On February 9, 2016 CEL-SCI Corporation (NYSE MKT: CVM) reported financial results for the quarter ended December 31, 2015 (Press release, Cel-Sci, FEB 9, 2016, View Source [SID:1234509023]).

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Key corporate and clinical developments during first quarter fiscal year 2016 include:

Enrolled an additional 98 patients in the global pivotal Phase 3 head and neck cancer trial. As of January 31, 2016, 697 patients have been enrolled in the study.

Raised net proceeds of approximately $10.6 million to finance the Phase 3 head and neck cancer trial.
Increased Ergomed’s investment in our Phase 3 head and neck cancer trial. Ergomed added another $2 million to its existing $10 million investment for a total investment of $12 million. Ergomed will receive its funds back only from future sales of Multikine* (Leukocyte Interleukin, Injection).

Secured litigation funding for our ongoing arbitration against the former CRO of our Phase 3 trial from Lake Whillans, a firm that specializes in litigation funding. Pursuant to the agreement, CEL-SCI will receive up to $5.0 million in funding and Lake Whillans will get its funds back only from the proceeds derived from the arbitration.

Granted a new European patent on Multikine. The patent is important because it recites a mechanism of action of Multikine by which Multikine changes the type of T cells that enter the tumor microenvironment. This change results in the tumor becoming "visible" to the immune system, resulting in a robust and sustainable anti-tumor immune response.

Received acceptance into a new program for technology commercialization and niche analysis of our LEAPS rheumatoid arthritis vaccine candidate from the U.S. National Institutes of Health (NIH).

Continued patient enrollment in the Phase 1 trial of Multikine in HIV/HPV co-infected men and women with peri-anal warts at San Diego Naval Medical Center and University of California, San Francisco (UCSF). The study in expected to be complete in the second half of 2016.

"During the first quarter of fiscal 2016 we received funding from two strategic sources, Ergomed and Lake Whillans, which we believe affirm the strength of both our Phase 3 trial and our arbitration claims against the former CRO. We are confident in our partners as we move towards the completion of enrollment in our Phase 3 trial," stated CEL-SCI Chief Executive Officer Geert Kersten.

CEL-SCI reported an operating loss of ($5,783,132) for the quarter ended December 31, 2015 versus an operating loss of ($9,995,741) for the quarter ended December 31, 2014. Research and development expenses remained relatively consistent and increased by approximately $286,000 compared to the three months ended December 31, 2014. General and administrative expenses decreased by approximately $4,560,000 compared to the three months ended December 31, 2014. Major components of the decrease include approximately $2,296,000 in a gain on the derecognition of legal fees recognized pursuant to the agreement with Lake Whillans offset by approximately $316,000 net increase in other general and administrative expenses. Also during the quarter ended December 31, 2014, there was approximately $2,620,000 in additional employee compensation costs related to the issuance of shareholder approved shares of restricted stock released upon meeting predetermined milestones.

CEL-SCI’s net income (loss) available to common shareholders for the quarter ended December 31, 2015 was $2,341,813 or $0.02 per basic share, versus ($7,845,318) or ($0.11) per basic share during the quarter ended December 31, 2014. The income was primarily attributable to an unrealized gain on the fair value of warrants, as a result of the change in the stock price between reporting periods.

About Multikine

Multikine 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.

On February 9, 2016 Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, reported its fourth quarter and full year 2015 financial results and accomplishments(Press release, Sangamo BioSciences, FEB 9, 2016, View Source [SID:1234509022]).

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"2015 was an important and very productive year for Sangamo, and we enter 2016 poised to initiate the first human clinical trials of in vivo therapeutic genome editing," said Edward Lanphier, Sangamo’s president and chief executive officer. "Our zinc finger nuclease (ZFN) technology leads the therapeutic genome editing field and we have established the core competencies necessary to move our ground-breaking genome editing programs through IND enabling studies and into clinical trials. We believe our IVPRP clinical studies will provide fundamental proof-of-concept data and significantly differentiate the technical advantages of our ZFN platform from other genome editing technologies of bacterial origin as well as conventional gene therapy approaches. In addition to our two new open IVPRP INDs we plan to file six more IND applications in 2016 for our other IVPRP-based programs, and our hemoglobinopathies programs which we are developing in collaboration with Biogen. We began the year with approximately $210 million in cash, which puts Sangamo in a strong financial position and will allow us to accomplish all of our goals in 2016."

