On Aug. 3, 2016 bluebird bio, Inc. (Nasdaq: BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported business highlights and financial results for the second quarter ended June 30, 2016 (Press release, bluebird bio, AUG 3, 2016, View Source;p=RssLanding&cat=news&id=2192581 [SID:1234514211]).

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"We continue to advance our LentiGlobinTM, Lenti-D and bb2121 programs through clinical trials, and in recent months, we’ve been pleased to showcase some of the innovative platform work we are doing to continuously improve upon our therapies. At the ASH (Free ASH Whitepaper) Workshop on Genome Editing last month, we provided more detail on our proprietary megaTAL genome editing platform, and at the ASGCT (Free ASGCT Whitepaper) annual meeting, we highlighted our progress in developing next-generation T cell-based immunotherapies and improving manufacturing, transduction efficiency and assay development," said Nick Leschly, chief bluebird. "In the second half of 2016, we look forward to continued progress on our clinical programs, including initiation of the LentiGlobin HGB-207 Phase 3 study in non-β0/β0 transfusion-dependent thalassemia (TDT), the integration of manufacturing process improvements into our LentiGlobin clinical trials, and presenting updated LentiGlobin clinical data at ASH (Free ASH Whitepaper)."

Recent Highlights

MANUFACTURING AGREEMENT WITH LONZA – In June, bluebird and Lonza announced a strategic manufacturing agreement providing for the future commercial production of bluebird bio’s Lenti-D and LentiGlobin drug products. This agreement follows a successful multi-year clinical manufacturing relationship and provides bluebird bio with a path to commercial supply including dedicated production suites within Lonza’s state-of-the-art facility. Under this multi-year agreement, Lonza will complete the suite design, construction and validation along with process validation prior to anticipated commercial launch.

GENOME EDITING DATA PRESENTED AT ASH (Free ASH Whitepaper) WORKSHOP ON GENOME EDITING – In July, pre-clinical data from bluebird’s megaTAL genome editing platform was presented at the ASH (Free ASH Whitepaper) Workshop on Genome Editing in Washington, DC. The data reported at the ASH (Free ASH Whitepaper) workshop highlight recent progress bluebird has made in:
Expanding the number of megaTAL targetable sites in the genome to permit the precise placement of an editing event within a target gene

Refining the specificity of megaTALs to eliminate undesirable off-target activity
Combining megaTALs targeting different target genes to achieve the knockout of multiple genes simultaneously
ORPHAN DRUG DESIGNATION GRANTED FOR BB2121 IN MULTIPLE MYELOMA – In May, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for bb2121 in multiple myeloma. bb2121 is a chimeric antigen receptor T cell (CAR T) therapy targeting B cell maturation antigen (BCMA), and is being developed by bluebird bio in collaboration with Celgene Corporation. In February, the first patient was infused in the CRB-401 study of anti-BCMA CAR T therapy bb2121 in relapsed/refractory multiple myeloma. Additionally, Celgene exercised its option to exclusively license bb2121.

PRESENTED INTERIM DATA FROM STARBEAM STUDY AT AAN ANNUAL MEETING – In April, Dr. Florian Eichler of Massachusetts General Hospital for Children presented interim clinical data from the Starbeam study of Lenti-D in CALD at AAN. Initial Starbeam results suggest Lenti-D gene therapy may have similar efficacy to allogeneic hematopoietic stem cell transplant (HCT), the current standard of care, with a more favorable safety profile. As of March 31, 2016, three of the 17 patients enrolled in the study have reached two years of follow-up and remain free of major functional disabilities (MFDs), the primary endpoint of the study. Sixteen of the 17 patients had stabilization of their neurological function score (NFS), and 14 of 17 had a stable Loes score. The safety profile of Lenti-D treatment appeared consistent with myeloablative conditioning.

