A pharmaceutical company looking to develop a new drug typically needs to earmark several billion dollars and 10 to 12 years of research and development before it can see any return on investment.
But nine out of 10 times, the treatment doesn't make it to market, Novoheart Holdings Inc. CEO Ronald Li told S&P Global Market Intelligence.
"Can we potentially flip the ratio around? Instead of failing 95% of the time, can we accomplish a more respectable success rate? I think that's the most important thing we want to achieve," he said.
Heart tissue grown with stem cells.
The Toronto-listed stem cell company has been developing a technology to create miniature hearts that allow drugmakers to test their potential candidates early on in the development process, potentially shaving off years of R&D cost on compounds which may not have worked.
This area of research is not only emerging but booming, Li said.
"For investors, this is an emerging area. It's growing very rapidly because in general, technology in three-dimensional mini-organs is happening in a big way," he said. "What people are most excited about is the ability to custom-tailor [treatments] for individuals."
Taking a few drops of blood from a patient, Novoheart's technology platform — called MyHeart — can grow jars of mini hearts based on the genetic material in the blood.
Since the mini-hearts are genetically and immunologically identical to the patient's cells, they are essentially the patient's cloned parts that can be used to test whether a drug is safe for the patient, he said.
With a more accurate screening method, the company hopes its platform can help drug companies find promising drug compounds, while filtering out the ones that do not work or are unsafe.
A drug can fail anywhere throughout the drug development process, even after it has been marketed, the CEO said, citing Merck & Co. Inc.'s Vioxx as an example.
The pain relief drug was approved by the U.S. Food and Drug Administration in 1999, but was pulled from the market in 2004 after studies showed it increased the risk of heart problems.
Bridging the gap in drug screening
One of the main reasons why most drug compounds fail to work is because half of them are toxic to the heart and another 30% to 40% are toxic to the liver, the CEO said.
"That is the reality. That's why there are very few blockbuster [drugs] these days, because pharmaceutical companies have simply run out of candidates in the pipeline," Li said.
The current system of using animal models to test early drug compounds is also flawed, he added.
"Drug companies and scientists have been depending on animals — mice, rats — to tell them what candidates potentially work in humans," he said. "Once you prove your concept in animals, you take that leap of faith and test in humans, and there's absolutely nothing in between."
"We are trying to use our technology to bridge the gap between animal models and human trials," Li said.
Market growth and direction
With the MyHeart platform gaining attention, interest in Novoheart's technology has been growing, Li said.
In January, Novoheart partnered with a global pharmaceutical company to develop mini hearts that carry a mutation that causes a neurological disorder. About two-thirds of the patients die mainly due to heart problems associated with this disorder, the company said.
On Feb. 14, Novoheart signed a deal with privately held biotech company Sumocor LLC to develop new therapies for heart failure.
In reporting its latest financial results on Feb. 26, the company said it was awarded C$150,000 from the Innovation and Technology Commission of Hong Kong to support its expansion of R&D operations and facility, and another C$1.8 million to develop its MyHeart platform.
"In the not-too-distant future, stem cell mini-organs technology is going to allow us and pharmaceutical companies to discover new drugs," the CEO said. "Everything is coming together but obviously it takes time for the market to absorb and adapt and this is what we think is happening right now."