Zafgen - 2009 Fierce 15

FierceBiotech 2009 Fierce 15 winner

Based: Cambridge, MA
Founded: 2005
CEO: Tom E. Hughes (photo)

The Scoop: Zafgen is as far from the me-too drug trail that a developer can get. It's taking a radically novel approach to fighting fat and hopes to blaze a trail to a new class of drugs that will combat an epidemic of obesity. It faces a daunting series of challenges at every step through the clinical trials and on to regulatory review. But if they're right, Zafgen is on to the next big thing in obesity.

What makes it Fierce: Zafgen has a whole new approach to fighting obesity. In animal studies, the approach burned off body weight at the rate of 25 percent. "Once you treat animals, you see a retraction in the size of fat tissue," says CEO Tom Hughes. Fuel is burned off, appetite goes down, fat is released and the metabolism is stimulated. Dangerous liver fat dwindles and there's an improvement in insulin activity.

Most experimental drugs for obesity are targeted at centers of the brain. But Zafgen is going right for the fat tissue. Hughes, though, isn't providing a lot of details at this stage about exactly what the therapy does.

"We have a pretty good idea, but we're not sharing it," says the CEO. "There's a unifying theory of why all this is working. I've never seen anything that puts all the pieces together quite like this does.

"We will be starting our first clinical trial later this year," says Hughes, on a drug that was originally tested in cancer but never panned out as an oncology product. "We're taking that and repurposing it for obesity. What's cool about this is that not every anti-angiogenetic mechanism has an equivalent effect on fat tissue. We went through and picked the best approaches and we're working with the best of them."

Because Zafgen already has safety data on the lead program, researchers can go directly to a Phase Ib proof-of-concept test on patients. And if the therapeutic approach works in humans like it has in animal models, with something in the range of a 25 percent loss in body weight, then Zafgen has an opportunity to advance a therapeutic alternative to bariatric surgery.

"We should have data by the spring of next year, in the tens, not hundreds of patients," says the CEO. "It's our intention to partner this program out. We hope to have a deal in time by the top data." And there's more work being done to identify other therapies that can work even better.

And Hughes has overseen plenty of other programs. He comes straight from the labs of big pharma, having held a senior-level research position with Novartis before he was wooed away by the venture crowd backing the start-up biotech.

Zafgen raised $14 million in a Series B late last year and has brought in a total of about $22 million so far.

What to look for: It's all up ahead for Zafgen, but the developer has a leg up in its quest to gain proof-of-concept data. Hard data demonstrating that Zafgen is on the right track would be very valuable.

Venture backers: Atlas Ventures--which incubated the company--and Third Rock Ventures.

Zafgen - 2009 Fierce 15

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