TEN ABSTRACTS PRESENTED AT ASGCT (Free ASGCT Whitepaper) 19th ANNUAL MEETING – In April, two oral presentations given by bluebird’s academic collaborators highlighted previously presented data from bluebird bio’s ongoing gene therapy clinical trials, including interim data from the Starbeam Study of Lenti-D in cerebral adrenoleukodystrophy, and interim data from the HGB-205 study of LentiGlobin in severe sickle cell disease and TDT. Eight additional presentations were featured at the meeting, highlighting progress across the company’s preclinical, research and process development activities in both HSC gene therapy and T cell immunotherapy.
Second Half 2016 Anticipated Milestones

Update on LentiGlobin process improvements
Initiation of the HGB-207 study in patients with TDT with the non-β0/β0 genotype
Presentation of updated clinical data for LentiGlobin at the ASH (Free ASH Whitepaper) annual meeting in December 2016
Second Quarter 2016 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2016 were $779.0 million, compared to $865.8 million as of December 31, 2015, a decrease of $86.8 million.

Revenues: Collaboration revenue was $1.6 million for the second quarter of 2016 compared to $4.9 million for second quarter of 2015. The decrease is a result of an amendment to our collaboration agreement with Celgene in June 2015.
R&D Expenses: Research and development expenses were $41.8 million for the second quarter of 2016 compared to $44.3 million for the second quarter of 2015. The decrease in research and development expenses was primarily attributable to decreased in-licensing milestones and fees and stock-based compensation expense partially offset by increased employee payroll and facilities costs due to increased headcount, and increased manufacturing, clinical, and information technology costs to support the advancement of our clinical and pre-clinical programs.

G&A Expenses: General and administrative expenses were $18.4 million for the second quarter of 2016 compared to $10.7 million for the second quarter of 2015. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting costs to support our overall growth.
Net Loss: Net loss was $58.8 million for the second quarter of 2016 compared to $51.8 million for the second quarter of 2015.
Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $779.0 million as of June 30, 2016 will be sufficient to fund its current operations through 2018.

On August 03, 2016 Aduro Biotech, Inc. (NASDAQ:ADRO) reported financial results for the second quarter 2016 (Press release, Aduro BioTech, AUG 3, 2016, View Source;p=RssLanding&cat=news&id=2192550 [SID:1234514210]). Net income for the three months ended June 30, 2016 was $2.3 million, or $0.04 per share, and for the six months ended June 30, 2016 net loss was $26.5 million, or $0.41 per share, compared to a net loss of $26.3 million, or $0.50 per share, and $42.9 million, or $1.61 per share respectively, for the same periods in 2015.

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Cash, cash equivalents and marketable securities totaled $396.9 million at June 30, 2016, compared to $431.0 million at December 31, 2015.

"We are uniquely positioned in the field of immunotherapy with three distinct, proprietary technology platforms, as well as a strong cash position," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "As we continue to advance our programs, we anticipate a number of upcoming milestones across our three platforms, including data from our LADD platform in multiple tumor types, initial clinical results from ADU-S100, our first STING Pathway Activator, and IND-enabling studies for multiple B-select monoclonal antibodies."

Recent Progress

Preclinical data published in Blood highlighting the potential of Aduro’s proprietary monoclonal antibody BION-1301 targeting a proliferation-inducing ligand (APRIL) for the treatment of multiple myeloma
Initiated a Phase 1 clinical trial of ADU-S100, the first STING Pathway Activator compound to enter the clinic, for the treatment of cutaneously accessible tumors
Reported results from the Phase 2b ECLIPSE trial in pancreatic cancer
Reported data from the Phase 1b clinical trial in mesothelioma at ASCO (Free ASCO Whitepaper) 2016
Second Quarter 2016 Financial Results

Revenue was $39.0 million for the second quarter of 2016 and $43.0 million for the six months ended June 30, 2016, compared to $9.9 million and $19.5 million, respectively, for the same periods in 2015. The increase was primarily due to the receipt of a $35.0 million milestone payment from Novartis in connection with the initiation of the Phase 1 ADU-S100 trial in the second quarter of 2016.

Research and development expenses were $26.9 million for the second quarter of 2016 and $47.8 million for the six months ended June 30, 2016, compared to $13.5 million and $24.2 million, respectively, for the same periods in 2015. This increase was primarily due to clinical development expenses associated with our ongoing trials in pancreatic cancer, ovarian cancer and mesothelioma, including manufacturing and personnel costs.

