Argos Therapeutics to Participate in World Stem Cells and Regenerative Medicine Congress

On May 11, 2016 Argos Therapeutics, Inc. (Nasdaq:ARGS), an immuno-oncology company focused on the development and commercialization of truly individualized immunotherapies for the treatment of cancer based on the Arcelis technology platform, reported the company will participate in the World Stem Cells and Regenerative Medicine Congress to be held May 18-20, 2016 at the Business Design Centre in London (Press release, Argos Therapeutics, MAY 11, 2016, View Source [SID:1234512276]).

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Fred Miesowicz, Ph.D., special advisor and former chief operating officer of Argos, will join a panel titled, "Thinking beyond efficacy — considerations for commercial success," beginning at 5pm BST on Wednesday, May 18th. Dr. Miesowicz will also participate in a roundtable discussion, "Biotech updates: from head-to-toe," at 11:25am BST on Thursday, May 19th.

For more information about the Congress, visit View Source

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

Q1 2016 Interim Financial Report

On May 11, 2016 Bionor Pharma ASA reported Q1 2016 Interim Financial Report (Press release, Bionor Pharma, MAY 11, 2016, View Source [SID:1234512274]).

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HIGHLIGHTS Q1 2016
In the first quarter of 2016, Bionor continued its preparation of the BIOSKILL trial and other clinical activities. Additional clinical trial applications for BIOSKILL were forwarded to competent authorities, and the company also reported further data from the REDUC Part B trial. The company’s working capital was strengthened with the support from existing and new shareholders in a NOK 45 million private placement and a subsequent NOK 16 million repair offering.

REDUC Part B results, primarily related to latent reservoir size, were given as an oral presentation at the prestigious Conference on Retroviruses and Opportunistic Infections (CROI), in Boston, MA (USA)
Bionor announced that the third and final assay for measuring latent HIV reservoir size, the primary endpoint in the REDUC Part B trial, supports that the combination of Vacc-4x and the latency reversing agent romidepsin (Istodax, Celgene) leads to a reduction in latent viral reservoir
Clinical trial applications for the BIOSKILL clinical trial were submitted to authorities in France and Australia, and approval was obtained in Denmark and the United Kingdom. In addition, a request for a pre-IND meeting with the U.S. Food and Drug Administration (FDA) was submitted
Bionor was granted up to NOK 9.2 million from Research Council of Norway to further advance Vacc-4x in a combination treatment regimen
The company’s shareholders approved the completion of a private placement raising NOK 45 million in gross proceeds, which is expected to fund the company through the first half of 2016, and a subsequent repair offering for existing shareholders raising gross proceeds of NOK 16 million
A new Chairman and two new board members were elected at an extraordinary general meeting on 11 March 2016
Net cash flow in Q1 2016 was NOK -31.2 million (Q1 2015: NOK -17.8 million)
Cash and cash equivalents at 31 March 2016 was NOK 41.8 million (31 March 2015: NOK 75.3 million).
Events after the balance sheet date

At the company’s annual general meeting on 22 April 2016, a number of resolutions were rejected by the shareholders. Mr. Steen Krøyer was elected as new board member, replacing Thomas Hofstaetter. An extraordinary general meeting will be convened shortly to handle the rejected proposals
On 26 April 2016, the company announced that Dr. David Horn Solomon and Bionor’s Board of Directors had agreed that Dr. David Horn Solomon on the same day would leave his position as Chief Executive Officer of Bionor Pharma ASA, owing to a shareholder led change in company strategy. The Board of Directors will initiate a search process for a new CEO. Until such search is concluded, the Board has appointed MSc Pharm Unni Hjelmaas as acting CEO
Three board members, Benedicte Fossum, Kirsten Drejer and Jerome B. Zeldis resigned from the Board on 28 April 2016.
Financial guidance for 2016
For the full year 2016, Bionor maintains its financial guidance of a Core cost base in the range of NOK 58-66 million. The Core cost base is defined as Employee Benefit Expenses plus Other operating expenses.

Per S. Thoresen, Chairman of the Board of Directors, commented:
"The new Board maintains its focus on HIV immunotherapy and the overall strategy to advance Vacc-4x in combination with other medicines in order to contribute to a possible functional HIV cure. With 36 million people world-wide living with HIV, it is of paramount importance that treatment options for HIV-positive individuals are improved. We have a strong asset in Vacc-4x, and as Board and company, we look forward to advancing our functional HIV cure strategy to the future benefit of HIV-positive individuals and their caregivers as well as to our shareholders. The timing and details in the clinical development program as well as the core cost base are under consideration in light of the company’s cash position and market capitalization. Any possible changes will be disclosed in due course in a stock exchange announcement."

