Eagle Pharmaceuticals Receives Sixth Patent for Bendamustine

On February 23, 2016 Eagle Pharmaceuticals, Inc. (NASDAQ:EGRX) ("Eagle" or the "Company") reported that the United States Patent and Trademark Office (USPTO) has granted U.S. Patent No. 9,265,831, which pertains to liquid bendamustine hydrochloride (HCl) formulations(Press release, Eagle Pharmaceuticals, FEB 23, 2016, View Source [SID:1234509140]). The patent issued today expires on January 28, 2031. This new patent, along with five previously issued Patents (Nos. 8,609,707, 8,791,270*PED, 9,000,021, 9,034,908, and 9,144,568), further expands and protects Eagle’s bendamustine HCI intellectual property estate. This grant brings the total number of Orange Book listed patents to six, with this latest patent to be included shortly.

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"Eagle continues to strengthen its bendamustine patent portfolio with now six Orange Book listed patents running from 2026-2033 and several more pending. The issuance of this new patent supports the long-term earnings potential of the bendamustine franchise products and further protects its longevity. Given the nature of our patent portfolio, the Company believes that it will be very difficult for any ANDA filers to design around these patents," said Scott Tarriff, President and Chief Executive Officer of Eagle Pharmaceuticals.

Under a February 2015 exclusive license agreement for BENDEKA (bendamustine hydrochloride) Injection, Teva Pharmaceuticals is responsible for all U.S. commercial activities for the product including promotion and distribution.

BioCryst Reports Fourth Quarter & Full Year 2015 Financial Results

On February 23, 2016 BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported financial results for the fourth quarter and full year ended December 31, 2015(Press release, BioCryst Pharmaceuticals, FEB 23, 2016, View Source [SID:1234509139]).

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"In 2015, we gained clarity on both of our HAE drug candidates and brought in additional capital by out-licensing RAPIVAB. Our efforts are now focused on completing the bioavailability study of a solid dosage form of avoralstat and completing the APeX-1 clinical trial of BCX7353," said Jon P. Stonehouse, President & Chief Executive Officer. "We ended 2015 with a balance sheet that enables us to achieve these two near-term data events without the need to raise capital. These two programs give us two shots at achieving our goal of developing a highly effective, conveniently dosed, oral drug candidate for the prophylactic treatment of HAE patients."

Fourth Quarter Financial Results

For the three months ended December 31, 2015, total revenues decreased to $4.6 million from $5.4 million in the fourth quarter of 2014. The decrease resulted primarily from lower 2015 RAPIVAB royalty revenue, as well as lower collaborative revenue associated with BCX4430 development under contracts with the National Institute of Allergy and Infectious Diseases (NIAID) and the Biomedical Advanced Research and Development Authority (BARDA).

Research and Development (R&D) expenses for the fourth quarter 2015 of $19.0 million were consistent with the $18.5 million incurred in the fourth quarter 2014. R&D expense in the fourth quarter 2015 was more heavily concentrated in the Company’s hereditary angioedema (HAE) programs, and to a lesser extent, BCX4430 development costs under the NIAID and BARDA contracts, as compared to R&D expenses in the fourth quarter 2014.

Selling, general and administrative (SG&A) expenses for the fourth quarter 2015 increased to $2.7 million compared to $2.0 million in the fourth quarter 2014, largely due to the initiation of activities in preparation for the future commercialization of the Company’s HAE product candidates.

Interest expense, which is related to non-recourse notes, was $1.3 million for the fourth quarter of both 2015 and 2014. Also, a $229,000 mark-to-market gain on the Company’s foreign currency hedge was recognized in the fourth quarter 2015, compared to a $4.8 million mark-to-market gain in the fourth quarter 2014. These gains result from periodic changes in the U.S. dollar/Japanese yen exchange rate and the related mark-to-market valuation of our underlying hedge arrangement.

The net loss for the fourth quarter of 2015 was $18.1 million, or $0.25 per share, compared to a net loss of $11.7 million, or $0.16 per share, for the fourth quarter 2014.