General and administrative expenses were $8.7 million for the second quarter of 2016 and $17.7 million for the six months ended June 30, 2016, compared to $5.9 million and $12.1 million, respectively, for the same periods in 2015. This increase was primarily due to continued growth of the company and the associated increased expenses related to personnel, facilities and professional services.

There was no loss from remeasurement of fair value of warrants for either the second quarter of 2016 or six months ended June 30, 2016, compared to $16.7 million and $26.1 million, respectively, for the same periods in 2015. In April 2015, all such warrants ceased being liability-classified as the contingency surrounding the number of shares issuable upon the warrant exercise expired. All outstanding warrants were equity-classified and not subject to future remeasurement.

Provision for income taxes was $1.5 million for the second quarter of 2016 and $4.7 million for the six months ended June 30, 2016. There was no provision for income taxes in the same periods in 2015. The income tax expense recorded for the second quarter of 2016 was primarily related to current and deferred federal income taxes.

On August 3, 2016 Nektar Therapeutics (Nasdaq: NKTR) reported its financial results for the second quarter ended June 30, 2016 (Press release, Nektar Therapeutics, AUG 3, 2016, View Source [SID:1234514207]).

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Cash and investments in marketable securities at June 30, 2016 were $274.9 million as compared to $308.9 million at December 31, 2015. This balance includes the $28.0 million payment received from AstraZeneca in April of 2016 for the sublicense of MOVENTIG (naloxegol) to ProStraken in Europe. The balance does not include the $20 million upfront payment for the licensing of European rights for ONZEALD to Daiichi Sankyo Europe, which occurred in Q2 2016.

"We continue to execute on the development and business objectives for Nektar," said Howard W. Robin, President and CEO of Nektar. "Following our licensing agreement with Daiichi Sankyo Europe, the MAA for ONZEALD was accepted by the EMA in July, and with an accelerated assessment review granted by the CHMP, we expect a decision on the recommendation for conditional approval in Q1 2017. Our Phase 3 study of NKTR-181 in patients with chronic low back pain has now completed enrollment and is on track to have topline data in the first quarter of 2017. Finally, NKTR-214 continues to advance in its Phase 1/2 study in cancer patients at MD Anderson and Yale Cancer Centers, with initial topline data expected before the end of this year. As the first medicine designed to selectively stimulate the in vivo growth of endogenous tumor-killing T cells and natural killer cells within the tumor micro-environment, we are extremely excited about the potential of NKTR-214 to transform the immuno-oncology landscape."

Year-to-date revenue for 2016 was $91.6 million as compared to $131.5 million in the first half of 2015. Revenue in 2016 included recognition of the $28.0 million cash payment received from AstraZeneca for the sublicense of MOVENTIG (naloxegol) to ProStrakan (Kyowa Kirin) in Europe. In addition, product sales, royalty revenue, and non-cash royalty revenue increased in the first half of 2016 compared to the first half of 2015. Revenue in the first half of 2015 included recognition of $90.0 million of the $100.0 million milestone payment from AstraZeneca following the first commercial sale of MOVANTIK in the U.S. in Q1 2015. Revenue in the second quarter of 2016 was $32.8 million as compared to $22.7 million in the second quarter of 2015.

Revenue included non-cash royalty revenue, related to our 2012 royalty monetization, of $8.1 million and $14.7 million in the second quarter and first half of 2016, respectively, and $4.7 million and $8.7 million in the second quarter and first half of 2015, respectively. This non-cash royalty revenue is offset by non-cash interest expense incurred in connection with the 2012 royalty monetization of $5.0 million and $10.0 million in the second quarter and first half of 2016, respectively and $5.2 million and $10.2 million in the second quarter and first half of 2015, respectively.

Total operating costs and expenses in the first half of 2016 were $139.5 million as compared to $131.9 million in the first half of 2015. Total operating costs and expenses in the second quarter of 2016 were $71.1 million as compared to $66.1 million in the second quarter of 2015. Total operating costs and expenses increased primarily as a result of increased research and development (R&D) expense.

