Entrepreneur with a vision to cure blindness

Acucela CEO Ryo Kubota talks about building a biotech startup to cure blindness.

Japanese-born Ryo Kubota has an unassuming presence about him. I met the Seattle-based Acucela executive at the JP Morgan Healthcare Conference in San Francisco in January.

He told me about how he was developing a drug to treat age-related macular degeneration, or AMD, a progressive disease of the retina that causes loss of central vision.

This is where the scientist in Kubota emerged. He used to be a professor at the University of Washington before deciding to go the commercial route.

Now, Kubota is a businessman -- but he stays true to his Japanese roots. The president and & CEO of biotech firm Acucela, Kubota says he's running his business with Japanese money to have more control over the destiny of his firm.

SmartPlanet: What is Acucela trying to do? Can you explain the difference between dry and wet macular degeneration?

RK: Ultimately, we are trying to cure blindness. Our initial realistic goal is to slow down the progression of disease that is destined to go blind like AMD.

Dry AMD always precedes wet AMD. Some people can go blind by just the terminal dry form of AMD called geographic atrophy, or GA; some people go blind after conversion to wet with leaky vessels that cause edema and hemorrhage. We are hoping to slowdown the progression of dry AMD, as well as hoping to reduce the rate of conversion from dry to wet.

SmartPlanet: What do you hope to accomplish? When did you get the idea to develop a daily pill that could slow down vision loss?

RK: We are interested in helping people maintain good vision. We may develop a Google car with Google if that helps blinded people to move around.

Our initial approach is the drug. We continue to do this, but we may not be limited to the pharmaceutical approach to help people with vision.

A device, stem cell therapy and anything goes once we have a success with the visual cycle modulation that we are developing. VCM was conceptualized originally by Dr. Paul Sieving currently heading the National Institutes of Health's National Eye Institute who is a good friend of mine, followed up with Kris Palczewski [formerly Seattle UW, now at Case Western Reserve University; as well as Gabriel Travis at University of California, Los Angeles and others].

We were the first company to work with non-retinoid visual cycle modulators. The original work was done with Accutane [retinoid] as we know about the side effects that are associated with this molecule which inhibit us from using it chronically.

SmartPlanet: What happened? You were a professor and decided to start Acucela? Can you talk about what motivated you to do so?

RK: At the time when I was doing research at the University of Washington as a professor, I felt that starting a company should empower me most to drive the science towards the benefit of patients. The university setting is fantastic for the basic research, but not necessarily best suited for applied or translational research. I wanted to hire a multi-disciplinary team of chemists, biologists, clinical development experts, regulatory experts, and marketing experts to drive the development of the drug.

I had a choice of licensing to other pharma to develop the technology, but it was important for me to be fully responsible to make sure that we put our best effort in advancing this science. When you try to bring game changing technology or disruptive technology, there are a lot of people skeptical about your technology because it is too much outside the box. I strongly believe this technology and so far we have proven to be true by advancing to phase 2. I am sure many skeptics assumed that we would fail in phase 1.

SmartPlanet: Since you never had to raise money, how has this impacted the growth of your company? Are you public?

RK: We had to raise money [$40 million --Ed.] from Japanese VC in the past. We came up with a creative deal structure with Otsuka such that we don't need to raise capital anymore in exchange for their commercialization right in the half of U.S. and Asia. We retained half of the U.S., South America and E.U.

We recently secured a third exciting and promising glaucoma program from Otsuka in a similar structure, so we don't need to raise money until we opt in. We are hoping to raise money by going public once we have our products in phase three in the near future. We are not public yet.

We are planning to go public in Japan where there are long term investors not necessarily looking for quarter to quarter numbers which do not apply and are devastating to the developmental stage of biotech.

SmartPlanet: You said you are developing three products, AMD drug, dry eye drug, and glaucoma drug. That's a large part of the market. There's really no preventative treatment for these currently. Is that right? Are all of these drugs used to prevent vision loss or can the drugs reverse vision loss? What age of people are you targeting?

RK: As you know the retina is part of the central nervous system, which is not possible to regenerate at this point despite the efforts of stem cell research. The best we can do at this point is to stop the disease progression.

As we know, our vision will take about 10 years from birth to mature fully, just like motor neurons [e.g. humans can't walk at birth --Ed.], so regenerating neuronal cells in the eye and educating cells to mature [10 years in natural development] is very difficult at this point.

Having newly born cells will not likely be useful in terms of vision, unless they undergo rigorous training. It is almost like a computer without software, and it takes 10 years in the natural course of development to install this software and firmware.

SmartPlanet: How exactly does your drug, ACU-4429, work?

RK: ACU-4429 works by inhibiting the enzyme called isomerase or RPE65. This enzyme is not only responsible for the visual cycle which is a physiological process, but it is responsible for the generation of retinoid related toxic by-products.

We want to stop the generation of toxic by-products by modulating this enzyme.

SmartPlanet: Why did you partner with a drug company in Japan for the clinical tests? What's the latest in clinical tests?

RK: I am Japanese. Yes, it is partly due to my relationship and partly due to their generous support of, and understanding what is important for our company.

Many U.S. and E.U. companies were interested in our program, but Otsuka understood our needs most. I want to build the company to last, and I don't want to just flip the company and sell, which many financial advisors adviced for me to sell and live a happy retirement. I am not interested in doing so, I want to go beyond pharmaceuticals to help blindness.

My company has people from all over the world in addition to the people from U.S., and I am good at putting people together for common goal that they can be proud of and wanting to be part of. I would like to expand this model for global diplomacy to make the world better place to live and co-exist in a respectful way. This is why I work closely with the Japanese Embassy or the Department of State and other government entity as an entry point for my ultimate goal.

SmartPlanet: Do you actually believe you can cure blindness?

RK: Of course, 100 percent. I was able to discover and name the glaucoma gene despite all the negative odds against me. Many reputable biotech companies disappeared in the past eight years, yet we are still running. I think we are doing things right to continue to make success. I have wonderful people who are a lot more capable than I am in my company to realize my vision and dream, I cannot thank them more.

This post was originally published on Smartplanet.com

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