On May 2,2017 Celyad (Euronext Brussels and Paris, and NASDAQ:CYAD), a leader in the discovery and development of cell therapies, reported a non-exclusive license agreement with Novartis for Celyad’s US patents for the production of allogeneic CAR-T cells (Press release, Celyad, MAY 2, 2017, View Source [SID1234518791]). This license agreement is related to two targets currently under development by Novartis.

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The agreement includes Celyad’s intellectual property rights under United States Patent No. 9,181,527 related to allogeneic human primary T-Cells that are engineered to be T-Cell Receptor (TCR) deficient and express a Chimeric Antigen Receptor (CAR). The granted claims are not limited to specific CARs or specific methods of generating allogeneic CAR T-cells, such as genome editing or genetic engineering.

Under the terms of the agreement Celyad receives an upfront payment and is eligible to receive success based clinical, regulatory and commercial milestone payments. If all success based milestones are achieved, Celyad is eligible to receive payments, including the upfront payment, totalling $96 million. In addition, Celyad will receive single digit royalties based on net sales of the licensed target associated products. Novartis has the option to extend the agreement to additional targets and/or to convert its license into an exclusive license. Celyad retains all rights to grant further licenses to third parties for the use of allogeneic CAR-T cells.

Celyad will not be involved in the development of Novartis’ CAR-T cells. Celyad will continue to focus on the development of its CAR-T pipeline, including its allogeneic NKR-2 T-cell immunotherapy in the EU and US territories and in collaboration with Ono Pharmaceuticals, its partner in Japan, Taiwan and Korea.

Dr. Christian Homsy, CEO of Celyad, said: "This non-exclusive agreement with Novartis recognizes the importance of our IP for companies developing allogeneic CAR-T therapies".

On May 2, 2017 Aduro Biotech, Inc. (NASDAQ:ADRO) reported financial results for the first quarter ended March 31, 2017 (Press release, Aduro Biotech, MAY 2, 2017, View Source [SID1234518790]). Net loss for the first quarter of 2017 was $21.8 million, or $0.32 per share, compared to a net loss of $28.8 million, or $0.45 per share for the same period in 2016.

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Cash, cash equivalents and marketable securities totaled $356.0 million at March 31, 2017, compared to $361.9 million at December 31, 2016.

"This will be an important year for Aduro, as we generate data in our ongoing ADU-S100/STING monotherapy trial and our planned Phase 2 trial in mesothelioma, as well as look for data from Janssen’s Phase 1 trials in lung and prostate cancers evaluating LADD therapeutic candidates," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "We also plan to advance our STING program into additional clinical studies in collaboration with Novartis, and the first antibody from our B-select platform, the novel anti-APRIL antibody, is expected to be cleared for clinical testing this year. With ten product candidates in our diversified portfolio and a healthy balance sheet, we are in a strong position to continue to advance our pipeline and build a leading immunotherapy company."

Key Recent Accomplishments

Established a clinical collaboration with Merck to evaluate the combination of Aduro’s LADD agent CRS-207 with Merck’s KEYTRUDA (pembrolizumab) in a Phase 2 study in gastric cancer
Entered into an exclusive license agreement with Stanford University for the use of neoantigen identification technology in therapeutics using modified Listeria for our personalized LADD program, pLADD
Expanded Aduro’s Scientific Advisory Board with leading immunotherapy and oncology experts
Presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting on BION-1301, anti-APRIL antibody, and ADU-S100 STING agonist
Presented at the Keystone Symposia on Cancer Immunology and Immunotherapy Conference on ADU-S100 and pLADD
Anticipated 2017 Milestones

Initiate Phase 2 mesothelioma trial with CRS-207 in combination with anti-PD1 in the first half of 2017 and report early results in the second half of 2017
Initiate Phase 2 gastric trial with CRS-207 in combination with anti-PD1 in the first half of 2017
Initiate Phase 1 pLADD (personalized LADD) trial in advanced gastro-intestinal cancers in the second half of 2017
Janssen expected to initiate Phase 1b/2 trial of ADU-214 in lung cancer and determine next steps for ADU-741 in prostate cancer in the second half of 2017
Report top-line findings from Phase 1 monotherapy trial of ADU-S100 in the second half of 2017
In collaboration with Novartis, initiate Phase 1b trial of ADU-S100 in combination with anti-PD1 in the second half of 2017
File Investigational New Drug Application for BION-1301, anti-APRIL antibody, in the second half of 2017
Initiate Phase 1 multiple myeloma trial with anti-APRIL antibody in the second half of 2017
First Quarter 2017 Financial Results
Revenue for the first quarter of 2017 was $3.8 million, compared to $4.0 million for the same period in 2016. The revenue recognized in both quarters primarily relates to deferred upfront payments under the Novartis collaboration agreement. In addition, revenue in the first quarter of 2016 included reimbursed research services of $0.2 million.