2015 Financial Results

For the year ended December 31, 2015, total revenues increased to $48.3 million from $13.6 million in 2014. The increase in 2015 revenues was primarily due to the recognition of $21.8 million of revenue associated with a $33.7 million upfront payment from the RAPIVAB out-licensing transaction, $6.3 million of RAPIVAB product revenue, and increased collaboration revenue associated with BCX4430 development.

R&D expenses increased to $72.8 million for 2015 from $51.8 million for 2014. This increase was primarily the result of significantly higher HAE development costs and slightly higher RAPIVAB and BCX4430 development costs incurred in 2015, as compared to 2014.

SG&A expenses increased to $13.0 million in 2015 from $7.5 million in 2014, due primarily to increased marketing, medical affairs activities associated with HAE programs, as well as unrestricted grants awarded to the U.S. and international HAE patient advocacy groups.

Interest expense, which is related to non-recourse notes, was $5.2 million in 2015 and $5.0 million in 2014. In addition, a $564,000 mark-to-market loss on the Company’s foreign currency hedge was recognized in 2015, compared to a $5.5 million mark-to-market gain in 2014. These gains result from periodic changes in the U.S. dollar/Japanese yen exchange rate and the related mark-to-market valuation of our underlying hedge arrangement. We also realized a currency hedge gain of $1.7 million from the exercise of a U.S. Dollar/Japanese yen currency option.

The 2015 net loss decreased to $43.0 million, or $0.59 per share, compared to a net loss of $45.2 million, or $0.68 per share for 2014.

Cash, cash equivalents and investments totaled $100.9 million at December 31, 2015 and represented a $13.1 million decrease from $114.0 million at December 31, 2014. Net operating cash use for 2015 was $42.2 million, as compared to $33.3 million utilized in 2014.

Clinical Development Update & Outlook

On February 8, 2016, we announced results from OPuS-2 (Oral P rophylaxiS-2), a clinical trial of avoralstat administered three times daily in a liquid-filled soft gel formulation for the prophylactic treatment of HAE attacks. The primary efficacy endpoint was angioedema attack frequency. Treatment with 500 mg and 300 mg of avoralstat three times daily failed to demonstrate a statistically significantly lower mean attack rate versus placebo. Statistically significant improvements in duration of attacks and in the Angioedema Quality of Life total score were observed comparing the 500 mg three times a day avoralstat arm to placebo. Following the analysis of OPuS-2 results, the decision was made to discontinue further development of softgel avoralstat formulation in order to focus development efforts on a novel solid dosage form of avoralstat.

BioCryst expects to report results from a relative bioavailability study testing the novel solid dosage form of avoralstat by mid-year 2016. The primary goal of this study is to achieve meaningfully better drug exposure in a twice daily dosing regimen.

BioCryst expects to report results from the BCX7353 APeX-1 dose ranging study in HAE patients by year end. The design of APeX-1 trial will be described once it is initiated.

Financial Outlook for 2016

Based upon development plans and our awarded government contracts, BioCryst expects its 2016 net operating cash use to be in the range of $55 to $75 million, and its 2016 operating expenses to be in the range of $78 to $98 million. Our operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the Company’s stock, as well as by the vesting of the Company’s outstanding performance-based stock options.

ARIAD Reports 2015 Financial Results, Provides 2016 Product Revenue Guidance and Outlines Company Progress

On February 23, 2016 ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) reported financial results for the fourth quarter and full year ended December 31, 2015 and issued 2016 product revenue guidance (Press release, Ariad, FEB 23, 2016, View Source;p=RssLanding&cat=news&id=2142086 [SID:1234509137]). Additionally, the Company provided an update on corporate developments and key objectives for 2016.

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"We had a strong fourth quarter of sales for Iclusig and 102 percent growth year over year," stated Paris Panayiotopoulos, president and chief executive officer of ARIAD. "This is a very important year for ARIAD as we work to complete our ongoing review of strategic initiatives to deliver patient and shareholder value. In addition to maximizing top-line growth of Iclusig, a key event this year will be the presentation of pivotal, registration data on brigatinib at ASCO (Free ASCO Whitepaper), along with our planned filing for marketing approval of brigatinib in the U.S."

