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bluebird bio and TC BioPharm Announce Strategic Collaboration to Research and Develop Gamma Delta CAR T Cell Product Candidates for Cancer Immunotherapy

On December 7, 2017 bluebird bio, Inc. (Nasdaq: BLUE) and Scottish immunotherapy company TC BioPharm, Ltd. (TCB) reported a strategic collaboration and license agreement focused on gamma delta CAR T cells (Press release, bluebird bio, DEC 7, 2017, View Source [SID1234522423]). The companies will work together to advance TC BioPharm’s lead CAR-engineered gamma delta T cell program into clinical trials as well as on additional hematologic and solid tumor targets.

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"Emerging research suggests that gamma delta T cells may constitute a powerful platform for CAR T cell therapies," said Philip Gregory, D.Phil., chief scientific officer, bluebird bio. "TCB is a leader in the gamma delta T cell field, with extensive capabilities spanning early research, clinical development and manufacturing. The combination with our deep expertise in CAR T cell biology, translational and clinical experience with leading CAR T cell drug products, and powerful gene therapy toolbox, offers a high degree of synergy. This partnership aims to help realize the full potential of the gamma delta T cell platform to bring novel and transformative therapies to cancer patients with high unmet medical need."

Commenting on the partnership with bluebird bio, TCB’s chief executive – Michael Leek, PhD, said, "We are delighted to be working alongside bluebird bio to discover and develop next-generation CAR T cell therapies based on our innovative ImmuniCAR platform. Both companies share the same dynamic culture, passion and drive, spearheaded by an overwhelming desire to treat cancer patients – with the potential to dramatically improve each individual’s prognosis and quality of life."

"We believe our gamma delta T cell platform has broad therapeutic potential," added Artin Moussavi, PhD, chief business officer of TC BioPharm. "The collaboration with bluebird bio, a leader in cell and gene therapy, recognizes the enormous potential of ImmuniCAR to deliver life-changing medicines."
"bluebird bio is leveraging its industry-leading toolbox of advanced cell and gene therapy technologies to accelerate immuno-oncology targets from concept to clinic," said Joanne Smith-Farrell, bluebird’s senior vice president, corporate development and strategy. "The agreement with TCB complements bluebird bio’s growing immuno-oncology development program, which includes clinical and pre-clinical CAR T and T Cell Receptor programs that leverage bluebird bio’s leading translational research and deep vector technology expertise to rapidly accelerate from target identification to clinical development."

Under the terms of the agreement, bluebird bio and TCB will collaborate to discover and develop CAR-engineered gamma delta T cells for cancer targets and indications. TCB is responsible for development of all targets through Phase 1/2, at which point bluebird has the exclusive option to assume sole responsibility for further clinical development and commercialization on a global basis.

Financial terms of the agreement include a $16 million upfront payment and subsequent potential R&D and commercial milestone payments. The Company is also eligible to receive undisclosed tiered royalties on product sales.

OncoCyte Presents Positive Data from its Liquid Biopsy Breast Cancer Diagnostic Test at the San Antonio Breast Cancer Symposium

On December 7, 2017 OncoCyte Corporation (NYSE American:OCX), a developer of novel, non-invasive liquid biopsy tests for the early detection of cancer, reported positive data from its most recent breast cancer diagnostic test study at the 2017 San Antonio Breast Cancer Symposium (SABCS) (Press release, BioTime, DEC 7, 2017, View Source;p=RssLanding&cat=news&id=2321631 [SID1234522422]). The data were presented by Philip McQuary, Ph.D., Director of Product Development at OncoCyte.

This study revealed that a novel blood based diagnostic test may allow for the non-invasive and sensitive detection of breast cancer in BI-RADS category 4 patients, thereby differentiating women who have breast cancer from those who do not. A 19-marker model resulted in an AUC of 0.935 with a sensitivity of 90% and specificity of 82%. The data from this study are consistent with data reported at the San Antonio Breast Cancer Symposium in December 2016.

The poster, titled "Assessment of an Immune Response Panel of Serum Protein Biomarkers for the Non-Invasive Detection of Breast Cancer," discusses details of OncoCyte’s study in which serum samples were collected at four U.S. sites from 136 women with suspicious diagnostic mammography findings undergoing biopsy to determine if they have breast cancer. All 136 subjects had mammograms and were classified as BI-RADS category 4 and had pathology confirmation of their diagnosis (malignant or benign). Statistical screening methodologies were used to identify markers with the potential to distinguish benign from malignant pathology. The candidate markers were further studied and combined to develop a potential diagnostic test.

BI-RADS (Breast Imaging and Reporting Data System) is a scoring system developed by the American College of Radiologists to help clinicians assess the risk of cancer in women with a lump or mass. A BI-RADS category 4 classification indicates a suspicious result, and women in this category are generally referred for a breast biopsy. The AUC of a test is a measure that combines sensitivity and specificity to express its total accuracy, with 1.0 being perfect accuracy and 0.50 being a random result. Sensitivity and specificity are statistical measures of test performance, with sensitivity measuring the percentage of malignant lumps or lesions that are identified correctly by the test and specificity measuring the percentage of benign lumps or masses correctly identified.

