New BGB324 study points to novel mechanism to enhance immunotherapy efficacy

On September 25, 2016 Leading oncology biopharmaceutical company BerGenBio AS, reported important new preclinical study data on its first-in-class AXL inhibitor, BGB324 (Press release, BerGenBio, SEP 25, 2016, View Source [SID:SID1234515416]). The study showed AXL to be a key factor in tumour resistance to the emerging class of new immune checkpoint inhibitors that can be targeted through combination therapy with BGB324. The study data was presented in a poster today at CRI-CIMT-EATI-AACR – The 2nd International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper), in New York.

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The new study evaluated whether BGB324 used in combination with immune checkpoint inhibitors (anti-CTLA-4 and anti-PD-1) in mouse carcinoma models enhanced the effect of immune checkpoint blockade in aggressive adenocarcinomas displaying limited immunogenicity. BGB324 is a highly selective small molecule inhibitor of the AXL receptor tyrosine kinase. An important regulator of the tumour cell plasticity related to EMT (Epithelial-to-Mesenchymal Transition), AXL signalling is also a key suppressor of the innate anti-tumour immune response. Thus AXL contributes uniquely to both tumour intrinsic and extrinsic mechanisms that suppress anti-tumour immunity. The new study shows that AXL was induced in tumours by immune checkpoint inhibitor treatment and postulated that this could limit their efficacy.

BGB324 is the only selective AXL inhibitor currently in clinical development. Phase Ib clinical trials are underway as single agent and in combination with standard of care drug (cytarabine) in acute myeloid leukaemia (AML), and in combination with erlotinib in non-small cell lung cancer (NSCLC).

Richard Godfrey, Chief Executive Officer of BerGenBio, commented:
"We believe this strong new preclinical data clearly demonstrates the rationale for combining BGB324 with immune checkpoint inhibitors to treat aggressive cancers. This study shows that these inhibitors actually increase AXL expression that supports immune evasion. Treatment with BGB2324 counters this, increasing tumor immunogenicity and promoting the anti-tumour response. In addition to the ongoing development of BGB324 in AML and NSCLC, this data suggests that BGB324 could also be used in combination with cancer immunotherapeutic agents to enhance their efficiency."

Navidea Achieves $1 Million in Lymphoseek® Commercial Milestones

On September 22, 2016 Navidea Biopharmaceuticals, Inc. (NYSE MKT:NAVB) reported it will receive payments totaling $1 million from two recently achieved Lymphoseek commercial milestones under its distribution agreements with U.S. partner Cardinal Health, Inc. (Cardinal) and European partner SpePharm AG, an affiliate of Norgine B.V. (Norgine) (Press release, Navidea Biopharmaceuticals, SEP 22, 2016, View Source;p=RssLanding&cat=news&id=2205270 [SID:SID1234515279]). Navidea will collect a $500,000 milestone payment from Cardinal based on the sale of a 100,000th patient dose of Lymphoseek (technetium Tc 99m tilmanocept) injection since launch. The Company will also receive a $500,000 payment from Norgine resulting from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) positive opinion for the Lymphoseek 50 microgram kit for radiopharmaceutical preparation, a reduced-mass, single-dose vial appropriate for the radiopharmaceutical distribution model in Europe.

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"We are pleased with the meaningful progress that Navidea and its partners have made in our commercial distribution efforts in both the U.S. and Europe," said Jed Latkin, interim Chief Operating Officer and Chief Financial Officer. "These milestones reflect the growing acceptance of Lymphoseek in the U.S. for improving the outcomes in patients with melanoma, breast and oral cavity cancers and signal the expected European launch of Lymphoseek in Q42016."

Lymphoseek is approved in the U.S. by the U.S. Food and Drug Administration (FDA) for use in lymphatic mapping to locate lymph nodes draining a primary tumor site in patients with solid tumors for which this procedure is a component of intraoperative management and for guiding Sentinel Lymph Node Biopsy (SLNB) using a handheld gamma counter in patients with node negative squamous cell carcinoma of the oral cavity, breast cancer or melanoma. Lymphoseek is indicated in the EU for imaging and intraoperative detection of sentinel lymph nodes draining a primary tumor in adult patients with breast cancer, melanoma, or localized squamous cell carcinoma of the oral cavity.1

Xenetic Biosciences to Present at the Ladenburg Thalmann 2016 Healthcare Conference

On September 22, 2016 Xenetic Biosciences, Inc. (OTCQB: XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company developing next-generation biologic drugs and novel orphan oncology therapeutics, reported that Scott Maguire, CEO of Xenetic Biosciences, will present at the Ladenburg Thalmann 2016 Healthcare Conference on Tuesday, September 27, 2016 at 3:30 p.m. ET in New York at the Sofitel New York (Press release, Xenetic Biosciences, SEP 22, 2016, View Source [SID1234537812]).

