Two years ago, Xconomy pinpointed a growing “autism mini-cluster” in Boston—a bevy of startups seeking to develop drugs to treat the condition and other brain disorders. Now one resident of that cluster has a major partner on board to help it meet that goal: Cambridge, MA-based Seaside Therapeutics, which announced on June 19 that it had formed a licensing deal with Swiss drug giant Roche.
Under the deal, Roche purchased an option to commercialize Seaside’s lead drug, STX209, which is currently in late-stage trials to treat fragile X syndrome, a genetic disease with symptoms similar to what’s seen in autism. Those include cognitive delays, attention deficit disorder, and antisocial behaviors. STX209 is also in earlier trials to treat autism spectrum disorders. Seaside will continue to develop the drug, and if it meets certain milestones, Roche could exercise the option, providing critical infrastructure necessary to market the drug in the U.S. and overseas, says Randy Carpenter, CEO of Seaside.
In addition, Roche has licensed Seaside’s intellectual property around a class of drugs called mGluR5 antagonists, which target a pathway in the brain that’s believed to play a major role in fragile X. Roche, which had been working on its own mGluR5 drugs, will lead both the R&D and commercialization of compounds in this class that the two companies are examining for developmental disorders.
Seaside did not reveal the financial details of the deal but, says Carpenter, the money isn’t what’s most important to his company. Roche and Seaside had actually been working together informally over the last decade, trying to define the molecular causes of developmental brain disorders and discover potential drug targets, he says. “We’ve shown a lot of what can be shared at the edges of open innovation,” Carpenter says. “Now we can share confidential information, like regulatory documents and relationships with patient advocacy groups.”
The Roche support comes at a crucial juncture for STX209. Seaside is currently completing enrollment for two pivotal late-stage trials of the drug in fragile X, both of which should be completed next year, Carpenter says. The company is primarily looking to prove that its drug improves aberrant behaviors, such as extreme social anxiety that often causes children with the disease to spend hours alone in their rooms instead of interacting with friends and family.
STX209 stimulates receptors in the brain called GABA-B, which play a key role in modulating excitatory and inhibitory signals. “In both fragile X and autism, there’s too much excitation and not enough inhibition,” Carpenter explains. “We can get that back into balance.”
Results from early trials have been promising, Carpenter says. “The children on the drug are less likely to spend all their time in solitary play,” he says. “They’re now interacting much more with their siblings, parents, and friends. For parents, this is really important, because that’s the main thing they worry about. Does my child have friends? Are they willing to play with their siblings? Do they like me? We’re getting really encouraging reports.”
The second class of drugs that Seaside is working on with Roche—mGluR5 antagonists—also target brain signaling, but in a different way. Patients with fragile X have a genetic abnormality that disrupts a particular protein in the brain involved in learning and memory. Inhibiting mGluR5 seems to restore the proper balance of that protein. “It allows the brain to determine more effectively what’s important information and what’s noise,” Carpenter says. “That allows it to learn better from experience.”
Seaside has an unusual list of investors that reside outside of the venture capital world, and therefore are more interested in finding cures for brain disorders than they are in making money, Carpenter says. Since the company was founded in 2005, it has raised more than $66 million from a private family investment fund and organizations such as the Fragile X Research Foundation, Autism Speaks, and the National Institutes of Health. “Because our investors have been focused on the societal returns and not the financial returns, it has allowed us to be more openly collaborative than is possible in a venture-backed or public company,” Carpenter says.
Roche and Seaside have crossed paths at several venues, including the Banbury Meeting, an annual gathering at Cold Spring Harbor Laboratory in New York, where researchers trade notes on fragile X research. And earlier this year, Carpenter spoke at a symposium on autism hosted by Roche.
Carpenter says Seaside will benefit from Roche’s worldwide leadership in neurology research. Roche is one of seven major pharmaceutical companies leading a $38.7 million initiative to bring academic scientists and industry together for autism research, for example. It is also leading the effort to search for biomarkers—molecular signatures that may contribute to developmental disorders and serve as targets for new drugs. “We’re comfortable we share the same mission and have the same passion,” Carpenter says. “I think they’re an ideal partner for us.”