On November 2, 2015 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP) reported financial results for the third quarter ended September 30, 2015, and provided an update on the company’s recent activities (Press release, Ziopharm, NOV 2, 2015, View Source [SID:1234507894]).

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"The third quarter saw an important expansion of our pipeline with the signing of a new Exclusive Channel Collaboration with our longtime partner Intrexon to include immunotherapies for the treatment of graft-versus-host-disease, or GvHD," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM. "This adds to our research programs with Intrexon in virotherapies, T-cell therapies and natural killer cell therapies for the treatment of cancer, all of which are advancing in the lab and clinic. We look forward to presenting a variety of data at scientific meetings before the end of this year."

Recent Highlights

Ad-RTS-hIL-12

Ad-RTS-hIL-12 is a gene therapy candidate for the controlled expression of interleukin 12 (IL-12), a critical protein for stimulating an anti-cancer T cell immune response, using the RheoSwitch Therapeutic System (RTS) gene switch. ZIOPHARM is currently enrolling patients in two studies of Ad-RTS-hIL-12: a Phase 1b/2 study for the treatment of patients with locally advanced or metastatic breast cancer following standard chemotherapy and a multi-center Phase 1 study in patients with recurrent or progressive glioblastoma multiforme, a form of brain cancer. The Company expects that early results from each study will be presented at scientific meetings prior to year’s end.

In September, ZIOPHARM announced the presentation of preclinical and clinical data from the Company’s Ad-RTS-IL-12 program in various malignancies at the CRI-CIMT-EATI-AACR Inaugural International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) in New York City.

These included a presentation highlighting additional evidence of systemic immune activation with Ad-RTS-hIL-12 and veledimex in advanced melanoma and breast cancer patients. Among other findings, treatment with Ad-RTS-hIL-12 and veledimex in patients with melanoma was found to increase the immune cytokine IL-12 and downstream cytokines, interferon gamma (IFN-g), interferon gamma-inducible protein 10 (IP-10) and interleukin 10 (IL-10), resulting in a significant increase in tumor infiltrating lymphocytes both locally, in injected lesions, and systemically, in non-injected lesions. These data provide clinical evidence of an abscopal response, in which local treatment affects other tumor sites at a distance, and confirm that localized therapy can lead to beneficial systemic anti-tumor effects.

A second presentation demonstrated the anti-tumor effects and tolerability of Ad-RTS-mIL-12 in mouse models of glioblastoma (brain cancer), colon cancer and melanoma. These data showed dose-related increases in veledimex in both plasma and brain tissue, leading to a corresponding elevation in expression of IL-12 mRNA. Ad-RTS-mIL-12 and veledimex also demonstrated systemic memory upon rechallenge in multiple syngeneic mouse models, providing further evidence of systemic anti-tumor immunity elicited by Ad-RTS-mIL-12.

ZIOPHARM announced in July 2015 that the U.S. Food and Drug Administration granted Orphan Drug Designation for Ad-RTS-hIL-12 and veledimex in the treatment of patients with malignant glioma. The FDA’s Office of Orphan Products grants orphan drug status to support development of medicines for underserved patient populations or rare disorders affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides eligibility for a seven-year period of market exclusivity in the United States after product approval, an accelerated review process, accelerated approval where appropriate, grant funding, tax benefits and an exemption from user fees.

Adoptive Cell Therapies

ZIOPHARM is developing various immuno-oncology programs, including chimeric antigen receptor T-cell (CAR-T), T-cell receptor (TCR) and natural killer (NK) adoptive cell based therapies. These programs are being advanced in collaboration with Intrexon, the biopharmaceutical business of Merck KGaA, Darmstadt, Germany, and the MD Anderson Cancer Center.

ZIOPHARM expects that early results from its adoptive cell therapy programs, including data highlighting the Sleeping Beauty non-viral gene transfer technology, will be presented to the medical and scientific community prior to year end.

