Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX), an ophthalmic pharmaceutical company based in California, and the Choroideremia Research Foundation (CRF), a non-for-profit patient organisation focused on the search for a cure for choroideremia (CHM), have partnered to advance a potential new treatment for patients with choroideremia (CHM). The collaboration will accelerate Kiora’s development of “KIO-301”, a small molecule designed to restore vision in patients with later-stage retinal degeneration. Kathi Wagner, Executive Director of the Choroideremia Research Foundation, said, “this collaboration with Kiora represents a significant step forward in our mission to bring new options to patients living with choroideremia. There is only one other active clinical stage program in CHM, which is simply not enough. We believe that KIO-301 has the potential to make a significant difference in the lives of those affected by this disease.”
Choroideremia (CHM) is an X-linked retinal degeneration that impacts an estimate of 1/50,000 males worldwide, exhibiting retinal thickening, photoreceptor degeneration, RPE depigmentation, and retinal remodelling. Males develop nyctalopia in their teens progressing to a loss of peripheral visual field leading to profound blindness in the fifth and sixth decades. CHM encodes Rab Escort Protein 1 (REP-1) which is a ubiquitously protein required for geranylgeranylation of ras-related GTPases, or Rab proteins, trafficking of vesicles in endocytic and exocytic pathways. Currently, there are several gene therapy studies focused on CHM treatment using both primary and secondary outcomes. In a recent natural history study in CHM, performed at Yale, research showed that BCVA was a function of age, followed with a 2-phase decline, slow followed by rapid decline, with an estimated transition age of 39.1 years (95% CI 33.5 to 44.7). BCVA followed a 2-phase decline until it reached 0 letters (r2=0.90) thereafter. The BCVA decline rate was 0.33 letters/year (95% CI −0.38 to 1.05) before 39 years, and 1.23 letters/year (95% CI 0.55 to 1.92) after 39 years (p=0.004).
According to Kiora, there are an estimated 12,000 CHM patients in the United States and Europe, and qualifies as a rare disease under the US Orphan Drug Act. CHM shares traits with retinitis pigmentosa (RP), including several inherited gene defects and the death of photoreceptor cells. Under the collaboration between Kiora and CRF, access to clinical and scientific thought leaders will assist in further development of “KIO-301” – a small molecule that acts as a “photoswitch” designed to restore vision in patients with inherited and age-related degenerative retinal diseases. KIO-301 will be aimed to selectively transport the small molecule, viable downstream retinal ganglion cells, “to turn them into light sensing cells, capable of signalling the brain as to the presence or absence of light”. Following the announcement of the collaboration, Dr. Christine Kay, a vitreoretinal surgeon and recognized international expert in inherited retinal diseases, stated, “the need to develop new therapies for patients with CHM is clear, and with the potential behind KIO-301, it’s very exciting to see the CRF and Kiora collaborate towards this goal”. In follow up, Brian Strem, Ph.D., President & CEO of Kiora commented that, “with the early clinical evidence and understanding of how KIO-301 works, we see a clear path for its application to choroideremia. By joining forces with CRF, we hope to make significant strides in the fight against this disease and accelerate the path to bringing this therapy to all of those in need.”