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Clinical data of Medigene’s dendritic cell (DC) vaccines in AML presented at CIMT conference

On May 11, 2016. Medigene AG (MDG1, Frankfurt, Prime Standard), a clinical stage immune-oncology company focusing on the development of T cell immunotherapies for the treatment of cancer, reported that the academic group of Prof. Gunnar Kvalheim at the Department of Cellular Therapy at the Oslo University Hospital, Norway, has presented clinical data on Compassionate Use[1] patients receiving dendritic cell (DC) vaccines for the treatment of acute myeloid leukaemia (AML) utilizing Medigene’s DC vaccine technology at the 14th Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) in Mainz, Germany (Press release, MediGene, MAY 11, 2016, View Source [SID:1234512273]). CIMT (Free CIMT Whitepaper) is Europe’s largest meeting focused on cancer immunotherapy research and development.

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The poster presentation of the Oslo University Hospital showed preliminary data from five AML patients receiving DC vaccines targeting the antigens Wilm’s tumor-1 (WT-1) and preferentially expressed antigen in melanoma (PRAME) employing Medigene’s new generation monocyte-derived fast dendritic cells. The poster is entitled "WT-1 and PRAME mRNA transfected TLR 7/8 polarized fast DCs vaccines in AML patients raise specific immune responses that correlate with clinical outcome".

AML is frequently diagnosed in elderly patients, who normally cannot tolerate intensive chemotherapy and/or stem cell transplantation, making curative treatment difficult and rates of early relapse high.

Results reported here are from five patients, where DC vaccination was started after hematopoietic recovery from first line chemotherapy treatment.

Data from the first four patients has already been reported at the 57th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2015. Those four patients have now been treated between 16 and 26 months.

Prof. Gunnar Kvalheim, Head of Department of Cellular Therapy, Oslo University Hospital, explains: "Altogether, these results show that fast TLR- polarized DCs can induce or enhance specific T cell responses with a patient individual pattern. Clinical responses are related to immune responses and can result in prolonged survival in AML patients not eligible for curative treatment."

Prof. Dolores J. Schendel, CEO and CSO of Medigene AG, adds: "These positive results encourage us in pursuing our proprietary DC vaccine development program for which we are currently conducting our own Phase I/II clinical AML trial, expanding the ongoing academic clinical studies."

The Oslo University Hospital has an agreement with Medigene for the use of Medigene`s new generation DC vaccines for their ongoing academic clinical studies. Medigene’s DC vaccines are produced according to GMP guidelines at the Department of Cellular Therapy at the Oslo University Hospital. Acute myeloid leukaemia is Medigene’s lead indication in its DC vaccine program.

About Medigene’s DC vaccines: The platform for the development of antigen-tailored DC vaccines is the most advanced platform of the highly innovative and complementary immunotherapy platforms of Medigene Immunotherapies. Currently, Medigene evaluates its DC vaccines in a company-sponsored Phase I/II clinical trial in acute myeloid leukaemia (AML). Further studies utilizing Medigene’s DC vaccine technology include two ongoing clinical investigator-initiated trials (IITs): a clinical Phase I/II trial for treating acute myeloid leukaemia (AML) at Ludwig Maximilians University Hospital Grosshadern, Munich, and a clinical Phase II trial of a treatment for prostate cancer at Oslo University Hospital. Moreover, compassionate use patients are treated with DC vaccines at the Department of Cellular Therapy at Oslo University Hospital.

Dendritic cells (DCs) are the most potent antigen presenting cells of our immune system. Their task is to take up, process and present antigens on their cell surface, which enables them to activate antigen-specific T cells for maturation and proliferation. This way T cells can recognize and eliminate antigen-bearing tumor cells. Dendritic cells can also induce natural killer cells (NK cells) to attack tumor cells. The team of Medigene Immunotherapies GmbH’s scientists has developed new, fast and efficient methods for generating dendritic cells ex-vivo, which have relevant characteristics to activate both T cells and NK cells. The DC vaccines are developed from autologous (patient-derived) precursor cells, isolated from the patient’s blood, and can be loaded with tumor-specific antigens to treat different types of cancer. Medigene’s DC vaccines are in development for the treatment of minimal residual disease or use in combination therapies.

argenx Reports First Quarter 2016 Financial Results and Provides Business Update

On May 11, 2016 argenx N.V. (Euronext Brussels: ARGX), a clinical-stage biopharmaceutical company focused on creating and developing differentiated therapeutic antibodies for the treatment of cancer and severe autoimmune diseases, reported financial results for the first quarter ended 31 March 2016 (Press release, arGEN-X, MAY 11, 2016, View Source [SID:1234512271]).

