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Halozyme Reports First Quarter 2015 Financial Results

On May 11, 2015 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results for the first quarter ended March 31, 2015 (Press release, Halozyme, MAY 11, 2015, View Source [SID:1234506620]). Financial highlights include revenues of $18.7 million and a net loss of $15.1 million, or $0.12 per share, compared to revenues of $12.0 million and a net loss of $26.5 million, or $0.22 per share, for the first quarter of 2014.

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"We continued to execute well in the first quarter against a focused strategy in oncology, ramping investments in our core program and achieving significant milestones toward the initiation of a Phase 3 study in pancreatic cancer patients early next year," said Dr. Helen Torley, president and chief executive officer. "In addition to this strategic progress, we exited the quarter in a much stronger financial position than last year due to a steady increase in royalty revenues from our rHuPH20 platform."

First Quarter 2015 Highlights and Subsequent Events

Announced plans to initiate a Phase 3 clinical study (Study 301) in metastatic pancreatic cancer patients with high-hyaluronan (HA) tumors: Based on feedback following a Type B meeting with the FDA, the company intends to initiate a Phase 3 study that will allow for a potential marketing application based on either progression free survival (PFS) or overall survival. Use of PFS as the basis for marketing approval will be subject to the overall benefit and risk associated with Halozyme’s investigational new drug, PEGPH20, combined with nab-paclitaxel (ABRAXANE) and gemcitabine therapy, including the:
Magnitude of the PFS treatment effect observed;
Toxicity profile; and
Interim overall survival.

The company plans to use a companion diagnostic test to prospectively identify and select patients with high levels of HA for its Phase 3 trial. The FDA provided feedback supporting the selection of high-HA patients and confirmed that an Investigational Device Exemption – or IDE – will be required prior to initiating the Phase 3 study. An IDE is a regulatory application that summarizes the methodology, validation and proposed cut-point for patient selection. The company plans to submit the IDE to support the late Q1 2016 Study 301 start.

Presented in January the interim results of Study 202 evaluating PEGPH20 with gemcitabine and ABRAXANE (nab-paclitaxel) in metastatic pancreatic cancer patients: In a retrospectively defined sub-population of patients, the data showed a statistically significant doubling in median PFS in metastatic pancreatic cancer patients with high levels of HA who were treated with PEGPH20 combined with nab-paclitaxel (ABRAXANE) and gemcitabine (9.2 months vs. 4.3 months in patients treated with nab-paclitaxel ABRAXANE and gemcitabine alone). The potential risk profile, including rate of thromboembolic events, was also evaluated.

In April, the company also announced plans to present the interim results of the study during an oral presentation by Dr. Sunil Hingorani, a principal investigator, at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).

Initiated enrollment in a Phase 1b/2 clinical trial of investigational PEGPH20 in non-small cell lung cancer (NSCLC): The company began enrollment in the Phase 1b/2 randomized clinical trial (PRIMAL) of PEGPH20 in combination with docetaxel as a second-line therapy for patients with locally advanced and metastatic NSCLC.

Published preclinical study results of PEGPH20 in Molecular Cancer Therapeutics (Singha et al., February 2015 14:523-532): Data highlights the use and potential of PEGPH20 to enhance the anti-cancer activity of the accompanying immunotherapy (trastuzumab and immune cells) in high-HA breast and ovarian cancer preclinical models. The paper expands on previously reported data in a poster presentation at the ASCO (Free ASCO Whitepaper) 2015 Gastrointestinal Cancers Symposium in San Francisco. The manuscript was written and funded by Halozyme.

Daratumumab selected as the first product candidate under Janssen collaboration: In March, Genmab A/S announced plans for a Phase 1 clinical trial of a subcutaneous formulation of the anti-CD38 antibody daratumumab using the ENHANZE technology. Daratumumab is being developed under a collaboration between Janssen and Genmab A/S since August 2012 when Genmab granted Janssen an exclusive worldwide license to develop, manufacture, and commercialize daratumumab. Daratumumab, a human monoclonal antibody that targets CD38, is in clinical development as a single agent and in combination with standard of care therapies in several settings of multiple myeloma.

