On May 12, 2016 ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE MKT: IMUC) reported financial results for the first quarter of 2016 (Press release, ImmunoCellular Therapeutics, MAY 12, 2016, View Source [SID:1234512328]).

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Andrew Gengos, ImmunoCellular Chief Executive Officer, commented: "We continue to make progress in all aspects of implementing our ICT-107 registration trial in patients with newly diagnosed glioblastoma. Over half of the targeted clinical sites in the US have been activated and are screening patients. We anticipate that all sites in the US can be activated by the middle of 2016. In Europe, the process of gaining regulatory approval for the trial and bringing clinical trial sites online is going very well. Clinical trial applications have been approved by the regulatory authorities in the Netherlands and the UK, and we are involved in interactions with the six other country authorities where the trial will be conducted. We also have received regulatory approval to start the trial in Canada. We expect to manufacture clinical supplies for the first qualifying patients in Canada and Europe in the third quarter. As the competitive landscape in newly diagnosed glioblastoma evolves, we continue to think that our phase 3 registration program, with the strong foundational support of placebo-controlled phase 2 data, is the best designed program underway in this indication. We are pleased with the progress we made in the first quarter, and believe that 2016 will be a year of accomplishment and value creation for our company."

For the quarter ended March 31, 2016, ImmunoCellular incurred a net loss of $5.6 million, or $0.06 per basic and diluted share, compared to a net loss of $1.4 million, or $0.02 per basic and diluted share, for the quarter ended March 31, 2015.

During the first quarter 2016, the Company incurred $4.7 million of research and development expenses compared to $2.1 million in the prior year quarter while general and administrative expenses remained relatively constant between periods. The $2.6 million increase in research and development expenses primarily reflects the additional expenses associated with the phase 3 trial of ICT-107. During the first quarter of 2016, the Company recorded a credit to other income of $500,000 related to reflect a write-down of the Company’s warrant liability, compared to a credit to other income of $1.8 million during the same period in 2015.

The Company also reported that cash used in operations in the first quarter of 2016 was $5.4 million compared to $3.0 million in the prior year quarter. The increase primarily reflects that additional research and development expenditures in the current year. As of March 31, 2016, the Company had $17.5 million in cash.

On May 12, 2016 OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported ts first quarter 2016 financial results and provided a summary of anticipated milestones (Press release, OncoGenex Pharmaceuticals, MAY 12, 2016, View Source [SID:1234512331]).

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Financial Results and Anticipated Near-term Milestones

As of March 31, 2016, the company’s cash, cash equivalents, and short-term investments were $46.1 million compared with $55.2 million as of December 31, 2015.

Based on current expectations, OncoGenex believes that its cash, cash equivalents, and short-term investments will be sufficient to fund its currently planned operations into the third quarter of 2017. Depending on timing of enrollment or event-driven final analyses, the expected key milestones and activities are as follows:

Custirsen
Announcing results from the AFFINITY trial, the phase 3 trial evaluating a survival benefit for custirsen in combination with cabazitaxel as second-line chemotherapy in approximately 630 patients with castrate-resistant prostate cancer. The final analysis for the intent-to-treat population is expected in the third quarter of 2016.
Announcing results from the ENSPIRIT trial, the phase 3 trial evaluating a survival benefit for custirsen in combination with docetaxel as second-line chemotherapy in approximately 700 patients with non-small cell lung cancer. The final survival analysis is expected in the first half of 2017.
Apatorsen
Announcing results from the Borealis-2 trial, an investigator-sponsored, randomized phase 2 trial evaluating apatorsen in combination with docetaxel treatment compared to docetaxel treatment alone in patients with advanced or metastatic bladder cancer. Final results are expected in the second half of 2016.
Announcing survival results from the Spruce trial, the investigator-sponsored, randomized, placebo-controlled phase 2 trial evaluating apatorsen treatment with carboplatin and pemetrexed chemotherapy in patients with previously untreated advanced non-squamous NSCLC. Overall survival results, including evaluation of patients with high baseline serum Hsp27 levels, are expected in the second half of 2016.
Completing a submission-ready investigational new drug application regarding apatorsen via intravesical administration in combination with Bacillus Calmette-Guerin (BCG) treatment in patients with non-muscle invasive bladder cancer.
Revenue for the three months ended March 31, 2016 and 2015 was $2.9 million and $1.4 million, respectively. The increased revenue in 2016 as compared to 2015 was due to higher collaboration revenue recognized on the deferred revenue as a result of higher ENSPIRIT costs. This was partially offset by a decrease in collaboration revenue recognized for the AFFINITY trial as a result of a decrease in associated clinical activities.

