Key Takeaways

  • Kiora has obtained exclusive rights outside Asia to KIO-401, an investigational oral calpain inhibitor being developed for CRAO and NAION.
  • Clinical studies conducted in Japan reported improvements in visual acuity, including statistically significant findings in a phase 2a comparison with an observational registry and a phase 3 subgroup of patients who were legally blind at baseline.
  • Kiora plans a multinational phase 3 CRAO trial beginning in 2027 and a phase 2b NAION trial in 2028, subject to funding and regulatory considerations.

Kiora Pharmaceuticals has obtained exclusive development and commercialization rights outside Asia to KIO-401 (mitucapistat), an investigational oral calpain inhibitor for central retinal artery occlusion (CRAO), from Senju Pharmaceutical. The company plans to initiate a multinational phase 3 registration trial in CRAO in 2027 and expand development into nonarteritic anterior ischemic optic neuropathy (NAION).

Announcing the agreement, Shuhei Yoshida, president and CEO of Senju Pharmaceutical, said, “The clinical results generated in Japan support continued global development of this compound as a potential treatment for acute retinal ischemia.”

Senju has completed phase 2a and phase 3 clinical studies of the compound, designated SJP-0008 in Japan. In a phase 2a study, KIO-401 demonstrated a statistically significant improvement from baseline in ETDRS visual acuity compared with outcomes from a prospective observational registry (P = .0398), according to Kiora. A subsequent randomized, double-masked phase 3 study demonstrated improvement in BCVA. According to Kiora, the study showed a statistically significant improvement among patients who were legally blind at baseline under World Health Organization criteria (P = .0195).

KIO-401 is designed to inhibit activated calpain, a calcium-dependent protease implicated in cellular injury following ischemia. According to Kiora, inhibiting calpain may help limit downstream cellular damage and preserve visual function following ischemic injury.

The planned multinational phase 3 CRAO trial will be randomized, double-masked, and controlled, with an anticipated enrollment of more than 100 patients. The final study design, including the primary endpoint and enrollment criteria, remains subject to regulatory discussions. Trial initiation is contingent on funding and regulatory alignment.

Kiora also plans to initiate a phase 2b trial of KIO-401 in NAION in 2028, subject to funding and regulatory considerations. The company also identified glaucoma and other ophthalmic disorders involving calpain-mediated injury as potential future areas of investigation.