On December 12, 2016 IGNITE Immunotherapy Inc. (IGNITE), a new company focused on oncolytic virus vaccine design, discovery and development reported its formation (Press release, IGNITE Immunotherapy, DEC 12, 2016, View Source [SID1234518751]).

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Along with strategic collaborative partner and lead investor Pfizer Inc. (NYSE: PFE), IGNITE will focus on the discovery and development of targeted and proprietary next-generation intravenous oncolytic (cancer cell lysing) virus vaccines for the immunotherapy of cancer. These biotherapeutics may be optimized for use in combination with immune checkpoint inhibitors. IGNITE is developing a robust and proprietary vector discovery platform, Oncolytic Vaccine Evolution, to potentially discover novel vectors for use in its lytic cancer vaccine products.

Key terms of the agreement with Pfizer include Pfizer holding a 50 percent equity investment in IGNITE, Pfizer providing full research and development funding for three years, and Pfizer having an exclusive option to acquire IGNITE after the initial research program is completed. Pfizer also has two seats on IGNITE’s board of directors. Financial terms were not disclosed.

"We are excited to announce the formation of IGNITE Immunotherapy, which we hope will emerge as a leader in the oncolytic virus cancer vaccine and immunotherapy fields. Our founding scientific team has deep expertise in the fields of oncolytic virus design and development, cancer immunotherapy, gene therapy and biotech entrepreneurship", said Dr. David Kirn, co-founder and Executive Chairman of IGNITE. "I’m thrilled and honored to be working with my co-Founders and internationally-recognized scientific leaders Dr. David Schaffer and Dr. Douglas Hanahan. Together with our collaborators at Pfizer, we have highly complimentary skill sets that will help enable our success in this complex and promising field. Our mission is to create intravenous oncolytic cancer vaccines that will be safe and highly effective in combination with immune checkpoint inhibitors, which we hope will ultimately cure patients with metastatic cancers."

"This partnership with IGNITE represents a significant advancement in Pfizer’s investment in oncolytic viruses, which we believe strengthens our position as a leader in next-generation immuno-oncology," said James Merson, Ph.D., Chief Scientific Officer, Vaccine Immunotherapeutics at Pfizer and a member of IGNITE’s scientific advisory board and board of directors. "Pfizer has a strong and growing portfolio of immuno-oncology assets, and we remain committed to developing unique cancer therapies and novel combination therapies that may benefit patients around the world.

About the Senior Scientific co-Founders and Board of IGNITE Immunotherapy Inc.

David Kirn, MD: co-Founder & Executive Chairman
Dr. Kirn is a physician-scientist, biotech entrepreneur and pioneer in oncolytic virus design, research and development with over 20 years in the field. IGNITE is his 4th start-up company in the field. He is also currently co-founder, Chairman and CEO of 4D Molecular Therapeutics, an AAV gene therapy company, and adjunct Professor of Bioengineering at UC Berkeley. Dr. Kirn has led the preclinical or clinical development of over 10 oncolytic virus therapeutics, including clinical trials involving over 800 patients in first-in-man through Phase 3 trials (including products from Onyx, Jennerex & Novartis/Cell Genesys). He has co-authored over 100 publications in the field. He has degrees from UC Berkeley (BA), UCSF (MD; Clinical Research & Biostatistics), and Haas Business School at UC Berkeley, and trained in internal medicine at Harvard (Brigham & Women’s Hospital) and in oncology at UCSF.

Dave Schaffer, PhD: co-Founder & SAB co-Chair
Dr. Schaffer is a leader in viral vector gene therapy and stem cell discovery, research and development. IGNITE is his 2nd start-up company in the field. At UC Berkeley, he is Professor of Chemical and Bioengineering, and Director of the Stem Cell Center. He is also currently co-founder & acting CSO of 4D Molecular Therapeutics, an AAV gene therapy company developing his directed vector evolution discovery platform. He has co-authored over 100 publications. He has degrees from Stanford (BS) and MIT (PhD), and his post-doctoral training was at The Salk Institute with Rusty Gage.

