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]).

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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.

On November 9, 2015 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the third quarter of 2015 and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, NOV 9, 2015, View Source;p=RssLanding&cat=news&id=2110660 [SID:1234508146]).

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"During the third quarter we continued to make good progress in the advancement of our stem cell transplant, CAR-T and TCR programs," said Tom Farrell, President and Chief Executive Officer of Bellicum. "We are particularly excited about the interim data that will be presented at ASH (Free ASH Whitepaper) 2015 that underscore the potential of our lead product candidate BPX-501 to improve outcomes for patients with blood cancers and inherited blood diseases undergoing haplo transplantation. BPX-501 could be used in the treatment of over 60 rare diseases, enabling a potential curative transplant for many of those disorders, including sickle cell disease, where treatment-related mortality has severely limited the adoption of allogeneic transplantation."

Added Mr. Farrell, "We are also looking forward to multiple presentations at ASH (Free ASH Whitepaper) highlighting our CAR-T and TCR adoptive cell therapy programs and the power of our cellular control technologies. We continue to anticipate that three new product candidates, BPX-601, BPX-701, and BPX-401 will be initiating clinical development in the first half of 2016."

Program Updates:

BPX-501 at ASH (Free ASH Whitepaper) 2015: Company to present interim data from the BP-004 ongoing Phase 1/2 clinical trial. Bellicum will present initial data in malignant and non-malignant blood diseases at the upcoming 26th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in early December in Orlando, Florida. The open label dose escalation trial in pediatric patients is evaluating whether BPX-501 T cells from a haploidentical donor, administered following a T-depleted hematopoietic stem cell transplant (HSCT), are safe and can enhance immune reconstitution.

Enrollment in the BP-004 trial continues at a strong pace at sites in Europe and in the U.S., with 53 pediatric patients enrolled as of October 31st, including one patient with sickle cell disease.

Among non-malignant patients in the trial treated to date are four children with beta thalassemia major (in its most severe form, the β0/β0 type) who have successfully undergone the HSCT transplant procedure with the add-back of BPX-501 gene-modified T cells. Within two weeks of the transplant procedure all patients became blood transfusion-independent. All four patients are alive, disease-free and remain transfusion-independent.

At a medical symposium in Parma, Italy in early September, principal investigator Dr. Franco Locatelli shared initial outcomes from this ongoing trial. His presentation included the first 15 children enrolled in the clinical study with non-malignant inherited disorders (four with severe combined immunodeficiency, or SCID; three with Wiskott-Aldrich Syndrome; four with Fanconi anemia; three with beta thalassemia, and one with hemophagocytic lymphohistiocytosis) who received the BPX-501 T cell add-back following HLA-partially matched family donor HSCT. In all cases, the BPX-501 T cells engrafted and expanded with no secondary graft failures. Grade II skin-only graft versus host disease (GvHD) was observed in one patient, and promptly resolved with topical steroids. None of the patients so far have developed chronic GvHD, and all are alive and disease-free.

BPX-601 preclinical data to be presented at ASH (Free ASH Whitepaper) 2015. Bellicum continues to advance its first GoCAR-T product candidate, containing our proprietary iMC (inducible MyD88/CD40) activation switch, designed to treat solid tumors expressing prostate stem cell antigen (PSCA). The Company expects to file an IND for the initial indication of pancreatic cancer by the end of 2015. In addition to pancreatic cancer, PSCA is also expressed in prostate, ovarian, bladder, esophageal and gastric cancers. BPX-601 is differentiated from traditional CAR-T therapies with an MC co-stimulatory domain that is activated by administration of rimiducid.
BPX-401 CIDeCAR preclinical data to be highlighted in an oral presentation at ASH (Free ASH Whitepaper) 2015. BPX-401, a CIDeCAR product candidate incorporating Bellicum’s proprietary MC co-stimulatory domain and the CaspaCIDe safety switch, is designed to target blood cancers expressing CD19. BPX-401 is expected to enter the clinic in the first half of 2016.

