Sage Therapeutics Shifts Gears, Focuses on Rare Form of Epilepsy

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financing beyond the $35 million Series A Third Rock provided when it started the company 15 months ago.

The plan begins with Sage’s most advanced program behind the SE compound. The drug is a treatment for traumatic brain injury, which can result from a severe blow to the head – typically in car accidents, sports collisions or combat situations. Taking a cue from animal models that show that a pregnant animal is protected from brain trauma because of elevated levels of certain neuroactive steroids designed to protect the fetus, Sage has crafted a drug to mimic that protective mechanism. The drug will enter mid-stage trials this year, Starr says.

Sage is also producing an anesthetic agent designed to get rid of the liabilities associated with propofol (Diprivan)—best known as the drug that caused Michael Jackson’s death. As little as double the usual dose of propofol can immediately shut down a patient’s lungs and heart. Sage’s drug dials in a specific plane of anesthesia – either light, mid or general – and keeps a patient there regardless of dose level. That program will be in the clinic in 2014, according to Starr.

The last program targets Fragile X syndrome, a form of autism in which children are born with the FMR-1 gene—a protein essential for cognitive development – “knocked out,” or unexpressed. There are no approved treatments for Fragile X Syndrome, though Sage hopes to change that. Starr says that Sage’s GABA modulator has shown preclinical evidence of changing the structure of the malformed synapses present in Fragile X Syndrome patients.

Behind those programs, Sage wants to use its NMDA modulators in more broad applications such as postpartum depression, anxiety, post-traumatic stress disorder, and schizophrenia. If everything goes according to plan, Sage would sign a lucrative deal with Big Pharma to tap into that discovery program and find a commercial partner for its anesthesia drug candidate while the company pushes its lead drugs through development.

Starr estimates that it will take between $70 million to $75 million to get the evidence it needs to file an FDA application to start marketing the SE drug, to prove its concept for anesthesia, to get some preliminary data in Fragile X syndrome, and to advance its discovery platform. So far, Sage has used about half of its $35 million Series A round and received $18 million in grants for its Fragile X and traumatic brain injury programs, but that still means it needs to come up with some more money.

“Our goal would be to see where we get to in business development discussions on a big early discovery alliance or on an anesthesia type alliance. If we can do that in the next year, then there’s a chance we don’t need to do any more funding,” Starr says. “But if the clinical data continues to roll and we’re making progress, we may be excited at Third Rock or bring in another syndicate partner to do a B round and keep the ownership up and develop these ourselves.”

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