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SRI International Receives $19.8 Million National Cancer Institute Contract for PREVENT Cancer Program

On March 10, 2016 SRI International reported that it has been awarded a contract of up to $19.8 million from the National Cancer Institute (NCI) PREVENT Cancer Preclinical Drug Development Program to support the development of potential cancer preventive agents or vaccines (Press release, SRI International, MAR 4, 2016, View Source [SID:1234509760]). Under the contract, SRI Biosciences, a division of SRI International, will conduct preclinical studies to assess the efficacy of specific compounds or vaccines for preventing invasive-cancer development. Researchers will also identify biomarkers to help quantify the effectiveness of the experimental compounds and vaccines.

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The PREVENT Cancer Drug Development Program is an NCI-supported pipeline to bring new cancer PREVENTing interventions and biomarkers through preclinical development towards clinical trials. PREVENT enables the milestone-driven progression of novel cancer PREVENTive chemical or biological agents (singly or in combination), and biomarkers from the lab bench towards proof-of principle preclinical and clinical testing and registration or validation.

"The discovery and development of cancer preventative agents is an area that is underserved, primarily because the length of required clinical trials can be resource-prohibitive for many companies. Our work in biomarker discovery may provide validated surrogate endpoints that can help to shorten clinical trials in cancer prevention," said Lidia Sambucetti, Ph.D., senior director of Cancer Biology, Center for Discovery Technologies, SRI Biosciences, and principal investigator for the NCI contract. "The PREVENT program is an opportunity to identify and advance novel strategies for cancer prevention."

Under the three-year contract, SRI Biosciences will conduct detailed preclinical pharmacological studies to determine the efficacy of experimental agents, as well as test biomarkers that may parallel the effectiveness of response to these agents.

Under the PREVENT program, for efficacy and biomarker testing, two NCI task orders in the area of cancer prevention have already been awarded to SRI Biosciences: The first to develop a mesothelin-based vaccine against ovarian cancer, and another to develop novel models for testing preventative agents against ovarian cancer.

For the ovarian cancer program, SRI Biosciences optimized a vaccine strategy designed to mount both antibody-based and cellular immunity against mesothelin tumor antigen. SRI researchers are currently testing whether the vaccine can help prevent ovarian cancer. In addition, SRI generated encouraging data supporting the development of a new model that will be used to test experimental drugs for ovarian cancer prevention.

"Efforts toward cancer prevention are the best long-term investments to protect the health of society as a whole while also reducing the healthcare costs," said Nathalie Scholler, M.D., Ph.D., director Cancer Immunology, Center for Cancer, SRI Biosciences and lead on the ovarian cancer program.

SRI has broad experience with similar studies for NCI and for many other divisions of the National Institutes of Health, as well as for private sponsors.

This project has been funded in whole or in part with Federal funds from the National Cancer Institute (NCI), National Institutes of Health, Department of Health and Human Services, under Contract No. HHSN261201500041I & HHSN261201200014I"

The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. – See more at: View Source#sthash.Gwxi65Wc.dpuf

3DMed and Alphamab Start Joint Development of China PD-L1 Cancer Drug

3DMed, a Shanghai biotech, and Suzhou’s Alphamab have decided to join forces to develop KN035, a PD-L1 immuno-oncology drug discovered by Alphamab (Article, ChinaBio, MAR 4, 2016, View Source [SID1234523603]). Unlike most PD-L1 mAbs, KN035 is an injected product that the companies hope will be more effective and cause fewer side effects than the competition. 3DMed and Alphamab will develop KN035 equally. They expect to file for China clinical trial approval in Q2 and begin the trials before the end of the year, with US trials following later. Further details of the agreement were not disclosed.

