1stOncology™: Cancer Intelligence Service – Page 16322 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

ZIOPHARM Announces Publication in Scientific Reports Describing Genetic Editing of Human Leukocyte Antigen in Cell Therapies to Broaden Their Human Application

On February 23, 2016 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, reported the publication of a study in Scientific Reports, a journal of the Nature Publishing Group, describing the genetic editing of human leukocyte antigen (HLA) in hematopoietic stem cells as a means of broadening the human application of these and other cell therapies (Press release, Ziopharm, FEB 23, 2016, View Source [SID:1234509153]). The article, titled "Genetic editing of HLA expression in hematopoietic stem cells to broaden their human application", is available online first at View Source

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!

Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM and senior author of the journal article, noted: "Genetic editing of HLA expression is a step towards generating universal biological products, where one donor’s cells may become suitable for sustained engraftment in multiple unrelated recipients. Unlocking the method by which HLA repertoire can be modified is one key to achieving this goal and fully harnessing the potential for off-the-shelf (OTS) therapies in immuno-oncology applications. Together with our partners at Intrexon and MD Anderson, we are bringing to bear multiple technologies to advance the findings of these studies and achieve this objective, with the goal of deploying it across our T-cell and natural killer (NK)-cell therapy platforms."

Transplantation of allogeneic, or donor-derived, hematopoietic stem cells (HSCs) into recipients with hematologic disorders is used to restore bone marrow function, termed hematopoiesis. Finding a suitable donor can be challenging due to the need to match the constellation of HLA with the recipient. For the study, researchers at The University of Texas MD Anderson Cancer Center eliminated expression of one set of HLA molecules, termed HLA-A, on donor HSC using artificial zinc finger nucleases. Other HLA molecules, such as HLA B and C remained expressed to help prevent elimination by resident NK cells. Following genetic editing, the HSCs maintained their ability to engraft and reconstitute hematopoiesis. This paper reveals that genetically altered HSC harvested from a small pool of donors will then match with a large number of unrelated recipients, which has two implications. First, it broadens the number of recipients who might benefit from bone marrow transplantation, which has particular appeal for racial minorities underserved by the current genetic makeup of unrelated donors. Second, it paves the way for generating OTS cells that are HLA matched with multiple recipients, even though they were obtained from one donor.

ZIOPHARM is developing various cell-based immuno-oncology programs, including CAR-T, TCR and NK adoptive cell-based therapies, in collaboration with its partner Intrexon Corporation (NYSE:XON) and MD Anderson.

Merck Announces Second-Quarter 2016 Dividend

On February 23, 2016 Merck (NYSE:MRK), known as MSD outside the United States and Canada, reported that the Board of Directors has declared a quarterly dividend of $0.46 per share of the company’s common stock for the second quarter of 2016 (Press release, Merck & Co, FEB 23, 2016, View Source [SID:1234509150]). Payment will be made on April 7, 2016, to shareholders of record at the close of business on March 15, 2016.

8-K – Current report

On February 23, 2016 Insys Therapeutics, Inc. (NASDAQ:INSY) ("Insys" or "the Company") reported financial results for the three- and twelve-month periods ended December 31, 2015 (Filing, 8-K, Insys Therapeutics, FEB 23, 2016, View Source [SID:1234509148]).

Highlights of and subsequent to the fourth quarter of 2015 include:

● Total net revenue increased to $91.1 million, compared to $66.5 million for the fourth quarter of 2014;

● Revenue from Subsys (fentanyl sublingual spray) was $91.1 million, up 38% compared with fourth quarter 2014 revenue of $66.1 million;

● Net income was $17.0 million, or $0.24 per basic and $0.22 per diluted share, compared to net income of $9.3 million, or $0.13 per basic and diluted share, for the fourth quarter of 2014;

● Cash, cash equivalents and investments were $202.3 million as of December 31, 2015; and

● Enrolled the first patient in our Phase II clinical study for the treatment of infantile spasms (IS) using our pharmaceutical CBD (CBD) product candidate.

