On November 4, 2015 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, reported two poster presentations at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 30th Anniversary Annual Meeting & Associated Programs being held November 4-8, in National Harbor, Maryland (Press release, Verastem, NOV 4, 2015, View Source;p=RssLanding&cat=news&id=2106370 [SID:1234507953]).

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The details of the presentations at SITC (Free SITC Whitepaper) are as follows:

Title: Targeting Focal Adhesion Kinase Reprograms the Pancreatic Tumor Microenvironment and Renders Pancreas Cancer Responsive to Checkpoint Immunotherapy
Date and time: Friday Nov. 6 from 12:30 – 2:00 PM ET
Track: Tumor Microenvironment
Poster #: 419

Title: FAK/PYK2 Inhibitors Defactinib and VS-4718 Enhance Immune Checkpoint Inhibitor Efficacy
Date and time: Friday Nov. 6 from 12:30 – 2:00 PM ET
Track: Optimizing Combination Immunotherapy
Poster #: 371

The posters will be on display starting at 3:00pm ET on Thursday, November 5th and will remain accessible through 2:00pm ET on Saturday, November 7th. The posters will also be available on Verastem’s website at http://bit.ly/R3M6wc starting at 6:00pm ET on Thursday, November 5th.

About VS-6063
VS-6063 (defactinib) is an orally available compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). Cancer stem cells are an underlying cause of tumor resistance to chemotherapy, recurrence and ultimate disease progression. Research has demonstrated that FAK activity is critical for the growth and survival of cancer stem cells. VS-6063 is currently being studied in the "Window of Opportunity" study in patients with mesothelioma prior to surgery, a Phase 1/1b study in combination with paclitaxel in patients with ovarian cancer, a trial in patients with KRAS-mutated non-small cell lung cancer and a trial evaluating the combination of VS-6063 and VS-5584 in patients with relapsed mesothelioma.

About VS-4718
VS-4718 is an orally available compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). VS-4718 is currently being studied in a Phase 1 dose escalation study in patients with advanced cancers.

On November 4, 2015 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, reported financial results for the three-month period ended September 30, 2015 (Press release, Spectrum Pharmaceuticals, NOV 4, 2015, View Source [SID:1234507977]).

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"The highest priority of the Company remains SPI-2012, which is a late-stage drug that could compete in the multi-billion dollar neutropenia market," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "We had a productive meeting last week with the FDA and expect to finalize our SPA on SPI-2012 quickly. We have a meeting with the FDA this Friday to discuss EVOMELA’s Complete Response Letter and we feel confident in bringing EVOMELA to the market for patients. Before the end of this year, we look forward to filing the NDA for our bladder cancer drug apaziquone and initiating a Phase 2 breast cancer trial for poziotinib in the U.S. shortly. We believe the infrastructure that we have built over the years serves as a strong foundation for continued future growth."

Pipeline Update- Two Potential Blockbusters and One Near-term NDA Submission:

SPI-2012, a novel long-acting GCSF: In a Phase 2 dose ranging study, SPI-2012 was shown to be superior at the higher dose tested and non-inferior at the middle dose in decreasing the duration of severe neutropenia compared to the blockbuster drug pegfilgrastim. SPI-2012 was also shown to have an acceptable safety profile with no significant dose-related or unexpected toxicities. The Phase 2 data will be presented at the San Antonio Breast Cancer Symposium. Spectrum has continued to have productive discussions with the FDA, expects to finalize the pivotal study design this year, and start the study shortly after reaching SPA agreement with the Agency. Over 80 study sites have already been qualified.

Apaziquone, a potent tumor-activated pro-drug for non-muscle invasive bladder cancer: By year end, Spectrum expects to file the NDA based on the previous Phase 3 studies. The Company has also initiated enrollment in an additional randomized, placebo-controlled Phase 3 trial under the SPA agreement, and treated the first patient in late October. This Phase 3 study has been specifically designed to address important lessons learned from the previous apaziquone Phase 3 studies, as well as recommendations made by the FDA.

