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8-K – Current report

On November 9, 2015 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the third quarter ended September 30, 2015 and recent company developments (Filing, 8-K, TG Therapeutics, NOV 9, 2015, View Source [SID:1234508125]).

Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "During the third quarter, we achieved another major milestone for the Company in obtaining a Special Protocol Assessment for our UNITY-CLL trial, a study evaluating the safety and efficacy of our proprietary ‘1303’ combination regimen in patients with front-line as well as previously treated CLL. This is a very important and exciting clinical trial for the Company, as it represents our first pivotal trial for our proprietary combination and, if successful, should provide a broad approval in CLL offering patients in both first-line and relapsed/refractory setting, a novel, non-chemotherapy treatment option. Further, it would provide us a broad label for building additional three and, possibly, four drug proprietary combinations to further improve outcomes for patients with CLL. With Phase 3 programs in oncology now underway for both TG-1101 and TGR-1202, we’re excited to begin exploring the potential of our pipeline products for the treatment of autoimmune disease, an area where B-cell targeted therapies have proven highly effective, and anticipate commencing our first trial in Multiple Sclerosis in the near-term." Mr. Weiss continued, "We also remain focused on aggressively enrolling into our ongoing GENUINE Phase 3 clinical trial, and expect top-line data from this study in the second half of 2016. Finally, from a financial perspective, with more than $115 million in cash and investments we have enough cash to execute on our business plan."

Recent Developments and Highlights

· In September 2015, we announced a Special Protocol Assessment (SPA) agreement with the FDA for the first Phase 3 clinical trial of our proprietary combination regimen of TG-1101 (ublituximab) with TGR-1202 ("1303") for patients with chronic lymphocytic leukemia, the UNITY-CLL study.

· In September 2015, we announced the initiation of a Phase 1/2 clinical trial investigating the use of TG-1101 and TGR-1202 in combination with pembrolizumab, the anti-PD-1 immune checkpoint inhibitor, in patients with relapsed or refractory CLL, the first clinical trial evaluating the safety, tolerability and effectiveness of the triple combination of a PI3K delta inhibitor with an anti-CD20 mAb and an anti-PD-1 checkpoint inhibitor.

Goals/Objectives for the Remainder of 2015

· Continue to aggressively recruit into the GENUINE Phase 3 Clinical Trial of TG-1101 in combination with ibrutinib

· Enroll the first patient by year end in our UNITY-CLL Phase 3 clinical trial of TG-1101 plus TGR-1202 in front-line and relapsed/refractory CLL

· Announce our next registration trial evaluating 1303 in patients with NHL

· Continue to recruit into the triple combination cohort of 1303 plus ibrutinib as well as the triple combination study of 1303 plus pembrolizumab, as well as seek to evaluate additional novel triple combinations of interest

· Expand into autoimmune diseases with the first Phase 2 trial in Multiple Sclerosis to commence in the near-term

· Continue to advance our pre-clinical compounds, including IRAK4, anti PD-L1 and anti-GITR forward towards clinical development

· Continue to seek additional compounds to further complement our current portfolio

Financial Results for the Third Quarter 2015

At September 30, 2015 the Company had cash, cash equivalents, investment securities, and interest receivable of $115.4 million, which includes approximately $67.0 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the year (all of which was previously disclosed in connection with our last quarterly update), as compared to December 31, 2014 when we had $78.9 million.

Our consolidated net loss for the third quarter ended September 30, 2015, excluding non-cash items, was approximately $12.4 million, which included approximately $6.9 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The consolidated net loss for the third quarter ended September 30, 2015, inclusive of non-cash items, was $13.7 million, or $0.28 per diluted share, compared to a consolidated net loss of $17.5 during the comparable quarter in 2014, representing a decrease in consolidated net loss of $3.8 million. The decrease in consolidated net loss during the third quarter ended September 30, 2015 was primarily the result of $8.1 million of expense ($4.1 million of which was non-cash stock expense) recorded during the quarter ended September 30, 2014 in conjunction with the Company’s licensing agreement for TGR-1202, and a $2.9 million decrease in non-cash compensation expense related to equity incentive grants over the comparable period in 2014. Partially offsetting the aforementioned decreases, other research and development expenses for TG-1101 and TGR-1202 increased $4.8 million and $2.1 million, respectively, over the comparable period in 2014.

