RXi Pharmaceuticals Corporation a biotechnology company focused on discovering, developing and commercializing innovative therapies based on its proprietary, next-generation RNAi platform, has initiated clinical study with an RNAi compound, RXI-109, using their proprietary self-delivering RNAi technology. RXI-109 has been shown to effectively silence a key regulator of scarring, connective tissue growth factor (CTGF), in vitro in cell culture and in vivo in rodent skin models.
After completion of the necessary toxicological studies and clearance of their IND submission by the US Food and Drug Administration (FDA), they have now successfully dosed their first subject in a dose escalation study.
“This is an exciting time for RXi Pharmaceuticals, and for RNAi technology in general,” said Pamela Pavco, Chief Development Officer for the company. “After an intense period of preparation, we are happy to announce the achievement of this significant milestone by RXi.” She added that “Thanks to the potential of our proprietary self-delivering technology, we believe that RXI-109 will provide a highly selective and efficacious solution to an important unmet medical need. RXI-109 is designed to reduce or prevent skin scarring following trauma or surgery and is intended to reduce disfiguring hypertrophic scarring and keloids.” RXi’s phase I clinical trial is a dose escalation study designed to evaluate safety and tolerability of RXI-109 in humans and may provide preliminary evidence of the reduction of surgical scars.
RXi Pharmaceuticals first clinical programme centres around RXI-109, a self-delivering RNAi compound (sd-rxRNA) developed by RXi for the reduction of dermal scarring in planned surgeries. RXI-109 is designed to reduce the expression of CTGF (connective tissue growth factor), a critical regulator of several biological pathways involved in fibrosis, including scar formation in he skin. The first clinical trial of RXI-109, initiated in June 2012, will evaluate the safety and tolerability of several dose levels of RXI-109 in humans and may provide preliminary evidence of surgical scar reduction. As there are currently no FDA-approved drugs to prevent scar formation, a therapeutic of this type could have great benefit for trauma and surgical patients (especially relating to raised or hypertrophic scarring), as a treatment during the surgical revision of existing unsatisfactory scars, and in the treatment, removal and inhibition of keloids (scars which extend beyond the original skin injury).
RXi’s sd-rxRNA compounds are designed for therapeutic use and have drug-like properties, such as high potency, target specificity, serum stability, reduced immune response activation, and efficient cellular uptake. They 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. The combination of these features has been shown in vitro and in animal tissues, to allow sd-rxRNAs to achieve efficient spontaneous cellular uptake and potent, long-lasting intracellular activity.
RXi Pharmaceuticals Corporation is a biotechnology company focused on discovering, developing and commercializing innovative therapies based on its proprietary, next-generation RNAi platform.