U.S. FDA Approves IMBRUVICA® (ibrutinib) for First-line Treatment of Chronic Lymphocytic Leukemia

On March 4, 2016 The U.S. Food and Drug Administration (FDA) reported that it has approved IMBRUVICA (ibrutinib) capsules for treatment-naïve patients with chronic lymphocytic leukemia (CLL) (Press release, Johnson & Johnson, MAR 4, 2016, View Source [SID:1234509383]).1

The approval is based on data from the Phase 3 RESONATE-2 (PCYC-1115) study, the first head-to-head clinical trial comparing IMBRUVICA to a chemotherapy agent. Results showed IMBRUVICA significantly extended progression-free survival (PFS; the primary endpoint) and increased overall response rate (ORR; a key secondary endpoint) compared to chlorambucil in previously untreated patients with CLL age 65 or older. IMBRUVICA is now approved for use in all lines of CLL therapy, considerably expanding the number of patients who may benefit from this treatment. This broadens the indication beyond the initial CLL approval in February 2014 for the treatment of patients with CLL who have received at least one prior therapy and in July 2014 for CLL patients with del 17p,1 a genetic mutation typically associated with poor treatment outcomes.2 IMBRUVICA is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company.

On a related front, the National Comprehensive Cancer Network (NCCN) published an update on February 17 to its Clinical Practice Guidelines for non-Hodgkin’s lymphomas recommending IMBRUVICA for certain first-line CLL patients.

"People living with CLL who have not been previously treated now have an option that significantly improved progression-free survival when compared to the oral chemotherapy used in the RESONATE-2 trial," said Jan Burger, M.D., Ph.D., Associate Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX and RESONATE-2 study lead investigator. "The results seen in the RESONATE-2 clinical trial are truly compelling and make this medicine an attractive first-line treatment option for clinicians in the hematology space."

The expanded IMBRUVICA indication is based on data from the Phase 3, randomized, open-label RESONATE-2 trial, which showed IMBRUVICA significantly improved PFS and ORR versus chlorambucil in treatment-naïve patients aged 65 or older with CLL or small lymphocytic lymphoma (SLL). The PFS as assessed by an Independent Review Committee (IRC) according to the clarified International Workshop on Chronic Lymphocytic Leukemia (iWCLL) criteria indicated an 84 percent statistically significant reduction in the risk of death or progression in the IMBRUVICA arm versus the chlorambucil arm (HR=0.161 [95 percent CI, 0.091-0.283]). Median PFS was not reached for IMBRUVICA versus 18.9 months for chlorambucil (95 percent CI: 14.1, 22.0). Data from RESONATE-2 were presented in an oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 7, 2015, in addition to being featured in the official ASH (Free ASH Whitepaper) press program and simultaneously published online in The New England Journal of Medicine.

CLL is a slow-growing blood cancer that most commonly arises from B cells, a type of white blood cell (lymphocyte) that originates in the bone marrow.3,4 CLL is predominantly a disease of the elderly, with a median age of 71 at diagnosis.5

"The IMBRUVICA story gets better and better. The results of RESONATE-2 demonstrate how IMBRUVICA can change the treatment strategies for many patients with CLL in the treatment-naïve setting. We anticipate this approval will give clinicians and more of their patients the opportunity to explore the efficacy and safety of treatment with IMBRUVICA for this disease," said Peter F. Lebowitz, M.D., Ph.D., Global Oncology Head, Janssen Research & Development, LLC.

Janssen and Pharmacyclics continue to support an extensive clinical development program for IMBRUVICA, including 16 Phase 3 study commitments in multiple patient populations.

IMBRUVICA in First-line, Elderly CLL Patients
The safety and efficacy of IMBRUVICA were evaluated in the randomized, international, multi-center, open-label Phase 3 RESONATE-2 trial in 269 treatment-naïve patients with CLL/SLL* aged 65 years or older. Patients were randomized to receive either IMBRUVICA 420 mg orally, once daily until progression or unacceptable toxicity or chlorambucil 0.5 to 0.8 mg/kg on days 1 and 15 of each 28-day cycle for up to 12 cycles, with an allowance for intrapatient dose increases up to 0.8mg/kg based on tolerability.

The primary endpoint of the study was met, with IMBRUVICA demonstrating a longer PFS versus chlorambucil as determined by the IRC per clarified iWCLL criteria. The hazard ratio was 0.161 (95 percent CI, 0.091-0.283, P<0.0001), which represents a reduction of risk of progression or death by 84 percent versus chlorambucil (median PFS not reached for IMBRUVICA vs. 18.9 months for chlorambucil [95 percent CI: 14.1, 22.0]); IMBRUVICA was associated with a significantly higher ORR (a composite of complete and partial responses [82.4 percent vs. 35.3 percent; P<0.0001]) as assessed by the IRC per modified iWCLL criteria. Notably, five patients (3.7 percent) in the IMBRUVICA arm and two patients (1.5 percent) in the chlorambucil arm achieved a complete response.

