Kiora Pharmaceuticals has secured a significant partnership with Japanese pharmaceutical company Senju Pharmaceutical for the development and commercialization of KIO-301, a novel therapy for inherited retinal diseases, across key Asian markets. The exclusive option agreement covers Japan, China, and other Asian territories, with a total potential deal value of $110 million plus tiered royalties on sales.
Under the terms of the agreement, Kiora will receive an immediate $1.25 million exclusive option fee from Senju. If Senju exercises its option following topline data from the ongoing ABACUS-2 Phase 2 clinical trial, Kiora will receive an additional mid-single digit million upfront payment, along with development, regulatory, and commercial milestones.
Novel Molecular Photoswitch Technology
KIO-301 represents a breakthrough approach to treating inherited retinal diseases, functioning as what the company describes as a "molecular photoswitch." The therapy targets retinal ganglion cells (RGCs), which connect the eye to the brain, in patients whose light-sensing cells (rods and cones) have degenerated due to conditions like retinitis pigmentosa.
The small molecule enters surviving RGCs and, when activated by light, enables these cells to send signals to the brain with the aim of restoring visual function. This mechanism offers potential treatment for patients with ultra-low vision or no light perception due to inherited retinal diseases.
Phase 2 Clinical Development
KIO-301 is currently being evaluated in ABACUS-2, a multi-center, double-masked, randomized, controlled, multi-dose Phase 2 study involving 36 patients with ultra-low vision or no light perception due to retinitis pigmentosa. The trial design allows Senju to make its licensing decision based on concrete clinical data.
"Like elsewhere in the world, patients in Asia with inherited retinal diseases, including retinitis pigmentosa, have no therapeutic options to slow their disease progression nor restore what has already been lost," said Shuhei Yoshida, President of Senju. "KIO-301 is a very promising, novel therapeutic with the potential to give vision back to affected individuals."
Strategic Partnership Framework
The agreement structure allows Senju to collaborate with Théa Open Innovation, Kiora's exclusive global development and commercialization partner for KIO-301 outside of Asia. Kiora established its partnership with Théa Open Innovation in January 2024, receiving a $16 million upfront payment and potential milestone payments of up to $285 million, plus tiered royalties of up to 20% on net sales.
Under the potential Senju agreement, the Japanese company would assume responsibility for all development, regulatory, and commercial activities within the licensed Asian territory, including country-specific regulatory and commercial milestones.
Market Impact and Financial Implications
Brian Strem, PhD, CEO of Kiora, emphasized the strategic importance of the partnership: "This option agreement represents a critical step toward establishing a long-term strategic partnership with Senju and extends the global commercial reach of KIO-301 by building on the foundation established by Théa."
The combined relationships with Théa and Senju bring the total potential value of Kiora's strategic partnerships to over $400 million, significantly strengthening the company's financial position. The agreement is expected to extend Kiora's cash runway into late 2027, beyond the projected readout of both ongoing Phase 2 clinical trials.
As a leading ophthalmic pharmaceutical company in Asia, Senju's interest in KIO-301 validates the significant unmet medical need for treatments addressing inherited retinal conditions like retinitis pigmentosa and other retinal diseases in the region.
