On November 21, 2016 Syros Pharmaceuticals (NASDAQ: SYRS) reported that new data on its lead candidate, SY-1425, a selective retinoic acid receptor alpha (RARα) agonist, will be highlighted in a late-breaking presentation at the San Antonio Breast Cancer Symposium (SABCS) taking place December 6-10, 2016, in San Antonio (Press release, Syros Pharmaceuticals, NOV 21, 2016, View Source;p=irol-newsArticle&ID=2224844 [SID1234516732]).

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The presentation will feature new preclinical data showing that SY-1425 represents a potentially promising therapeutic approach for defined subsets of breast cancer patients whose tumors are driven by abnormally high expression of the RARA gene.

Details on the presentations are as follow:

Date & Time: Saturday, December 10, from 7:30 – 9:00 am CST
Presentation Title: A novel subgroup of estrogen receptor positive breast cancer may benefit from super-enhancer guided patient selection for retinoic acid receptor α agonist treatment
Session: Treatment: Novel Targets and Targeted Agents
Presenter: Michael R. McKeown, Ph.D., Senior Scientist, Syros Pharmaceuticals
Program Number: P6-11-18
Location: Henry B. Gonzalez Convention Center, Hall 1

SY-1425 is currently in a Phase 2 clinical trial in genomically defined subsets of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) patients. Using its gene control platform, Syros discovered subsets of AML, MDS and breast cancer patients whose tumors have a highly specialized regulatory region of non-coding DNA, known as a super-enhancer, associated with the RARA gene, which codes for the RARα transcription factor. The super-enhancer is believed to lead to over-expression of the RARA gene, locking cells in an immature, undifferentiated and proliferative state. Treatment with SY-1425 in cancer cells with this super-enhancer promotes differentiation of these cells.

About Syros Pharmaceuticals

Syros Pharmaceuticals is pioneering the understanding of the non-coding region of the genome to advance a new wave of medicines that control expression of disease-driving genes. Syros has built a proprietary platform that is designed to systematically and efficiently analyze this unexploited region of DNA in human disease tissue to identify and drug novel targets linked to genomically defined patient populations. Because gene expression is fundamental to the function of all cells, Syros’ gene control platform has broad potential to create medicines that achieve profound and durable benefit across a range of diseases. Syros is currently focused on cancer and immune-mediated diseases and is advancing a growing pipeline of gene control medicines. Syros’ lead drug candidates are SY-1425, a selective RARα agonist in a Phase 2 clinical trial for genomically defined subsets of patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-1365, a selective CDK7 inhibitor with potential in a range of solid tumors and blood cancers. Led by a team with deep experience in drug discovery, development and commercialization, Syros is located in Cambridge, Mass.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including without limitation statements regarding the potential therapeutic benefits of treatment with SY-1425 in genomically defined subsets of AML, MDS and breast cancer patients. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: Syros’ ability to: advance the development of its programs, including SY-1425, under the timelines it projects in current and future clinical trials; obtain and maintain patent protection for its drug candidates and the freedom to operate under third party intellectual property; demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; replicate scientific and non-clinical data in clinical trials; successfully develop a companion diagnostic test to identify patients with biomarkers associated with the RARA super-enhancer; obtain and maintain necessary regulatory approvals; identify, enter into and maintain collaboration agreements with third parties; manage competition; manage expenses; raise the substantial additional capital needed to achieve its business objectives; attract and retain qualified personnel; and successfully execute on its business strategies; risks described under the caption "Risk Factors" in the company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which is on file with the Securities and Exchange Commission; and risks described in other filings that the company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Syros expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

On November 21, 2016 RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) ("RedHill" or the "Company"), a biopharmaceutical company primarily focused on development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, reported the presentation of a poster relating to YELIVA (ABC294640), the Company’s proprietary, first-in-class, orally-administered sphingosine kinase-2 (SK2) selective inhibitor, at the 2016 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Molecular Targets and Cancer Therapeutics Symposium, on November 29, 2016, in Munich, Germany (Press release, RedHill Biopharma, NOV 21, 2016, View Source [SID1234516722]).

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The poster, entitled ‘Antitumor and Anti-Inflammatory Effects of the Sphingosine Kinase-2 Inhibitor ABC294640 in Combination with Radiation,’ was authored by scientists from Apogee Biotechnology Corporation ("Apogee"), the original developers of YELIVA.

