On November 09, 2015 Portola Pharmaceuticals (Nasdaq:PTLA) reported a corporate update and reported its financial results for the third quarter ended September 30, 2015 (Press release, Portola Pharmaceuticals, NOV 9, 2015, View Source;p=RssLanding&cat=news&id=2110682 [SID:1234508172]).

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"We achieved a number of significant clinical, regulatory and manufacturing milestones over the past quarter," said William Lis, chief executive officer of Portola. "We completed enrollment in the APEX study of betrixaban, our Fast Track-designated product. For andexanet alfa, our breakthrough Factor Xa reversal agent, we initiated our rolling submission of our Biologics License Application, or BLA, to the U.S. Food and Drug Administration, or FDA. That comes on the heels of two important andexanet manufacturing milestones: we completed the commercial manufacturing process validation in support of our submission and commercial launch, and successfully scaled-up our first Generation 2 process, which will allow us to increase our production capacity at lower costs. These achievements keep us on track for our planned launch of andexanet alfa in 2016 followed by betrixaban in 2017, assuming clinical and regulatory success."

Other Recent Achievements, Upcoming Events and Milestones

Betrixaban

Completed APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) study enrollment.
Successfully completed a sixth and final review of the APEX study Data and Safety Monitoring Committee, to correspond with an increase in sample size from 6,850 to 7,500 patients.
Received Fast Track designation from the FDA for prevention of VTE in acute medically ill patients.
APEX Study metrics, including pooled blinded aggregate event rates, remain on target.
Plan to report topline APEX study data late in the first quarter of 2016.

Plan to submit a New Drug Application to the FDA in 2016, subject to positive data.

Andexanet Alfa

Initiated rolling submission of BLA; we expect the application to be complete by the end of 2015 under Accelerated Approval pathway.

Completed commercial manufacturing process validation at CMC Biologics to support the BLA and commercial launch at 2,500 liter scale.

Successfully scaled up our first Generation 2, 10,000 liter batch product at Lonza.

Announced topline data from the second part of the Phase 3 ANNEXATM-R study, which demonstrated that andexanet alfa administered as an intravenous bolus followed by a continuous two-hour infusion produced rapid reversal of the anticoagulant effect of rivaroxaban and sustained it for the duration of the infusion. The full data set from Part 2 of the study will be presented during a Late-Breaking Clinical Trial session at the American Heart Association’s Scientific Sessions 2015 on November 11 in Orlando.
Enrollment remains on track in ANNEXA-4, a Phase 4 confirmatory study of patients receiving apixaban, rivaroxaban, edoxaban or enoxaparin who present with an acute major bleed.

Initiated a Phase 2 proof-of-concept study in healthy volunteers to evaluate the safety and efficacy of andexanet alfa in reversing the anticoagulant effect of betrixaban, and define the dose of betrixaban required for inclusion in ANNEXA-4.
Plan to present two abstracts at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December in Orlando.

Cerdulatinib – Oral, dual Syk/JAK kinase inhibitor for hematologic cancers

Continued to dose-escalate in the Phase 1 part of the ongoing Phase 1/2a study in patients with relapsed/refractory B-cell malignancies who have failed multiple therapies to determine the maximum tolerated dose. Exploring alternate dosing regiments and formulations to get to a higher exposure. Cerdulatinib continues to show activity and is well tolerated.
Plan to present three abstracts at the ASH (Free ASH Whitepaper) Annual Meeting in December in Orlando.

Corporate

Elected David C. Stump, M.D., former executive vice president, research and development, at Human Genome Sciences, Inc., to Portola’s board of directors.

Third Quarter Financial Results

Collaboration revenue earned under Portola’s collaborations with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, Daiichi Sankyo and Lee’s Pharmaceutical was $2.9 million for the third quarter of 2015 compared with $2.4 million for the third quarter of 2014.

Total operating expenses for the third quarter of 2015 were $58.5 million compared with $38.2 million for the same period in 2014. Total operating expenses for the third quarter of 2015 included $6.1 million in stock-based compensation expense compared with $2.2 million for the third quarter of 2014. Research and development expenses were $48.4 million for the third quarter of 2015 compared with $31.8 million for the third quarter of 2014 as the Company continued to support its manufacturing scale-up of andexanet alfa in preparation for BLA submission and commercial launch and work on its larger scale Generation 2 manufacturing process at Lonza; its Phase 3 and Phase 4 ANNEXA studies of andexanet alfa; completing enrollment in the Phase 3 APEX Study of betrixaban and its Phase 1/2a clinical study of cerdulatinib. Selling, general and administrative expenses for the third quarter of 2015 were $10.1 million compared with $6.4 million for the same period in 2014 as the Company increased headcount to support its growth, resulting in higher headcount-related costs including stock-based compensation expense, and increased pre-commercial launch activities.

