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Arrowhead Research Changes Name to Arrowhead Pharmaceuticals

On April 6, 2016 Arrowhead Research Corporation (NASDAQ:ARWR) reported that it has changed its corporate name to Arrowhead Pharmaceuticals, Inc (Press release, Arrowhead Research Corporation, APR 6, 2016, View Source [SID:1234510482]). The company’s common stock will continue to trade on the NASDAQ Global Select Market under the existing stock ticker symbol, ARWR. These shares have been assigned a new CUSIP number of 04280A100. Holders of stock certificates with the prior corporate name, need not take any action.

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As part of this name change, the company has also launched a new corporate website at www.arrowheadpharma.com and a new Twitter handle @ArrowheadPharma.

Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals, said: "Now is the time to acknowledge that our key priorities are increasingly focused on advancing products through clinical development to bring innovative new medicines to patients. The name Arrowhead Pharmaceuticals is more consistent with our stage of development as a company and reflects the great progress we’re making on our broad pipeline of RNAi-based drugs."

Janssen Enters Worldwide Collaboration and License Agreement with TESARO, Inc., for Niraparib in Prostate Cancer

On April 6, 2016 Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, reported it has entered a worldwide collaboration and license agreement with TESARO, Inc., for exclusive rights to the investigational compound niraparib in prostate cancer (Press release, Johnson & Johnson, APR 6, 2016, View Source [SID:1234510457]). Niraparib is an orally administered poly polymerase (PARP) inhibitor, currently in late-stage development for patients with metastatic breast cancer and ovarian cancer.

According to terms of the license agreement and collaboration arrangement, Janssen will have global rights and be responsible for all development and commercialization activities for niraparib for use in prostate cancer, except in Japan. TESARO will maintain global development, manufacturing and commercial rights for all other indications.

In addition to an upfront payment, TESARO will be eligible to receive milestone payments, based upon the achievement of specified development, regulatory and commercial milestones, as well as royalties on future worldwide sales.

Separate to the exclusive license and collaboration arrangement, Johnson & Johnson Innovation – JJDC, Inc. will make an equity investment in TESARO.

"PARP inhibitors are an exciting, emerging class of medicines in prostate cancer, and we believe niraparib will perfectly complement our existing portfolio," said Peter F. Lebowitz, M.D., Ph.D., Oncology Therapeutic Area Head, Janssen Research & Development, LLC. "Our team is eager to apply its prostate cancer expertise to niraparib, and enthusiastic about its potential to expand our impact on the lives of men with this disease."

PARP proteins play a key survival role in DNA repair in cancer cells. By inhibiting PARP, certain defective cancer cells are not able to repair themselves, leading to cell death. A portion of men with prostate cancer have these defective cancer cells and may benefit from use of a PARP inhibitor, either alone, or in combination with other treatments.

BIND Therapeutics Reports Phase 2 Data from iNSITE 1 and iNSITE 2 Trials with BIND-014 and Provides Strategic Update

On April 6, 2016 BIND Therapeutics, Inc. (NASDAQ: BIND), a biotechnology company developing targeted and programmable therapeutics called ACCURINS, reported preliminary top-line results from the BIND-014 (PSMA-targeted docetaxel nanoparticles) phase 2 iNSITE 1 trial in advanced non-small cell lung cancer (NSCLC) of squamous histology and the iNSITE 2 trial in cervical and head and neck cancers (Press release, BIND Therapeutics, APR 6, 2016, View Source [SID:1234510456]).

In the iNSITE 1 trial, for the primary endpoint, BIND-014 demonstrated a 52.5 percent 6-week disease control rate (6wDCR) for the intent-to-treat population (n=40) and a 70.0 percent 6wDCR in the per protocol population (n=30), which exceeded the protocol defined criteria for success of 65 percent. BIND intends to seek licensing or collaboration opportunities for further development of BIND-014 in NSCLC. In the first stage of the iNSITE 2 trial, for the primary endpoint, BIND-014 demonstrated an objective response rate of 10 percent in the head and neck cancer cohort (n=20); there were no objective responses in the cervical cancer cohort (n=23). Based on these results, BIND has decided to halt further enrollment in the iNSITE 2 trial in advanced cervical and head and neck cancers. Additional details from the iNSITE 1 and iNSITE 2 trials are listed below and the Company expects to present further details at an upcoming medical meeting.

Data from these trials suggest that BIND-014 continues to provide meaningful improvements in safety and tolerability, and at least similar efficacy when administered at a 20 percent lower dose in comparison to historical studies with docetaxel. As a result, the Company believes that these new data further validate the potential of the ACCURINS platform.

