReGenTree said it would be attending upcoming ARVO (The Association for Research in Vision and Ophthalmology) 2018 annual meeting on April 29-May 3 in the US "to work with key opinion leaders as well as meet with potential strategic partners."