EVOMELA, a propylene-glycol free melphalan formulation with improved stability: Spectrum is actively addressing the non-clinical issues raised in the Complete Response Letter regarding the EVOMELA NDA. FDA has granted a Type A meeting for November 6, 2015 and the company believes these issues can be swiftly resolved. Spectrum plans to launch this drug with our existing sales force.

Poziotinib, a potential best-in-class, novel, pan-HER inhibitor: The Company plans to initiate a breast cancer program in the U.S., based on compelling Phase 1 efficacy data in breast cancer patients who had failed multiple other HER-2 directed therapies. In addition, multiple Phase 2 studies funded by our partner, Hanmi Pharmaceuticals, are currently ongoing in South Korea.
Three-Month Period Ended September 30, 2015 (All numbers are approximate)

GAAP Results

Total product sales were $28.5 million in the third quarter of 2015. Total product sales decreased 41% from $47.9 million in the third quarter of 2014.

Product sales in the third quarter included: FUSILEV (levoleucovorin) net sales of $11.1 million, FOLOTYN (pralatrexate injection) net sales of $8.7 million, ZEVALIN (ibritumomab tiuxetan) net sales of $4.8 million, MARQIBO (vinCRIStine sulfate LIPOSOME injection) net sales of $1.3 million and BELEODAQ (belinostat for injection) net sales of $2.6 million.

Spectrum recorded net loss of $18.7 million, or $(0.28) per basic and diluted share in the three-month period ended September 30, 2015, compared to net loss of $11.5 million, or $(0.18) per basic and diluted share in the comparable period in 2014. Total research and development expenses were $9.9 million in the quarter, as compared to $14.4 million in the same period in 2014. Selling, general and administrative expenses were $19.4 million in the quarter, compared to $24.1 million in the same period in 2014.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $7.9 million, or $(0.12) per basic share and diluted share in the three-month period ended September 30, 2015, compared to non-GAAP net income of $5.3 million, or $0.08 per basic and $0.07 per diluted share in the comparable period in 2014. Non-GAAP research and development expenses were $9.4 million, as compared to $14.0 million in the same period of 2014. Non-GAAP selling, general and administrative expenses were $17.2 million, as compared to $21.3 million in the same period in 2014.

2015 Financial Guidance

Spectrum raises guidance on year-end cash to over $125 million, up from the Company’s previous guidance of $110 million excluding any new business development transactions.

On November 4, 2015 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies, reported business highlights and financial results for the third quarter ended September 30, 2015 (Press release, bluebird bio, NOV 4, 2015, View Source;p=RssLanding&cat=news&id=2106830 [SID:1234507954]).

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"2015 continues to be a year of significant advancement for our programs in beta-thalassemia, sickle cell disease and oncology, which is reflected in our strong presence at ASH (Free ASH Whitepaper) this year. We are excited about the progress we’ve made in the third quarter in enrolling the HGB-206 study in severe sickle cell disease, and we have made the decision to increase the enrollment target in order to gather more data and provide additional regulatory options," said Nick Leschly, chief bluebird. "Our balance sheet remains strong, allowing us to continue to execute on our goals heading into 2016. This includes initiating the Phase 1 study for our first clinical oncology program, bb2121, in early 2016 and our plans to present data from the Starbeam study of LentiD in CCALD in the first half of 2016."

Recent Highlights

DATA FROM HGB-204, HGB-205 AND HGB-206 CLINICAL STUDIES OF LENTIGLOBIN TO BE PRESENTED AT ASH (Free ASH Whitepaper) – Updated data from the HGB-204 study of LentiGlobin in beta-thalassemia major and the HGB-205 study in beta-thalassemia major and severe sickle cell disease (SCD) will be highlighted in separate oral presentations at the ASH (Free ASH Whitepaper) annual meeting in December. Initial early data from the HGB-206 study of LentiGlobin in severe SCD will be presented in a poster.

