On May 12, 2016 GenVec, Inc. (NASDAQ: GNVC) reported financial results for the first quarter ended March 31, 2016 (Press release, GenVec, MAY 12, 2016, View Source [SID:1234512307]). For the three months ended March 31, 2016, the company reported a net loss of $1.9 million, or $0.11 per share, compared with a net loss of $1.5 million, or $0.09 per share, for the three months ended March 31, 2015. The company ended the first quarter with $6.9 million in cash, cash equivalents, and liquid investments.

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"The recommendation to continue the Phase 1/2 study of CGF166 by the trial’s Data Safety Monitoring Board (DSMB) was an essential hurdle to pass," said Douglas J. Swirsky, president and CEO of GenVec. "The decision was based on a thorough and objective review of the available safety and efficacy data from all patients enrolled in the study. As a result, we believe the trial is on track to be completed in 2017 as previously expected."

"Our partnered pipeline continues to advance, and we expect our partner TheraBiologics to advance a second-generation neural stem cell-based cancer treatment utilizing our technology into the clinic later this year," Mr. Swirsky continued. "Our recent financing further enables GenVec to remain focused on finding new collaborations to maximize the value of our AdenoVerse gene delivery platform and we are excited by the response from potential partners."

Cash Position

As of May 10, 2016, the company had $10.6 million in cash, cash equivalents, and liquid investments (unaudited), which includes the proceeds of the company’s May 2016 financing.

Updated 2016 Guidance

For 2016, GenVec anticipates a cash burn between $6.0 million and $6.5 million and believes its existing resources are sufficient to fund operations into 2018.

Financial Results for the Three Months Ended March 31, 2016

Revenues for the three-month period ended March 31, 2016 were $0.3 million, which represents a decrease of 28% as compared to revenues of $0.4 million in the comparable prior year period.

The decrease in revenue for the three-month period ended March 31, 2016 is primarily attributable to the completion of our contract with the DHS related to our animal health program in February 2015. In connection with this contract we recognized $0.2 million in revenue in 2015 with no corresponding revenue in 2016. Also contributing to the decrease was a reduced work scope under our hearing loss and balance disorders program, which resulted in a $0.1 million reduction in revenue in the current period as compared to the same period in 2015. Partially offsetting these decreases was an increase in revenue from our malaria program of $0.2 million primarily attributable to our grant with the NIH. Work under this grant was completed in March 2016.

Operating expenses were $2.1 million for the three-month period ended March 31, 2016, which represents an increase of 11% as compared to $1.9 million in the comparable prior year period.

General and administrative expenses for the three-month period ended March 31, 2016 increased 9% with expense of approximately $1.4 million in 2016 as compared to $1.3 million in 2015. The increase is primarily attributable to higher personnel costs due to the expansion of our workforce by three full-time employees as compared to the same period in 2015.

Research and development expenses for the three-month period ended March 31, 2016 increased 14% with expense of approximately $0.7 million in 2016 as compared to $0.6 million in 2015. The increase is primarily attributable to higher professional, material, and facility costs.

On May 12, 2016 Mirna Therapeutics, Inc. (Nasdaq:MIRN), a clinical stage biopharmaceutical company developing a broad pipeline of microRNA-based oncology therapeutics, reported financial results for the first quarter of 2016 and provided an update on recent developments (Press release, Mirna Therapeutics, MAY 12, 2016, View Source [SID:1234512339]).

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"During the first quarter we continued to make progress advancing our lead product candidate MRX34, the first microRNA therapeutic in clinical development in cancer," commented Paul Lammers, M.D., M.Sc., Mirna’s President and CEO. "To date, MRX34 has produced clinically significant responses in patients with various types of late-stage cancers, demonstrating its ability to affect multiple pathways involved in cancer growth and immune evasion. We are continuing to advance MRX34 toward Phase 2 in late 2016 and also pushing ahead with our preclinical program to study its potential in combination with other cancer drugs."