Doug Hanahan: co-Founder & SAB co-Chair
Dr. Hanahan is a leader in cancer research with over 25 years of experience in the fields of cancer biology, mouse tumor models, tumor resistance mechanisms and experimental therapeutics (including oncolytic viruses). IGNITE is his 3rd start-up company in the field (previous SAB member at Jennerex and Onyx). He is Director of the Swiss Institute for Cancer Research (ISREC), and Professor at EPFL (Lausanne, Switzerland); he previously was on the faculty at UCSF Medical School. He has co-authored over 100 publications, including the seminal Cell paper "The Hallmarks of Cancer" with Robert Weinberg (2000 and 2011). He serves on the cancer advisory board for Pfizer. He has degrees from MIT (BS) and Harvard (PhD), and post-doctoral training at Cold Spring Harbor.
Theresa Janke: co-Founder & Board member
Ms. Janke has over 15 years of clinical research and operations, alliance and program management, and business operations start-up experience in the biopharmaceutical industry, including work in immunotherapy, gene therapy and oncolytic virus therapy. She is currently SVP of Operations and Alliance/ Program Management at 4D Molecular Therapeutics.

James Merson: Board member
James Merson, Ph.D. is Senior Vice President and Chief Scientific Officer of the Vaccine Immunotherapeutics Research Unit at Pfizer. Prior to his current role, Dr. Merson was Chief Scientific Officer of Pfizer’s Vaccine Research Unit, Head of the Antivirals Therapeutic Area, and leader of Pfizer’s first efforts into immuno-gene therapy. Dr. Merson received his B.A. in Biology from Bellarmine College in Louisville, Kentucky, and his Ph.D. in Microbiology and Immunology from Baylor College of Medicine in Houston, Texas. He is a member of the British Society for Immunology, International Society of Vaccines, and is an adjunct professor at the Scripps Research Institute.

Bob Smith: Board member
Bob Smith is Pfizer’s Senior Vice President, Gene Therapy Business and Early Commercial Development, Rare Disease, for Pfizer’s Innovative Health Business. Prior to his current role, Bob was SVP of Business Development for Pfizer’s Worldwide Research and Development organization, and SVP of Global Business Development and Mergers & Acquisitions at Wyeth. Bob received his B.S. in Neuroscience from the University of Rochester, NY, and his M.B.A. in Finance and Corporate Accounting from the William E. Simon Graduate School of Business Administration at the University of Rochester, NY.

For more information on IGNITE and its product design and discovery efforts, please visit www.igniteimmunotherapy.com.

About IGNITE Immunotherapy Inc.
IGNITE is focused on the discovery and development of targeted oncolytic virus vaccines for the intravenous immunotherapy of cancer. IGNITE’s founding team, led by Drs. David Kirn (Executive Chairman), David Schaffer (SAB co-Chair) and Douglas Hanahan (SAB co-Chair), has deep expertise in oncolytic virus design and development, cancer immunotherapy, gene therapy vector discovery, experimental cancer therapeutics and biotech entrepreneurship. Our discovery platform, termed Oncolytic Vaccine Evolution, is designed to discover optimized gene and immunotherapy delivery vehicles to target cancer cells in diverse patient populations with common metastatic tumor types. These products may be designed for intravenous administration, antibody resistance, tumor-specificity and combination efficacy with immune checkpoint inhibitors.

About IGNITE’s Oncolytic Vaccine Evolution
Oncolytic virus cancer vaccines have demonstrated promising antitumoral activity and tolerability, and the oncolytic virus IMLYGIC (Amgen; talimogene laherperepvec) was approved by the US FDA in 2015 for the local treatment of unresectable cutaneous, subcutaneous and nodal lesions in patients with melanoma recurrent after initial surgery. While oncolytic viruses represent a promising new approach to cancer immunotherapy, hurdles to this approach still exist. First, IMLYGIC and many other clinical-stage agents require direct intratumoral injection, a method with significant disadvantages versus standard intravenous (IV) infusions that are used for blockbuster cancer biotherapeutics such as monoclonal antibodies (e.g. most patients with metastatic cancer have tumor metastases that are not directly injectable in the clinic). Second, if administered IV, many of these immunotherapeutic viruses are rapidly cleared by the immune system (e.g. by antibodies and/or complement). Finally, the number of immune-activating transgenes that can be expressed from the vector is limited by these viruses’ transgene-encoding capacities. Novel oncolytic vaccine vectors are needed for the IV delivery of diverse immunostimulatory transgenes to metastatic cancers.