Our consolidated net loss for the nine months ended September 30, 2015, excluding non-cash items, was approximately $32.5 million, which included approximately $16.0 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The consolidated net loss for the nine months ended September 30, 2015, inclusive of non-cash items, was $45.3 million, or $1.01 per diluted share, compared to a consolidated net loss of $37.0 million during the comparable period in 2014, representing an increase in consolidated net loss of $8.3 million. The increase in consolidated net loss during the nine months ended September 30, 2015 was primarily the result of other research and development expenses for TG-1101 and TGR-1202 increasing approximately $14.6 million and $4.5 million, respectively, over the comparable period in 2014. This was offset by $9.3 million of expense ($5.3 million of which was non-cash stock expense) recorded in conjunction with the Company’s licensing agreements for TGR-1202 and the IRAK4 inhibitors program during the nine months ended September 30, 2014, and a decrease of $3.2 million in non-cash compensation expense related to equity incentive grants over the comparable period in 2014.

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Inovio Pharmaceuticals Reports 2015 Third Quarter Results

On November 09, 2015 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported financial results for the quarter ended September 30, 2015 (Press release, Inovio, NOV 9, 2015, View Source [SID:1234508123]).

Total revenue was $24.2 million and $34.6 million for the three and nine months ended September 30, 2015, compared to $1.8 million and $8.0 million for the same periods in 2014.

Total operating expenses were $20.5 million and $54.4 million for the three and nine months ended September 30, 2015, compared to $10.2 million and $36.5 million for the same periods in 2014.

The net income (loss) attributable to common stockholders for the three and nine months ended September 30, 2015, was $5.6 million, or $0.08 per share, and $(11.2 million), or $(0.17) per share, compared to $(7.2 million), or $(0.12) per share, and $(28.7 million), or $(0.49) per share, for the same periods in 2014.

Revenue

The increase in revenue for the comparable periods was primarily due to development payments from our DARPA Ebola grant as well as $15.0 million of revenue recognized in the third quarter 2015 from the up-front payment received from our partnership agreement with MedImmune. Accounting recognition of the remainder of the $27.5 million upfront payment has been deferred and will be triggered by future events. The net income achieved during the third quarter was attributable to the increase in revenue and may not be reflective of future quarters.

Operating Expenses

Research and development expenses for the three and nine months ended September 30, 2015, were $16.1 million and $42.2 million, compared to $7.0 million and $24.9 million for the same periods in 2014. The increase for the three and nine month periods was primarily related to increased investment in our product development programs. General and administrative expenses for the three and nine months ended September 30, 2015, were $4.4 million and $13.2 million versus $3.2 million and $11.6 million for the same periods in 2014.

Capital Resources

As of September 30, 2015, cash and short-term investments were $170.8 million compared with $93.6 million as of December 31, 2014. At quarter end the company had 72.2 million shares outstanding and 78.9 million fully diluted.

Inovio’s balance sheet and statement of operations are provided below. Form 10-Q providing the complete 2015 third quarter financial report can be found at: View Source

Corporate Update

Corporate Development

On August 7, 2015, Inovio entered into a strategic cancer vaccine collaboration and license agreement with MedImmune, the global biologics research and development arm of AstraZeneca. MedImmune acquired exclusive rights to Inovio’s INO-3112 immunotherapy, which targets cancers caused by human papillomavirus (HPV) types 16 and 18. MedImmune intends to study INO-3112 in combination with selected immunotherapy molecules within its pipeline in HPV-driven cancers. Emerging evidence suggests that the benefits from immuno-oncology molecules, such as those in MedImmune’s portfolio, can be enhanced when they are used in combination with cancer vaccines that generate tumor-specific T-cells.

MedImmune paid Inovio $27.5 million in the third quarter and will make potential future payments totaling up to $700 million upon reaching development and commercial milestones. MedImmune will fund all development costs. Inovio is entitled to receive up to double-digit tiered royalties on INO-3112 product sales.

Inovio and MedImmune will also develop two additional DNA-based cancer vaccine products not included in Inovio’s current product pipeline, which MedImmune will have the exclusive rights to develop and commercialize. Inovio will be eligible to receive development, regulatory and commercialization milestone payments and royalties on net sales for these cancer vaccines.

This is the second major pharmaceutical partnership for Inovio’s DNA-based immunotherapy technology, adding to its existing license agreement with Roche for the INO-1800 hepatitis B immunotherapy.

Inovio initiated a partnership with the European Organization for Research and Treatment of Cancer to evaluate INO-3112 in combination with traditional chemo-radiotherapy for the treatment of patients with locally advanced stage cervical cancer. The primary endpoint of this phase II study is to evaluate progression free survival at 18 months. It is expected to begin in 2016 and will be part of MedImmune’s development plans.

