On March 9, 2016 Trillium Therapeutics Inc. (NASDAQ:TRIL; TSX: TR) a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial results for the year ended December 31, 2015 and provides a corporate update (Press release, Trillium Therapeutics, MAR 9, 2016, View Source [SID:1234509480]).

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!

"2015 was a pivotal year for Trillium with the successful advancement of TTI-621, our checkpoint inhibitor targeting CD47, into human clinical trials in patients with advanced hematologic malignancies and we look forward to providing an update on this trial by the end of this year," commented Trillium’s Chief Executive Officer, Dr. Niclas Stiernholm. "Another important event that took place in 2015 was the release of our in vitro data demonstrating markedly low binding of TTI-621 to human red blood cells, which may be an important differentiation factor from other agents targeting CD47, such as monoclonal antibodies."

2015 Highlights:

Filed our investigational new drug application with the FDA and initiated our multi-center, open-label Phase 1a/1b trial, evaluating TTI-621 as a single-agent in patients with relapsed or refractory hematologic malignancies. During the dose escalation phase set to enroll up to 36 subjects, we intend to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics in order to determine the optimal dose for subsequent enrollment in the expansion phase. In the second part of the trial, we intend to explore the safety and preliminary antitumor activity of TTI-621 at the optimal dose identified in the escalation phase in 12–15 subjects per hematologic malignancy type, which includes indolent B-cell lymphoma, aggressive B-cell lymphoma, T-cell lymphoma, Hodgkin lymphoma, chronic lymphocytic leukemia, multiple myeloma, acute myeloid leukemia, and myelodysplastic syndrome.

Subsequent to the end of the year, Trillium acquired privately-held Fluorinov Pharma Inc. to gain an in-house proprietary fluorine-based chemistry platform and several preclinical oncology programs.

Presented data from an expanded pool of human donors that conclusively demonstrated that TTI-621, which targets the CD47 "do not eat" signal, exhibits only minimal binding to human red blood cells (RBCs) despite their high expression of CD47. These data are in direct contrast to anti-CD47 monoclonal antibodies, which bind strongly to RBCs. Consequently, compared to antibodies TTI-621 may be less likely to induce anemia in patients, may result in improved pharmacological properties due to the avoidance of the antigen "sink" effect caused by RBCs, and may avoid interference with laboratory blood typing tests.

Presented data at the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) demonstrating that TTI-621 triggers macrophage-mediated phagocytosis of a broad range of human B cell tumors and was effective at controlling the growth of aggressive B cell lymphoma xenografts in mice.

Strengthened our research and drug development teams, including the appointment of Eric L. Sievers, MD, who joined us as Chief Medical Officer from Seattle Genetics.

Completed an underwritten public offering of US$55 million in April providing funds enabling the clinical advancement and expansion of our TTI-621 program. Participating investors included several premier US healthcare funds.
2015 Financial Results:

Our cash increased to $86,770,542 at December 31, 2015 compared to $26,165,056 at December 31, 2014 due mainly to funds received in the April 2015 public offering, warrant exercises and foreign exchange gains, partially offset by cash used in operations.

Net loss for the year ended December 31, 2015 of $14,733,699 exceeded the loss of $12,881,820 for the comparable prior year due mainly to higher costs for our SIRPaFc development program including increased personnel costs.

For the year ended December 31, 2015, research and development costs increased over the comparable prior year as we completed IND-enabling toxicology studies, incurred manufacturing costs to supply our clinical trial, completed the IND submission and initiated the Phase I trial in 2015. Personnel costs were also higher in 2015 as we added staff to manage our expanded research and development activities.

General and administrative expenses for the year ended December 31, 2015 were higher than the comparable prior year due mainly to higher insurance expenses, Fluorinov acquisition costs, and personnel-related costs, partially offset by lower stock exchange filing fees.

Finance income for the year ended December 31, 2015 was higher than the comparable prior year due mainly to a net foreign currency gain of $6,106,703 due mainly to holding U.S. dollar denominated cash with a strengthening U.S. dollar.

