1stOncology™: Cancer Intelligence Service – Page 16169 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Galena Biopharma Reports Positive Outcome from the Data Safety Monitoring Board on the Two NeuVax™ (nelipepimut-S) Clinical Trials in Combination with Trastuzumab

On February 6, 2017 Galena Biopharma, Inc. (NASDAQ:GALE), a biopharmaceutical company committed to the development and commercialization of hematology and oncology therapeutics that address unmet medical needs, reported the results from a meeting of the Data Safety Monitoring Board (DSMB) for the two investigator-sponsored (IST) combination clinical trials with NeuVax (nelipepimut-S) plus trastuzumab (Press release, Galena Biopharma, FEB 6, 2017, View Source [SID1234517647]). The trials are being run in breast cancer patients to assess the ability of the combination of trastuzumab and the HER2 vaccine nelipepimut-S (administered with the immunoadjuvant granulocyte macrophage-colony stimulating factor) to prevent recurrence in the adjuvant setting.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

In light of the early termination of the NeuVax Phase 3 PRESENT (Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer with Low to Intermediate HER2 Expression with NeuVax Treatment) trial last year, the independent DSMB performed separate futility assessments on each of the NeuVax plus trastuzumab combination trials. The DSMB reported that there are no safety concerns with either trial and neither was found to be futile.

For the Phase 2b trial in patients with low-to-intermediate HER2 expression (HER2 1+/2+), n=242 patients were evaluated, and the recommendation from the DSMB is to continue the trial with one revision to the statistical analysis plan regarding the timing of the pre-specified interim analysis. Given the lengthy duration of enrollment for the trial, the DSMB determined that the pre-specified interim efficacy analysis be moved up from 12 months to 6 months after the last patient is enrolled. Completion of enrollment is expected in the second quarter of 2017; therefore, the DSMB expects to perform the interim efficacy analysis near the end of 2017.

For the Phase 2 trial in high-risk, HER2 3+ patients, and per the trial protocol, the pre-specified interim safety analysis was also completed on n=50 patients and demonstrated that the agent is well tolerated with no increased cardiotoxicity associated with giving NeuVax in combination with trastuzumab. These findings were similar to the findings presented in October 2016 from the HER2 1+/2+ trial. The recommendation from the DSMB is to continue the HER2 3+ trial unmodified.

"The recommendations from the DSMB are very positive for NeuVax in combination with trastuzumab as there were no safety concerns and the studies were not deemed futile. Given these findings, the trials can continue per the DSMB recommendation," said Bijan Nejadnik, M.D., Executive Vice President and Chief Medical Officer. "We are also pleased that we will be able to reach the interim efficacy analysis in our Phase 2b HER2 1+/2+ trial earlier than expected with that readout now planned by the end of this year. It is important to note that these IST trials are designed with a different methodology to identify recurrences clinically and not via proactive imaging as was done in the PRESENT trial. We are grateful to our investigators running these trials and their continued dedication to breast cancer patients as they study potential new therapeutic options to prevent recurrence."

About NeuVax (nelipepimut-S)

NeuVax (nelipepimut-S) is a first-in-class, HER2-directed cancer immunotherapy under evaluation to prevent breast cancer recurrence after standard of care treatment in the adjuvant setting. NeuVax contains the immunodominant peptide derived from the extracellular domain of the HER2 protein, a well-established target for therapeutic intervention in breast carcinoma. The nelipepimut-S sequence stimulates specific CD8+ cytotoxic T lymphocytes (CTLs) following binding to specific HLA molecules on antigen presenting cells (APC). These activated specific CTLs recognize, neutralize and destroy, through cell lysis, HER2 expressing cancer cells, including occult cancer cells and micrometastatic foci. The nelipepimut-S immune response can also generate CTLs to other immunogenic peptides through inter- and intra-antigenic epitope spreading. In clinical studies, NeuVax is combined with recombinant granulocyte macrophage-colony stimulating factor (GM-CSF).

NeuVax is currently in two breast cancer studies in combination with trastuzumab (Herceptin; Genentech/Roche): a Phase 2b trial in node positive and triple negative HER2 IHC 1+/2+ (clinicaltrials.gov identifier: NCT01570036); and, a Phase 2 trial in high risk, node positive or negative HER2 IHC 3+ patients (clinicaltrials.gov identifier: NCT02297698). A Phase 2 clinical trial is also ongoing with NeuVax in patients with ductal carcinoma in situ (DCIS) (clinicaltrials.gov identifier: NCT02636582), and a Phase 2 trial is planned in patients with gastric cancer.