BPX-701 progressing toward the clinic. Bellicum continues to advance its proprietary T cell receptor (TCR) product candidate designed to target solid tumors expressing the preferentially-expressed antigen in melanoma, or PRAME. The Company has identified clinical sites for its BPX-701 CaspaCIDe-enabled TCR product candidate and expects to file an IND by the end of 2015, initially for the indications of PRAME-expressing sarcomas and uveal melanoma.

Third Quarter and Nine Months Ended September 30, 2015 Financial Results:

Bellicum reported a net loss of $13.4 million for the third quarter of 2015 and $31.7 million for the nine months ended September 30, 2015, compared to a net loss of $4.1 million and $9.7 million for the comparable periods in 2014. The results included non-cash, share-based compensation charges of $2.3 million and $5.9 million for the third quarter and nine months ended September 30, 2015, respectively, and $0.1 million and $0.2 million for the comparable periods in 2014. As of September 30, 2015, cash and investments totaled $163.2 million.

Grant revenues were $0.1 million and $0.2 million for the three and nine months ended September 30, 2015, respectively, and $0.7 million and $1.8 million during the comparable periods in 2014. The decrease in grant revenues was primarily due to the June 2014 expiration of the Company’s grant award from the Cancer Prevention and Research Institute of Texas.

Research and development expenses were $9.8 million and $23.5 million, respectively, for the three and nine months ended September 30, 2015, compared to $2.3 million and $7.9 million during the comparable periods in 2014. The higher expenses in the 2015 periods were primarily due to an increase in manufacturing and clinical expenses as a result of increased patient enrollment in our BPX-501 clinical trials, increased expenses for the IND-enabling activities on the Company’s CAR-T and TCR product candidates and increased personnel and infrastructure costs.

General and administrative expenses were $3.9 million and $8.9 million, respectively, for the three and nine months ended September 30, 2015, compared to $1.3 million and $2.3 million during the comparable periods in 2014. The increased G&A expenses in 2015 were due to the growth of the organization and the costs associated with operating as a public company.

(Filing, 10-Q, Bio-Path Holdings, NOV 9, 2015, View Source [SID:1234508148])

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(Filing, 10-Q, Progenics Pharmaceuticals, NOV 9, 2015, View Source [SID:1234508174])

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On November 09, 2015 ArQule, Inc. (Nasdaq:ARQL) reported the results of preclinical and clinical studies focusing on tivantinib, ARQ 087, ARQ 092, and ARQ 751 (Press release, ArQule, NOV 9, 2015, View Source [SID:1234508101]). The data were presented at the AACR (Free AACR Whitepaper)-NCI-EORTC meeting on November 7th, 2015. The poster presentations can be accessed in the "Investor and Media" section of our website, www.arqule.com, under "Recent Data Presentations."

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For the first time, data from an exploratory sub-analysis of the MARQUEE trial with tivantinib in non-small cell lung cancer in patients with advanced disease and epidermal growth factor receptor (EGFR) mutations were presented. The data showed tivantinib, when added to erlotinib, increased progression-free survival to 13 months compared to 7.5 months in the erlotinib plus placebo arm. The sub-analysis included 109 patients of which 56 were in the combination tivantinib plus erlotinib arm of the trial. The data were highlighted in an AACR (Free AACR Whitepaper) press release and press conference.

"The data from the sub-analysis of the MARQUEE trial supports our focus on precision medicine," said Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "Although the study did not meet its primary endpoint, this analysis is encouraging and offers evidence that tivantinib when dosed in a specific patient population can provide substantial benefit."

Additionally, the company presented pre-clinical and clinical data on its early stage proprietary pipeline that support ArQule’s efforts to address the needs of patients in therapeutic areas of high unmet need through precision medicine. Data presented on FGFR inhibitor, ARQ 087, demonstrate that FGFR2 dysregulation correlates with efficacy and supports the on-going phase 2 trial in intrahepatic cholangiocarcinoma. Similarly, data presented on AKT inhibitors, ARQ 092 and ARQ 751, demonstrate that both drugs inhibit AKT and provide strong rationale for further studies in patients harboring AKT1 and PI3K mutations.

