Ember Therapeutics – 2012 Fierce 15

Lou Tartaglia, interim CEO of Ember Therapeutics--image courtesy of Third Rock Ventures

Based: Boston
Founded: 2011
Interim CEO: Lou Tartaglia
Focus: Metabolic diseases/brown fat
Website: www.embertx.com

The Scoop: Obesity and diabetes are two of the biggest boogeymen of medicine, chasing after millions of overweight individuals and pushing them toward ugly health outcomes such as heart attacks and strokes. But the so-called "diabesity" tandem must beware of calorie-crunching brown fat. Brown fat fights the calorie-storing white fat people carry around their waistlines. And Ember Therapeutics jumped onto the biotech scene late last year with a $34 million Series A round from Third Rock Ventures to research drugs that harness brown fat to treat metabolic diseases.

What Makes it Fierce?

Brown fat opens an unexploited pathway for pharma to wage war on the scary obesity epidemic, and Ember Therapeutics has amassed the talent and intellectual property to be a player in this emerging area. Its scientific founders have generated some of the pioneering evidence of approaches to activate beneficial fats to the detriment of the bad kind. And Interim CEO Lou Tartaglia has recruited a staff of biotech vets to translate their science into potential drugs for metabolic diseases such as diabetes and obesity.

Scientists used to think that brown adipose tissue or fat, typically found in human infants, vanished before adulthood. Then in 2009 researchers revealed for the first time that brown fat exists in human adults, too, providing a new hope for targeting the fat that burns calories into heat. Bruce Spiegelman, a scientific founder of Ember from the Dana-Farber Cancer Institute, piloted a study published in Nature that described the ability of the natural hormone irisin in turning white fat into the brown variety in mice. And fellow Ember scientific founder C. Ronald Kahn, of the Joslin Diabetes Center, helped discover the role of BMP7 protein in promoting the development of brown fat.

Ember has licensed in assets from those seminal studies, jockeying for position in the brown fat race that involves multiple startup and pharma players. All hunger for pieces of the blockbuster market for drugs against obesity, which afflicts more than 1 in 3 U.S. adults and millions of children. This year, the FDA stamped approvals on two closely watched weight-loss treatments--Arena Pharmaceuticals' ($ARNA) Belviq and Vivus' ($VVUS) Qsymia--after the developers endured a gantlet of regulatory hoops and scrutiny of potential side effects.

Ember hatched in part from Third Rock's best bet on one of the most exciting new targets in obesity--and Tartaglia calls it a "brown fat company."

"Brown fat is an innate organ that was evolved specifically to dissipate excess caloric energy and to augment this I think is the most attractable way of generating weight-loss drugs through the mechanism of energy expenditure," says Tartaglia, who doubles as a partner at Third Rock. "And I think they will be the perfect complement to the appetite suppressants we see being approved today."

Unlike some appetite suppressants, the hormone irisin doesn't slip through the blood-brain barrier and may not cause the suicidal thoughts and other CNS side effects associated with other treatments. This is the hope at Ember, which is researching the use of irisin as a weight-loss option. At this point, the company's injections of the hormone have been tested in mice. Within two years, the startup's irisin could be studied in man after planned studies in primates, Tartaglia says. As with many young drug programs, human studies could come sooner or later.

Ember is entertaining serious thought about both biologic and small-molecule approaches and a variety of brown fat-related targets. For instance, Spiegelman and others recently found that so-called beige fat, genetically distinct from white and brown fat, offers therapeutic potential in humans. It's too early to say whether irisin or copies of the hormone would make good drugs for humans, but there's a huge incentive for giving the science a chance.

A bevy of companies is exploring brown fat for obesity. As Bloomberg reported last year, Roche/Genentech produced evidence that its antibody candidate stimulates brown fat activity and helped mice drop weight. And at least one other startup in the Boston area, Energesis Pharmaceuticals, has formed in recent years to focus on this field. Among brown fat startups, however, Ember has the advantage of deep-pocketed VC support from Third Rock and a unique lineup of biotech and scientific heavyweights on hand.

Third Rock is underwriting the entire brown-fat crusade at Ember--for now.

"Almost every large pharmaceutical company has approached us for discussions," Tartaglia says, "and it makes sense that they would because they are trying to make decisions about whether to work with us or to try to compete in this area."

At the moment, according to the CEO, his company plans to stay in touch with potential pharma partners until its programs mature to the point when one or more are ripe for a deal. "Obesity is an area [in which] a small company like Ember doing Phase III clinical trials just isn't realistic," he says. So it's more likely that the biotech, like many others, will seek a partnership before late-stage studies begin.

Investor: Third Rock Ventures.

-- Ryan McBride (Email | Twitter)

Ember Therapeutics – 2012 Fierce 15
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