BASEL, Switzerland—Roche and Seaside Therapeutics haveannounced a collaboration for the development of disease-modifying treatmentsfor fragile X syndrome and autism spectrum disorders. The alliance is intendedto accelerate research and development in the field of these neurodevelopmentaldisorders and hopefully change the treatment paradigm for the diseases byproducing therapeutics that target the molecular basis of fragile X syndromeand autism spectrum disorder.
Per the terms of the agreement, Seaside will license patentscovering the use of mGluR5 antagonists for the treatment of neurodevelopmentaldisorders exclusively to Roche, who will be responsible for leading thedevelopment and commercialization of the compounds for fragile X syndrome and autismspectrum disorders. Patients are currently being enrolled for a Phase IIclinical trial of RG7090, Seaside's mGluR5 drug candidate, in fragile Xsyndrome.
"This collaboration is a real win for patients andcaregivers—aligning leading minds and organizations committed to rapidlyadvancing transformational drugs to treat autism and fragile X syndrome," RandyCarpenter, M.D., president and CEO of Seaside Therapeutics, said in a pressrelease. "Importantly, this collaboration also provides Seaside with additionalresources to complete late-stage clinical development of STX209, which webelieve has the potential to change the treatment paradigm for fragile X andautism and thereby help patients and their families achieve an improved qualityof life."
Seaside will develop its GABA-B agonist program, retainingexclusive rights to issued and pending patnets covering the use of GABA-Bagonists for the treatment of fragile X syndrome and autism spectrum disorders.Roche may exercise options to commercialize STX209, Seaside's lead GABA-Bcandidate, once specific clinical development phases in both indications arecomplete, but Seaside will continue to lead the clinical development of theprograms. Additional terms and financial details were not disclosed.
GABA-B was first identified by Seaside as a drug target intreating fragile X syndrome and autism, as recent studies have shown thatfragile X syndrome and autism both exhibit deficient inhibitoryneurotransmission. STX209, a GABA-B agonist and the company's lead GABA-Bcandidate, "has the potential to normalize this deficiency," Seaside notes onits website. In addition, modulating the GABA-B pathway has the potential toindirectly reduce activation of the mGluR5 pathway, and mGluR5 negativeallosteric modulators serve to normalize the excessive protein synthesis thatcan lead to symptoms associated with fragile X syndrome.
"Roche is committed to finding new treatments in areas ofhigh unmet medical need such as autism spectrum disorders," Luca Santarelli,global head of Roche Neuroscience, said in a press release. "Recent discoveriesin genetics have shed light on the biological underpinnings of these conditionsthus providing a basis for mechanistic drug discovery. To establish aleadership position in this field we sought to build a solid partnership withSeaside Therapeutics, a company that has successfully pioneered the researchand development in this novel and uncharted area."
Autism spectrum disorders encompass a group of cognitivedisorders, including autism and Asperger's syndrome, that lead to impairedsocial interaction and communication. Fragile X syndrome is a rare geneticdisease with similar symptoms, and no therapies exist to address the coresymptoms for either condition.