"While the single-arm design of these trials precludes definitive conclusions, we remain especially intrigued by the iNSITE 1 data in squamous histology non-small cell lung cancer patients," said Hagop Youssoufian, M.D., chief medical officer, BIND Therapeutics. "We believe these data provide additional clinical validation that ACCURINS successfully widen the therapeutic window of conventional docetaxel. Taken together, we believe these data justify further development of BIND-014 in clinical settings where improved safety and tolerability may be valuable to patients."

Following these results and in connection with the previously announced shift in research and development strategy, BIND also announced a restructuring plan designed to streamline operations and reduce the Company’s operating expenses. The restructuring will include a workforce reduction of 38 percent, and is expected to be substantially complete by the end of April 2016, after which the Company will have 61 employees. In addition, BIND will evaluate options for the Company’s wholly owned subsidiary in Moscow. Collectively, these actions are expected to bring BIND’s quarterly cash burn rate to approximately $6 million per quarter by the third quarter of 2016.

"This workforce reduction is a necessary action to bring our operating costs to a more sustainable level, allowing the ongoing development of our pipeline of innovative therapeutic candidates that address challenges small molecule chemistry or antibody engineering have not been able to overcome," said Andrew Hirsch, president and chief executive officer, BIND Therapeutics. "We truly appreciate the efforts of all of the employees affected by this action and thank them for their dedicated service and contributions to the development of our ACCURINS technology. Furthermore, our decision to continue development of BIND-014 with a collaborator was based on the fact that recent advances in the treatment paradigm for second-line solid tumors have shifted the development, regulatory and reimbursement pathway for BIND-014 such that the capital and resources required to execute on this strategy are more appropriate in collaboration with a larger company."

In addition to the workforce reduction initiatives, the Company is actively working with an investment bank to initiate a review of financial and strategic alternatives with the goal of maximizing stockholder value. Potential alternatives to be explored and evaluated during the review process may include raising additional capital, a strategic collaboration with one or more parties, or the licensing, sale or divestiture of some of the Company’s proprietary technologies. Pending a decision to undertake any financial or strategic alternatives, BIND is continuing its development and collaboration activities in accordance with its current innovative medicines strategy while managing its cash position. There is no finite timetable for completion of the financial and strategic review process.

Shift in Research & Development Focus
In December 2015, BIND announced the appointment of Jonathan Yingling, Ph.D., to the role of chief scientific officer. BIND believes Dr. Yingling has provided the scientific expertise necessary to accelerate BIND’s transformation from a technology platform business focused on improving the therapeutic index of chemotherapeutics to an innovative medicines company focused on leveraging the unique advantages of ACCURINS to maximize clinical benefit for patients. As a result, BIND recently announced a shift in its research and development efforts to focus on developing innovative medicines that incorporate unique combinations of novel tumor-directed targeting ligands and new classes of payloads, including oligonucleotides and molecularly targeted therapies. BIND’s innovative medicines strategy aims to create nanoparticles with synergistic properties by leveraging three differentiating attributes of the ACCURINS technology platform:

ACCURINS can potentially be functionalized with ligands that elicit a biological response and enhance disease tissue accumulation;
ACCURINS are able to incorporate therapeutic payloads with diverse physical and chemical properties, including highly charged APIs; and
ACCURINS can be engineered to optimize the efficacy of the encapsulated drug by controlling its release rate.
"We’ve made significant advances in the ACCURINS technology since BIND-014 was developed in 2009 and we are leveraging those advances to accelerate our new innovative medicines pipeline," said Jonathan Yingling, Ph.D., chief scientific officer at BIND. "In addition to ongoing collaborations that have resulted in molecularly targeted kinase inhibitor ACCURINS, one of which is in phase 1 trials, several new product concepts are being pursued internally. We believe our ACCURINS technology has the potential to create a pipeline of products that elicit tumor cell death, modulate the tumor microenvironment, or both, as a means to maximize clinical benefit for patients."

The Company currently has one kinase inhibitor Accurin, AZD2811, in phase 1 clinical trials in collaboration with AstraZeneca. A second kinase inhibitor Accurin is currently in IND (investigational new drug) -enabling activities through a collaboration with Pfizer. Following optimization of lead product candidates and completion of preclinical studies, BIND anticipates initiation of clinical testing for one or more proprietary innovative product candidates as early as 2018.

iNSITE 1 in squamous histology NSCLC
The Company’s decision to pursue additional development of BIND-014 through a collaboration or licensing opportunity follows an analysis of 40 patients in the intent-to-treat (ITT) population and 30 patients in the per-protocol (PP) population with squamous histology NSCLC.

