On August 8, 2016 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") reported that it is expanding its promotion of APIFINY, the only cancer-specific, non-PSA blood test available to assess the risk for the presence of prostate cancer, into the important Florida market pursuant to its exclusive promotional agreement with Armune BioScience, Inc. ("Armune"), the owner of the product (Press release, AEterna Zentaris, AUG 8, 2016, View Source [SID:1234514371]). The expansion into Florida follows Armune’s receipt of a clinical laboratory license from the State of Florida.

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"Our sales and marketing teams are well positioned to leverage the opportunity in the Florida market. We believe APIFINY provides significant value in aiding clinicians to more accurately determine the optimal clinical pathway for men at risk of prostate cancer by means of its non-PSA based measurement of risk for the presence of prostate cancer. APIFINY helps meet the needs of achieving value in today’s shifting healthcare environment that involves improving outcomes and patient experiences while lowering overall costs," commented Jude Dinges, Chief Commercial Officer of the Company.

"We are excited to enter the Florida market with the recent approval of our laboratory license by the State of Florida," said David Esposito, President and Chief Executive Officer of Armune. "With over 7,000 tests ordered since the launch of APIFINY, we anticipate significant demand being generated from the Florida market. Given the current concerns of PSA testing throughout the world, APIFINY is well positioned to offer clinicians additional information in the assessment of prostate cancer risk. In addition, we are confident that APIFINY will help to address our healthcare system’s demand for improved outcomes at lower costs."

About Prostate Cancer

Other than skin cancer, prostate cancer is the most common cancer in American men. The American Cancer Society predicted that 220,800 new cases of prostate cancer in the United States would occur in 2015, that there would be 27,540 prostate cancer deaths during the year and that one man in seven would be diagnosed with prostate cancer during his lifetime. Approximately 60% of prostate cancer is diagnosed in men aged 65 or older, and it is rare before age 40. Prostate cancer is the second leading cause of cancer death in American men, behind only lung cancer. Prostate cancer can be a serious disease, but most men diagnosed with prostate cancer do not die from it. In fact, more than 2.9 million men in the United States who have been diagnosed with prostate cancer at some point are still alive today.

About APIFINY

APIFINY is the only cancer-specific, non-PSA blood test that may aid clinicians in the assessment of risk for the presence of prostate cancer. APIFINY technology measures specific biological markers known to be associated with the immune system’s response to prostate cancer and is based on the measurement of eight prostate-cancer-specific autoantibodies in human serum. These autoantibodies are produced and amplified by the immune system in response to the presence of prostate cancer cells. The autoantibodies are stable and, because of their amplification, are likely to be abundant and easy to detect, especially with small tumors characteristic of early-stage cancers. The autoantibody markers span a range of biological functions integral to prostate cancer progression. APIFINY is offered as a lab developed test by Armune BioScience in its CLIA regulated laboratory located in Ann Arbor, MI.

About Armune BioScience

Armune is a medical diagnostics company that develops and commercializes unique proprietary technology exclusively licensed from the University of Michigan for diagnostic and prognostic tests for cancer. The Armune BioScience Laboratory is a commercial reference laboratory, certified and regulated by the federal Clinical Laboratory Improvement Amendments (CLIA) law established in 1988. The laboratory’s CLIA Certificate permits it to perform APIFINY and other high-complexity medical tests. Armune, a private company, has a corporate headquarters in Kalamazoo, MI and a research and commercial laboratory in Ann Arbor, MI. For more information, visit www.armune.com.

On August 8, 2016 Adaptimmune Therapeutics plc (Nasdaq:ADAP) ("Adaptimmune" or the "Company"), a leader in T-cell therapy to treat cancer, reported financial results for the second quarter ended June 30, 2016 (Press release, Adaptimmune, AUG 8, 2016, View Source;p=RssLanding&cat=news&id=2193633 [SID:1234514347]).