First Quarter 2015 Financial Highlights

Revenues for the first quarter of 2015 were $18.7 million, compared to $12.0 million for the first quarter of 2014. Revenues in the first quarter included $6.8 million in royalty revenue from sales of products under collaboration agreements, $6.1 million in product sales of bulk rHuPH20 for use in manufacturing collaboration products for Roche, $3.8 million in Hylenex recombinant (hyaluronidase human injection) product sales, and $2.0 million in collaboration revenues. Royalty revenues represent October to December 2014 sales as a result of the one quarter lag in royalty reports.

Research and development expenses for the first quarter of 2015 were $16.7 million, compared to $21.4 million for the first quarter of 2014. The decrease was primarily due to a planned decrease in expenses associated with the diabetes program.

Selling, general and administrative expenses for the first quarter of 2015 were $9.4 million, compared to $10.3 million for the first quarter of 2014. The decrease was primarily due to a decrease in compensation expenses.

The net loss for the first quarter of 2015 was $15.1 million, or $0.12 per share, compared to a net loss for the first quarter of 2014 of $26.5 million, or $0.22 per share.

Cash, cash equivalents and marketable securities were $128.5 million at March 31, 2015, compared to $135.6 million at December 31, 2014. Net cash used in the first quarter of 2015 was approximately $7.1 million.
Financial Outlook for 2015

For the full year 2015, the company reiterated its previously disclosed guidance of:

Net revenues to be in the range of $85 million to $95 million.
Operating expenses to be in the range of $145 million to $155 million.
Net cash burn to be between $35 million to $45 million.

OPKO Announces First Quarter Operating and Financial Results

On May 11, 2015 OPKO Health, Inc. (NYSE:OPK), a multi-national biopharmaceutical and diagnostics company, reported operating and financial results for its first quarter ended March 31, 2015 (Filing, Q1, Opko Health, MAY 11, 2015, View Source [SID:1234506585]).

Business Highlights

Pfizer Collaboration Agreement for Long Acting Human Growth Hormone Closed in January 2015; OPKO Received Up-Front Payments Totaling $295 million for Global Commercialization Rights to hGH-CTP: In connection with the collaboration, OPKO received up-front payments of $295 million and will receive an additional $275 million upon achievement of development-related milestones. In addition, OPKO will receive initial royalty payments upon the commercialization of hGH-CTP for Adult growth hormone deficiency (GHD). Upon the launch of hGH-CTP for Pediatric GHD, the royalties will transition to gross profit sharing among all indications for both hGH-CTP and Pfizer’s Genotropin. OPKO will lead clinical development and will be responsible for funding the development programs for Adult and Pediatric GHD and growth failure in children born small for gestational age (SGA). Pfizer will be responsible for all development costs for additional indications as well as all post-marketing studies. In addition, Pfizer will fund the commercialization activities for all indications and lead the manufacturing activities covered by the global development plan.

hGH-CTP for Pediatric GHD Data Presented at 2015 ENDO; Adult Phase 3 Clinical Trial Continues to Advance: OPKO presented positive twelve-month data from its ongoing Phase 2 clinical trial for pediatric GHD at the 97th Annual Meeting of the Endocrine Society (ENDO) on March 5th, 2015 in San Diego, California.

Acquired EirGen Pharma; A Growing, Profitable Specialty Pharmaceutical Developer and Manufacturer: OPKO acquired EirGen Pharma, Ltd., a growing, profitable and cash flow positive specialty pharmaceutical company focused on the development and commercial supply of high potency, high barrier to entry pharmaceutical products for sale in the U.S., Canada, Japan, Australia, most European countries, and more than 40 others around the world. EirGen, based in Waterford, Ireland, was founded by two former executives of IVAX Pharmaceuticals, Tom Brennan and Patsy Carney. The company, situated in a state of the art high containment research and development and manufacturing facility, is approved by the FDA, EMEA (European Health Authorities) and the PMDA (Japanese Health Authorities). To date, EirGen and its commercial partners have filed 10 product applications with the FDA and 5 each in Europe and Japan. EirGen has a strong research and development portfolio of over 20 niche, high barrier to entry drugs. EirGen will rapidly expand its drug portfolio with access to additional capital from OPKO, together with the benefits of Irish government programs encouraging research and development in Ireland. EirGen also offers significant synergies and benefits to OPKO through its ability to manufacture OPKO’s current and future products.

