Emerging Drug Developer: Transgeneron Therapeutics

Transgeneron Therapeutics is a tiny company on a big mission.

The fledgling biotech believes it has found a new approach to diabetes that has the potential to replace insulin therapy, a multibillion-dollar market.

The developer is based in Gainesville, FL, not far from the lab of Dr. Li-Jun Yang at the University of Florida. Yang, an M.D., found that injecting a recombinant transcription factor called Pdx-1 in mice models engineered to develop diabetes reversed hyperglycemia, regenerating insulin-producing cells. And now a tiny band of staffers--allied to an outsourced team of consultants and researchers--is pushing to find the funds to advance from animal studies to proof-of-concept in man.

Dr. Yang's "research has been in liver control of glucose and hormonal aspects of diabetes to change the glucose system and how it's regulated," says Leslie Molony, PhD, a veteran scientist who co-founded and now runs the company. "She was looking at the liver in terms of a potential source for cells, to reprogram cells to reverse diabetes. She's been working on how to get those cells to differentiate into pancreatic islet cells--the factors and genes required--and she's been working on that for quite some time."

"Pdx-1 does work," says Molony. "And once it works it at least looks like it has cured the diabetes in those animals as long as we've carried out the research so far. We're not saying this is a cure. It can reduce or eliminate the need for insulin. And we're not sure how long the therapy may work. If you can replace the hormonal regulation of the pancreas for a year--which is what happens in islet transplants--without giving an islet transplant, that's a huge advantage to patients. They could go about their normal lives and once a year have a treatment.

"This is not cell therapy or gene therapy," stresses Molony. ‘We're injecting a therapeutic protein that transdifferentiates cells."

Takeda Research Investments in Palo Alto, CA provided Transgeneron with $1.25 million in seed funds. But it's going to take a lot more than that to get into the clinic.

"Our goal is to get $10 million for at least the first tranche of a Series A," says Molony, which would allow researchers to obtain proof-of-concept data in man. An IND could be completed in 18 months.

Proof of concept in man could be obtained in a Phase Ib trial, she adds, "provided the FDA approves of the clinical protocol."

Those are big goals, she concedes. And raising funds hasn't been made any easier by the credit crunch. For now, the hunt for investment capital also includes every source of alternative capital imaginable, including possible partnership deals, foundation money and grants.

Transgeneron's quest has been helped by some positive attention. The company was singled out in late 2007 as SEBio's winner of the Early-Stage Shootout. Investors have been intrigued by researchers' initial success regenerating diseased organs.

And they've done it all on a shoestring budget.

"We're semi-virtual," says Molony, with three full-time workers and a development team composed of consultants. Even with additional funds, she adds, the company would remain semi-virtual, but additional resources would open the way to more product management, quality control work and validation.

But even with a small staff and a lean budget, a developer can think and act globally. Transgeneron has turned to a research protein production facility in Scotland and a clinical research group in Taiwan to conduct animal research.

"It doesn't matter where you are," says Molony, adding that it's helped that Florida has become recognized as a hotbed of research activity.

Emerging Drug Developer: Transgeneron Therapeutics

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