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Recent Corporate and Clinical Highlights:

Received access to Priority Medicines (PRIME) regulatory support from the European Medicines Agency for NY-ESO SPEAR (Specific Peptide Enhanced Affinity Receptor) T-cell therapy;
Received orphan medicinal product designation for SPEAR T-cell therapy targeting NY-ESO for the treatment of soft tissue sarcoma in the European Union from the European Commission;
Received orphan drug designation for SPEAR T-cell therapy targeting NY-ESO for the treatment of soft tissue sarcoma from the U.S. Food and Drug Administration (FDA);
Finalized commercial development and supply agreement for Thermo Fisher Scientific’s DynaBeads CD3/CD28 Cell Therapy System for use in manufacturing Adaptimmune’s SPEAR T-cell therapies;
Announced new preclinical and clinical data at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting, including: data showing that the incidence of cytokine release syndrome appears to be of lower frequency and severity with NY-ESO SPEAR T-cell therapy compared to that reported with CD19 CAR-T therapy; data describing robust clinical responses including a 50 percent response rate (60 percent at the target dose) in synovial sarcoma, and a 91 percent response rate in multiple myeloma; and that Adaptimmune’s extensive preclinical safety package is capable of preclinically validating TCRs with enhanced affinity for target proteins; and
Expanded synovial sarcoma trials to sites outside of the United States with submissions made to the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom, and to Health Canada which has now approved the clinical trial application.
"The last six months were a period of strong progress for Adaptimmune on a number of fronts," commented James Noble, Adaptimmune’s Chief Executive Officer. "Among our accomplishments, we continued to advance our NY-ESO SPEAR T-cell program toward pivotal studies in sarcoma, received orphan drug and Breakthrough Therapy designation in the U.S. and received both PRIME regulatory eligibility and orphan medicinal product designation in Europe. We also presented important new clinical data at the 2016 ASCO (Free ASCO Whitepaper) meeting and at our analyst and investor day in April 2016 that continue to differentiate our SPEAR T-cell products from other therapies in the space."

Mr. Noble continued, "Still, this period has not been without its challenges. As we previously announced, the FDA placed a partial clinical hold on our planned pivotal study of NY-ESO SPEAR T-cell therapy in myxoid round cell liposarcoma and requested additional information prior to trial commencement. We will provide a full response to the FDA shortly. Separately, the initiation of the pivotal study in synovial sarcoma has been delayed until mid-2017. In addition, slower than anticipated enrollment in non-small cell lung cancer (NSCLC) for our MAGE-A10 and NY-ESO SPEAR T-cell therapies has also pushed the timelines for these data readouts into 2017. Notwithstanding these challenges, the breadth and depth of our clinical pipeline mean that, by early 2017, our clinical efforts could involve SPEAR T-cell therapies directed against four separate targets in as many as 10 different tumor types; we anticipate that our data readouts throughout 2017 may validate the targets themselves and, more broadly, our SPEAR T-cell platform in additional solid tumors beyond sarcoma."

Financial Results for the three-month period ended June 30, 2016

Cash / liquidity position: As of June 30, 2016, Adaptimmune had $150.9 million of cash and cash equivalents and $55.0 million of short-term deposits representing a total liquidity position1 of $205.9 million. For the three months ended June 30, 2016, the decrease in cash and cash equivalents was $12.9 million and the decrease in short-term deposits was $7.3 million, representing a decrease in total liquidity position of $20.2 million.
Revenue: For the three months ended June 30, 2016, revenue was $0.3 million compared to $2.8 million for the three months ended June 30, 2015. This decrease was primarily due to a change in the estimate of the period over which the revenue relating to the GSK Collaboration and License Agreement is being recognized.
Research and development ("R&D") expenses: R&D expenses increased to $16.2 million for the three months ended June 30, 2016 from $8.4 million for the three months ended June 30, 2015, primarily due to increased period-over-period costs associated with: ongoing clinical trials of the Company’s NY-ESO and MAGE-A10 SPEAR T-cell therapies; preparation for a study with the Company’s SPEAR T-cell therapy targeting AFP; and personnel expenses for an increased number of employees engaged in R&D.
General and administrative ("G&A") expenses: G&A expenses were $6.8 million for the three months ended June 30, 2016 compared to $5.5 million for the three months ended June 30, 2015. The increase was primarily due to increased personnel costs, increased property costs and other costs associated with being a public company, partially offset by a decrease in share-based compensation expenses.
Net loss: Net loss attributable to holders of the Company’s ordinary shares was $22.1 million for the three months ended June 30, 2016. This equates to $(0.05) per ordinary share or $(0.31) per American Depositary Share.
1 Total liquidity position is a non GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.

Financial Guidance

Adaptimmune is reiterating its guidance. For the full year 2016, the Company expects its decrease in total liquidity position to be between $80 and $100 million and expects its total liquidity position at December 31, 2016, including cash, cash equivalents and short term deposits, to be at least $150 million. This guidance excludes the effect of any potential new business development activities.

