On December 7, 2017 Sanofi reported that it has launched two new late-stage clinical studies to determine if an investigational biologic called isatuximab, when used in combination with other commonly used cancer treatments, might be an effective treatment option for certain people with multiple myeloma, a rare blood cancer related to lymphoma and leukemia. Isatuximab is an investigational anti-CD38 monoclonal antibody being studied for the treatment of patients with relapsed and previously untreated multiple myeloma (Press release, Sanofi Genzyme, DEC 7, 2017, View Source [SID1234522429]).

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"The start of two new Phase 3 trials will provide further clinical data as we continue to advance the development of isatuximab," said Joanne Lager, M.D. Head of Oncology Development, Sanofi. "Our multi-study program across major multiple myeloma segments aims to demonstrate the value of isatuximab in combination with emerging standard treatment regimens. We are committed to developing a potential new treatment option for a continuum of patients with multiple myeloma, a population with high unmet need."

Late-stage studies include approximately 750 patients with multiple myeloma

IKEMA study is a 325-patient randomized, open-label, global multicenter Phase 3 trial that will compare isatuximab in combination with carfilzomib and dexamethasone against carfilzomib and dexamethasone in patients with relapsed and refractory multiple myeloma that have previously been treated with one-to-three lines of therapy.
IMROZ study is a 425-patient randomized, open-label, global multicenter Phase 3 trial that will compare isatuximab in combination with bortezomib, lenalidomide and dexamethasone against bortezomib, lenalidomide and dexamethasone in newly diagnosed multiple myeloma patients not eligible for transplant.
Both studies will evaluate progression-free survival as the primary endpoint. Key secondary endpoints include overall survival, overall response rate, depth of response, safety and quality of life.

Isatuximab granted orphan designation
Isatuximab has been granted orphan designation in the U.S. and European Union. In December 2016, Sanofi started an additional Phase 3 study (ICARIA), comparing isatuximab in combination with pomalidomide and dexamethasone against pomalidomide and dexamethasone in patients with relapsed and refractory multiple myeloma. The development program for isatuximab will now total three Phase 3 studies.

Other isatuximab data being presented at upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) meeting
Findings from additional ongoing studies of isatuximab will be presented during poster sessions at this year’s American Society of Hematology (ASH) (Free ASH Whitepaper) meeting, December 8-12, in Atlanta, GA, including the following abstracts:

Saturday, December 9, 5:30 p.m.-7:30 p.m.:

Updated Results from a Phase Ib Study of Isatuximab Plus Pomalidomide (Pom) and Dexamethasone (dex) in Relapsed/Refractory Multiple Myeloma (RRMM)
Abstract: 1887
Presenter: Dr. Paul Richardson
"In Vivo Vaccination" Effect in Clinical Responders to Anti-Myeloma Monoclonal Antibody Isatuximab
Abstract: 1830
Presenter: Dr. Tim Luetkens
Sunday, December 10, 6:00 p.m. -8:00 p.m.:

A Phase Ib Study of Isatuximab in Combination with Bortezomib, Cyclophosphamide, and Dexamethasone (VCDI) in Patients with Newly Diagnosed Multiple Myeloma Non-Eligible for Transplantation
Abstract: 3160
Presenter: Dr. Enrique M. Ocio
Pre-Clinical Efficacy of the Anti-CD38 Monoclonal Antibody (mAb) Isatuximab in Acute Myeloid Leukemia (AML)
Abstract: 2655
Presenter: Tomas Jelinek

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.

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 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 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.