Kubota Vision Inc. Signs Supply and Licensing Agreement with Laboratoires KÔL for Stargardt Disease Treatment Candidate Under Compassionate Use

Kubota Vision Inc. (“Kubota Vision”), a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Minato-ku, Tokyo, Japan) announced signing of a Supply and Licensing Agreement with Laboratoires KÔL (Clermont-Ferrand, France) for the provision of a Stargardt disease (STGD1) treatment candidate under compassionate use authorization.

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The purpose of this Agreement is for Kubota Vision and KÔL to collaborate on providing “Emixustat” for treatment of Stargardt Disease (STGD1) through compassionate use authorization in France.

Kubota Vision will exclusively manufacture and supply Emixustat final products to KÔL and KÔL will have exclusive rights to distribute Emixustat in France under compassionate use access.

Ryo Kubota, Chairman, President, and CEO of Kubota Pharmaceutical Holdings, stated, “We are extremely pleased to enter into this Agreement with Laboratoires KÔL for the development, supply and distribution of Emixustat for Stargardt disease (STGD1). By combining Kubota Vision’s clinical development and visual cycle modulation technology with KÔL’s expertise in rare ocular diseases, we aim to deliver meaningful impact for patients and the global ophthalmic community.”

Sophie Momège, Founder and CEO of Laboratoires KÔL, commented, “We are honored to collaborate with Kubota Vision to explore the development and commercialization of Emixustat for Stargardt disease, a condition that currently has no cure. By combining our expertise with Kubota Vision’s proven clinical capabilities, we aim to bring one of the first effective treatments to patients worldwide and help reduce vision loss among children and adults.”

About Stargardt disease (STGD1)

Stargardt disease (STGD1) is a rare hereditary retinal disorder that typically develops in childhood or adolescence and causes gradual loss of vision. It is also known as Stargardt macular dystrophy or juvenile macular degeneration. The condition is primarily caused by mutations in the ABCA4 gene, which lead to progressive damage of the photoreceptor cells and subsequent decline in visual acuity. Symptoms include central vision loss, blurred or distorted vision, reduced visual field, and color vision abnormalities. While most cases appear early in life, symptoms may also emerge in adulthood. The retina enables vision through the visual cycle, a process that converts light into electrical signals sent to the brain. During this cycle, light absorption by visual pigments generates vitamin A–derived byproducts that are toxic if not properly cleared. In a healthy retina, these compounds are removed from photoreceptor cells by a membrane transporter, preventing damage to the retinal pigment epithelium (RPE).

In Stargardt disease, mutations in the ABCA4 gene impair this protective mechanism. As a result, toxic metabolites accumulate within RPE cells, leading to cellular dysfunction and triggering apoptosis. The progressive death of RPE cells causes secondary loss of photoreceptors, ultimately resulting in progressive vision loss and, in severe cases, blindness. The accumulation of these toxic metabolites within RPE is considered to be the direct cause of Stargardt Disease.

Currently, no approved treatment is available for Stargardt disease.

About Emixustat

Emixustat is expected to suppress the progression of Stargardt disease (STGD1) by selectively inhibiting RPE65, a key enzyme in the visual cycle, through Kubota group’s proprietary visual cycle modulation (VCM) technology. This selective inhibition reduces the accumulation of metabolic waste products generated during the visual cycle.

Visual Cycle Modulation (VCM) is a therapeutic approach designed to reduce the accumulation of toxic byproducts in the retina during the visual cycle. These toxic compounds contribute to oxidative stress and light‑induced retinal damage, particularly within retinal pigment epithelium (RPE) cells, which continuously phagocytose photoreceptor outer segments while accumulating visual cycle byproducts.

Emixustat hydrochloride modulates this process by selectively targeting rod cells without affecting cone cells. It inhibits key enzymes involved in the visual cycle, thereby reducing rod cell activity and slowing the formation of toxic metabolites. By decreasing their accumulation in RPE cells, visual cycle modulation helps protect the retina and may delay disease progression.

About Laboratoires KÔL

Laboratoires KÔL is a French pharmaceutical company focused on rare diseases, with a strong emphasis on ophthalmology. The company possesses deep expertise in corneal grafting, access authorization pathways (AAC/AAP), and preventive eye-care therapies, pioneering innovative treatments for rare corneal disorders. Drawing inspiration from ancient eye-care traditions such as Egyptian “khôl” and integrating cutting-edge medical science, Laboratoires KÔL collaborates with hospitals, research institutions, and regulatory bodies to deliver patient-centric therapeutic solutions worldwide. The company engages in the research, development, manufacturing, and commercialization of drugs for rare diseases. Its proprietary antisense oligonucleotide platform is developed into 2 different eyedrops formulations respectively for corneal disease (Olisens^®), and retina. A large clinical program is on-going for this antiangiogenic messenger RNA technology with preliminary promising results in both indications. For more information, visit https://www.laboratoires-kol.com/

About Kubota Vision Inc.

Kubota Vision Inc. is a wholly owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596), committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. Kubota Pharmaceutical Group’s research and development pipeline includes Emixustat hydrochloride as a therapeutic candidate for Stargardt disease (STGD1) and proliferative diabetic retinopathy (PDR), as well as a VAP-1 inhibitor targeting Alzheimer’s disease and metabolic dysfunction-associated steatohepatitis (MASH). As medical device and related product portfolio includes Kubota Glass®, a wearable device designed to suppress the progression of myopia, and eyeMO®, a retinal monitoring device developed for home- and tele-ophthalmology use. Kubota Glass® is currently being marketed in both the Japanese and Chinese markets, further expanding the Kubota Pharmaceutical Group’s commitment to advancing ophthalmic innovation and accessible vision care solutions globally. For more information, visit kubotavision.com

The “Kubota” logo is registered trademark of Kubota Pharmaceutical Holdings Co., Ltd.

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