On November 3, 2017 Pharmaceuticals plc (Nasdaq: JAZZ) reported the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for Vyxeos (daunorubicin and cytarabine) powder for concentrate for infusion to treat adults with high-risk acute myeloid leukemia (AML) defined as therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC) (Press release, Jazz Pharmaceuticals, NOV 3, 2017, View Source [SID1234521543]).

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The CHMP granted Vyxeos accelerated assessment, which is designed to reduce the review timeline for products of major interest for public health and therapeutic innovation. Vyxeos also received Promising Innovative Medicine (PIM) designation from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom. A PIM designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS), intended for the treatment, diagnosis or prevention of a life-threatening or seriously debilitating condition with the potential to address an unmet medical need.

“If approved, Vyxeos will become the first new chemotherapy treatment option specifically for European patients with therapy-related AML or AML with myelodysplasia-related changes,” said Karen Smith, M.D., Ph.D., executive vice president, research and development and chief medical officer at Jazz Pharmaceuticals. “We are passionate about bringing a new treatment option for high-risk AML to the appropriate patients in the EU as quickly as possible and look forward to working with the CHMP during this review process.”

The MAA for Vyxeos includes clinical data from five studies, including the pivotal Phase 3 study. Data from the Phase 3 study, which demonstrated a statistically significant improvement in overall survival for Vyxeos versus standard of care, were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2016.

“Despite numerous advances in the treatment of cancer in Europe, patients with high-risk AML defined as t-AML or AML-MRC have limited treatment options and some of the lowest survival rates compared to people with other forms of AML,” said Dr. Nigel Russell, Professor of Haematology, Faculty of Medicine & Health Sciences at the University of Nottingham. “The hematology oncology community has worked tirelessly to better understand the causes and disease pathways associated with AML and we look forward to the arrival of innovative new treatment options.”

About Vyxeos
Vyxeos daunorubicin 2.2 mg/mL and cytarabine 5 mg/mL powder for concentrate for infusion is an advanced liposomal formulation that delivers a fixed-ratio (1:5) of daunorubicin and cytarabine to the bone marrow that has been shown to have synergistic effects at killing leukemia cells in vitro and in animal models. Vyxeos is the first product developed with the company’s proprietary CombiPlex platform, which enables the design and rapid evaluation of various combinations of therapies. Vyxeos received Orphan Drug Designation by the European Commission in January 2012 and by the U.S. Food and Drug Administration (FDA) in September 2008 for the treatment of AML. Vyxeos received U.S. FDA approval on August 3, 2017 for the treatment of adults with newly-diagnosed t-AML or AML-MRC.

About AML
Acute myeloid leukemia (AML) is a blood cancer that begins in the bone marrow, which produces most of the body’s new blood cells.1 AML cells crowd out healthy cells and move aggressively into the bloodstream to spread cancer to other parts of the body.2 The median age at diagnosis is 68 years old, with rising age associated with a progressively worsening prognosis.3-4 There is also a reduced tolerance for intensive chemotherapy as patients age.5 Patients with t-AML or AML-MRC have few treatment options and some of the lowest survival rates compared to people with other forms of leukemia.6-7 A hematopoietic stem cell transplant (HSCT) may be a curative treatment option for patients.8

On November 3, 2017 AbbVie (NYSE: ABBV) reported that it will participate in the Jefferies 2017 London Healthcare Conference on Thursday, Nov. 16, 2017. Bill Chase, executive vice president and chief financial officer, will present at 3:20 a.m. Central time (Press release, AbbVie, NOV 3, 2017, View Source [SID1234521535]).

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A live audio webcast of the presentation will be accessible through AbbVie’s Investor Relations website at investors.abbvie.com. An archived edition of the session will be available later that day.

On November 3, 2017 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare diseases, reported that the first data from its ongoing Phase 1b clinical study of X4P-001-IO in patients with melanoma will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting on November 8-12 in National Harbor, MD (Press release, X4 Pharmaceuticals, NOV 3, 2017, View Source [SID1234521562]). A second poster highlighting the effects of CXCR4 inhibition in a syngeneic model of melanoma will also be presented.

