On October 1, 2019 Checkmate Pharmaceuticals Inc., a clinical stage biopharmaceutical company focused upon activation of innate immunity to treat advanced cancer, reported the first patient was treated in a Phase 1b/II study of BAVENCIO (avelumab) in multi-drug combinations with CMP-001 in patients with Squamous Cell Cancer of the Head and Neck (SCCHN) as part of the JAVELIN Medley study sponsored by Pfizer Inc. (NYSE:PFE) in collaboration with Merck KGaA, Darmstadt, Germany (Press release, Checkmate Pharmaceuticals, OCT 1, 2019, View Source [SID1234539993]). JAVELIN Medley is designed to evaluate BAVENCIO, a human anti-programmed death ligand (PD-L1) co-developed and co-commercialized by Merck KGaA Darmstadt, Germany and Pfizer, in combination with other immune modulators in patients with locally advanced or metastatic solid tumors.

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"The dosing of patients in this trial represents an important milestone for Checkmate as we seek to build upon promising data we have generated to date with CMP-001, particularly in PD-1-refractory advanced melanoma. Based upon the preclinical and clinical work conducted to date, we believe CMP-001 can effectively convert cold tumors to hot and thereby improve response to PD-1/L1-based immunotherapy in a variety of solid tumors. We look forward to the results of this clinical trial," commented Barry Labinger, President and CEO of Checkmate Pharmaceuticals.

This Phase 1b/2 study in up to 60 patients is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of the doublet combination of avelumab and CMP-001; the triplet combination of CMP-001, avelumab and utomilumab (4-1BB agonist); and the triplet combination of CMP-001, avelumab and PF-04518600 (OX40 agonist), in patients with locally advanced SCCHN. For more information about this trial, please visit clinical trials www.clinicaltrials.gov. (Identifier: NCT02554812)

About CMP‐001

CMP‐001 is a first‐in‐class CpG‐A Toll‐like receptor 9 (TLR9) agonist that is encapsulated in a virus‐like particle. CMP‐001 is designed to induce both innate and adaptive anti‐tumor immune responses, thereby converting immunologically "cold" tumors into immunologically "hot" tumors, with the potential to mediate tumor regression. It is the only CpG‐A class TLR9 agonist in clinical trials and differs from other CpG classes in clinical development by having a native DNA backbone that induces the highest levels of type I Interferon (IFN). Based on analyses of gene expression in human tumors showing that increased IFN and related immune gene expression is associated with better response to PD‐1 inhibition, it is believed that this mechanism of action may restore, enable or improve responses to anti‐PD‐1/PD‐L1 therapeutics. CMP‐001 is being evaluated in multiple tumor types to assess safety, activity, alternative routes of administration and combination with other immunotherapies and modalities. For information on CMP‐001 trials that are currently recruiting patients, please visit www.clinicaltrials.gov.

Avelumab Approved Indications

Avelumab (BAVENCIO) in combination with axitinib is indicated in the US for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

The US Food and Drug Administration (FDA) also granted accelerated approval for avelumab (BAVENCIO) for the treatment of (i) adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (mMCC) and (ii) patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. These indications are approved under accelerated approval based on tumor response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

Avelumab is currently approved for patients with MCC in more than 45 countries globally, with the majority of these approvals in a broad indication that is not limited to a specific line of treatment.

Avelumab Important Safety Information from the US FDA-Approved Label

The warnings and precautions for avelumab (BAVENCIO) include immune-mediated adverse reactions (such as pneumonitis and hepatitis [including fatal cases], colitis, endocrinopathies, nephritis and renal dysfunction and other adverse reactions [which can be severe and have included fatal cases]), infusion-related reactions, hepatotoxicity, major adverse cardiovascular events (MACE) [which can be severe and have included fatal cases], and embryo-fetal toxicity.

Common adverse reactions (reported in at least 20% of patients) in patients treated with BAVENCIO monotherapy include fatigue, musculoskeletal pain, diarrhea, nausea, infusion-related reaction, peripheral edema, decreased appetite/hypophagia, urinary tract infection and rash. Common adverse reactions (reported in at least 20% of patients) in patients receiving BAVENCIO in combination with axitinib include diarrhea, fatigue, hypertension, musculoskeletal pain, nausea, mucositis, palmar-plantar erythrodysesthesia, dysphonia, decreased appetite, hypothyroidism, rash, hepatotoxicity, cough, dyspnea, abdominal pain and headache. Clinical chemistry and hematology laboratory value abnormalities reported in at least 10% of patients include hyponatremia, lymphopenia, GGT increased, blood triglyceride increased and lipase increased, and grade 3-4 lymphopenia, anemia, elevated cholesterol and liver enzymes.

For full Prescribing Information and Medication Guide for BAVENCIO, please see www.BAVENCIO.com.

On October 1, 2019 Tmunity Therapeutics, Inc., a private clinical-stage biotherapeutics company focused on saving and improving lives by delivering the full potential of next-generation T-cell immunotherapy, reported it has entered into an exclusive license and research collaboration agreement with Children’s Hospital of Philadelphia (CHOP) to advance a glypican 2 (GPC2) chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of neuroblastoma, a rare cancer that affects the developing nervous system (Press release, Tmunity Therapeutics, OCT 1, 2019, View Source [SID1234539992]). The therapy also has expansion potential in medulloblastoma and small cell lung cancer, among several other pediatric and adult cancers that express an abundance of the GPC2 protein on their cell surface.