"2015 was another year of solid growth coupled with steady R&D progress, and we believe 2016 will be a pivotal year for the Company," said Dr. John N. Kapoor, Chairman, President and Chief Executive Officer, of Insys Therapeutics. "We are focused on the anticipated regulatory approval and subsequent launch of our second commercial product, Syndros, which has a PDUFA date of April 1st of this year. We believe Syndros can become a new treatment option for patients suffering from chemotherapy induced nausea and vomiting, as well as those fighting anorexia associated with AIDS.

"With respect to our pipeline, we continue to make progress across our platform of sublingual spray products. We also continue to advance our research and clinical efforts using our CBD product candidates. "As we focus on building a solid foundation, we are confident in our ability to take Insys to the next level and drive value for our shareholders," concluded Dr. Kapoor.

Fourth Quarter 2015 Financial Results

Net revenue for the fourth quarter of 2015 was $91.1 million compared to $66.5 million for the fourth quarter of 2014, an increase of 38%.

Gross margin was 93% for the fourth quarter of 2015 compared with 91% for the fourth quarter of 2014.

Sales and marketing expense was $18.5 million during the fourth quarter of 2015, or 20% of net revenue, compared to $17.3 million, or 26% of net revenue, for the fourth quarter of 2014.

Research and development expense increased to $14.6 million for the fourth quarter of 2015, compared to $12.9 million for the fourth quarter of 2014, mainly as a result of Insys’ continued pipeline development investments during 2015.

General and administrative expense increased to $21.8 million for the fourth quarter of 2015, and included a non-cash equity compensation charge of $5.0 million incurred in connection with the departure of the former CEO, compared to $14.2 million for the fourth quarter of 2014.

Income tax expense was $12.9 million for the fourth quarter of 2015.

Net income for the fourth quarter of 2015 was $17.0 million, or $0.24 per basic and $0.22 per diluted share, compared to net income of $9.3 million, or $0.13 per basic and diluted share, for the fourth quarter of 2014. Non-GAAP adjusted net income for the fourth quarter of 2015 was $27.2 million, or $0.36 per diluted share, compared to non-GAAP adjusted net income of $19.5 million, or $0.26 per diluted share, in the prior year quarter. The reconciliation of net income to non-GAAP adjusted net income is included at the end of this press release.

2015 Financial Results

Net revenue for the year ended December 31, 2015 was $330.8 million compared to $222.1 million for the year ended December 31, 2014, an increase of 49%.

Gross margin for 2015 was 91%, compared with 90% for 2014.

Sales and marketing expense was $80.7 million during 2015, or 24% of net revenue, compared to $58.1 million, or 26% of net revenue, for 2014.

Research and development expense increased to $55.3 million for 2015, or 17% of net revenue, compared to $33.1 million, or 15% of revenue, for 2014, mainly as a result of Insys’ continued pipeline development investments during 2015.

General and administrative expense increased to $64.1 million for 2015, or 19% of net revenue, compared to $44.3 million, or 20% of net revenue, for 2014.

Income tax expense was $34.5 million for 2015, reflecting an effective corporate tax rate of 37%.

Net income for 2015 was $58.5 million, or $0.82 per basic and $0.77 per diluted share, compared to net income of $38.0 million, or $0.55 per basic and $0.52 per diluted share, for 2014. Non-GAAP adjusted net income, which adjusts for non-cash stock compensation expense and non-cash income tax expense, was $104.2 million, or $1.38 per diluted share, compared to $78.7 million, or $1.07 per diluted share, in the prior year. The reconciliation of net income to Non-GAAP adjusted net income is included at the end of this press release.

Liquidity

The Company had $202.3 million in cash, restricted cash, cash equivalents, and short-term and long-term investments, no debt, and $253.7 million in stockholders’ equity as of December 31, 2015.

As previously disclosed, on November 5, 2015, the Insys’ Board of Directors approved the repurchase of up to $50 million of the Company’s common stock. As of February 22, 2016, the Company had expended approximately $27.4 million to repurchase approximately 1.1 million shares of common stock outstanding. Insys intends to finance the remainder of the share repurchase program through available cash on hand.

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.

"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]).

"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.