"Data presented at this year’s AACR (Free AACR Whitepaper)-NCI-EORTC conference are part of ArQule’s translational effort to connect preclinical and clinical research and guide a true precision medicine endeavor," said Giovanni Abbadessa, Vice President of Clinical Development, Translational Medicine and Medical Affairs at ArQule. "Both ARQ 092 and ARQ 087 have shown single-agent activity in vitro, in vivo and in patients in cancers driven by genetic alterations of their respective targets, AKT and FGFR. In addition, these pre-clinical data find confirmation in the clinical results achieved by ARQ 087 and ARQ 092 in their respective phase 1 clinical trials in genetically-altered endometrial and breast cancer and in FGFR2-driven cholangiocarcinoma, respectively. Combinability data reported for both experimental drugs with standard therapies may allow even more development options for the future."

Precision Medicine

Tivantinib is enrolling in two biomarker-driven phase 3 trials, METIV-HCC and JET-HCC. ARQ 087 is enrolling in a biomarker-driven phase 2 trial in intrahepatic cholangiocarcinoma (iCCA) with FGFR translocations. ARQ 092 is enrolling in a phase 1b biomarker-driven trial in patients with AKT and PI3K activating mutations including patients with breast, endometrial and ovarian cancers.

About MET and tivantinib (ARQ 197)

Tivantinib is an orally administered, selective inhibitor of MET, a receptor tyrosine kinase, which is currently in Phase 3 clinical trials. In healthy adult cells, MET can be present in normal levels to support natural cellular function, but in cancer cells, MET can be inappropriately and continuously activated. When abnormally activated, MET plays multiple roles in aspects of human cancer, including cancer cell growth, survival, angiogenesis, invasion and metastasis. The activation of certain cell signaling pathways, including MET, has also been associated with the development of resistance to anti-EGFR (epidermal growth factor receptor) antibodies such as cetuximab and panitumumab.

Pre-clinical data have demonstrated that tivantinib inhibits MET activation in a range of human tumor cell lines and shows anti-tumor activity against several human tumor xenografts. In clinical trials to date, treatment with tivantinib has been generally well tolerated and has shown clinical activity in a number of tumors. Tivantinib has not yet been approved for any indication in any country.

In December 2008, ArQule and Daiichi Sankyo signed a license, co-development and co-commercialization agreement for tivantinib in the U.S., Europe, South America and the rest of the world, excluding Japan, China (including Hong Kong), South Korea and Taiwan.

About the AKT Pathway, ARQ 092 and ARQ 751

ARQ 092 and ARQ 751 are orally available, selective small molecule inhibitors of the AKT kinase. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications.

ARQ 092, the lead compound in ArQule’s AKT program, has completed Phase 1a clinical testing and has advanced into Phase 1b expansion testing in cohorts of patients with endometrial cancer, lymphoma and tumors harboring either AKT or PI3K mutations. A number of next-generation compounds in the Company’s AKT program are in early to late stages of pre-clinical development. The company plans to file an Investigational New Drug (IND) application by the end of 2015 for ARQ 751, a next generation AKT inhibitor.

About FGFR and ARQ 087

ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor ("FGFR") family with demonstrated efficacy in FGFR2 amplified tumors. The FGFR pathway is disrupted in several ways in human cancer, thus providing numerous therapeutic targets for an inhibitor of this pathway. ARQ 087 has demonstrated inhibition of tumor growth and downstream signaling in vivo in tumors whose growth is driven by these targets.

Signals of single agent activity with this compound were observed in Phase 1a testing. Phase 1b expansion cohorts with ARQ 087 include patients with cholangiocarcinoma and adrenocortical tumors, as well as those with FGFR translocations, amplification and mutations. Clinical development of ARQ 087 has advanced into Phase 2 for intrahepatic cholangiocarcinoma ("iCCA") following the observation of two confirmed partial responses in this patient population in the Phase 1 portion of the program.