European Commission Grants Marketing Authorization for Gilead’s Zydelig® (Idelalisib) for the Treatment of Chronic Lymphocytic Leukemia and Follicular Lymphoma

On September 19, 2014 Gilead Sciences reported that the European Commission has granted marketing authorization for Zydelig (idelalisib), 150 mg tablets, a first-in-class oral treatment for two incurable blood cancers – chronic lymphocytic leukemia (CLL) and follicular lymphoma (FL) (Press release Gilead Sciences, SEP 19, 2014, View Source [SID:1234500758]). For the treatment of CLL, Zydelig has been approved for use in combination with rituximab for patients who have received at least one prior therapy; or as first-line treatment in the presence of 17p deletion or TP53 mutation in patients unsuitable for chemo-immunotherapy. For the treatment of FL, Zydelig has been approved as a monotherapy in patients who are refractory to two prior lines of treatment. Zydelig inhibits PI3K delta, a protein that is overexpressed in many B-cell malignancies and plays a role in the viability, proliferation and migration of these cancer cells.

CLL and FL are slow-growing incurable blood cancers that can lead to life-threatening complications such as anemia, serious infection and bone marrow failure requiring treatment. The goal of therapy for patients with these cancers is to improve overall survival and quality of life.

“Although chemo-immunotherapy is initially used to treat both CLL and FL, relapse is common and many patients run out of treatment options to treat the disease as it progresses,” said Peter Hillmen, PhD, Professor of Experimental Haematology and Honorary Consultant Haematologist at Leeds Teaching Hospitals NHS Trust. “Further, CLL patients with the 17p deletion or TP53 mutation are not suitable for chemo-immunotherapy, requiring alternative first-line treatment options. Thus, Zydelig is a welcomed treatment option that offers a new approach in the management of these cancers.”

A chromosome 17 deletion – del (17p) or a mutation in the TP53 gene in CLL cells have been linked to poor prognosis and a more rapid disease progression. For these patients most conventional chemotherapy treatments are not effective and deliver poor responses of relatively short duration. Treatment options are limited for these patients.

“Zydelig represents an important therapeutic advance for patients living with CLL and FL,” said John C. Martin, PhD, Chairman and Chief Executive Officer, Gilead Sciences. “Gilead is pleased to be making a difference in the lives of people living with these blood cancers and we are committed to helping ensure timely access to the treatment for patients who may benefit from therapy.”

The approval of Zydelig in CLL is supported primarily by data from a randomized, placebo-controlled Phase 3 trial (Study 116) of Zydelig plus rituximab in 220 patients with relapsed CLL who were not able to tolerate standard chemotherapy. Study 116 was stopped early in October 2013 by an independent Data Monitoring Committee due to a highly statistically significant benefit in progression-free survival (PFS) in the Zydelig plus rituximab arm compared with the rituximab only treatment arm (hazard ratio = 0.18 (95 percent CI: 0.10, 0.32), p<0.0001). Median PFS was not reached in the Zydelig plus rituximab arm (95 percent CI: 10.7 months, NR) and was 5.5 months in the placebo plus rituximab arm (95 percent CI: 3.8, 7.1). The approval in FL, the most common type of indolent non-Hodgkin lymphoma (iNHL), is supported by data from a single-arm Phase 2 study (Study 101-09) of Zydelig monotherapy in 125 iNHL patients refractory to rituximab and alkylating-agent-containing chemotherapy. In the 72 patients with FL in this study, Zydelig achieved an overall response rate of 54 percent and the median duration of response was not reached (range: 0.0, 14.8+ months). Results of Study 116 and Study 101-09 were published in The New England Journal of Medicine in March 2014. Adverse drug reactions (including Grade ≥3) reported in clinical studies in patients with hematological malignancies receiving Zydelig included infections, neutropenia, pneumonitis, diarrhea/colitis, increased transaminase (indicator of liver function), rash and pyrexia. For additional safety information, see the Summary of Product Characteristics at www.ema.europa.eu.