Pipeline Review
Adaptimmune is providing an update below on each cohort of its clinical pipeline and the timing of anticipated milestones.

NY-ESO SPEAR T-cell Therapy
Synovial sarcoma:
Cohort 1 (high NY-ESO expression, cyclophosphamide + fludarabine) is complete, and was initially reported at the 2015 American Association for Cancer Research (AACR) (Free AACR Whitepaper) meeting. Updates on cohorts 2 (low NY-ESO expression, cyclophosphamide + fludarabine) and 3 (cyclophosphamide) will be presented at the 2016 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) meeting. Cohort 4 (modified cyclophosphamide + fludarabine) is on schedule to initiate in 2H 2016.

The Company submitted to the IND its proposed pivotal study in myxoid round cell liposarcoma and received a partial clinical hold notice from the FDA. The notice was received prior to the study being opened at any clinical site. This communication from the FDA was not related to concerns on safety, but rather to the request for additional information on CMC about manufacturing changes introduced in the pivotal program, and information on and modification of some clinical trial design features. The Company intends to submit its full response shortly; the agency will then have 30 days to respond. In addition, enrollment into the pivotal synovial sarcoma study will begin in mid-2017 to allow for the submission of a Special Protocol Assessment requested by FDA and characterization of the revised commercial manufacturing process to be employed in the pivotal studies. If the discussions with the FDA on MRCLS are concluded within the 30-day review window, the Company would expect to initiate the study as planned in Q4 2016/Q1 2017; if it takes longer, then this study will be on a similar timeline to the synovial sarcoma study.

Multiple myeloma:
The 25-patient study with autologous stem cell transplant is complete and has been published (Rapoport; Nat Med, 2015). The Company expects to agree terms for a combination study with a PD-1 receptor inhibitor using cyclophosphamide and fludarabine conditioning in 2016, with initiation of the study occurring in 1H 2017.

Ovarian cancer:
The Company reported data from this six-patient study at the 2016 ASCO (Free ASCO Whitepaper) meeting showing no objective response in the absence of fludarabine. The Company is evaluating a preconditioning regimen consisting of modified doses of cyclophosphamide and fludarabine and intends to enroll patients in 2H 2016 with this regimen.

Melanoma:
The Company reported data at the 2016 ASCO (Free ASCO Whitepaper) meeting showing no objective response in the absence of fludarabine in six patients who had progressed after treatment with immune check point inhibitors. The Company is considering a study with a new preconditioning regimen including fludarabine in combination with check point inhibitors in 2017.

Non-small cell lung cancer:
A study is open and actively screening patients; data are anticipated in 2017. The chemotherapy conditioning for this trial is being modified in an amendment to consist of cyclophosphamide and fludarabine instead of cyclophosphamide alone.

MAGE-A10 SPEAR T-cell Therapy
Non-small cell lung cancer:
A study is open and actively screening patients; data are anticipated in 2017. Chemotherapy conditioning for this trial is being modified in an amendment to consist of cyclophosphamide and fludarabine instead of cyclophosphamide alone.

Bladder, melanoma, and ovarian cancer:
The Company is on track to initiate this study, including a preconditioning regimen of cyclophosphamide and fludarabine, in 2016 with data anticipated in 2017.

AFP SPEAR T-cell Therapy
Hepatocellular cancer:
The investigational new drug application (IND) is open, and the Company anticipates that enrollment will begin in 1H 2017.

MAGE-A4 SPEAR T-cell Therapy
Multiple tumor types:
The Company is on track for an IND submission in 1Q 2017.

Generation 2 TCRs
Multiple tumor types:
The Company is on track to submit INDs from 2017 onwards.

On August 8, 2016 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported financial results for the second quarter of 2016, recapped recent progress against the Company’s multi-year vision and provided an update on clinical data timelines (Press release, Epizyme, AUG 8, 2016, View Source [SID:1234514351]).

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"We are pleased with our continued momentum in the clinical development of tazemetostat in non-Hodgkin lymphoma, or NHL, and genetically defined solid tumors, as well as the expansion of the tazemetostat clinical development program into mesothelioma," said Robert Bazemore, President and Chief Executive Officer of Epizyme. "For the remainder of the year, our focus is on the continued enrollment in and execution of our Phase 2 NHL and solid tumor studies to generate mature objective response and durability data, and to assess the differences in activity between study arms. With a robust clinical program underway for tazemetostat, and advancements in our preclinical and discovery efforts, we believe we are further solidifying Epizyme’s position as the leader in epigenetic drug discovery and development."