Recent Highlights

Announcement of FDA clearance of IND application for Phase 1/2 clinical trial of MPS I (Hurler syndrome) program. In February 2016, Sangamo announced that its Investigational New Drug (IND) application for the Company’s SB-318 program was cleared by the U.S. Food and Drug Administration (FDA) and is now active. SB-318 is an application of the Company’s proprietary In Vivo Protein Replacement Platform (IVPRP) genome editing approach, for the treatment of MPS I. In December 2015, the NIH Recombinant DNA Advisory Committee (RAC) unanimously approved the clinical protocol for SB-318.
Announcement of FDA clearance of IND application for Phase 1/2 clinical trial of hemophilia B program. In December 2015, Sangamo announced that an IND application for SB-FIX, the Company’s IVPRP genome editing approach for the potential cure of hemophilia B, has been cleared by the FDA and is now active.

Presentation of Phase 2 clinical data from SB-728-T HIV studies demonstrating superiority of adenoviral delivery of zinc finger nucleases to T-cells for viral load control and reservoir reduction. In December 2015, Sangamo presented Phase 2 clinical data from ongoing clinical trials of the Company’s SB-728-T HIV program, SB-728-1101 Cohort 3* and SB-728mR-1401. The preliminary comparative data suggest that adenoviral delivery of ZFNs to T-cells may be uniquely immune-stimulatory for both acute viral load control and HIV reservoir reduction. The trial is currently ongoing with the accrual of five additional subjects in ‘1101 Cohort 3*.
Presentation of data at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) meeting (ASH) (Free ASH Whitepaper) highlighting ZFP Therapeutic programs for hemophilia and hemoglobinopathies. In December 2015, Sangamo presented data at ASH (Free ASH Whitepaper) demonstrating the production of therapeutic levels of Factor IX (FIX) clotting protein in non-human primates (NHPs) from its hemophilia B program, and clinical scale manufacturing and engraftment of ZFN-modified hematopoietic stem and progenitor cells (HSPCs) for the treatment of beta-thalassemia.

Publication of improved method for efficient targeted integration in HSPCs and T-cells. In November 2015, Sangamo announced the publication in Nature Biotechnology of data demonstrating efficient ZFN-mediated, targeted gene insertion in HSPCs, as well as a study in Nucleic Acids Research, demonstrating a similarly efficient process in primary human T-cells.
Internal Organization. Sangamo promoted Stewart Craig, Ph.D., from Vice President to Senior Vice President of Technical Operations. Dr. Craig joined Sangamo in May 2014 and has led the development of the Company’s successful and growing manufacturing capabilities. Fyodor Urnov, Ph.D., Senior Scientist, was promoted to Vice President of Discovery & Translational Research. Dr. Urnov is a key contributor to the development of Sangamo’s ZFP Therapeutic technology platform and leads Sangamo’s hemoglobinopathies research collaboration with Biogen Inc. (Biogen). Nathalie Dubois-Stringfellow, Ph.D. was promoted from Senior Director to Vice President of Product Development & Management. Dr. Dubois-Stringfellow, with extensive experience in pre-clinical drug development and project management, established an effective cross-functional team-based culture at Sangamo, enabling the Company’s successful and timely IND submissions.

Upcoming Events in the First Half of 2016

Initiation of Phase 1/2 clinical trials for IVPRP-based SB-FIX-1501 (hemophilia B) and SB-318-1502 (MPS I / Hurler syndrome) programs. The trials will be the first two in vivo clinical studies of genome editing in humans and the first clinical programs based on Sangamo’s IVPRP approach. Sangamo expects to initiate the Phase 1/2 trial for hemophilia B in the first half of 2016, and the Phase 1/2 trial for MPS I in mid-2016.