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"On the strategic side we were very pleased to announce our collaboration with AbbVie last month for the exclusive opt-in right to ARGX-115 which targets the novel immune checkpoint GARP. The collaboration not only provides an upfront payment of $40 million further strengthening our financial position but also provides validation from a global oncology player of our first immuno-oncology asset from our Innovative Access program (IAP), which has quickly generated novel and valuable compounds," said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"We remain very focused on advancing our pipeline programs and on driving forward our ongoing studies in cancer and autoimmune diseases including several upcoming milestones. We have defined the doses for our multiple ascending dose escalation study for ARGX-113; we expect data from this study by the end of July of this year and to announce our Phase 2 plan in the second half of 2016. We will also have data from our two lead oncology programs this year with top-line Phase 1 data of ARGX-110 in T-cell lymphoma in the third quarter of 2016 and top-line data from the Phase 1 safety expansion cohort of ARGX-111 by mid-2016."

FIRST QUARTER 2016 AND RECENT HIGHLIGHTS

In the first quarter of 2016, the Company

Announced initial results from a Phase 1 single ascending dose (SAD) study of ARGX-113, a potential breakthrough therapy for severe autoimmune diseases. Results showed compound to be safe and well-tolerated across all doses in healthy volunteers. Additionally, observed promising pharmacodynamic (PD) effects for speed, depth and duration of IgG reduction.
Launched three clinical sites in South Korea to recruit MET-amplified cancer patients for Phase 1 safety expansion cohort of ARGX-111.
Received milestone payment from LEO Pharma collaboration (initiated in May 2015) to develop antibody-based treatments for inflammatory skin conditions.
Received EUR 16 million investment by U.S. funds advised by subsidiaries of Federated Investors.
argenx partner, Bird Rock Bio (formerly RuiYi), a company focused on the discovery and development of novel biologic therapies, announced that gerilimzumab, a novel SIMPLE AntibodyTM equipped with argenx’s proprietary NHance technology neutralizing the IL-6 cytokine, demonstrated safety and pharmacokinetics that support low, infrequent dosing and the potential for favorable pricing.

More recently, the Company

Initiated a collaboration with AbbVie to develop and commercialize ARGX-115. ARGX-115, a preclinical-stage human antibody program targeting the novel immuno-oncology target GARP, is a protein that is believed to contribute to immunosuppressive effects of T-cells. argenx will receive an upfront payment of $40 million and near-term preclinical milestones of $20 million from AbbVie in return for the exclusive option to license ARGX-115. argenx is also eligible to receive additional development, regulatory and commercial payments up to $625 million upon achievement of pre-determined milestones as well as tiered, up to double-digit royalties on net sales upon commercialization.

FINANCIAL HIGHLIGHTS (as of 31 March 2016) (compared to financial highlights as of 31 March 2015)

Operating income of EUR 2.8 million (31 March 2015: EUR 1.8 million).
Net loss of EUR 3.0 million (31 March 2015: EUR 3.0 million).
Cash position of EUR 53.8 million (cash, cash-equivalents and financial assets) (31 March 2015: EUR 52.2 million) allowing Company to pursue development of its product portfolio in line with its communicated business plan.

UPCOMING CLINICAL MILESTONES

ARGX-113

Top-line data from Phase 1 Multiple Ascending Dose (MAD) study by the end of July 2016
Announcement of two indications for Phase 2 proof-of-concept studies in second half of 2016, including myasthenia gravis

ARGX-110

Interim data from Phase 1 study in T-cell lymphoma (TCL) in the third quarter of 2016 and top-line data by the end of 2016
Initiate first combination study in 2H 2016
Ongoing enrollment in Phase 1 safety expansion cohort with Stage 4 nasopharyngeal carcinoma patients; enrollment completion by the end of 2016

ARGX-111

Interim data of Phase 1b safety expansion cohort by mid- 2016

KEY FIGURES (CONSOLIDATED)

Financial overview

Period ended
Period ended

in thousands of euros
March 31, 2016
March 31, 2015
Variance

Revenue
2,216
1,210
1,006
Other operating income
619
614
5
Total operating income
2,835
1,825
1,011
Research and development expenses
(4,408)
(3,950)
(458)
General and administrative expenses
(1,401)
(1,105)
(296)
Operating profit/(loss)
(2,974)
(3,230)
257
Financial income
42
91
(50)
Exchange gains/(losses)
(38)
160
(198)
Profit/loss for the period
(2,970)
(2,979)
9
Net increase (decrease) in cash, cash-equivalents and financial assets
11,520
(3,809)
15,329
Cash, cash-equivalents and financial assets at the end of the period
53,847
52,164
1,683

DETAILS OF THE FINANCIAL RESULTS

For the three-month period ended 31 March 2016, operating income reached EUR 2.8 million compared to EUR 1.8 million during the same period in 2015. The higher operating income in the first quarter of 2016 is mainly explained by the partial recognition of the upfront payment received following the signature of a partnership with LEO Pharma in May 2015.