Advancing the Clinical Development of Tazemetostat for the Treatment of Non-Hodgkin Lymphoma (NHL) and Genetically Defined Solid Tumors

In June, Epizyme presented positive, early findings from its ongoing Phase 2 study of tazemetostat in patients with relapsed or refractory NHL at the American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting on Lymphoma Biology. As of the data cutoff date in late May, approximately 30 percent of the planned 270 patients were enrolled into the study. Tazemetostat demonstrated clinical activity, including objective responses, in all five arms of the study and a favorable safety profile in a heavily pre-treated, multiply relapsed and highly refractory patient population. Overall patient enrollment is proceeding according to internal projections, and the study is now open for enrollment in the U.S.

Overall patient enrollment is also on-track in the Company’s ongoing Phase 2 clinical study in adult patients and Phase 1 study in pediatric patients with INI-1 negative tumors or synovial sarcoma.

Epizyme plans to provide data on the efficacy and safety of tazemetostat from both the NHL and solid tumor Phase 2 studies in the first half of 2017. The Company plans to meet with regulatory authorities, beginning with the U.S. Food and Drug Administration (FDA) in mid-2017, to review data from all cohorts of its Phase 2 solid tumor study and discuss registration strategies based on these data. Epizyme also expects to meet with FDA to review Phase 2 NHL data and discuss registration strategies in 2017.

In June, Epizyme participated in FDA’s Pediatric Subcommittee of the Oncologic Drugs Advisory Committee (ODAC) meeting, which reviewed select novel, targeted agents for the treatment of pediatric cancer. Epizyme was one of five invited innovators, and presented on its ongoing pediatric clinical program. The Company received valuable feedback and support from Committee members on the development of tazemetostat as a treatment for rare and extremely aggressive cancers in pediatric patients.
Broadening Scope of Tazemetostat Clinical Program

In August, Epizyme initiated a global study of tazemetostat in patients with mesothelioma. This is a multi-center Phase 2 study evaluating tazemetostat as a monotherapy treatment for patients with mesothelioma characterized by BAP1 loss-of-function. The study is open for enrollment in the U.S., with an accepted Investigational New Drug application, and is expected to open in international sites shortly. This study marks the first of five new indications into which the Company plans to expand tazemetostat in the clinic by the end of 2020.

In June, Epizyme entered into a collaboration agreement with Genentech, a member of the Roche Group, to conduct a clinical trial to investigate tazemetostat and Genentech’s recently approved anti-PD-L1 cancer immunotherapy, Tecentriq (atezolizumab), when used in combination for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma. The study will be conducted by Genentech and is expected to begin in the second half of 2016.

In May, Epizyme entered into a collaboration agreement with the Lymphoma Study Association (LYSA), a premier cooperative group in France dedicated to clinical and translational research for lymphoma, to investigate the combination of tazemetostat with R-CHOP as a front-line treatment in elderly, high-risk patients with DLBCL. The study will be conducted by LYSA and is expected to be initiated in the second half of 2016.

Second Quarter 2016 Financials Results and Guidance

Collaboration revenue was $0.5 million and $0.9 million for the three and six months ended June 30, 2016, respectively, compared to $0.7 million and $1.6 million for the three and six months ended June 30, 2015, respectively. The period-over-period decreases reflect decreased recognition of deferred revenue from upfront payments and research and development revenue related to the Company’s collaboration with GlaxoSmithKline, partially offset by increased recognition of deferred revenue from upfront payments from its Celgene collaboration.

Research and development (R&D) expenses were $21.5 million and $39.2 million for the three and six months ended June 30, 2016, respectively, compared to $20.6 million and $77.6 million for the three and six months ended June 30, 2015, respectively. During the three months ended June 30, 2016, R&D expenses increased due to external and internal costs associated with the expansion of the tazemetostat program, which more than offset the period-over-period reduction of pinometostat-related costs. The period-over-period decrease from the six months ended June 30, 2015 was driven by the first quarter 2015 payment to Eisai of $40.0 million for the reacquisition of the worldwide rights, excluding Japan, to tazemetostat. Epizyme expects that R&D expenses will increase significantly in the remainder of 2016. This will be driven by the planned expansion of clinical trial activity for tazemetostat, including initiation of the mesothelioma study, initiation of the two combination trials in DLBCL, and expansion of the Company’s ongoing studies in NHL and solid tumors. In addition, discovery and preclinical research costs are expected to increase as Epizyme advances its wholly owned small molecule programs against multiple novel epigenetic targets and continues its research efforts under its Celgene collaboration.