ZIOPHARM announced in September the publication of a preclinical study in Cancer Research, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), demonstrating the preferential targeting of solid tumor cells over healthy cells using engineered chimeric antigen receptor (CAR) T cells. The publication described how tuning the binding affinity of CARs to activate T cells based on the density of EGFR expression allows for targeting of cancer cells with high levels of EGFR, while sparing normal cells with low levels of EGFR. The study demonstrates an approach for translating the effects of adoptive CAR T-cells from liquid to solid tumors.

GvHD

In September, Intrexon Corporation announced that it has formed a new Exclusive Channel Collaboration with ZIOPHARM for the treatment and prevention of graft-versus-host disease (GvHD). GvHD is a major complication of allogeneic hematopoietic stem-cell transplantation which significantly impairs the quality of life and survival of many recipients, and is unaddressed by existing treatments, which have limited efficacy and increased toxicity. The collaboration will focus on addressing the underlying pathologies of GvHD through engineered cell platforms to express and control delivery of interleukin-2 (IL-2), a cytokine critical for modulation of the immune system. The companies plan to employ two treatment approaches: infusion of regulatory T cells (Tregs) conditionally expressing IL-2 utilizing Intrexon’s proprietary gene control approaches such as its RheoSwitch platform; and deployment of orally-delivered microbe-based ActoBiotics therapeutics expressing IL-2 to modulate immune function.

Under the terms of the agreement, Intrexon is to receive a technology access fee of $10 million in cash and reimbursement for all research and development costs. The agreement also provides for equal sharing of operating profits.

Corporate

In September, ZIOPHARM announced the appointment of Scott Tarriff to the Company’s Board of Directors. Mr. Tarriff, who serves as President, Chief Executive Officer and a Director of Eagle Pharmaceuticals, brings more than 25 years of pharmaceutical industry experience to ZIOPHARM.

Third-Quarter 2015 Financial Results

Net loss for the third quarter of 2015 was $18.2 million, or $(0.14) per share, compared to a net loss of $6.1 million, or $(0.06) per share, for the third quarter of 2014. Included in the loss for the third quarter of 2014 was non-cash income of $5.8 million, or $(0.06) per share for the change in fair value of warrants.

Research and development expenses were $17.0 million for the third quarter of 2015 compared to $9.7 million for the third quarter of 2014. The increase in research and development is due to a $10.0 million charge for in-process research and development with Intrexon for GvHD, and of $4.3 million related to CAR-T programs. These increases were offset by $5.7 million in savings related to discovery and nonclinical activities, $625 thousand in clinical study costs related to small molecule programs and $775 thousand savings in payroll, employee related and other expenses.

General and administrative expenses were $3.1 million for the third quarter of 2015 compared to $2.8 million for the third quarter of 2014. The increase of $0.3 million in general and administrative expenses is primarily attributable to non-cash equity compensation and other employee related expenses.

The Company ended the quarter with cash and cash equivalents of approximately $163.8 million. Given current development plans, the Company anticipates that current cash resources will be sufficient to fund our planned operations into the first quarter of 2018.

On November 2, 2015 BIND Therapeutics, Inc. (NASDAQ: BIND), a clinical-stage nanomedicine company developing targeted and programmable therapeutics called AccurinsTM, reported financial results and business highlights for the third quarter ended September 30, 2015 (Press release, BIND Therapeutics, NOV 2, 2015, View Source [SID:1234507868]). Management will host a conference call today at 8:30 a.m. ET to provide a business update and review the Company’s third quarter financial results.