Spectrum Pharmaceuticals Out-Licenses Rights for Greater China to CASI Pharmaceuticals for Three of Its Drugs

On September 18, 2014 Spectrum Pharmaceuticals, Inc. (Nasdaq: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, and CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company dedicated to the acquisition, development and commercialization of innovative therapeutics addressing cancer and other unmet medical needs for the global market with a primary focus on China, reported the signing of license agreements whereby CASI has been granted exclusive rights to two of Spectrum Pharmaceuticals’ commercial oncology drugs, Zevalin (ibritumomab tiuxetan) Injection for intravenous use and Marqibo (vinCRIStine sulfate LIPOSOME injection) for intravenous infusion, and a Phase 3 drug candidate, Captisol-EnabledTM Melphalan (CE melphalan), for development and commercialization in China, including Taiwan, Hong Kong and Macau (Press release, Spectrum Pharmaceuticals, SEP 18, 2014, View Source [SID1234533645]).

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ZEVALIN is used in the treatment of non-Hodgkin’s lymphoma (NHL) and MARQIBO is used in the treatment of acute lymphoblastic leukemia (ALL). CE melphalan has met the endpoints in a pivotal trial for use as a conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma. Spectrum plans to file a New Drug Application with the U.S. Food and Drug Administration (FDA) for CE melphalan in the second half of 2014.

CASI will be responsible for the development and commercialization of the three drugs, including the submission of import drug registration applications to regulatory authorities and conducting any confirmatory clinical studies in greater China, if and as required.

"We are delighted to see our anticancer drugs to be developed and marketed in greater China through CASI, a NASDAQ-listed Company focused on China," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "The management of CASI has a track record of successfully developing anticancer drugs in China. We are pleased to be a shareholder of CASI at this early stage of their development and look forward to CASI creating value for our shareholders as they grow. China’s pharmaceutical market is growing at a rapid pace and is already approaching second place to only the United States in the world. The greater China drug market for anticancer drugs is projected to become the world’s largest in the next decade and CASI has the opportunity to take a leading position to address these significant unmet medical needs. We are impressed with the management team at CASI and their expertise in China, and look forward to sharing in the success of our drugs in this important market."

Commenting on the transaction, Ken K. Ren, Ph.D., CASI’s Chief Executive Officer, said, "We are very excited to have entered into this transaction with Spectrum, a Company with a successful track record of developing and commercializing drugs expeditiously in the U.S. The addition of these three drugs transforms our pipeline and significantly expands our market share potential in China. Our transaction is structured rather uniquely in that the shares and note represent the purchase price to Spectrum and is in lieu of royalties and milestones normally associated with traditional licenses, thereby aligning Spectrum’s interest with our shareholders. We look forward to a productive relationship with Spectrum."

Dr. Ren added, "These drug products come with strong intellectual property protection and significant technology barriers. We are currently preparing the import drug registration applications in greater China, initially for ZEVALIN and MARQIBO, and since both drugs are approved for sale in the U.S., we anticipate that confirmatory clinical trials will be required for marketing approval in our territory. The submission of the import drug registration for CE melphalan will follow immediately after its approval by the U.S. FDA. The annual incidence in China for NHL, ALL and multiple myeloma is increasing each year with high mortality rates, it is our goal to have these innovative products available to patients in greater China as soon as possible to address these unmet medical needs, and as Spectrum expands these drugs into additional indications in the U.S., we too will apply for expanded labels in our territory."

In addition to its initial stake in CASI, Spectrum Pharmaceuticals will have certain rights to maintain its post-transaction ownership position. Spectrum Pharmaceuticals also will have the opportunity to designate a member to CASI’s board of directors. Detailed information on the transaction can be found in CASI’s Report on Form 8-K, which will be filed with the Securities and Exchange Commission.

H.C. Wainwright & Co., LLC acted as Spectrum’s advisor.

Boehringer Ingelheim and CureVac announce collaboration to develop next generation lung cancer immunotherapy

On September 18, 2014 Boehringer Ingelheim and CureVac reported jointly announce an exclusive global license and development collaboration (Press release Boehringer Ingelheim, SEP 18, 2014, View Source [SID:1234500827]). The new collaboration focuses on CureVac’s CV9202, a novel investigational therapeutic mRNA vaccine, in early clinical development for the treatment of lung cancer. Boehringer Ingelheim will start clinical investigation of CV9202 in at least two different lung cancer settings, in combination with afatinib in patients with advanced or metastatic epidermal growth factor (EGFR) mutated non-small cell lung cancer (NSCLC) and in combination with chemo-radiation therapy in patients with unresectable stage III NSCLC. Upon signature, CureVac receives EUR 35 million. Further, CureVac can achieve milestone payments of up to EUR 430 million and royalties on sales.