General and administrative (G&A) expenses were $7.4 million and $13.3 million for the three and six months ended June 30, 2016, respectively, as compared to $6.0 million and $11.2 million for the three and six months ended June 30, 2015, respectively. The period-over-period increases in G&A expenses were largely due to staffing key leadership roles in the second quarter. Epizyme expects that G&A spend will increase modestly over the remainder of 2016.

Net loss was $28.0 million and $50.9 million for the three and six months ended June 30, 2016, respectively, compared to a net loss of $25.8 million and $87.1 million for the three and six months ended June 30, 2015, respectively.

Cash, cash equivalents and marketable securities were $288.6 million as of June 30, 2016, as compared to $208.3 million as of December 31, 2015.

Epizyme has established an operating plan and investment strategy designed to support its objectives through 2020 and extend its runway. Based on the updated operating plan, the Company now believes that its cash, cash equivalents and marketable securities as of June 30, 2016 will be sufficient to fund the Company’s planned operations into at least the second quarter of 2018.

On August 8, 2016 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported financial results for the quarter ended June 30, 2016 (Press release, Inovio, AUG 8, 2016, View Source [SID:1234514352]). The following financial results provide a year-over-year comparison of the second quarter in 2016 and 2015. Total revenue was $6.2 million compared to $5.3 million. Total operating expenses were $24.4 million compared to $20.4 million. The net loss attributable to common stockholders was $18.7 million, or $0.26 per share, compared to $6.2 million, or $0.09 per share.

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Revenue

The increase in revenue was primarily due to an increase in development payments from our DARPA Ebola grant.

Operating Expenses

Research and development expenses were $19.6 million compared to $16.7 million. The increase was primarily related to increased investment in our product development programs. General and administrative expenses were $5.8 million compared to $4.7 million.

Capital Resources

As of June 30, 2016, cash and cash equivalents and short-term investments were $134.5 million compared with $163.0 million as of December 31, 2015. There were 73.5 million shares outstanding and 81.2 million fully diluted.

The Company sold 119,400 shares of common stock at an average price of $11.12 per share, for net proceeds of $1.3 million, under the ATM common stock sales agreement implemented during the period.

Inovio’s balance sheet and statement of operations are provided below. Form 10-Q providing the complete 2016 second quarter financial report can be found at: View Source

Corporate Update

Clinical Development

The FDA and European Medicines Agency provided an affirmative path toward an indication for VGX-3100 to treat HPV-16/18-related high grade cervical dysplasia in a pivotal phase III registration study. We completed major commercial device design and manufacturing process development efforts, and are in the final stage of testing. Completion of this extensive work will enable us to then submit our final package to the FDA in order to start the phase III in 4Q 2016.
Received approval from the FDA to initiate a phase I human trial to evaluate Inovio’s Zika DNA vaccine (GLS-5700). This phase I, open-label, dose-ranging study with 40 healthy subjects is evaluating the safety, tolerability and immunogenicity of GLS-5700. Subsequent to the quarter Inovio announced the dosing of the first subject in this study. We expect to report interim immune response and safety data in 4Q 2016.
Inovio will continue to develop its hepatitis B DNA immunotherapy (INO-1800) independently following Roche’s notice that it will discontinue its collaboration with Inovio and its development of INO-1800. INO-1800 was licensed to Roche from Inovio in 2013. All of Roche’s rights to INO-1800, including the right to license the product to other parties, will be returned. Inovio will continue to advance its current phase I study of INO-1800, which is enrolling as planned in 30 clinical sites in the U.S. and Asia-Pacific regions. Inovio anticipates completing enrollment in the first half of 2017 and expects results in the second half of 2017.
The study has completed interim safety reviews with a favorable safety profile to date. Immunology analyses are planned after completion of enrollment.
Partnered with the National Cancer Institute and Mayo Clinic to initiate a phase I trial of our immunotherapy for hepatitis C (INO-8000). The dose escalation study will enroll patients in the early stages of chronic HCV infection to determine the therapy’s ability to decrease and potentially eliminate HCV viral load, measure HCV specific immune responses and durability of these immune responses, and evaluate safety and tolerability.
Completed enrollment of 94 subjects in the phase I study of our PENNVAX-GP HIV immunotherapy. After completing extensive immunogenicity analyses, we expect to report data in 1H 2017.
Completed enrollment of 22 subjects in the phase I study of our HPV-driven cancer immunotherapy, INO-3112, in head & neck cancer patients. We expect to report additional immune response and safety data in 4Q 2016.
Completed enrollment of 62 subjects in the phase I study of our INO-5150 prostate cancer immunotherapy. We expect to report interim immune response and safety data in 4Q 2016.
Completed enrollment of 75 subjects in the phase I study of our GLS-5300 MERS vaccine. We expect to report interim immune response and safety data in 4Q 2016.
Corporate Development