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"This was an important quarter for BIND as we made significant progress with our proprietary pipeline, collaboration programs and research initiatives that have the potential to lead to breakthrough therapies," said Andrew Hirsch, president and chief executive officer at BIND Therapeutics. "Interim phase 2 data is forthcoming on our lead product BIND-014 in two studies across six tumor types with limited treatment options, starting with a readout by the end of 2015 in KRAS mutant and squamous histology non-small cell lung cancers. In addition, Pfizer exercised an option in our collaboration to move a kinase inhibitor towards clinical development and we expect our collaboration with AstraZeneca to result in a second Accurin to enter the clinic in the fourth quarter. We have also made tremendous progress in developing Accurins to target diseased immune cells, infectious disease, and to deliver oligonucleotides. Collectively, we are now positioned to potentially have at least four Accurins in clinical development within the next two years."

Recent Pipeline/Business Highlights:

Appointed Dr. Arthur Tzianabos, current president and chief scientific officer at OvaScience, to the Board of Directors.
Received notification from Pfizer that it would exercise its option to obtain an exclusive license to develop and commercialize an Accurin drug candidate for the treatment of solid tumors under the companies’ global collaboration agreement. BIND expects to receive the $2.5 million option fee in the fourth quarter of 2015.

Dosed the first patient in the iNSITE 2 trial in patients with four tumor histologies, each affecting fewer than 200,000 patients in the U.S.: cholangiocarcinoma, advanced cervical cancer, advanced bladder cancer, and advanced squamous cancer of the head and neck.

Completed build out of dedicated manufacturing space, through an arrangement with a large contract manufacturing organization, that is capable of producing Accurins at the double-digit kilogram scale.

Continued preclinical work in collaboration with Macrophage Therapeutics, completing derivatization of the Manocept targeting agent and producing multiple versions of Manocept-targeted Accurins for in vivo testing.
Continued preclinical work, and generated promising initial data, on Accurin versions of anti-infective and oligonucleotide-based therapies.
Anticipated upcoming milestones and activities include:

Dose the first patient in the phase 1 trial of AZD2811 with collaborator AstraZeneca. BIND expects to receive the $4.0 million milestone payment in the fourth quarter of 2015.

Present preclinical and clinical pharmacokinetic data from BIND’s clinical stage Accurin BIND-014, data from BIND’s preclinical stage Accurin BIND-510, and new data from a previously unannounced feasibility study with an Accurin formulation of Merck’s AKT inhibitor, MK-2206, at the upcoming AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper).
Report initial data from the first stage of the iNSITE 1 trial in KRAS mutant or squamous histology non-small cell lung cancer in the fourth quarter of 2015.

Report the results of ongoing proof-of-concept work with Macrophage Therapeutics before year-end.

Report overall survival data from the completed phase 2 non-small cell lung cancer study with BIND-014 in the broader patient population following occurrence of all survival events.

Report final results from the phase 2 prostate cancer study with BIND-014 following occurrence of 75 percent of the survival events.

Report initial stage 1 data readout from iNSITE 2 trial in the first half of 2016.
Initiate in vivo studies with Accurin containing an antibiotic that targets gram negative bacteria.
Initiate in vitro and in vivo studies aimed at characterizing the ability of Accurins to impact PK, biodistribution, cellular uptake and gene silencing activity of oligonucleotide drugs.

Third Quarter 2015 Financial Results
Revenue for the third quarter of 2015 was $2.1 million, compared to $3.4 million in the third quarter of 2014. Revenues are primarily related to collaborations with AstraZeneca, Pfizer and Roche. The decrease in 2015 third quarter revenue compared to 2014 third quarter revenue was primarily due to lower revenue recognized from the up-front license fee from the AstraZeneca collaboration. The Company uses a proportional performance model to recognize revenue related to the up-front license fees, milestones, option exercise fee, and research and development services. Revenue is recognized over estimated performance obligations over time – at times much later than when cash is received.

Research and development expenses totaled $9.7 million for the third quarter of 2015, compared to $7.1 million for the third quarter of 2014. The increase in research and development expenses was primarily driven by higher manufacturing and related expenses as we scaled up and completed the clinical manufacturing batch needed for Accurin AZD2811, as well as greater compensation expense to support the development of BIND’s internal pipeline and collaborations.