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This new agreement is part of Boehringer Ingelheim’s long-term commitment to delivering tomorrow’s cancer therapies through the discovery of novel treatment options with high therapeutic value for patients. The company’s oncology portfolio includes Giotrif (afatinib)*, an irreversible ErbB family blocker, approved in a number of markets, including the EU, the US and Japan for the treatment of distinct types of EGFR mutation-positive NSCLC. Boehringer Ingelheim’s oncology pipeline covers a broad range of solid tumors and haematologic malignancies (blood cancer), including two investigational compounds in Phase III clinical development: nintedanib in distinct types of NSCLC and colorectal cancer, and volasertib in acute myeloid leukemia.

"At Boehringer Ingelheim we are proud of our commitment to help improve the treatment of cancers with a high medical need. In our collaboration with CureVac, we will investigate combining existing treatments with the approach of sustained activation of the immune system. With this we hope to be able to develop new treatments and further expand our broad pipeline in lung cancer." said Professor Klaus Dugi, Chief Medical Officer, Boehringer Ingelheim.

CureVac’s mRNA-based technology represents a novel approach in cancer treatment. For the first time, mRNA could be optimised to mobilise the patient’s own immune system to fight the tumour with a specific immune response elicited through the RNActive vaccine. Cancer immunotherapy has been chosen as the "Breakthrough of the year 2013" by SCIENCE magazine. CV9202 is a combination of mRNA molecules coding for six antigens overexpressed in lung cancer, designed to induce an immune response against the tumour. CV9202 and the preceding RNActive cancer vaccine CV9201 tested in initial clinical trials by CureVac demonstrated clinical safety and activity in generating immune responses against all anti-tumour antigens.

Ingmar Hoerr, co-founder and CEO of CureVac GmbH commented: "This collaboration is extremely relevant for CureVac because, as a biotech enterprise, we rely on collaboration with strong partners for the clinical development and commercialisation of our compounds. Cancer immunotherapy represents one of the biggest innovations in cancer treatment of recent times and we are delighted to now be working with Boehringer Ingelheim. The out-licensing and clinical development of our promising therapeutic vaccine CV9202 represents the logical next step in developing this novel treatment for cancer patients and the significant commitment from Boehringer Ingelheim underscores the relevance of the mRNA technology."

Boehringer Ingelheim and CureVac announce collaboration to develop next generation lung cancer immunotherapy

On September 18, 2014 Boehringer Ingelheim and CureVac, leader in mRNA (messenger ribonucleic acid)-based drug development, reported jointly an exclusive global license and development collaboration (Press release, Boehringer Ingelheim, SEP 18, 2014, View Source [SID1234518779]). The new collaboration focuses on CureVac’s CV9202, a novel investigational therapeutic mRNA vaccine, in early clinical development for the treatment of lung cancer. Boehringer Ingelheim will start clinical investigation of CV9202 in at least two different lung cancer settings, in combination with afatinib in patients with advanced or metastatic epidermal growth factor (EGFR) mutated non-small cell lung cancer (NSCLC) and in combination with chemo-radiation therapy in patients with unresectable stage III NSCLC. Upon signature, CureVac receives EUR 35 million. Further, CureVac can achieve milestone payments of up to EUR 430 million and royalties on sales.

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This new agreement is part of Boehringer Ingelheim’s long-term commitment to delivering tomorrow’s cancer therapies through the discovery of novel treatment options with high therapeutic value for patients. The company’s oncology portfolio includes Giotrif (afatinib)*, an irreversible ErbB family blocker, approved in a number of markets, including the EU, the US and Japan for the treatment of distinct types of EGFR mutation-positive NSCLC. Boehringer Ingelheim’s oncology pipeline covers a broad range of solid tumors and haematologic malignancies (blood cancer), including two investigational compounds in Phase III clinical development: nintedanib in distinct types of NSCLC and colorectal cancer, and volasertib in acute myeloid leukemia.