Inovio completed the acquisition of all of Bioject Medical Technologies Inc.’s assets, including pioneering needle-free jet injection technology, devices, and intellectual property, for $5.5 million in cash and stock.
Inovio’s DNA-based monoclonal antibody technology will be deployed to develop new immunotherapy approaches to treat HIV. This work will be funded by a $23 million grant, called BEAT-HIV: Delaney Collaboratory to Cure HIV-1 Infection by Combination Immunotherapy, from the National Institutes of Health to The Wistar Institute, an Inovio collaborator, and more than 30 of the nation’s leading HIV investigators.
Preclinical Development

Preclinical testing of our Zika virus synthetic vaccine induced robust and durable immune responses in mice and in non-human primates (monkeys).

On August 8, 2016 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the second quarter ended June 30, 2016 (Press release, Celldex Therapeutics, AUG 8, 2016, View Source [SID:1234514393]).

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"Celldex continues to build one of the most robust pipelines in immuno-oncology, most recently advancing CDX-014 into the clinic in renal cell carcinoma," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "In collaboration with our investigators, we also presented a significant body of data in the second quarter with eight presentations across both AACR (Free AACR Whitepaper) and ASCO (Free ASCO Whitepaper) that spoke to the broad utility of our product candidates in combination immunotherapy and highlighted a number of the novel targets we are pursuing."

"We continue to enroll patients to the pivotal METRIC study of glembatumumab vedotin in triple negative breast cancer, with a focus on a number of new sites in Europe that were added over the last quarter and look forward to presenting data from the Phase 2 study of glembatumumab vedotin in metastatic melanoma later this year," concluded Marucci.

Program Updates:

Glembatumumab vedotin ("glemba"; CDX-011), an antibody-drug conjugate (ADC) targeting gpNMB in multiple cancers

Enrollment continues in the Company’s Phase 2b randomized study (METRIC) of glembatumumab vedotin in patients with metastatic triple negative breast cancers that overexpress gpNMB, a molecule associated with poor outcomes for triple negative breast cancer patients and the target of glembatumumab vedotin. Enrollment is open across the United States, Canada, and Australia and opened in the European Union in April, with close to 25 sites added in the EU in the second quarter. Additional sites continue to be added to support enrollment completion.

Patient enrollment is complete, and the primary endpoint has been met in the Phase 2 single-agent study of glembatumumab vedotin in metastatic melanoma (post-progression on checkpoint therapy). The primary endpoint of the study, objective response rate, required a minimum of six responses in the first 52 patients to be deemed successful. Celldex plans to present data from this study at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in October 2016. As previously announced, the Company has amended the protocol to add a second cohort of patients to a glembatumumab vedotin and varlilumab combination arm to assess the potential clinical benefit of the combination and to explore varlilumab’s potential biologic and immunologic effect when combined with an ADC. This additional cohort is open to enrollment.

Celldex is also evaluating glembatumumab vedotin in other cancers in which gpNMB is expressed.

Celldex has entered into a collaborative relationship with PrECOG, LLC, which represents a research network established by the Eastern Cooperative Oncology Group (ECOG), and PrECOG, LLC is conducting a Phase 1/2 study in squamous cell lung cancer. This study opened to enrollment in April 2016.

Celldex and the National Cancer Institute (NCI) have entered into a Cooperative Research and Development Agreement (CRADA) under which the NCI is sponsoring two studies of glembatumumab vedotin—one in uveal melanoma and one in pediatric osteosarcoma. Both studies are currently open to enrollment.
Varlilumab ("varli"; CDX-1127), a fully human monoclonal agonist antibody that binds and activates CD27, a critical co-stimulatory molecule in the immune activation cascade

The Phase 2 portion of the varlilumab and nivolumab (Opdivo) study opened to enrollment in April 2016. A protocol amendment was recently finalized to include additional arms evaluating alternate dosing schedules in both renal cell carcinoma and squamous cell head and neck cancer. The non-small cell lung cohort was removed prior to enrolling any patients to accommodate the addition of these new arms. As amended, the overall study size has increased and includes cohorts in colorectal cancer (n=18), ovarian cancer (n=18), head and neck squamous cell carcinoma (n=48), renal cell carcinoma (n=75) and glioblastoma (n=20). The study is being conducted by Celldex under a clinical trial collaboration with Bristol-Myers Squibb Company. The companies are sharing development costs.