DATA FROM PRE-CLINICAL STUDIES OF BB2121 TO BE PRESENTED AT ASH (Free ASH Whitepaper) — bluebird bio will also present data from its lead oncology program, bb2121 at ASH (Free ASH Whitepaper) – the first bluebird bio oncology data to be presented at a major medical meeting. Three poster presentations will highlight preclinical data and manufacturing improvements made to the anti-BCMA CAR-T program.

INCREASED ENROLLMENT TARGET FOR HGB-206 STUDY IN SEVERE SCD – Announced plan to increase enrollment in the U.S.-based HGB-206 study in severe SCD from eight patients to 20 patients to gain additional data and experience with LentiGlobin in SCD. bluebird bio plans to provide an update on enrollment in all three LentiGlobin clinical studies at the ASH (Free ASH Whitepaper) meeting in December.

PUBLISHED GENOME EDITING PAPER IN SCIENCE TRANSLATIONAL MEDICINE – Along with collaborators at the Center for Immunity and Immunotherapy at Seattle Children’s Research Institute, published "Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template" in Science Translational Medicine. The findings showed that it is possible to edit the CCR5 gene to enable T cells to both resist and kill HIV. They also showed that the megaTAL nuclease platform allows researchers to place edited genes very precisely in the genome, leading to precise location and control of expression of the edited gene.

Third Quarter 2015 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2015 were $901.7 million, compared to $492.0 million as of December 31, 2014, an increase of $409.7 million, which was primarily driven by the June 2015 equity financing.

Revenues: Collaboration revenue was $1.3 million for the third quarter of 2015 compared to $6.3 million for the third quarter of 2014. The decrease is a result of an amendment to our collaboration agreement with Celgene in the second quarter of 2015.

R&D Expenses: Research and development expenses were $30.4 million for the third quarter of 2015, compared to $16.6 million for the same period in 2014, an increase of $13.8 million. The increase in research and development expenses was primarily attributable to a $6.6 million increase in employee compensation and benefit expense to support our overall growth and a $3.6 million increase in expenses necessary to support the advancement of our clinical and pre-clinical programs.

G&A Expenses: General and administrative expenses were $13.7 million for the third quarter of 2015, compared to $6.6 million for the same period in 2014, an increase of $7.1 million. The increase in general and administrative expenses was primarily attributable to a $5.7 million increase in employee compensation and benefit expense to support our overall growth.

Net Loss: Net loss was $42.9 million for the third quarter of 2015, compared to net loss of $17.0 million for the third quarter of 2014.

Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $901.7 million as of September 30, 2015 will be sufficient to fund its current operations through 2018.

On November 4, 2015 Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) reported the presentation of a responder survival analysis from the Phase 3 VALOR trial at the 33rd Annual Chemotherapy Foundation Symposium (CFS) taking place at the Marriott Marquis in New York City (Press release, Sunesis, NOV 4, 2015, View Source;p=RssLanding&cat=news&id=2106984 [SID:1234507978]). The results are being presented today, Wednesday, November 4th, at 6:00 p.m. as a poster during the general poster session in the Exhibit Hall.

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The poster presentation, titled "Impact of Complete Remission on Overall Survival in Patients with Refractory/Relapsed Acute Myeloid Leukemia Treated with Vosaroxin Plus Cytarabine or Placebo Plus Cytarabine: Responder Analysis for the Phase 3 VALOR Trial," will be available on the Sunesis website at www.sunesis.com, following the conclusion of the symposium.
VALOR is a randomized, double-blind, placebo-controlled Phase 3 trial which enrolled 711 adult patients with first relapsed or refractory AML at 124 leading sites in 15 countries. Patients were stratified for age, geographic region and disease status and randomized one to one to receive either vosaroxin and cytarabine or placebo and cytarabine. The full results from VALOR were recently published in the September 2015 print issue of The Lancet Oncology.