Research and development expenses were EUR 4.4 million and EUR 4 million for the three-month period ended 31 March 2016 and 2015, respectively. The EUR 0.4 million increase in R&D expenses in the first three months of 2016 corresponds principally to increased clinical trial activities and to the recruitment of additional R&D personnel.

General and administrative expenses amounted to EUR 1.4 million in the first three months of 2016 compared to EUR 1.1 million on 31 March 2015. The EUR 0.3 million increase in the first quarter of 2016 is principally explained by expenses related to the move of the company to its new premises, the implementation of a new IT infrastructure and the recruitment of additional G&A personnel for supporting the operational activities of the company.

The Company generated a net loss of EUR 3 million in the three-month periods ended 31 March 2016 and 31 March 2015.

The Company’s cash, cash equivalents and financial assets amounted to EUR 53.8 million on 31 March 2016 compared to EUR 42.3 million on 31 December 2015 and EUR 52.2 million on 31 March 2015.

FINANCIAL CALENDAR

26 August 2016: Half year 2016 business update and financial results
27 October 2016: Q3 2016 business update and financial results

CytRx Reports First Quarter 2016 Financial Results

On May 11, 2016 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported financial results for the three months ended March 31, 2016, and provided an overview of recent accomplishments and upcoming milestones for its research and development programs (Press release, CytRx, MAY 11, 2016, View Source;p=RssLanding&cat=news&id=2167453 [SID:1234512256]).

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"So far, 2016 has been a very productive year for CytRx," said Steven A. Kriegsman, Chairman and CEO of CytRx. "On the clinical front, we reached the 191 progression events in our global, pivotal Phase 3 trial with aldoxorubicin in second-line soft tissue sarcoma to trigger the data verification and analysis. We look forward to announcing top-line results at the end of June 2016. We announced that we will present updates on three aldoxorubicin clinical trials at the ASCO (Free ASCO Whitepaper) Annual Meeting in June 2016. Additionally, our team recently presented data at the American Association of Cancer Research Annual Meeting on our LADRTM technology platform and on DK049, the first drug to emerge from that platform."

Mr. Kriegsman continued: "On the corporate side, we added commercial oncology expertise with the addition of Ms. Olivia Ware to our executive management team. We also raised an additional $25 million in non-dilutive capital to fund clinical and pre-commercial activities for aldoxorubicin."

First Quarter 2016 and Recent Highlights

Bolstered the Management Team. In January 2016, CytRx hired Ms. Olivia Ware as Chief Commercial Officer. Ms. Ware brings more than 20 years of biotechnology and pharmaceutical experience, including thirteen years at Genentech where she was responsible for key aspects of the launches of the oncology drugs Rituxan, Herceptin and Avastin. While at Genentech, Ms. Ware was a Senior Director of Oncology and was responsible for the initial commercial launch of Avastin in the U.S., which reached $1 billion in sales during its first year on the market. Prior to this, she was head of Herceptin marketing and held a number of other positions in the commercial organization. Later, Ms. Ware was a Senior Director in the Product Portfolio Management Group, managing approximately 20 senior leaders responsible for building and leading the cross-functional drug development teams that developed and implemented strategic plans and guided the drug development processes for all oncology products at Genentech.

Strengthened the Balance Sheet. In February 2016, CytRx entered into a long-term loan for up to $40 million with Hercules Technology Growth Capital. The initial tranche of $25 million was received, and CytRx can access an additional $15 million, subject to certain research and development milestones.

Reached the Target Number of Events in the Global Pivotal Phase 3 Trial. In April 2016, CytRx achieved the target number of progression events in the aldoxorubicin global, pivotal Phase 3 trial in patients with second-line soft tissue sarcomas. Our contract research organization started the collection and verification of the trial data from all 433 patients enrolled at 79 sites around the globe. CytRx expects to report top-line results following the analysis of the data in June 2016.

Presented Data on LADRTM Technology Platform and DK049. In April 2016, CytRx presented two posters at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting detailing both our Linker Activated Drug Release (LADRTM) Platform and DK049, an albumin-binding derivative of the widely used chemotherapy agent gemcitabine combined with our LADRTM technology. DK049 was nominated for clinical development in 2015.

