Eyes On Kiora - ABACUS-2 And KLARITY Data Readouts Due In 2027

(RTTNews) - The eyes are often called the windows to the soul, but they also reflect our overall health. One of the most important parts of our eyes is the retina, a layer of cells at the back of the eyeball. The retina converts light into electric signals, which are sent to the brain through the optic nerve, where these signals are processed into the images that we see.

The company we are profiling today is Kiora Pharmaceuticals (KPRX), a clinical-stage biopharmaceutical company developing advanced treatments for retinal diseases. It has two drug candidates - KIO-301 and KIO-104 - both of which are under clinical development.

-- KIO-301 is being developed for the treatment of inherited retinal diseases, including retinitis pigmentosa, which causes the eye's light-sensing cells (rods and cones) to degenerate, leading to vision loss. It works by targeting surviving cells in the retina (the Retinal Ganglion Cells or RGCs), which connect the eye to the brain. KIO-301 enters these cells and, when activated by light, allows them to send signals to the brain, aiming to restore visual function. This compound has "on" and "off" positions controlled by the presence or absence of light and acts as a "molecular photoswitch."

A phase 2 clinical trial of KIO-301 for vision restoration in patients with retinitis pigmentosa, dubbed ABACUS-2, is underway. The goal of the study is to investigate the safety, tolerability and efficacy of up to 3 doses of KIO-301 administered by intravitreal (IVT) injection bilaterally every 6 weeks in patients with late-stage retinitis pigmentosa.

The study is designed to enrol 36 patients with ultra-low vision or no light perception, regardless of their underlying gene mutation associated with retinitis pigmentosa.

Retinitis pigmentosa is an inherited eye disease that leads to gradual vision loss over time. It typically begins with night blindness, followed by a progressive narrowing of the visual field, often described as tunnel vision. As the condition advances, patients experience an eventual loss of central vision, significantly impacting daily activities and independence. The progression of vision loss varies among individuals, but on average, half of those affected are no longer eligible to drive by the age of 37, and many become legally blind by around 55 years old. At present, there is no definitive cure or treatment for this retinal disease.

In the United States, roughly 100,000 people are affected by retinitis pigmentosa.

Under an agreement signed in January 2024, Kiora granted Théa Open Innovation, an independent European pharmaceutical company, exclusive worldwide development and commercialization rights, excluding Asia, to KIO-301 for the treatment of degenerative retinal diseases. The company has partnered with Senju Pharmaceutical Co., Ltd., on KIO-301 for the treatment of ophthalmic diseases in Asia, including Japan and China.

-- Next up is KIO-104, being developed for the treatment of patients with retinal inflammation. It works by suppressing certain T-cells and cytokines in the eye that drive damaging inflammation.

A phase 2 clinical trial evaluating KIO-104 for the treatment of patients with retinal macular edema, dubbed KLARITY, is underway. In the study, patients with macular edema secondary to one of the diseases, say, diabetic retinopathy, non-infectious uveitis, retinal vein occlusion, or post-pseudophakic cataract surgery, will be enrolled.

The study, designed to enrol up to 28 patients, will be conducted in 2 parts:

Part A (Dose Optimization) will assess the safety and efficacy of three injections administered once every two weeks in 8 subjects. Cohort 1 will receive 3.5 µg doses of KIO-104 while Cohort 2 will receive 10 µg doses of KIO-104. Part B (Cohort Expansion) will investigate different dosing regimens (2-week versus 4-week intervals) in the remaining subjects at a dose selected from Part A.

Kiora Pharma holds the worldwide rights to KIO-104.

The trial readouts of ABACUS-2 and KLARITY are anticipated in early 2027.

Cash Position

Kiora ended the second quarter of 2025 with cash, cash equivalents, and short-term investments of $20.7 million, along with an additional $3.1 million in receivables and tax credits. This financial position is expected to provide the company with a cash runway extending through late 2027.

Formerly known as EyeGate Pharmaceuticals Inc., the company rebranded as Kiora Pharmaceuticals Inc. on the Nasdaq under the symbol KPRX on November 8, 2021, to reflect its sharpened focus on developing novel ophthalmic therapeutics.

KPRX has traded in a range of $2.21 to $4.18 in the last 1 year. The stock closed yesterday's trading at $2.47, up 6.93%.