A post hoc landmark analysis was performed comparing overall survival (OS) by complete remission (CR) status. To mitigate the potential bias that early death would preclude CR, only patients alive at 60 days were included. Of the 711 patients in the VALOR intent-to-treat population, 570 patients were alive at the 60-day mark, including 285/356 (80%) in the vosaroxin/cytarabine arm and 285/355 (80%) in the placebo/cytarabine arm. At 60 days, the CR rate was 33.0% and 15.4% in the respective treatment arms. The addition of vosaroxin produced the greatest percentage increase in CR rate compared to the control arm in patients ≥ 60 y of age, patients with high blast count, and patients with refractory or early relapsed disease. These same patient groups also show the greatest OS benefit with the addition of vosaroxin. In both treatment arms and all study-strata, achievement of CR was associated with consistently longer median OS; patients with CR at 60 days had a median OS of 20.1 months (21.2 months with vosaroxin/cytarabine and 19.8 months with placebo/cytarabine), and patients without CR had a median OS of 7.1 months (7.3 and 7.1 months, respectively). Irrespective of treatment arm, OS was consistently prolonged in patients with CR. The stratified Chi-square statistical analysis of survival demonstrated a HR for CR of 0.42.

"These data underscore the long-held clinical understanding that CR status is the strongest independent predictor of overall survival in patients with AML, with VALOR demonstrating a two-fold increase in CR with the addition of vosaroxin," said Harry Erba, MD, PhD, Professor of Medicine and Director of the Hematologic Malignancy Program at the University of Alabama at Birmingham. "Importantly, the CR benefit, and thus survival benefit, conveyed by the addition of vosaroxin is most evident in patients over 60 years of age and those with refractory or early relapsed disease. These data further demonstrate the potential of vosaroxin as a much needed new treatment option for patients with relapsed or refractory AML."

About QINPREZO (vosaroxin)
QINPREZO (vosaroxin) is an anti-cancer quinolone derivative (AQD), a class of compounds that has not been used previously for the treatment of cancer. Preclinical data demonstrate that vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis. Both the U.S. Food and Drug Administration (FDA) and European Commission have granted orphan drug designation to vosaroxin for the treatment of AML. Additionally, vosaroxin has been granted fast track designation by the FDA for the potential treatment of relapsed or refractory AML in combination with cytarabine. Vosaroxin is an investigational drug that has not been approved for use in any jurisdiction.
The trademark name QINPREZO is conditionally accepted by the FDA and the EMA as the proprietary name for the vosaroxin drug product candidate.

About AML
AML is a rapidly progressing cancer of the blood characterized by the uncontrolled proliferation of immature blast cells in the bone marrow. The American Cancer Society estimates that there will be approximately 20,830 new cases of AML and approximately 10,460 deaths from AML in the U.S. in 2015. Additionally, it is estimated that the prevalence of AML across major global markets (U.S., France, Germany, Italy, Spain, United Kingdom and Japan) is over 75,000. AML is generally a disease of older adults, and the median age of a patient diagnosed with AML is about 67 years. AML patients with relapsed or refractory disease and newly diagnosed AML patients over 60 years of age with poor prognostic risk factors typically die within one year, resulting in an acute need for new treatment options for these patients.

On November 4, 2015 Eagle Pharmaceuticals, Inc. (NASDAQ:EGRX) ("Eagle" or the "Company") and Spectrum Pharmaceuticals (NASDAQ:SPPI) ("Spectrum") reported that they have entered into a co-promotion agreement ("the agreement") under which the Spectrum 32-person Corporate Accounts Sales Team will dedicate 80 percent of its time to selling and marketing up to six Eagle products over a period of at least 18 months (Press release, Eagle Pharmaceuticals, NOV 4, 2015, View Source [SID:1234507955]). Spectrum will continue to maintain a separate sales force to market existing and potential hematology products.