Rayaldee Met Primary Endpoints in Both Pivotal Phase 3 Trials; NDA Submission Scheduled Later in May: OPKO announced successful top-line results from both of its pivotal Phase 3 trials with Rayaldee in September 2014. These trials were identical randomized, double-blind, placebo-controlled, multi-site studies intended to establish the safety and efficacy of Rayaldee as a new treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency. OPKO has scheduled submission of NDA to the FDA later in May.

Positive Rayaldee Results Presented at the National Kidney Foundation Spring Clinical Meetings and 2015 ENDO Conference: The presentations of positive Rayaldee Phase 3 trial data were entitled "Modified-Release Calcifediol Is Effective for SHPT in Both Stages 3 and 4 CKD" (March 2015 in Dallas TX) and "Modified-Release Calcifediol Controls Elevated iPTH, Corrects 25(OH)D Levels and Reduces Bone Markers in CKD Patients" (March 2015 in San Diego, CA).

IND for Long Acting Factor VIIa-CTP for Hemophilia Filed and Accepted: In March 2015, the FDA accepted OPKO’s IND application to Initiate a Phase 2a Trial for its Long-Acting Coagulation Factor VIIa-CTP to Treat Hemophilia. Clinical Trials are expected to commence shortly.

Clinical Studies for Long Acting Oxyntomodulin for Obesity and Diabetes Expected to Begin During 2015; OPKO expects to commence studies for its long acting Oxyntomodulin for diabetes and obesity in the second half of 2015.

Launched 4Kscore Test in Mexico and Expects Further Latin American Launches in 2015; Adoption of 4Kscore Test Continues to Grow in the U.S. and Europe: OPKO launched the 4Kscore Test in Mexico in January 2015. OPKO also expects to launch the 4Kscore Test in additional Latin America markets through its subsidiaries during 2015. OPKO is working to obtain reimbursement for the 4Kscore Test by payers in the U.S. and abroad and expects adoption to rapidly increase once reimbursement is received.
Announced Publication of a Study Entitled "Predicting High-Grade Cancer at Ten-Core Prostate Biopsy Using Four Kallikrein Markers Measured in Blood in the ProtecT Study.": The ProtecT study is a prospective randomized controlled trial conducted in the UK for the purpose of evaluating the cost effectiveness of conventional treatments in PSA-detected, clinically localized prostate cancer. Of the 82,428 men recruited for the trial, a total of 6,129 men with elevated PSA (≥3.0 ng/mL) who underwent prostate biopsy and provided an adequate blood sample were tested for the four kallikreins and their 4Kscore results were determined. The study showed that the four kallikrein panel enhanced aggressive prostate cancer detection compared with PSA and age alone. The area under the curve (AUC) for the 4K model was 0.820 (95% CI = 0.802 to 0.838) while the PSA model was 0.738 (95% CI = 0.716 to 0.761) for high-grade cancer. The ProtecT study is the latest in a series of peer-reviewed publications demonstrating the superior clinical value that testing with four kallikrein markers adds to risk prediction for aggressive prostate cancer.

Rolapitant NDA Filing Submitted in September and Accepted for Review by FDA in November: OPKO’s partner, TESARO, submitted a NDA to the FDA for approval of oral rolapitant, an investigational neurokinin-1 (NK-1) receptor antagonist in development for the prevention of chemotherapy-induced nausea and vomiting (CINV). The NDA is supported by data from four controlled studies covering a spectrum of patients receiving chemotherapy that commonly causes nausea and vomiting. The top-line results of three of the Phase 3 studies were previously announced by TESARO and were presented in detail at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June 2014. On November 5, 2014, TESARO announced the FDA accepted its NDA filing for rolapitant, with a PDUFA date of September 5, 2015. Upon approval and following commercialization, OPKO will receive up to $110 Million in additional milestones and tiered, double digit royalties.

