On November 8, 2017 RXi Pharmaceuticals Corporation (NASDAQ: RXII), a clinical-stage RNAi company developing innovative therapeutics that address significant unmet medical needs, reported that it has selected two self-delivering RNAi (sd-rxRNA) compounds from its immuno-oncology pipeline for preclinical development (Press release, RXi Pharmaceuticals, AUG 8, 2017, View Source [SID1234531518]). 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.

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"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:

Single therapeutic agent with one or multiple immune checkpoints attenuated
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
Only small alterations needed in cell manufacturing process: facilitates adaptation of existing cell technologies