Inovio and collaborators are advancing multiple treatment and prevention approaches against Ebola. Inovio received an initial $20 million award from the Defense Advanced Research Projects Agency (DARPA). In September, DARPA awarded Inovio an additional $25 million for the successful completion of pre-clinical and clinical development milestones. This funding supports the development of a DNA-based vaccine, a therapeutic DNA-based monoclonal antibody treatment (dMAb), and a conventional monoclonal antibody treatment. Inovio has completed enrollment of 75 healthy subjects in a phase I study of the Ebola DNA vaccine.

Clinical Development

Inovio’s manuscript detailing the broad study findings of its phase II study of VGX-3100 in patients with high-grade cervical dysplasia (CIN 2/3) was published in The Lancet, a top peer-reviewed medical journal. This publication describes that VGX-3100, a first-in-class product for treating high grade cervical neoplasia associated with HPV, is the first therapy to demonstrate that activated killer T cells induced in the body have the power to clear neoplastic lesions as well as the virus which caused the disease. These findings provide proof of principle not only for this disease indication but for the broad utility of Inovio’s technology across cancers and infectious diseases.

Results of the trial were reported in the article entitled, "Safety, efficacy, and immunogenicity of VGX-3100, a therapeutic synthetic DNA vaccine targeting human papillomavirus 16 and 18 E6 and E7 proteins for cervical intraepithelial neoplasia 2/3: a randomized, double-blind, placebo-controlled phase 2b trial."

Inovio continues to make preparations to launch a phase III registration study of VGX-3100 in 2016. Necessary steps include scaling to commercial-level production of its immunotherapy product and delivery devices. The company expects its end-of-phase-II meeting with the FDA, which will review Inovio’s phase II data and proposed phase III clinical trial design, to take place in early 2016.

Inovio launched a phase I study of INO-5150, its SynCon immunotherapy targeting prostate-specific membrane antigen and prostate-specific antigen, in men with biochemically relapsed prostate cancer. This study is evaluating the safety, tolerability, and immunogenicity of INO-5150 alone or in combination with Inovio’s DNA-based IL-12 immune activator. The company expects to report interim data from this study in 2016.

The first patient was dosed in Inovio’s phase I trial to evaluate safety and tolerability of PENNVAX-GP, the company’s "universal" DNA vaccine for HIV. The trial will measure immune responses following administration of the vaccine in four groups of healthy subjects receiving the vaccine with and without an immune activator (DNA IL-12) and delivered into muscle or skin using Inovio’s CELLECTRA delivery technology. This 94-patient study is being conducted by the HIV Vaccines Trial Network (HVTN) and funded by the National Institute of Allergy and Infectious Diseases (NIAID).

Inovio’s partner for its DNA vaccine for Middle East Respiratory Syndrome (MERS), GeneOne Life Science Inc., filed an Investigational New Drug Application (IND) for GLS-5300 with the United States Food and Drug Administration in October and intends to launch a clinical trial in healthy volunteers by the year end.

8-K – Current report

On November 05, 2015 Caladrius Biosciences, Inc. (NASDAQ:CLBS) ("Caladrius"), a company combining a leading cell therapy service provider with a development pipeline including a Phase 3 clinical program in immuno-oncology and a portfolio of projects in immune modulation and ischemic repair, reported financial results for the three months ended September 30, 2015 (Filing, 8-K, Caladrius Biosciences, NOV 9, 2015, View Source [SID:1234508122]).

Business Highlights

• Total revenues for the third quarter of 2015 increased 43% to $5.9 million when compared with $4.1 million in the year-ago third quarter, driven by increases in Clinical Service revenue at the Company’s PCT subsidiary, an external development and manufacturing partner for the growing cell therapy industry

• PCT entered into a long-term dedicated capacity agreement with IRX Therapeutics, Inc. to support their anticipated clinical trial manufacturing needs including the manufacture of EU-compliant cell therapy product, adding to a growing list of immuno-oncology clients with similar arrangements including Kite Pharma and ImmunoCellular Therapeutics

• Continued patient enrollment in a Phase 3 clinical study of lead product candidate CLBS20 for the treatment of recurrent Stage III or Stage IV metastatic melanoma

• Presented data elucidating the mechanism of action of CLBS20, suggesting correlations between a distinct immune response triggered by CLBS20 and overall survival of melanoma patients

• Received an initial award of $300,000 out of a potential award of up to $2.3 million from the National Cancer Institute to fund the first phase of a project for process optimization of CLBS20

• Entered into a collaboration agreement with Sanford Research to develop CLBS03, the Company’s T regulatory cell therapy product candidate, for the treatment of adolescents with recent-onset type 1 diabetes, which the Company expects to advance to a Phase 2 clinical study in early 2016

• Promoted Joseph Talamo to Chief Financial Officer and Todd Girolamo to General Counsel.