Prof. Dr. Klaus Dugi
Prof. Dr. Klaus Dugi

"At Boehringer Ingelheim we are proud of our commitment to help improve the treatment of cancers with a high medical need. In our collaboration with CureVac, we will investigate combining existing treatments with the approach of sustained activation of the immune system. With this we hope to be able to develop new treatments and further expand our broad pipeline in lung cancer." said Professor Klaus Dugi, Chief Medical Officer, Boehringer Ingelheim.

CureVac’s mRNA-based technology represents a novel approach in cancer treatment. For the first time, mRNA could be optimised to mobilise the patient’s own immune system to fight the tumour with a specific immune response elicited through the RNActive vaccine. Cancer immunotherapy has been chosen as the "Breakthrough of the year 2013" by SCIENCE magazine. CV9202 is a combination of mRNA molecules coding for six antigens overexpressed in lung cancer, designed to induce an immune response against the tumour. CV9202 and the preceding RNActive cancer vaccine CV9201 tested in initial clinical trials by CureVac demonstrated clinical safety and activity in generating immune responses against all anti-tumour antigens.

Ingmar Hoerr, co-founder and CEO of CureVac GmbH commented: "This collaboration is extremely relevant for CureVac because, as a biotech enterprise, we rely on collaboration with strong partners for the clinical development and commercialisation of our compounds. Cancer immunotherapy represents one of the biggest innovations in cancer treatment of recent times and we are delighted to now be working with Boehringer Ingelheim. The out-licensing and clinical development of our promising therapeutic vaccine CV9202 represents the logical next step in developing this novel treatment for cancer patients and the significant commitment from Boehringer Ingelheim underscores the relevance of the mRNA technology."

About CureVac
CureVac, a clinical stage biopharmaceutical company from Tübingen, Germany, is pioneering the field of mRNA-based technology platforms for medical purposes with which mRNA is specifically optimised and formulated. Since 2000 the company develops novel mRNA-based cancer immunotherapies and prophylactic vaccines against infectious diseases – both under the brand RNActive. CureVac has successfully established the first GMP (good manufacturing practice) facility worldwide for the manufacture of RNA and mRNA and has pioneered mRNA-based drugs in clinical studies.

The company has successfully completed Phase I/IIa clinical studies with its RNActive cancer vaccines in prostate cancer and non-small cell lung cancer (NSCLC). Results so far have shown that mRNA-based products showed a favorable safety profile and induced immune responses including humoral and cellular, helper (both Th1 and Th2) and effector and memory responses. CureVac is currently conducting a number of clinical trials with its RNActive vaccines. A large randomised Phase IIb clinical trial in castrate resistant prostate cancer with CV9104 has been fully enrolled in December 2013. In the field of cancer immunotherapy CureVac is already collaborating with the Ludwig Cancer Research Institute to enable clinical testing of novel cancer immunotherapy treatment options.

CureVac’s RNActive technology is also used to develop prophylactic vaccines for infectious diseases. In March 2014, CureVac received the EUR 2 million Vaccine Prize from the European Commission for its RNActive vaccine technology. In particular, the jury acknowledged the fact that the RNActive vaccines represent a novel technology enabling the production of safe, efficacious and cost-effective vaccines that are protected against elevated temperature as well as inadvertent freezing. In the field of prophylactic vaccines CureVac is amongst others collaborating with Sanofi Pasteur, In-Cell-Art, DARPA and Janssen Pharmaceuticals.

For more information please visit www.curevac.com (link is external)

About Boehringer Ingelheim
The Boehringer Ingelheim group is one of the world’s 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, Boehringer Ingelheim operates globally with 142 affiliates and a total of more than 47,400 employees. The focus of the family-owned company, founded in 1885, is researching, developing, manufacturing and marketing new medications of high therapeutic value for human and veterinary medicine. In 2013, Boehringer Ingelheim achieved net sales of about 14.1 billion euros. R&D expenditure corresponds to 19.5% of its net sales.

About Boehringer Ingelheim in Oncology
Boehringer Ingelheim has a long-term commitment to deliver tomorrow’s cancer therapies by discovering and developing novel treatment options that combine ground-breaking science with the intent of high therapeutic value for patients. It runs an extensive and diverse study programme involving investigators and patients from around the world. This is supported by a significant financial investment, with the aim of developing treatments, which will make a difference to the lives of patients and their families.