Research and development expenses were $20.6 million for the first quarter of 2017, compared to $20.9 million for the same period in 2016. Research and development expenses incurred in the first quarter of 2016 included GVAX Pancreas manufacturing and pancreatic cancer clinical trial expenses, which did not occur in 2017. The decrease in expenses was partially offset by increased costs to manufacture our B-select antibodies and increased research and development expenses for the STING platform, as well as higher personnel and facility related costs in first quarter of 2017.

General and administrative expenses were $8.3 million for the first quarter of 2017, compared to $9.0 million for the same period in 2016. This decrease was primarily due to lower consulting and professional fees.

Income tax benefit was $2.8 million for the first quarter of 2017, compared to a provision for income taxes of $3.2 million for the same period in 2016. The income tax benefit recorded for the first quarter of 2017 was due to the current benefit of federal income taxes paid in 2016.

On May 2, 2017 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, reported results for the three-month period ended March 31, 2017 (Press release, Spectrum Pharmaceuticals, MAY 2, 2017, View Source [SID1234518788]).

“We remain focused on our advanced stage pipeline and look forward to several important milestones in the near future,” said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. “Based on the preclinical results and clinical data from the first compassionate-use patient, treated at MD Anderson Cancer Center by Dr. John Heymach under a compassionate-use protocol approved by the FDA, enthusiasm is building in the scientific community about the potential of poziotinib in non-small cell lung cancer patients with exon 20 insertion mutations. There is immense need for effective therapies in this disease as the current progression free survival is under 2 months. In addition, I am delighted with the recent pace of enrollment of the ROLONTIS Phase 3 program. Since the beginning of this year, we have enrolled over 135 patients in the pivotal trial. We are looking forward to Phase 3 results and a BLA filing next year. With three advanced stage drugs being studied in multiple tumors, I believe Spectrum is poised for transformational growth.”

Pipeline Update:

ROLONTIS (eflapegrastim), a novel long-acting GCSF: A pivotal Phase 3 study (ADVANCE) was initiated under an SPA from the FDA in 2016 to evaluate ROLONTIS in the management of chemotherapy-induced neutropenia. Based on the amended SPA, the size of the ADVANCE study was reduced to 400 from 580 evaluable patients. The ADVANCE study is now 75% enrolled and the Company expects to complete enrollment in the second half of this year. To strengthen the regulatory package in the U.S. and Europe, the Company has initiated the 218-patient RECOVER study, which is expected to include sites not only from the U.S., but also from Europe, Canada and South Korea. For the RECOVER Study, sites have been initiated and first patient enrollment is imminent. The Company continues to expect to file the BLA next year.
Poziotinib, a potential best-in-class, novel, pan-HER inhibitor: An investigator sponsored trial has been initiated at the University of Texas MD Anderson Cancer Center in non-small cell lung cancer patients with EGFR exon 20 insertion mutations. The study is expected to yield interim results before year end. Spectrum is also conducting a Phase 2 breast cancer study in the U.S., based on promising Phase 1 study efficacy data in breast cancer patients who had failed multiple HER2-directed therapies. Further, multiple Phase 2 studies are being conducted in South Korea by Hanmi Pharmaceuticals and National OncoVenture to study breast, lung, head-and-neck and gastric cancer indications.
QAPZOLA, a potent tumor-activated drug being investigated for low and intermediate risk non-muscle invasive bladder cancer: The Company received a new SPA from the FDA for a new Phase 3 study incorporating learnings from the previous studies, as well as recommendations from the FDA. Compared to the previous program, this new Phase 3 study will include fewer evaluable patients (n=425 versus 1,557 patients), use a higher dosage of QAPZOLA (8 mg versus 4 mg), and will evaluate time-to-recurrence as the primary endpoint. The Phase 3 trial is expected to start enrolling patients in the third quarter.
Three-Month Period Ended March 31, 2017 (All numbers are approximate)

GAAP Results

Total product sales were $25.8 million in the first quarter of 2017. Product sales in the first quarter included: FUSILEV (levoleucovorin) net sales of $2.6 million, FOLOTYN (pralatrexate injection) net sales of $9.3 million, ZEVALIN (ibritumomab tiuxetan) net sales of $2.8 million, MARQIBO (vinCRIStine sulfate LIPOSOME injection) net sales of $2.0 million, BELEODAQ (belinostat) for injection net sales of $2.9 million, and EVOMELA (melphalan) for injection net sales of $6.3 million.