ITT [n=40] PP[n=30]
6-week Disease Control Rate
(6wDCR) 52.5%
[36.1-68.5]
70.0%
[50.6-85.3]
6wDCR ± 7 days* 70.0%
[53.5-83.4]
86.7%
[69.3-96.2]
Overall Response Rate (ORR) 10.0%
(1 confirmed, 3 unconfirmed)
13.3%
(1 confirmed, 3 unconfirmed)

*Per the study protocol, scans were performed every 6 weeks after Cycle 1 Day 1 and had a ± 7 day allowance. The primary endpoint was strictly defined as disease control rate of 6 weeks or greater which had the effect of excluding patients whose data was gathered within the 7 day allowance of the 6 week timepoint. As a result, patients whose first post-treatment scan occurred between week 5 and week 6 were excluded from the primary analysis. Including these patients in a sensitivity analysis results in an 86.7 percent response rate in the PP population. While the study protocol does not permit the inclusion of these patients in the primary endpoint, the Company believes these data are meaningful.

iNSITE 2 in advanced cervical and head and neck cancers
The primary endpoint of the two-stage iNSITE 2 trial was ORR with a target of at least one response in the first 20 patients for advancement from stage 1 to stage 2. In the head and neck cancer cohort (20 evaluable patients), the ORR was 10 percent; there were no responses in the cervical cancer cohort (23 evaluable patients). Based on these results, the Company has decided to halt further enrollment in the iNSITE 2 trial in advanced cervical and head and neck cancers.
Safety data in more than 300 patients treated with BIND-014 to date continue to demonstrate meaningful improvements in hematologic and non-hematologic toxicities when compared to historical docetaxel data.

MorphoSys Initiates Phase 2 Combination Trial of MOR208 and Lenalidomide in Patients with Relapsed or Refractory Diffuse Large B Cell Lymphoma (DLBCL)

On April 6, 2016 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) reported that the first patient was dosed in a phase 2 combination trial of MOR208 with lenalidomide (Revlimid) (Press release, MorphoSys, APR 6, 2016, View Source [SID:1234510438]).

The trial, which has been named L-MIND (Lenalidomide-MOR208 IN DLBCL), is designed to evaluate the safety and efficacy of MOR208 in combination with the immunomodulatory drug lenalidomide in adult subjects with relapsed or refractory diffuse large B cell lymphoma (DLBCL), the most common form of non-Hodgkin’s lymphoma (NHL). MOR208 is a potent anti-CD19 antibody with a proprietary modification to the Fc portion, and is being developed to treat B cell malignancies.

The single-arm, open-label, multicenter L-MIND study is expected to enroll 80 patients in 56 centers in 9 European countries and the USA. At the time of study entry, patients must present with relapsed or refractory DLBCL, which had previously been treated with at least one and not more than two prior lines of therapy, including one anti-CD20 targeting therapy (e.g. rituximab). Patients must not be candidates for high-dose chemotherapy with autologous stem cell transplantation.

Patients will receive weekly intravenous infusions of 12mg/kg MOR208 for 12 weeks, followed by administration every second week for up to 2 years or until disease progression or unacceptable toxicity, whichever comes first. In addition to MOR208, lenalidomide will be administered orally, for up to one year.

The study’s primary end point is overall response rate (ORR), comprising complete responses (CR) and partial responses (PR). Secondary outcome measures include duration of response (DoR), progression-free survival (PFS) and overall survival (OS), as well as an evaluation of the drug combination’s safety and pharmacokinetic parameters of MOR208.

"We are pleased to kick off the L-MIND trial as the first in a series of planned clinical studies evaluating combination therapies with MOR208 in hemato-oncological indications with high medical need. MOR208 has already demonstrated very encouraging single-agent activity and was well tolerated by patients in our phase 2a NHL trial as presented at the ASH (Free ASH Whitepaper) 2015 conference in December 2015. In addition, an ongoing Phase 2 investigator-initiated trial has already shown good preliminary safety and activity of the combination of MOR208 and lenalidomide in patients suffering from chronic lymphocytic leukemia (CLL). These recent findings encourage us to explore the therapeutic potential of MOR208 in different combinations", said Dr. Arndt Schottelius, Chief Development Officer of MorphoSys AG.

Detailed information on the trial can be found at clinicaltrials.gov.

About MOR208:
MOR208 is an Fc-enhanced antibody targeting CD19, a more widely and earlier expressed target across multiple lymphomas and leukemias than CD20, the therapeutic target of the most commonly used lymphoma and leukemia targeted treatments. MOR208 has been engineered to possess significantly enhanced antibody-dependent cell-mediated cytotoxicity (ADCC), thus improving a key mechanism for tumor cell killing. By targeting CD19 and being Fc-enhanced, MOR208 could therefore become an important and attractive alternative to multiple current treatment options for some of the sickest cancer patients.