Selected Consolidated Financial Information:

Consolidated Statements of Loss and Comprehensive Loss

Amounts in Canadian dollars ​Year ended December 31, 2015 Year ended December 31, 2014
Research and development expenses 18,050,091 10,595,808
General and administrative expenses 3,184,347 2,577,460
Net finance income (6,510,241) (291,448)
Net loss and comprehensive loss for the year 14,733,699 12,881,820
Basic and diluted loss per common share (2.22) (3.06)

Consolidated Statements of Financial Position

Amounts in Canadian dollars As at December 31, 2015 As at December 31, 2014
Cash 86,770,542 26,165,056
Total assets 90,039,468 28,186,032
Total equity 85,803,868 24,304,294

On March 09, 2016 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, reported the publication of an article describing the use of Sleeping Beauty non-viral gene transfer technology to modify T cells for the targeting of neoantigens present within solid tumors (Press release, Ziopharm, MAR 9, 2016, View Source [SID:1234509448]). This approach unlocks the potential for rapid and inexpensive personalized T-cell receptor (TCR) gene therapy aimed at the unique mutations within a patient’s cancer cells. The article, titled "Stable, non-viral expression of mutated tumor neoantigen-specific T-cell receptors using the Sleeping Beauty transposon/transposase system," was published in the journal Molecular Therapy (5 March 2016, doi:10.1038/mt.2016.51), and is available online.

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 DNA plasmids from the Sleeping Beauty platform provide a customizable solution to personalized TCR gene therapy, which is needed to open the door to safely target solid tumors," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM and an author of the paper. "There is growing consensus that a non-viral approach to gene therapy will be essential to targeting individual neoantigens. In Sleeping Beauty, we have the only clinically validated non-viral gene transfer platform. This compels us to bring this important technology to patients."

Neoantigens unique to each patient’s tumor can be recognized by autologous T cells through their T-cell receptors, but the low frequency and/or terminal differentiation of mutation-specific T cells may limit their utility as adoptive T-cell therapies. The publication describes how the Sleeping Beauty platform can be used to transfer and stably express TCR genes into T cells while retaining their proliferative potential. Indeed, the Sleeping Beauty gene transfer occurs without the need for cell division thus facilitating the genetic modification of minimally-differentiated T cells. The authors note that the Sleeping Beauty platform "can facilitate the use of personalized T cell therapy targeting unique neoantigens."

The Sleeping Beauty transposon-transposase system was exclusively licensed by Intrexon Corporation (NYSE:XON) through the University of Texas MD Anderson Cancer Center and accessed as part of ZIOPHARM’s collaboration with Intrexon.

On March 9, 2016 Mirati Therapeutics, Inc. (NASDAQ: MRTX) reported financial results for the fourth quarter and full year ended December 31, 2015 and provided an update on its drug development programs (Filing, Q4/Annual, Mirati, 2015, MAR 9, 2016, View Source [SID:1234509450]).

"We made significant progress across our entire pipeline in 2015, setting up a potentially transformative year for the Company in 2016," said Charles M. Baum, M.D., Ph.D., president and CEO of Mirati. "After reporting initial results in our Phase 1b dose expansion clinical trial for glesatinib last fall, which included two confirmed responses in non-small cell lung cancer patients, we have quickly moved into a Phase 2 clinical trial utilizing our diagnostic collaborations with Guardant Health and Foundation Medicine to help us identify which NSCLC patients we believe are most likely to respond."

"We expect to see updated data from both of those trials later this year, as well as data from our Phase 1b dose expansion clinical trial for sitravatinib, and we are looking forward to starting our Phase 2 combination trial in immuno-oncology for mocetinostat in the second quarter. Following our successful financing last September, we are now poised to capitalize on the significant potential of our targeted cancer therapies for patients in need of better treatment options."

2015 Operational Highlights

Glesatinib (MGCD265): Molecularly targeted kinase inhibitor

· In September 2015, presented data at the World Conference on Lung Cancer 2015 demonstrating the favorable tolerability and clinical efficacy of MGCD265 in a Phase 1b dose expansion clinical trial
· In December 2015, initiated a single arm, open-label Phase 2 clinical trial in NSCLC patients with driver alterations in MET — which occur in up to 7% of NSCLC patients
· In December 2015, announced a collaboration with Guardant Health to use the Guardant360 diagnostic tool in the Phase 2 clinical trial to screen NSCLC patients for certain genetic alterations to the MET pathway in order to identify those patients we believe are most likely to respond to MGCD265
· In December 2015, announced a separate collaboration with Foundation Medicine for the development of a companion diagnostic test for MGCD265 in NSCLC

