On September 19, 2016 Diffusion Pharmaceuticals Inc. (OTCQX:DFFN), a clinical stage biotechnology company focused on the development of novel small molecule therapeutics for cancer and other hypoxia-related diseases, reported the successful completion of dosing of two animal toxicology studies required by the U.S. Food and Drug Administration (FDA) to support Diffusion’s planned Phase 3 clinical trial testing its lead molecule trans sodium crocetinate (TSC) in newly diagnosed Glioblastoma (GBM) patients (Press release, Diffusion Pharmaceuticals, SEP 19, 2016, View Source [SID:SID1234515214]).

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As outlined at the end of Phase 2 FDA meeting, the number of TSC dosing exposures per patient will double in the planned Phase 3 trial as compared to the Phase 2 trial. As part of the agreement reached on a trial design for the Phase 3 study, the FDA requested that Diffusion augment its existing TSC toxicology studies with additional three month TSC toxicology data in both rats and dogs. The animal dosing for these three month studies has now been successfully completed.

David G. Kalergis, Diffusion’s Chairman and Chief Executive Officer, said, "The successful completion of dosing for these three month animal toxicology studies is an important milestone in support of Diffusion’s ability to conduct a Phase 3 pivotal trial of TSC in newly diagnosed GBM patients."

On September 19, 2016 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported that it has entered into a new five-year strategic manufacturing agreement with PCT, a Caladrius company, ("PCT") a subsidiary of Caladrius Biosciences (Nasdaq:CLBS) for the supply of Adaptimmune’s SPEAR T-cell therapies (Press release, Adaptimmune, SEP 19, 2016, View Source [SID:SID1234515212]). Under the contract, Adaptimmune will benefit from exclusive access to an EU and FDA compliant manufacturing unit at PCT as well as dedicated, specialist staff.

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Adaptimmune’s relationship with PCT, a contract manufacturer of patient cell therapy products, is intended to provide Adaptimmune with maximum operational flexibility for the manufacture of its SPEAR T-cell products from development, through clinical manufacturing and ultimately, subject to marketing authorizations, into commercialization.

"PCT is an elite contract manufacturing organization in the field of patient-specific cell therapies, and we are very pleased to strengthen and develop our existing relationship," said Dr. Gwendolyn Binder-Scholl, Adaptimmune’s Chief Technology Officer. "We have worked with PCT over the past three years and their commitment to high quality manufacturing, allied to timely delivery, makes them an ideal manufacturing partner for Adaptimmune. This arrangement will also complement well our new manufacturing plant currently under construction in Philadelphia."

"We are pleased to significantly advance our relationship with Adaptimmune, which began with earlier-phase clinical trials. PCT is committed to adapting our service arrangements to support our client’s evolving needs as they proceed through late-stage trials and into commercial manufacturing," said Robert A. Preti, PhD, President of PCT and Senior Vice President, Manufacturing and Technical Operations of Caladrius Biosciences. "We appreciate Adaptimmune’s continued trust in PCT’s ability to support the distribution of their groundbreaking technologies in the U.S. and Europe."

Adaptimmune’s SPEAR T-cell therapies are novel cancer immunotherapies that use enhanced affinity T-cell receptors (TCRs) to target and destroy cancer cells by strengthening a patient’s natural T-cell response. T-cells are a type of white blood cell that play a central role in a person’s immune response to disease.

The manufacturing process for Adaptimmune’s SPEAR T-cell therapies consists of isolating T-cells from the blood of cancer patients; transferring affinity enhanced TCRs, which have been modified to recognize cancer cells, into the cells; activating and expanding the T-cells; and, introducing the affinity enhanced cells back into the patient to enable the patient’s immune system to attack cancer.

On September 19, 2016 Seattle Genetics, Inc. (NASDAQ: SGEN) reported enrollment of the first patient in a multicenter phase 1 clinical trial of SGN-CD123A for patients with relapsed or refractory acute myeloid leukemia (AML) (Press release, Seattle Genetics, SEP 19, 2016, View Source [SID:SID1234515205]). SGN-CD123A is an investigational antibody-drug conjugate (ADC) targeted to CD123 utilizing Seattle Genetics’ proprietary technology, an engineered cysteine antibody (EC-mAb) stably linked to a highly potent DNA binding agent called a pyrrolobenzodiazepine (PBD) dimer. CD123 is expressed across AML subtypes, including leukemic stem cells, which are difficult to kill and may be responsible for high relapse rates even following intensive therapy. The trial is designed to assess the safety and anti-leukemic activity of SGN-CD123A. SGN-CD123A represents Seattle Genetics’ second clinical-stage program in development for AML, reflecting the company’s commitment to addressing the significant unmet need for these patients.

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"AML is an aggressive, life-threatening disease with at least 33,000 newly diagnosed cases in the U.S. and Europe each year, and those patients have few effective treatment options," said Jonathan Drachman, M.D., Chief Medical Officer and Executive Vice President, Research and Development at Seattle Genetics. "ADCs are an established treatment modality designed to specifically target cancer cells and minimize toxic side effects. The significant unmet need for AML and frequent expression of the validated target CD123 on leukemic stem cells support initiating this phase 1 clinical trial. Through our SGN-CD123A program, alongside vadastuximab talirine targeted to CD33 and currently in a phase 3 study, we are making a substantial effort to develop novel treatment options for AML patients."