The safety of IMBRUVICA in this patient population was consistent with previously reported studies. The adverse reactions (AR) reported in the U.S. Prescribing Information reflect exposure to IMBRUVICA with a median duration of 17.4 months versus a median exposure to chlorambucil of 7.1 months: nearly 2.5 times longer exposure for IMBRUVICA. Warnings and Precautions include hemorrhage, infections, cytopenias, atrial fibrillation, hypertension, second primary malignancies, tumor lysis syndrome and embryo-fetal toxicity. The most common ARs (≥20 percent) of any Grade in the RESONATE-2 trial for IMBRUVICA were diarrhea (42 percent), musculoskeletal pain** (36 percent), cough (22 percent) and rash** (21 percent). The most common Grade 3/4 AR (≥five percent) was pneumonia** (eight percent). Four to 10 percent of patients receiving IMBRUVICA in the studies supporting the CLL indications (PCYC-1102, RESONATE [PCYC-1112] and RESONATE-2) discontinued treatment due to ARs. These included pneumonia, subdural hematomas and atrial fibrillation (one percent each). ARs leading to dose reduction occurred in approximately four percent of patients.

*IMBRUVICA is not approved by the FDA to treat SLL.

**includes multiple ADR terms

About IMBRUVICA
IMBRUVICA was one of the first therapies to receive U.S. approval after having received the FDA’s Breakthrough Therapy Designation. IMBRUVICA works by blocking a specific protein called Bruton’s tyrosine kinase (BTK).1 The BTK protein transmits important signals that tell B cells to mature and produce antibodies and is needed by specific cancer cells to multiply and spread.1,6 IMBRUVICA targets and blocks BTK, inhibiting cancer cell survival and spread.1 For more information, visit www.IMBRUVICA.com.

Access to IMBRUVICA
Janssen and AbbVie are striving to make access to IMBRUVICA easy by helping patients understand their insurance benefits for IMBRUVICA. The YOU&i Support Program is a personalized program that includes information on access and affordability, nurse call support and resources for patients being treated with IMBRUVICA. This includes the YOU&i Instant Savings program, which provides co-pay support to eligible commercially insured IMBRUVICA patients. This program is not valid for patients with Medicare or Medicaid. Patients can access the program by contacting 1-877-877-3536, option 1 or by visiting www.IMBRUVICA.com .

IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Hemorrhage – Fatal bleeding events have occurred in patients treated with IMBRUVICA. Grade 3 or higher bleeding events (intracranial hemorrhage [including subdural hematoma], gastrointestinal bleeding, hematuria, and post-procedural hemorrhage) have occurred in up to 6% of patients. Bleeding events of any grade, including bruising and petechiae, occurred in approximately half of patients treated with IMBRUVICA.

The mechanism for the bleeding events is not well understood. IMBRUVICA may increase the risk of hemorrhage in patients receiving antiplatelet or anticoagulant therapies and patients should be monitored for signs of bleeding. Consider the benefit-risk of withholding IMBRUVICA for at least 3 to 7 days pre- and postsurgery depending on the type of surgery and the risk of bleeding.

Infections – Fatal and nonfatal infections have occurred with IMBRUVICA therapy. Grade 3 or greater infections occurred in 14% to 26% of patients. Cases of progressive multifocal leukoencephalopathy (PML) have occurred in patients treated with IMBRUVICA. Evaluate patients for fever and infections and treat appropriately.

Cytopenias – Treatment-emergent Grade 3 or 4 cytopenias including neutropenia (range, 19% to 29%), thrombocytopenia (range, 5% to 17%), and anemia (range, 0% to 9%) occurred in patients treated with IMBRUVICA. Monitor complete blood counts monthly.