Management Commentary

"PCT continues to post strong revenue growth, which we expect to continue as the immuno-oncology sector is experiencing industry-wide advances and there are increasing numbers of such products on the market and in development. We expect PCT to continue to capitalize on its unmatched cell development and manufacturing expertise as industry demand for cell therapy services continues to grow," said David J. Mazzo, Ph.D., Chief Executive Officer of Caladrius. "In addition, we continued to advance our own clinical development programs in immuno-oncology (CLBS20) and immune modulation (CLBS03), while maintaining financial discipline that allowed us to lower our cash burn."

2015 Third Quarter Financial Highlights
Total revenue for the third quarter of 2015 increased 43% to $5.9 million compared with $4.1 million for third quarter of 2014, primarily due to higher reported Clinical Services revenues at PCT.

Research and development expenses in the third quarter of 2015 decreased 24% to $6.3 million compared with $8.5 million for the third quarter of 2014. The decrease was primarily related to lower costs associated with the Company’s ischemic repair and immune modulation programs. The lower costs were partially offset by increased expenses associated with the Intus Phase 3 clinical trial that initiated in 2015.

Selling, general and administrative expenses were approximately $5.1 million for the third quarter of 2015 compared with $7.9 million for same period in 2014. The decrease was primarily due to lower equity-based compensation expenses in the current quarter compared with a year ago and, to a lesser extent, lower expenses associated with other general and administrative activities.

The net loss for the third quarter of 2015 was approximately $11.4 million or $0.21 per share, compared with a net loss of $17.2 million or $0.48 per share for same period in 2014.

As of September 30, 2015, Caladrius had cash, cash equivalents and marketable securities of $29.4 million.

Verastem Reports Third Quarter 2015 Financial and Corporate Results

On November 9, 2015 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, reported financial results for the third quarter ended September 30, 2015, and also provided an overview of certain corporate developments (Press release, Verastem, NOV 9, 2015, View Source;p=RssLanding&cat=news&id=2110303 [SID:1234508120]).

"Our mission is to improve survival and the quality of life for patients battling cancer through the combination of our cancer stem cell-targeting agents with current and emerging standard of care treatments," said Robert Forrester, President and Chief Executive Officer of Verastem. "We are supported by a strong balance sheet, three clinical compounds and a team of talented and dedicated professionals. We continue to believe in our pipeline candidates and are focused on executing their clinical development."
Mr. Forrester continued: "We are working with thought-leading researchers on innovative anti-cancer applications for our compounds. For example, researchers from the University of Edinburgh recently published a ground-breaking paper in "Cell" which highlights the potential of focal adhesion kinase (FAK) inhibition to enable the body’s immune system to better fight cancer. While this is early stage data, it provides support for the thesis that FAK inhibitors may be useful in combination with immuno-oncology agents with the goal of yielding more durable responses for a greater number of cancer patients."

Recent Developments
Stopped Enrollment in the COMMAND Study Evaluating VS-6063 in Mesothelioma Due to Futility – In September 2015, Verastem announced its decision to stop enrollment in the Phase 2 registration-directed, double-blind, placebo-controlled study (COMMAND) of VS-6063 for patients with mesothelioma. The decision to stop enrollment followed a Data Safety Monitoring Board (DSMB) review of a pre-planned interim analysis. The results of the analysis demonstrated that VS-6063 had a generally well tolerated safety profile but that there was not a sufficient level of efficacy to warrant continuation of the study.

New Research Published in the Journal "Cell" Highlighting the Potential of FAK Inhibition to Enhance the Efficacy of Anti-Tumor Immunotherapy – In September 2015, Verastem announced that researchers from the University of Edinburgh published a study in the journal "Cell" which discusses results from preclinical research showing that focal adhesion kinase (FAK), a protein which is often overproduced in tumors, enables cancer cells to evade attack by the body’s immune system. In this study, researchers discovered that FAK inhibition can favorably modulate the balance of immune cells in the tumor, inducing immune-mediated tumor elimination in preclinical models.

Presented Data on VS-6063, VS-4718 and VS-5584 at Key Oncology-Focused Medical Meetings – In November 2015, Verastem presented a total of six posters at the 2015 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) and the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 30th Anniversary Annual Meeting. The presented data described the results of preclinical research showing that FAK inhibitors may enhance the effects of anti-cancer immunotherapy for both typically responsive and non-responsive tumors.

