On November 10, 2016 Bio-Path Holdings, Inc. (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported its financial results for the third quarter ended September 30, 2016 and also provided an update on recent corporate developments (Filing, Q3, Bio-Path Holdings, 2016, NOV 10, 2016, View Source [SID1234516508]).

"Throughout the third quarter and in recent weeks, we have made meaningful progress across our clinical and corporate development programs. Earlier this month, we were pleased to announce the enrollment and dosing of the first patient in the efficacy portion of our Phase 2 clinical study of BP1001 for the treatment of acute myeloid leukemia," said Peter Nielsen, President and CEO of Bio-Path Holdings. "Recently, the European Medicines Agency granted orphan drug designation to BP1001 for the treatment of AML, which recognizes the potential of our DNAbilize technology, underscores the significant unmet medical need in this debilitating disease, and provides regulatory provisions that can accelerate the review process and expand our market exclusivity."

Recent Corporate Highlights

· Announced First Patient Dosed in Phase 2 Trial Evaluating BP1001 in Acute Myeloid Leukemia. In November, the Company announced the enrollment and dosing of the first patient in the efficacy portion of its Phase 2 clinical study of BP1001, a liposomal Grb2 antisense for the treatment of acute myeloid leukemia (AML). The primary endpoint of the study is the number of patients who achieve Complete Remission (CR), including CR with incomplete hematologic recovery (CRi) and CR with incomplete platelet recovery (CRip). Secondary endpoints assessing the safety and efficacy of BP1001 include overall survival, time to response, duration of response, and adverse events as evaluated by physical examination findings, vital signs and clinical laboratory tests.

· Granted Orphan Drug Designation in the European Union for BP1001 for the Treatment of AML. In November, the Company announced that the European Medicines Agency (EMA) granted orphan drug designation to BP1001 for the treatment of AML. To receive orphan drug designation from the EMA, a therapy must be intended for the treatment of a life-threatening or chronically debilitating rare condition with a prevalence of less than five in 10,000 in the European Union. Orphan drug designation provides incentives designed to facilitate development including fee reductions for protocol assistance, scientific advice and importantly, may provide up to ten years of market exclusivity in the EU following product approval.

Financial Results for the Third Quarter Ended September 30, 2016

The Company reported a net loss of $1.6 million, or $0.02 per share, for the three months ended September 30, 2016, compared to a net loss of $1.5 million, or $0.02 per share, for the same period last year. The increase was primarily due to the release of drug material for our Phase 2 clinical trial for BP1001 in AML and associated clinical trial costs.

Research and development expenses for the three months ended September 30, 2016 increased to $2.3 million, compared to $1.0 million for the same period in 2015. General and administrative expenses for the three months ended September 30, 2016 increased to $0.7 million, compared to $0.5 million for the three months ended September 30, 2015.

As of September 30, 2016, the Company had cash of $11.3 million, compared to $8.9 million at December 31, 2015. Net cash used in operating activities for the nine months ended June 30, 2016 was $6.5 million compared to $4.0 million for the comparable period in 2015.

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On November 10, 2016 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported financial results for the third quarter ended September 30, 2016 (Press release, Adaptimmune, NOV 10, 2016, View Source;p=RssLanding&cat=news&id=2221279 [SID1234516506]).

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"Adaptimmune has delivered strong momentum since our last update," said James Noble, Adaptimmune’s Chief Executive Officer. "We have initiated the first site for a triple tumor study with our wholly-owned MAGE-A10 SPEAR T-cells under our new partnership with MD Anderson, and initiated Cohort 4 in our NY‑ESO synovial sarcoma study, as well as commenced recruitment of new patients in our NY-ESO ovarian cancer study under an amended protocol. In addition, we have executed a number of strategic agreements to accelerate our ability to develop, evaluate, and manufacture our affinity enhanced T-cell therapies for patients suffering from a wide array of solid tumor cancers. We are well placed for continued execution and to generate data from studies in multiple cancers with our SPEAR T-cell therapies in 2017."

Mr. Noble continued, "As we recently announced, the FDA has lifted the partial clinical hold on the planned NY-ESO MRCLS study, and we expect to start screening patients shortly. Our goal remains to be the first company to file for approval with a TCR therapy."