The current focus of research includes compounds in signal transduction inhibition, angiogenesis inhibition and cell-cycle kinase inhibition. Boehringer Ingelheim’s first oncology compound afatinib, approved as Giotrif/Gilotrif, is available to patients with distinct types of Epidermal Growth Factor Receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC). Two investigational compounds are in Phase III clinical development: nintedanib in distinct types of NSCLC and colorectal cancer, and volasertib in acute myeloid leukemia. A growing pipeline of early-stage oncology compounds in areas such as growth/survival signaling, immunotherapy and epigenetics adds to the later stage compounds and the CV9202 RNActive vaccine will now be investigated in combination with afatinib and chemo-radiation therapy for the treatment of patients with NSCLC.

More than 400 employees around the world (including 250 highly skilled scientists in Vienna, Austria at the Boehringer Ingelheim cancer research centre) are dedicated to the discovery and development of new cancer treatments. They work with the Research Institute of Molecular Pathology (IMP) in Austria and experts in universities, hospitals and companies around the world.

CTI BioPharma And Servier Announce Exclusive License And Collaboration Agreement To Develop And Commercialize PIXUVRI

On September 17, 2014 CTI BioPharma Corp. (CTI) (NASDAQ and MTA: CTIC) and Servier reported that they have entered into an exclusive license and collaboration agreement to develop and commercialize PIXUVRI (pixantrone) in a transaction valued at up to €103.0 million (approximately $133.5 million) if all milestones are achieved (Press release, CTI BioPharma, SEP 17, 2014, View Source;p=irol-newsArticle&ID=1968777 [SID1234529057]). Under the agreement, CTI retains full commercialization rights for PIXUVRI in Austria, Denmark, Finland, Germany, Israel, Norway, Sweden, Turkey, the United Kingdom and the U.S., with Servier having exclusive rights to commercialize PIXUVRI in all other countries.

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PIXUVRI is conditionally approved in the European Union for patients with aggressive B-cell non-Hodgkin lymphoma (NHL) who failed two or three prior lines of therapy. PIXUVRI is the first monotherapy treatment option for this patient group and the only therapy licensed for third and fourth line use in aggressive B-cell NHL patients, which includes diffuse large B-cell lymphoma (DLBCL). As of this announcement, PIXUVRI was available in 11 countries and has achieved reimbursement decisions under varying conditions in England/Wales, Italy, France, Germany and the Netherlands. Under the terms of the agreement, CTI will receive an upfront payment of €14 million (approximately $18.1 million) and is eligible to receive additional sales, clinical and regulatory milestone payments, as well as royalties on sales, such royalty payments being subject to certain reductions.

"We believe Servier represents the ideal strategic partner to achieve the full potential of PIXUVRI, particularly in those regions of the world where CTI does not currently have, or plan to have a presence," said James A. Bianco, M.D., President and CEO of CTI. "Our two companies share a vision for bringing PIXUVRI to patients and believe this collaboration will not only maximize the development, commercialization and market potential of PIXUVRI, but will also help accelerate potential development expansion into new indications. We believe Servier’s development expense contributions could help us achieve a net positive contribution margin for PIXUVRI this year and profitability in 2015 and beyond."

"Servier is conducting a comprehensive chemistry and biology research program in the field of oncology with the aim to develop and bring novel effective therapies to patients with cancer," said Jean Pierre Abastado, Head of the Oncology Therapeutic Innovation Center at Servier. "In addition, Servier has entered into several scientific collaborations with Academic Institutions as well as a number of other partnerships in the field of oncology and hematology. This new partnership will nicely fit within Servier’s portfolio by bringing an immediate therapeutic solution for patients suffering from aggressive B-cell non-Hodgkin lymphoma (NHL) who failed two or three prior lines of therapy."

"This partnership around PIXUVRI will also enable Servier to build its hemato-oncology capabilities for market access and medical information in many countries, thereby preparing for the arrival of an extensive portfolio of innovative treatments that are currently in clinical development," said Pascal Touchon, Vice President Scientific Collaboration and Business development at Servier.