CEO Message


Dear Shareholders,

"A World without Blindness" - the goal of the company hasn't changed since the company was founded in 2002. Our greatest mission is to translate innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. We at Kubota Pharmaceutical group feel that it is our privilege and duty to help improve patients' quality of life with our innovative therapeutics and medical technologies.

We are also excited and proud to be working with NASA's research and development program, making great progress with our phase 1 Swept Source-OCT ("SS-OCT") device to be used in space. We look forward to seeing what the future holds. Additionally, our ongoing Phase 3 clinical trial investigating emixustat in patients with macular atrophy secondary to Stargardt disease has also been making great strides and will complete enrollment by the end of April 2020. We are one step closer to helping patients with such a devastating disease as Stargardt disease.

While we continue to evaluate new opportunities to expand our ophthalmic product pipeline, we are currently focusing on three programs in development: emixustat hydrochloride, our leading investigational drug candidate; optogenetics, gene therapy to restore vision; and a remote retinal monitoring device, home-based miniature OCT (Optical Coherence Tomography).

Emixustat Hydrochloride

The common treatments that are currently being used for retinal diseases are invasive. A needle is utilized to administer drugs directly into the eye. This method of administration increases the anxiety and recovery burden of the patients. Because emixustat is being developed as an oral treatment, we believe this innovative drug candidate has a high safety profile and will diminish the patient's frustration by minimizing the burden of the treatment on the patient's quality of life.

Emixustat is designed to specifically target the visual cycle in an effort to reduce accumulation of toxic by-products and reduce oxidative stress. We are exploring emixustat's potential to stop or slow the progression of vision loss in patients diagnosed with Stargardt disease in an ongoing clinical study. The target total number of subjects for this study is 162; that goal was met on March 30 and a total of 171 patients had been enrolled as of April 14, 2020. We will focus on completing the study and look forward to top-line results after a 24-month treatment for all subjects.

Optogenetics, gene therapy to restore vision

Our gene therapy program is an optogenetic approach for the treatment of Retinitis Pigmentosa (RP). Symptoms of RP most commonly occur in early childhood and affect nearly 1 in 4,000 people worldwide. Approximately 1.4 million people worldwide are affected by RP. There are currently no treatment options for patients with RP, and the disease's progression to blindness is inevitable. Our approach, which is independent of genotype, inserts the human rhodopsin gene (hRho) into retinal cells that are not normally light sensitive to enable them to sense light, with the goal of restoring vision. Because hRho is an endogenous human protein native to the retina, it could lower the immunological risk of such a therapy, enabling us to develop a potentially safe and effective treatment for vision restoration in advanced RP patients.

The area of gene therapy is anticipated to grow at a fast pace. In 2017, Luxturna became the first FDA-approved gene therapy for a genetic disease. It has since been available to patients at a cost of $425,000 per eye. Luxturna was developed by Spark Therapeutics, which has since been acquired by Novartis Pharmaceuticals.

Remote Retinal Monitoring Device, home-based miniature OCT (Optical Coherence Tomography)

Our lead development project for mobile Health (mHealth) is the Patient Based Ophthalmology Suite (PBOS). Key features of PBOS are low cost and a patient-friendly design that will enable the monitoring of disease progression directly by patients in their homes, supporting an effective and optimal treatment regimen via network connectivity and cloud-based technologies. The recent outbreak of COVID-19 has triggered more demand for remote medical devices, and we will advance the program to bring it to market as quickly as possible.

Relying on our PBOS technology, we aim to develop a compact OCT device for NASA's deep space mission that can diagnose, monitor and eventually enable treatment of Spaceflight Associated Neuro-ocular Syndrome (SANS) for spaceflight crewmembers, of which approximately 69% of long-duration spaceflight crewmembers present one or more signs. In January 2020, NASA provided us with positive feedback on our development program, commenting they recognize the tremendous potential of our device.

Thank you for supporting our vision and together we will march towards fulfilling that goal of having a world without blindness.

April 2020
Ryo Kubota, MD, PhD
Representative Executive Officer, Chairman, President and CEO
Kubota Pharmaceutical Holdings Co., Ltd.