Recent Corporate and R&D Highlights:

Partial clinical hold lifted by FDA of NY-ESO SPEAR T-cell therapy study in MRCLS;
Initiation of screening in up to 15 MRCLS patients expected in 4Q 2016 with results from this revised study informing a potential future registration trial;
Established collaboration and supply agreement for combination study of Merck’s PD-1 inhibitor and the Company’s NY-ESO SPEAR T-cell therapy in multiple myeloma; initiation expected in 1H 2017;
Secured strategic agreement with PCT for dedicated manufacturing capacity;
Entered strategic alliance with MD Anderson to expedite T-cell therapy development;
Initiated MD Anderson as the first site for MAGE-A10 SPEAR T-cell therapy triple tumor study in urothelial cancer, melanoma, or squamous cell carcinoma of the head and neck;
Presented data demonstrating response to NY-ESO SPEAR T-cell therapy in synovial sarcoma patients with low NY-ESO expression (Cohort 2) (ESMO 2016);
Presented data indicating that fludarabine is required in preconditioning (Cohort 3) (ESMO 2016);
Commenced enrollment in NY-ESO synovial sarcoma Cohort 4 with a modified preconditioning regimen including fludarabine;
Started recruitment of additional ovarian cancer patients under an amended protocol using NY‑ESO SPEAR T-cell therapy with a modified preconditioning regimen including fludarabine;
Completed preclinical evaluation of MAGE-A4 SPEAR T-cells, with data demonstrating that MAGE-A4 is an attractive target with widespread expression in multiple tumor types; IND planned to be filed in 2017 (data to be presented at SITC (Free SITC Whitepaper) 2016); and
Completed initial evaluation of a second generation NY-ESO SPEAR-T cell expressing a dominant negative TGF-Beta receptor, with data indicating that these SPEAR T-cells may overcome TGF-Beta tumor-mediated immunosuppression (to be presented at SITC (Free SITC Whitepaper) 2016).
Financial Results for the Three-Month Period ended September 30, 2016

Cash / liquidity position: As of September 30, 2016, Adaptimmune had $140.4 million of cash and cash equivalents and $47.1 million of short-term deposits representing a total liquidity position1 of $187.5 million. For the three months ended September 30, 2016, the decrease in cash and cash equivalents was $10.5 million and the decrease in short-term deposits was $7.9 million, representing a decrease in total liquidity position of $18.4 million.
Revenue: For the three months ended September 30, 2016, revenue was $2.4 million compared to $4.9 million for the three months ended September 30, 2015. This decrease was primarily due to the impact of development milestones achieved in the three months ended in September 30, 2015 under the GSK Collaboration and License Agreement.
Research and development ("R&D") expenses: R&D expenses increased to $15.6 million for the three months ended September 30, 2016 from $8.9 million for the three months ended September 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 increased personnel expenses.
General and administrative ("G&A") expenses: G&A expenses were $5.4 million for the three months ended September 30, 2016 compared to $4.4 million for the three months ended September 30, 2015. The increase was primarily due to increased personnel costs.
Net loss: Net loss attributable to holders of the Company’s ordinary shares was $18.5 million for the three months ended September 30, 2016. This equates to $(0.04) per ordinary share or $(0.26) 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.

On November 10, 2016 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported a new myeloid gene expression collaboration to expand the company’s immuno-oncology portfolio (Press release, NanoString Technologies, NOV 10, 2016, View Source [SID1234516482]). The Company, in conjunction with Lisa Coussens, Ph.D., Professor & Chair, Developmental & Cancer Biology Department, OHSU Knight Cancer Institute, Portland, Oregon, is developing two new myeloid focused research panels for the study of the innate immune response to cancer. An early version of the Myeloid Innate Immunity Panel will be made available to Dr. Coussens and her collaborators, as well as the Stand Up To Cancer – Lustgarten Foundation Pancreatic Dream Team members in an exclusive, advance offering during the month of November in conjunction with Pancreatic Cancer Awareness Month, after which the panels will be available to all researchers.

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"I am thrilled to be partnering with NanoString to create these novel myeloid-focused panels," said Coussens. "We anticipate that through these efforts, we will enable a more complete understanding of the local interplay between myeloid immune components and neoplastic cells in tumors."

Myeloid cells play a key role in modulating activities fundamental to cancer development and are known to have both tumor promoting and anti-tumor functions. As myeloid cells are affected by and can have an impact on many types of cancer therapy, they are broadly applicable within immuno-oncology research. A heightened awareness of the importance of the mechanisms of immunotherapy resistance has brought the myeloid immune response into focus as a key modulator of the adaptive immune response. NanoString is currently working with Coussens on her efforts in understanding recruitment of myeloid cells into neoplastic tissue, and the subsequent regulation exerted by those myeloid cells on neoplastic cells and other cells within dynamic tumor microenvironments.

The Myeloid Innate Immunity panel includes approximately 700 genes representing all major categories of myeloid cells, enabling quantitative evaluation of heterogeneous myeloid cell populations based on recruitment, differentiation, maturation status, and functional activities. The panels are optimized to work across a range of sample types including fresh frozen tissues, formalin-fixed paraffin-embedded (FFPE) samples, peripheral blood mononuclear cells and cell lysates.

