On July 15, 2015 Argos Therapeutics reported the pivotal phase 3 ADAPT clinical trial of AGS-003 in combination with standard targeted therapy for the treatment of metastatic renal cell carcinoma (mRCC) has reached its enrollment goal of at least 450 randomized patients (Press release, Argos Therapeutics, JUL 15, 2015, View Source [SID:1234506335]).

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"Strong partnerships and coordination across our global study base to identify eligible patients and collect tumor samples have led to successful enrollment for the largest clinical trial ever conducted in patients with newly diagnosed, unfavorable risk, synchronous metastatic RCC," said ADAPT trial co-principal investigator and lead medical oncologist Dr. Robert Figlin, the Steven Spielberg Family chair in hematology oncology and professor of medicine and biomedical sciences at the Cedars-Sinai Samuel Oschin Comprehensive Cancer Institute. "With enough patients screened with successful tumor collection to meet and exceed our target of 450 randomized patients, we look forward to shifting our full attention to the treatment phase of the study and expected data readouts in 2016."

AGS-003 is an autologous dendritic-cell based immunotherapy designed to induce a memory T-cell response specific to each patient’s unique tumor antigens. It is produced using a small sample from a patient’s own tumor and dendritic cells derived from a leukapheresis procedure. In an open-label phase 2 study, treatment with AGS-003 plus sunitinib yielded a median overall survival of more than 30 months in newly diagnosed, unfavorable risk mRCC patients.

To qualify for the ADAPT trial, patients were required to be good candidates for standard surgery and targeted drug therapy. During the enrollment process approximately 55 percent of patients who consented for tumor collection and screening for the treatment phase of the trial were found to be ineligible for treatment because of non-clear cell histology, lack of suitability to initiate standard targeted drug therapy, poor performance status, poor prognosis after surgery, a lack of evaluable metastatic disease, or other factors.

"By concluding enrollment in the ADAPT trial, we have reached an exciting milestone by demonstrating the ability to rapidly screen and collect tumor samples for more than 1,000 newly diagnosed metastatic RCC patients over the course of approximately two years," said ADAPT trial co-principal investigator and lead urologic oncologist Dr. Christopher Wood, professor of urology and deputy chairman of the department of urology, division of surgery at the University of Texas MD Anderson Cancer Center. "This would not have been possible without a strong multidisciplinary collaboration among urologists and oncologists, which positions us well to advance our evaluation of AGS-003 in addition to standard treatment through trial completion."

"The ADAPT trial is focused on a population with significant unmet needs as the expected median survival based upon International mRCC Database Consortium (IMDC) benchmarks is approximately 15 months after diagnosis, even with standard surgery and approved targeted therapies," said Doug Plessinger, vice president of clinical and medical affairs for Argos Therapeutics. "We continue to believe that the combination of standard treatment with our personalized immunotherapy has the potential to bring new hope to mRCC patients, and we look forward to the next interim data review from the ADAPT trial in the first part of 2016."

About the Arcelis Technology Platform

Arcelis is a fully personalized immunotherapy technology that captures mutated and variant antigens that are specific to each patient’s disease. It is designed to overcome immunosuppression by producing a durable memory T-cell response without adjuvants that may be associated with toxicity. The technology is potentially applicable to a wide range of different cancers, and is designed to overcome many of the manufacturing and commercialization challenges that have impeded other personalized cancer immunotherapies. The Arcelis process uses only a small tumor or blood sample and the patient’s own dendritic cells, which are collected and optimized following a single leukapheresis procedure. The proprietary process uses RNA isolated from the patient’s disease sample to program dendritic cells to target disease specific antigens. The activated, antigen-loaded dendritic cells are then formulated into the patient’s plasma and administered via intradermal injection.

On July 14, 2015 Mersana Therapeutics, Inc. and Recepta Biopharma S.A. reported that they have entered into an exclusive license agreement in which Mersana will use its proprietary Fleximer technology to develop and commercialize an immunoconjugate with the undisclosed cancer antibody licensed from Recepta (Press release, Mersana Therapeutics, JUL 14, 2015, View Source [SID1234609623]).