General and administrative expenses totaled $3.9 million for the third quarter of 2015, which was essentially flat when compared to the third quarter of 2014.

Net loss for the third quarter of 2015 was $10.2 million, compared to a net loss of $7.3 million for the third quarter of 2014.

Cash, cash equivalents and short-term investments were approximately $41 million as of September 30, 2015. Including the $2.5 million Pfizer option exercise fee and the anticipated R&D reimbursement for the next stage of the Pfizer collaboration, the Company expects that its cash, cash equivalents and short-term investments will fund operating expense and capital expenditure requirements into the fourth quarter of 2016. This expectation is based on BIND’s current operating plans and research and development funding that it expects to receive under its existing collaborations, but excludes any potential milestone payments under its collaboration agreements.

We expect to earn a $4.0 million milestone from AstraZeneca during the fourth quarter of 2015 for the first patient in a phase 1 trial for Accurin AZD2811. We only include milestones in our cash guidance once they are achieved.

On November 2, 2015 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported the presentation of data from the Company’s HEAT Study, highlighting the curative potential for ThermoDox plus optimized radiofrequency ablation (RFA) in intermediate primary liver cancer, also known as hepatocellular carcinoma (HCC), as well as preclinical data on the correlation of heating duration during RFA in combination with ThermoDox (Press release, Celsion, NOV 2, 2015, View Source [SID:1234507870]). The clinical data were presented by two leading liver cancer experts from South Korea and Taiwan, Professor Won Young Tak, MD, Ph.D., Division of Gastroenterology and Hepatology, Department of Internal Medicine, School of Medicine, Kyungpook National University, Daegu, Republic of Korea and Dr. Shi-Ming Lin, MD, co-chair ACTA 2015 and vice chairman, Department of Gastroenterology and Hepatology, Chang Gung Memorial Hospital, Taipei, Taiwan, on October 30-31, 2015 at the 2015 Asian Conference on Tumor Ablation (ACTA) in Fukuoka, Japan.

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"There is clear evidence that the duration of the RFA regimen is critical when treating patients with ThermoDox," said Professor Tak, lead investigator in South Korea for the Company’s HEAT and OPTIMA studies. "Findings from the data presented at ACTA, including the multivariate analysis, HEAT Study data demonstrating compelling survival outcomes and supportive preclinical data, underscore the importance of Celsion’s ongoing OPTIMA Study, which is designed to demonstrate the potential of ThermoDox with an optimized RFA regimen in this setting."

"The incidence rate of HCC within Asian countries is growing at an alarming rate, with current estimates projecting that Asia will account for approximately 75% of newly diagnosed cases annually," said Professor Lin, lead investigator in Taiwan for the Company’s HEAT and OPTIMA studies. "The totality of the data presented demonstrate that ThermoDox plus optimized RFA has a strong potential to serve as a curative therapy for patients with liver cancer, where there exists a strong unmet need for effective treatment options."

The three presentations included:

"Effect of Radiofrequency Ablation (RFA) Dwell Time (+/-) ThermoDox on Safety and Overall Survival (OS) Among 452 Intermediate Solitary HCC Patients With Lesions 3 to 7 cm: HEAT Study Data," by Professor Won Young Tak, MD, Ph.D., Division of Gastroenterology and Hepatology, Department of Internal Medicine, School of Medicine, Kyungpook National University, Daegu, Republic of Korea and lead investigator in South Korea for the Company’s HEAT and OPTIMA studies. Professor Tak discussed data from Celsion’s latest HEAT Study post-hoc analysis, which suggests an overall survival benefit of over two years in the large subgroup of patients treated with ThermoDox plus optimized RFA (RFA > 45 minutes).