2015 Fourth Quarter and Full-Year Financial Results

Revenues

Net product revenues from sales of Iclusig were $33.3 million for the fourth quarter of 2015, an increase of 56% from the fourth quarter of 2014.

U.S. sales of Iclusig were $25.1 million for the fourth quarter, an increase of 48% from the fourth quarter of 2014.

European sales of Iclusig were $8.2 million for the fourth quarter, an increase of 86% from the fourth quarter of 2014.

Net product revenues from sales of Iclusig for the year ended December 31, 2015 were $112.5 million, an increase of 102% from 2014. In the U.S., net product revenue totaled $85.7 million in 2015, an increase of 114% from 2014, while in the EU, net product revenue totaled $26.8 million in 2015, an increase of 71% from 2014.

Net Loss

Quarter Ended December 31, 2015

Net loss for the fourth quarter ended December 31, 2015 was $59.9 million, or $0.32 per share, compared to a net loss of $5.8 million, or $0.03 per share, for the same period in 2014. The net loss for 2014 included $50 million in non-recurring income resulting from an amendment to our license agreement with Bellicum Pharmaceuticals, Inc.

R&D expenses were $44.8 million for the fourth quarter of 2015, an increase of 37% from the fourth quarter of 2014, reflecting an increase in Iclusig clinical-trial costs, as well as increased manufacturing and other supporting costs related to Iclusig.

Selling, general and administrative expenses were $43.8 million for the fourth quarter of 2015, an increase of 9% from the fourth quarter of 2014, reflecting an increase in personnel and commercial-related expenses.

Year Ended December 31, 2015

Net loss for the full year 2015 was $231.2 million, or $1.23 per share, compared to a net loss of $162.6 million, or $0.87 per share, for the full year 2014. The net loss for 2014 included $50 million in non-recurring income resulting from an amendment to our license agreement with Bellicum, noted above. The net loss for 2015 includes approximately $14.5 million in non-recurring expenses related to the retirement of our chief executive officer and costs associated with our 2015 proxy and legal matters.

The 2015 results include an increase in Iclusig product revenue of $56.8 million as well as an increase in operating expenses of $70.5 million in 2015 compared to 2014, reflecting an increase in R&D expenses related to an increase in clinical-trial costs, as well as manufacturing and other support costs related to Iclusig and brigatinib clinical trials, and an increase in personnel and related costs reflecting a larger number of R&D employees in 2015 compared to 2014.

Cash Position

As of December 31, 2015, cash, cash equivalents and marketable securities totaled $242.3 million, compared to $352.7 million in cash and cash equivalents at December 31, 2014.

Cash, cash equivalents and marketable securities include $50 million we received in the third quarter of 2015 from PDL BioPharma, Inc. (PDL) pursuant to a royalty financing agreement. In July 2016, we will receive an additional $50 million from PDL. Under the agreement, we also have an option, in our sole discretion, to receive up to an additional $100 million through July 2016.

2016 Product Revenue Guidance

Net product revenues from sales of Iclusig are expected to be in the range of $190 million to $200 million. This guidance includes sales of Iclusig in the U.S., Europe, and other select countries where ARIAD has distributorships in place.

ARIAD will provide guidance on 2016 operating expenses at the time of completing its ongoing strategic review expected in the second quarter of 2016.

Research and Development Progress and Key Objectives

Iclusig Clinical Development

Late last year we initiated the OPTIC-2L trial, a Phase 3 trial of Iclusig in patients with chronic-phase chronic myeloid leukemia (CP-CML) who have experienced treatment failure after imatinib therapy. This second-line study of Iclusig is aimed at expanding the indication for Iclusig in patients with resistant and intolerant CML.

Patient enrollment is ongoing in the OPTIC (Optimizing Ponatinib Treatment In CML) trial of Iclusig. This randomized, dose-ranging trial is designed to evaluate three different starting doses of ponatinib in patients with refractory CP-CML and is expected to inform the optimal use of Iclusig in these patients.

