On May 04, 2016 ArQule, Inc. (Nasdaq:ARQL) reported its financial results for the first quarter of 2016 (Press release, ArQule, MAY 4, 2016, View Source [SID:1234511911]).

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For the quarter ended March 31, 2016, the Company reported a net loss of $4,981,000 or $0.08 per share, compared with a net loss of $4,551,000 or $0.07 per share, for the quarter ended March 31, 2015.

At March 31, 2016, the Company had a total of approximately $47,642,000 in cash, equivalents and marketable securities.

Key Highlights

ARQ 092, our proprietary AKT inhibitor in phase 1 for Proteus syndrome, has demonstrated AKT knockdown in the first three patients dosed: Our collaborators, the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH), have completed the safety, pharmacokinetics and biomarker evaluation of the first cohort of three patients in the phase 1 trial for ARQ 092 in Proteus syndrome. In all of these patients, ARQ 092 was well tolerated and successfully achieved the pre-specified decrease in AKT signaling. The NIH has opened enrollment to patients ages 12 to 18.
ARQ 092 phase 1b trial in oncology has completed enrollment: The final cohort of patients with AKT1/PI3K mutations has completed enrollment. In total, 10 patients with AKT1 mutations have been enrolled in the phase 1b portion of the trial. We anticipate presenting data from the study by year-end.
ARQ 087, our proprietary FGFR inhibitor in phase 2 portion of the trial for intrahepatic cholangiocarcinoma (iCCA), is expected to complete enrollment in the third quarter of 2016: ArQule recently received a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) on orphan drug designation for ARQ 087 for biliary tract cancer. The phase 2 portion of the study continues as planned and preliminary data will be available this summer.
ARQ 761, our NQO1 inhibitor, in phase 1b/2 for pancreatic cancer completed enrollment of the first cohort: Our collaborators at the University of Texas Southwestern Medical Center have enrolled the first cohort of patients in combination with gemcitabine and abraxane.
ARQ 531, our proprietary BTK inhibitor, proceeds into Good Laboratory Practice (GLP) toxicology studies: Pre-clinical experiments, including toxicity studies, for ARQ 531 are proceeding as planned.
Tivantinib phase 3 trial in second-line hepatocellular carcinoma, METIV-HCC, completed its planned interim assessment and will continue to the final analysis: The independent data monitoring committee conducted the planned interim assessment and it was determined that the trial will continue to its final analysis. The biomarker-driven phase 3 trial is expected to be completed by year end. The METIV-HCC trial is randomized 2:1 against best supportive care and enrolled approximately 300 MET-high patients with the primary end-point of overall survival.
"We were particularly pleased to report progress with our proprietary pipeline this quarter, and we remain on track for additional data read-outs this year," said Paolo Pucci, Chief Executive Officer of ArQule. "The completion of enrollment for the oncology trial with ARQ 092 and the anticipated completion of the ARQ 087 trial in iCCA next quarter set us up nicely to achieve our 2016 goals. With the METIV-HCC interim analysis behind us, we look forward to concluding the trial by year-end."

"We are pleased to hear from our collaborators at the NIH that the data collected from the first cohort of patients provides compelling in vivo evidence of the effect of ARQ 092 in Proteus syndrome," said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "The opportunity for ArQule to add rare diseases to its established oncology clinical development program is a significant step forward in our efforts in precision medicine."

Revenues and Expenses

Revenues for the quarter ended March 31, 2016, were $1,227,000 compared with revenues of $2,785,000 for the quarter ended March 31, 2015. Research and development revenue in 2016 and 2015 includes revenue from the Daiichi Sankyo tivantinib development agreement and the Kyowa Hakko Kirin exclusive license agreement. The revenue decreases in the quarter ended March 31, 2016 of $0.6 million from our Daiichi Sankyo METIV-HCC trial and $1.0 million from our Kyowa Hakko Kirin JET-HCC trial were due to the extension of the development period through December 31, 2016 for both programs.