Sitravatinib (MGCD516): Molecularly targeted kinase inhibitor

· In September 2015, presented interim clinical data from the ongoing Phase 1 dose escalation clinical trial of MGCD516 in patients with advanced solid tumors at the European Cancer Congress (ECC) 2015:
· Demonstrated that MGCD516 is well tolerated with a favorable pharmacokinetic profile
· Established recommended Phase 2 dose of 150 mg QD

· In December 2015, initiated the Phase 1b dose expansion clinical trial in genetically selected patients
· Initial focus on NSCLC in patients with genetic driver mutations, including RET, CHR4q12, CBL, Trk and DDR with exploratory cohorts in other solid tumors where the MGCD516 profile may provide benefit

Mocetinostat (MGCD103): Class I & IV HDAC inhibitor

· In August 2015, announced an immuno-oncology clinical trial collaboration with MedImmune, the global biologic research and development arm of AstraZeneca, to evaluate the safety and efficacy of mocetinostat in combination with durvalumab, an investigational anti-PD-L1 immune checkpoint inhibitor
· Phase 2 clinical trial will be conducted in patients with NSCLC including those who are PD-L1 low and who have failed prior checkpoint inhibitor treatment, two significant areas of unmet medical need

Corporate:

· Executed two successful financings in 2015, generating net proceeds of $143.3 million for the Company
· In February 2015, completed offering of 2.6 million shares of common stock at $20.00 per share
· In September 2015, completed offering of 2.3 million shares of common stock at $45.00 per share

2016 Milestones

· Update on Phase 1b dose expansion clinical trial of MGCD265 expected in the second quarter of 2016
· Initial data from Phase 2 clinical trial of MGCD265 in NSCLC patients with driver alterations in MET is expected by the end of 2016
· Preliminary data from the Phase 1b dose expansion clinical trial for MGCD516 in genetically selected patients is expected in the second half of 2016
· Phase 2 clinical trial for mocetinostat in combination with durvalumab in patients with NSCLC is expected to begin in the second quarter of 2016

Fourth Quarter and Fiscal Year 2015 Financial Results

Cash, cash equivalents, and short-term investments were $122.3 million at December 31, 2015, compared to $29.3 million at December 31, 2014. In September 2015, the Company completed a public offering of 2.3 million shares of its common stock, generating net proceeds of $94.9 million. In February 2015, the Company completed a public offering of 2.6 million shares of its common stock, generating net proceeds of $48.4 million.

Research and development expenses for the fourth quarter of 2015 were $14.9 million, compared to $7.1 million for the same period in 2014. Research and development expenses for the year ended December 31, 2015 were $49.0 million, compared to $26.1 million for the same period in 2014. The increases in research and development expenses primarily reflect costs to advance the clinical development of the Company’s three oncology development programs, MGCD265, MGCD516 and mocetinostat. General and administrative expenses for the fourth quarter of 2015 were $3.6 million, compared to $3.4 million for the same period in 2014. General and administrative expenses for the year ended December 31, 2015 were $15.8 million, compared to $12.7 million for

the same period in 2014. The increases in general and administrative expenses primarily reflect higher non-cash stock-based compensation expense.

Other income and expense, net, was income of $0.1 million for both the fourth quarter of 2015 and 2014. Other income and expense, net, for the year ended December 31, 2015 was income of $0.2 million compared to expense of $4.6 million for the same period in 2014. Other income and expense, net, for the year ended December 31, 2014 primarily reflects losses arising from the change in fair value of our warrant liability. During 2014, we amended the warrant agreements to allow for the warrants to be denominated in U.S. dollars. The amended warrants qualified for equity classification and were reclassified into stockholders’ equity.

Net loss for the fourth quarter of 2015 was $18.4 million, or $0.96 per share basic and diluted, compared to net loss of $10.4 million, or $0.77 per share basic and diluted for the same period in 2014. Net loss for the year ended December 31, 2015 was $64.5 million, or $3.82 per share basic and diluted, compared to net loss of $43.7 million, or $3.24 per share basic and diluted for the same period in 2014.

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 March 9, 2016 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 financial results for the three-month period and year ended December 31, 2015 (Press release, Spectrum Pharmaceuticals, MAR 9, 2016, View Source [SID:1234509452]).