About Breast Cancer1

New cases of breast cancer occur at an annual rate of 125 per 100,000 women in the U.S., with an estimated 246,660 new cases and 40,450 deaths in 2016. Approximately 89.7% of breast cancer patients are expected to survive five years after diagnosis. Approximately 12.4% of women will be diagnosed with breast cancer at some point during their lifetime (2011 – 2013 data). Of the women diagnosed with breast cancer annually, about 25% are HER2 positive (IHC 3+) and approximately 50%-60% of these women express HER2 at a low to intermediate range (IHC 1+/2+ or FISH < 2.0). The prevalence data from 2013 showed an estimated 3,053,450 women living with breast cancer in the United States.

1National Cancer Institute Surveillance, Epidemiology, and End Results Program

Arrowhead Reports Fiscal 2017 First Quarter Results

On February 6, 2017 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2017 first quarter ended December 31, 2016 (Press release, Arrowhead Pharmaceuticals, FEB 6, 2017, View Source [SID1234517646]). The company is hosting a conference call at 4:30 p.m. EST to discuss results.

Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and enter Conference ID 59701860.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 404-537-3406 and enter Conference ID 59701860.

Selected Fiscal 2017 First Quarter and Recent Events

Took steps to redeploy company resources to support development of RNAi therapeutics that utilize the company’s new proprietary subcutaneous and extra-hepatic delivery systems, including:
Discontinued the development of ARC-520, ARC-521, and ARC-AAT which utilized the intravenously administered DPCiv, or EX1, delivery vehicle
Reduced the workforce by approximately 30%, while maintaining resources necessary to support current and potential partner-based programs and the Company’s pipeline
Continued progress on preclinical pipeline including ARO-HBV, ARO-AAT, ARO-F12, ARO-HIF2, and programs partnered with Amgen, ARO-LPA and ARO-AMG1
Continued progress on former drug candidates prior to the discontinuations
Presented preclinical and clinical data on former drug candidate ARC-AAT at the Liver Meeting, providing validation of the potential of RNAi in alpha-1 liver disease
Advanced former drug candidate ARC-521 into a Phase 1/2 study
Conducted multiple dose and combination studies of former drug candidate ARC-520
Selected Fiscal 2017 First Quarter Financial Results

ARROWHEAD PHARMACEUTICALS, INC.
CONSOLIDATED CONDENSED FINANCIAL INFORMATION (unaudited)

Three Months Ended
December 31,
OPERATING SUMMARY
2016 2015

REVENUE $ 4,365,496 $ 43,750
OPERATING EXPENSES
Research and development 9,527,051 10,338,833
Salaries and payroll-related costs 4,276,105 3,919,886
General and administrative expenses 1,854,174 1,951,609
Stock-based compensation 2,424,442 2,380,343
Depreciation and amortization 1,185,611 794,349
TOTAL OPERATING EXPENSES 19,267,383 19,385,020
OPERATING LOSS (14,901,887 ) (19,341,270 )
OTHER INCOME/(EXPENSE), PROVISION FOR INCOME TAXES 2,815,779 76,856
NET LOSS $ (12,086,108 ) $ (19,264,414 )

EARNINGS PER SHARE (BASIC AND DILUTED): $ (0.17 ) $ (0.32 )
WEIGHTED AVERAGE SHARES OUTSTANDING 71,444,600 59,548,672

FINANCIAL POSITION SUMMARY
December 31, September 30,

2016 2016

CASH AND CASH EQUIVALENTS 102,105,569 85,366,448
OTHER ASSETS 42,152,537 42,810,057
TOTAL ASSETS 144,258,106 128,176,505
TOTAL LIABILITIES 47,049,685 33,152,246
TOTAL STOCKHOLDERS’ EQUITY 97,208,421 95,024,259
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY 144,258,106 128,176,505

SHARES OUTSTANDING 74,413,040 69,746,685

ARIAD Announces Submission of Marketing Authorization Application for Brigatinib to the European Medicines Agency

On February 6, 2017 ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) reported the submission of a Marketing Authorization Application (MAA) for its investigational oral anaplastic lymphoma kinase (ALK) inhibitor, brigatinib, to the European Medicines Agency (EMA) (Press release, Ariad, FEB 6, 2017, View Source;p=RssLanding&cat=news&id=2243225 [SID1234517645]). ARIAD is seeking marketing approval in the European Union of brigatinib in adult patients with anaplastic lymphoma kinase (ALK+) non-small cell lung cancer (NSCLC) who have been previously treated with crizotinib. The U.S. Food and Drug Administration (FDA) is currently reviewing a New Drug Application for brigatinib filed by ARIAD and has set an action date of April 29, 2017 under the Prescription Drug User Fee Act (PDUFA).

"ARIAD’s submission of the brigatinib MAA to the EMA is one of many recent milestones highlighting our strong investment in internally discovered rare cancer therapies," said Paris Panayiotopoulos, president and chief executive officer of ARIAD. "Since announcing our definitive agreement to combine with Takeda, we remain focused on our accountability to our patients by propelling brigatinib forward and by preparing for its anticipated U.S. launch."