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The details of the poster presentations are as follows:

X4P-001, an Orally Bioavailable CXCR4 Antagonist, Increases T-cell Infiltration in Human Metastatic Melanoma
Friday, November 10, Abstract # P367, Session Category: Immune Modulation, Cytokines, and Antibodies

Efficacy and Mechanism of Action of CXCR4 Inhibition in B16-OVA Melanoma Model
Friday, November 10, Abstract # P356, Session Category: Immune Modulation, Cytokines, and Antibodies

About X4P-001-IO in Cancer

X4P-001-IO is an investigational selective, oral, small molecule inhibitor of CXCR4 (C-X-C receptor type 4) that regulates the tumor microenvironment, thereby enhancing endogenous anti-tumor responses. CXCR4 is a chemokine receptor that modulates immune function and angiogenesis through the trafficking of key immune cells such as T- cells, dendritic cells, and myeloid derived suppressor cells. CXCR4 signaling is disrupted in a broad range of cancers, facilitating tumor growth by allowing cancer cells to evade immune detection and creating a pro-tumor microenvironment. X4P-001-IO is being investigated in three separate clinical studies in solid tumors.

About Melanoma

Cutaneous malignant melanoma is the fifth most common cancer in men and the sixth most common cancer in women in the United States. When discovered early, melanoma is highly curable with 10-year overall survival rates approaching 95% for stage I melanoma and 45-77% for stage II melanoma.1 However, for patients with stage III and IV melanoma, the prognosis is much worse. The 10-year survival rate for stage IV melanoma is 10-15%.2 Adjuvant therapies for patients with resectable stage III melanoma include immunomodulating drugs, such as high dose interferon-α therapy and anti-CTLA-4 or PD-1 antibody therapy. Unmet medical needs remain to establish and improve overall survival in patients with advanced resectable melanoma, as well as improving objective response rates in patients who do not respond to existing treatments.

On November 3, 2017 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported that it will present two trials in progress posters at the 2017 SITC (Free SITC Whitepaper) annual meeting November 8-12, 2017 at the Gaylord National Hotel & Convention Center in National Harbor, Maryland, United States (Press release, Adaptimmune, NOV 3, 2017, View Source [SID1234521553]).

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In addition, Adaptimmune will have a corporate exhibition booth (#718), and the company will also be hosting a dinner for investors on November 10th with representatives from Adaptimmune’s leadership team and key opinion leader Dr. David S. Hong from The University of Texas MD Anderson Cancer Center. Please reach out to Adaptimmune Investor Relations ([email protected]) for more information on attending the dinner.

The trials in progress posters will summarize the study designs for Adaptimmune’s ongoing MAGE‑A4 multi‑tumor study in urothelial (“bladder”), melanoma, head and neck, ovarian, non-small cell lung cancer (NSCLC), esophageal, and gastric cancers; and, the combination study with NY-ESO and NY‑ESO and KEYTRUDA (pembrolizumab) in multiple myeloma Details are below.

Trials in Progress Posters:

Title: Study Design: Phase 1 dose escalation, multi-tumor study to assess safety, tolerability and antitumor activity of genetically engineered MAGE-A4 SPEAR T-cells in HLA-A2+ subjects with MAGE-A4+ tumors
– Poster Number: P247
– Session Time: November 10, 2017 from 12:30 PM to 2:00 PM (lunch) and 6:30 PM to 8:00 PM (reception)
– Location: Prince George’s Exhibition Hall DE

Title: Study Design: An Open-label Randomized Pilot Study of NY-ESO-1 SPEAR T-cells Alone or in Combination with Pembrolizumab in HLA-A2+ Subjects with Relapsed and Refractory Multiple Myeloma (NCT03168438)
– Poster Number: P248
– Session Time: November 11, 2017 from 12:30 PM to 2:00 PM (lunch) and 6:30 PM to 8:00 (reception)

On November 3, 2017 Dynavax Technologies Corporation(NASDAQ:DVAX) reported financial results for the third quarter ended September 30, 2017. Cash, cash equivalents and marketable securities were $191.7 million at September 30, 2017 compared to $81.4 million at December 31, 2016. The increase was primarily due to net proceeds of approximately $169 million during the year from an underwritten public offering and sales of common stock under an at-the-market sales agreement (Press release, Dynavax Technologies, NOV 3, 2017, View Source [SID1234521558]).