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The agreement leverages the experience and network of the Spectrum Corporate Accounts Sales Team in order to maximize the respective product sales of both companies by targeting infusion centers, hospitals, and oncology purchasing groups.

The Spectrum Corporate Accounts Sales Team is a group of 32 proven and seasoned professionals focused in the hematology and oncology space, calling on infusion centers and hospitals. Each team member has extensive pharmaceutical industry experience, including years of oncology-specific experience. This cohesive team has demonstrated strong performance and they are poised to commercialize Eagle’s promising assets.

Under the terms of the agreement, the Spectrum Corporate Accounts Sales Team will promote up to six products. This will potentially include Eagle’s current pipeline plus products that may be in-licensed over time. Eagle’s approximately 20 direct sales representatives will focus on promoting Eagle products with an initial emphasis on the launch of RTU Bivalirudin and RYANODEX (dantrolene sodium).

As outlined in the agreement, Eagle will pay Spectrum a base fee of $12.8 million over 18 months. Spectrum will also have the opportunity to earn an additional $9 million in specific identified sales milestone payments if its Corporate Accounts Sales Team surpasses sales projections, for a potential total payment of up to $22 million in base fee and specified milestones over 18 months. The agreement grants both companies the opportunity for six-month renewal periods upon mutual agreement.

"Our agreement with Spectrum is a very positive step at this exciting and critical time for Eagle as we transition from a development-stage pharmaceutical company to a commercial organization with a portfolio of differentiated, in-market products and a continued promising pipeline," said Scott Tarriff, President and Chief Executive Officer of Eagle Pharmaceuticals. "Driven by our commitment to building sustainable, long-term shareholder value, this agreement allows Eagle to make this transition with little commercial risk and minimal financial obligation while we deliberately build our own commercial sales team."

"By partnering with Spectrum, we are capitalizing on a unique opportunity to strategically secure a highly-successful sales team that will spend 80 percent of its time calling exclusively on Eagle’s customers, infusion centers, hospitals, and oncology purchasing groups. We are confident that Spectrum’s proven sales team will help Eagle maximize the success of new product launches and achieve our increased revenue goals while reducing the expense burden prior to and during the launch process," concluded Tarriff.

"Partnering with Eagle is a value-enhancing opportunity as we continue our efforts to bring innovative products for patients and enhance value for Spectrum shareholders," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "The timing of the agreement allows us to quickly leverage our existing infrastructure to launch and market Eagle’s drugs. Moreover, maintaining a cohesive and engaged sales team has long-term strategic value for Spectrum. By working with Eagle to promote its expanding portfolio of marketed products, we are providing the members of the Spectrum Corporate Accounts Sales Team an opportunity to strengthen their partnerships with key stakeholders in the oncology marketplace and participate in the engaging work of launching important therapeutics. We look forward to working closely with Eagle to bring improved specialty products to health care professionals and patients."

Eagle to Hire Approximately 20 Direct Sales Representatives as Part of Long-Term Commercialization Strategy; Sales Team to Form Company’s Internal Commercial Organization

As part of the co-promotion agreement and long-term strategy to build an internal commercial team, Eagle will also hire approximately 20 direct sales representatives who will be a part of Eagle’s independent commercial organization. These representatives will be managed under the Spectrum sales team infrastructure for the duration of the co-promotion agreement. This arrangement serves to ensure a well-trained sales team and facilitates a seamless, low-cost transition with minimal risk.

Eagle expects its in-market portfolio will significantly expand in 2016, assuming FDA approvals for Docetaxel Injection, RTU bivalirudin, and the tentatively approved 500 mL RTD bendamustine. Anticipated approval dates for these products are in December 2015, March 2016, and May 2016, respectively. Combined with anticipated royalties from Teva for rapidly administered bendamustine 50 mL, revenues from RYANODEX, and royalties from commercial partner net product sales for argatroban, the Company expects an increasing revenue stream and expedited ability to deliver long-term, sustainable growth.