IGNITE Immunotherapy is advancing the field of oncolytic cancer vaccines by taking advantage of evolution to help discover vectors that are designed to efficiently and selectively target cancer cells after IV administration. Our Oncolytic Vaccine Evolution platform empowers us to potentially discover and engineer optimized and proprietary oncolytic vectors for use in cancer immunotherapy. The resulting products may be evolved and designed for intravenous infusion, resistance to immune-mediated clearance (e.g. by antibodies, complement), tumor-specific replication and cell lysis, and immune-activating transgene expression.

On December 13, 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 idelalisib (Zydelig) (Press release, MorphoSys, DEC 12, 2016, View Source [SID1234517058]). The trial, which has been named COSMOS (CLL patients assessed for ORR & Safety in MOR208 Study), is designed to evaluate the safety and efficacy of MOR208 in combination with the PI3K delta inhibitor idelalisib in adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Patient enrolled must have been refractory or shown relapse or intolerance to a prior, most recent, therapy with a Bruton’s Tyrosine Kinase (BTK) inhibitor (e.g. ibrutinib). MOR208 is an investigational Fc-engineered monoclonal antibody targeting CD19 that is being developed for the treatment of patients with B cell malignancies. CLL is the most common type of leukemia in Western populations.

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"We are pleased to kick off the COSMOS trial. This is the third in a series of clinical studies we have initated this year investigating combination therapies with our CD19 antibody MOR208 in hemato-oncological indications", commented Dr. Arndt Schottelius, Chief Development Officer of MorphoSys AG. "There is a particularly high medical need for chronic lymphocytic leukemia (CLL) patients, especially following discontinuation of a BTK inhibitor therapy. We look forward to exploring the potential of MOR208 in this indication in combination with idelalisib. In addition, we are also planning to investigate MOR208 with a second combination partner in this patient population and will provide more details soon."

The single-arm, open-label, multicenter COSMOS study will enroll patients in Europe and in the USA. Patients will receive intravenous infusions of MOR208 for up to 24 cycles of 28 days each. Idelalisib is taken orally, 150 mg twice-daily for the study duration. The study will include a safety run-in phase consisting of a safety evaluation by an independent data monitoring committee (IDMC).

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

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

About CD19
CD19 is broadly and homogeneously expressed across different B cell malignancies including DLBCL and CLL. CD19 enhances B cell receptor (BCR) signaling, which is important for B cell survival, making CD19 a potential target in B cell malignancies.

About MOR208
MOR208 (previously Xmab5574) is an Fc-engineered ("Fc-enhanced") monoclonal antibody targeting CD19. Fc-modification of MOR208 is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus possibly improving a key mechanism of tumor cell killing. Furthermore, MOR208 induces direct apoptosis by binding to CD19, which is a crucial component for B cell receptor (BCR) signaling.
MorphoSys is currently investigating MOR208 as an immunotherapeutic treatment option in several phase 2 combination studies in patients with B cell malignancies. A phase 2 study namend L-MIND (Lenalidomide-MOR208 IN DLBCL) is investigating the safety and efficacy of MOR208 in combination with lenalidomide in patients with relapsed or refractory DLBCL. A phase 2/3 study named B-MIND (Bendamustine-MOR208 IN DLBCL) is evaluating the safety and efficacy of MOR208 in combination with the chemotherapeutic agent bendamustine in comparison to rituximab plus bendamustine in patients with relapsed or refractory DLBCL. The B-MIND trial, which is currently in the safety part, is planned to be transitioned into a pivotal phase 3 part in 2017. A third trial namend COSMOS (CLL patients assessed for ORR & Safety in MOR208 Study) is investigating safety and efficacy of MOR208 together with idelalisib in patients with relapsed or refractory CLL or SLL after discontinuation of BTK inhibitor therapy.

