DELSIA, Seaside Therapeutics announce biomarker partnership

NEW YORK—Autism Speaks has announced the grant of a $2million award to Delivering Scientific Innovation for Autism (DELSIA), itsnot-for-profit affiliate, for the establishment of a partnership between DELSIAand Seaside Therapeutics, a Cambridge, Mass.-based company working toward thedevelopment of therapeutics for the treatment of autism, fragile X syndrome andother neurodevelopmental disorders. The two organizations will work toward thediscovery of biomarkers for the development of safe, effective and personalizedtreatments for autism spectrum disorder.

Under the agreement, DELSIA will provide SeasideTherapeutics with $2 million in funding to support Seaside's autism biomarkerdiscovery program, which seeks to identify gene and protein biomarkers capableof predicting patients' response to autism treatments.

"Autism Speaks is extremely pleased to be funding DELSIA'sfirst award of $2 million to Seaside Therapeutics, a company that has shown along-standing commitment to developing treatments for neurodevelopmentaldisorders, including autism spectrum disorder," Geri Dawson, Ph.D., chair ofDELSIA's board of managers and chief science officer at Autism Speaks, said ina press release.

DELSIA's partnership with SeasideTherapeutics represents the first award from the newly established venturephilanthropy affiliate of Autism Speaks. The initial goal of the partnership isto identify patients that are likely to benefit from being treated with STX-209(arbaclofen), Seaside Therapeutics' lead compound, and identify biomarkers thatcould indicate an increased risk for side effects. STX-209 is a GABA-B receptoragonist, currently in Phase III development for fragile X syndrome and Phase IIdevelopment for autism, two conditions that present with abnormalities insynaptic transmission. By acting through the GABA-B receptor, the compound "mayserve to restore the normal balance at the synapse."

"We are enormously excited to be partnering withSeaside, a company at the forefront of autism medicines development," RobertRing, Ph.D., president of DELSIA and vice president of translational researchfor Autism Speaks, said in a statement. "Our partnership will help generatecritical information that can be used to pinpoint which individuals with autismare most likely to respond to arbaclofen, one of the first treatments that hasthe potential to address the core social impairment associated with autism.This knowledge will have broader implications down the road for developingeffective treatments for people with autism."

Previous studies have demonstrated STX-209 to have promisein improving social and communication functions in those with autism andfragile X syndrome.

Ring called the program "an investment towards personalizedmedicine in autism." STX-209 represents the most advanced program in the autismclinical pipeline today, and if successful, could provide the first therapy forone of autism's core symptoms.

"This represents an exciting new avenue for moving autismresearch out of the laboratory and into the real world," said Ring in apress release. "By accelerating the development of effective treatments,we are following in the footsteps of successful endeavors by organizations suchas the Cystic Fibrosis Foundation."

SOURCE:Autism Speaks press release