Portola reported a net loss of $55.2 million, or $(1.05) net loss per share, for the third quarter of 2015 compared with a net loss of $35.8 million, or $(0.86) net loss per share, for the third quarter of 2014. Shares used to compute net loss per share attributable to common stockholders were approximately 52.6 million for the third quarter of 2015 compared with approximately 41.4 million for the same period in 2014.

As of September 30, 2015, cash, cash equivalents and investments totaled $356.0 million compared with cash, cash equivalents and investments of $392.3 million as of December 31, 2014.

(Filing, 10-Q, Galena Biopharma, NOV 9, 2015, View Source [SID:1234508155])

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On November 09, 2015 OncoMed Pharmaceuticals Inc. (NASDAQ:OMED) reported clinical and preclinical data related to three of its clinical-stage 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) (Press release, OncoMed, NOV 9, 2015, View Source [SID:1234508139]). Data from three posters covered Phase 1a safety, biomarker and anti-tumor activity of brontictuzumab (anti-Notch1, OMP-52M51), novel biomarker discoveries related to vantictumab (anti-Fzd7, OMP-18R5) in non-small cell lung cancer (NSCLC) and preclinical characterization of safety and efficacy for anti-DLL4/VEGF bispecific (OMP-305B83).

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Brontictuzumab Phase 1a: Single-agent Activity in Biomarker-positive Patients; Manageable Safety Profile

Among the data presented were Phase 1a clinical trial results for brontictuzumab in patients with advanced solid tumors. Brontictuzumab demonstrated single-agent activity in a biomarker-selected refractory patient population. Among 15 patients whose tumors overexpressed the activated form of Notch1, as measured by OncoMed’s proprietary immunohistochemistry (IHC) test, eight patients achieved stable disease or partial response for an overall clinical benefit rate of 53 percent. Anti-tumor activity was observed in adenoid cystic carcinoma, colorectal cancer and HER2 negative breast cancer. Partial responses were observed in two patients with adenoid cystic carcinoma after just one dose of brontictuzumab. Among patients whose tumors measured high in Notch1 activation, five have survived 100 days or longer as of the data cut off. There are five additional Notch1 high patients that are currently ongoing on the study and OncoMed plans to present follow-up data on those patients when the data matures. In biomarker negative subjects, only one of 11 had clinical benefit (9%). Brontictuzumab was generally well tolerated, with the most common adverse event being on-target, manageable diarrhea. Notch pathway and cancer stem cell pathway markers were reduced in serial tumor biopsies and in surrogate patient samples (blood) at doses above 1.5 mg/kg every three weeks. The single agent Phase 2 dose of brontictuzumab was established as 1.5mg/kg every three weeks.

"We are very encouraged by this early evidence of single-agent activity of brontictuzumab in a refractory biomarker-selected patient population. Notch1 has been shown to be elevated in a variety of solid tumor types, including breast, colorectal, esophageal, cholangiocarcinoma, pancreatic, small cell lung and adenoid cystic carcinomas," said Jakob Dupont, M.D., OncoMed’s Senior Vice President, Chief Medical Officer. "Based on these results and the manageable safety profile, Phase 1b combination studies of brontictuzumab in combination with chemotherapy are being contemplated with a particular emphasis on the Notch1 biomarker positive subjects."

These data were presented by Pamela Munster, M.D., Professor of Medicine, Program Leader Development Therapeutics, and Director of Early Phase Clinical Trials’ Program Helen Diller Cancer Center of the University of California, San Francisco in a poster titled: Safety and preliminary efficacy results of a first-in-human Phase I study of the novel cancer stem cell (CSC) targeting antibody brontictuzumab (OMP-52M51, anti-Notch1) administered intravenously to patients with certain advanced solid tumors (Abstract #C42).

"This successful Phase 1 study of brontictuzumab has provided much insight into this drug candidate. We have learned about the safety profile that can be managed; we have evidence of on-target effects against the Notch pathway; and we have observed single-agent activity in biomarker-selected patients," said Dr. Munster. "Taken together, data from this study suggests that brontictuzumab has the potential to benefit patients and should be developed in future studies with standard of care, particularly in biomarker selected patients."