Spectrum recorded net loss of $23.0 million, or $0.29 per basic and diluted share in the three-month period ended March 31, 2017, compared to net loss of $9.3 million, or $0.14 per basic and diluted share in the comparable period in 2016. Total research and development expenses were $14.7 million in the quarter, as compared to $15.5 million in the same period in 2016. Selling, general and administrative expenses were $18.6 million in the quarter, compared to $22.0 million in the same period in 2016.

The Company ended the quarter with Cash and Cash Equivalents of $137 million.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $11.4 million, or $0.14 per basic and diluted share in the three-month period ended March 31, 2017, compared to non-GAAP net income of $0.3 million, or $0.01 per basic share and less than $0.01 per diluted share in the comparable period in 2016. Non-GAAP research and development expenses were $14.3 million, as compared to $13.0 million in the same period of 2016. Non-GAAP selling, general and administrative expenses were $15.7 million, as compared to $16.7 million in the same period in 2016.

On May 2, 2017 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, reported results for the three-month period ended March 31, 2017 (Press release, Spectrum Pharmaceuticals, MAY 2, 2017, View Source [SID1234518788]).

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"We remain focused on our advanced stage pipeline and look forward to several important milestones in the near future," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "Based on the preclinical results and clinical data from the first compassionate-use patient, treated at MD Anderson Cancer Center by Dr. John Heymach under a compassionate-use protocol approved by the FDA, enthusiasm is building in the scientific community about the potential of poziotinib in non-small cell lung cancer patients with exon 20 insertion mutations. There is immense need for effective therapies in this disease as the current progression free survival is under 2 months. In addition, I am delighted with the recent pace of enrollment of the ROLONTIS Phase 3 program. Since the beginning of this year, we have enrolled over 135 patients in the pivotal trial. We are looking forward to Phase 3 results and a BLA filing next year. With three advanced stage drugs being studied in multiple tumors, I believe Spectrum is poised for transformational growth."

Pipeline Update:

ROLONTIS (eflapegrastim), a novel long-acting GCSF: A pivotal Phase 3 study (ADVANCE) was initiated under an SPA from the FDA in 2016 to evaluate ROLONTIS in the management of chemotherapy-induced neutropenia. Based on the amended SPA, the size of the ADVANCE study was reduced to 400 from 580 evaluable patients. The ADVANCE study is now 75% enrolled and the Company expects to complete enrollment in the second half of this year. To strengthen the regulatory package in the U.S. and Europe, the Company has initiated the 218-patient RECOVER study, which is expected to include sites not only from the U.S., but also from Europe, Canada and South Korea. For the RECOVER Study, sites have been initiated and first patient enrollment is imminent. The Company continues to expect to file the BLA next year.
Poziotinib, a potential best-in-class, novel, pan-HER inhibitor: An investigator sponsored trial has been initiated at the University of Texas MD Anderson Cancer Center in non-small cell lung cancer patients with EGFR exon 20 insertion mutations. The study is expected to yield interim results before year end. Spectrum is also conducting a Phase 2 breast cancer study in the U.S., based on promising Phase 1 study efficacy data in breast cancer patients who had failed multiple HER2-directed therapies. Further, multiple Phase 2 studies are being conducted in South Korea by Hanmi Pharmaceuticals and National OncoVenture to study breast, lung, head-and-neck and gastric cancer indications.
QAPZOLA, a potent tumor-activated drug being investigated for low and intermediate risk non-muscle invasive bladder cancer: The Company received a new SPA from the FDA for a new Phase 3 study incorporating learnings from the previous studies, as well as recommendations from the FDA. Compared to the previous program, this new Phase 3 study will include fewer evaluable patients (n=425 versus 1,557 patients), use a higher dosage of QAPZOLA (8 mg versus 4 mg), and will evaluate time-to-recurrence as the primary endpoint. The Phase 3 trial is expected to start enrolling patients in the third quarter.
Three-Month Period Ended March 31, 2017 (All numbers are approximate)

GAAP Results

Total product sales were $25.8 million in the first quarter of 2017. Product sales in the first quarter included: FUSILEV (levoleucovorin) net sales of $2.6 million, FOLOTYN (pralatrexate injection) net sales of $9.3 million, ZEVALIN (ibritumomab tiuxetan) net sales of $2.8 million, MARQIBO (vinCRIStine sulfate LIPOSOME injection) net sales of $2.0 million, BELEODAQ (belinostat) for injection net sales of $2.9 million, and EVOMELA (melphalan) for injection net sales of $6.3 million.