Data from the Phase 1 portion (n=36) of the varlilumab and nivolumab study were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April. The combination showed acceptable tolerability and safety across all dose levels without any evidence of increased autoimmunity or inappropriate immune activation. Combination therapy led to marked changes in the tumor microenvironment including increased infiltrating CD8+ T cells and increased PD-L1 expression, which have been shown to correlate with a greater magnitude of treatment effect from checkpoint inhibitors in other clinical studies. Additional favorable immune biomarkers, such as increase in inflammatory chemokines and decrease in T regulatory cells, were also noted. In a subset of patients (n=17) on study who had both pre- and post-tumor biopsies available, preliminary evidence also suggested a correlation between biomarker data and stable disease or better in seven of these patients (4 ovarian cancer, 2 colorectal cancer, 1 squamous cell carcinoma of the head and neck).

Enrollment has been completed in the Phase 1 dose-escalation portion of the Phase 1/2 study of varlilumab and atezolizumab (Tecentriq; anti-PDL1) in patients with multiple solid tumors. The Company anticipates the Phase 2 portion of the study in renal cell carcinoma will be initiated in the third quarter of this year. This study is being conducted by Celldex under a clinical trial collaboration with Roche. Roche is providing study drug, and Celldex is responsible for conducting and funding the study.

Additional combination studies of varlilumab continue to enroll patients including:

A Phase 1/2 safety and tolerability study examining the combination of varlilumab and sunitinib (Sutent) in patients with metastatic clear cell renal cell carcinoma. The Company anticipates the Phase 1 portion of the study will complete enrollment in the next few months and that the Phase 2 portion of the study will initiate by year-end.
A Phase 1/2 safety and tolerability study examining the combination of varlilumab and ipilimumab (Yervoy) in patients with stage III or IV metastatic melanoma. In the Phase 2 portion of the study, patients with tumors that express NY-ESO-1 will also receive Celldex’s CDX-1401, an NY-ESO-1-antibody fusion protein for immunotherapy.
As discussed above, a Phase 2 study of varlilumab and glembatumumab vedotin in metastatic melanoma (post-progression on checkpoint therapy).
CDX-1401, an NY-ESO-1-antibody fusion protein for immunotherapy

As discussed above, a Phase 1/2 study examining the combination of varlilumab and ipilimumab continues to enroll patients with stage III or IV metastatic melanoma. In the Phase 2 portion of the study, patients with tumors that express NY-ESO-1 will also receive CDX-1401.

Celldex continues to support several external collaborations, including an NCI sponsored Phase 2 study of CDX-1401 and CDX-301 for patients with metastatic melanoma, which has completed enrollment (n=60 patients; not selected for NY-ESO-1 expression). Initial data from this study were presented at the at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data confirmed that CDX-1401 is effective at driving NY-ESO-1 immunity and further demonstrated the value of CDX-301 as a combination agent for enhancing tumor-specific immune response. Based on results to date, plans for additional studies are being considered, including a targeted study in NY-ESO-1 positive disease to determine if these enhanced immune responses can translate to improved clinical outcomes.

Additionally, Roswell Park Cancer Center is conducting an investigator sponsored study evaluating CDX-1401, poly-ICLC (Hiltonol) and the IDO1 inhibitor epacadostat (INCB24360) in patients in remission with ovarian, fallopian tube or primary peritoneal cancer. Patients’ tumors must have expressed NY-ESO-1 or the LAGE-1 antigen to be eligible for the study. Celldex is providing CDX-1401 and poly-ICLC in support of this study.
CDX-301 (recombinant human Flt3L), a potent hematopoietic cytokine that uniquely expands the number of dendritic cells to prime the immune system for more robust immune responses to cancer antigens

As outlined above, data were presented from the Phase 2 study of CDX-1401 and CDX-301 in metastatic melanoma that further demonstrated the value of CDX-301 as a combination agent for enhancing tumor-specific immune response. CDX-301 greatly expanded peripheral blood dendritic cells and was highly effective at increasing cancer antigen specific T cells and antibodies when combined with CDX-1401. These results, which also showed rapid cellular immune responses in a majority of patients, suggests that pre-treatment with CDX-301 could provide a highly applicable, effective immunologic approach.

