Oncbiomune has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, OncBioMune Pharmaceuticals, 2017, NOV 21, 2016, View Source [SID1234522125]).

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On November 21, 2016 Mustang Bio, Inc. ("Mustang"), a Fortress Biotech (NASDAQ: FBIO) Company, reported that Phase 1 clinical data and pre‐clinical data on its MB‐101 (IL13Rα2‐specific Chimeric Antigen Receptor–engineered CAR T cells (CAR T cells)) product candidate in development for the treatment of glioblastoma were presented by investigators from the City of Hope ("COH") in oral sessions at the 21st Annual Meeting and Education Day of the Society for Neuro‐Oncology ("SNO") in Scottsdale, AZ (Press release, Fortress Biotech, NOV 21, 2016, View Source;FID=1500093635 [SID1234516780]).   

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Dr. Lindsay A. Rosenwald, Fortress Biotech’s Chairman, President and Chief Executive Officer commented, "CAR T cell therapy has shown promise in treating certain forms of hematological cancers.  However, translating that activity into solid tumors has been challenging to date.  The clinical data presented at SNO by the investigators from COH suggest MB‐101 is safe and well‐tolerated, and capable of eliciting a potent anti‐tumor response in patients with glioblastoma (GBM), a disease that is almost universally fatal.  We believe this is the first evidence of activity of CAR T cells in the treatment of GBM.  We are very encouraged by the early evidence of anti‐tumor activity with MB‐101 with five of seven post‐surgical resection patients showing stable disease for >8 weeks and, in particular, one patient showing complete response for 7.5 months. Interestingly, this patient was the only patient of the seven to receive, on a compassionate use basis, the dual delivery of CAR T cells by both intracavitary and via intraventricular administration, which provides systemic CNS delivery of the CAR T cells.  We look forward to continuing to advance MB‐101 in Phase 1 studies and further exploring the dual delivery approach beyond this single patient experience."

The following summarizes the oral presentations on November 18, 2016 at SNO:

Phase I Study of Chimeric Antigen Receptor–Engineered T cells Targeting IL13Rα2 for the Treatment of Glioblastoma

Presenter: Christine E. Brown, PhD, Heritage Provider Network Professor of Immunotherapy, Associate Director, T Cell Therapeutics Research Laboratory, City of Hope National Medical Center/Beckman Research Institute

The Phase I study presented showed early clinical data evaluating IL13Rα2‐targeted CAR T cell therapy for the treatment of glioblastoma.  On this study, patients are treated on a four‐week therapeutic regimen consisting of three weekly intracranial infusions of IL13Rα2‐specific CAR T cells followed by one rest week for toxicity and disease assessment.  To date, seven patients have been treated with local intracavitary delivery of the CAR T cells following surgical resection.
Some highlights from the presentation included:
The treatment was well‐tolerated in all patients treated – with No DLTs or therapy‐related SAEs
No grade 3 or higher toxicities attributed to the therapy were observed
No CRS or Neurotoxicity was observed
Only grade  < 2 fevers, headaches, myalgia, chills
Best Response: 2 PD, 4 SD for >8‐weeks, 1 SD CR following intraventricular CAR T therapy for 7.5 months

Development of murine IL13Rα2‐targeted CAR T cells (mIL13BBζ) for assessment of CAR T cell therapy in syngeneic glioma models

Presenter: Darya Alizadeh, PhD, City of Hope National Medical Center/Beckman Research Institute

The pre‐clinical research program presented discussed a murine IL13Rα2‐targeted CAR T cell platform that was developed to evaluate parameters that impact the efficacy of CAR T cell therapy. Overall, the development of mIL13BBζ CAR T cells and its applications will allow researchers to assess factors that may impact the efficacy of CAR T cells and provide invaluable information critical for combination therapies and clinical trial design.  These studies may also provide important insights for improving therapeutic outcomes for patients with glioblastoma.

About Glioblastoma multiforme (GBM)
Glioblastomas (GBM) are tumors that arise from astrocytes cells that make up the supportive tissue of the brain. These tumors are usually highly malignant (cancerous) because the cellsreproduce quickly and they are supported by a large network of blood vessels. GBM isthe most common brain and central nervoussystem (CNS) malignancy, accounting for 15.1 percent of all primary brain tumors and 55.1 percent of all gliomas. There will be an estimated 12,120 new glioblastoma casesin the U.S. in 2016. Malignant brain tumors are the most common cause of cancer‐ related deaths in adolescents and young adults aged 15‐39, and the most common cancer occurring among 15‐19 year olds in the U.S. (Brain Tumor Statistics. American Brain Tumor Association.  December 2015). While GBM is a rare disease (2‐3 cases per 100,000 person life years in the U.S. and EU), it is quite lethal with five‐year survival rates historically lessthan 10 percent. Chemotherapy with temozolomide and radiation are shown to extend mean survival from approximately 12 to 15 months, while surgery remains the standard of care. GBM remains difficult to treat due to the inherent resistance of the tumor to conventional therapies. Treatment is further complicated by the susceptibility of the brain to damage, difficulty of the brain to repair itself and limitation to drugs crossing the blood brain barrier. Immunotherapy approaches targeting brain tumors offer promise over conventional treatments.

