RXi Pharmaceuticals Corporation (RXi) and privately-held Thera Neuropharma, Inc. (Thera) have entered into an exclusive license agreement for RXi's novel and proprietary sd-rxRNA platform to develop therapeutics for neurodegenerative diseases.
Under the terms of the agreement, Thera will be responsible for all research, development, manufacturing, regulatory and commercialization activities for the licensed products. The initial focus of the agreement will be on sd-rxRNA compounds targeting superoxide dismutase 1 (SOD1) for use in developing innovative treatments for amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease. Furthermore, there are potential synergies between RXi's SOD1 sd-rxRNA compounds and Thera's small molecule regenerative therapeutics (SMRT), to target multiple factors involved in the pathogenesis of ALS. During the past several months, the companies have jointly filed provisional patents on the mechanism of action of the combined compounds for the management of neurodegenerative diseases associated with SOD1.
RXi's novel and proprietary self-delivering RNAi (sd-rxRNA) compounds are designed with unique features that result in robust cellular uptake and target-specific mRNA reduction. RXI's most advanced sd-rxRNA compound, RXI-109, is currently in phase 2 human clinical trials for dermal scarring and phase 1/2 human clinical trials for subretinal fibrosis associated with late stage age-related macular degeneration.
RXi's SOD1-targeting sd-rxRNA compounds share similar cellular uptake features with RXI-109 and have been found to successfully enter the cells of the spinal cord and brain when administered by intrathecal injection in rodent. SOD1 protein misfolding is one of the key contributing factors associated with ALS progression and reduction of SOD1 levels by sd-rxRNA treatment could provide therapeutic benefits. Thera Neuropharma's broad-spectrum neuroprotective agents are small molecule compounds shown to be well tolerated in vivo and designed to up-regulate critical cell functioning pathways. Thera's clinical candidates have a direct effect on protecting motor neurons against protein misfolding and oxidative toxicity. Their neuroprotective and regenerative effects are achieved by inducing axonal regeneration, thus restoring the viability of the neuronal network and activating modulatory/compensatory mechanisms that delay progression of ALS symptoms.