Total operating expenses for the three months ended March 31, 2016 and 2015 were $7.4 million and $6.4 million, respectively. Net loss for the three months ended March 31, 2016 and 2015 was $3.7 million and $4.5 million, respectively.

As of May 12, 2016 OncoGenex had 29,914,226 shares outstanding.

On May 11, 2016 Ability Pharmaceuticals, SL reported that the company has entered into an agreement with the NASDAQ-listed US company SciClone Pharmaceuticals, Inc. (SCLN) granting SciClone an exclusive license to develop and market the novel anticancer ABTL0812 in China and some adjacent territories (Press release, Ability Pharmaceuticals, MAY 11, 2016, View Source [SID1234562099]).

Under the terms of the agreement, AbilityPharma has granted SciClone exclusive rights to develop, promote, market, distribute and sell ABTL0812 in China, as well as Hong Kong, Macau, Taiwan and Vietnam, getting access to follow-up compounds. AbilityPharma will receive an upfront payment and research funding as well as development, regulatory and sales milestone payments, potentially totaling more than $20 million; AbilityPharma will also be eligible to receive royalties on sales. SciClone will be responsible for all aspects of development, product registration and commercialization in the partnered regions, having access to data generated by AbilityPharma.

Carles Domènech, Chief Executive Officer and co-founder of AbilityPharma, stated: "We are excited to partner with SciClone, a NASDAQ US company that has a strong presence in such a rapidly growing market like China, which shares our enthusiasm and sense of urgency for bringing important new cancer therapeutics to patients. The collaboration with SciClone represents meaningful support for our development efforts. This agreement represents the first one signed for China by the Catalan and Spanish Biotech Industry, and we are very proud of it."

About ABTL0812

ABTL0812 is a first-in-class PI3K/Akt/mTOR signaling pathway inhibitor for the treatment of solid tumors. ABTL0812 has a novel mechanism of action, which has shown both in vitro and in vivo high efficacy in tumor settings, including resistant cancer models. It up-regulates TRIB3 leading to inhibition of Akt phosphorylation, which results in decreased activity of mTORC1 (Published in Clinical Cancer Research, Erazo et al., December 2015). ABTL0812 is active in models of multiple solid tumors as single agent and potentiates the anti-cancer effect of chemotherapy as well. The compound plays also a relevant role in tumor stem cells.

AbilityPharma has completed a Phase 1/1b clinical trial of orally administered ABTL0812 in 29 advanced cancer patients with solid tumors and presented the study results at AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) (Vidal et al.) in Boston in November 2015. This first-in-humans clinical study was designed to explore ABTL0812’s safety and efficacy profile. Outstanding safety and tolerability with several long-term disease stabilizations were demonstrated in patients progressing after previous chemotherapy lines, two of them stabilized over one year. The study showed the inhibition of the PI3K/Akt/mTOR pathway based on biomarkers, with correlation with ABTL0812 plasma levels. A full Phase 2 clinical program has been designed in several advanced tumor indications including endometrial and lung cancer (ongoing). US FDA and Europe EMA granted ABTL0812 Orphan Drug Status for the pediatric cancer neuroblastoma.

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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

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.