Taking the fight to cancer’s stem cells

By GREGORY ZELLER //

Many have said them, tried their best and fallen short – but the words still have unique and powerful meaning, in medical parlance and beyond.

A cure for cancer.

James Egan doesn’t say that, specifically. But the “former mad scientist” – he does say that – and his team at Targagenix Inc., a 2013 startup residing inside Stony Brook University’s Long Island High-Technology Incubator, are developing a drug that’s unique in the fight against cancer. Not because it kills tumors, which other pharmaceuticals can also do, but for its ability to target and neutralize highly resistant cancer stem cells, the tiny buggers that tend to revive the disease.

And yes, Egan does use the word “cure.”

“We’ve done extensive animal testing,” he told Innovate LI. “And we’ve been able to cure many difficult and aggressive cancer indications in murine, or mouse, models.”

Meet DHA-SBT-1214 – they’re working on a stage name – a revolutionary drug that’s been 20 years in the making already. Egan, a former vice president at New York City oncology company IRX Therapeutics, incorporated Targagenix in Delaware specifically to commercialize the work of Iwao Ojima, an SBU distinguished professor of chemistry and director of the university’s Institute of Chemical Biology & Drug Development.

ojima

Iwao Ojima

Ojima, who also heads SBU’s chapter of the National Academy of Inventors, discovered the drug while screening “multi-drug-resistant tumor cell lines,” as Egan put it, and has spent the better part of two decades researching its effectiveness against both tumors and those stubborn stem cells.

The highly regarded researcher, whose findings have been published in numerous peer-reviewed scientific journals, now serves as Targagenix’s senior scientific advisor, alongside Mansoor Amiji, a distinguished professor of pharmacy at Boston’s Northeast University.

Egan, who earned an MBA and a PhD in molecular pharmacology at SBU, came on the scene when the university’s Center for Biotechnology asked him to review Ojima’s technology – specifically, to assess its commercial potential.

At IRX Therapeutics, a clinical-stage bioresearch company, Egan’s job was to raise investments and form strategic partnerships, including academic and industry collaborations. Those experiences convinced him, beyond any doubt, that Ojima had the goods.

“I’ve never seen data so compelling,” Egan recalled. “‘I thought, ‘This is amazing technology and I’m going to run with it.’”

What impressed him first about the platform technology, the scientist noted, was its effectiveness against multiple cancer indications – including pancreatic, breast, prostate, lung and a host of other common cancers.

But what impressed him most, “what’s really exciting,” he noted, is DHA-SBT-1214’s ability to destroy both cancerous tumors and those tenacious stem cells.

“There are plenty of drugs that kill tumors, but not many that are effective against both populations,” he said. “Cancer stem cells are highly drug-resistant, and they’re responsible for metastasizing after frontline treatment.

“They’re the cells that repopulate the tumors after you first treat them,” Egan added. “And that’s what makes cancer treatment so difficult.”

A personal $20,000 investment later – covering the Targagenix incorporation and related legal fees – the founder and CEO is now anticipating an FDA Investigational New Drug designation. He expects the designation, a key step toward initiating human trials, will come in 2017, and his research team is already writing up the trial’s clinical protocols.

Egan, meanwhile, is also considering a number of potential trial sites, “depending on economics” and other factors.

Economics, naturally, weigh heavily on long-term research projects like this – Egan estimates another seven to 10 years before a commercially viable project is announced – and in that regard, Targagenix has already done well. The startup has landed over $2.4 million in grants from the National Cancer Institute and matching funds from SBU’s Sensor CAT, the Center for Advanced Technology in Diagnostic Tools and Sensor Systems.

Now it’s going pro: This month, Targagenix kicked off an aggressive fundraising campaign, with Egan traveling the country to meet personally with high-net-worth individuals, investor groups and pharmaceutical companies. His goal: $8 million, raised in two distinct tranches.

“The first $2 million gets you to the Investigational New Drug designation and lets you start the human trials,” Egan noted. “Another $6 million will get us through the proof-of-concept trial in humans.”

While it could be a decade or longer before doctors are prescribing a Targagenix miracle drug, the CEO – who’s scheduled to meet in the coming weeks with various Long Island-based investors – suggested “significant value” for investors over a shorter term.

“That could mean an IPO or there could be a licensing deal,” he noted.

For now, according to Egan, the focus is on raising that first tranche and earning the “regulatory accelerants” that will move Ojima’s work from the lab to the clinic. Targagenix plans to launch a website in June explaining DHA-SBT-1214; from there, its potential – both economically and as a fabled cancer cure – is “extremely exciting,” the CEO noted.

“After years of Dr. Ojima’s research, this really needed to move forward,” he said. “It was an opportunity I couldn’t resist, for the reasons we all get into this field, to try to help patients.

“And it could be a multi-blockbuster,” Egan added. “A blockbuster is generally thought of as a billion a year in revenue. Well, this could be a $10-billion-a-year drug.”

Targagenix Inc.

What’s It? Oncology startup developing a revolutionary pharmaceutical cancer treatment

Brought To You By: “Mad scientist” James Egan and the quite sane, long-honored SBU researcher Iwao Ojima

All In: $20,000, personally invested by Egan, to incorporate and cover other legal fees

Status: Cancer cured – really – in mice; anticipating human trials in late 2017


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