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Under the terms of the agreement, Recepta will provide Mersana exclusive rights to its novel monoclonal antibody to an undisclosed target, and Mersana will leverage Fleximer to develop an immunoconjugate against the target. Financial terms of the agreement include an upfront payment and subsequent payments to Recepta, which together could total $86 million plus royalties if certain development, regulatory and commercial milestones are achieved. Mersana will conduct and fund clinical development and regulatory activities. Recepta will have rights to commercialize in Brazil, while Mersana will have rights to commercialize in the rest of the world. Mersana will be eligible to receive royalties from Recepta on sales in Brazil.

"This licensing deal with Mersana follows pioneering R&D conducted by Recepta with this antibody, which was discovered by Ludwig Cancer Research, our partner and a global nonprofit research organization. It will enable the development of a novel immunoconjugate that has the potential to improve patient outcomes in oncology," said José Fernando Perez, PhD, Chief Executive Officer of Recepta.

"We are excited to develop a Fleximer-based immunoconjugate with this antibody to address unmet needs in cancer," said Anna Protopapas, President and Chief Executive Officer of Mersana. "This will expand our pipeline of oncology therapies that address the limitations of currently available antibody-drug conjugates and complement our objective to pursue one IND each year, starting with XMT-1522 later this year."

(Filing, 10-K, Peregrine Pharmaceuticals, JUL 14, 2015, View Source [SID:1234506333])

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On July 14, 2015 Fate Therapeutics, Inc. reported that it has entered into a research collaboration with Regents of the University of Minnesota for the development of natural killer (NK) cell-based cancer immunotherapeutics (Press release, Fate Therapeutics, JUL 14, 2015, View Source [SID1234516709]). The collaboration will foster the advancement of two distinct therapeutic programs, both of which aim to leverage the inherent ability of NK cells to rapidly detect and effectively destroy malignant cells without prior antigen exposure or administration of a patient’s own immune cells. While adoptive transfer of NK cells has demonstrated anti-tumor activity, the isolation and generation of clinically-relevant quantities of homogeneous populations of highly-persistent NK cells has been challenging. Fate Therapeutics will utilize its cell programming approach and proprietary induced pluripotent stem cell technology under the collaboration to pursue the development of optimized "off-the-shelf" NK cell-based cancer therapeutics.

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"Cell-based immunotherapies are rapidly emerging as one of the most promising treatment paradigms for many oncology indications, and NK cell-based therapeutics in particular may offer a compelling off-the-shelf therapeutic approach to adoptive cancer immunotherapy," said Christian Weyer, M.D., M.A.S., President and Chief Executive Officer of Fate Therapeutics. "The University of Minnesota has pioneered the basic research and clinical investigation of NK cell-based therapeutics, and we look forward to collaborating with their expert team in the development of NK cell-based immunotherapies that may provide distinct advantages in transforming the treatment of cancer."

The antibody-dependent cellular cytotoxicity program will be led by renowned NK cell biologist Jeffrey S. Miller, M.D., Deputy Director of the Masonic Cancer Center and the Deputy Director of the Clinical and Translational Science Institute at the University of Minnesota. Dr. Miller and his team have recently identified an "adaptive" NK cell phenotype that exhibits a unique metabolic program shown in preclinical studies to promote long-term persistence in vivo, and that has an epigenetic profile similar to that of cytotoxic T lymphocytes, which may induce potent anti-tumor activity against a variety of tumors. Under the collaboration, Dr. Miller and Fate Therapeutics will apply the Company’s cell programming approach with the intent to optimize NK cell persistence and cytotoxicity and accelerate the development of a programmed "adaptive" NK cellular therapeutic for use in combination with tumor-specific monoclonal antibodies.

