Date: 2016-05-03
Type of information: Licensing agreement
Compound: self-delivering RNAi (sd-rxRNA®) compounds targeting superoxide dismutase 1 (SOD1)
Company: RXi Pharmaceuticals (USA - MA) Thera Neuropharma (USA - PA)
Therapeutic area: Neurodegenerative diseases
Type agreement: licensing
Action mechanism: RNAi/antisense oligonucleotide. RXi's self-delivering RNAi (sd-rxRNA) compounds are hybrid oligonucleotide molecules that combine the beneficial properties of both conventional RNAi and antisense technologies. The sd-rxRNAs have a single-stranded phosphorothioate region, a short duplex region, and contain a variety of nuclease-stabilizing and lipophilic chemical modifications. 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 amyotrophic lateral sclerosis 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 amyotrophic lateral sclerosis symptoms.
Disease: amyotrophic lateral sclerosis (ALS)
Details: * On May 3, 2016, RXi Pharmaceuticals and Thera Neuropharma announced that they 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 Neuropharma 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). 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.
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