Presentation of clinical data from Sangamo’s HIV program at the 2016 Annual Conference on Retroviruses and Opportunistic Infections (CROI). Sangamo’s collaborator, Rafick Pierre Sekaly, Ph.D., will present further immunologic and viral reservoir analyses of clinical data from the Company’s SB-728-1101 study, suggesting potential mechanisms of viral control post-treatment with SB-728-T.

Preclinical data presentation from Sangamo’s MPS I and MPS II programs at the 2016 Annual WORLDSymposium Meeting. Sangamo expects to present data from its animal model studies for the Company’s IVPRP-based MPS I and MPS II (Hunter syndrome) programs for lysosomal storage disorders (LSDs). The meeting is being held in San Diego, CA from February 29 to March 4, 2016.
Submission of IND applications for Sangamo’s SB-913 (MPS II) program and beta-thalassemia program. Sangamo expects to file both IND applications in the first half of 2016. SB-913, for the treatment of MPS II, is the second LSD application of the Company’s proprietary IVPRP approach. The beta-thalassemia program, which is being developed in collaboration with Biogen, employs Sangamo’s ZFN-mediated ex vivo genome editing approach to knockout the BCL11A Enhancer.

Fourth Quarter 2015 Results

For the fourth quarter ended December 31, 2015, Sangamo reported a consolidated net loss of $14.0 million, or $0.20 per share, compared to a net loss of $4.3 million, or $0.06 per share, for the same period in 2014. As of December 31, 2015, the Company had cash, cash equivalents, marketable securities and interest receivable of $209.3 million.

Revenues for the fourth quarter of 2015 were $9.1 million, compared to $15.0 million for the same period in 2014. Fourth quarter 2015 revenues were generated from the Company’s collaboration agreements with Biogen, Shire International GmbH (Shire), and Dow AgroSciences, enabling technology agreements and research grants. The revenues recognized for the fourth quarter of 2015 consisted of $9.0 million in collaboration agreements and approximately $0.2 million in research grants, compared to $14.5 million and approximately $0.4 million, respectively, for the same period in 2014.

The decrease in collaboration agreement revenues was primarily a result of an amendment to the Company’s collaboration and license agreement with Shire in the third quarter of 2015, returning the rights to the hemophilia programs to Sangamo. In the fourth quarter of 2015, Sangamo recognized $1.9 million of revenues related to research services performed under the collaboration agreement with Shire, and $1.9 million of revenues related to research services performed under the collaboration agreement with Biogen. In addition, pursuant to the agreements entered into with Shire in January 2012 and Biogen in January 2014, Sangamo received upfront payments of $13.0 million and $20.0 million, respectively. These payments are being recognized as revenue on a straight-line basis over the initial six-year research term for Shire and approximately 40 months for Biogen. The Company recognized $0.5 million of the Shire upfront payment and $1.6 million of the Biogen upfront payment as revenue for the fourth quarter of 2015.

Research and development expenses were $19.9 million for the fourth quarter of 2015, compared to $15.1 million for the same period in 2014. The increase was primarily due to increases in manufacturing expenses, external research expenses associated with our preclinical programs, and personnel-related expenses, including stock-based compensation. General and administrative expenses were $4.9 million for the fourth quarter of 2015, compared to $4.3 million for the same period in 2014.

Total operating expenses for the fourth quarter of 2015 were $24.8 million, compared to $19.4 million for the same period in 2014.

Full Year 2015 Results

For the year ended December 31, 2015, the consolidated net loss was $40.7 million, or $0.58 per share, compared to a consolidated net loss of $26.4 million, or $0.39 per share, for the year ended December 31, 2014. Revenues were $39.5 million for the year ended December 31, 2015, compared to $45.9 million for the same period in 2014. Total operating expenses were $86.4 million for the year ended December 31, 2015, compared to $72.7 million for the same period in 2014.