CDX-301’s potential activity is also being explored in a Phase 1/2 study of CDX-301 and poly-ICLC in combination with low-dose radiotherapy in patients with low-grade B-cell lymphomas conducted by the Icahn School of Medicine at Mount Sinai.
CDX-014, an antibody-drug conjugate (ADC) targeting the transmembrane protein T-cell immunoglobulin mucin-1 (TIM-1) in renal cell carcinoma

In July 2016, Celldex announced that enrollment had opened in the Phase 1 dose-escalation portion of the Company’s Phase 1/2 study of CDX-014 in advanced clear cell and papillary renal cell carcinoma (RCC). The Phase 1 study will evaluate cohorts of patients receiving increasing doses of CDX-014 to determine the maximum tolerated dose and a recommended dose for Phase 2 study.
RINTEGA ("rindopepimut"; "rindo"; CDX-110), an EGFRvIII(v3)-specific therapeutic vaccine for glioblastoma (GBM)

As previously disclosed, in March, during a pre-planned interim analysis, the independent Data Safety and Monitoring Board (DSMB) recommended discontinuation of the Phase 3 ACT IV study of RINTEGA (rindopepimut) in patients (n=745) with newly diagnosed EGFRvIII-positive glioblastoma. Study closure activities are substantially complete, and Celldex continues to anticipate that the Company will not incur substantial additional costs related to RINTEGA at this time. Celldex is in the process of conducting a thorough review of the data and plans to present the ACT IV results at the Society for Neuro-Oncology Annual Meeting in November of 2016. All patients on the RINTEGA arm of the ACT IV study, prior Phase 2 studies and existing compassionate use recipients have been offered ongoing access to RINTEGA on a compassionate use basis.
Second Quarter and First Six Months 2016 Financial Highlights and Updated 2016 Guidance

Cash position: Cash, cash equivalents and marketable securities as of June 30, 2016 were $220.1 million compared to $254.0 million as of March 31, 2016. The decrease was primarily driven by our second quarter cash used in operating activities of $33.8 million, $5.9 million of which were RINTEGA-related payments. At June 30, 2016, Celldex had 99.4 million shares outstanding.

Revenues: Total revenue was $1.4 million in the second quarter of 2016 and $2.7 million for the six months ended June 30, 2016, compared to $2.2 million and $2.7 million for the comparable periods in 2015. Total revenue was primarily derived from our clinical trial collaboration with Bristol-Myers Squibb and our research and development agreement with Rockefeller University.

R&D Expenses: Research and development (R&D) expenses were $25.7 million in the second quarter of 2016 and $53.2 million for the six months ended June 30, 2016, compared to $26.5 million and $51.6 million for the comparable periods in 2015.

The decrease in R&D expenses of $0.8 million between the three-month periods was primarily due to lower clinical costs of $3.2 million, offset in part by increased contract manufacturing costs of $0.8 million and personnel costs of $1.6 million, including higher stock-based compensation of $0.8 million.

The increase in R&D expenses of $1.6 million between the six-month periods was primarily due to higher contract manufacturing and other contract service costs and personnel costs, including higher stock-based compensation of $1.3 million, offset by lower clinical costs.

G&A Expenses: General and administrative (G&A) expenses were $7.8 million in the second quarter of 2016 and $17.1 million for the six months ended June 30, 2016, compared to $8.2 million and $14.3 million for the comparable periods in 2015.

The decrease in G&A expenses of $0.4 million between the three-month periods was primarily due to lower commercial planning costs of $1.1 million, partially offset by higher stock-based compensation of $0.7 million.

The $2.8 million increase in G&A expenses between the six-month periods was primarily due to higher stock-based compensation of $1.8 million, facility costs and legal costs.

Net loss: Net loss was $32.0 million, or ($0.32) per share, for the second quarter of 2016 and $66.6 million, or ($0.67) per share, for the six months ended June 30, 2016, compared to a net loss of $32.4 million, or ($0.33) per share and $62.5 million, or ($0.65) per share for the comparable periods in 2015.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at June 30, 2016 combined with the anticipated proceeds from future sales of our common stock under our $60 million sales agreement with Cantor Fitzgerald & Co. are sufficient to meet estimated working capital requirements and fund planned operations through 2018.