About MB‐101 (IL13Rα2‐specific CAR T cells)
IL13Rα2 is an attractive target for CAR T therapy as it has limited expression in normal tissue but is over‐expressed on the surface of the majority of GBM. CAR T cells are designed to express a membrane‐tethered IL‐13 receptor ligand (IL‐13) incorporating a single point mutation that provides high affinity for IL13Rα2 and reduces binding to IL13Rα1 in order to reduce healthy tissue targeting.  

Mustang is developing an optimized CAR T product incorporating enhancements in CAR design and T cell engineering to improve antitumor potency and T cell persistence. We include a second‐generation hinge optimized CAR containing mutations in the IgG4 linker to reduce off‐target Fc interactions, as well as the 41BB (CD137) co‐stimulatory signaling domain forimproved persistence of CAR T cells, and extracellular domain of CD19 as a selection/safety marker. In order to further improve persistence, central memory T cells are enriched and genetically engineered using a manufacturing process that limits ex vivo expansion in order to reduce T cell exhaustion and maintain a memory T cell phenotype.

On November 21, 2016 Ruga Corporation reported the company’s name change to Aravive Biologics, Inc., and the relocation of its business operations to Houston, Texas (Press release, Aravive Biologics, NOV 21, 2016, View Source [SID1234516733]). The move follows the company’s award of a $20 million grant from the Cancer Prevention & Research Institute of Texas (CPRIT), which is supporting the development of a novel drug candidate, Aravive-S6, as a potential treatment for acute myelogenous leukemia (AML) and solid tumors including ovarian, pancreatic, and breast cancers.

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"We are very pleased to be selected to receive this significant funding from CPRIT and look forward to building our business in Houston’s Texas Medical Center," said Ray Tabibiazar, M.D., President and Chief Executive Officer of Aravive Biologics. "Houston’s vibrant biomedical community is home to many of the top cancer researchers in the United States, including noted experts on AML, our lead hematologic cancer indication. We look forward to accessing this outstanding expertise and growing our presence within the local biomedical community as we advance Aravive-S6 into clinical trials."

"Investing in Aravive Biologics’ clinical development program was an easy decision for CPRIT," said Michael Lang, Chief Product Development Officer of CPRIT. "Aravive-S6 is an innovative compound that has exhibited strong preclinical proof-of-principal, and it addresses a critical unmet medical need. The company also has experienced management with an excellent track record in oncology drug development. Aravive team members are well positioned for success, and we welcome them to the Houston biomedical community."

Aravive-S6 is a novel high-affinity, soluble Fc-fusion protein designed to block the activation of the GAS6- AXL signaling pathway by serving as a decoy that prevents the binding of GAS6 to the AXL receptor on the surface of tumor cells. The AXL receptor, when activated through GAS6 binding, has been shown to act as a "survival switch," a key driver of invasiveness and metastasis, and a critical regulator of therapeutic resistance to cytotoxic chemotherapeutic drugs.

Aravive Biologics has robust and compelling data demonstrating the in vivo efficacy and tolerability of its lead drug candidate in preclinical models of ovarian, renal, breast, and pancreatic cancer, and AML. Aravive-S6 provides high specificity and selectivity for the AXL/GAS6 pathway that other anti-AXL and anti-GAS6 inhibitors have been unable to match; it has greater than 100-fold tighter affinity for GAS6 compared to other anti-AXL and anti-GAS6 antibody candidates in development. Aravive Biologics has also developed a proprietary complementary diagnostic tool that may enable the identification of patients with cancers exhibiting elevated GAS6 levels, which would allow the company to match its drug candidate to those patients most likely to benefit from therapy.

AML is a cancer that begins in bone marrow and affects cells intended to mature into different types of blood cells. Research shows that interaction between the AXL receptor and its GAS6 ligand leads to more severe and invasive cases of AML.

"As patients with AML tend to be older (over 60 years of age) and possibly also in poorer health, they are often unable to tolerate standard, intensive chemotherapy regimens and thus must undergo less rigorous treatment, said Amato Giaccia, Ph.D., Chief Scientific Officer and co-founder of Aravive. "We envision that Aravive-S6 might be administered either as a single agent or as a complement to standard chemotherapy that assists in reducing the survival of cancer cells, which have become "addicted" to AXL/GAS6 signaling, while attempting to achieve or maintain remission."

Each year, approximately 19,950 new cases of AML are diagnosed, primarily in adults, and about 10,430 deaths from the disease, nearly all in adults. About 35% of AML cases exhibit active GAS6/AXL signaling, an incidence which may potentially qualify Aravive-S6 for Orphan Drug Designation.

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.