Research and development expenses in the first quarter of 2016 were $4,198,000, compared with $4,413,000 for the first quarter of 2015. The $0.2 million decrease in research and development expense in the first quarter of 2016 was primarily due to lower facility costs of $0.3 million and lower labor related costs of $0.2 million from reduced headcount. These decreases were partially offset by increased outsourced clinical and product development costs of $0.3 million.

General and administrative costs were $2,044,000 in the first quarter of 2016 compared with $3,187,000 for the first quarter of 2015. General and administrative expense decreased by $1.2 million in the first quarter of 2016 primarily due to lower facility costs of $0.9 million and labor related cost of $0.2 million.

2016 Financial Guidance

For 2016, ArQule expects net use of cash to range between $23 and $25 million. Revenues are expected to range between $4 and $5 million. Net loss is expected to range between $24 and $27 million, and net loss per share to range between $(0.34) and $(0.39) for the year. ArQule expects to end 2016 with between $29 and $31 million in cash and marketable securities. Our guidance has been updated to include the issuance of 8,027,900 shares of common stock related to the stock offering completed during the quarter.

On MAY 4, 2016 GlycoMimetics, Inc. (NASDAQ: GLYC) reported financial results for the first quarter ended March 31, 2016.
"In February of 2016, we announced the topline readout of data from the first two cohorts in our ongoing Phase 1/2 clinical trial evaluating our drug candidate GMI-1271 in relapsed/refractory acute myeloid leukemia (AML) patients (Filing, Q1, GlycoMimetics, 2016, MAY 4, 2016, View Source [SID:1234511914]). Of the first 13 evaluable patients dosed with GMI-1271 in combination with chemotherapy, investigators observed clinical responses in eight patients, for an overall response rate of 62 percent. This group of 13 patients also tolerated GMI-1271 well. This is an important finding, and it is our plan to present the data in greater detail at a major medical meeting," said Rachel King, GlycoMimetics’ Chief Executive Officer. "In addition, we plan soon to initiate clinical development of GMI-1359, our third product candidate, also with a novel mechanism of action targeting E-selectin and CXCR4, an important target believed to play a key role in cancer cell migration and infiltration. Here our initial indications will likely also be hematological cancers, but in the future we may expand into solid tumors as well. We anticipate filing an investigational new drug application (IND) with the U.S. Food & Drug Administration for GMI-1359 in the third quarter of 2016."

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As of March 31, 2016, GlycoMimetics had cash and cash equivalents of $38.8 million. GlycoMimetics reported no revenue for the quarter ended March 31, 2016 or the quarter ended March 31, 2015. The company’s research and development expenses increased to $5.5 million for the quarter ended March 31, 2016 as compared to $5.2 million for the first quarter of 2015. During the three months ended March 31, 2016, our research and development expense increased by approximately $300,000 compared to the same period in 2015, an increase of 6 percent that was primarily attributable to an increase in GMI-1271 clinical trial costs, offset in part by a decrease in expenses related to manufacturing and process development for GMI-1271. In addition, the preclinical development of GMI-1359 has resulted in increased expenses in manufacturing and for toxicology studies required to support a potential IND filing. The company’s general and administrative expenses increased slightly to $2.1 million for the quarter ended March 31, 2016 as compared to $1.9 million for the first quarter of 2015. These increases were primarily due to increased headcount and associated salaries and stock-based compensation expense.

Insufficient tissue oxygenation, or hypoxia, contributes to tumor aggressiveness and has a profound impact on clinical outcomes in cancer patients. At decreased oxygen tensions, hypoxia-inducible factors (HIFs) 1 and 2 are stabilized and mediate a hypoxic response, primarily by acting as transcription factors. HIFs exert differential effects on tumor growth and affect important cancer hallmarks including cell proliferation, apoptosis, differentiation, vascularization/angiogenesis, genetic instability, tumor metabolism, tumor immune responses, and invasion and metastasis. As a consequence, HIFs mediate resistance to chemo- and radiotherapy and are associated with poor prognosis in cancer patients. Intriguingly, perivascular tumor cells can also express HIF-2α, thereby forming a "pseudohypoxic" phenotype that further contributes to tumor aggressiveness. Therefore, therapeutic targeting of HIFs in cancer has the potential to improve treatment efficacy. Different strategies to target hypoxic cancer cells and/or HIFs include hypoxia-activated prodrugs and inhibition of HIF dimerization, mRNA or protein expression, DNA binding capacity, and transcriptional activity. Here we review the functions of HIFs in the progression and treatment of malignant solid tumors. We also highlight how HIFs may be targeted to improve the management of patients with therapy-resistant and metastatic cancer.
Copyright © 2016. Published by Elsevier Inc.

