On December 7, 2017 Heat Biologics, Inc. ("Heat") (NASDAQ: HTBX), a biopharmaceutical company developing drugs designed to activate a patient’s immune system against cancer, reported that it received written responses from the U.S. Food and Drug Administration (FDA) following its Type C meeting regarding its planned registrational HS-110 clinical trial design for the treatment of non-small cell lung cancer (NSCLC) (Press release, Heat Biologics, DEC 7, 2017, View Source [SID1234522426]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The discussion focused on elements of proposed clinical trial designs, both single-arm and controlled, which the FDA agreed would be appropriate to support a registrational trial of HS-110. Clinical endpoints and post-marketing commitments were also discussed in the context of accelerated approval.

"We are very pleased with the outcome of our recent Type C guidance meeting with the FDA," said George Peoples, M.D., Chief Medical Officer for Heat. "The FDA clearly understands the significant unmet need for more effective second-line treatments for NSCLC. We look forward to incorporating their guidance as we prepare to advance HS-110 into registrational trials."

HS-110 is currently in Phase 2 as a treatment for NSCLC. Trial design details and next steps are expected to be announced following the read-out of the Phase 2 data, anticipated in 2H 2018.

On December 7, 2017 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or "the Company") reported that it has begun dosing subjects in its pivotal study for the Company’s fulvestrant formulation intended as a monotherapy treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer in postmenopausal women not previously treated with endocrine therapy, or HR-positive advanced breast cancer in postmenopausal women with disease progression following endocrine therapy or as a combination therapy with palbociclib for the treatment of HR-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer in women with disease progression after endocrine therapy (Press release, Eagle Pharmaceuticals, DEC 7, 2017, View Source [SID1234522424]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This pharmacokinetic and safety pivotal study is an open label trial in which healthy female volunteers across multiple U.S. sites will be randomized 1:1 to receive either the Company’s fulvestrant formulation or the reference drug Faslodex. The Company anticipates dosing its last subject during the first half of 2018, with study completion within twelve months, and an expected NDA filing in the fourth quarter of 2018.

"We began dosing the first cohort of subjects in our pivotal study fulvestrant trial on November 30th, following guidance from the U.S. Food and Drug Administration (FDA) regarding the study design. We believe our fulvestrant formulation holds the potential to be a best-in-class treatment option for thousands of patients," stated Scott Tarriff, Chief Executive Officer.

"Our innovative formulation, if approved, could offer multiple potential benefits compared to the current branded fulvestrant product, Faslodex. Our formulation allows the therapy to be administered at the recommended dose with one intramuscular injection instead of two high-viscosity intramuscular injections, and in far less time – seconds instead of minutes. In addition, it does not contain castor oil, and our formulation’s lower viscosity allows for administration with a 23-gauge needle, which is 25% thinner than the current needle required to administer Faslodex," added Tarriff.

Faslodex, manufactured by AstraZeneca, generated worldwide sales of $925 million in the twelve months ended September 30, 2017.

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.