Research and development expense in the second quarter of 2016 was $52.4 million as compared to $45.4 million in the second quarter of 2015. For the first half of 2016, R&D expense was $101.6 million as compared to $92.4 million in the first half of 2015. R&D expense was higher in the second quarter and first half of 2016 as compared to the same periods in 2015 primarily due to expenses for the NKTR-181 Phase 3 studies and the initiation of the Phase 1/2 study of NKTR-214.

General and administrative expense was $11.0 million in the second quarter of 2016 as compared to $10.2 million in the second quarter of 2015. G&A expense in the first half of 2016 was $21.3 million as compared to $20.5 million in the first half of 2015.

Net loss in the second quarter of 2016 was $48.6 million or $0.36 loss per share as compared to $52.7 million or $0.40 loss per share in the second quarter of 2015. Net loss in the first half of 2016 was $68.1 million or $0.50 loss per share as compared to $18.8 million or $0.14 loss per share in the first half of 2015.

The company also announced the following upcoming presentations and events:

Fourth Annual Immuno-Oncology Summit, Boston, MA:

Oral Abstract: "Of Mice and Men: Translating the Immune Oncology Mechanism of Action of NKTR-214." Presented by: Jonathan Zavelsky, Ph.D.
Date: August 31, 2016, 5:15 p.m. Eastern Time
Second CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper), New York, NY:

Abstract/Poster #311: "The CD122-biased immunostimulatory cytokine NKTR-214 combined with checkpoint blockade leads to mobilization of antitumor immunity and synergistic activity", Langowski, J., et al.
Date: September 26, 2016, 5:15 – 7:45 p.m. Eastern Time
ESMO 2016 Congress, Copenhagen, Denmark:

Abstract #3048: "Combining Complementary Mechanisms of Immune Activation: NKTR-214, a Biased IL-2 Pathway Agonist, and Immune Checkpoint Antagonists", Charych, D., et al.
Date: October 9, 2016, 1:00 – 2:00 p.m. Central European Time
10th Annual Pain and Migraine Therapeutics Conference, Chicago, IL:

Oral Abstract: "Clinical Development of a Novel Opioid Molecule with Inherent Anti-abuse Properties", Presented by Carlo DiFonzo, Ph.D.
Date: October 19, 2016, 1:00 – 1:30 p.m. Central Time

On August 03, 2016 Mateon Therapeutics, Inc. (Nasdaq:MATN), a biopharmaceutical company developing vascular disrupting agents (VDAs) for the treatment of orphan oncology indications, reported a corporate update and reported financial results for the second quarter of 2016 (Press release, Mateon Therapeutics, AUG 3, 2016, View Source [SID:1234514204]).

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Recent Corporate Highlights

Presented new data reflecting improved survival outcomes for CA4P-treated patients from Study GOG-0186I at an investor event in New York. These data showed an improvement of 5.6 months in overall survival and 3.7 months in progression-free survival in patients with measurable disease.
Published positive results from Study GOG-0186I, an open-label randomized Phase 2 study evaluating CA4P in recurrent ovarian cancer, in the Journal of Clinical Oncology, the official journal of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).
Announced name change to Mateon Therapeutics, Inc. to reflect the Company’s new focus on combination vascular targeted therapy in orphan oncology indications.
Initiated the FOCUS Study, a Phase 2/3 clinical trial of CA4P in combination with bevacizumab (Avastin) and chemotherapy, for the treatment of patients with platinum-resistant ovarian cancer.
Enrolled the first patient into the Phase 2 portion of the PAZOFOS Study, a Phase 1b/2 clinical trial of CA4P in combination with pazopanib (Votrient), in patients with recurrent ovarian cancer.
Received orphan drug designation for CA4P for the treatment of glioma from the U.S. Food and Drug Administration (FDA).
Completed enrollment of the first cohort and initiated the second cohort of OX1222, an open-label dose ranging study of OXi4503 in combination with cytarabine, in patients with relapsed/refractory acute myeloid leukemia (AML).
"We have accomplished a great deal over the last several months and believe we are well-positioned for long-term success," said William D. Schwieterman, M.D., Mateon’s President and Chief Executive Officer. "Most importantly, we announced significant new overall survival and progression-free survival findings from the Phase 2 GOG-0186I Study. I am confident in our strategic direction, pleased with the continued development of our clinical pipeline and look forward to creating long-term shareholder value."