By inhibiting the SK2 enzyme, YELIVA blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid signaling molecule that promotes cancer growth and pathological inflammation.

RedHill is pursuing and evaluating, with YELIVA, multiple clinical programs in oncology, inflammatory and gastrointestinal indications, as well as potential collaboration opportunities with larger pharmaceutical companies to evaluate YELIVA as an add-on therapy to their existing oncology treatments.

Results from a Phase I study with YELIVA in patients with advanced solid tumors confirmed that the study, conducted at the Medical University of South Carolina Hollings Cancer Center (MUSC), successfully met its primary and secondary endpoints, demonstrating that the drug is well-tolerated and can be safely administered to cancer patients at doses that provide circulating drug levels that are predicted to have therapeutic activity.

A Phase II study with YELIVA for the treatment of advanced hepatocellular carcinoma (HCC) was initiated at MUSC Hollings Cancer Center. The study protocol is under FDA review and enrolment is expected to begin by year end 2016. The study is supported by a $1.8 million grant from the National Cancer Institute (NCI) awarded to MUSC and is intended to fund a broad range of studies on the feasibility of targeting sphingolipid metabolism for the treatment of a variety of solid tumor cancers, with additional funding from RedHill.

A Phase Ib/II study with YELIVA for the treatment of refractory or relapsed multiple myeloma was initiated at Duke University Medical Center. The study is supported by a $2 million grant from the NCI Small Business Innovation Research Program (SBIR) awarded to Apogee, in conjunction with Duke University, with additional support from RedHill.

A Phase I/II clinical study evaluating YELIVA in patients with refractory/relapsed diffuse large B-cell lymphoma was initiated at the Louisiana State University Health Sciences Center in New Orleans in June 2015 and was recently amended to address overall recruitment prospects. The study, which will now also include Kaposi sarcoma patients, is expected to resume by the end of 2016, pending regulatory approval. The study is supported by a grant from the NCI, as well as additional support from RedHill.

A Phase Ib study to evaluate YELIVA as a radioprotectant for prevention of mucositis in head and neck cancer patients undergoing therapeutic radiotherapy is planned to be initiated in the first quarter of 2017.

The ongoing studies with YELIVA (ABC294640) are registered on www.ClinicalTrials.gov, a web-based service by the U.S. National Institute of Health, which provides public access to information on publicly and privately supported clinical studies.

About YELIVA (ABC294640):
YELIVA (ABC294640) is a Phase II-stage, proprietary, first-in-class, orally-administered, sphingosine kinase-2 (SK2) selective inhibitor with anti-cancer and anti-inflammatory activities. RedHill is pursuing with YELIVA multiple clinical programs in oncology, inflammatory and gastrointestinal indications. By inhibiting the SK2 enzyme, YELIVA blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid signaling molecule that promotes cancer growth and pathological inflammation. SK2 is an innovative molecular target for anticancer therapy because of its critical role in catalyzing the formation of S1P, which is known to regulate cell proliferation and activation of inflammatory pathways. YELIVA was originally developed by U.S.-based Apogee Biotechnology Corp. and completed multiple successful pre-clinical studies in oncology, inflammation, GI and radioprotection models, as well as the ABC-101 Phase I clinical study in cancer patients with advanced solid tumors. The development of YELIVA was funded to date primarily by grants and contracts from U.S. federal and state government agencies awarded to Apogee Biotechnology Corp., including the U.S. National Cancer Institute, the U.S. Department of Health and Human Services’ Biomedical Advanced Research and Development Authority (BARDA), the U.S. Department of Defense and the FDA Office of Orphan Products Development.

On November 21, 2016 ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE MKT:IMUC) reported the presentation of updated immune monitoring data from the phase 2 trial of ICT-107 in patients with newly diagnosed glioblastoma (Press release, ImmunoCellular Therapeutics, NOV 21, 2016, View Source [SID1234516718]). Also presented were updated long-term survival data from the phase 1 trial of ICT-107. ICT-107 is a dendritic cell-based immunotherapy targeting multiple tumor-associated antigens on glioblastoma stem cells. ICT-107 is currently being tested in a phase 3 registration trial in patients with newly diagnosed glioblastoma. The updated phase 1 and phase 2 data were presented in two oral sessions on Friday, November 18th, at the 21st Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology, held in Scottsdale, AZ.

