MARLBOROUGH, MA, USA I August 8, 2017 I RXi Pharmaceuticals Corporation (NASDAQ: RXII), a clinical-stage RNAi company developing innovative therapeutics that address significant unmet medical needs, today announced that it has selected two self-delivering RNAi (sd-rxRNA®) compounds from its immuno-oncology pipeline for preclinical development. For oncology treatments based on adoptive cell transfer (ACT), compounds RXI-762 and RXI-804 suppress the expression of immune checkpoint proteins PD-1 and TIGIT respectively, which can result in an improved efficacy to the targeted tumors.  This decision triggered the selection of a manufacturing facility to initiate production of cGMP grade material, initially for the first of these two compounds (RXI-762). The latter also supports moving RXI-762 into clinical development as early as 2018 as part of an ACT therapy.

“It is our mission, using our novel technology platform, to improve patients’ lives through the development of a new class of treatments,” said Dr. Gerrit Dispersyn, Chief Development Officer. He further added that, “The selection of these two sd-rxRNA compounds, as well as securing cGMP manufacturing for such compounds, brings us closer to achieving this goal.”

RXi’s immuno-oncology program with sd-rxRNA provides a versatile approach to improve upon well-established ACT methodologies. The Company has identified lead compounds for a number of immune checkpoint targets that provide a long-lasting effect, individually and in combination, with target gene silencing demonstrated in various immune effector cells relevant in cancer immunotherapy, including CAR-T cells, TILs, and NK cells. 

Also, the Company’s ongoing discovery programs include, but are not limited to, the evaluation of sd-rxRNA compounds to silence targets related to cytokine release syndrome (CRS).  One of the development goals is to enhance the therapeutic potential and reduce the toxicity profile of current immuno-oncology treatments.

The potential advantages of RXi’s approach using sd-rxRNA for immunotherapy are:

  1. Single therapeutic agent with one or multiple immune checkpoints attenuated
  2. Streamlined regulatory path: no multiple combination trials required; newly discovered checkpoint targets can be rapidly tackled; ex vivo application of sd-rxRNA; clinically proven safety of sd-rxRNA
  3. Only small alterations needed in cell manufacturing process: facilitates adaptation of existing cell technologies

About RXi Pharmaceuticals

RXi Pharmaceuticals Corporation (NASDAQ: RXII) is a clinical-stage company developing innovative therapeutics that address significant unmet medical needs.  Building on the pioneering discovery of RNAi, scientists at RXi have harnessed the naturally occurring RNAi process which can be used to “silence” or down-regulate the expression of a specific gene that may be overexpressed in a disease condition.  RXi developed a robust RNAi therapeutic platform, including self-delivering RNA (sd-rxRNA®) compounds, that have the ability to highly selectively block the expression of any target in the genome, thus providing applicability to many therapeutic areas. Our current programs include dermatology, ophthalmology, and cell-based immunotherapy.  RXi’s extensive patent portfolio provides for multiple product and business development opportunities across a broad spectrum of therapeutic areas, and we actively pursue research collaborations, partnering and out-licensing opportunities with academia and pharmaceutical companies.  For additional information, visit the Company’s website,  

SOURCE: RXi Pharmaceuticals