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On May 04, 2016 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported it will exhibit the breadth and promise of its cancer and infectious disease immunotherapy, vaccine and DNA-based monoclonal antibody (dMAb) pipeline in 10 presentations at the 19th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) held in Washington, DC from May 4-7 (Press release, Inovio, MAY 4, 2016, View Source;Cell-Therapy/default.aspx [SID:1234511916]).

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The ASGCT (Free ASGCT Whitepaper) abstract review committee selected Inovio’s presentation of its successful preclinical Ebola vaccine development for one of only three presentations to be given at its prestigious Presidential Symposium. In the selected abstract, out of 768 submitted and accepted, the presented preclinical testing in mice and non-human primates showed Inovio’s Ebola vaccine protected 100% of immunized animals from sickness and death following exposure to a lethal dose of Ebola virus. Those results led to a study of 75 healthy subjects which showed the vaccine was safe, tolerable, and generated strong T cell and antibody responses in human as well.

Inovio’s Ebola results will be offered on Friday, May 6, at 2:50 p.m. in a presentation entitled: "479 – An Optimized DNA Vaccine Formulation Protects Against Lethal Ebola Makona Virus Challenge in Non-Human Primates and Elicits Robust Immune Responses."

Dr. J. Joseph Kim, President and CEO, said, "The results we will present at the ASGCT (Free ASGCT Whitepaper) global scientific forum demonstrate the potential and breadth of the three pillars of Inovio’s broad pipeline: Immunotherapies and vaccines against cancers and challenging infectious diseases and our DNA-encoded monoclonal antibodies (dMAbs), which we believe will offer clear advantages over conventional monoclonal antibody technologies including faster development, easier product manufacturing, and more favorable pharmacokinetics."

Inovio and its collaborators will present clinical and preclinical data that supports its on-going trials exploring the next generation of immunotherapies, vaccine and monoclonal antibodies at the following times over the next two days (link to abstracts):

19th Annual Meeting of the American Society of Gene & Cell Therapy

May 5

264 – In Vivo DNA-Monoclonal Antibody (DMAb) Gene Delivery Protects Against Lethal Bacterial and Viral Challenges in Mice
5:30 – 5:45 p.m.

428 – Generation of DNA Plasmid-Encoded Neutralizing Monoclonal Antibodies In Vivo
6:00 – 8:00 p.m.

433 – DNA Monoclonal Antibodies Target Influenza Virus In Vivo
6:00 – 8:00 p.m.

435 – Skin Delivery of a RSV Vaccine with Surface Electroporation Provides Full Protection from Lower Respiratory Disease in the Cotton Rat
6:00 – 8:00 p.m.

436 – Developing a Synthetic DNA Vaccine for an Emerging Pathogen – Middle East Respiratory Syndrome
6:00 – 8:00 p.m.

440 – Extreme Polyvalency Induces Potent Cross-Clade Cellular and Humoral Responses in Rabbits and Non-Human Primates
6:00 – 8:00 p.m.

401 – In Vivo Expression of Plasmid Encoded IgG for PD-1 or LAG3 by Synthetic DNA as a New Tool for Cancer Immunotherapy
6:00 – 8:00 p.m.

May 6

479 – An Optimized DNA Vaccine Formulation Protects Against Lethal Ebola Makona Virus Challenge in Non-Human Primates and Elicits Robust Immune Responses
2:50 – 3:10 p.m.