Financial Guidance for 2016

Cash and Investments: Sangamo expects that its cash, cash equivalents and marketable securities will be at least $150 million at the end of 2016, inclusive of research funding from existing collaborators but exclusive of funds arising from any additional new collaborations or partnerships, equity financings or other new sources.
Revenues: In light of the amendment to our collaboration and licensing agreement with Shire, that returned the rights of the hemophilia programs to Sangamo, the Company expects that revenues will be in the range of $20 million to $25 million in 2016, inclusive of research funding from existing collaborations.
Operating Expenses: Sangamo expects that operating expenses will be in the range of $85 million to $95 million for 2016.

On February 9, 2016 OPKO Health, Inc. (NYSE: OPK) reported the results of a study for the 4Kscore Test’s clinical utility in reducing the number of prostate biopsies performed, while increasing the probability of detecting aggressive prostate cancer in men with abnormal prostate-specific antigen (PSA) levels and or digital rectal examination (DRE) results (Press release, Opko Health, FEB 9, 2016, View Source [SID:1234509014]). The peer-reviewed study, "The 4Kscore Test Reduces Prostate Biopsy Rates in Community and Academic Urology Practices", written by Badrinath Konety, MD, et al. and published in the January 2016 edition of Reviews in Urology, a MedReviews, LLC. Publication, which included 611 patients seen by 35 academic and community urologists across the United States, indicated that consideration of results from the 4Kscore tests led to 64.6% fewer prostate biopsies being performed among participating patients.

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"The 4Kscore test is a useful tool in identifying men with a significant risk of having an aggressive form of prostate cancer, who are most likely to benefit from a prostate biopsy and selective treatment or intensive intervention, while avoiding biopsies in men who are at low risk for developing aggressive disease," commented lead researcher Badrinath Konety, M.D., Dougherty Family Chair in Uro-Oncology and director of the Institute for Prostate and Urologic Cancers at the University of Minnesota. "Our findings suggest that PSA screening, when coupled with the 4Kscore test, can be made more specific, reduce biopsy complications and overtreatment, and be a more cost-effective solution for managing a patient’s prostate health."

Dr. Konety and colleagues evaluated the influence of the 4Kscore test on urologist-patient decisions about whether to perform a biopsy in men who had an abnormal PSA and or DRE result. Test results for patients were stratified into low risk ( < 7.5%), intermediate risk (7.5%-19.9%) and high risk (≥20%) for developing aggressive prostate cancer. Nearly half (49.3%) of the men were categorized as low risk; 25.7% and 25.0% fell into the intermediate-risk and high-risk categories, respectively. Notably, the 4Kscore test results influenced biopsy decisions in 88.7% of the men. In the three risk groups, a biopsy was avoided in 94.0%, 52.9%, and 19.0% of men in the low, intermediate, and high-risk categories, respectively.

A higher 4Kscore test result was significantly associated with a greater likelihood of having a prostate biopsy (P < 0.001). Among the 171 men who had a biopsy, 45 of the 104 cases (43.3%) with a high-risk 4Kscore test result (≥20% risk) were found to have aggressive prostate cancer upon prostate biopsy.

About the 4Kscore Test

The 4Kscore is the only blood test that accurately identifies an individual patient’s risk for aggressive prostate cancer, the lethal form of prostate cancer. The 4Kscore test uses a proprietary algorithm that incorporates the blood levels of four different prostate-derived kallikrein proteins: Total PSA, Free PSA, Intact PSA and Human Kallikrein-2 (hK2), plus the patient’s age, and other clinical information to calculate the percentage risk (probability) of finding a Gleason Score 7 or higher grade of prostate cancer. The four kallikrein panel of biomarkers utilized in the 4Kscore Test is based on over a decade of research conducted by scientists at Memorial Sloan-Kettering Cancer Center and leading European institutions and is included as a standard of care in the 2015 NCCN Prostate Cancer Early Detection Guidelines. The 4Kscore test provides individualized risk for the presence of aggressive prostate cancer and adds new information to the shared decision making discussion between the Urologist and the patient.