On December 12, 2016 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and DarwinHealth reported a strategic partnership to use quantitative systems biology-based algorithms and novel, validated approaches focused on tumor checkpoints — a new class of cancer targets — to help prioritize investigational compounds in the Daiichi Sankyo Cancer Enterprise pipeline for clinical development (Press release, Daiichi Sankyo, DEC 12, 2016, View Source [SID1234517057]).

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Utilizing DarwinHealth’s proprietary, oncotecture-based technology, the vast majority of investigational compounds in the Daiichi Sankyo Cancer Enterprise portfolio will be evaluated and compared against an extensive repository of patient-derived tumor samples to measure their efficacy in disrupting the master regulator proteins that represent the most critical vulnerabilities of each specific tumor. These studies will provide a comprehensive readout of each compound’s potential clinical value, including its genome-wide mechanism of action, its tumor-specific biomarkers of sensitivity and resistance, and its ability to synergize with other drugs for combination therapy applications. Through quantitative modeling, the developmental trajectory of each potential treatment will be predicted to help Daiichi Sankyo design more effective, successful and focused clinical studies to leverage key opportunities that are currently being missed or may not be identified using traditional methodologies.

"As we begin to see quantitative science revolutionize the approach to clinical development, we are very excited about entering into this partnership with DarwinHealth," said Igor Matushansky, MD, PhD, Global Head Oncology Translational Development, Daiichi Sankyo. "These insights will help us to focus on compounds with a higher likelihood of success to ultimately ensure the best treatment options are reaching patients with cancer sooner."

"Genetics has been the basis of the development of precision medicine, but even more targeted and integrative approaches are now needed to deal with the complexity of cancer," explained Dr. Andrea Califano, Clyde and Helen Wu Professor and Chair of Systems Biology at Columbia University and co-founder of DarwinHealth. "The evolution of cancer research has expanded our universe from gene mutations to the ultimate effects these genomic alterations have on protein activity. Virtual, computation-based methods have finally achieved the accuracy necessary to systematically detect aberrantly activated proteins representing the master regulators of a cancer cell. We have created sophisticated tools, such as the VIPER algorithm, to identify tightly-knit modules of master regulator proteins – which we call tumor checkpoints – that represent a new class of actionable therapeutic targets in cancer."

On December 12, 2016 Cellectar Biosciences, Inc. (Nasdaq: CLRB) (the "company"), an oncology-focused clinical stage biotechnology company, reported that the United States Patent and Trademark Office ("USPTO") has granted patent number 9,480,754 covering the method of use for CLR 1603 to treat breast, lung, colorectal and prostate cancers as well as their associated cancer stem cells (Filing, 8-K, Cellectar Biosciences, DEC 12, 2016, View Source [SID1234517049]). CLR 1603 consists of Cellectar’s proprietary phospholipid drug conjugate ("PDC") delivery platform technology using a unique chemical linker, conjugated to the chemotherapeutic agent paclitaxel.

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This patent and the previously granted composition of matter patent provide intellectual property protection for CLR 1603 to the end of 2035. Earlier this year, the company announced that CLR 1603 showed an increased delivery of between 20 – 30 times that of paclitaxel alone to a tumor in a preclinical xenograft cancer model. Additionally, CLR 1603 did not require the use of Cremophor in its formulation, a desired feature as Cremophor has been linked to a number of serious adverse events. As a result, CLR 1603 improved formulation, combined with its targeted delivery, has the potential to significantly reduce adverse events typically associated with other paclitaxel-based therapies.

"This patent provides intellectual property protection for one of our most interesting paclitaxel PDCs," said Jim Caruso, president and CEO of Cellectar. "The chemotherapeutic conjugate program is an exciting addition to CLR 131, our lead radiotherapeutic PDC currently in a Phase I clinical study for relapsed or refractory multiple myeloma. We plan to initiate a Phase II trial for CLR 131 in patients with relapsed or refractory multiple myeloma and select hematologic malignancies in early 2017. We are enthusiastic about the potential of each of these programs and look forward to reporting continued progress."