Atrial Fibrillation – Atrial fibrillation and atrial flutter (range, 6% to 9%) have occurred in patients treated with IMBRUVICA, particularly in patients with cardiac risk factors, hypertension, acute infections, and a previous history of atrial fibrillation. Periodically monitor patients clinically for atrial fibrillation. Patients who develop arrhythmic symptoms (eg, palpitations, lightheadedness) or new-onset dyspnea should have an ECG performed. Atrial fibrillation should be managed appropriately and if it persists, consider the risks and benefits of IMBRUVICA treatment and dose modification.

Hypertension – Hypertension (range, 6% to 17%) has occurred in patients treated with IMBRUVICA with a median time to onset of 4.5 months (range, 0.03 to 18.40 months). Monitor patients for new-onset hypertension or hypertension that is not adequately controlled after starting IMBRUVICA. Adjust existing antihypertensive medications and/or initiate antihypertensive treatment as appropriate.

Second Primary Malignancies – Other malignancies (range, 5% to 16%) including non-skin carcinomas (range, 1% to 4%) have occurred in patients treated with IMBRUVICA. The most frequent second primary malignancy was non-melanoma skin cancer (range, 4% to 13%).

Tumor Lysis Syndrome – Tumor lysis syndrome has been infrequently reported with IMBRUVICA therapy. Assess the baseline risk (eg, high tumor burden) and take appropriate precautions. Monitor patients closely and treat as appropriate.

Embryo-Fetal Toxicity – Based on findings in animals, IMBRUVICA can cause fetal harm when administered to a pregnant woman. Advise women to avoid becoming pregnant while taking IMBRUVICA and for 1 month after cessation of therapy. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

ADVERSE REACTIONS
The most common adverse reactions (≥20%) in patients with B-cell malignancies (MCL, CLL, WM) were thrombocytopenia* (57%, 53%, 43%), diarrhea (51%, 48%, 37%), anemia* (41%, 37%, 13%), neutropenia* (47%, 46%, 44%), musculoskeletal pain (37%, 32%†, NA‡), fatigue (41%, 29%, 21%), bruising (30%, 25%†, 16%†), nausea (31%, 24%, 21%), rash (25%, 23%†, 22%†), and upper respiratory tract infection (34%, 19%, 19%).

*Based on adverse reactions and/or laboratory measurements (noted as platelets, neutrophils, or hemoglobin decreased).
†Includes multiple ADR terms.
‡Not applicable; no associated ADRs.

The most common Grade 3 or 4 non-hematologic adverse reactions (≥5%) in MCL patients were pneumonia (7%), abdominal pain (5%), atrial fibrillation (5%), diarrhea (5%), fatigue (5%), and skin infections (5%).

Approximately 4% (CLL), 14% (MCL), and 11% (WM) of patients had a dose reduction due to adverse reactions.

Approximately 4-10% (CLL), 9% (MCL), and 6% (WM) of patients discontinued due to adverse reactions. Most frequent adverse events leading to discontinuation were pneumonia, subdural hematomas, and atrial fibrillation (1% each) in CLL patients and subdural hematoma (1.8%) in MCL patients.

DRUG INTERACTIONS
CYP3A Inhibitors – Avoid coadministration with strong and moderate CYP3A inhibitors. If a moderate CYP3A inhibitor must be used, reduce the IMBRUVICA dose.

CYP3A Inducers – Avoid coadministration with strong CYP3A inducers.

SPECIFIC POPULATIONS
Hepatic Impairment – Avoid use in patients with moderate or severe baseline hepatic impairment. In patients with mild impairment, reduce IMBRUVICA dose.

Please see full Prescribing Information: View Source

ProNAi Reports 2015 Year-End Financial and Operational Results

On March 3, 2016 ProNAi Therapeutics, Inc. (NASDAQ: DNAI), a clinical-stage oncology company advancing novel therapeutics for patients with cancer and hematological diseases, reported its financial and operational results for the year ended December 31, 2015 (Press release, ProNAi Therapeutics, MAR 3, 2016, View Source [SID:1234509951]).

