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EISAI AND PIQUR SIGN LANDMARK COLLABORATION AGREEMENT TO INVESTIGATE
HALAVEN® (ERIBULIN) AND PQR309 IN HARD TO TREAT FORM OF BREAST CANCER

On February 17, 2016 Eisai and PIQUR Therapeutics reported a landmark agreement to conduct a Phase 1/2b clinical study to investigate PQR309 in combination with Halaven (eribulin) in patients with triple-negative breast cancer (TNBC) (Press release, PIQUR Therapeutics, FEB 17, 2016, View Source [SID1234527273]).

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A significant number of HER2-negative breast cancer patients are expected to have activated PI3K, de novo or induced by prior chemotherapy administration. PQR309 is currently engaged in multiple Phase 1 and Phase 2 studies as a single agent and has shown promising activity. The combination of a PI3K/mTOR inhibitor with eribulin may prove to be an effective treatment in second line therapy for locally advanced or metastatic TNBC patients.

The Phase 1/2b study is scheduled to begin in early 2016. The initial Phase 1 dose-escalation part of the study will assess the safety and tolerability of PQR309 combined with eribulin in patients with locally advanced or metastatic HER2-negative and triple-negative breast cancer. The Phase 2b expansion part of the study will enroll patients with advanced or metastatic TNBC. The primary objective of this part of the study is to evaluate the efficacy of the PQR309 in combination with eribulin. In total, the Phase1/2b study will enroll approximately 60 patients. PIQUR will be responsible for conducting the Phase 1/2b clinical trial and the parties may extend the collaboration to include a Phase 3 clinical trial as well as additional trials in new indications of mutual interest.

"We are delighted to enter into this collaboration with PIQUR Therapeutics, which has rapidly established itself as one of the leading experts in the field of PI3K/mTOR inhibitors. Based on convincing pre-clinical data and on the promising results of PIQUR’s front runner compound PQR309 shown in Phase 1 study, we believe that combining eribulin with PQR309 may provide new treatment options for patients with TNBC," said Dr Takashi Owa, Chief Innovation Officer, Vice President Eisai Co., Ltd.

"We are very pleased to enter into this collaboration with Eisai, one of the worldwide leaders in oncology and to jointly explore new avenues to address an unmet need in breast cancer. We look forward to a fruitful partnership and to pursue the development of this combination treatment following the completion of this first study," said Hervé Girsault, Chief Business Officer of PIQUR Therapeutics.

Eribulin is the first in the halichondrin class, microtubule dynamics inhibitors with a novel mechanism of action. Structurally eribulin is a simplified and synthetically produced version of halichondrin B, a natural product isolated from the marine sponge Halichondria okadai. Eribulin is believed to work by inhibiting the growth phase of microtubule dynamics which prevents cell division.1 Eribulin remains the only single agent chemotherapy to significantly improve overall survival in women with advanced breast cancer after anthracycline and taxane treatment. Metastatic breast cancer is a very difficult condition to treat and only 15% of women will survive beyond five years.2

This collaboration underscores Eisai’s human health care (hhc) mission, the company’s commitment to innovative solutions in disease prevention, cure and care for the health and well-being of people worldwide. Eisai is committed to oncology to address the unmet medical needs of patients and their families.

Ipsen enters into a licensing agreement with 3B Pharmaceuticals to develop novel radiopharmaceuticals in oncology

On February 17, 2016 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven biotechnological group, and 3B Pharmaceuticals GmbH (3B Pharmaceuticals), a German private life sciences company focusing on targeted radiopharmaceutical drugs and diagnostics for oncology indications, reported the signature of an exclusive license agreement for novel radiopharmaceuticals in oncology (Press release, Ipsen, FEB 17, 2016, View Source [SID:1234509084]).

Ipsen acquires exclusive worldwide rights to develop and commercialize novel radiopharmaceuticals targeting the neurotensin receptor. Ipsen will focus on the development of the lead program currently in preclinical development for the treatment of pancreatic adenocarcinoma and potentially other oncology indications.

Claude Bertrand, Executive Vice president R&D, Chief Scientific Officer at Ipsen stated: "Ipsen is very pleased to enter into an agreement with 3B Pharmaceuticals. This program is fully aligned with our strategy to strengthen our pipeline in niche oncology indications as well as in the field of radiopharmaceuticals following our recent acquisition of OctreoPharm GmbH."

Ulrich Reineke, Managing Director of 3B Pharmaceuticals, added: "We are very pleased to enter into this agreement with Ipsen, a company with a strong commitment to radiopharmaceuticals. This agreement emphasizes the growing importance of nuclear medicine in targeted cancer therapy, and we hope that the neurotensin receptor-targeting radiopharmaceuticals will improve the lives of seriously ill cancer patients."