About Phospholipid Drug Conjugates (PDCs)
Cellectar’s product candidates are built upon its patented cancer cell-targeting delivery and retention platform of optimized phospholipid ether-drug conjugates (PDCs). The company deliberately designed its phospholipid ether (PLE) carrier platform to be coupled with a variety of payloads to facilitate both therapeutic and diagnostic applications. The basis for selective tumor targeting of our PDC compounds lies in the differences between the plasma membranes of cancer cells compared to those of normal cells. Cancer cell membranes are highly enriched in lipid rafts, which are glycolipoprotein microdomains of the plasma membrane of cells that contain high concentrations of cholesterol and sphingolipids, and serve to organize cell surface and intracellular signaling molecules. PDCs have been tested in over 70 different xenograft models of cancer.

On December 12, 2016 Genmab A/S (Nasdaq Copenhagen: GEN) reported that the European Commission (EC) has granted a marketing authorization for the use of ofatumumab (Arzerra) in combination with fludarabine and cyclophosphamide (FC) for the treatment of adult patients with relapsed chronic lymphocytic leukemia (CLL) in the European Union (Press release, Genmab, DEC 12, 2016, View Source [SID1234517045]). The variation to the Marketing Authorization for this indication was submitted to the European Medicines Agency (EMA) in March 2016 by Novartis under the ofatumumab collaboration between Novartis and Genmab. Subsequently, on November 10, the Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending that Arzerra be approved in this indication.

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"We welcome this decision by the European Commission to expand the use of Arzerra, as this further broadens the treatment options for CLL patients in Europe," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The EC’s approval was based on results from the Phase III COMPLEMENT 2 study that evaluated ofatumumab in combination with FC versus FC alone in patients with relapsed CLL. Top-line results from COMPLEMENT 2 were reported in April 2015.

About CLL
CLL is the most commonly diagnosed adult leukemia in Western countries, and accounts for approximately 1 in 4 cases of leukemia.1 Most CLL patients experience disease progression despite initial response to therapy and may require additional treatment.2

About COMPLEMENT 2
COMPLEMENT 2 (NCT00824265) is an open-label, two-arm, randomized, Phase III study, which included 365 patients in 18 countries with relapsed CLL. Patients in the study were randomized 1:1 to treatment with up to six cycles of ofatumumab in combination with fludarabine and cyclophosphamide (FC) or up to six cycles with fludarabine and cyclophosphamide alone.

The primary endpoint of the study was progression free survival (PFS), which was assessed by an Independent Review Committee (IRC) according to the International Workshop for Chronic Lymphocytic Leukaemia (iwCLL) updated 2008 National Cancer Institute-sponsored Working Group (NCIWG) guidelines.3 The study met the primary endpoint with a median progression free survival in patients receiving ofatumumab in combination with FC of 28.9 months, compared to 18.8 months in patients receiving FC alone (HR =0.67, p=0.0032). Secondary endpoints included overall response rate, overall survival, patient reported outcomes, time to response, duration of response, time to progression, time to next therapy, safety assessments and quality of life. The safety profile observed in this study was consistent with other trials of ofatumumab and no new safety signals were observed.

About Ofatumumab (Arzerra)
Ofatumumab is a human monoclonal antibody that is designed to target the CD20 molecule found on the surface of chronic lymphocytic leukemia (CLL) cells and normal B lymphocytes.
In the United States, Arzerra is approved for use in combination with chlorambucil for the treatment of previously untreated patients with CLL for whom fludarabine-based therapy is considered inappropriate, for use in combination with fludarabine and cyclophosphamide for the treatment of patients with relapsed CLL, and for extended treatment of patients who are in complete or partial response after at least two lines of therapy for recurrent or progressive CLL. In the European Union, Arzerra is approved for use in combination with chlorambucil or bendamustine for the treatment of patients with CLL who have not received prior therapy and who are not eligible for fludarabine-based therapy. In more than 50 countries worldwide, Arzerra is also indicated as monotherapy for the treatment of patients with CLL who are refractory after prior treatment with fludarabine and alemtuzumab.

Under the collaboration with Novartis, a subcutaneous formulation of ofatumumab is in Phase III development for relapsing multiple sclerosis.

Please see full Prescribing Information, including Boxed WARNING for Arzerra (ofatumumab).

Arzerra is marketed under a collaboration agreement between Genmab and Novartis. Novartis has rights to develop ofatumumab in autoimmune indications, including multiple sclerosis.