On October 12, 2016 RXi Pharmaceuticals Corporation (NASDAQ: RXII), a clinical-stage RNAi company developing innovative therapeutics that address significant unmet medical needs, reported that it has entered into an exclusive option agreement to acquire all outstanding capital stock of MirImmune Inc., a privately-held company focused on the development of next generation immunotherapies for the treatment of cancer, in consideration for a number of shares equal to 19.99% of the then outstanding shares of common stock of RXi, plus additional potential consideration contingent on MirImmune reaching certain milestones (Press release, RXi Pharmaceuticals, OCT 12, 2016, View Source [SID:SID1234515779]). RXi Pharmaceuticals can exercise the option to acquire MirImmune on the terms set forth in the option agreement at any time prior to April 5, 2017 but has no obligation to do so. Schedule your 30 min Free 1stOncology Demo! MirImmune was co-founded by Tim Barberich, founder and former Chairman and CEO of Sepracor, Inc. In early 2015, RXi and MirImmune entered into an exclusive license agreement to RXi’s novel and proprietary sd-rxRNA technology for use in developing innovative cell-based cancer immunotherapies. MirImmune’s progress in cell therapy using RXi’s RNAi technology during the past 18 months forms a strong foundation for therapeutic development in the immuno-oncology space. If RXi exercises its option to acquire MirImmune, the acquisition would enable RXi to expand its pipeline and enter into the rapidly expanding field of immuno-oncology using the unique competitive advantages of sd-rxRNA technology.
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The advantages of RXi’s RNAi technology, listed below, have been verified in in vitro testing by MirImmune and their partners, and offer support that sd-rxRNA technology is uniquely suited for immune checkpoint modulation in cellular immuno-oncology therapies, such as CAR T-cells.
Multiple immune checkpoints can be targeted at the same time using a combination of sd-rxRNA compounds;
sd-rxRNA have been able to block both extracellular immune check points as well as intracellular targets that are not accessible to antibody therapies; and
Rapid and efficient transfection of T-cells with sd-rxRNA compounds in cell culture (>98%) is obtained while maintaining close to 100% cell viability.
In addition to demonstrating the effectiveness of our sd-rxRNA platform for cell therapy, MirImmune’s progress to date also includes:
Selection of six lead sd-rxRNA compounds against six different extracellular and intracellular immune check points;
Preclinical data that demonstrate silencing of all six targets in vitro, singly and in combinations;
Efficient and long-lasting knockdown of immune checkpoints in vivo;
Demonstrating the applicability of sd-rxRNA transfection in cell therapy to solid tumors in addition to blood cancers by:
Providing preclinical data demonstrating that mesothelin-targeted CAR T-cells modified with the anti-PD1 targeted sd-rxRNA significantly slow in vivo tumor growth in a human ovarian cancer model in mice compared to vehicle control
Generating promising results in ex vivo testing on melanoma cells obtained from a patient-donor, using sd-rxRNA reduction of PD1 in tumor infiltrating lymphocytes (TILs) resulting in destruction of those melanoma cells; and
Filing of intellectual property that covers the use of RNAi compounds for use in cell therapy.
"We are very pleased to have the opportunity to welcome MirImmune into our Company," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He added that, "This acquisition would not only allow us to create value for our shareholders; it more importantly sets the stage for what could potentially be a transformational change in the way we treat patients with various malignancies, including solid tumors. Our ultimate goal, through our own efforts as well as through partnerships, is the development of more tolerable treatments resulting in better quality of life and extended survival of family members we would otherwise lose prematurely. This approach is a key first step into the field of cell-based therapies, where sd-rxRNA has numerous advantages over other gene-modulating technologies."
"We are very excited to join our efforts with RXi in the development of RNAi for immunotherapy of cancer," said Dr. Alexey Eliseev, CEO of MirImmune, Inc. He added that, "We at MirImmune have taken first steps in improving the properties of cell-based immunotherapies, such as CAR T‑cells, by RNAi and enabling them to work in the immunosuppressive environment of solid tumors. CAR T‑cell treatments have shown an enormous promise in the treatment of hematological malignancies, but they have been only marginally effective with solid tumors. We treat therapeutic immune cells with RNAi compounds ex-vivo to knock down immune checkpoints, such as PD-1, LAG-3 and others. This treatment boosts the anti-tumor activity of the cells when they are subsequently administered to the patients. RXi’s sd-rxRNA is arguably the best RNAi technology for such ex vivo treatment of cells. Our plan is to develop sd-rxRNA as a more safe and effective alternative to gene editing of the immune cells or checkpoint blockade with monoclonal antibodies."
About RXi’s Proprietary Self-delivering RNAi (sd-rxRNA) Technology Platform
RNAi is a powerful molecular tool that has the ability to "silence" or down-regulate the expression of a specific gene that may be overexpressed in a disease condition. Scientists at RXi developed a robust RNAi therapeutic platform that includes self-delivering RNA (sd-rxRNA) compounds where drug-like properties are built into the RNAi compound itself. These proprietary compounds are novel RNAi compounds with enhanced properties for therapeutic use including: efficient spontaneous cellular uptake, stability, reduced potential for immune stimulation, and potent, long-lasting intracellular activity. All cell types tested (primary, neuronal and non-adherent) internalize sd-rxRNA compounds uniformly and efficiently, resulting in potent and long lasting silencing. sd‑rxRNA compounds have the ability to selectively block the expression of any target in the genome providing applicability to a broad spectrum of therapeutic areas.