A fix for malfunctioning mitochondria

Farmingdale's MitoGenetics team.
By GREGORY ZELLER //

A tri-state connection may soon unravel the mystery of mitochondrial disease – a potential breakthrough for patients with type 2 diabetes, Alzheimer’s disease and a host of other life-threatening conditions.

Headquartered in South Dakota, MitoGenetics LLC – brainchild of former Iowa State University researcher William Switzer – is knee-deep in lab work at Broad Hollow Bioscience Park in Farmingdale, where it enjoys the benefits of Empire State Development’s Start-Up NY tax-incentives program.

The company’s Broad Hollow research, according to CEO Marcia Hendrickson, could soon rewrite medical texts regarding malfunctioning mitochondria, tiny bacteria-based structures that share a symbiotic relationship with human cells.

Marcia

MitoGenetics CEO Marcia Hendrickson

Quick biology lesson: Thousands of mitochondria live in every cell, each containing over 16,000 base pairs, the building blocks of the DNA double helix. Those base pairs form genes and proteins primarily responsible for turning glucose into adenosine triphosphate, the so-called “molecular unit of currency” that essentially fuels cellular reactions.

When the mitochondria malfunction, the cells mutate, leading to any number of potentially serious disorders. As a large energy consumer, the human brain is especially susceptible to mitochondrial disease, leading to a high association with Alzheimer’s, Parkinson’s disease and other neurodegenerative conditions. The mitochondrial focus on glucose also leads to a high percentage of diabetes diagnoses.

Other mitochondrial-related conditions include heart, liver and kidney diseases; gastrointestinal and respiratory disorders; and a host of maladies with symptoms ranging from muscle failure to sight and hearing problems.

Switzer launched MitoGenetics in 2010 specifically to develop pharmaceutical therapies for misbehaving mitochondria. In his mid-80s at the time, Switzer was friends with SUNY College at Old Westbury neuroscience professor George Stefano, who agreed to take the mitochondrial baton and run with it.

Through a sponsored research agreement, Stefano – now a MitoGenetics vice president – led years of research at Old Westbury. By 2013, the work seemed promising enough to start taking the business side a little more seriously. Enter Hendrickson, a microbiologist, CPA and self-described “corporate clean-up person.”

Hendrickson had spent nine years as the founding director of the Enterprise Institute, a South Dakota-based business accelerator that helps researchers commercialize technology by focusing on funding, IP and strategic planning.

As a veteran “restructurer of companies,” as she put it, the scientist/accountant knew immediately that MitoGenetics had the goods.

“I could tell the Old Westbury team was brilliant, and I could see where the science was heading,” Hendrickson told Innovate LI. “If their theories were right and their early science was right, they could do astounding things for health.

“The mitochondria are really the drivers of our health,” Hendrickson added. “If they could actually do something to keep the mitochondria healthy and functioning properly, it would be a real blockbuster.”

To accommodate the new CEO, MitoGenetics’ headquarters was “moved” to South Dakota. The meat of the business, however, remains on Long Island, and in March shifted to Broad Hollow, where 11 researchers led by Stefano are now “refining” a therapeutic compound that should be ready for human trials in 2016.

With a patent pending, the company has already scored with investors. So far, it’s raised about $6 million in venture capital, including buy-ins from South Dakota-based angels familiar to the CEO and multiple individual Long Island investors. It’s now embarking on a $2 million funding round that will cover the early human trials and the toxicity studies that precede them.

“It will pretty much be spent in New York,” Hendrickson said of the $2 million, which MitoGenetics would like to raise by March. “It will all be spent on the science.”

The Start-Up NY designation, Hendrickson noted, has been a big help for a company whose M.O. from the start has been spending on the science.

“It effectively helps us get more cash into the hands of scientists, because we don’t have to pay those taxes,” she said.

Ever more importantly, the Start-Up NY designation helps legitimize Switzer’s startup – a critical differentiator, Hendrickson added, for a company that’s been completely investor-funded to date, sans private or government grants.

“It shows the world that the State of New York is investing in MitoGenetics,” she said. “It gives us much more credibility.”

Hendrickson said she would be happy to pitch the Long Island Angel Network – “if they’re interested” – and any other potential investor eager to learn more about the company’s research and business plan. Whoever does or doesn’t get on board, however, Hendrickson is confident that her company will raise that $2 million and begin human testing in the next several months, starting first with type 2 diabetes patients.

How long those trials will last and when researchers may move on to other mitochondrial disease-related disorders “are among the questions we’re figuring out now,” Hendrickson said, though the CEO is already predicting a fully commercialized MitoGenetics therapeutic remedy by 2020.

“That’s really not that long, in pharmaceutical-company terms,” she noted. “We’re pretty far down the science road already, and we’ve already got pharmaceutical companies talking to us.

“We understand this is still a high-risk investment, which is why we’re using a lot of smaller angel investments,” Hendrickson added. “We don’t expect somebody to put $500,000 into the company at this point. But whoever does invest … is buying into what will be some of the biggest blockbuster drugs ever to come out of Long Island.”