On May 6, 2016 La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported first quarter 2016 financial results and highlighted recent corporate progress (Filing, Q1, La Jolla Pharmaceutical, 2016, MAY 11, 2016, View Source [SID:1234512493]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Recent Corporate Progress

• La Jolla’s ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial continued to enroll as planned through the first quarter of 2016. The ATHOS 3 trial is La Jolla’s multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501, the Company’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH), which was initiated in 2015. The Company initiated the ATHOS 3 trial after reaching an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA), in which the agreed-upon primary efficacy endpoint in ATHOS 3 is an increase in blood pressure. Results of ATHOS 3 are expected by the end of 2016.

• La Jolla reported interim results in January 2016 of its multicenter, open-label, dose-escalation Phase 1 clinical trial of LJPC-401, the Company’s novel formulation of hepcidin, in patients at risk for iron overload due to conditions such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. These results suggested a dose-dependent reduction in serum iron following a single dose of LJPC-401. Complete results from this Phase 1 clinical trial, which was initiated in October 2015, are expected in the second half of 2016.
"The first quarter was a productive start to 2016 for La Jolla, highlighted by the continued enrollment of our ATHOS 3 trial and encouraging interim data from our Phase 1 clinical trial of LJPC-401," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "We look forward to continuing the advancement of each of our exciting programs during the rest of 2016, culminating with the results from the ATHOS 3 trial that are expected by the end of the year."
Results of Operations

As of March 31, 2016, La Jolla had $113.1 million in cash and cash equivalents, compared to $126.5 million as of December 31, 2015. The decrease in cash and cash equivalents was primarily due to net cash used for operating activities. Based on current operating plans and projections, La Jolla believes that its current cash and cash equivalents are sufficient to fund operations into 2018.

La Jolla’s net cash used for operating activities for the three months ended March 31, 2016 was $13.0 million, compared to net cash used for operating activities of $5.5 million for the same period in 2015. La Jolla’s net loss for the three months ended March 31, 2016 was $16.5 million, or $0.96 per share, compared to a net loss of $9.0 million, or $0.59 per share, for the same period in 2015. During the three months ended March 31, 2016, La Jolla recognized contract revenue of approximately $0.2 million, which was pursuant to a services agreement initiated in 2015 under which La Jolla provides research and development services to a related party. The net loss includes non-cash, share-based compensation expense of $3.7 million for the three months ended March 31, 2016, compared to $3.4 million for the same period in 2015.

The increases in net cash used for operating activities and net loss in 2016 as compared to 2015 were primarily due to increased development costs associated with the ATHOS 3 trial and the costs associated with the initiation of the Phase 1 clinical trial of LJPC-401 in iron overload. In addition, there were increases in personnel and related costs, which were mainly due to the hiring of additional personnel and increased facility costs to support the increased development activities.

On May 11, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported a corporate update and reported financial results for the first quarter ended March 31, 2016 (Press release, Onconova, MAY 11, 2016, View Source [SID:1234512298]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We recently reported two important advances in the development of rigosertib for the unmet medical needs of patients with myelodysplastic syndromes (MDS). Foremost is the elucidation of the novel mechanism of action of rigosertib targeting the RAS pathway, recently published in Cell, and the release of detailed clinical data from our ONTIME Phase 3 study in Lancet Oncology. These peer-reviewed papers provide important validation of the mechanistic rationale for rigosertib as a novel treatment option for patients with MDS," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "We are encouraged by the continued progress in the global Phase 3 INSPIRE trial which is now enrolling patients in the U.S. and Europe."

Recent Business Highlights:

Progress in INSPIRE Pivotal Trial of Rigosertib in Higher-risk MDS (HR-MDS)

The global INSPIRE trial is now enrolling patients in the United States and Europe. As of May 3, 2016, 51 sites, including 13 in the U.S., are open and recruiting patients. This pivotal trial was initiated in 4Q2015.

Results from Onconova’s ONTIME trial were published in the top-tier, peer-reviewed journal, Lancet Oncology. The article, entitled, "Rigosertib versus best supportive care for patients with high-risk myelodysplastic syndromes after failure of hypomethylating drugs (ONTIME): a randomised, controlled, phase 3 trial," appeared in the March 8, 2016 online edition of the journal.
Rigosertib Mechanism of Action

The unique RAS-targeted mechanism of action for rigosertib was published in the prestigious peer-reviewed experimental biology journal, Cell. The paper, entitled "A small molecule RAS-mimetic disrupts RAS association with effector proteins to block signaling," appeared in the April 21, 2016 edition of the journal.
Proprietary NCE targeting ARK5 and CDK4/6

A presentation at the 2016 American Association of Cancer Research Annual meeting demonstrated the important differentiating features of ON 123300, Onconova’s novel pre-clinical ARK5 and CDK4/6 targeted agent. Results indicated that the inhibitory activity of ON 123300 on ARK5 and CDK4/6 resulted in the activation of programmed cell death in colorectal cancer cells, while treatment with an approved CDK4/6 inhibitor merely resulted in cytostasis.
Upcoming Events

Enrollment of the first patient in the Phase 3 INSPIRE trial in Japan: 2Q2016

Presentation of updated Phase 2 data from oral rigosertib combination trial in MDS: 2Q2016

End of Phase 2 meeting with FDA to discuss data from oral rigosertib combination trial: 2H2016
First Quarter 2016 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2016 totaled $16.8 million, compared to $19.8 million as of December 31, 2015.
Total net revenue was $1.5 million for the first quarter of 2016 compared to $0.1 million for the first quarter of 2015.
Research and development expenses were $5.8 million for the first quarter of 2016 compared to $9.5 million for the first quarter of 2015.
General and administrative expenses were $3.2 million for the first quarter of 2016 compared to $3.0 million for the first quarter of 2015.

About Rigosertib

Rigosertib is a small molecule inhibitor of cellular signaling and acts as a RAS mimetic. These effects of rigosertib appear to be mediated by direct binding of the compound to the RAS-binding domain (RBD) found in many RAS effector proteins, including the Raf kinases and PI3K. The therapeutic focus for rigosertib is myelodysplastic syndromes (MDS), a group of bone marrow disorders characterized by ineffective formation of blood cells that often converts into acute myeloid leukemia (AML). Clinical trials for rigosertib are being conducted at leading institutions in the United States, Europe, and the Asia-Pacific region. Rigosertib is protected by issued patents (earliest expiry in 2026) and has been awarded Orphan Designation for MDS in the United States, Europe and Japan.

About INSPIRE

The INternational Study of Phase III IV RigosErtib, or INSPIRE, is based on guidance received from the U.S. Food and Drug Administration and European Medicines Agency and derives from the findings of the ONTIME Phase 3 trial. INSPIRE is a multi-center, randomized controlled study to assess the efficacy and safety of IV rigosertib in HR-MDS patients who had progressed on, failed to respond to, or relapsed after previous treatment with an HMA within the first nine months of initiation of HMA treatment. This time frame optimizes the opportunity to respond to treatment with an HMA prior to declaring treatment failure, as per NCCN Guidelines. The trial will enroll approximately 225 patients randomized at a 2:1 ratio into two treatment arms: IV rigosertib plus Best Supportive Care versus Physician’s Choice plus Best Supportive Care. The primary endpoint of INSPIRE is overall survival and an interim analysis is anticipated. Full details of the INSPIRE trial, such as inclusion and exclusion criteria, as well as secondary endpoints, can be found on clinicaltrials.gov (NCT02562443).

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

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

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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."

– See more at: View Source#sthash.XFHBDKIF.dpuf