"We began 2015 with a number of significant activities including the closing of the Pfizer transaction, the initiation of our clinical development program for Factor VIIa-CTP and the imminent submission of our NDA for Rayaldee," said Phillip Frost, M.D., Chairman and CEO. "The 4Kscore Test continues to gain acceptance in the US, Mexico and Europe and we are pleased with the recently published studies further supporting the long-term outcomes of using this potentially revolutionary blood test. With the EirGen acquisition, we added a growing, profitable, cash flow positive business to our complement of operating companies which we believe will lead to further growth and profitability of our other businesses," Dr. Frost continued.

Financial Highlights

Cash, cash equivalents and marketable securities were $348.2 million as of March 31, 2015.
Reflects receipt of Pfizer upfront payments of $295.0 million, partially offset by a non-recurring $25.9 million payment to the Office of the Chief Scientist in Israel related to the Pfizer transaction.

Consolidated revenues increased to $30.1 million during the three months ended March 31, 2015 from $22.3 million in the comparable period of 2014. Revenue for the three months ended March 31, 2015 included $12.5 million of revenue resulting from OPKO’s collaboration agreement with Pfizer. OPKO is recording revenue in connection with the Pfizer transaction on a straight-line basis over the expected development period.

Net loss for the three months ended March 31, 2015 included increased operating expenses including a $4.5 million increase in research and development expense to $25.5 million, and non-recurring and non-cash charges, such as:
$25.9 million of non-recurring operating expense related to the repayment of a grant to the Office of the Chief Scientist in Israel related to the Pfizer transaction; and
$49.8 million in non-cash charges related to the change in value of embedded derivatives which are part of our January 2013 convertible senior notes due in 2033 (the "2033 Senior Notes"). This non-cash charge is principally a result of the increased market price of our common stock since December 31, 2014.

CEL-SCI RECEIVES AUTHORIZATION TO CONDUCT ITS PHASE 3 MULTIKINE TRIAL IN SPAIN

On May 11, 2015 CEL-SCI Corporation (NYSE MKT: CVM)("CEL SCI" or the "Company") reported that Spain’s Agency for Medicinal Products and Medicinal Devices has authorized the Company to commence patient enrollment for its ongoing Phase 3 trial of its investigational immunotherapy Multikine* (Leukocyte Interleukin, Injection) in patients with advanced primary squamous cell carcinoma of the oral cavity/soft palate, a type of head and neck cancer. Spain is the 22nd country to authorize CEL-SCI’s Phase 3 trial for patient enrollment (Press release, Cel-Sci, MAY 11, 2015, View Source [SID:1234506570]).

CEL-SCI recently announced it had reached a key milestone of receiving authorization to conduct the Phase 3 study from 21 countries, as originally planned. The Company is aiming to expand the trial into a total of approximately 100 clinical centers in about 25 countries. As of April 30, 2015, 437 patients had been enrolled in the global Phase 3 study.

About the Multikine Phase 3 Study

The Multikine Phase 3 study is enrolling patients with advanced primary squamous cell carcinoma of the head and neck. The objective of the study is to demonstrate a statistically significant improvement in the overall survival of enrolled patients who are treated with the Multikine treatment regimen plus standard of care ("SOC") vs. subjects who are treated with SOC only.

About Multikine

Multikine (Leukocyte Interleukin, Injection) is an investigational immunotherapeutic agent that is being tested in an open-label, randomized, controlled, global pivotal Phase 3 clinical trial as a potential first-line treatment for advanced primary squamous cell carcinoma of the head and neck. Multikine is designed to be a different type of therapy in the fight against cancer: one that appears to have the potential to work with the body’s natural immune system in the fight against tumors.

Multikine is also being tested in a Phase 1 study under a Cooperative Research and Development Agreement ("CRADA") with the U.S. Naval Medical Center, San Diego, as a potential treatment for peri-anal warts in HIV/HPV co-infected men and women. CEL-SCI has also entered into two co-development agreements with Ergomed Clinical Research Limited to further the development of Multikine for cervical dysplasia/neoplasia in women who are co-infected with HIV and HPV and for peri-anal warts in men and women who are co-infected with HIV and HPV.

