On February 6, 2017 Kiadis Pharma N.V. ("Kiadis Pharma" or the "Company") (Euronext Amsterdam and Brussels: KDS), a clinical stage biopharmaceutical company developing innovative T-cell immunotherapy treatments for blood cancers and inherited blood disorders, reported a progress update on the clinical and regulatory status of ATIR101, the Company’s lead product to address the key risks and limitations of hematopoietic stem cell transplantation (HSCT) in blood cancer (Press release, Kiadis, FEB 6, 2017, View Source [SID1234517649]).

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Initiation of pivotal Phase III trial with ATIR101
Following regulatory approval from the national authority in Canada (Health Canada), the Company is pleased to announce the initiation of its randomized, controlled, transatlantic Phase III trial with ATIR101 (CR-AIR-009). Approximately 195 patients with acute leukemia will be enrolled in total in the Phase III trial and randomized 1:1 to receive a haploidentical allogeneic HSCT using either the Kiadis Pharma approach with a single dose of ATIR101 or the post-transplant cyclophosphamide approach (also known as the Baltimore Protocol). The trial protocol has also been submitted for approval and is being evaluated by the United States FDA (Food and Drug Administration) as well as several European regulatory authorities. Following approval the trial will be rolled out to additional study centers in the United States and Europe.

CTI Clinical Trial and Consulting Services, Inc., an international clinical contract research organization, has been appointed to work with Kiadis Pharma to support the clinical part of the trial and PCT, LLC, A Caladrius Company, a leading external manufacturing partner to the cell therapy industry, will manufacture ATIR101 for the United States and Canada. The German Red Cross Blood Donor Service, Baden-Wuerttemberg-Hessen, will remain the manufacturer in Europe. All 15 study centers that participated in the Company’s Phase II trials with ATIR101 (CR-AIR-007 and CR-AIR-008) have confirmed their intention to participate in the Phase III trial (CR-AIR-009). Kiadis Pharma is actively and rapidly aligning more sites with the aim of having more than 40 sites in North America and Europe participating in the Phase III trial.

Ongoing CR-AIR-008 trial continues to confirm safety profile of ATIR101
Infusing a single dose of ATIR101 continues to be safe in the ongoing Phase II trial with ATIR101 (CR-AIR-008) which, as previously announced, is continuing to treat patients with a single dose of ATIR101 according to the clinical protocol and the recommendation of the Independent Data Monitoring Committee (IDMC). Five patients in this trial have now been treated with a single dose of ATIR101 of which three were infused more than 120 to 150 days ago. None of these patients have shown any symptoms of severe Graft-versus-Host-Disease (GVHD), with none having received any prophylactic immunosuppression.

MAA submission to EMA for ATIR101 progressing well
Following the Company’s decision to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the use of ATIR101 in blood cancers, Kiadis Pharma confirms that the preparations for submitting the dossier are progressing well. The MAA will be based on the results from the Company’s successful single dose Phase II trial (CR-AIR-007) which confirmed that ATIR101 can be safely infused and shows statistically significant benefits on overall survival and reducing death from GVHD and infections, compared to an observational control group of patients having received a similar T-cell depleted stem cell transplant from a haploidentical family member but without the addition of ATIR101.

Kiadis Pharma has previously received an Advanced Therapy Medicinal Product (ATMP) certificate from EMA for manufacturing quality and pre-clinical data, recognizing that this data generated for ATIR101 meets the stringent standards imposed by the agency in evaluating an MAA.

Contingent on approval from EMA, Kiadis Pharma anticipates launching ATIR101 in to the European market in 2018.

Phase I/II trial with ATIR201
As previously announced, the Company’s Phase I/II clinical trial with its product ATIR201 for thalassemia (CR-BD-001) has received regulatory approval from the national authority in the United Kingdom (the MHRA, the Medicines and Healthcare products Regulatory Agency) as well as approval from the Ethics Committees of the Royal Manchester Children’s Hospital and the Birmingham Children’s Hospital. In addition, approval from the Ethics Committee of the University of Regensburg in Germany has now been received, with regulatory approval from the national authority in Germany pending. Kiadis Pharma expects the first clinical trial data to become available in the second half of 2017.

