Roche and Seaside Therapeutics announce landmark alliance to advance novel treatments for FXS and ASD

Roche (SIX: RO, ROG; OTCQX: RHHBY) and Seaside Therapeutics announced today that they have entered into a collaboration to develop disease modifying treatments for fragile X syndrome (FXS) and autism spectrum disorders (ASD), both neurodevelopmental disorders for which there are currently no effective pharmacological treatments that address core symptoms. The alliance aims to speed up research and development in this field and lead a fundamental change in the treatment paradigm for FXS and ASD by developing therapeutics that target the molecular basis and, in turn, core symptoms of these neurodevelopmental disorders.

Under the terms of the agreement, Seaside will license patents covering the use of mGluR5 antagonists for the treatment of neurodevelopmental disorders exclusively to Roche. Roche will subsequently lead the development and commercialization of these compounds for the treatment of FXS and ASD. Its mGluR5 drug candidate RG7090, is currently enrolling patients in a Phase 2 clinical trial in FXS.

Seaside will develop its GABA-B agonist program and retains exclusive rights to issued and pending patents covering the use of GABA-B agonists for the treatment of FXS and ASD. Seaside’s lead GABA-B candidate STX209 is currently enrolling patients in Phase 3 trials in FXS and recently completed enrollment in a Phase 2b trial in ASD. Roche may exercise options to commercialize STX209 upon completion of certain clinical development phases in FXS and ASD, but Seaside will continue to lead the clinical development of these programs. Additional terms of the transaction will not be disclosed.

“Roche is committed to finding new treatments in areas of high unmet medical need such as autism spectrum disorders,” comments Luca Santarelli, Global Head of Roche Neuroscience. “Recent discoveries in genetics have shed light on the biological underpinnings of these conditions thus providing a basis for mechanistic drug discovery. To establish a leadership position in this field we sought to build a solid partnership with Seaside Therapeutics, a company that has successfully pioneered the research and development in this novel and uncharted area.”

“This collaboration is a real win for patients and caregivers—aligning leading minds and organizations committed to rapidly advancing transformational drugs to treat autism and fragile X syndrome,” said Randy Carpenter, MD, President and Chief Executive Officer of Seaside Therapeutics. “Importantly, this collaboration also provides Seaside with additional resources to complete late-stage clinical development of STX209, which we believe has the potential to change the treatment paradigm for fragile X and autism and thereby help patients and their families achieve an improved quality of life.”

ASD refers to a group of enigmatic cognitive disorders, including autism and Asperger’s syndrome, which impair social interaction and communication, whereas FXS is a rare genetic disease, whose symptoms closely resemble ASD, and whose underlying mechanism may be similar. With no approved pharmacological therapies that address core symptoms for either condition, the unmet medical need remains high. The compounds that Roche and Seaside Therapeutics are developing hold the promise to become the best-in-class treatments by targeting aberrant glutamate signaling and GABA, thereby restoring synaptic transmission in ASD and FXS patients.

About mGluR5

The most commonly inherited form of autism involves the gene encoding fragile X mental retardation protein (FMRP). Loss of FMRP function disrupts signaling between neurons, leading to widespread brain abnormalities and mental retardation. Normally, FMRP is balanced by mGluR5, an important receptor in the brain that is involved in learning and memory. Without normal FMRP, this balance is lost, leaving mGluR5 function unopposed. Early results from a clinical trial suggest that children with fragile X syndrome can be helped by drugs that inhibit mGluR5 activity. (Source: Sfari)

Reference

1. Michalon et al., Chronic Pharmacological mGlu5 Inhibition Corrects Fragile X in Adult Mice. Neuron – Volume 74, Issue 1, 12 April 2012, Pages 49–56 (full text)