"Effect of Standardizing Radiofrequency Ablation and Lyso-Thermosensitive Liposomal Doxorubicin (LTLD, ThermoDox) on Overall Survival (OS) Among Patients with a Solitary 3 to 7 cm HCC Lesion: A HEAT Study Multivariate Analysis," by Dr. Shi-Ming Lin, MD, co-chair ACTA 2015, vice-chairman, Department of Gastroenterology and Hepatology, Chang Gung Memorial Hospital, Taipei, Taiwan, and lead investigator in Taiwan for the Company’s HEAT and OPTIMA studies. Dr. Lin reviewed the extensive data from Celsion’s HEAT Study, including the results of multivariate analyses performed which clearly suggests that RFA heating or dwell time greater than 45 minutes was the only statistically significant variable that explained the significant improvement in overall survival (79 months in the optimized RFA plus ThermoDox subgroup versus 53.6 months in the optimized RFA only subgroup) in a large, well bounded subgroup of 285 patients (41% of the HEAT Study patients).

"Importance of Heating Time on the Local Drug Deposition During RFA in Combination with Lyso-Thermosensitive Liposomal Doxorubicin (LTLD) in a Porcine Model," by Nicholas Borys, MD, Celsion’s senior vice president and chief medical officer. Dr. Borys reviewed findings from a preclinical study demonstrating that in a porcine model, a direct correlation was observed between the duration of RFA heating, or dwell time, and the concentration of doxorubicin localized to the liver.
The presentation abstracts will be available on Celsion’s website at View Source

About Celsion’s Phase III OPTIMA Study

Celsion’s Phase III OPTIMA Study is a global pivotal, double-blind, placebo-controlled study evaluating ThermoDox, its proprietary heat-activated liposomal encapsulation of doxorubicin, in combination with optimized radiofrequency ablation (RFA) in HCC. The study is expected to enroll up to 550 patients in over 75 clinical sites in the North America, Europe, China and Asia Pacific, and will evaluate ThermoDox in combination with optimized RFA, which will be standardized to a minimum of 45 minutes across all investigators and clinical sites for treating lesions three to seven centimeters, versus standardized RFA alone. The primary endpoint for the trial is overall survival, which is supported by post-hoc analysis of data from the Company’s 701 patient HEAT Study, where optimized RFA has demonstrated the potential to significantly improve survival when combined with ThermoDox. The statistical plan for the OPTIMA Study calls for two interim efficacy analyses by an independent Data Monitoring Committee (iDMC).

(Filing, 10-Q, Amgen, NOV 2, 2015, View Source [SID:1234507867])

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On November 2, 2015 Curis, Inc. (NASDAQ:CRIS), a biotechnology company focused on the development and commercialization of innovative drug candidates for the treatment of human cancers, reported that its collaborator, Aurigene Discovery Technologies Limited, will present data from two programs at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) to be held from November 5 – 9, 2015, in Boston, MA (Press release, Curis, NOV 2, 2015, View Source [SID:1234507871]).

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Curis’ collaborator, Aurigene will present data from CA-170 (previously AUPM-170), a first-in-class oral, small molecule immune checkpoint antagonist targeting programmed death ligand-1 (PD-L1) and V-domain Ig suppressor of T cell activation (VISTA) as well as from the interleukin-1 receptor association kinase4 (IRAK4) inhibitor program. Curis recently exercised options to license both these programs under a collaboration agreement with Aurigene established earlier this year.

Additional information on the presentations can be found below and abstracts can be accessed at www.aacr.org.

Poster Presentations

Date/Time: Friday, Nov. 6, 2015, 12:15 PM – 3:15 PM
Session Title: Immune Modulators
Presentation Title: First-in-class orally available immune checkpoint antagonists for cancer therapy
Location: Session A, Hall C-D
Abstract Number: A96

Date/Time: Sunday, Nov. 8, 2015, 12:30 PM – 3:30 PM
Session Title: Therapeutic Agents: Small Molecule Kinase Inhibitors
Presentation Title: Efficacy of novel IRAK4 inhibitors in ABC-DLBCL and AML models
Location: Session C, Hall C-D
Abstract Number: C191