"We are pleased and excited with the results that have been generated over the course of this year in our business development and clinical programs," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He added that, "As stated throughout this press release, you will see that we have continued to advance our clinical programs according to projected timelines and remain on track for final readouts during 2017. In fact, new data in the RXI-109 dermal trial confirm and extend the 3-month data previously reported for subjects treated with 5 mg/cm and indicate a positive effect of RXI-109 out to 6 months after the last dose. In addition, we look forward to completing the proposed MirImmune transaction that will allow the integration of our self-delivering RNAi compounds into a new therapeutic area. sd-rxRNA has the potential to become a key component in the rapidly expanding field of cellular immunotherapy, in what may be a transformational change in the way we treat patients with various malignancies, including solid tumors."
Business Development – Option to Acquire MirImmune: Recently MirImmune has provided new data demonstrating silencing of a number of undisclosed immunosuppressive targets in natural killer cells (NK cells) using RXi's sd-rxRNA compounds. This adds to a remarkable set of immune checkpoint modulation studies in human T cells, including CAR-T cells and tumor Infiltrating lymphocytes (TILs). In most cell types, the sd-rxRNA treatment results in potent silencing while maintaining close to 100% transfection efficiency and nearly full cell viability. Moreover, the silencing effect has been validated in a number of clinically used cell treatment protocols. The acquisition of MirImmune is the first step into the field of cell-based therapies for RXi where its sd-rxRNA platform has numerous advantages over other RNAi technologies.
RXI-109-1402 – Hypertrophic Scarring: The first two cohorts in RXI-109-1402 (3-month treatment regimen) are complete and a blinded assessment of paired clinical photographs taken at 3, 6 and 9 months has been conducted. These data, based on 14 reviewers resulting in a data set of ~1,200 observations, confirm the positive differentiation from untreated surgery incisions in hypertrophic scars previously presented for a subset of subjects treated with 5 mg/cm RXI-109 at 3 months. In addition, these data extend this observation to all time points, including the post-treatment follow-up period through 9 months post-surgery. Given the choices of whether one scar was better, not different from or worse, than its paired scar, the reviewers selected the RXI-109 treated sites as better >60% of the time at all time points and remarkably this differentiation was maintained even at 6 months after the last dose. RXI-109 was safe and well tolerated, with adverse events being typical to the healing process following scar revision surgery and intradermal injections, including mild to moderate redness, pain and tenderness. There were no RXI-109-related serious adverse events.
In the second half of the study, two cohorts were added to evaluate a 6-month treatment regimen. As compared to six doses in Cohorts 1 and 2, these cohorts include either nine doses (Cohort 3) or eight doses (Cohort 4). As expected, the limited 3-month data available from Cohort 3 appear to align with that of Cohorts 1 and 2 as these subjects all had the same dosing schedule through month 3. A complete read-out of the whole study including all four cohorts with follow-up until 9 months post- surgery is expected in the middle of 2017. At that time the complete data set will include conclusions based on scar evaluations from the principal investigators, demographics, and comparisons across cohorts and times.
RXI-109-1501 – Retinal Scarring in Advanced Age-related Macular Degeneration: The first two cohorts in RXI-109-1501 are completely enrolled and dosing in the 3rd cohort at the highest planned dose level has begun. Subjects in this study receive four intraocular doses of RXI-109 on a monthly basis followed by a 4-month observation period, and to date there have been no safety issues that precluded continuation of dosing. Complete enrollment is anticipated for early in 2017, ahead of our original plan, with a complete safety read out of the study in the second half of 2017.
RXI-SCP-1502 – Treatment of Cutaneous Warts: This study has completed enrollment of its first cohort of subjects, including the pharmacokinetic portion of this ongoing study. A preliminary review of sensitization and wart clearance data from the subset of subjects that have completed the 10-week treatment phase of the study was performed. Results show that greater than 90% of the subjects demonstrated a sensitization response, a prerequisite to be able to develop a therapeutic response. Additionally, more than 60% of the subjects responded to the treatment by exhibiting either complete or greater than 50% clearance of all treated warts with up to 10 weekly treatments. Samcyprone™ treatment has been generally safe and well tolerated with drug-related adverse events being most typically expected local reactions due to the sensitization and challenge responses in the skin. An additional cohort will be added in January 2017 to evaluate an optimized treatment/dosing regimen with a reduced initial sensitizing dose applied to the inner arm and all warts which may result in a more streamlined treatment phase. Whereas the read-out of the first cohort data will happen in the first half of 2017, the complete readout of the final study is anticipated in the second half of 2017.
Consumer / Functional Health Care: Efficacy and toxicity testing in cell culture and skin equivalents has been successfully completed for RXI-231, an sd-rxRNA that targets tyrosinase, a key enzyme in melanin synthesis. RXi is coordinating with a US clinical testing site to initiate human testing in Q1 2017 and RXI-231 has been manufactured in sufficient quantities to support this activity. In addition to evaluating safety, the effect of RXI-231 on the appearance of skin pigmentation will be assessed.
About RXi's Proprietary Self-delivering RNAi (sd-rxRNA) Technology Platform
RXi's proprietary sd-rxRNA technology has many advantages over its competitors in the RNAi space. Scientists at RXi have designed chemically-modified RNAi compounds with improved drug-like properties that are potent, stable and specific. These proprietary compounds have built-in delivery properties and therefore do not require a delivery vehicle for local therapeutic applications. The enhanced properties of sd-rxRNA include: 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.
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, the Company's discovery and clinical development programs are based on its proprietary self-delivering RNAi (sd-rxRNA®) platform and Samcyprone™, a small molecule topical immunomodulator. Current clinical development programs include RXI-109, an sd-rxRNA, for the treatment of dermal and ocular scarring, and Samcyprone™ for the treatment of such disorders as warts, alopecia areata, non-malignant skin tumors and cutaneous metastases of melanoma. RXi's robust pipeline, coupled with an extensive patent portfolio, provides for multiple product and business development opportunities across a broad spectrum of therapeutic areas. We are committed to being a partner of choice for academia, small companies, and large multinationals. We welcome ideas and proposals for strategic alliances, including in- and out-licensing opportunities, to advance and further develop strategic areas of interest. Additional information may be found on the Company's website, www.rxipharma.com.
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding the Company's plans or other expectations, goals, objectives, strategies, timelines and legal matters are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially, including the risks and uncertainties associated with: risks that the potential acquisition of MirImmune may not proceed; the actual realization of anticipated benefits of the purchase of MirImmune; risks related to our ability to control the timing of the purchase of MirImmune; 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; risks that other companies or organizations may assert patent rights preventing us from developing or commercializing our product candidates and other risks detailed from time to time in the Company's most recent Annual Report on Form 10-K and other documents subsequently filed with or furnished to the Securities and Exchange Commission. These forward-looking statements are based on current information that may change and you are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this filing, as actual results may differ from those contemplated by our forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement, and the Company undertakes no obligation to revise or update any forward-looking statement to reflect events or circumstances after the date of this press release.
RXi Pharmaceuticals Corporation
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