Manfred Rüdiger, PhD, Chief Executive Officer of Kiadis Pharma, commented: "I am very excited that we have initiated our pivotal Phase III trial with ATIR101. We have significant momentum now and the preparation of our MAA submission to EMA is progressing well. This work will bring our potentially life-saving treatment, which is also protected by Orphan Drug Designations in both Europe and the US, one step closer to patients with an anticipated launch in Europe in 2018."

About ATIR101
For patients suffering from blood cancers, an allogeneic hematopoietic stem cell transplantation (HSCT) is generally regarded as the most effective curative approach. During an HSCT treatment, the bone marrow, harboring the diseased cancer cells, is completely destroyed and subsequently replaced by stem cells in the graft from a healthy donor. After an HSCT treatment it usually takes the patient at least six to twelve months to recover to near-normal blood cell levels and immune cell functions. During this period, the patient is highly vulnerable to infections caused by bacteria, viruses and fungi but also to disease relapse.

ATIR101 (Allodepleted T-cell ImmunotheRapeutics) provides for a safe donor lymphocyte infusion (DLI) from a partially matched (haploidentical) family member without the risk of causing severe Graft-versus-Host-Disease (GVHD). The T-cells in ATIR101 will help fight infections and remaining tumor cells and thereby bridge the time until the immune system has fully re-grown from stem cells in the transplanted graft.

In ATIR101, T-cells that would cause GVHD are eliminated from the donor lymphocytes using Kiadis Pharma’s photodepletion technology, minimizing the risk of GVHD and eliminating the need for prophylactic immune-suppression. At the same time, ATIR101 contains potential cancer killing T-cells from the donor that could eliminate residual cancer cells and help prevent relapse of the disease, known as the Graft-versus-Leukemia (GVL) effect.

Therefore, ATIR101, administered as an adjunctive immuno-therapeutic on top of HSCT, provides the patient with functional, mature immune cells from a partially matched family donor that can fight infections and tumor cells but that do not cause GVHD. ATIR101 thus has the potential to make curative HSCT a viable option to many more patients.

The Company estimates that approximately 35% of patients who are eligible and in urgent need of HSCT will not find a matching donor in time. A partially matched (haploidentical) family donor, however, will be available to over 95% of patients.

ATIR101, consisting of donor T-cells that fight infections and residual tumor cells while not eliciting severe GVHD, is designed to result in low relapse rates and low rates of death due to infections, in the absence of severe acute GVHD.

On February 2, 2017 TapImmune, Inc. (NASDAQ: TPIV), a clinical-stage immuno-oncology company specializing in the development of innovative peptide and gene-based immunotherapeutics and vaccines for the treatment of cancer and metastatic disease, reported it has successfully completed a multi-gram scale-up and GMP manufacturing of a second clinical lot of TPIV 200, the company’s multi-epitope T-cell vaccine targeting folate receptor alpha (Press release, TapImmune, FEB 6, 2017, View Source;orderby=date&order=DESC [SID1234517648]). The manufactured vaccine product will be used to supply an ongoing Phase 2 study of TPIV 200 for the treatment of platinum-sensitive ovarian cancer, as well as a planned Phase 2 study sponsored by the Mayo Clinic for treating triple-negative breast cancer.

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"The successful release of our second lot of TPIV 200 represents another important milestone in the progression of our product pipeline and technologies," said Dr. Glynn Wilson, Chairman and CEO of TapImmune. "Improvements to the manufacturing process include a process change to improve scalability and a formulation change to improve the physical appearance and consistency of the final vialed product. The end result is a superior formulation that is more amenable to large scale manufacturing and commercialization."

"Our first TPIV 200 lot was manufactured in early 2016 to fully supply a TapImmune-sponsored Phase 2 trial evaluating the vaccine for the treatment of triple-negative breast cancer as well as a Phase 2 trial evaluating the vaccine in combination with a checkpoint inhibitor for platinum-resistant ovarian cancer, both of which are currently enrolling patients," said Dr. John Bonfiglio, President and COO of TapImmune. "The current, larger clinical batch of TPIV 200 will fully supply the first TapImmune-sponsored Phase 2 trial in platinum-sensitive ovarian cancer, for which a number of clinical sites are currently being screened and initiated. The batch will also supply a planned Mayo Clinic-sponsored Phase 2 trial for triple-negative breast cancer, which is fully funded by a grant from the Department of Defense."

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.

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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

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.