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During his presentation, Mr. Maguire will provide a corporate update and discuss the Company’s clinical and regulatory strategies for its product candidates currently in development in-house and with Xenetic’s biotechnology and pharmaceutical partners. The Company’s product pipeline currently includes Virexxa (sodium cridanimod), which is being evaluated for the treatment of endometrial cancer and triple negative breast cancer, ErepoXen, a polysialylated form of erythropoietin (EPO), a hormone produced by the kidneys to maintain red blood cell production and prevent anemia, and OncoHist, which is being evaluated for the treatment of acute myeloid leukemia (AML) in refractory patients and refractory non-Hodgkin lymphoma (NHL).

Mr. Maguire will also discuss Xenetic’s $100 million license deal with Shire and provide an overview of the product candidate and clinical status.

A live webcast of the presentation will be available by accessing the IR Calendar in the Investors section of the Xenetic website (www.xeneticbio.com). The webcast replay will be available approximately two hours after the presentation ends and will be accessible for 90 days.

Nicox first half 2016 business and financial update

On September 21, 2016 Nicox S.A. (Euronext Paris: FR0013018124, COX), the international ophthalmic R&D company, reported its financial results for the six months ended June 30, 2016, and provided an update on its activities (Press release, NicOx, SEP 21, 2016, View Source [SID:SID1234515282]).

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"Following the recently completed transaction with GHO Capital involving our European and International commercial assets, we are now well positioned to refocus our resources on our promising R&D pipeline as we await FDA approval decisions for latanoprostene bunod and AC-170" commented Michele Garufi, Chairman and Chief Executive Officer of Nicox. "This transaction allows us to reduce our infrastructure costs by €6 million (approximately 66%) and, together with the recent financing of €18 million, to accelerate the development of our proprietary pipeline assets, including obtaining clinical proof-of-concept data for NCX 4251 and NCX 470 by the end of 2018."

Operational highlights for the six months to June 30, 2016 and post-reporting period

In August 2016, Nicox completed the transfer of its European and International commercial operations to a new pan-European ophthalmic specialty pharmaceutical company led by GHO Capital. The transaction was valued at up to €26 million and the Company received a €9 million upfront cash payment upon closing of the transaction and a minority stake in the new company.
In July 2016, Nicox completed a financing through a reserved capital increase of ordinary shares of the Company with a specific category of investors. Gross proceeds from the financing were €18 million.
On July 21, 2016, latanoprostene bunod licensee Bausch + Lomb (a wholly-owned subsidiary of Valeant Pharmaceuticals International, Inc.) announced its receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for the use of latanoprostene bunod for the treatment of glaucoma. The CRL cited concerns pertaining to a Current Good Manufacturing Practice (CGMP) inspection at Bausch + Lomb’s manufacturing facility in Tampa, Florida. The FDA’s letter did not identify any efficacy or safety concerns with respect to the latanoprostene bunod NDA or additional clinical trials needed for the approval of the NDA. Bausch + Lomb is currently working with the FDA to resolve and address the CGMP concerns. Latanoprostene bunod has the potential to generate significant revenue for Nicox through milestones (up to $132.5 million net, mainly on commercial sales targets) and royalties (potential net tiered royalties on sales from 6% to 11%).
In June, Nicox announced that the U.S. FDA had accepted the Company’s NDA for AC-170, a novel, proprietary eye drop formulation of cetirizine, targeting the treatment of ocular itching associated with allergic conjunctivitis. The FDA also granted Priority Review and assigned a Prescription Drug User Fee (PDUFA) goal date of October 18, 2016 (contingent upon the information and data provided by Nicox during the review period). Approval of the AC-170 NDA prior to December 1st, 2016 will trigger a milestone payment of $35 million in Nicox shares to former Aciex shareholders or $10 million in Nicox shares if approval of the NDA is received after this date. Nicox is currently in partnering discussions in the United States for this program.
Other Updates

• Philippe Masquida, EVP, Managing Director European & International Operations, will be leaving the company at the end of September 2016, following the successful completion of the transfer of the European and International commercial operations. Nicox sincerely thanks Philippe for his years of service and his significant contributions to the commercial business.

First-half financial summary2

The Group’s revenues have been retreated following reclassification of the European commercial business as Discontinued Operations. For information, the European and International product sales of the operations transferred to VISUfarma BV, the new Group led by GHO Capital, were €7.1 million over the first half of 2016, an increase of 50% compared to the same period in 2015.

Excluding Discontinued Operations the operating expenses for the period were €12.0 million compared to €8.8 million for the six months to June 2015. The increase in operating expenses over the period is mainly explained by costs related to the submission of the AC-170 NDA.

