On May 4, 2016 Nordic Nanovector ASA (OSE: NANO), a biotechnology company focusing on the development of novel targeted therapeutics in haematology and oncology, reported that the first patient has been enrolled into one of the two new arms of its expanded Lymrit 37-01 clinical study with Betalutin (Press release, Nordic Nanovector, MAY 4, 2016, View Source [SID:1234511950]).

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Betalutin is a novel anti-CD37 targeting Antibody Radionuclide Conjugate in development for the treatment of major types of non-Hodgkin’s lymphoma (NHL), including Follicular Lymphoma (FL).

The new arm (Arm 3) is designed to investigate the safety and efficacy of Betalutin in up to 12 patients with relapsed FL pre-dosed with standard anti-CD20 immunotherapy (rituximab) on Day 0, a few hours prior to the administration of Betalutin.

Luigi Costa, Nordic Nanovector CEO, commented: "We are pleased to initiate the first of the two new cohorts in our ongoing clinical study, which represents a significant step forward in Betalutin’s development plan. Data we have seen to date suggest that we can achieve strong clinical efficacy with a regimen that controls haematological side effects. The two new arms are investigating if different pre-dosing regimens will allow the use of higher doses of Betalutin to potentially achieve even higher efficacy than that so far observed, and therefore an even more compelling product profile."

The Lymrit 37-01 study is a Phase 1/2 open label, single injection ascending dose study investigating three dose levels of Betalutin and different dosing regimens in patients with relapsed NHL with the aim of identifying an optimal dose regimen to take into the Phase 2 PARADIGME study, which is expected to start in 2H 2017.

Patient recruitment into the Phase 2 part of Arm 1 (15Mbq/kg plus 50mg/ml unconjugated "cold" HH1 anti-CD37 antibody) is progressing as planned with dose-escalation expected to begin in 2H 2016. Patient screening is also underway for the final arm in the expanded Lymrit 37-01 study (Arm 4), in which escalating doses of Betalutin plus pre-treatment with a higher dose of cold anti-CD37 antibody than in Arm 1 will be evaluated in relapsed FL patients.

A decision to increase the dose of Betalutin to 17.5 MBq/kg in Arm 1 can be made based on the evaluation of the safety and efficacy data observed in the 15 patients treated with 15 MBq/kg. A decision to increase the dose of Betalutin to 17.5 MBq/kg or 20 MBq/kg in one or the other of Arms 3 and 4 can be made based on the evaluation of the safety and efficacy data observed in the first three patients of both cohorts.

Data and analysis recently published at the American Association of Cancer Research annual meeting (16-20 April) confirmed that Betalutin was generally well tolerated and showed a 63.2% Overall Response Rate (ORR) and a 31.6% Complete Response (CR) in evaluable patients. Clinical responses observed were sustained, with Duration of Response exceeding 12 months in most responders in the 15 MBq/kg group who have been followed up for at least 12 months.

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."

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:

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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.

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.