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.

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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

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).

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"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.

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]).

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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.

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 .

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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.

On February 5, 2017 Abbisko Therapeutics Co., Ltd. reported the successful completion of its series A round financing at $28 million (Press release, Abbisko Therapeutics, FEB 5, 2017, View Source;article_id=70 [SID1234556289]). This round was led by Lilly Asia Ventures and followed by Sinopharm Capital, Jianxin Capital, and TF Capital.

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The fund raised in this round will be used to support new drug research of Abbisko Therapeutics Co., Ltd. (hereinafter referred to as Abbisko), including setting up a new drug R&D center, building an innovative drug R&D team and establishing antitumor drug R&D pipelines.

Abbisko is a new innovative biopharmaceutical company established in Zhangjiang of Shanghai. The company’s founding members previously held significant R&D leadership and management positions in several Fortune 500 pharmaceutical companies (Novartis, Johnson & Johnson, Merck, Eli Lilly and AbbVie) and China’s leading pharmaceutical companies (Hengrui and Hansoh). They have extensive project leadership and team management experience in novel drug development and clinical trials. Abbisko will focus on unmet needs of patients especially those of Chinese patients, follow international standards for new drug development, and dedicate itself to the research and development of innovative therapeutics with global intellectual property rights.

Dr. Yao-chang Xu, Abbisko’s founder, board chairman and CEO, commented on this round of financing: "We are gratified that several of the world’s leading investment institutions participated in this very important round of financing. We are a new but highly experienced drug development team. The investors agree with our strategy and philosophy. As a result, our team can set sail quickly and seize the opportunities brought by the rapid development of biotech sector in China. We’ll work hard to bring more revolutionary treatment solutions to suffering patients as soon as possible."

Mr. Aimin Wu, Sinopharm Capital’s president and partner, said: "At present, China’s innovative drug R&D are in a golden age of development. Abbisko has established a top-notch R&D team with a fast growing cutting-edge product pipelines. Sinopharm fully recognize and appreciates Abbisko’s R&D strength and supports its direction. Led by Dr. Yao-chang Xu, Abbisko is capable of becoming a super star company in the biomedical sector. Sinopharm is honored to participate in the establishment and development of Abbisko, helping domestic biomedical companies to compete with international innovative enterprises."

With Abbisko’s successful establishment and completion of this round of investment, an important new force has risen for China’s innovative drug R&D.