The current standard of care for breast cancer diagnosis – annual or biannual mammogram screenings – does not meet the needs of large populations of women for whom mammography alone is not sufficient. These populations include women with dense breast tissue, genetic mutations (BRCA), a family history of breast cancer, or those who have suspicious mammogram screening results (BIRADs 3 or 4). The Company’s non-invasive liquid biopsy breast cancer diagnostic is intended to be a confirmatory, post-mammogram test that potentially would reduce the number of patients subjected to invasive breast biopsy procedures. Further R&D and clinical utility studies are required to determine whether the confirmatory test would be accurate and commercially viable.

According to published reports, there are about 38 million mammograms performed annually in the U.S., resulting in 1.6 million breast biopsies per year. Of these, only 260,000 (16%) result in a cancer diagnosis. The large number of suspicious findings in diagnostic mammograms leads to a significant amount of unnecessary invasive follow-up procedures. The financial burden to the healthcare system imposed by the follow-up testing of false-positive mammograms and breast cancer over-diagnosis is estimated to be $4 billion a year.

About Breast Cancer

Breast cancer is the second most common cancer among US women. Current screening guidelines set forth by the American Cancer Society recommend screening mammography for the early detection of breast cancer in women at average risk. Specifically, guidelines call for annual mammography for asymptomatic women age 45 to 54 and once every two years for women age 55 and older. Suspicious screening mammograms are generally followed up with a diagnostic mammogram and sometimes by an MRI (Magnetic Resonance Image) or an ultrasound. Ultimately, suspicious findings unresolved by imaging typically result in the recommendation of a breast biopsy.

Alexion and Halozyme Enter License Agreement for ENHANZE Technology

On December 7, 2017 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Halozyme Therapeutics, Inc. (NASDAQ:HALO) reported a collaboration and license agreement that enables Alexion to use Halozyme’s ENHANZE drug-delivery technology in the development of subcutaneous formulations for their portfolio of products (Press release, Alexion, DEC 7, 2017, View Source [SID1234522420]). The agreement provides Alexion with the opportunity for exclusive development of up to four targets, including a next generation subcutaneous formulation of ALXN1210 (ALXN1210 SC), the company’s investigational long-acting C5 complement inhibitor, to potentially further extend the dosing interval of ALXN1210 SC to once every two weeks or once per month.

"Alexion’s goal is to provide continued innovation and more treatment options that can significantly improve the lives of patients with rare diseases," said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. "We are excited to partner with Halozyme and look forward to utilizing its ENHANZE technology, which enables rapid injection of subcutaneous treatments and potentially increases bioavailablity, in our development programs."

"We are delighted to support Alexion’s innovative development initiatives focused on improving the lives of patients with rare diseases," said Dr. Helen Torley, president and CEO of Halozyme. "ENHANZE has become the industry standard for converting intravenous therapies to a subcutaneous delivery, helping partners and health care providers reduce the treatment burden and administration time for patients."

Under the terms of the agreement, Halozyme will receive an initial $40 million with the potential to earn additional payments of up to $160 million for each target developed, subject to achievement of specified development, regulatory and sales-based milestones. Halozyme will also receive mid-single digit royalties on sales of commercialized products.

The Halozyme ENHANZE technology is based on a proprietary recombinant human hyaluronidase enzyme (rHuPH20) that temporarily degrades hyaluronan — a glycosaminoglycan or chain of natural sugars in the body — to aid in the dispersion and absorption of other injected therapeutic drugs. For Halozyme partners, this technology may allow for more rapid delivery of injectable medications through subcutaneous injection (just under the skin). This delivery has been shown in studies to reduce health care practitioner time required for administration and shorten time for drug administration.

Alexion is Halozyme’s eighth global collaboration and license partner for the ENHANZE technology, and the third partnership formed in 2017. These partnerships cover nearly 50 therapeutic targets and include three commercialized products.

AVEO Oncology Announces Clinical Updates to Tivozanib and Ficlatuzumab Programs

On December 7, 2017 AVEO Oncology (NASDAQ:AVEO) reported clinical updates for two of its oncology programs: FOTIVDA (tivozanib), the Company’s potent, selective, long half-life inhibitor of all three vascular endothelial growth factor (VEGF) receptors, and ficlatuzumab, the Company’s humanized IgG1 antibody that binds to the hepatocyte growth factor (HGF) ligand with high affinity and specificity to inhibit the biological activities of the HGF/c-Met pathway (Press release, AVEO, DEC 7, 2017, View Source;p=RssLanding&cat=news&id=2321570 [SID1234522414]).

"We continue to build strong momentum in our oncology programs, including progress with our lead program, tivozanib, and the advancement of our other oncology programs, including ficlatuzumab and AV-203," said Michael Bailey, president and chief executive officer of AVEO. "With approval of tivozanib and commercial sales underway in Europe, we are squarely focused on the next two pillars of our tivozanib strategy: the potential for U.S. registration and additional immunotherapy combination studies. As these strategies unfold, 2018 is expected to be another transformative year, with anticipated top-line results in the TIVO-3 study of tivozanib in third line advanced renal cell carcinoma (RCC), as well as development of our earlier-stage programs, including the TiNivo study of tivozanib in combination with Opdivo, and the initiation of two ficlatuzumab investigator-sponsored studies."