"It has been a pleasure to collaborate with Dr. Coussens and we are excited to share this work with the broader community of cancer researchers. The Myeloid panel is a collection of genes that encompass the many characteristics of the innate immune response that will help advance cancer research with obvious applications in infectious disease as well," said Joseph Beecham, Ph.D., senior vice president of R&D at NanoString. "These myeloid panels are highly complementary to NanoString’s 770 gene PanCancer Immune Profiling Panel, layering a unique dimension of gene expression information that will provide insights into the modulation activities of the innate immune response."

Dr. Coussens is chair of the Department of Cell, Developmental & Cancer Biology at OHSU. Her research is focused on revealing the role that immune cells play in regulating solid tumor development. Coussens is a principal investigator on the Stand Up To Cancer – Lustgarten Foundation Pancreatic Cancer Convergence Dream Team in which her work is focused on clinical evaluation of immune-based therapies in pancreatic cancer. She has received numerous awards, including: the V Foundation Scholar Award, the AACR (Free AACR Whitepaper)-Women in Cancer Research Charlotte Friend Memorial Lectureship, and the 2015 recipient of the 13th Rosalind E. Franklin Award from the National Cancer Institute.

This is the latest in a series of research partnerships NanoString has with global leaders in immuno-oncology. NanoString and Coussens will be presenting independently at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) conference taking place Wednesday, November 9 through Sunday, November 13 at the Gaylord National Hotel & Convention Center in National Harbor, Maryland.

Results from NanoString’s previously announced collaborations with Merck and MD Anderson Cancer Center will also be presented this week at AMP and SITC (Free SITC Whitepaper).

– Title: Beyond PD-L1 IHC: A Gene Expression Based Test in development for anti-PD-1 response on the nCounter Dx Analysis System
– Speaker: Dr. Matthew Marton, Director of Genomics and Companion Diagnostics, Merck
– Date/time: Wednesday, November 9th, 8 AM – 9 AM.

– Title: The increasing clinical relevance of predictive biomarkers in cancer immunotherapy: can we afford to move forward without them?
– Speakers: Alessandra Cesano, Alex Rueben (MDACC) & Jared Lunceford (Merck).
– Date/time: Saturday, November 12th, 12:00 PM – 1:00 PM.

On November 10, 2016 Celsion Corporation (NASDAQ:CLSN) reported data from the third cohort of patients in its Phase Ib dose escalating clinical trial (the OVATION Study) combining GEN-1, the Company’s DNA-based immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery (Press release, Celsion, NOV 10, 2016, View Source [SID1234516462]). In the first nine patients dosed, GEN-1 plus standard chemotherapy produced impressive results, with no dose limiting toxicities and highly promising efficacy signals in this difficult to treat cancer.

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"While the patient number in this trial may be small, the consistency and robust nature of the data across all three cohorts and the encouraging clinical responses underscore the potential of GEN-1 to serve as an effective, safe IL-12 immunotherapy in ovarian cancer," said Nicholas Borys, M.D., Celsion’s chief medical officer. "I am particularly impressed with the pathological response data, which is known to be associated with prolonged survival in this patient population. The data generated support continued evaluation of GEN-1 in ovarian cancer, and we look forward to seeing how GEN-1 performs in the fourth and final study cohort."
The OVATION Study is designed to enroll three to six patients per dose cohort with the goal of identifying a safe, tolerable and therapeutically active dose of GEN-1 by recruiting and maximizing an immune response. The first three cohorts each enrolled three patients. Enrollment in the fourth cohort is ongoing, and Celsion expects to complete the OVATION Study this year and report data in early 2017. Future studies of GEN-1 will include a Phase I/II study combining GEN-1 with Avastin and Doxil.

OVATION Study – Totality of Results in the First Three Cohorts
Of the first nine patients dosed, one patient demonstrated a complete response (CR), five patients demonstrated partial response (PR) and three patients demonstrated stable disease (SD), as measured by RECIST criteria. This translates to a 100% disease control rate (DCR), and 66% objective response rate (ORR).

Eight patients had successful resections of their tumors, with four patients having an R0 resection, which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed, and three patients with a R1 resection, indicating microscopic residual tumor. One patient had an R2, indicating macroscopic residual tumor. One patient in the second cohort was ineligible for debulking surgery due to a medical complication unrelated to the study or the study drug.

