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.

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]).

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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 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]).

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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 Ablynx NV [Euronext Brussels and Nasdaq: ABLX] reported that the Ablynx management team will host a conference call and webcast to present additional data from the Phase III HERCULES study of caplacizumab in acquired thrombotic thrombocytopenic purpura (aTTP), following its presentation by Professor Marie Scully in the late-breaking abstracts session at the 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Atlanta, GA, USA (Press release, Ablynx, DEC 7, 2017, View Source [SID1234522437]).

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The late-breaking data presentation will include positive topline results from the Phase III HERCULES study of caplacizumab announced on 2 October 2017 together with important data from additional analyses on the use of plasma exchange (PEX) and length of intensive care unit and hospital stay. The slides will be made available on the Ablynx website under Events & Presentations immediately after the presentation on 12 December 2017 at 7.30 am ET/1.30 pm CET.

Following the late-breaking data presentation, the Ablynx management team will host a conference call and webcast on 12 December 2017 at 4.00 pm CET/10.00 am ET. The live webcast and replay will be available via this link. If you wish to participate in the Q&A session, please dial +32(0)2 400 69 26 or +1 646 828 8193 and use confirmation code 9477994.

Late-breaking abstracts session at ASH (Free ASH Whitepaper), Atlanta, GA, USA
Date: Tuesday 12 December 2017
Abstract (LBA-1): Results of the Randomized, Double-Blind, Placebo-Controlled, Phase III HERCULES study of Caplacizumab in Patients with Acquired Thrombotic Thrombocytopenic Purpura
Presentation Time: 7.30 am ET (1.30 pm CET)
Presenter: Professor Marie Scully, M.D., Department of Haematology, University College London Hospitals NHS Trust, London, UK
Room: Building C, Level 1, Hall C2-C3 (Georgia World Congress Center)

About HERCULES
The HERCULES study recruited 145 patients and is the largest randomised, double-blind, placebo-controlled study conducted in patients with aTTP. Patients with an acute episode of aTTP were randomised 1:1 to receive either caplacizumab or placebo in addition to daily plasma exchange and immunosuppression. Patients received a single intravenous bolus of 10mg caplacizumab or placebo followed by a daily subcutaneous dose of 10mg caplacizumab or placebo for 30 days after the last daily PEX. If at the end of this treatment period there was evidence of persistent underlying disease activity (indicative of an imminent risk for recurrence), treatment could be extended for additional seven-day periods up to a maximum of 28 days and was to be accompanied by optimisation of immunosuppression. Patients were followed for a further 28 days after discontinuation of treatment.
A three-year follow-up study (NCT02878603) of patients who have completed the HERCULES study is in progress and will further evaluate the long-term safety and efficacy of caplacizumab and repeated use of caplacizumab, as well as characterising the long-term impact of aTTP.

About caplacizumab
Caplacizumab is a bivalent anti-vWF Nanobody that received Orphan Drug Designation in Europe and the United States in 2009. Caplacizumab blocks the interaction of ultra-large vWF multimers (ULvWF) with platelets and, therefore, has an immediate effect on platelet aggregation and the ensuing formation and accumulation of the micro-clots that cause the severe thrombocytopenia, tissue ischemia and organ dysfunction in aTTP. This immediate effect of caplacizumab has the potential to protect the patient from the manifestations of the disease while the underlying disease process resolves.

In February 2017, based on the Phase II study results, a Marketing Authorisation Application (MAA) was submitted to the European Medicines Agency (EMA) for approval of caplacizumab in aTTP. In July 2017, Ablynx received Fast Track designation from the Food and Drug Administration (FDA) for caplacizumab for the treatment of aTTP. In October 2017, positive results from the Phase III HERCULES study, meeting primary and two key secondary endpoints, were announced. These data are expected to further support the MAA, as well as a planned Biologics License Application (BLA) filing in the United States in 2018. If approved by regulatory authorities, caplacizumab would be the first therapeutic specifically indicated for the treatment of aTTP.
About aTTP

aTTP is a rare, acute, life-threatening, autoimmune blood clotting disorder. It is caused by impaired activity of the ADAMTS13 enzyme, leaving ULvWF molecules uncleaved (vWF is an important protein involved in the blood clotting process). These ULvWF molecules spontaneously bind to blood platelets, resulting in severe thrombocytopenia (very low platelet count) and clot formation in small blood vessels throughout the body1, leading to ischemia and widespread organ damage2.

Despite the current standard-of-care treatment consisting of PEX and immunosuppression, episodes of aTTP are still associated with a mortality rate of up to 20%, with most deaths occurring within 30 days of diagnosis3. Furthermore, patients are at risk of acute thromboembolic complications (e.g. stroke, myocardial infarction) and of recurrence of disease. Some patients are refractory to therapy1, which is associated with a poor prognosis for survival of an acute episode of aTTP. Long term, patients are at increased risk for hypertension, major depression, and premature death4.

On December 7, 2017 Neon Therapeutics, a clinical-stage immuno-oncology company developing neoantigen-based therapeutics, reported that it has entered a clinical trial collaboration with Merck (known as MSD outside the United States and Canada) to evaluate Neon Therapeutics’ proprietary personal neoantigen vaccine, NEO-PV-01, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), along with chemotherapy (Press release, Neon Therapeutics, DEC 7, 2017, View Source [SID1234522428]).

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NEO-PV-01 is Neon Therapeutics’ most advanced product candidate, and is a personal neoantigen vaccine based on DNA mutations from each patients’ tumor. Neon Therapeutics and Merck will collaborate on a Phase 1b clinical trial that will examine the safety, tolerability and preliminary efficacy of NEO-PV-01 in combination with KEYTRUDA, pemetrexed and carboplatin in patients with untreated advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC). Additionally, the trial will assess neoantigen-specific immune responses in peripheral blood and tumor tissue, and other markers of immune response.

"We believe there is a strong mechanistic rationale to explore the combination of a personal neoantigen cancer vaccine, anti-PD-1 therapy and chemotherapy," said Richard Gaynor, M.D., president of research and development at Neon Therapeutics. "Together with a growing set of clinical collaborators, we are working to amass a diverse set of clinical data to understand the potential of NEO-PV-01 to improve durability and response rates in combination with multiple immuno-oncology targets."

The collaboration agreement is between Neon Therapeutics, Inc. and Merck, through a subsidiary. Additional details were not disclosed.