On February 09, 2016 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported that the updated American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Clinical Practice Guideline on the appropriate use of breast tumor biomarker assay results recommends the use of the Prosigna Breast Cancer Prognostic Gene Signature Assay (PAM50) to guide decisions on adjuvant systemic therapy for women with early-stage invasive breast cancer with known hormone receptor and HER2 status (Press release, NanoString Technologies, FEB 9, 2016, View Source [SID:1234509010]). The Prosigna assay was one of only two such assays to receive a "high" rating of evidence quality together with a "strong" recommendation.
"Prosigna’s inclusion in the ASCO (Free ASCO Whitepaper) Clinical Practice Guideline, specifically for use in guiding decisions on adjuvant systemic therapy for women with early stage breast cancer, places Prosigna on equal footing with the Oncotype DX test," said Brad Gray, President and Chief Executive Officer of NanoString Technologies. "This is the sixth major breast cancer treatment guideline to include Prosigna in the past twelve months, and it further strengthens our case for reimbursement and market adoption."

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The ASCO (Free ASCO Whitepaper) Clinical Practice Guideline provides evidence-based recommendations based on a comprehensive review and analyses of the relevant literature for each recommendation. The Guideline states that if a patient has hormone receptor-positive, HER2-negative (node-negative) breast cancer, the clinician may use the PAM50 (Prosigna) risk of recurrence (ROR) score, known as the Prosigna Score in the United States, in conjunction with other clinicopathologic variables, to guide decisions on adjuvant systemic therapy.

"We applaud the rigorous scientific methodology and transparency that the ASCO (Free ASCO Whitepaper) multidisciplinary expert breast cancer panel applied in reviewing and weighing the available scientific evidence," said Alessandra Cesano, M.D., Ph.D., Chief Medical Officer of NanoString. "By focusing on the clinical utility of sparing women unnecessary chemotherapy, the Committee has helped future breast cancer patients avoid the risk of fatal, life-threatening, or permanently changing toxicities."

The Guideline, together with additional information including details of the methodology and review of the scientific literature, is available at: View Source

About the Prosigna Breast Cancer Prognostic Gene Signature Assay and nCounter Dx Analysis System
The Prosigna Assay provides a risk category and numerical score for assessment of the risk of distant recurrence of disease at 10 years in postmenopausal women with node-negative (Stage I or II) or node-positive (Stage II), hormone receptor-positive (HR+) breast cancer. Based on the PAM50 gene signature initially discovered by Charles Perou, Ph.D. and colleagues, the Prosigna Assay is an in vitro diagnostic tool that utilizes gene expression data weighted together with clinical variables to generate a risk category and numerical score to assess a patient’s risk of distant recurrence of disease. The Prosigna Assay measures gene expression levels of RNA extracted from formalin-fixed paraffin embedded (FFPE) breast tumor tissue previously diagnosed as invasive breast carcinoma.

The Prosigna Assay requires minimal hands-on time and runs on NanoString’s proprietary nCounter Dx Analysis System, which offers a reproducible and cost-effective way to profile many genes simultaneously with high sensitivity and precision.

The nCounter Dx Analysis System is a highly automated and easy-to-use platform that utilizes a novel digital barcoding chemistry to deliver high precision multiplexed assays. The system is available in the multi-mode FLEX configuration, which is designed to meet the needs of high-complexity clinical laboratories seeking a single platform with the flexibility to run the Prosigna Breast Cancer Assay and, when operated in the "Life Sciences" mode, process translational research experiments and multiplexed assays developed by the laboratory.

In the United States, the Prosigna Assay is available for diagnostic use when ordered by a physician. The Prosigna Assay has been CE-marked and is available for use by healthcare professionals in the European Union and other countries that recognize the CE Mark, as well as Canada, Israel, Australia, New Zealand and Hong Kong.

In the U.S., the Prosigna Assay is indicated in female breast cancer patients who have undergone surgery in conjunction with locoregional treatment consistent with standard of care, either as:
(1) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-negative, Stage I or II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors or (2) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-positive (1-3 nodes), Stage II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors. The device is not intended for patients with four or more positive nodes.

For more information, please visit www.prosigna.com.