Of the eight surgically treated and evaluable patients, one patient demonstrated a complete pathological response (cPR), three patients demonstrated a micro pathological response (microPR), and four patients demonstrated a macroPR. These data compare favorably to historical data, which indicate that cPRs are typically seen in less than 7% of patients receiving neoadjuvant chemotherapy followed by surgical resection. cPRs have been associated with a median overall survival of 72 months, which is more than three years longer than those who do not experience a cPR. In addition, microPRs are seen in approximately 30% of patients, and are associated with a median overall survival of 38 months¹.

Seven patients who completed treatment follow-up experienced a dramatic (greater than 90%) drop in their CA-125 protein levels as of their most recent study visit. CA-125 is used to monitor certain cancers during and after treatment. CA-125 is present in greater concentrations in ovarian cancer cells than in other cells. A 50% reduction in CA-125 levels is considered meaningful. Six patients maintained CA-125 levels below the standard cutoff level of 35 U/mL.

OVATION Study – Top Line Translational Data from First Two Cohorts
Celsion also reported initial translational data from the first two cohorts of the OVATION study. Tumor and blood samples collected before the start of the neoadjuvant chemotherapy (NACT) and after the completion of GEN-1 treatment at debulking surgery are being analyzed for immune cell populations. Top line data demonstrates intriguing immunological changes in the tumor that are consistent with the activation of the immune system. Specifically,
In tumor tissue, there was an increase in cytotoxic CD8+ T-cell density in three out of four evaluable patients at debulking surgery. There was a decrease in immunosuppressive FoxP3+ T-cells in two out of those 4 patients. The ratio of CD8+/FoxP3+ cells was increased in all four evaluable patients. High tumor infiltrating CD8+ T-cell density, low FoxP3+ T-cell density or high CD8+/FoxP3+ ratio demonstrate a potential shift in tumor environment to favoring immune stimulation following NACT + GEN-1 therapy. For the remaining two patients the post-treatment tumor tissue was not available. In one of those two patients there was complete pathological response hence no tumor tissue was present to provide a post-treatment comparison. In the other patient the debulking surgery was not performed due to disease related complications.

In plasma samples, there appeared to be no significant change in T-cell density following the treatment. The density of myeloid derived suppressor cells that are associated with immunosuppression in ovarian cancer were either decreased or did not increase in post-treatment samples.

Additional immune analysis of biological tissue including cytokine ELISA from the first two patient cohorts and a complete analysis of the two higher dose cohorts is in progress.

"The clinical and translational data generated to date are meaningful and reinforce our confidence in the potential of GEN-1 to address advanced Stage III and IV ovarian cancer, a population clearly in need of effective therapies," said Dr. Khursheed Anwer, Ph.D., MBA, Celsion’s chief scientific officer. "We anticipate completion of enrollment in the fourth patient cohort in the coming weeks, and will continue to assess a potential accelerated clinical development path for GEN-1. In parallel, we are currently evaluating translational data from the study, which we expect to report before the end of the fourth quarter."

On November 10, 2016 Argos Therapeutics Inc. (Nasdaq:ARGS) ("Argos"), an immuno-oncology company focused on the development and commercialization of individualized immunotherapies based on the Arcelis technology platform, reported that the company will be presenting at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 31st Annual Meeting to be held November 11-13 at the Gaylord National Hotel and Convention Center in National Harbor, Maryland.

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Mark DeBenedette, Ph.D., director of immunology for Argos, will present two posters on Friday, November 11th from 12:15-1:30pm ET in Prince George’s Exhibition Hall AB:

"Immunological impact of check point blockade on dendritic cell driven T cell responses; A cautionary tale" (205)
"Multi-Kinase Inhibitors for the Treatment of mRCC: Implications for Combined Therapy with AGS-003; an Autologous Dendritic Cell Immunotherapy" (259)
The SITC (Free SITC Whitepaper) 31st Annual Meeting provides a multidisciplinary educational and interactive environment focused on improving outcomes for current and future patients with cancer by incorporating strategies based on basic and applied cancer immunotherapy. For more information visit View Source

About the Arcelis Technology Platform
Arcelis is a precision immunotherapy technology that captures both mutated and variant antigens that are specific to each patient’s individual disease. It is designed to overcome immunosuppression by producing a specifically targeted, durable memory T-cell response without adjuvants that may be associated with toxicity. The technology is potentially applicable to the treatment of a wide range of different cancers and infectious diseases, and is designed to overcome many of the manufacturing and commercialization challenges that have impeded other personalized immunotherapies. The Arcelis process uses only a small disease sample or biopsy as the source of disease-specific antigens, and the patient’s own dendritic cells, which are optimized from cells collected by a leukapheresis procedure. The proprietary process uses RNA isolated from the patient’s disease sample to program dendritic cells to target disease-specific antigens. These activated, antigen-loaded dendritic cells are then formulated with the patient’s plasma, and administered via intradermal injection as an individualized immunotherapy.