"A World without Blindness" - the goal of the company since Acucela 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 have been evaluating opportunities to expand our ophthalmic product pipeline through initial research to further advance our program and are currently in development for emixustat hydrochloride, our leading investigational drug candidate, Optogentics, gene delivery tools for ocular gene therapy, and remote medical monitoring device.
(1) 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.
(2) Optogentics, gene delivery tools for ocular gene therapy
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 is independent of genotype. Since human rhodopsin (hRho) is an endogenous human protein native to the retina, hRho 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.
Luxturna, the first FDA-approved gene therapy for a genetic disease, becomes available to patients in 2017 for $850,000 treatment cost on both eyes. The area of gene therapy development is anticipated to grow at a fast pace.
(3) Remote Medical Monitoring Device, home-based miniature OCT (Optical Coherence Tomography)
Our lead development project for mobile Health (mHealth) applications is the Patient Based Ophthalmology Suite (PBOS). Key features of PBOS are low cost and patient-friendly design, to enable it to be used directly by patients at home.
We are also developing a compact OCT device for NASA's Deep Space missions. With our unique approach, we are able to create a solid-state OCT yielding high resolution imagery. The final flight-ready device will allow NASA to replace their current COTS OCT devices with smaller, lighter, easier to use, durable and radiation hardened instruments that are practical for use in smaller spacecrafts, while providing required image quality from astronauts during flight.
By using network connectivity and cloud-based technologies, physicians will have a remote access to monitor disease progression and re-treatment needs for the patients on ground and even astronauts in space.
The unfortunate truth is that millions of patients suffer from sight debilitating disorders and we at Acucela feel that it is our privilege and duty to help them. Our company is made up of talented scientists and professionals who are dedicated in advancing and developing innovative therapeutics and medical technologies to help patients improve their quality of life. The strides we have made so far show our commitment to this journey and we will not stop.
Thank you for supporting our vision and together we will march towards fulfilling that goal of having a world without blindness.
Ryo Kubota, MD, PhD
Representative Executive Officer, Chairman, President and CEO
Kubota Pharmaceutical Holdings Co., Ltd.