Vantictumab in Non-Small Cell Lung Cancer — Novel Biomarker Identified

In a preclinical patient-derived xenograft non-small cell lung cancer (NSCLC) model, OncoMed researchers confirmed the anti-tumor activity of vantictumab both as a single-agent and in combination with taxane treatment. Sequential dosing (the administration of vantictumab two days prior to treatment with paclitaxel) was more efficacious than same-day dosing. Pharmacodynamic biomarkers showed that vantictumab inhibits genes in cancer stem cell pathways that support its mechanism of action. Importantly, LEF1, a key transcription factor in the Wnt pathway was identified as a potential predictive biomarker for treatment response in patient-derived xenograft models. LEF1 is being retrospectively evaluated as a predictive biomarker in OncoMed’s ongoing Phase 1b clinical trial of vantictumab in combination with docetaxel in patients with previously treated NSCLC. Results of these studies were presented on Friday, November 6 in a poster titled: Predictive and pharmacodynamic biomarkers of vantictumab (OMP-18R5; anti-Frizzled7) in non-small cell lung cancer (Abstract #A30) by Ann Kapoun, Ph.D. of OncoMed.

Anti-DLL4/VEGF Bispecific — Superior Inhibition of Tumor Growth

Preclinical studies of OncoMed’s dual-targeting anti-DLL4/VEGF bispecific antibody in xenograft tumor models demonstrated superior anti-tumor activity compared to either anti-DLL4 and anti-VEGF antibodies alone. The combination of anti-DLL4 and anti-VEGF resulted in broad-spectrum activity in many different tumor types including breast, colon, ovarian and pancreatic tumors. In serial transplantation studies, the anti-DLL4/VEGF bispecific antibody showed a greater effect than anti-DLL4 alone in delaying tumor recurrence following the termination of treatment and reducing the frequency of cancer stem cells in the tumors. Researchers also observed that simultaneous inhibition of DLL4 and VEGF induced a down-regulation of vasculature-related genes and decreased vasculature density. The anti-DLL4/VEGF bispecific antibody showed an improved cardiac profile in cynomolgus monkeys compared to anti-DLL4 which may translate to an improved safety profile in the clinic. OncoMed is currently testing the anti-DLL4/VEGF bispecific in a Phase 1a clinical trial for patients with advanced refractory solid tumors. These data were presented by Wang-Ching Yen, Ph.D., of OncoMed in a poster titled: Dual targeting of the DLL4 and VEGF pathways with a bispecific monoclonal antibody inhibits tumor growth and reduces cancer stem cell frequency (Abstract #C134) on November 8, 2015.

"The results of studies presented at the AACR (Free AACR Whitepaper)-NCI-EORTC meeting provide valuable information on optimal dosing of vantictumab plus taxane therapy and have identified a promising biomarker in the non-small cell lung cancer indication," said John Lewicki, Ph.D., Executive Vice President, Chief Scientific Officer. "And, in preclinical models, our bispecific is demonstrating robust anti-tumor activity against a number of tumor types and the combination of anti-DLL4 and anti-VEGF appears to have the potential to improve upon the cardiotoxicity profiles of either agent alone. We look forward to evaluating how these data translate into the clinic in our ongoing trials."

Brontictuzumab, vantictumab and the anti-DLL4/VEGF bispecific are novel investigational therapeutics currently being evaluated in ongoing clinical trials. Patients interested in learning more about participating in one of OncoMed’s many clinical trials may learn more by calling 1-866-914-7347 or emailing [email protected].

On November 9, 2015 Puma Biotechnology, Inc. (NYSE: PBYI), a biopharmaceutical company, reported financial results for the third quarter ended September 30, 2015 (Filing, 8-K, Puma Biotechnology, NOV 9, 2015, View Source [SID:1234508176]).

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Unless otherwise stated, all comparisons are for the third quarter and nine months ended September 30, 2015, compared to the third quarter and nine months ended September 30, 2014.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss of $60.4 million, or $1.87 per share, for the third quarter of 2015, compared to a net loss of $35.9 million, or $1.19 per share, for the third quarter of 2014. Net loss for the nine months ended September 30, 2015 was $177.6 million, or $5.55 per share, compared to $94.5 million, or $3.16 per share, for the nine months ended September 30, 2014.

