On October 13, 2016 Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) reported that it has submitted its responses to European Medicines Agency (EMA) Day 120 List of Questions issued by the Committee for Medicinal Products for Human Use (CHMP) as part of the centralized review process of the Marketing Authorization Application (MAA) for vosaroxin as a treatment for relapsed/refractory acute myeloid leukemia (AML) in patients aged 60 years and older (Press release, Sunesis, OCT 13, 2016, View Source [SID:SID1234515808]). Sunesis expects to receive the EMA Day 180 List of Outstanding Issues before year-end.

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 are pleased with the comprehensive set of answers and additional analyses that our team has provided in response to the EMA’s Day 120 list of questions," said Daniel Swisher, President and Chief Executive Officer of Sunesis. "As our regulatory efforts progress to bring vosaroxin to market in Europe as a treatment for relapsed/refractory AML, we are advancing active dialogues with potential pharma collaborators toward the goal of supporting a market launch in 2017. With the clock now restarting, we anticipate being one step closer to that goal."

About QINPREZO (vosaroxin)
QINPREZO (vosaroxin) is an anti-cancer quinolone derivative (AQD), a class of compounds that has not been used previously for the treatment of cancer. Preclinical data demonstrate that vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis. Both the U.S. Food and Drug Administration (FDA) and European Commission have granted orphan drug designation to vosaroxin for the treatment of AML. Vosaroxin is an investigational drug that has not been approved for use in any jurisdiction.

Vosaroxin’s Marketing Authorization Application for relapsed refractory AML is currently under review by the European Medicines Agency, and a regulatory decision regarding approval is expected in 2017.

The trademark name QINPREZO is conditionally accepted by the FDA and the EMA as the proprietary name for the vosaroxin drug product candidate.

On October 13, 2016 VBL Therapeutics (NASDAQ:VBLT), reported that it has engaged in a long-term lease contract of a new stand-alone facility in Modiin, Israel. The site will house VBL’s local biological drugs manufacturing facility, as the company plans ahead for potential commercialization of VB-111.

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 site design enables modular expansion of the manufacturing capacity, to supply growing demand following commercialization, while requiring only limited capital resources in the immediate stage. The near-term investment is not expected to materially impact VBL’s cash position. The Company projects that its current cash will suffice to support operations into 2019, beyond the readout of the pivotal GLOBE trial, while supporting a potential registration trial in ovarian cancer and the investment in the new facility. In addition, VBL expects that the establishment of the new facility will qualify for increased governmental financial incentives.

"Securing this new facility in Modiin fits strategically with our development plans and is an important step as we advance multiple clinical trials and move closer to potential commercialization of VB-111," stated Dror Harats, Chief Executive Officer of VBL Therapeutics.

The new facility will also include the company’s headquarters, discovery research and clinical development. VBL intends to operate and relocate to the new site in the second half of 2017.

VBL said that future commercial supply of VB-111 will likely involve a complementary source of supply, probably via a Contract Manufacturing partner in North America, although those plans have not yet been set.

On October 13, 2016 Heat Biologics, Inc. (Nasdaq:HTBX), an immuno-oncology company developing novel therapies that activate a patient’s immune system against cancer, reported that it continues to remain on track to report topline data this quarter from its Phase 2 trials evaluating HS-410 and HS-110 for the treatment of non-muscle invasive bladder cancer (NMIBC) and non-small cell lung cancer (NSCLC), respectively, as well as its Phase 1b trial evaluating HS-110 in combination with Bristol-Myers Squibb’s anti-PD-1 checkpoint inhibitor, Opdivo, for the treatment of NSCLC (Press release, Heat Biologics, OCT 13, 2016, View Source [SID:SID1234515797]).

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 are pleased with the progress we are making and look forward to reporting top-line data in both our NMIBC and NSCLC trials later this quarter," commented Jeff Wolf, Heat’s Founder and CEO. "We are encouraged by our early data in bladder cancer that suggests we are activating a robust antigen-specific immune response and our data in lung cancer that suggest HS-110 may improve response rates for patients with ‘cold tumors’ who typically have lower response rates to checkpoint inhibitor monotherapy."

On October 13, 2016 TG Therapeutics, Inc. (NASDAQ:TGTX) reported that it has filed with the FDA an amended protocol for the GENUINE Phase 3 trial (Press release, TG Therapeutics, OCT 13, 2016, View Source [SID:SID1234515796]). Prior to the amendments, the GENUINE study consisted of two parts:

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!

Part I to evaluate the effect of the addition of TG-1101 to ibrutinib on overall response rate (ORR) in approximately the first 200 patients enrolled, to support a filing for accelerated approval of TG-1101; and
Part II to evaluate the effect of the addition of TG-1101 to ibrutinib on progression-free survival (PFS) in all study patients (approximately 330), to support a filing for full approval of TG-1101.
The amended protocol contains the following substantive changes:

Part II of the study has been eliminated, and accordingly, the study’s sole primary endpoint will be ORR as originally contemplated in Part I; and
Target enrollment has been reduced to approximately 120 randomized patients.