"During 2015, we continued to transform ProNAi into a world-class oncology drug development company, securing both the talent and capital required to pursue our vision of developing and commercializing a pipeline of promising clinical-stage oncology assets with the potential to provide meaningful therapeutic outcomes to patients with cancer," said Dr. Nick Glover, President and CEO of ProNAi Therapeutics. "Concurrent with building our company, we continued to advance our lead asset PNT2258, operationalizing two Phase 2 trials in 2015, Wolverine and Brighton, that are at the forefront of a concerted registration-oriented clinical development program planned for the drug. In addition to PNT2258, during 2015 we began evaluating novel product candidates available for licensing or acquisition, with the goal of maximizing our clinical development capabilities and leveraging the full potential of our team by advancing a broad and diversified pipeline of assets."

"We anticipate reporting initial interim data from the Wolverine trial in third-line DLBCL in the second quarter of 2016. This trial has been designed to identify and characterize patient populations who respond to PNT2258 on the basis of their genetics and disease characteristics and will be essential to determining potential paths to registration for the drug," added Dr. Glover. "We recently started enrolling the Brighton trial in Richter’s transformation and expect to report interim data from this trial before the end of 2016. We are also designing a number of additional Phase 2 trials that could support the registration and commercialization strategies for PNT2258. Two planned trials, Cypress and Granite, will evaluate PNT2258 in combination with standard-of-care treatment regimens for the treatment of second-line DLBCL in the "transplant eligible" and "transplant ineligible" patient populations respectively. We are also designing trials evaluating PNT2258’s potential in DLBCL in combination with a targeted anti-cancer drug, and in other hematological malignancies as well. Although we maintain a strong balance sheet, we are carefully considering the timing of these additional trials with a view to maximizing PNT2258’s potential while deploying our capital prudently."

2015 Operational Highlights:

Operationalized the Wolverine Phase 2 trial evaluating single-agent PNT2258 in third-line relapsed or refractory DLBCL, opening more than 20 clinical sites during 2015 that are now actively recruiting patients.

Initiated the Brighton Phase 2 trial evaluating single-agent PNT2258 in Richter’s transformed CLL.

Completed a successful IPO in July 2015, raising more than $158 million in gross proceeds. ProNAi shares began trading on the NASDAQ Global Market on July 16, 2015 under the symbol "DNAI."

Strengthened the ProNAi management team, appointing six additional experienced senior executives including Dr. Barbara Klencke, M.D., as Chief Development Officer. Dr. Klencke is an accomplished oncology drug developer with a demonstrable track record of success, having made substantial contributions to the development and approval of several important oncology products including Kyprolis, Kadcyla, Avastin and Tarceva.

Expanded the company from approximately 25 employees to more than 50 employees and opened our corporate headquarters in Vancouver, Canada.

ProNAi (NASDAQ:DNAI) was selected for addition to the NASDAQ Biotechnology Index (NASDAQ:NBI)
2015 Financial Results (all amounts reported in U.S. currency)

Total operating expenses for the year ended December 31, 2015 were $35.8 million compared to $22.6 million for the year ended December 31, 2014. Total operating expenses include non-cash stock based compensation of $3.2 million and $0.3 million for the years ended December 31, 2015 and 2014, respectively.

Research and development expenses increased to $26.4 million for the year ended December 31, 2015 from $19.1 million for the year ended December 31, 2014. These increases were primarily due to expenses related to the continuation of our PNT2258 clinical trials, the manufacture of PNT2258 and an increase in personnel-related costs. Expenses for the year ended December 31, 2014 include a one-time $11.0 million milestone payment that was made to Novosom AG during the second quarter of 2014.

General and administrative expenses increased to $9.5 million for the year ended December 31, 2015 from $3.5 million for the year ended December 31, 2014. These increases were primarily due to increased personnel-related costs and professional fees incurred in support of activities as a public company and corporate growth.

For the year ended December 31, 2015, ProNAi incurred a net loss of $53.3 million compared to a net loss of $23.9 million for the year ended December 31, 2014. The net loss includes a non-cash charge related to the change in fair value of preferred stock warrants of $17.4 million and $1.4 million for the years ended December 31, 2015 and 2014, respectively.

At December 31, 2015, ProNAi had $150.2 million in cash and cash equivalents compared to $39.2 million in cash, cash equivalents and short-term investments at December 31, 2014.

At December 31, 2015, there were approximately 30,058,105 shares of common stock issued and outstanding and 3,522,813 options issued and outstanding.

