Kubota Vision Partners with Laboratoires KÔL to Provide Emixustat for Stargardt Disease Under Compassionate Use in France
核心洞察
Kubota Vision Inc. (搜索) and Laboratoires KÔL (搜索) signed a supply and licensing agreement to provide Emixustat for Stargardt disease (搜索) treatment through compassionate use authorization in France.
Emixustat works by selectively inhibiting RPE65 (搜索), a key enzyme in the visual cycle, using Kubota's proprietary visual cycle modulation technology to reduce toxic metabolite accumulation.
The collaboration aims to address Stargardt disease (搜索), a rare hereditary retinal disorder with no currently approved treatments that causes progressive vision loss in children and adults.
Kubota Vision Inc. (搜索), a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (搜索), has announced a strategic partnership with French pharmaceutical company Laboratoires KÔL (搜索) to provide Emixustat for Stargardt disease (搜索) treatment under compassionate use authorization in France. The supply and licensing agreement represents a significant step toward addressing an unmet medical need for patients with this rare hereditary retinal disorder.
Partnership Details and Distribution Framework
Under the terms of the agreement, Kubota Vision will exclusively manufacture and supply Emixustat final products to Laboratoires KÔL (搜索), while KÔL will hold exclusive rights to distribute the treatment candidate in France under compassionate use access. This collaboration combines Kubota Vision's clinical development expertise and visual cycle modulation technology with KÔL's specialized knowledge in rare ocular diseases.
Ryo Kubota, Chairman, President, and CEO of Kubota Pharmaceutical Holdings, stated, "We are extremely pleased to enter into this Agreement with Laboratoires KÔL (搜索) for the development, supply and distribution of Emixustat for Stargardt disease (搜索) (STGD1 (搜索)). 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 (搜索), commented, "We are honored to collaborate with Kubota Vision to explore the development and commercialization of Emixustat for Stargardt disease (搜索), 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."
Understanding Stargardt Disease
Stargardt disease (搜索) (STGD1 (搜索)) is a rare hereditary retinal disorder that typically develops in childhood or adolescence and causes gradual loss of vision. Also known as Stargardt macular dystrophy (搜索) or juvenile macular degeneration (搜索), the condition is primarily caused by mutations in the ABCA4 (搜索) 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 disease affects the retina's visual cycle, a process that converts light into electrical signals sent to the brain.
In healthy retinas, light absorption by visual pigments generates vitamin A-derived byproducts that are toxic if not properly cleared. These compounds are normally removed from photoreceptor cells by a membrane transporter, preventing damage to the retinal pigment epithelium (RPE). However, in Stargardt disease (搜索), mutations in the ABCA4 (搜索) gene impair this protective mechanism, causing toxic metabolites to 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.
Currently, no approved treatment is available for Stargardt disease (搜索).
Emixustat's Mechanism of Action
Emixustat is expected to suppress the progression of Stargardt disease (搜索) by selectively inhibiting RPE65 (搜索), 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 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 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.
Company Profiles
Laboratoires KÔL (搜索) 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, and preventive eye-care therapies, pioneering innovative treatments for rare corneal disorders. The company engages in the research, development, manufacturing, and commercialization of drugs for rare diseases, with a proprietary antisense oligonucleotide platform developed into two different eyedrops formulations for corneal disease and retina applications.
Kubota Vision Inc. (搜索) is committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. The Kubota Pharmaceutical Group's research and development pipeline includes Emixustat hydrochloride as a therapeutic candidate for Stargardt disease (搜索) and proliferative diabetic retinopathy (搜索), as well as a VAP-1 inhibitor targeting Alzheimer's disease (搜索) and metabolic dysfunction-associated steatohepatitis (搜索). The company's medical device portfolio includes Kubota Glass, a wearable device designed to suppress the progression of myopia (搜索), and eyeMO, a retinal monitoring device developed for home- and tele-ophthalmology use.