During the third quarter, Verastem also gave several oral and poster presentations at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper)/18th European Cancer Congress (ECC) and at the 16th World Conference on Lung Cancer (WCLC) describing clinical and preclinical data relating to its pipeline product candidates.

All of the posters and presentations can be found on the Presentations page of Verastem’s website.

Third Quarter 2015 Financial Results
As of September 30, 2015, Verastem had cash, cash equivalents and investments of $120.5 million compared to $92.7 million as of December 31, 2014. Verastem used $11.6 million for operating activities in the third quarter ended September 30, 2015 (the "2015 Quarter").

Net loss for the 2015 Quarter was $15.4 million, or $0.42 per share, as compared to a net loss of $13.3 million, or $0.52 per share, for the same period in 2014 (the "2014 Quarter"). Net loss includes stock-based compensation expense of $2.1 million and $2.8 million for the 2015 Quarter and 2014 Quarter, respectively.

Research and development expense for the 2015 Quarter was $11.3 million compared to $9.0 million for the 2014 Quarter. The $2.3 million increase from the 2014 Quarter to the 2015 Quarter was primarily related to an increase of $2.3 million in contract research organization expense for outsourced biology, chemistry, development and clinical services, which includes our clinical trial costs, an increase in consulting fees of approximately $299,000, an increase in personnel related costs of approximately $115,000 due to increased headcount and salaries, and an increase of approximately $102,000 in lab supplies due to increased research activity. These increases were partially offset by a decrease of approximately $537,000 in stock-based compensation.

General and administrative expense. General and administrative expense for the 2015 Quarter was $4.2 million compared to $4.3 million for the 2014 Quarter. The decrease of approximately $100,000 from the 2014 Quarter to the 2015 Quarter primarily resulted from a decrease in professional fees of approximately $249,000, primarily related to lower IP and general legal costs, and a decrease in stock-based compensation expense of approximately $245,000. These decreases were partially offset by increases in personnel related costs of approximately $247,000, primarily due to an increase in headcount and salaries, and in consulting fees of approximately $164,000.

The number of outstanding common shares as of September 30, 2015, was 36,934,804.

Financial Guidance
We expect our existing cash, cash equivalents and investments will enable us to fund our current operating plan and capital expenditure requirements at least through the first half of 2017.

About VS-6063
VS-6063 (defactinib) is an orally available compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). Cancer stem cells are an underlying cause of tumor resistance to chemotherapy, recurrence and ultimate disease progression. Research has demonstrated that FAK activity is critical for the growth and survival of cancer stem cells. VS-6063 is currently being studied in the "Window of Opportunity" study in patients with mesothelioma prior to surgery, a Phase 1/1b study in combination with paclitaxel in patients with ovarian cancer, a trial in patients with KRAS-mutated non-small cell lung cancer and a trial evaluating the combination of VS-6063 and VS-5584 in patients with relapsed mesothelioma.

About VS-4718
VS-4718 is an orally available compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). VS-4718 is currently being studied in a Phase 1 dose escalation study in patients with advanced cancers.

About VS-5584
VS-5584 is an orally available compound that has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in chemotherapy-resistant models. Verastem is currently conducting a dose escalation trial of VS-5584 in patients with advanced solid tumors as a single agent and a combination trial of VS-5584 and VS-6063 in patients with relapsed mesothelioma. VS-5584 has been granted orphan drug designation for use in mesothelioma in the U.S. and EU.

NewLink Genetics Corporation Announces Promising Clinical Data on Indoximod at the Society for Neuro-Oncology Annual Meeting

On November 09, 2015 NewLink Genetics Corporation (NASDAQ:NLNK), a biopharmaceutical company at the forefront of discovering, developing and commercializing novel immuno-oncology product candidates, including both cellular immunotherapy and checkpoint inhibitor platforms, to improve the lives of patients with cancer, reported that a study update from a Phase 1b/2 study of indoximod in combination with temozolomide in temozolomide-refractory glioblastoma patients will be presented at the 20th Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology on November 19-22, 2015 at the San Antonio Marriott Rivercenter (Press release, NewLink Genetics, NOV 9, 2015, View Source [SID:1234508119]).

Details for the presentation follow:

Abstract Title: Updates on phase 1b/2 combination study of the IDO pathway inhibitor indoximod with temozolomide for adult patients with temozolomide-refractory primary malignant brain tumors
Abstract Number: IMCT-21
Time: 7:30-9:30 p.m.
Date: Friday, November 20
Location: Ballroom B
Session Type: Poster