Upcoming Milestones

Present updated aldoxorubicin clinical trial results at the ASCO (Free ASCO Whitepaper) Annual Meeting in June 2016 from the ongoing Phase 2 clinical trial in patients with unresectable glioblastoma, the Phase 2 clinical trial in Kaposi’s sarcoma and from the Phase 1b combination trial with gemcitabine in patients with advanced cancers
Announce top-line results from the global pivotal Phase 3 clinical trial with aldoxorubicin as a treatment for patients with second-line soft tissue sarcomas
Present aldoxorubicin pivotal Phase 3 clinical trial results at a major medical meeting
Complete enrollment in the global Phase 2b clinical trial evaluating aldoxorubicin in patients with second-line small cell lung cancers
First Quarter 2016 Financial Results

CytRx reported cash, cash equivalents and short term investments of $68.2 million as of March 31, 2016.

Net loss for the quarter ended March 31, 2016 was $12.6 million, or $0.19 per share, compared with a net loss of $17.5 million, or $0.31 per share, for the quarter ended March 31, 2015. During the first quarter of 2016, CytRx recognized a non-cash loss on warrant derivative liability of $0.2 million, compared to a non-cash loss of $1.9 million for the three-month period ended March 31, 2015. The Company did not recognize revenues for the first quarters of 2016 or 2015.

Research and development (R&D) expenses were $8.2 million for the first quarter of 2016, and included development expenses of $5.8 million for the aldoxorubicin program. R&D expenses were $12.6 million for the first quarter of 2015.

General and administrative (G&A) expenses were $4.0 million for the first quarter of 2016, compared to $3.1 million for the first quarter of 2015.

About Soft Tissue Sarcoma
Soft tissue sarcoma is a cancer occurring in muscle, fat, blood vessels, tendons, fibrous tissues and connective tissue, and can arise anywhere in the body at any age. According to the American Cancer Society, there are approximately 50 types of soft tissue sarcomas. In 2013 more than 11,400 new cases were diagnosed in the U.S. and approximately 4,400 Americans died from this disease. In addition, approximately 40,000 new cases and 13,000 deaths in the U.S. and Europe are part of a growing underserved market.

About Small Cell Lung Cancer
An estimated 1.6 million new cases of lung cancer are diagnosed worldwide each year. In the Western world, approximately 13-15% of cases are SCLC, a deadly form of lung cancer associated with tobacco use. The five year survival rate is less than 7%, in part because an estimated 70% of patients have extensive disease at diagnosis. According to the National Cancer Institute, more than 30,000 new cases will be diagnosed in the USA in 2014. The estimated 2014 SCLC incidences for Europe and Asia are over 58,000 and 136,000, respectively.

About Glioblastoma Multiforme
Glioblastoma is the most common and most malignant primary brain tumor in adults and afflicts more than 12,000 new patients in the U.S. annually. The median survival after diagnosis is approximately 14 months, despite patients subsequently receiving surgical resection, radiotherapy and chemotherapy. Limited efficacy of chemotherapeutic agents has been attributed to several contributing factors including insufficient drug delivery to the tumor site through the blood:brain barrier.

About Aldoxorubicin
The widely used chemotherapeutic agent doxorubicin is delivered systemically and is highly toxic, which limits its dose to a level below its maximum therapeutic benefit. Doxorubicin also is associated with many side effects, especially the potential for damage to heart muscle at cumulative doses greater than 450 mg/m2. Aldoxorubicin combines doxorubicin with a novel single-molecule linker that binds directly and specifically to circulating albumin, the most plentiful protein in the bloodstream. Protein-hungry tumors concentrate albumin, thus increasing the delivery of the linker molecule with the attached doxorubicin to tumor sites. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released. This allows for greater doses (3 ½ to 4 times) of doxorubicin to be administered while reducing its toxic side effects. In studies thus far there has been no evidence of clinically significant effects of aldoxorubicin on heart muscle, even at cumulative doses of drug well in excess of 2,000 mg/m2.

About DK049
DK049 is a novel LADRTM-enhanced derivative of the commonly used chemotherapeutic agent gemcitabine. Like aldoxorubicin, DK049 binds to circulating albumin for transport to the tumor. It incorporates a novel dual-releasing linker that is cleaved in an acidic environment and requires a specific enzyme for prolonged release of the active drug at the tumor site. DK049 appears to overcome the major deactivation and resistance mechanisms that limit the effectiveness of gemcitabine. Preclinical studies in animal models of non-small cell lung, pancreatic and ovarian cancers showed superior anti-tumor activity compared to gemcitabine. CytRx recently presented preclinical data on DK049 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2016.