"We are excited about the prospects of utilizing cell programming to optimize the anti-tumor properties of the phenotype, and look forward to collaborating with Fate Therapeutics in the development of programmed adaptive NK cell-based immunotherapeutics to treat cancer," said Dr. Miller. "Our data demonstrate that the adaptive phenotype is functionally distinct from conventional NK cells upon triggering through the CD16 receptor, which may make these cells ideal effectors to elicit an enhanced antibody-mediated cytotoxic effect."

The second program, focusing on induced pluripotent stem cell (iPSC)-derived targeted cancer immunotherapy, will be led by Dan Kaufman, M.D., Ph.D., Professor of Medicine and a member of the Masonic Cancer Center at the University of Minnesota. Dr. Kaufman has pioneered the derivation of NK cells from pluripotent stem cells (iNK cells), including the establishment of a clinically-compatible culture system and differentiation protocol that enable the efficient generation of large quantities of cytotoxic NK cells. Leveraging the Company’s proprietary iPSC technology, Dr. Kaufman and Fate Therapeutics will genetically-modify iPSCs to express tumor cell-targeting modalities, creating an immune-engineered pluripotent cell source for use in the derivation of "off-the-shelf" NK cell-based targeted immunotherapies.

"The introduction of antigen-specificity by genetically engineering induced pluripotent stem cells, combined with the unlimited proliferative potential and differentiation capacity of such cells, may prove to be the cornerstone of off-the-shelf targeted cancer immunotherapy," said Dr. Kaufman. "We look forward to developing engineered iNK cell-based cancer therapeutics in collaboration with Fate Therapeutics that may overcome key limitations of adaptive autologous cell therapy including the requirement to isolate and engineer cells for each individual patient."

In consideration for funding the collaboration activities, Fate Therapeutics has the option to secure exclusive patent rights to all intellectual property arising under the collaboration. Additionally, Fate Therapeutics has secured an exclusive option to certain background intellectual property of the University of Minnesota. Drs. Miller and Kaufman will serve as advisors to the Company in the development of hematopoietic cell-based immunotherapies, including those derived from induced pluripotent stem cells.

On July 14, 2015 Celator Pharmaceuticals reported that patients have been enrolled in an investigator-initiated Phase 2 clinical study evaluating CPX-351 (cyatarabine:daunorubicin) Liposome Injection as a treatment for patients with newly diagnosed Acute Myeloid Leukemia (AML) at high risk for induction treatment mortality (Press release, Celator Pharmaceuticals, JUL 14, 2015, View Source [SID:1234506326]).

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"Patients with newly diagnosed AML at increased risk of early death urgently need safer and more effective treatment," said Jorge Cortes, M.D., Deputy Department Chair, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. "Our earlier experiences with CPX-351 in newly diagnosed and first relapse AML patients make us optimistic about this trial."

Phase 2 studies with CPX-351 observed improved efficacy and reduced early mortality in the majority of patients studied directly supporting evaluating CPX-351 in this population of newly diagnosed AML patients at high risk for induction treatment mortality.

The Phase 2 study will enroll up to 55 patients, with the primary objective to assess the preliminary efficacy and safety of multiple dose levels of CPX-351 in patients with newly diagnosed AML at high risk for induction treatment mortality. High risk for induction treatment mortality is defined as 30%-50% predicted risk of expiring by day 60. Estimation of risk for induction treatment mortality will be based on factors associated with lower likelihood of AML response with or without factors that reduce tolerance to treatment-associated adverse events. Every patient must have at least one AML-related factor (adverse cytogenetics, secondary AML, MDR phenotype, etc.) that contributes to elevated risk of induction treatment mortality. Patients may or may not have patient-related factors (poor performance status, co-morbidities, poor organ function, etc.) that also contribute to elevated risk of induction treatment mortality.

"We are pleased that Dr. Cortes and MD Anderson Cancer Center chose to study CPX-351 in this patient population," said Arthur Louie, Chief Medical Officer of Celator Pharmaceuticals. "As we await overall survival data from our CPX-351 Phase 3 trial in patients with high-risk AML, we believe there is significant potential for CPX-351 in other AML populations as well as other blood cancers, and we believe it important to evaluate these opportunities."