Medigene announces milestone payment in phase I/II trial with dendritic cell (DC) vaccines for AML

On May 11, 2015 Medigene AG (MDG1, Frankfurt, Prime Standard) reported that the progress achieved in its recently initiated clinical phase I/II trial with dendritic cell (DC) vaccines for the treatment of acute myeloid leukaemia (AML) gives rise to a milestone payment amounting to EUR 700,000 to former contributing shareholders of Medigene Immunotherapies GmbH (formerly: Trianta Immunotherapies GmbH) to be made within the next five months (Press release, MediGene, MAY 11, 2015, View Source [SID:1234506569]). Medigene intends to settle this payment through the issuance of new shares from authorised capital. The number of new shares will be calculated based on the average value of Medigene’s share price over the 30 days both before and from today’s announcement. The milestone payment was an agreed part of the purchase price in the acquisition of Trianta in January 2014.

Medigene’s phase I/II multi-centre, open-label trial will include a total of 20 AML patients after completion of standard chemotherapy to reduce the risk of recurrence of leukaemia by using Medigene’s DC vaccines. To date, the first patients have been enrolled and a number of additional patients have been identified for inclusion in the trial. The primary objective is to prove feasibility and safety of active immunotherapy with Medigene’s DCs. Secondary objectives are induction of immune responses, control of minimal residual disease (MRD) and clinical response/time to progression (TTP).

Dr Frank Mathias, Chief Executive Officer (CEO) of Medigene AG, comments: "Reaching this milestone is once again proof of the progress Medigene has made since acquiring Trianta. The start and continuation of the clinical phase I/II trial enables us to further validate our new generation of DC vaccines in the clinic and will therefore contribute to the intrinsic value of this technology and our company."

Further studies utilizing Medigene’s DC vaccine technologies include two ongoing clinical investigator-initiated trials (IITs), a clinical phase II trial (prostate cancer) at Oslo University Hospital and a clinical phase I/II trial (AML) at the Ludwig-Maximilians University Hospital Großhadern, Munich, as well as a compassionate use programme[1] including patients with diverse malignancies.

About Medigene’s DC vaccines: The platform for the development of antigen-tailored DC vaccines is the most advanced platform of the three highly innovative and complementary immunotherapy platforms of Medigene Immunotherapies.

Medigene’s dendritic cell product platform allows the design of new generation dendritic cell vaccines. Dendritic cells can take up antigens efficiently, process them and present them on their surface in a form that can induce antigen-specific T cells to proliferate and mature. This way T cells can recognize and eliminate antigen-bearing tumour cells. Dendritic cells can also induce natural killer cells (NK cells) to become active and attack tumour cells. Scientists of Medigene Immunotherapies have developed new, fast and efficient methods for preparing autologous (patient-specific) mature dendritic cells which have relevant characteristics to activate both T cells and NK cells. The dendritic cells can be loaded with various tumour antigens to treat different types of cancer and are designed for treatment of minimal residual disease or use in combination therapies.

Further audio-visual education about Medigene’s DCs at: View Source

CEL-SCI RECEIVES AUTHORIZATION TO CONDUCT ITS PHASE 3 MULTIKINE TRIAL IN ITALY

On May 11, 2015 CEL-SCI Corporation (NYSE MKT: CVM)("CEL SCI" or the "Company") reported that the Italian Medicines Agency (AIFA) has authorized the Company to commence patient enrollment for its ongoing Phase 3 trial of its investigational immunotherapy Multikine* (Leukocyte Interleukin, Injection) in patients with advanced primary squamous cell carcinoma of the oral cavity/soft palate, a type of head and neck cancer (Press release, Cel-Sci, MAY 11, 2015, View Source [SID:1234506568]). Italy is the 23rd country to authorize CEL-SCI’s Phase 3 trial for patient enrollment.

Having surpassed its originally planned milestone of receiving authorization to conduct the Phase 3 study from 21 countries, CEL-SCI is now aiming to expand the trial into a total of approximately 100 clinical centers in about 25 countries. As of April 30, 2015, 437 patients had been enrolled in the global Phase 3 study.