European Medicines Agency issues Orphan Medicinal Product Designation for KIO-310 from Kiora Pharmaceuticals

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Kiora Pharmaceuticals announced the European Medicines Agency (EMA) issued an Orphan Medicinal Product Designation for KIO-310, for the treatment of inherited retinal diseases (IRDs) including retinitis pigmentosa (RP) and choroideremia.

The designation covers KIO-30, a small molecule photoswitch that selectively confers light-sensing capabilities to retinal ganglion cells following the degeneration of photoreceptors in inherited retinal diseases, for the treatment of non-syndromic rod-dominant retinal dystrophies.¹

The designation is granted after a thorough review from the EMA and only if a drug candidate meets specific criteria: the investigational therapy is medically plausible and intended to treat a serious or life-threatening disease, and the condition affects no more than 5 in 10,000 people in the EU.

“This designation provides Kiora and our development and commercialization partner, Théa Open Innovation, with at least 10 years of market exclusivity, exclusive of patent protection, in Europe,” Eric Daniels, MD, chief development officer of Kiora Pharmaceuticals, said in a press release.¹ “This is in addition to other regulatory and market access benefits.”

Alongside market exclusivity, the designation will provide many benefits for the company, including a centralized process for EU market approval, reduced or waived fees for regulatory activities and EMA scientific advice and protocol assistance to optimize trial design.

Kiora Pharmaceuticals also stated that the upcoming ABACUS-2 trial of KIO-301 for vision restoration in patients with RP will be a multi-center, double-masked, randomized, controlled and multiple-dose study. Data from ABACUS reported in November 2023 demonstrated meaningful vision improvements in patients with late-stage RP.

Earlier this year, Kiora and Théa Open Innovation (TOI), a sister company of the global ophthalmic specialty company Laboratories Théa, signed an agreement to the exclusive worldwide co-development and commercialization, excluding Asia, for KIO-301 in the treatment of retinal diseases. At the time, Brian Strem, PhD, CEO of Kiora, spoke about the Phase I/II ABACUS trial findings.

“Based on the Phase I/II (ABACUS) data of KIO-301 in retinitis pigmentosa, we have started to implement our plan to initiate our Phase 2, multicentre, controlled clinical trial for retinitis pigmentosa, in early 2024 with the goal of reporting results in H1 2025 and explore other retinal disease where KIO-301 may be applicable,” he said in a news release at the time.²

Reference:

1. Kiora Pharmaceuticals Receives European Orphan Medicinal Product Designation for KIO-301 for the Treatment of Inherited Retinal Dystrophies. News release. Kiora Pharmaceuticals. Accessed July 30, 2024. Kiora Pharmaceuticals Receives European Orphan Medicinal Product Designation for KIO-301 for the Treatment of Inherited Retinal Dystrophies :: Kiora Pharmaceuticals, Inc. (KPRX)

2. Hutton D. Kiora Pharmaceuticals and Théa Open Innovation announce partnership, will target IRDs. Ophthalmology Times Europe. Published 2 February, 2024. Accessed 30 July, 2024. https://europe.ophthalmologytimes.com/view/kiora-pharmaceuticals-and-th-a-open-innovation-announce-partnership