Boston-based Third Rock Ventures has committed a $34 million chunk of its funds to a biotech upstart that promises to take some new insights into brown fat and insulin sensitivity and turn them into new treatments for diabetes and obesity. Initially helmed by Third Rock partner Lou Tartaglia, Ph.D., who pieced the deal together, Ember Therapeutics will get started with a small virtual crew challenged to point the newly launched developer toward the clinic with enough cash to see if their theories can deliver some solid proof-of-concept data.
In many ways a classic Third Rock startup, Ember is being built on a scientific foundation provided by a trio of top scientists: Bruce Spiegelman, Ph.D., from the Dana Farber Cancer Institute, Patrick Griffin, Ph.D., chairman of the department of molecular therapeutics at The Scripps Research Institute in Florida and C. Ronald Kahn, M.D., professor of medicine, Joslin Diabetes Center and Harvard Medical School.
"We're well financed to move things along for a number of years," Tartaglia tells FierceBiotech, "moving into the clinic and looking to partner with pharma for later-stage development." Tartaglia adds that he's already hired a pair of key employees as Ember builds up to a virtual staff of six.
As FierceBiotech reported last October, when Third Rock was pondering whether to go ahead with a big first round, Spiegelman and Griffins garnered significant attention in scientific circles last fall when they unveiled new research they believe pinpoint the reasons why Avandia and Actos sometimes cause weight gain, the erosion of bone density and water retention among diabetics. While the drugs activate PPAR-gamma, making cells more sensitive to insulin and improving blood sugar, the scientists confirmed just weeks ago that when PPAR-gamma undergoes a process called phosphorylation by the kinase Cdk5, it disrupts various genes. Their new compound, SR1664, tested in mice, provided preclinical confirmation of the science behind a new drug program that blocks the Cdk5 enzyme, eliminating the side effects.
Obesity and diabetes are both rampant in the United States and indeed around the world, but recent attempts to gain approvals for new weight drugs have experienced some stiff headwinds at the FDA, where marginal weight loss data and prospective safety issues have kept regulators leery of new treatments intended for long-term use in a mass audience. But Tartaglia believes that the brown fat approach is "completely different from classic appetite suppressants."
By avoiding pathways in the central nervous system, Tartaglia believes that Ember has the potential to avoid many of the safety issues associated with obesity drugs by focusing on a natural mechanism for burning fat.
"Having said that," he adds, "I hope that appetite suppressants are approved and wish them well if they turn out safe." They would, after all, make great additions to a potential combination therapy with Ember's treatments. "We're certainly looking for game-changing weight loss, greater than 10%."
- here's the press release