The study is a phase 1, open-label, multicenter, dose-escalation clinical trial. It will initially evaluate the maximum tolerated dose of SGN-CD123A, followed by an expansion cohort to further define safety and estimate anti-leukemic activity. In addition, the trial will assess pharmacokinetics, remission rate, duration of complete remission and overall survival. The study is designed to evaluate SGN-CD123A administered every three weeks and will enroll up to approximately 100 relapsed or refractory patients at multiple centers in the United States.

Preclinical SGN-CD123A data presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated enhanced anti-leukemic activity across multiple AML disease models, including those typically resistant to chemotherapy. With more than 15 years of experience and innovation, Seattle Genetics is the leader in ADC development. ADCs are designed to selectively deliver cell-killing agents to tumor cells, and thus reduce many of the toxic effects of traditional chemotherapy while enhancing antitumor activity.

For more information about the trial, including enrolling centers, please visit www.clinicaltrials.gov.

About Acute Myeloid Leukemia

Acute myeloid leukemia, also called acute myelocytic leukemia or AML, is an aggressive type of cancer of the bone marrow and blood that progresses rapidly without treatment. AML starts in the bone marrow (the interior part of bones, where new blood cells are made) and quickly moves into the blood. According to the SEER database and Kantar Health Sciences, in 2016 approximately 33,000 new cases of AML (mostly in adults) will be diagnosed in the U.S. and Europe. In the U.S. alone, nearly 10,500 deaths will occur from AML this year.

About SGN-CD123A

SGN-CD123A is a novel ADC targeted to CD123 utilizing Seattle Genetics’ proprietary technology. CD123 is expressed across AML subtypes, including on leukemic stem cells, which are difficult to kill and may be responsible for high relapse rates even following intensive therapy. The CD123 antibody is attached to a highly potent DNA binding agent, a pyrrolobenzodiazepine (PBD) dimer, via a proprietary site-specific conjugation technology to a monoclonal antibody with engineered cysteines (EC-mAb). PBD dimers are significantly more potent than systemic chemotherapeutic drugs and the site-specific conjugation technology (EC-mAb) allows uniform drug-loading of the cell-killing PBD agent to the anti-CD123 antibody. The ADC is designed to be stable in the bloodstream and to release its potent DNA binding agent upon internalization into CD123-expressing cells.

On September 19, 2016 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported that EORTC, the European Organisation for Research and Treatment of Cancer, is expanding its collaborative agreement with Ignyta (Press release, Ignyta, SEP 19, 2016, View Source [SID:SID1234515203]). Under this collaboration, Ignyta will serve as a diagnostic laboratory performing fusion testing for EORTC’s SPECTA (Screening Patients for Efficient Clinical Trial Access) initiative.

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Part of the testing will include identifying patients who harbor a gene fusion for the NTRK, ROS1 or ALK genes for potential inclusion of these patients in Ignyta’s global STARTRK-2 Phase 2 clinical study of entrectinib. The genetic data obtained from this screening will also be available for potential future academic research as part of EORTC’s SPECTA initiative.

"EORTC’s SPECTA network includes a broad pool of clinical trial sites, tissue samples and patient data, which is helping us identify patients with a variety of tumor histologies across Europe who could potentially be enrolled in our STARTRK-2 clinical trial, and may benefit from entrectinib, our CNS-penetrant, targeted Trk, ROS1 and ALK inhibitor," said Zachary Hornby, COO at Ignyta. "We are happy to work with EORTC to demonstrate the value of biomarker-driven clinical trials to bring forward innovative, targeted cancer medicines."

EORTC’s SPECTA program is a pan-European biobank and molecular profiling platform for patients in colorectal, brain, lung, melanoma and rare cancers, which aims to optimize drug access, help advance the development of targeted treatments and accelerate new healthcare delivery. Built as a collaborative network, SPECTA provides access to large sets of patient-level clinical and biological data for international, multidisciplinary clinical research.

"Identification of specific genetic mutations provides opportunities for improving treatment efficacy in cancer patients," said Vassilis Golfinopoulos, Medical Director at EORTC. "This is at the heart of our SPECTA program, which helps oncologists find trials that are adapted to their patients’ needs. The collaboration with Ignyta is allowing more patients to be screened, and providing more data to advance the development of precision medicine in cancer."

The Ignyta STARTRK-2 Phase 2 study is evaluating the effectiveness of entrectinib, a selective tyrosine kinase inhibitor targeting tumors that harbor NTRK, ROS1 or ALK gene fusions. The STARTRK-2 trial is actively enrolling and working with leading cancer centers worldwide, including nine countries throughout Europe.

About EORTC

The European Organisation for the Research and Treatment of Cancer (EORTC) brings together European cancer clinical research experts from all disciplines for trans-national collaboration. Both multinational and multidisciplinary, the EORTC Network comprises more than 4,600 collaborators from all disciplines involved in cancer treatment and research in more than 640 hospitals in over 37 countries. A unique pan-European clinical research infrastructure, EORTC offers an integrated approach to drug development, drug evaluation programs and medical practices through translational and clinical research.