Non-GAAP adjusted net loss was $35.5 million, or $1.10 per share, for the third quarter of 2015, compared to $25.4 million, or $0.84 per share, for the third quarter of 2014. Non-GAAP adjusted net loss for the nine months ended September 30, 2015 was $104.3 million, or $3.26 per share, compared to $71.7 million, or $2.40 per share, for the nine months ended September 30, 2014. Non-GAAP adjusted net loss excludes stock-based compensation expense, which represents a significant portion of overall expense and has no impact on the cash position of the Company. For a reconciliation of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release.

Net cash used in operating activities for the third quarter of 2015 was $36.8 million. Net cash used in operating activities for the nine months ended September 30, 2015 was $121.4 million. At September 30, 2015, Puma had cash and cash equivalents of $23.6 million and marketable securities of $224.2 million, compared to cash and cash equivalents of $38.5 million and marketable securities of $102.8 million at December 31, 2014. Puma’s current level of cash and cash equivalents and marketable securities includes net proceeds of approximately $205.1 million from a public offering of the Company’s common stock, which was completed in January 2015.

"We continued to make significant progress with the neratinib clinical program in the third quarter," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "Puma achieved a number of milestones, including the presentation of additional data on patients with centrally confirmed HER2-positive disease from our Phase III ExteNET Trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2015 Breast Cancer Symposium and the publication in the Journal of the National Comprehensive Cancer Network of a patient with HER2 non-amplified (HER2-negative) metastatic breast cancer who also had a HER2 activating mutation and was successfully treated with PB272. We continue to anticipate filing for regulatory approval of PB272 for the extended adjuvant treatment of HER2-positive breast cancer in the first quarter of 2016.

"We anticipate a number of additional milestones through the end of 2015 and beyond. These include (i) presentations of additional data from the Phase III ExteNET Trial in the extended adjuvant treatment of early stage HER2-positive breast cancer (anticipated in the fourth quarter of 2015); (ii) publication of Phase III ExteNET Trial results (anticipated in the fourth quarter of 2015); (iii) presentation of the Phase II FB-7 trial of PB272 as a neoadjuvant treatment for patients with HER2-positive breast cancer (anticipated in the fourth quarter of 2015); (iv) presentation of data from our Phase II trial of PB272 in HER2 non-amplified breast cancer that has a HER2 mutation (anticipated in the fourth quarter of 2015); (v) reporting initial data from the Phase II trial of neratinib in extended adjuvant HER2-positive early stage breast cancer using loperamide prophylaxis (anticipated in the fourth quarter of 2015); (vi) publication of results to date on the use of prophylactic loperamide to reduce the neratinib-related diarrhea (anticipated in the fourth quarter of 2015); (vii) completing the ongoing Phase II trial of PB272 in patients with HER2-positive metastatic breast cancer that has metastasized to the brain (anticipated in the first half of 2016); and (viii) expanding additional cohorts in our Phase II basket trial of PB272 in patients with solid tumors with activating HER2 mutations (anticipated in the fourth quarter of 2015)."

Operating Expenses

Based on GAAP, operating expenses were $60.7 million for the third quarter of 2015, compared to $36.0 million for the third quarter of 2014. Operating expenses for the nine months ended September 30, 2015 were $178.2 million, compared to $94.7 million for the nine months ended September 30, 2014. The increase in operating expenses for the third quarter of 2015 compared to the third quarter of 2014 was primarily driven by an increase in stock-based compensation expense of $14.4 million, an increase in clinical trial expense of $5.1 million and an increase in internal research and development expense of $3.0 million. The increase in operating expenses for the nine months ended September 30, 2015 compared to the same period in 2014 was primarily driven by an increase in stock-based compensation of $50.5 million, an increase in clinical trial expense of $19.4 million and an increase in internal research and development expense of $8.2 million.

General and Administrative Expenses:

Based on GAAP, general and administrative expenses were $8.8 million for the third quarter of 2015, compared to $3.9 million for the third quarter of 2014. General and administrative expenses for the nine months ended September 30, 2015 were $22.2 million compared to $11.3 million for the nine months ended September 30, 2014.

Research and Development Expenses:

Based on GAAP, research and development expenses were $51.9 million for the third quarter of 2015, compared to $32.1 million for the third quarter of 2014. Research and development expenses for the nine months ended September 30, 2015 were $156.0 million, compared to $83.4 million for the nine months ended September 30, 2014.

(Filing, 10-Q, GTx, NOV 9, 2015, View Source [SID:1234508157])

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