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RXi Pharmaceuticals Advances Immuno-Oncology Pipeline by Selecting Two sd-rxRNA Compounds for Preclinical Development and Sourcing cGMP Manufacturing

Aug 8, 2017

MARLBOROUGH, Mass., Aug. 8, 2017 /PRNewswire/ -- 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.

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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:

  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,  

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements about: our ability to successfully develop RXI-109, Samcyprone™ and our other product candidates (collectively "our product candidates"); the future success of our clinical trials with our product candidates; the timing for the commencement and completion of clinical trials; our ability to enter into strategic partnerships and the future success of these strategic partnerships; and our ability to deploy our sd-rxRNA® technology through partnerships, as well as the prospects of these partnerships to provide positive returns. Forward-looking statements about expectations and development plans of RXi's product candidates and partnerships involve significant risks and uncertainties, including the following: risks that we may not be able to successfully develop and commercialize our product candidates; risks that product development and clinical studies may be delayed, not proceed as planned and/or be subject to significant cost over-runs; risks related to the development and commercialization of products by competitors; risks related to our ability to control the timing and terms of collaborations with third parties; and risks that other companies or organizations may assert patent rights preventing us from developing or commercializing our product candidates. Additional risks are detailed in our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q under the caption "Risk Factors."  Readers are urged to review these risk factors and to not act in reliance on any forward-looking statements, as actual results may differ from those contemplated by our forward-looking statements. RXi does not undertake to update forward-looking statements to reflect a change in its views, events or circumstances that occur after the date of this release.


RXi Pharmaceuticals Corporation
Tamara McGrillen

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SOURCE RXi Pharmaceuticals Corporation