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The ICT-107 phase 2 trial was a randomized, double-blind, placebo-controlled phase 2 study of the safety and efficacy of ICT-107 in patients with newly diagnosed glioblastoma following resection and chemoradiation. ICT-107 is an intradermally administered autologous immunotherapy consisting of the patient’s own dendritic cells pulsed with six synthetic tumor-associated antigens: AIM-2, MAGE-1, TRP-2, gp100, HER-2, IL-13Rα2. The placebo control consisted of the patient’s unpulsed dendritic cells. The data from the phase 2 trial indicated a survival advantage in the ICT-107 treated group compared to the control group. The data also showed an association between immune response and survival, especially in HLA-A2 positive (HLA-A2+) patients, which is the target patient population for the ongoing phase 3 registration trial.

The updated immune response data from the phase 2 trial showed that treatment with ICT-107 resulted in the development of a measurable anti-tumor T cell response in some patients, which was associated with survival. Patients that developed the anti-tumor T cell response which was measurable by both ELISpot (to detect viable T cells capable of binding to a target antigen) and multimer testing (to detect T cell binding with higher sensitivity than ELISpot) had improved survival. The data demonstrated that immuno-monitoring can provide an early indication of patients responding to immunotherapy. In the current ongoing phase 3 registration trial of ICT-107, ImmunoCellular plans to perform immuno-monitoring to support the trial.

The data were presented at SNO by Steven J. Swanson, PhD, Senior Vice President, Research, ImmunoCellular Therapeutics, in a presentation titled, "Categorizing immune responders with fusion metrics and simulation for association to survival and progression-free survival with immune response in HLA-A2+ patients with GBM from a phase 2 trial of dendritic cell (DC) immunotherapy (ICT-107)."

Dr. Swanson commented: "ICT-107 is designed to deliver therapeutic benefit by stimulating the patient’s immune system to attack tumor tissue. A first indicator that the immunotherapeutic is active is the production of tumor-specific T cells by the patient. In our SNO presentation, we described our ability to more clearly interpret the immune-monitoring data from the phase 2 trial. The ability to accurately identify negative and positive responses enabled us to better understand which of the patients in our trial generated T cells capable of attacking the tumor. We determined that patients with a T cell response measureable in both the ELIspot assay and through multimer analysis achieved longer survival as compared with patients who did not show a positive response. These data should enable us to better interpret the results of our ongoing phase 3 trial."

Andrew Gengos, ImmunoCellular Chief Executive Officer, said: "The phase 2 trial immune monitoring results indicate that patients who mount a T cell response appear to have improved survival over those without a detectable response. In designing the phase 3 trial, we have made important changes in the protocol to potentially enhance the immune response in ICT-107 treated patients with the goal of optimizing the potential survival outcomes in the trial."

Data from the phase 1 trial of ICT-107 were presented by Surasak Phuphanich, MD, Department of Neurology, Cedars-Sinai in Los Angeles, in a presentation titled "Ten-year follow up with long term remission in patients with newly diagnosed glioblastoma (GBM) treated with ICT-107 vaccine (phase 1)." The phase 1 open-label, single institution trial, which was completed in 2010, included 16 evaluable patients with newly diagnosed glioblastoma. Results of the study were initially published in 2012 (Cancer Immunol Immunother).

Updated survival data presented by Dr. Phuphanich at the 2016 SNO meeting showed that 19% of patients had long-term remission of greater than 8 years, with the longest remission being 9.6 years. Also, 38% of patients demonstrated long-term survival of greater than 8 years, with the longest survivor greater than 10.2 years. Immune response data showed a correlation between survival and cancer-stem-associated expression, and a trend toward greater CD8 T cell cytokine responses in long-term survivors.

ICT-107 Phase 3 Registration Trial Underway

The ongoing phase 3 registrational trial of ICT-107 is designed as a randomized, double-blind, placebo-controlled study of HLA-A2+ subjects, which is being conducted at about 120 sites in the US, Canada and the EU, with plans to randomize at least 500 patients with newly diagnosed glioblastoma. The primary endpoint in the trial is overall survival. Secondary endpoints include progression-free survival and safety, as well as overall survival in the two pre-specified MGMT subgroups.

For patients, families and physicians seeking additional information about the ICT-107 phase 3 trial, please consult www.clinicaltrials.gov.

About ImmunoCellular Therapeutics, Ltd.