"The brigatinib clinical trials have provided patients with refractory ALK+ NSCLC, including those patients who have metastatic brain lesions, with a potential important treatment option," said Maurice Perol, MD, Léon Bérard Cancer Center, Lyon, France. "Based on the clinical data we’ve seen to date, we are really excited by the prospect that appropriate patients in the EU may have access to brigatinib as a new targeted treatment."

ARIAD’s MAA submission includes clinical data from its Phase 1/2 and pivotal Phase 2 ALTA trials of brigatinib. Results from the ALTA trial and central nervous system (CNS) activity in the ALTA and Phase 1/2 trials were reported at the International Association for the Study of Lung Cancer (IASLC) 17th World Conference on Lung Cancer (WCLC) in December 2016. In the ALTA trial, 222 patients received either 90 mg of brigatinib once per day (QD) continuously or 180 mg QD, preceded by a lead-in dose of 90 mg QD for seven days. Data from the ALTA trial demonstrated that of 110 patients on the 180-mg regimen QD with a seven-day lead-in at 90 mg QD with a median follow-up of 11.0 months, 55 percent achieved confirmed objective response as assessed by the investigator. In this arm, the median progression-free survival (PFS) was 15.6 months in this post-crizotinib setting, by both investigator and independent review committee (IRC) assessment. Additionally, in this arm, 67 percent (12/18) of patients with measurable brain metastases achieved a confirmed intracranial objective response. The most common treatment-emergent adverse events (TEAEs; ≥ 25% of all patients in this arm, regardless of relationship to treatment), were nausea (43%), fatigue (40%), diarrhea (39%), cough (36%), increased blood creatine phosphokinase (CPK) (33%), headache (30%), rash (28%), and vomiting (26%). The most common serious adverse reactions other than neoplasm progression reported in 2% or more of patients included pneumonia (5.0%), pneumonitis (5.0%) and epilepsy (2.3%). A subset of pulmonary adverse events (AEs) with early onset (median: Day 2; range: Day 1-9) occurred in 6.4 percent of all patients (grade ≥3 in 3% of patients); no such events with early onset occurred after dose escalation to 180 mg QD following the lead-in dose of 90 mg for seven days.

About Brigatinib

Brigatinib is an investigational, targeted cancer medicine discovered internally at ARIAD. It is in development for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) non-small cell lung cancer (NSCLC). The global Phase 2 ALTA trial, in patients with locally advanced or metastatic ALK+ NSCLC who were previously treated with crizotinib, is the primary basis for brigatinib’s initial regulatory review. ARIAD has also initiated the Phase 3 ALTA 1L trial to assess the efficacy and safety of brigatinib in comparison to crizotinib in patients with locally advanced or metastatic ALK+ NSCLC who have not received prior treatment with an ALK inhibitor. More information on brigatinib clinical trials can be found here.

Brigatinib received Breakthrough Therapy designation from the FDA for the treatment of patients with ALK+ NSCLC whose tumors are resistant to crizotinib, and was granted Orphan Drug designation by the FDA for the treatment of ALK+, ROS1+ and EGFR+ NSCLC.

About ALK+ NSCLC

Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for approximately 80 to 85 percent of the estimated 222,500 new cases of lung cancer diagnosed each year in the United States, according to the American Cancer Society. Anaplastic lymphoma kinase (ALK) was first identified as a chromosomal rearrangement in anaplastic large-cell lymphoma (ALCL). Genetic studies indicate that chromosomal rearrangements in ALK are key drivers in a subset of NSCLC patients as well. Approximately five percent of patients with NSCLC have a rearrangement in the ALK gene, according to the American Cancer Society.

Advaxis Announces First Patient Dosed in Global Phase 3 AIM2CERV Trial for High-risk, Locally Advanced Cervical Cancer

On February 6, 2017 Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies, reported that the first patient has been enrolled and dosed in its AIM2CERV (Advaxis IMmunotherapy 2 prevent CERVical recurrence) trial, the only company-sponsored global phase 3 trial enrolling patients with advanced stage cervical cancer (Press release, Advaxis, FEB 6, 2017, View Source [SID1234517644]).

AIM2CERV is a global, randomized, 450-patient study evaluating axalimogene filolisbac as an adjuvant therapy following primary treatment with chemotherapy and radiation in patients with high-risk locally advanced carcinoma of the cervix (HRLACC). The primary objective of the trial is to compare disease-free survival (DFS) in patients receiving axalimogene filolisbac to DFS in patients receiving placebo, with secondary objectives including overall survival, safety and tolerability. AIM2CERV is being conducted in collaboration with the GOG Foundation, Inc. under a Special Protocol Assessment (SPA) agreement with U.S. Food and Drug Administration (FDA).