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 had solid operating performance this quarter and our pipeline has never been stronger with multiple drugs enrolling in late-stage trials," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "We believe each of our late-stage drugs have demonstrated strong clinical data, and can be transformative to the Company. We just started enrolling the pivotal Phase 3 study for SPI-2012 and a Phase 2 study for poziotinib. We have two drugs lined up for FDA decision this year: Evomela in May, and EOquin in December. We remain focused on bringing innovative oncology medicines to the market."

Pipeline Update:

SPI-2012, a novel long-acting GCSF: A pivotal Phase 3 study was initiated in Q1 2016 and will evaluate SPI-2012 as a treatment for chemotherapy-induced neutropenia in approximately 580 patients with breast cancer. In a Phase 2 dose ranging study, SPI-2012 was shown to have a shorter duration of severe neutropenia at the higher dose tested and comparable at the middle dose compared to the blockbuster drug pegfilgrastim. SPI-2012 was also shown to have an acceptable safety profile with no significant dose-related or unexpected toxicities.

Poziotinib, a potential best-in-class, novel, pan-HER inhibitor: Spectrum initiated a Phase 2 breast cancer program in the U.S., based on promising Phase 1 efficacy data in breast cancer patients who had failed multiple other HER2-directed therapies. The Company submitted the Phase 2 protocol to the FDA as part of an Investigational New Drug (IND) application in November 2015. In addition, multiple Phase 2 studies are being conducted by Hanmi Pharmaceuticals and National OncoVenture in South Korea.

EVOMELA, a propylene-glycol free melphalan formulation: After receiving a Complete Response Letter in October, Spectrum was granted a Type A meeting with the FDA on November 6, 2015. Within days, the company resubmitted the NDA and received a PDUFA date of May 9, 2016. If approved, we plan to launch Evomela with our existing sales force.

EOquin, a potent tumor-activated drug for non-muscle invasive bladder cancer: Spectrum filed an NDA based on the previous Phase 3 studies. The FDA accepted the NDA and has given Spectrum a PDUFA date of December 11, 2016. The FDA also indicated that it plans to hold an advisory committee meeting regarding the NDA. The Company is actively enrolling an additional randomized, placebo-controlled Phase 3 trial under the SPA agreement. The Phase 3 study has been specifically designed to build on learnings from the previous EOquin Phase 3 studies, as well as recommendations from the FDA.

Three-Month Period Ended December 31, 2015 (All numbers are approximate)

GAAP Results

Total product sales were $34.8 million in the fourth quarter of 2015. Total product sales decreased 33% from $51.7 million in the fourth quarter of 2014.

Product sales in the fourth quarter included: FUSILEV (levoleucovorin) net sales of $15.1 million, FOLOTYN (pralatrexate injection) net sales of $10.3 million, ZEVALIN (ibritumomab tiuxetan) net sales of $3.7 million, BELEODAQ (belinostat) for injection net sales of $3.0 million, and MARQIBO (vinCRIStine sulfate LIPOSOME injection) net sales of $2.7 million. FUSILEV sales exceeded our expectations in the fourth quarter, however, we continue to expect significant declines in the future due to additional competition and pricing pressure.

Spectrum recorded net loss of $4.2 million, or $0.06 per basic and diluted share in the three-month period ended December 31, 2015, compared to net loss of $3.0 million, or $0.05 per basic and diluted share in the comparable period in 2014. Total research and development expenses were $15.4 million in the quarter, as compared to $14.4 million in the same period in 2014. Selling, general and administrative expenses were $21.2 million in the quarter, compared to $24.5 million in the same period in 2014.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $4.6 million, or $0.07 per basic share and diluted share in the three-month period ended December 31, 2015, compared to non-GAAP net income of $7.5 million, or $0.12 per basic and $0.09 per diluted share in the comparable period in 2014. Non-GAAP research and development expenses were $14.8 million as compared to $14.0 million in the same period of 2014. Non-GAAP selling, general and administrative expenses were $18.1 million, as compared to $21.4 million in the same period in 2014.

Twelve-Month Period Ended December 31, 2015 (All numbers are approximate)

GAAP Results

Total product sales were $136.9 million for the twelve months ended December 31, 2015. Total product sales decreased 27% from $186.5 million in the same period of 2014.