505 – VGX-3100 Drives Regression of HPV16/18 CIN2/3 and Robust Cellular Immune Responses in Blood and Cervical Tissue in a Blinded, Randomized, Placebo-Controlled Phase 2B Study
4:00 – 4:15 p.m.

643 – Various Forms of CD40L Encoded as an Immune Plasmid Adjuvant Generate Unique Anti-Cancer DNA Vaccine Induced Responses
6:00 – 8:00 p.m.

About the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper)

ASGCT is the primary professional membership organization for gene and cell therapy. The Society’s members are scientists, physicians, patient advocates, and other professionals. Its members work in a wide range of settings including universities, hospitals, government agencies, foundations, biotechnology and pharmaceutical companies. Its mission is to advance knowledge, awareness, and education leading to the discovery and clinical application of gene and cell therapies to alleviate human disease.

On May 4, 2016 Yissum Research Development Company of the Hebrew University of Jerusalem reported that it had signed an exclusive world-wide licensing and research agreement with BioTheryX, Inc., developer of novel protein degradation and immunomodulatory drugs for cancer and immune dysfunction, for the development and commercialization of drug candidates representing first-in-class therapy for both hematologic and solid malignancies. Financial terms of the license were not disclosed (Press release, , MAY 4, 2016, View Source [SID1234641528]).

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The novel technology was invented by Yinon Ben-Neriah, MD, PhD, Blumenthal Professor of Cancer Research, Lautenberg Center for Immunology, Hebrew University-Hadassah Medical School, with generous support by AMRF (The Dr. Miriam and Sheldon G. Adelson Medical Research Foundation). Dr. Ben-Neriah’s and his team showed that inhibition of the clinically validated enzyme CKI-alpha, induces several tumor suppressor pathways, including a new type of DNA damage response and p53 activation. This provides a novel approach to treat a wide range of cancers, in particular selective types of hematological malignancies.

Prof. Yinon Ben-Neriah has recently received the 2016 Rappaport Prize for excellence in biomedical research, among others, for his ground breaking research on the relationship between chronic inflammation and cancer and the treatment of leukemia.

Based on their complementing expertise, BioTheryX and Yissum have agreed to join forces and focus on the selection and advancement of clinical candidates designed to inhibit CKI-alpha. Initial clinical focus for these candidates will be selective subtypes of myelodysplastic syndrome and acute myeloid leukemia that are not responsive to available cancer therapy.

In preclinical studies of acute leukemia, these clinical candidates showed a far greater therapeutic potential than any previously reported studies. Treatment of genetically-modified leukemic mice modeling poor-prognosis human acute myeloid leukemia, abolished the disease signs in the majority of the animals, without compromising the normal bone marrow, demonstrating that CKI-alpha inhibitors have a large therapeutic window and are unique in their capacity to specifically eliminate leukemia stem cells, including otherwise treatment-resistant stem cells – a strong indication of cancer cure.

Yaacov Michlin, President and Chief Executive Officer, Yissum, commented, "The treatment that was developed in Prof. Ben-Neriah’s lab is very different from other available therapies, both in its mechanism of action and its ability to eliminate leukemic stem cells, and thus in its therapeutic potential. In light of the successful pre-clinical studies, we believe that it offers a significant breakthrough, and we are very pleased to partner with BioTheryX in the development of new drug candidates for potential treatment of a variety of hematological indications. We believe that the combined know-how and research efforts of the teams at BioTheryX and the Hebrew University will facilitate new drug development, leading to significant advancement in the therapy of this class of devastating cancer diseases."

David Stirling, Chief Executive Officer, BioTheryX, commented, "BioTheryX is particularly pleased to partner with the Hebrew University on this important project. Our team, having developed the remarkable IMiD family of drugs while at Celgene, which have improved the quality of life of so many cancer patients and their loved ones, brings a deep wealth of experience to developing novel cancer therapies that modulate protein degradation and the immune system to target cancer causing proteins for destruction. We believe that these unique drug candidates from the Hebrew University will not only bring new treatment modalities to a variety of hematological cancers, but may provide the only option for those patients that relapse and become resistant to currently available therapies."