At the new study size, the study is 90% powered to show a statistically significant improvement in ORR, with the minimal detectable difference of approximately 20% (absolute difference between the arms). Additionally, patients will be followed until progression, but the study will no longer be powered for PFS.

The Company expects that it will complete enrollment in the revised trial by year end 2016, and will have topline data available in the first half of 2017. If the results of the study are positive, the Company plans to request a pre-BLA meeting to discuss the data and a filing strategy with the FDA. The Company has communicated with the FDA regarding its intention to file a BLA for accelerated approval if the results are positive and the FDA has agreed that a pre-BLA meeting can be requested based on ORR data from the GENUINE study. Assuming a positive outcome of a pre-BLA meeting, targeted to occur in the fourth quarter of 2017, the Company believes it could file a BLA in the first half of 2018.

Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "Today’s announcement marks an important milestone for the Company. Given the GENUINE enrollment challenges we’ve faced to date, we are very excited to accelerate the trial to a rapid conclusion, while also maintaining the ability to potentially file the data for accelerated approval. The GENUINE study, as amended, remains a robust, randomized clinical trial, which we believe, if positive, could support accelerated approval for patients with relapsed/refractory high-risk CLL. Moreover, we believe the amended study and revised regulatory strategy is consistent with the recent accelerated approvals for novel agents in CLL, which notably were not pursuant to an SPA but occurred after the finding of positive ORR results. Importantly, with completion of enrollment now expected by year end, we and our clinical trial sites can focus our resources on completing our UNITY-CLL Phase 3 trial as quickly as possible. Early enrollment in UNITY-CLL is very encouraging and we anticipate that study will be fully enrolled before filing a BLA for the GENUINE study. UNITY-CLL remains unchanged and unaffected by the amendments to the GENUINE study, and if positive, could support full approval for both TG-1101 and TGR-1202 based on its primary endpoint of PFS." Mr. Weiss continued, "We have greatly appreciated all of the guidance and counsel from the FDA in designing our clinical programs and we look forward to continuing our collaborative working relationship as we accelerate toward the conclusion of enrollment into the GENUINE study this year and ORR data in the first half of 2017."

On October 13, 2016 OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported results from the final analysis of the Phase 3 ENSPIRIT trial of custirsen in patients whose non-small cell lung cancer (NSCLC) has progressed following initial treatments (Press release, OncoGenex Pharmaceuticals, OCT 13, 2016, View Source [SID:SID1234515793]). The trial did not meet the primary endpoint of demonstrating a statistically significant improvement in overall survival for patients treated with custirsen in combination with docetaxel compared to docetaxel alone. The median overall survival for the custirsen arm was 9.0 months versus 7.9 months for the control arm with a hazard ratio of 0.915 (one-sided p=0.178). Safety results were consistent with those observed in previous trials of custirsen in combination with chemotherapy.

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!

"Following the negative results of previous custirsen trials, an early final analysis of the ENSPIRIT trial was conducted in an effort to conserve capital and extend our cash runway," said Scott Cormack, President and CEO of OncoGenex. "We will continue to take appropriate steps to realize the most value for our shareholders once we receive the results of our apatorsen Phase 2 Borealis-2 bladder cancer trial which are expected by the end of October."

OncoGenex is continuing to work with MTS Health Partners who has been advising the company in the exploration of strategic alternatives since mid-August.

"OncoGenex is grateful to the patients who participated in the ENSPIRIT trial and their families for their support, as well as our investigators and our employees for their commitment to improving cancer care for those who need it most," Cormack continued.

About the Phase 3 ENSPIRIT Trial
The Phase 3 ENSPIRIT trial is an international, randomized, open-label trial designed to evaluate custirsen for the treatment of advanced or metastatic NSCLC in patients who have progressed after initial chemotherapy treatment. The trial investigated if combining custirsen with docetaxel, a standard second-line NSCLC chemotherapy, has the potential to improve survival outcomes compared to docetaxel alone in these patients. The trial enrolled 664 patients at approximately 50 sites globally.

For more information on the ENSPIRIT trial, please visit View Source

About the Borealis-2 Trial
Borealis-2 is an investigator-sponsored, randomized Phase 2 trial evaluating a survival benefit with apatorsen in combination with docetaxel treatment compared to docetaxel treatment alone in approximately 200 patients with metastatic bladder cancer who have disease progression following first-line platinum-based chemotherapy. The trial is being coordinated by the Hoosier Cancer Research Network at 27 sites across the United States.