May 2016 – Page 86 – 1stOncology™: Cancer Intelligence Service

Inovio Pharmaceuticals Showcases its Broad Pipeline of DNA-based Immunotherapies, Vaccines and dMAbs at the American Society of Gene & Cell Therapy

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.

8-K – Current report

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."

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.

ArQule Reports First Quarter 2016 Financial Results

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]).

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.

Juniper Pharmaceuticals Reports First Quarter 2016 Financial Results

On May 4, 2016 Juniper Pharmaceuticals, Inc. (Nasdaq: JNP) ("Juniper" or the "Company"), a women’s health therapeutics company, reported financial results for the three-month period ended March 31, 2016 (Press release, Juniper Pharmaceuticals, MAY 4, 2016, View Source;p=RssLanding&cat=news&id=2164732 [SID:1234511899]). Highlights include:

Phase 2b trial of COL-1077 10% lidocaine vaginal gel in women undergoing a minimally invasive pipelle-directed endometrial biopsy on track for Q3 2016 data readout;
Pre-IND meeting with the U.S. Food and Drug Administration (FDA) confirmed development pathway for JNP-0101, an oxybutynin intra-vaginal ring (IVR) for the treatment of overactive bladder in women;
Management team, Board of Directors, and Scientific Advisory Board (SAB) strengthened with several key additions;
Product revenue increased 63% and service revenue increased 32% versus 2015; and,
Balance sheet remains strong.

Juniper Pharmaceuticals, Inc. (PRNewsFoto/Juniper Pharmaceuticals, Inc.)
"This was an exceptionally strong quarter for Juniper, with advances in our pipeline of proprietary products supported by solid revenue growth from both our product and service businesses," stated Frank Condella, Chief Executive Officer. "We continue to deliver on our strategy of growing our core business revenues and advancing our product pipeline through clinical development to create long-term shareholder value.

"We look forward to reporting results of our Phase 2b trial of COL-1077 this summer. There are over seven million minimally-invasive gynecologic procedures performed annually in the U.S. alone, and currently available analgesics do not adequately address the pain and cramping experienced by these women. Assuming positive results of the current study, we expect to initiate a pivotal Phase 3 trial of COL-1077 in 2017.

"During the quarter we completed a collaborative pre-IND meeting with the FDA for our first IVR program, JNP-0101 for the treatment of overactive bladder in women. We believe our oxybutynin IVR holds great potential to effectively treat this common condition while improving systemic side effects and health outcomes with improved compliance. We look forward to filing our IND application later this year, and plan to initiate a Phase 2 bioavailability and dose finding study once the IND is active," Condella concluded.

First Quarter Financial Results
First quarter total revenues increased 45% to $12.1 million, compared with $8.3 million for the quarter ended March 31, 2015.

Product revenues were $7.9 million, an increase of $3.1 million, or 63%, versus the first quarter of last year, driven by continued in-market growth and new market sales of CRINONE (progesterone gel) by Merck KGaA, Darmstadt, Germany.

Service revenues from Juniper Pharma Services were $3.3 million, an increase of $0.8 million, or 32%, versus the first quarter of last year, as we experienced strong growth in customer volumes. Royalty revenues, based on Allergan’s sales of CRINONE, were $0.9 million, a 9% decrease versus the first quarter of last year.

Gross profit increased to $5.8 million as compared with $3.4 million in the prior year quarter.

Total operating expenses were $5.5 million in the first quarter of 2016, a $1.2 million increase as compared to the prior year quarter.

The $0.9 million increase in general and administrative costs as compared to the prior year quarter was primarily driven by creation of an internal business development function that was not in place in 2015, in addition to non-recurring legal and professional service costs.

The $0.4 million increase in R&D spending as compared to the prior year quarter was driven by costs associated with our ongoing Phase 2b clinical trial of COL-1077 and the advancement of our IVR pipeline product candidates: JNP-0101 (oxybutynin IVR), JNP-0201 (estrogen + progesterone IVR for symptoms of menopause), and JNP-0301 (progesterone IVR for the prevention of preterm birth).

Juniper recorded net income of $0.4 million, or $0.03 per diluted share, in the first quarter of 2016, compared to a net loss of $0.7 million, or ($0.06) per diluted share, in the same period of 2015.

Additional Business Highlights

Herman Weiss, MD, MBA, FACOG, was appointed Vice President, Medical Affairs and Clinical Development. He was previously Global Medical Director of Women’s Health and Bone Health at Teva Pharmaceutical Industries, Ltd.
Mary Ann Gray, Ph.D., joined Juniper’s Board of Directors as Audit Committee Chair. Dr. Gray’s 20+ years in the biotechnology and biopharmaceutical industry includes Wall Street, financial, and scientific experience.
Prominent physicians Linda Giudice, MD, PhD, and Marianne Mann, MD, joined Juniper’s Scientific Advisory Board.
Liquidity
Cash and cash equivalents were $13.5 million as of March 31, 2016, versus $13.9 million at December 31, 2015. The decrease in cash and cash equivalents was primarily the result of capital expenditures and the timing of certain payments related to revenues recorded in the current quarter.

Conference Call
As previously announced, Juniper’s management will hold a conference call to discuss financial results for the first quarter ended March 31, 2016, as follows:

Date:
Wednesday, May 4, 2016
Time:
8:30 a.m. EDT
Dial-in numbers:
Toll free: (866) 374-4635 (U.S.), (855) 669-9657 (Canada), or

International: (412) 902-4218
Webcast (live & archive):
www.juniperpharma.com, under ‘Investors’ or click here
The teleconference replay will be available at approximately one hour after completion through Thursday, May 12, 2016, at (877) 344-7529 (U.S.), (855) 669-9658 (Canada) or (412) 317-0088 (International). The conference ID for the replay is 10083822.

The archived webcast will be available for one year via the aforementioned URLs.

The antibodies against 5-bromo-2′-deoxyuridine specifically recognize trifluridine incorporated into DNA.

Trifluridine (FTD) is a key component of the novel oral antitumor drug TAS-102 (also named TFTD), which consists of FTD and a thymidine phosphorylase inhibitor. FTD is supposed to exert its cytotoxicity via massive misincorporation into DNA, but the underlying mechanism of FTD incorporation into DNA and its correlation with cytotoxicity are not fully understood. The present study shows that several antibodies against 5-bromo-2′-deoxyuridine (BrdU) specifically cross-react with FTD, either anchored to bovine serum albumin or incorporated into DNA. These antibodies are useful for several biological applications, such as fluorescence-activated cell sorting, fluorescent immunostaining and immunogold detection for electron microscopy. These techniques confirmed that FTD is mainly incorporated in the nucleus during S phase in a concentration-dependent manner. In addition, FTD was also detected by immunohistochemical staining in paraffin-embedded HCT-116 xenograft tumors after intraperitoneal administration of FTD. Intriguingly, FTD was hardly detected in surrounding matrices, which consisted of fibroblasts with marginal expression of the nucleoside transporter genes SLC29A1 and SLC29A2. Thus, applications using anti-BrdU antibodies will provide powerful tools to unveil the underlying mechanism of FTD action and to predict or evaluate the efficacy and adverse effects of TAS-102 clinically.

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Month: May 2016

Inovio Pharmaceuticals Showcases its Broad Pipeline of DNA-based Immunotherapies, Vaccines and dMAbs at the American Society of Gene & Cell Therapy

8-K – Current report

ArQule Reports First Quarter 2016 Financial Results

Juniper Pharmaceuticals Reports First Quarter 2016 Financial Results

The antibodies against 5-bromo-2′-deoxyuridine specifically recognize trifluridine incorporated into DNA.