Financial Results for the Second Quarter of 2016

For the second quarter of 2016, Mateon reported a net loss of $3.6 million compared to a net loss of $3.3 million for the second quarter of 2015. R&D expenses increased to $2.4 million in the second quarter of 2016, compared to $2.0 million in the second quarter of 2015, while general and administrative expenses were $1.3 million for both the second quarter of 2016 and the second quarter of 2015.

At June 30, 2016, Mateon had cash, cash equivalents and short-term investments of $19.3 million, which the Company currently believes is sufficient to fund operations through the availability of key clinical data from the FOCUS Study, expected in the second half of 2017.

On August 03, 2016 Insys Therapeutics, Inc. (NASDAQ:INSY) ("Insys" or "the Company") reported financial results for the three-month period ended June 30, 2016 (Press release, Insys Therapeutics, AUG 3, 2016, View Source;p=RssLanding&cat=news&id=2192279 [SID:1234514202]).

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Highlights of and subsequent to the second quarter of 2016 include:

Total net revenue was $67.1 million, compared to $77.6 million for the second quarter of 2015;

Net income totaled $4.4 million, or $0.06 per basic and $0.06 per diluted share, compared to net income of $7.3 million, or $0.10 per basic and $0.10 per diluted share, for the second quarter of 2015;

Cash, cash equivalents and investments were $193.7 million as of June 30, 2016; and

Insys received FDA approval for the marketing of SyndrosTM (dronabinol oral solution), a proprietary, orally administered liquid formulation of dronabinol.
"We are pleased that despite the recent drop-off in volume, Subsys has maintained an approximately 44% market share. We believe that Subsys will continue to provide a very solid financial foundation for the Company, and we remain focused on restoring Subsys scripts to a growth path," said Dr. John N. Kapoor, Chairman, President and Chief Executive Officer of Insys Therapeutics. "The recent FDA approval to market Syndros is excellent news. We are eager to expand our commercial portfolio with a product that we believe has distinct advantages over the current formulation of dronabinol in soft gel capsule and one that will provide significant long-term growth opportunities for Insys. We are proud of our pipeline and believe that product candidates in both sprays and cannabinoids hold great promise. As always, we remain committed to serving the patients who rely on our compounds, while striving to deliver value to Insys stockholders," he concluded.

Second Quarter 2016 Financial Results

Net revenue for the second quarter of 2016 was $67.1 million compared to $77.6 million for the second quarter of 2015, a decrease of 13.5%. The results reflect a decline in Subsys prescription volumes due to softness in overall demand in the TIRF category, including Subsys, and continued pressure from third-party payers.

Gross margin was 91% for the second quarter of 2016 compared with 89% for the comparable quarter of 2015.

Sales and marketing expense was $19.7 million during the second quarter of 2016, or 29% of net revenue, compared to $22.0 million, or 28% of net revenue, for the second quarter of 2015.

Research and development expense increased to $22.9 million for the second quarter of 2016, compared to $17.8 million for the second quarter of 2015, as we continue to advance the multistage products in our pipeline.

General and administrative expense decreased to $13.9 million for the second quarter of 2016, down from $15.3 million for the second quarter of 2015.

Income tax expense was $240,000 for the second quarter of 2016, compared to $4.8 million during the second quarter of 2015.

Net income for the second quarter of 2016 was $4.4 million, or $0.06 per basic and $0.06 per diluted share, compared to net income of $7.3 million, or $0.10 per basic and $0.10 per diluted share, for the second quarter of 2015. Non-GAAP adjusted net income for the second quarter of 2016 was $9.5 million, or $0.13 per diluted share, compared to non-GAAP adjusted net income of $15.6 million, or $0.21 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.

Liquidity

The Company had $193.7 million in cash, cash equivalents, and short-term and long-term investments, no debt, and $259 million in stockholders’ equity as of June 30, 2016.