Under the financial terms of the agreement 3B Pharmaceuticals will receive a licensing upfront payment and is eligible to receive development and regulatory milestones of up to 82 million Euros for several indications as well as tiered royalties on world-wide annual net sales of products developed and commercialized by Ipsen.

bluebird bio Announces First Patient Treated with bb2121 in CRB-401 Phase 1 Study in Patients with Relapsed/Refractory Multiple Myeloma

On February 17, 2016 bluebird bio, Inc. (Nasdaq:BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported treatment of the first patient in a Phase 1 study of its product candidate bb2121 in patients with relapsed/refractory multiple myeloma (Press release, bluebird bio, FEB 17, 2016, View Source [SID:1234509080]).

bb2121 is a chimeric antigen receptor T cell (CAR T) therapy targeting B cell maturation antigen (BCMA), and bluebird bio is developing bb2121 in collaboration with Celgene Corporation. bluebird bio also announced today that Celgene has exercised its option to exclusively license bb2121, under the terms of the collaboration agreement between the two companies.

"bb2121 is bluebird bio’s first oncology program to enter the clinic, and the treatment of this first patient marks an important milestone for us as we build a broad, fully integrated T cell immunotherapy franchise," said Nick Leschly, chief bluebird. "We are pleased that Celgene has exercised their option to license bb2121. We believe our combined manufacturing, development and commercial expertise will enable us to rapidly advance bb2121 through clinical trials."

"Despite many recent advances in the field, multiple myeloma remains incurable, with almost all patients becoming refractory to therapy eventually," said James N. Kochenderfer, M.D., National Cancer Institute, an investigator for the CRB-401 study. "BCMA is one of the most exciting targets in multiple myeloma, and we are eager to explore the potential of bb2121 to become an important new treatment option for patients living with multiple myeloma."

bluebird bio and Celgene amended and restated their collaboration agreement in June 2015 to focus on developing product candidates targeting BCMA during a three-year collaboration term. By exercising its exclusive option under the terms of the agreement, Celgene will be responsible for worldwide development and commercialization of bb2121 after Phase 1. bluebird bio is responsible for the development of bb2121 through the completion of the CRB-401 Phase 1 study and has an option to share in the development, promotion and profits in the United States. bluebird bio will receive a $10 million option exercise payment from Celgene, and bluebird bio is also eligible to receive specified development, regulatory and commercial milestone payments and royalty payments on net sales.

About the CRB-401 Study

The primary objective of the CRB-401 study is to evaluate the maximum tolerated dose of bb2121 and determine the recommended Phase 2 dose. The secondary objective is patient response, measured using the International Myeloma Working Group (IMWG) Response Criteria for Multiple Myeloma. The first portion of the study includes a dose-escalation phase in which cohorts of patients will receive ascending doses of bb2121 to determine the maximum tolerated dose and establish a recommended Phase 2 dose. The second portion of the study is a dose expansion phase where patients will receive bb2121 to further evaluate the safety, tolerability and clinical activity at the recommended Phase 2 dose.

Durvalumab granted Breakthrough Therapy designation by US FDA for treatment of patients with PD-L1 positive urothelial bladder cancer

On February 17, 2016 AstraZeneca and MedImmune, its global biologics research and development arm, reported that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) for durvalumab (MEDI4736), an investigational human monoclonal antibody directed against programmed death ligand-1 (PD-L1), for the treatment of patients with PD-L1 positive inoperable or metastatic urothelial bladder cancer whose tumour has progressed during or after one standard platinum-based regimen (Press release, AstraZeneca, FEB 17, 2016, View Source [SID:1234509073]).

Robert Iannone, Senior Vice President, Head of Immuno-Oncology, Global Medicines Development at AstraZeneca said: "Metastatic bladder cancer is an area of enormous unmet medical need. We are encouraged by this Breakthrough Therapy designation. We look forward to working closely with the FDA to bring durvalumab to bladder cancer patients as soon as possible."

The Breakthrough Therapy designation is designed to expedite the development of new drugs which are intended to treat a serious condition and which have shown encouraging early clinical results, which demonstrate substantial improvement on a clinically significant endpoint over available therapies or when there is significant unmet medical need.

The Breakthrough Therapy designation for durvalumab was granted by the FDA on the basis of early clinical data from a Phase I trial (Study 1108) in patients with advanced metastatic urothelial bladder cancer whose tumour has progressed during or after one standard platinum-based regimen. This represents the third Breakthrough Therapy designation AstraZeneca has received from the FDA for medicines in Oncology. This designation offers the opportunity for further collaboration with the FDA for the durvalumab development programme. Data from study 1108 have been submitted for presentation at a future medical meeting.