Dr. Lammers continued, "On the corporate front, we were pleased to welcome Peter Greenleaf, Chief Executive Officer of Sucampo Pharmaceuticals, to our Board of Directors, and Dr. Vincent J. O’Neill as our new Chief Medical Officer. We look forward to their unique contributions as we advance our clinical development strategy in the promising new field of microRNA therapeutics."

FIRST QUARTER AND MRX34 PROGRAM UPDATES

Update from MRX34 Phase 1 trial planned at ASCO (Free ASCO Whitepaper). The Company expects to present additional clinical data from the ongoing Phase 1 trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in early June. To date, MRX34 has demonstrated compelling clinical results as a single agent therapy, including confirmed partial responses in patients with renal cell carcinoma, acral melanoma, and hepatocellular carcinoma. Additionally, several patients with solid tumors have achieved long-term stable disease during treatment with MRX34. These responses were observed in cancer patients with advanced Stage 4 metastatic disease, whose cancer had progressed after previously receiving multiple rounds of therapy. Top-line data from this study are expected in 2017.
Phase 1b translational medicine trial on track to begin in late 2016. This study will include serial tumor biopsies and is intended to develop deeper insights into the mechanism of action of MRX34 in melanoma patients and identify potential biomarkers of drug activity and treatment response.
MRX34 expected to begin Phase 2 by end of 2016. Studies in renal cell carcinoma and melanoma patients are being planned based on the responses observed to date in the ongoing Phase 1 trial, and on the high unmet medical need despite the availability of new therapies.
Preclinical studies ongoing of combination regimens with potential to enhance effectiveness of standard cancer therapies. At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April, Mirna researchers presented in vitro findings that demonstrated the synergistic anticancer effects between MRX34 and platinum and other commonly used cytotoxic chemotherapy drugs across a range of non-small cell lung cancer (NSCLC) cell lines. Synergistic anticancer effects were also shown between MRX34 and tyrosine kinase inhibitors. These results suggest a broad potential for combination of MRX34 with other standard of care drug classes.

Preclinical studies are also ongoing to support selection of a second microRNA product candidate from the Company’s pipeline, with an Investigational New Drug (IND) application planned in late 2017.
CORPORATE UPDATES

Further strengthened management with the appointments of Peter Greenleaf to the Company’s Board of Directors and Dr. Vincent J. O’Neill to the role of Chief Medical Officer. Mr. Greenleaf, an industry veteran with over 20 years of experience in drug development and commercialization, including three years as President of Medimmune, currently serves as the Chief Executive Officer of Sucampo Pharmaceuticals. Dr. O’Neill is a medical oncologist with 15 years of therapeutic and diagnostic product development experience, and has held senior leadership roles at several global pharmaceutical companies, including Sanofi, Genentech and GlaxoSmithKline.
FIRST QUARTER 2016 FINANCIAL RESULTS

Cash Position and Guidance: Cash, cash equivalents, and marketable securities totaled $80.6 million as of March 31, 2016, compared to $89.7 million as of December 31, 2015. The Company has no debt. Based on the current operating plan, the Company expects that current cash resources will be sufficient to meet operating requirements into 2018.
Research and Development Expenses: Research and development expenses were approximately $4.5 million for the three months ended March 31, 2016 as compared to $3.4 million for the same period in 2015. The increase was primarily attributable to increased employee compensation expense due to a higher headcount to support the advancement of the Company’s clinical and preclinical development programs, as well as increases in manufacturing, clinical, and intellectual property costs.
General and Administrative Expenses: General and administrative expenses were approximately $2.1 million for the three months ended March 31, 2016 as compared to approximately $0.9 million for the same period in 2015. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to a higher headcount and higher outside professional and consulting costs, the majority of which were costs to comply with public company operating and reporting requirements.
Net Loss: Net loss was approximately $6.6 million for the first quarter of 2016 compared to $4.3 million in the comparable period in 2015. The results included non-cash, stock-based compensation charges of approximately $447,000 in the three months ended March 31, 2016 and approximately $134,000 in the same period in 2015.

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.