Kiora Pharmaceuticals Receives Investigational New Drug Approval to Initiate ABACUS-2, a Phase 2 Clinical Trial of KIO-301 for the Treatment of Retinitis Pigmentosa

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[{"type":"text","content":"Encinitas, California--(Newsfile Corp. - October 29, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) (\"Kiora\" or the \"Company\") today announced it received regulatory approval to initiate a Phase 2 clinical trial to investigate KIO-301 for vision restoration in patients with retinitis pigmentosa. The ABACUS-2 trial will be a 36 patient, multi-center, double-masked, randomized, controlled, multiple dose study enrolling patients with ultra-low vision or no light perception regardless of their underlying gene mutation associated with retinitis pigmentosa. Dosing of the first patient with KIO-301 is expected to begin next year following validation of novel functional vision endpoints. These functional assessments may serve as approvable primary endpoints in subsequent registration studies in the United States, Europe and other major regions.\n\"There are unfortunately no approved therapies for patients with retinitis pigmentosa,\" said Eric Daniels, M.D., Chief Development Officer at Kiora. \"This study represents a significant step toward addressing this challenge. Prior to submission for approval, we engaged with European and US regulators to incorporate their expectations and guidance for approvable endpoints. Consistent with historical approval in other inherited retinal disease, both regulatory bodies emphasized the need to measure a therapy's effect on everyday functional vision. For this reason, we are investing time upfront to validate the functional endpoints for ABACUS-2, increasing our likelihood of success in a potential single Phase 3 trial for market approval in the US and Europe. This validation work is being performed, in collaboration with our partner Théa Open Innovation, with the support of the Choroideremia Research Foundation as part of a grant to design a standard endpoint for investigational therapies of inherited retinal diseases.\"\nKiora has identified and is now contracting with trial sites and clinical investigators at major inherited retinal disease reference centers across Australia. Patients will be randomized 2:1 to receive KIO-301 intravitreally or control. Treatment will be delivered to both eyes. Participants will be randomized to receive either a high dose (100 micrograms) or low dose (50 micrograms) of KIO-301 and after trial participants receive three consecutive doses (6 weeks apart), th...

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