Excluding the Discontinued Operations, the Group recorded a net loss of €12.3 million as of June 2016, compared to a net loss of €10.1 million3 at the same date in 2015.

The Group had cash, cash equivalents and financial instruments of €12.3 million as of June 30, 2016, compared to €29 million on December 31, 2015; however, considering the financing in July of this year of €18 million gross and the €8.9 million cash payment following the transfer of the European commercial operations to VISUfarma BV, our unaudited cash, cash equivalents and financial instruments at August 31, 2016 are estimated at €34.1 million.

The half-yearly financial report will be available in French by the end of September 2016 on Nicox’s website www.nicox.com in the section Investor Information > Regulated information > Financial Information.

The procedures relating to the limited review of the interim financial statements have been carried out. The limited review report will be issued after the finalisation of the procedures required for the publication of the-first half financial report.

Peregrine Pharmaceuticals Provides Update on Oral Presentation of Top-Line Data from Phase III SUNRISE Trial of Bavituximab at European Society for Medical Oncology (ESMO) 2016 Congress

On September 21, 2016 Peregrine Pharmaceuticals, Inc. (NASDAQ:PPHM) (NASDAQ:PPHMP), a biopharmaceutical company committed to improving patient lives by manufacturing high quality products for biotechnology and pharmaceutical companies and advancing its proprietary R&D pipeline, reported that top-line data from the Phase III SUNRISE trial of bavituximab in patients with previously treated locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) will be presented as a late-breaking oral presentation at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress (Press release, Peregrine Pharmaceuticals, SEP 21, 2016, View Source [SID:SID1234515262]). Presented data will include a biomarker in the SUNRISE trial that correlated with a statistically significant improvement in overall survival for patients treated with bavituximab in combination with docetaxel compared to patients treated with docetaxel alone. Peregrine will file a new patent application directed to the use of the biomarker prior to the presentation of results at the ESMO (Free ESMO Whitepaper) 2016 Congress, which is being held October 7-11, 2016 in Copenhagen, Denmark.

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Details of the Phase III SUNRISE trial data presentation are as follows:
Presentation Number: LBA45
Presentation Title: Top-line results from SUNRISE: A Phase III Randomized Double-Blind, Placebo-Controlled Multicenter Trial of Bavituximab Plus Docetaxel in Patients with Previously Treated Stage IIIb/IV Non-Squamous Non-Small Cell Lung Cancer
Date: Monday, October 10, 2016
Time: 9:15 a.m. (local time in Copenhagen)

"I want to start by thanking the patients, investigators and scientists involved in the SUNRISE trial that have made possible the continuing collection and analysis of important data from the study. While the current interim analysis is still ongoing, it is exciting to already see that a biomarker associated with positive outcomes for patients receiving the combination of bavituximab with docetaxel has been identified. In the evolving cancer therapeutic space, biomarker identification is playing an increasingly critical role in guiding clinical development strategies and trial designs," said Steven W. King, president and chief executive officer of Peregrine. "It is important to note that we are undertaking a broad biomarker analysis effort as part of the SUNRISE trial and this initial set of data is just the first of what we hope will be several findings that will help guide the bavituximab clinical program going forward. We look forward to presenting results from this ongoing analysis effort at the ESMO (Free ESMO Whitepaper) 2016 Congress, as well as other medical conferences as the additional data becomes available."

The primary goal of the biomarker analysis is to identify a biomarker profile for patients that receive the most benefit from a bavituximab-containing therapeutic regimen. As specified in the study protocol, thousands of patient samples were collected to potentially identify biomarkers associated with improved outcome for patients receiving bavituximab. Peregrine is in the process of filing a new patent application directed to the use of the initial biomarker discovery which will be presented at the ESMO (Free ESMO Whitepaper) 2016 Congress. Additional patient sample testing and analysis is ongoing and may result in other biomarkers of importance.

Bavituximab is an investigational chimeric monoclonal antibody that targets phosphatidylserine (PS). Signals from PS inhibit the ability of immune cells to recognize and fight tumors. Bavituximab is believed to override PS mediated immunosuppressive signaling by blocking the engagement of PS with its receptors as well as by sending an alternate immune activating signal. PS targeting antibodies have been shown to shift the functions of immune cells in tumors, resulting in multiple signs of immune activation and anti-tumor immune responses.

Peregrine’s clinical development strategy for bavituximab is currently focused on small, early-stage proof-of-concept trials evaluating the drug in combination with other cancer treatments. The intent behind this strategy is to control research and development costs, while continuing to generate clinical data to further validate bavituximab’s combination potential that will be critical to bringing onboard a partner to help advance the program.