Durvalumab is also being tested in first-line bladder cancer as a monotherapy as well as in combination with tremelimumab as part of the DANUBE Phase III trial which achieved first patient in during the final quarter of 2015.

NOTES TO EDITORS

About bladder cancer

Urothelial bladder cancers arise from the epithelium of the bladder and are the 9th most common form of cancer worldwide. Based on the Global Burden of Disease Cancer Collaboration, it is estimated that there were 400,000 incidents of bladder cancer and 173,000 deaths worldwide for the year 2013. Metastatic bladder cancer remains an area of great unmet medical need with 5-year overall survival rates of less than 15%.

About durvalumab (MEDI4736)

Durvalumab is an investigational human monoclonal antibody directed against programmed death ligand-1 (PD-L1). PD-L1 can be expressed by tumours to evade detection by the immune system through binding to PD-1 on cytotoxic T lymphocytes. Durvalumab blocks the PD-L1 interaction with PD-1, countering the tumour’s immune-evading tactics. Durvalumab is being developed, alongside other immunotherapies, to empower the patient’s immune system and attack the cancer. Durvalumab is being investigated in an extensive clinical trial programme, as monotherapy or in combination with tremelimumab, in NSCLC, head and neck, bladder, gastric, pancreatic, HCC and blood cancers.

BIND Therapeutics and Synergy Pharmaceuticals Announce Collaboration to Develop ACCURINS® with Proprietary Uroguanylin Analogs for Targeting Gastrointestinal Receptors on Tumors

On February 16, 2016 BIND Therapeutics, Inc. and Synergy Pharmaceuticals Inc. reported they have entered into a research collaboration to engineer ACCURINS incorporating Synergy’s proprietary uroguanylin analogs to explore the potential targeting of guanylate cyclase-C (GC-C) receptors expressed on tumors, specifically GI malignancies (Press release, BIND Therapeutics, FEB 16, 2016, View Source [SID:1234513873]). Upon achievement of proof-of-concept, the companies anticipate expanding the collaboration to enhance the potential effect of uroguanylin-based ACCURINS by incorporating therapeutic payloads.

"This collaboration represents another important advance as we develop ACCURINS with the potential to target a broad range of cells with novel therapeutic payloads," said Jonathan Yingling, Ph.D., chief scientific officer, BIND Therapeutics. "The versatility of our platform is demonstrated by our wide range of collaborations and the ability to leverage our technology platform in a wide variety of applications. We believe the complementary technologies of Synergy and BIND have the potential to generate novel uroguanylin-based therapies that could have a profound impact on the treatment of diseases."

Uroguanylin is a naturally occurring GI peptide and activator of the intestinal GC-C receptor, which is a known target for stimulating a variety of physiological responses. Plecanatide, Synergy’s lead uroguanylin analog for functional GI disorders, successfully completed phase 3 clinical trials for chronic idiopathic constipation (CIC) in 2015, and the Company recently filed a new drug application (NDA) for plecanatide to treat CIC. Synergy also recently announced positive data on its second uroguanylin analog, dolcanatide, in a phase 1b proof-of-concept study in ulcerative colitis patients.

"We are pleased to collaborate with BIND Therapeutics on this project, which we believe can broaden the potential therapeutic applications of our proprietary uroguanylin-based platform," said Kunwar Shailubhai, Ph.D., M.B.A., chief scientific officer and executive vice president of Synergy Pharmaceuticals. "BIND’s ACCURIN technology has been shown to target diseased tissues with a wide variety of therapeutic payloads. We have separately presented data showing that uroguanylin analogs specifically bind to different types of colorectal polyps and tumor in mice. Together, we believe that we can create uroguanylin-based ACCURINS that have the potential to provide new treatment options in GI cancer."

This early research collaboration is not expected to have a material financial impact on Synergy Pharmaceuticals or BIND Therapeutics.

About ACCURINS
ACCURINS, a new class of targeted therapeutics developed using BIND’s Medicinal Nanoengineering platform, are nanoparticles engineered to have a profound impact on the treatment of disease. The elegant and novel design of ACCURINS allow for prolonged circulation, controlled and tunable release and selective targeting of a therapeutic payload to diseased tissue or cells while avoiding immune surveillance detection and systemic toxicities.

ACCURINS can be engineered for multiple therapeutic applications and have the potential to integrate numerous payloads, including highly potent drugs with mechanism-based toxicities that limit therapeutic benefit, DNA, RNA, proteins and immunotherapy agents. This attribute enables ACCURINS to target multiple diseases, including cancer, inflammatory, vascular, and infectious disease.