Tivozanib Updates

Enrollment Complete in Phase 2 Portion of Phase 1/2 TiNivo Trial in Advanced RCC. AVEO announced today that enrollment of 21 patients is now complete, with one patient remaining in screening, in the Phase 2 portion of the TiNivo study, a Phase 1/2 multicenter trial of tivozanib in combination with Bristol-Myers Squibb’s OPDIVO (nivolumab), an immune checkpoint, or PD-1, inhibitor, for the treatment of advanced RCC. In the Phase 1 dose escalation portion of the trial, tivozanib was administered in two escalating dose cohorts, 1.0 and 1.5 mg daily, in combination with nivolumab at 240 mg every 2 weeks (n=6).

Phase 1 data from the study, which were presented at the 16th International Kidney Cancer Symposium, demonstrated that the combination of Opdivo and tivozanib was well tolerated up to the full dose and schedule of single agent tivozanib (1.5 mg daily), with no dose limiting toxicities. The most common adverse events (any grade) were hypertension, asthenia and decreased appetite. No grade 4 adverse events were reported. Two grade 3 events were reported beyond cycle 1 (stomatitis and increased ALT), which did not lead to study discontinuation and were managed concurrently. Best response at the time of presentation included a 67% (4/6) partial response (PR) rate and a 100% disease control rate (4 confirmed PR + 2 stable disease, 1 of which was unconfirmed). Additional results from the Phase 1 portion of the trial and initial results from the Phase 2 portion are expected to be presented at scientific meetings in the first half of 2018, including at the 2018 Genitourinary Cancers Symposium taking place February 8-10, 2018, and co-sponsored by the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).
Ficlatuzumab Updates

Phase 2 Study of Ficlatuzumab in Combination with Cetuximab in HNSCC Initiated. The Company announced today the initiation of an investigator-sponsored randomized, multicenter Phase 2 trial of ficlatuzumab and cetuximab (ERBITUX), an EGFR-targeted antibody, in patients with cetuximab-resistant, metastatic head and neck squamous cell carcinoma (HNSCC). AVEO is partnered with Biodesix, Inc. on the developments of ficlatuzumab. The study will seek to confirm findings from a Phase 1 study where the addition of ficlatuzumab to cetuximab resulted in a disease control rate of 67%, and prolonged progression free and overall survival compared to historical controls, in addition to being well tolerated. This Phase 2 multi-center study, which is being conducted under the direction of Julie E. Bauman, MD, MPH, Professor of Medicine, Chief, Division of Hematology/Oncology, Associate Director of Translational Research, University of Arizona Cancer Center, is expected to enroll approximately 60 patients randomized to receive either ficlatuzumab alone or ficlatuzumab and cetuximab.
Phase 1b Study of Ficlatuzumab in Combination with Gemcitabine and Nab-paclitaxel in Pancreatic Cancer Initiated. The Company announced today the initiation of an investigator-sponsored Phase 1b study to test the safety and tolerability of ficlatuzumab when combined with Nab-paclitaxel and Gemcitabine in previously untreated metastatic pancreatic ductal cancer (PDAC). The goal of the study, which is based on preclinical findings demonstrating a synergistic effect of these drugs in a preclinical model of PDAC, is designed to determine maximum tolerated dose of ficlatuzumab when combined with gemcitabine and nab-paclitaxel. Secondary outcome measures include response rate and progression free survival. The study, which is being conducted under the direction of Kimberly Perez, M.D. at the Dana-Farber Cancer Institute, is expected to enroll approximately 30 patients.
About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI) discovered by Kyowa Hakko Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma in the European Union plus Norway and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications. Tivozanib has been investigated in several tumors types, including renal cell, colorectal and breast cancers.

About Ficlatuzumab

Ficlatuzumab (formerly known as AV-299) is a potent hepatocyte growth factor (HGF) inhibitory antibody that binds to the HGF ligand with high affinity and specificity to inhibit HGF/c-Met biological activities. AVEO and Biodesix, Inc. currently divide all worldwide development costs for ficlatuzumab and are seeking a commercialization partner. Ficlatuzumab is currently being evaluated in investigator-sponsored trials in squamous cell carcinoma

Neurocrine Biosciences to Present at the BMO Capital Markets 2017 Prescriptions for Success Healthcare Conference

On December 7, 2017 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported that it will present at the BMO Capital Markets 2017 Prescriptions for Success Healthcare Conference at 11:00 a.m. ET on Thursday, Dec. 14, 2017, in New York City (Press release, Neurocrine Biosciences, DEC 7, 2017, View Source;p=RssLanding&cat=news&id=2321693 [SID1234522445]). Timothy P. Coughlin, Vice President Finance of Neurocrine Biosciences, will present at the conference.

The live presentation will be webcast and may be accessed on the Company’s website at View Source A replay of the presentation will be available on the website approximately one hour after the conclusion of the event and will be archived for one month.