8-K – Current report

On March 3, 2016 Fate Therapeutics, Inc. (NASDAQ: FATE), a biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported business highlights and financial results for the fourth quarter and full year ended December 31, 2015 (Filing, Q4/Annual, Fate Therapeutics, 2015, MAR 3, 2016, View Source [SID:1234509372]).

"During this past year, we significantly advanced our long-term corporate strategy, successfully building a ground-breaking preclinical pipeline of programmed adoptive immunotherapies, including a NK-cell therapy for solid tumors, a CD34+ cell therapy for autoimmune diseases and off-the-shelf cancer therapies derived from engineered pluripotent cell lines. We also formed a strategic research collaboration with Juno Therapeutics, leveraging our expertise in hematopoietic cell biology and ex vivo cell programming, to enhance the therapeutic function of Juno’s engineered T-cell immunotherapies," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We have continued this momentum into 2016, having secured FDA clearance to conduct a Phase 1/2 clinical trial of ProTmune, and we look forward to safety and efficacy data from this trial during 2016. Finally, we will begin sharing data across our entire preclinical pipeline at industry-leading scientific conferences during the first half of 2016."
Recent Highlights

• FDA Clearance of IND Application for ProTmune Phase 1/2 Study. In January 2016, Fate Therapeutics announced that its Investigational New Drug (IND) application for ProTmune (FT1050-FT4145 programmed mobilized peripheral blood cells) was cleared by the U.S. Food and Drug Administration (FDA). The Company expects to commence enrollment of a multi-center, randomized, controlled Phase 1/2 clinical trial of ProTmune for the prevention of acute graft-versus-host disease (GvHD) and cytomegalovirus (CMV) infection in mid-2016, both of which are severe life-threatening immunological conditions with no approved FDA therapies.

• ProTmune Preclinical Data Highlighted at 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting (ASH) (Free ASH Whitepaper) and 2016 BMT Tandem Meetings. Fate Therapeutics presented ProTmune preclinical data demonstrating that FT1050-FT4145 programmed immune cells reduce acute GvHD and retain anti-tumor, or graft-versus-leukemia (GvL), activity in vivo. Acute GvHD is a leading cause of morbidity and mortality in patients undergoing allogeneic HCT, and therapeutic strategies aimed at addressing GvHD that suppress or deplete the immune system can compromise or eliminate T cells, often leading to severe infections and disease relapse. GvL activity is critical to eradicating residual cancer and realizing the curative potential of allogeneic HCT.
• Pluripotent Cell Platform for Off-the-Shelf NK- and T-Cell Cancer Immunotherapies Highlighted at 2015 ASH (Free ASH Whitepaper) Meeting. Fate Therapeutics presented the Company’s patent-protected, pluripotent cell platform, which combines genetic engineering of pluripotent cells with rapid and efficient generation of immune cells, for developing off-the-shelf engineered cancer immunotherapies without requiring patient-sourced cells. Highlighted features of this platform include the precise integration of multiple genetic modifications into pluripotent cells, the efficient expansion of engineered pluripotent cell clones, and the derivation of CD34+ cells, NK cells and T cells using well-defined, small molecule-driven protocols.

• Patent Position Covering Pluripotent Cell Platform Significantly Expanded. In 2015, Fate Therapeutics and its exclusive licensors were granted 21 patents covering induced pluripotent cell technology, extending its formidable position to include over 60 issued patents and 90 pending patent applications. Most notably, in October 2015, the U.S. Patent and Trademark Office issued U.S. Patent No. 9,169,490 providing broad protection for cell compositions expressing a sufficient amount of octamer-binding transcription factor 4 (Oct4) to enable pluripotency. The production of Oct4 protein within a cell is critical to generate highly-stable, genetically-modified, clonal pluripotent cell lines for use in the unlimited production of off-the-shelf engineered cell therapies.

• NK- and CD34+ Cell Immunotherapy Collaborations Formed with Leading Medical Institutions. In July 2015, Fate Therapeutics entered into a multi-year agreement with the University of Minnesota, under which the Company is advancing toward clinical development an Adaptive NK-cell cancer immunotherapy for solid tumors in collaboration with Dr. Jeffrey Miller, M.D., Professor of Medicine and Deputy Director, University of Minnesota Cancer Center. Additionally, in June 2015, the Company entered into a multi-year research agreement with Boston Children’s Hospital to develop an immuno-regulatory CD34+ cell therapy, which is currently being assessed in several preclinical models of T cell-mediated immune dysfunction.