Otsuka Pharmaceutical Co., Ltd. (Otsuka), our commercial partner for Iclusig in Japan and several other Asian countries, submitted a new drug application (NDA) to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) seeking approval for Iclusig for the treatment of resistant or intolerant CML and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ALL). This marketing application was submitted in early 2016 and is expected to lead to an initial approval of Iclusig in Japan by the end of this year.

Brigatinib Clinical Development

We submitted clinical data from the Phase 2 ALTA trial of brigatinib to this year’s annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June, 2016. The ALTA trial enrolled approximately 220 patients at 71 sites in North America, Europe and Asia. In addition to an anticipated data presentation at ASCO (Free ASCO Whitepaper), we are on track to file for approval of brigatinib in the U.S. in the third quarter of this year. Brigatinib has received breakthrough-therapy designation from the U.S. Food and Drug Administration.

The randomized front-line clinical trial of brigatinib is expected to begin in the second quarter of 2016. This Phase 3 trial is designed to compare brigatinib and crizotinib in patients with ALK+ non-small cell lung cancer (NSCLC), who have not received prior ALK inhibitors, and is expected to serve as a confirmatory trial for accelerated approval of brigatinib.

Advancing the Pipeline

We submitted an investigational new drug (IND) application for AP32788 in late 2015 and received clearance from the U.S. Food and Drug Administration at the end of January to begin a Phase 1/2 proof-of-concept clinical trial which we expect to begin in 2016. AP32788 is an orally active tyrosine-kinase inhibitor (TKI), designed to address the unmet medical need in NSCLC patients with specific EGFR and HER2 kinase mutations. ARIAD estimates that there are approximately 6,000 patients in the United States living with EGFR exon 20 or HER2 point mutations.

Upcoming Investor Meetings

ARIAD management will be participating at the following investor conferences:

RBC Capital Markets’ Healthcare Conference, New York City, February 24, 2016

Cowen and Company Healthcare Conference, Boston, March 9, 2016

Barclays Global Healthcare Conference, Miami, March 17, 2016

About Iclusig (ponatinib) tablets

Iclusig is a kinase inhibitor. The primary target for Iclusig is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Iclusig was designed using ARIAD’s computational and structure-based drug-design platform specifically to inhibit the activity of BCR-ABL. Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

Iclusig is approved in the U.S., EU, Australia, Switzerland, Israel and Canada.

In the U.S., Iclusig is a kinase inhibitor indicated for the:

Treatment of adult patients with T315I-positive chronic myeloid leukemia (chronic phase, accelerated phase, or blast phase) or T315I-positive Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL).

Treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia or Ph+ ALL for whom no other tyrosine kinase inhibitor (TKI) therapy is indicated.

These indications are based upon response rate. There are no trials verifying an improvement in disease-related symptoms or increased survival with Iclusig.

IMPORTANT SAFETY INFORMATION, INCLUDING THE BOXED WARNING

WARNING: VASCULAR OCCLUSION, HEART FAILURE, and HEPATOTOXICITY

See full prescribing information for complete boxed warning

Vascular Occlusion: Arterial and venous thrombosis and occlusions have occurred in at least 27% of Iclusig treated patients, including fatal myocardial infarction, stroke, stenosis of large arterial vessels of the brain, severe peripheral vascular disease, and the need for urgent revascularization procedures. Patients with and without cardiovascular risk factors, including patients less than 50 years old, experienced these events. Monitor for evidence of thromboembolism and vascular occlusion. Interrupt or stop Iclusig immediately for vascular occlusion. A benefit risk consideration should guide a decision to restart Iclusig therapy.

Heart Failure, including fatalities, occurred in 8% of Iclusig-treated patients. Monitor cardiac function. Interrupt or stop Iclusig for new or worsening heart failure.

Hepatotoxicity, liver failure and death have occurred in Iclusig-treated patients. Monitor hepatic function. Interrupt Iclusig if hepatotoxicity is suspected.