Updated interim data, presented at the 2015 annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (ASH 2015) in December 2015, summarize efficacy and safety results for MOR208 monotherapy in 92 heavily pre-treated patients. The overall response rate was 28% across all four subtypes of non-Hodgkin’s lymphoma (NHL) and reached 36% in the diffuse large B cell lymphoma (DLBCL) subgroup (both based on evaluable patients). At the time of the analysis, several responders – 9 out of 21 – had an ongoing response to the single-agent treatment. The longest response duration observed so far exceeded 20 months in both DLBCL and follicular lymphoma (FL).

AstraZeneca to showcase clinical and scientific leadership in lung cancer at ELCC 2016

On April 6, 2016 AstraZeneca reported that it will report new clinical trial and scientific data from their industry-leading lung cancer franchise of marketed and pipeline medicines at the European Lung Cancer Conference (ELCC) together with its global biologics research and development arm, MedImmune, in Geneva, Switzerland, 13 -16 April, 2016 (Press release, AstraZeneca, APR 6, 2016, View Source [SID:1234510436]).

Fifteen abstracts will be presented at the meeting including eight oral presentations, two "best abstracts" and two "late-breakers". Five abstracts have been selected for the official press programme. Highlights will reinforce the potential of Tagrisso (osimertinib) for the treatment of specific types of advanced non-small cell lung cancer (NSCLC), compare plasma testing and tumour tissue biopsy for treatment decisions in advanced NSCLC, and demonstrate the progress of combination therapy in immuno-oncology.

Tagrisso in first- and second-line lung cancer treatment

Two oral, late-breaker presentations share data from the Tagrisso AURA studies. They build on evidence that supported the accelerated approval of Tagrisso as the first indicated treatment for epidermal growth factor receptor (EGFR) T790M mutation-positive metastatic NSCLC in the US, EU and Japan.

On Thursday 14 April, updated efficacy and safety data will be presented from two Phase I expansion cohorts exploring Tagrisso as first-line treatment for patients with EGFRm advanced NSCLC (Abstract #LBA1_PR). Phase I/II data in pre-treated patients with EGFR T790M advanced NSCLC (Abstract #LBA2_PR) will also be presented. Both presentations will feature in the ELCC press programme on Thursday 14 April.

Mondher Mahjoubi, Senior Vice President, Head of Oncology, Global Product and Portfolio Strategy at AstraZeneca, said: "ELCC’s recognition of the importance of our data for the lung cancer community is very encouraging and we are excited to present more mature data from the AURA programme for our new, first-in-class lung cancer medicine, Tagrisso. We are also reaffirming our commitment to deliver the right treatment to the right patient based on scientific research, with updates from our pioneering plasma circulating tumour DNA trials."

Plasma ctDNA testing: Delivering the right treatment to the right patient

Accurate identification of patients with tumours carrying key molecular mutations is essential for delivering next-generation targeted therapies to those most likely to benefit. Detection of plasma circulating tumour-derived DNA (ctDNA) in a simple blood test offers a minimally invasive alternative to tumour tissue biopsy, and is already available for identifying patients suitable for treatment with AstraZeneca’s Iressa.

Building on this work, the latest results from innovative research in minimally invasive plasma ctDNA analysis to identify patients with EGFRm T790M NSCLC and predict response to Tagrisso will be reported (Abstract #134O_PR and #135O_PR). Further data from the ASSESS study for EGFR mutation detection in plasma from this patient group will also be presented (Abstract #58O_PR). ASSESS is the first largescale "real-world" study comparing tumour biopsy with ctDNA testing for EGFRm in advanced NSCLC. These studies are highlighted in the ELCC press programme on Friday 15 April.

Marc Denis, Professor of Biochemistry and Molecular Biology at Nantes University Hospital, Nantes, France, said: "Plasma ctDNA testing has the potential to rapidly identify patients suitable for targeted therapy not just for lung cancer but across a wide range of tumour types. Availability of these simple blood tests may streamline diagnosis, including for patients where tumour samples are unavailable."

Immuno-oncology (IO): Combination focus

AstraZeneca has a broad programme of combination clinical trials underway in oncology and continues to explore novel combination therapies addressing the needs of difficult-to-treat patients with lung cancer. Safety and efficacy data from two exploratory trials combining immunotherapies and small-molecules will be reported at ELCC. These presentations have been designated "best abstracts" (Abstracts #57O and #136O) by the conference.

Robert Iannone, Head of Immuno-Oncology, Global Medicines Development, at AstraZeneca, said: "Durvalumab is the cornerstone of our IO portfolio and we have a rapidly-advancing development programme focused primarily on novel combinations. At ELCC, we are presenting the exceptional science behind our combination approach with abstracts describing the ongoing MYSTIC and NEPTUNE studies combining durvalumab with tremelimumab. These build on recent data showing the anti-tumour activity of this combination in patients with metastatic NSCLC irrespective of PD-L1 status, as published in The Lancet Oncology."