Product sales in 2015 included: FUSILEV (levoleucovorin) net sales of $60.7 million, FOLOTYN (pralatrexate injection) net sales of $40.6 million, ZEVALIN (ibritumomab tiuxetan) net sales of $17.5 million, BELEODAQ (belinostat) for injection net sales of $10.1 million, and MARQIBO (vinCRIStine sulfate LIPOSOME injection) net sales of $8.0 million.

Spectrum recorded net loss of $50.8 million, or $0.78 per basic and diluted share in the twelve-month period ended December 31, 2015, compared to net loss of $45.7 million, or $0.71 per basic and diluted share in the comparable period in 2014. Total research and development expenses were $50.8 million for the year, as compared to $69.7 million in the same period in 2014. Selling, general and administrative expenses were $86.5 million for the year, compared to $97.4 million in the same period in 2014.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $17.6 million, or $0.27 per basic and diluted share in the twelve-month period ended December 31, 2015, compared to non-GAAP net income of $21.4 million, or $0.33 per basic and $0.27 per diluted share in the comparable period in 2014. Non-GAAP research and development expenses were $45.7 million as compared to $50.0 million in the same period of 2014. Non-GAAP selling, general and administrative expenses were $77.9 million, as compared to $84.9 million in the same period in 2014.

On March 9, 2016 OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported year end 2015 financial results and provided a summary of anticipated milestones (Press release, OncoGenex Pharmaceuticals, MAR 9, 2016, View Source [SID:1234509458]).

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!

Financial Results and Anticipated Near-term Milestones

As of December 31, 2015, the company’s cash, cash equivalents and short-term investments increased to $55.2 million from $47.1 million as of December 31, 2014.

Based on current expectations, OncoGenex believes that its cash, cash equivalents and short-term investments will be sufficient to fund its currently planned operations into the third quarter of 2017. Depending on timing of enrollment or event-driven final analyses, the expected key milestones and activities are as follows:

Custirsen
Announcing AFFINITY trial results, the phase 3 trial evaluating a survival benefit for custirsen in combination with cabazitaxel as second-line chemotherapy in approximately 630 patients with castrate-resistant prostate cancer. The final analysis for the intent-to-treat population is expected in the third quarter of 2016.

Announcing ENSPIRIT trial results, the phase 3 trial evaluating a survival benefit for custirsen in combination with docetaxel as second-line chemotherapy in approximately 700 patients with non-small cell lung cancer. The final survival analysis is expected in the first half of 2017.

Apatorsen
Announcing Borealis-2 trial results, an investigator-sponsored, randomized phase 2 trial evaluating apatorsen in combination with docetaxel treatment compared to docetaxel treatment alone in patients with advanced or metastatic bladder cancer. Final results are expected in the second half of 2016.

Announcing Spruce trial results for the overall survival endpoint, the investigator-sponsored, randomized, placebo-controlled phase 2 trial evaluating apatorsen treatment with carboplatin and pemetrexed chemotherapy in patients with previously untreated advanced non-squamous NSCLC. Results, including evaluation of patients with high Hsp27 expression, are expected in the second half of 2016.

Preparing an investigational new drug application for FDA submission. The proposed Phase 1/2 study design would evaluate apatorsen for intravesical administration in combination with Bacillus Calmette-Guerin (BCG) treatment in patients with non-muscle invasive bladder cancer. In its feedback to OncoGenex at a pre-IND meeting, the FDA supported the study population and classification of subpopulations and deemed proposed definitions of primary and secondary endpoints acceptable.

Revenue for the fourth quarter and year ended December 31, 2015 was $6.0 million and $18.2 million, respectively. This compares with $5.7 million and $27.1 million, respectively, in the same periods in 2014. The decrease in 2015 as compared to 2014 was due primarily to lower collaboration revenue recognized for the reimbursement of expenses for the AFFINITY trial as a result of patients coming off treatment. This was partially offset by higher ENSPIRIT trial costs, which OncoGenex became responsible for pursuant to the Termination Agreement with Teva. Revenue recognized in 2015 is attributable to the advance reimbursement received in the second quarter of 2015, pursuant to the Termination Agreement with Teva, for research and development costs incurred by OncoGenex related to the custirsen development program.

Total operating expenses for the fourth quarter and year ended December 31, 2015 were $9.5 million and $36.9 million, respectively. Net loss for the fourth quarter and year ended December 31, 2015 was $1.7 million and $16.8 million, respectively.

As of March 9, 2016, OncoGenex had 29,812,998 shares outstanding.