Spectrum recorded net loss of $23.0 million, or $0.29 per basic and diluted share in the three-month period ended March 31, 2017, compared to net loss of $9.3 million, or $0.14 per basic and diluted share in the comparable period in 2016. Total research and development expenses were $14.7 million in the quarter, as compared to $15.5 million in the same period in 2016. Selling, general and administrative expenses were $18.6 million in the quarter, compared to $22.0 million in the same period in 2016.

The Company ended the quarter with Cash and Cash Equivalents of $137 million.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $11.4 million, or $0.14 per basic and diluted share in the three-month period ended March 31, 2017, compared to non-GAAP net income of $0.3 million, or $0.01 per basic share and less than $0.01 per diluted share in the comparable period in 2016. Non-GAAP research and development expenses were $14.3 million, as compared to $13.0 million in the same period of 2016. Non-GAAP selling, general and administrative expenses were $15.7 million, as compared to $16.7 million in the same period in 2016.

On May 2, 2017 Shire plc (Shire) (LSE: SHP, NASDAQ: SHPG) reported unaudited results for the three months ended March 31, 2017 (Press release, Shire, MAY 2, 2017, View Source [SID1234518784]).

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Flemming Ornskov, M.D., M.P.H., Shire Chief Executive Officer, commented:

"In the first quarter we delivered strong top-line growth with quarterly product sales of $3.4 billion. I am especially pleased to see that our sales growth came from across our broad portfolio, with genetic diseases growing 14%, our recently launched XIIDRA product achieving a 22% market share and the Baxalta business growing at 8% on a pro forma basis. We also improved our operational efficiency, and are ahead of plan on integrating Baxalta.

"Our priorities for the rest of 2017 remain unchanged: launching new products while driving commercial excellence, generating operational efficiencies, and advancing our pipeline of novel therapies. Additionally, we continue to prioritize paying down debt, and we are on track to achieve our full-year financial guidance.

"Looking ahead, I see tremendous opportunity for further growth as we continue to build on our position as the global leader in treating patients with rare diseases."

Financial Highlights Q1 2017(1) Growth(1) Non GAAP CER(1)(2)
Product sales $3,412 million +110% +110%
Product sales excluding legacy Baxalta $1,807 million +11% +11%
Total revenues $3,572 million +109% +110%

Operating income from continuing operations $497 million (9%)
Non GAAP operating income(2) $1,454 million +82% +82%

Net income margin(3)(4) 10% (15ppc)
Non GAAP EBITDA margin(2)(4) 44% (5ppc)

Net income $375 million (11%)
Non GAAP net income(2) $1,102 million +74%

Diluted earnings per ADS(5) $1.23 (42%)
Non GAAP diluted earnings per ADS(2)(5) $3.63 +14% +14%

Net cash provided by operating activities $459 million +18%
Non GAAP free cash flow (2) $247 million (27%)
(1) Results include Baxalta Inc. (Baxalta) (acquired on June 3, 2016) and Dyax Corp. (Dyax) (acquired on January 22, 2016), unless otherwise noted. Percentages compare to equivalent 2016 period.
(2) The Non GAAP financial measures included within this release are explained on pages 25 – 26, and are reconciled to the most directly comparable financial measures prepared in accordance with US GAAP on pages 19 – 21.
(3) US GAAP net income as a percentage of total revenues.
(4) Percentage point change (ppc).
(5) Diluted weighted average number of ordinary shares 911.8 million.

Financial Highlights

Delivered product sales growth of 110% with strong legacy Shire sales and the inclusion of legacy Baxalta sales.
Achieved combined pro forma sales growth of 9%; legacy Shire sales growth of 11% and legacy Baxalta pro forma sales growth of 8%.
Generated Non GAAP earnings per ADS of $3.63, reflecting strong business fundamentals and operational discipline.
Ahead of schedule with Baxalta integration and on-track to achieve at least $700 million in synergies by year 3.
Debt pay-down continued during the quarter and we remain on-track to achieve our year end debt target.
Product and Pipeline Highlights

Continued to drive expansion of the U.S. dry eye disease market, with XIIDRA increasing its market share to 22% as of March 2017.
Increased CINRYZE sales by 38% to $226 million, reflecting higher patient demand and improvements in available supply.
Increasing demand for our Immunology products, with pro forma sales growth for immunoglobulin therapies and bio therapeutics of 10% and 19% respectively.
Expect U.S. Food and Drug Administration (FDA) decision for SHP465 in Attention Deficit Hyperactivity Disorder (ADHD) on or before June 20, 2017.
Completed enrollment in SHP643 open-label extension study; topline pivotal study results expected in Q2 2017.
Initiated Phase 3 trials for SHP640 and SHP620 in patients with bacterial and adenoviral conjunctivitis and cytomegalovirus infection, respectively.