Please see the full U.S. Prescribing Information for Iclusig, including the Boxed Warning, for additional important safety information

Juno Therapeutics, Seattle Cancer Care Alliance and Fred Hutchinson Cancer Research Center Announce New Immuno-oncology Clinical Trials Unit

On February 23, 2016 Juno Therapeutics, Inc. (NASDAQ: JUNO), a biopharmaceutical company focused on re-engaging the body’s immune system to revolutionize the treatment of cancer, and Fred Hutchinson Cancer Research Center (Fred Hutch) reported the creation of a new, best-in-class clinical trials unit (CTU) dedicated to immuno-oncology (Press release, Juno, FEB 23, 2016, View Source [SID:1234509163]).

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University of Washington, the Seattle Cancer Care Alliance (SCCA), Fred Hutch, and Juno are all investing in the CTU, and Juno will also provide research funds to Fred Hutch in support of pre-clinical and clinical research focused on the development of improved cancer immunotherapies.

"Immuno-oncology, and in particular, T cell therapies are advancing at a rapid pace, and translational medicine and clinical care of patients need to move as quickly as the basic science," said Mark J. Gilbert, M.D., Juno’s Chief Medical Officer. "We are pleased to work with these world-leading institutions to accelerate the pace of learning for the field and the standard of care for patients. Our ongoing CAR T cell trials have highlighted the opportunity for strong translational medicine to improve our therapies and for dedicated clinical scientists to improve patient outcomes."

"Establishing this clinical trials unit is vital to accelerating our lifesaving research," said Fred Hutch President and Director Gary Gilliland, M.D., Ph.D. "We are on the precipice of multiple curative therapies, and the investment of Juno and the SCCA in our science will be catalytic in moving our discoveries into patients."

"Clinical trials are crucial steps in bringing safer, more effective cancer therapies to the patients who need them," said Fred Appelbaum, M.D., SCCA Executive Director and President. "The CTU will enable patients participating in clinical trials to get all of their care at a single site, with a team of dedicated surgeons, radiologists, pathologists and medical oncologists."

In addition to consolidating trial participants’ care into one location, the new unit will have dedicated space for research teams to handle specimens and collect trial data. The CTU is expected to be operational in mid-2016 and will be available for studies supported by Juno and other drugmakers.

Medigene strengthens its DC platform with patent for treatment of stem cell or bone-marrow transplanted patients

On February 23, 2016. Medigene AG (MDG1, Frankfurt, Prime Standard), a clinical stage immune-oncology company focusing on the development of T cell immuno-therapies for the treatment of cancer, reported the grant of US patent 9,238,063 by the US Patent Office (USPTO) covering semi-allogeneic anti-tumour vaccines with HLA[1] haplo-identical antigen presenting cells (APCs) (Press release, MediGene, FEB 23, 2016, View Source [SID:1234509154]). Medigene holds an exclusive license to the patent that was issued to Helmholtz Zentrum München (German Research Center for Environmental Health).

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Prof. Dolores Schendel, CEO and CSO of Medigene and co-inventor of this patent, explains: "This patent increases the applicability for Medigene’s DC vaccines and complements the IP portfolio for our DC vaccines already covered by granted patents in Europe, Australia and the US. It is highly relevant in the immunotherapy of stem cell or bone-marrow transplanted patients, who have received their transplants from parents, siblings or children."

For a stem cell or bone-marrow donation without an available HLA-identical "sibling", healthy relatives of the patient are considered meanwhile as the best potential donors. Those donors are at least HLA haplo-identical with respect to the patient, which means that 50% of the MHC/HLA molecules of a donor are identical to the MHC/HLA molecules of the patient.

Currently, there are limited treatment options for patients with haematological malignancies who suffer disease relapse after a stem cell or bone-marrow transplantation. The patent US9,238,063 claims a method of treating a tumour disease in patients by using semi-allogeneic antigen presenting cells of an HLA haplo-identical donor. The APCs are modified to express and present tumour-specific antigens which are in turn recognised by the T cells of the patient resulting in an immune reaction directed against the patient’s residual tumour cells.

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 utilising 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 recognise and eliminate antigen-bearing tumour cells. Dendritic cells can also induce natural killer cells (NK cells) to attack tumour 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 tumour-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.

Further audio-visual education about Medigene’s DC-Vaccines at:
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