Based on pre-clinical models, Quark believes that AMD can be treated by blocking the expression of the RTP-801 gene through RNA interference.An Israeli company which has set its sights on providing an effective drug to combat a cruel vision loss disease has just got one big step closer to its goal.
Quark Biotech, Inc. (QBI) has licensed its novel human gene RTP-801 and its related molecules to American pharmaceutical giant Pfizer Inc. During eight years of research, Quark’s scientists in its Ness Ziona R&D facilities discovered that RTP-801 is involved in the development of pathologic blood vessels which accelerate the progression of age-related macular degeneration (AMD).
AMD is the leading cause of blindness in the developed world, affecting about 15 million Americans over the age of 50. It is a disorder of the central retina, or macula, which is responsible for detailed vision and controls important functional visual activities like reading, recognizing faces, and watching television.
According to the National Eye Institute over 1.7 million Americans over age 50 suffer mild to profound vision loss from advanced AMD, which culminates as end-stage AMD. Patients affected in both eyes often experience a loss of independence, social interaction, and have difficulty with activities of daily living requiring detailed vision.
The target for RTP-801 is neovascular or wet AMD. Wet AMD is the most devastating form of the disease and occurs due to the formation of an abnormal vascular network beneath the retina of the eye. These blood vessels are excessively leaky and lead to an accumulation of fluid and blood beneath and within the retina resulting in a loss of visual acuity.
“Despite advances in research and the availability of new treatment options, there remains a need for new approaches to improve the lives of patients with AMD,” Martin Mackay, Ph.D., Pfizer senior vice president worldwide research and technology said in a release announcing the deal. “We are excited about the potential of RTP-801 to preserve vision in patients with wet AMD who have an increased risk of progressive eye damage and vision loss.”
“This agreement provides further recognition for Quark’s creative approach to discover conceptually new drugs to treat devastating diseases. We are dedicated to help society with innovative medicines by moving from novel gene targets to unique compounds and eventually commercial products,” added Daniel Zurr, CEO of Quark.
Based on pre-clinical models, Quark believes that AMD can be treated by blocking the expression of the RTP-801 gene through RNA interference or RNAi, a naturally occurring mechanism within cells for selectively silencing and regulating specific genes.
Speaking to ISRAEL21c from a business trip in Japan, Zurr said that the licensing agreement worth hundreds of millions of dollars was an important milestone for the company and justified the years of work that went into the research.
“The potential drug is based on a genetic pathway that we discovered. Once we discovered the pathway and its relevance to certain diseases including AMD, it was still an uphill struggle to find the drug that suppresses the gene, but we’re very fortunate to be on the right path,” he said.
Other drugs to treat AMD are also being developed in the pharmaceutical world – including two Pfizer drugs in clinical trial – but according to Zurr, they are all based on Vascular Endothelial Growth Factor (VEGF), a natural substance secreted by the body’s cells which encourages blood vessel growth.
“We’re coming with a totally new approach – and in the animal studies we’ve done, it looks like our drug is much more efficacious than the others,” he said, explaining that Pfizer is interested in exploring all options available for an AMD drug. “I think they’re excited about the possibilities.”
Zurr himself is excited about what the deal means for Israeli biotech in general, which he thinks has gotten the raw end of the deal compared to the investment that has gone into Israeli software and high tech development.
“Developing drugs from scratch takes a long time, and is very capital intensive.
Venture capitalists like to see brief exits – which can potentially go very well in software, but in drug development it’s a different ball game.
“I think Israel is in the first league in software development or in new instruments that you can develop in a relatively short time. Unfortunately, in drug development, we haven’t made it to the top. Since the creation of the country, we’ve only produced one effective ethical drug – Copaxone [developed at the Weizmann Institute and marketed by Teva as one of the most commonly prescribed drugs in the world for the treatment of multiple sclerosis],” he said.
Zurr is confident that Quark’s licensing agreement with Pfizer will be an important building block to developing a meaningful pharma industry in Israel, and help create jobs for young graduates in the biotech field.
“There’s no reason why we can’t create a real major industry for drugs – just like Switzerland has with Novartis and Roche. Hopefully, this is a step in that direction.”
Zurr credited QBI’s ability to work patiently on its research on the gene to the financial support and faith shown by Larry Ellinson, founder and CEO of Oracle and QBI’s main investor.
“What’s nice about Larry’s investment is that he wasn’t looking for a brief exit. He actually invested a lot into the company but allowed us to develop new drugs without putting pressure on to get it quickly,” he said. “We first discovered the gene in 1998, but then you have to find the exact function and pathway to develop the drug. It’s a long and tedious process.”
Now that the initial work has been completed, QBI is not abandoning its research. Zurr confirmed that the company will be working right alongside Pfizer in collaborating on clinical trials.