• Juno Therapeutics Strategic Research Collaboration Formed to Program T-Cell Immunotherapies. In May 2015, the Company entered into a research collaboration and license agreement with Juno Therapeutics, Inc. to identify small molecule modulators that enhance the therapeutic function of genetically-engineered chimeric antigen receptor (CAR) T-cell and T-cell receptor (TCR) immunotherapies. Under the collaboration, Juno paid the Company an upfront fee of $5.0 million, purchased one million shares of the Company’s common stock at $8.00 per share, and agreed to fund all of the Company’s collaboration activities. For the first five therapies developed by Juno that incorporate modulators identified through the collaboration, the Company is eligible to receive approximately $500 million upon the achievement of various clinical, regulatory and commercial milestones, plus royalties on net sales.
Upcoming Anticipated Milestones

• First patient to be administered ProTmune in Phase 1 stage of Phase 1/2 clinical trial in mid-2016

• Adaptive NK Cell cancer immunotherapy program update (The Innate Killer Summit, May 16-18, 2016, San Diego, CA)

• Programmed CD34+ Cell immuno-regulatory program update (American Diabetes Association’s 76th Scientific Sessions, June 10-14, 2016, New Orleans, LA)

• Off-the-Shelf, pluripotent cell-derived NK- and T-Cell cancer immunotherapy program updates (International Society for Stem Cell Research, June 22-25, 2016, San Francisco, CA)
• First patient to be administered ProTmune in Phase 2 stage of Phase 1/2 clinical trial in the fourth quarter of 2016

• Data update from Phase 1/2 ProTmune clinical trial (American Society of Hematology, December 3-6, 2016, San Diego, CA)
Financial Results & Guidance

• Cash Position: Cash and cash equivalents as of December 31, 2015 were $64.8 million, compared to $49.1 million as of December 31, 2014. The increase is primarily driven by net proceeds from the Company’s public offering of common stock in May 2015 and cash generated from entering into a research collaboration and license agreement with Juno Therapeutics in May 2015, offset by cash used to fund operating activities.

• Total Revenue: Revenue was $1.1 million for the fourth quarter of 2015 and $2.4 million for the year ended December 31, 2015. All revenue was derived from the Company’s collaboration with Juno.

• Total Operating Expenses: Total operating expenses were $8.0 million for the fourth quarter of 2015 and $30.2 million for the year ended December 31, 2015, compared to $5.9 million and $24.9 million in the comparable periods in 2014. Operating expenses for the fourth quarter of 2015 include $0.5 million of stock compensation expense, compared to $0.6 million for the fourth quarter of 2014.

• R&D Expenses: Research and development expenses were $5.4 million for the fourth quarter of 2015 and $19.9 million for the year ended December 31, 2015, compared to $3.9 million and $16.4 million in the comparable periods in 2014. The increase in R&D expenses is primarily related to an increase in third-party professional consultant and service provider fees to support the clinical development of our product candidates, and an increase in personnel expenses, including stock-based compensation expense, resulting from additional headcount to support the conduct of research activities.

• G&A Expenses: General and administrative expenses were $2.6 million for the fourth quarter of 2015 and $10.4 million for the year ended December 31, 2015, compared to $2.1 million and $8.5 million in the comparable periods in 2014. The increase in G&A expenses is primarily related to an increase in personnel expenses, including stock-based compensation expense, and an increase in costs related to our intellectual property portfolio.

• Common Shares Outstanding: Common shares outstanding as of December 31, 2015 were 28.7 million compared to 20.6 million as of December 31, 2014. Common shares outstanding increased primarily as a result of the 6.9 million shares of the Company’s common stock issued pursuant to the May 2015 financing, and the 1.0 million shares of the Company’s common stock issued and sold to Juno pursuant to the collaboration.

• Financial Guidance: The Company expects 2016 net cash burn to be between $38 million and $42 million.