"While some women with cervical cancer are considered cancer-free after chemotherapy and radiation, a significant number will experience a recurrence of the disease that is often more aggressive and results in a poor prognosis. The global reach of the AIM2CERV study is intended to determine whether treatment with axalimogene filolisbac after chemotherapy and radiation can help prevent or delay such recurrences," said Daniel J. O’Connor, President and Chief Executive Officer of Advaxis. "Following recently published data that revealed cervical cancer mortality rates may be exponentially higher in African American women and significantly underestimated for all women, coupled with research last year showing fewer than half of patients with locally advanced cervical cancer receive standard-of-care therapy, the need for new treatment options is more clear now than ever."

Cervical cancer occurs most often in women who have been infected by the human papillomavirus (HPV) and is the most common HPV-associated cancer in women. According to the American Cancer Society, approximately 12,000 women in the United States will be diagnosed with cervical cancer in 2017. Approximately 4,900 of these patients will be diagnosed with HRLACC, according to the Surveillance, Epidemiology and End Results (SEER) Program. While there are multiple vaccines available to prevent HPV, only one-third of the U.S. population has been vaccinated against the virus and the vaccination rate is lower worldwide. Cervical cancer largely affects women who have not received vaccines or regular screenings.

"Through our National Cervical Cancer Coalition (NCCC), every day we talk to women struggling with cervical cancer and the families of those who have lost loved ones to cervical cancer," said Deborah Arrindell, Vice President, Health Policy at the American Sexual Health Association, an umbrella organization which includes NCCC. "Just as we need options to prevent HPV-related cancers, there is a significant need for more therapeutic options to treat those with cancer. No woman should die from cervical cancer."

Axalimogene filolisbac, which received orphan drug designation and Fast Track designation for adjuvant therapy in HRLACC and orphan drug designation for stage II-IV cervical cancer from the FDA and was classified as an advanced therapy medicinal product (ATMP) for the treatment of cervical cancer by the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT), has shown encouraging activity and a manageable safety profile in phase 2 studies. Last year, the company presented positive data from the completed Phase 2 GOG-0265 study showing a promising rate of survival in persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix (PRmCC). Top-line data released in 2016 showed a 12-month overall survival rate of 38 percent observed in 50 patients in the trial. This was a 52 percent improvement over the 12-month overall survival rate that was expected in the trial’s patient population based on prognostic factors. The most commonly reported side effects were hypotension and symptoms related to cytokine release, including nausea, chills and fever. The full data set from the GOG-0265 study will be presented at the Society of Gynecologic Oncology’s 48th Annual Meeting on Women’s Cancer in March.

The company plans to enroll approximately 450 patients at 150 global sites. For additional information, please visit www.clinicaltrials.gov (NCT 02853604) or call 844-783-1529.

About Cervical Cancer

Cervical cancer is the fourth most common cancer in women worldwide and the second leading cause of cancer death in women under age 50. An estimated 500,000 new cases are diagnosed in globally and approximately 270,000 people die of the disease each year. Persistent HPV infection is the most important factor in the development of cervical cancer, research shows. According to the ICO Information Centre on HPV and Cervical Cancer, about 70 percent of cervical cancers are attributed to high-risk HPV strains. The prognosis for women with advanced and recurrent cervical cancer remains poor, with survival of only 4 to 7 months following failure of first-line treatment, research has shown. According to the American Cancer Society, the 5-year survival rate for metastatic disease is at just 15 percent, with the area continuing to be a high unmet medical need.

Genmab Announces Updated 2016 Financial Guidance

On February 6, 2017 Genmab A/S (Nasdaq Copenhagen: GEN) reported that it has updated its 2016 Financial Guidance (Press release, Genmab, FEB 6, 2017, View Source [SID1234517639]). Based on preliminary reporting, Genmab is improving its 2016 financial guidance published on December 20, 2016 .

The table below summarizes our revised and previous guidance.
MDKK Revised Guidance Previous Guidance
Revenue 1,790 — 1,840 1,720 — 1,770
Operating expenses (750) — (800) (800) — (850)
Operating income 1,015 — 1,065 895 — 945
Cash position at end of year* 3,850 — 3,950 3,650 — 3,750
*Cash, cash equivalents, and marketable securities
The revenue range has increased primarily due to higher royalties on net sales of DARZALEX by Janssen and achievement of additional DuoBody milestones. Among other items, the operating expense range has decreased due to timing of clinical and pre-clinical expenses. The operating income range has increased due to the combination of higher revenue and lower expenses. Our cash position has increased mainly due to timing of operating expenses and other working capital adjustments.
As previously announced, the annual report for 2016 will be published on February 22, 2017.