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Additional Financial Results

The net loss for the three months ended September 30, 2017 was $22.1 million, or $0.38 per share, compared to $34.7 million, or $0.90 per share, for the same period in 2016. The net loss for the nine months ended September 30, 2017 was $67.7 million, or $1.36 per share, compared to $90.7 million, or $2.36 per share, for the same period in 2016.

Research and development expenses for the quarter and nine months ended September 30, 2017 were $16.4 million and $47.6 million, respectively, compared to $23.2 million and $66.1 million for the same periods in 2016. The decrease in the 2017 periods reflect reduced compensation and related personnel costs as a result of the January 2017 restructuring and cost reduction initiative. Additionally, the 2017 periods reflect lower costs related to the investigational product HEPLISAV-B [Hepatitis B Vaccine (Recombinant), Adjuvanted] clinical and manufacturing activity partially offset by increased costs relating to seeking FDA approval for HEPLISAV-B and the ongoing development of SD-101, DV281 and earlier stage oncology programs.

General and administrative expenses for the quarter and nine months ended September 30, 2017 were $6.0 million and $18.1 million, respectively, compared to $11.8 million and $29.1 million for the same periods in 2016. The decrease in the 2017 periods reflect reduced compensation and related personnel costs as a result of the January 2017 restructuring and cost reduction initiative. Additionally, the 2016 periods included costs related to hiring of consultants for administrative and commercial development services for an anticipated commercial launch of HEPLISAV-B following FDA approval of this investigational product.

About HEPLISAV-B

HEPLISAV-B is an investigational adult hepatitis B vaccine that combines hepatitis B surface antigen with a proprietary Toll-like Receptor 9 agonist. Data from the Phase 3 trials which evaluated HEPLISAV-B administered as a two dose regimen over one month as compared to a currently licensed hepatitis B vaccine administered as 3 doses over a six month period are currently under review by FDA. Dynavax’s Biologics License Application for HEPLISAV-B has a Prescription Drug User Fee Act date of November 9, 2017. Dynavax has worldwide commercial rights to HEPLISAV-B.

About SD-101

SD-101 is Dynavax’s proprietary, second-generation, Toll-like receptor 9 (TLR9) agonist CpG-C class oligodeoxynucleotide. SD-101 is being studied for its multiple anti-tumor activities in innate immune cells and activation of plasmacytoid dendritic cells to stimulate T cells specific for antigens released from dying tumor cells. TLR9 agonists such as SD-101 enhance T and B cell responses and provide potent Type 1 interferon induction and maturation of plasmacytoid dendritic cells to antigen-presenting cells. SD-101 is being evaluated in several Phase 1/2 oncology studies to assess its safety and activity.

About DV281

DV281 is Dynavax’s proprietary investigational TLR9 agonist designed specifically for focused delivery to primary lung tumors and lung metastases. DV281 is similar in biological activity and mechanism of action to Dynavax’s Phase 2 immunotherapy candidate, SD-101, but has been optimized for administration as an aerosol. Both SD-101 and DV281 activate plasmacytoid dendritic cells which then stimulate T cells specific for antigens released from dying tumor cells. TLR9 agonists such as DV281 and SD-101 have been shown to stimulate potent Type 1 interferon induction along with maturation of dendritic cells to effective antigen-presenting cells; both activities are important for the induction of effective anti-tumor immunity. Dynavax has initiated dosing in a phase 1B dose escalation clinical trial of DV281 in patients with non-small cell lung cancer.

For information about SD-101 and DV281 trials that are currently recruiting patients, please visit www.clinicaltrials.gov.