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How MitoQ's developers navigated the pitfalls of biotech business

MitoQ CEO, Greg Macpherson.

After a tough start in clinical trials, excitement around the potential of University of Otago-developed compound MitoQ is once again strong.

In the age of the internet start-up the new mantra of business has become “fail fast” and “pivot”. Speed and agility are the essence of success.

But in biotech, different rules apply. Years of painstaking research, clinical trials and regulatory gymnastics precede any commercial launch. New products can fail at any of these hurdles.

For New Zealand developed anti-oxidant compound MitoQ, disappointment came early after the initial excitement of discovery when, over a decade ago, a trial on Parkinson’s disease patients found no discernible benefit.

The results were devastating, MitoQ CEO Greg Macpherson told Noted. It cost a lot of money and stopped the company in its tracks. Some good results from trials on hepatitis C were not enough to reclaim the momentum.

What didn’t slow down was international research on mitochondria – and researchers asking for MitoQ to investigate the link between cell dysfunction and various illnesses. Over the next five to seven years, links between disease and mitochondria were established and MitoQ was shown to have positive effects.

Clinical excitement around MitoQ has grown again, with a series of new trial results expected in coming months. Macpherson won’t detail those yet, but he clearly can’t wait for publication.

MitoQ was the brainchild of Otago University researchers Robin Smith and visiting Irish biochemist Mike Murphy.

The pair focused their research on mitochondria, the engine room of the cell, because there was a feeling that in certain medical conditions the mitochondria was damaged. The researchers wanted to find out what came first, the damage or the disease. Which caused which?

What they found was that, like a badly tuned engine, mitochondria failed to produce a natural antioxidant, coenzyme Q10, and overproduced harmful pollutants, free radicals, that cause oxidation damage to healthy cells.

The pair wondered what would happen if the antioxidant was introduced into the mitochondria. It had never been done before and required some smarts in the form of introducing a positive charge to coenzyme Q10 to allow it to penetrate.

Otago University patented the technique and soon business people came knocking. Ken Taylor, formerly of multinational Roche, and local investor Gary Lane raised funds, bought the patent and set MitoQ off on a long journey to commercial success.

The early clinical result may have been negative, but one thing was learned – MitoQ was safe. It was, after all, a natural compound and had been administered to a lot of people for a long time with no negative effects.

Because almost all our cells have mitochondria, the possible applications of MitoQ were also very broad, well beyond the initial Parkinson’s focus.

Over time, interesting benefits started to be found across a range of conditions affecting the lungs, liver, heart and kidneys.

By 2010 it was becoming clear that the initial failure was not the end of the road.

“We actually had something quite unique and quite significant,” Macpherson said.

So what’s the downside?

There isn’t any, said Macpherson, but he is at pains to say MitoQ is not a cure. Sold as a supplement, it supports the cells doing their job.

“On my first day at pharmacy school they talked about the perfect drug. That was one that went straight to the target, didn’t interfere with anything on the way through and was excreted unchanged.”

That’s a pretty good description of MitoQ, he said, though it is now being sold as a supplement rather than a drug.

MitoQ is available in a range of capsules for general purpose use and to target specific functions. It is also available as a skincare cream.

For Macpherson right now, the mission is to accelerate MitoQ’s global sales. While new patents have been registered to protect MitoQ, the original one is set to expire soon. The best defence in that scenario to be the incumbent, with a powerful brand and strong sales.

“We’re going through the stone age of medicine right now. What’s happening is amazing and what’s coming is going to knock your socks off,” Macpherson said.

The next age of medicine, powered by ability such as gene editing, is dawning.

So what causes the mitochondrial dysfunction in the first place? Well, Macpheron said, aging is part of it but so is our modern lifestyle and diet. Reducing sugar consumption and increasing exercise will help, but aging will have its impact in the end.

That’s when MitoQ comes in, tuning up the cellular engine to help it do its job again. Maybe it can even setting the clock of aging back a little.

In March, MitoQ was selected for study by the US National Institute of Aging’s government funded Interventions Testing Programme, the results of which should be released next year.