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John Maris, MD and Kristopher Bosse, MD, pediatric oncologists and physician-scientists at CHOP, identified and validated the oncoprotein GPC2. It is highly expressed on neuroblastomas but not detectable at appreciable levels in normal childhood tissues. In a seminal paper published in Cancer Cell in 2017, the CHOP team determined that GPC2 may be less susceptible to immune escape mechanisms due to its requirement for neuroblastoma proliferation. Their work validated GPC2 as a nonmutated neuroblastoma oncoprotein and established a strong foundation for GPC2 as an immunotherapeutic target. "This new collaboration builds upon our prior studies and pairs Tmunity’s experience in engineering and developing CAR-T cells with CHOP’s expertise in the biology of this family of cell surface molecules across neuroblastoma and several other cancers," said Dr. Bosse, an Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and CHOP.

"The collaboration with CHOP expands our network of world-class academic collaborators and progresses our strategy to build a pediatric oncology franchise and advance novel T-cell therapies for devastating diseases. By combining the scientific insights and research at CHOP with the T-cell development, regulatory, and manufacturing capabilities and expertise at Tmunity, we created a formidable team to attack this deadly cancer," said Usman "Oz" Azam, MD, President and Chief Executive Officer of Tmunity. "We look forward to working with Dr. Maris, Dr. Bosse, and their teams to advance GPC2 T-cell therapy and offer hope to patients with limited options."

Neuroblastoma is a rare cancer that affects the development of the nervous system by attacking immature nerve cells as early as the embryonic stage. Each year, about 800 children are diagnosed with neuroblastoma in the U.S., and overall survival rates are less than 50%. Current chemotherapy options for these patients are limited and highly toxic, which highlight the urgent need for new treatments for this disease, which improve overall survival and reduce long-term toxicity.

"We are excited to collaborate with Tmunity to accelerate the development of GPC2 for neuroblastoma," said John Maris, MD, co-head of the Pediatric Cancer Dream Team and Giulio D’Angio Chair in Neuroblastoma Research at CHOP. "Our studies have confirmed that GPC2 is an important target in neuroblastoma and other aggressive malignancies. We look forward to one day bringing, GPC2 T-cell therapies to the clinic and to patients in need."

Under the terms of the agreement, Tmunity will collaborate with CHOP to initiate IND-enabling studies and advance quickly into the clinic. Tmunity will be responsible for leading the development, manufacturing, regulatory, and commercialization of the GPC2 T-cell therapy. The majority of the preclinical studies will be performed by CHOP. Deal terms were not disclosed.

On October 1, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported that Gaurav Shah, M.D., Chief Executive Officer and President of Rocket is scheduled to present on Tuesday, October 8, 2019, at 11:00 a.m. Eastern Time at the Chardan 3rd Annual Genetic Medicines Conference, New York, N.Y (Press release, Rocket Pharmaceuticals, OCT 1, 2019, View Source [SID1234539991]).

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On October 1, 2019 Numab Therapeutics AG (Numab) reported the initiation of a partnership with Eisai, Co., Ltd (Eisai), under a research and option agreement, to discover and develop novel multi-specific antibody immunotherapies for cancer, using Numab’s proprietary MATCH platform (Press release, Numab, OCT 1, 2019, View Source [SID1234539990]).

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Under the terms of the agreement, Eisai has the option to acquire an exclusive license to develop and commercialize novel multi-specific antibody-based molecules that emerge from their research collaboration with Numab. In exchange, Numab will receive from Eisai an upfront payment and research funding and is eligible to receive success fees, milestone payments and tiered royalties on sales.

Dr. David Urech, CEO of Numab, said: "We are excited to enter into this partnership with Eisai, a company with a distinguished track-record of providing innovative treatment options for patients suffering from cancer. We look forward to collaborating with Eisai and applying Numab’s MATCH platform with the goal of leveraging multi-specific technology to generate cancer therapeutics that provide superior efficacy and safety compared to benchmark immunotherapies."

Dr. Takashi Owa, Vice President, Chief Medicine Creation and Chief Discovery Officer, Oncology Business Group at Eisai, said: "We highly value this partnership with Numab, whose versatile technology platform can produce plug-and-play multi-specific immunotherapies with outstanding biophysical properties and efficacy-to-safety profiles. Like Numab, we believe that engineering multi-specific drugs is a very promising strategy to enhance patient responses and overcome several limitations faced by mono-specific drugs and combinations thereof. We are excited to be working together with Numab to potentially bring next-generation antibody drugs to patients."

On October 1, 2019 Servier, an independent international pharmaceutical company, reported the acquisition of PIXUVRI from CTI BioPharma (Press release, Servier, OCT 1, 2019, View Source [SID1234539989]). PIXUVRI is a treatment for adult patients with multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphoma. Servier and CTI Biopharma completed an Asset Purchase Agreement which transferred worldwide rights of PIXUVRI to Servier. Servier commercialized PIXUVRI globally, in all countries where the drug was approved under an exclusive license from CTI BioPharma.

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"The acquisition of PIXUVRI is an important step towards Servier’s long-term strategy to become a key player in oncology. Within oncology, one of our key focuses is hematology, and we now have two medicines that are marketed globally alongside a strong and innovative pipeline of drug candidates which includes CAR-T therapies," said Claude Bertrand, Executive VP, Global Head of R&D at Servier. "As part of our strategy, we are committed to invest 50% of our R&D budget in oncology."

Non-Hodgkin lymphoma (NHL) is a blood cancer that affects the lymphatic system, it can occur in different parts of the body from the lymph nodes in the neck to the liver or spleen, but also in other organs such as the stomach, small bowel, bones, brain, testicles or skin.1 Globally there are over 500,000 new cases of NHL a year; it is both the 11th most commonly diagnosed cancer and cause of cancer death.2

"We have a strong commitment to providing effective solutions for patients living with cancer. PIXUVRI is an effective treatment that received standard European marketing authorization in June, and today marks a new step towards achieving Servier’s long term goals. We will ensure that patients will continue to have access to PIXUVRI", said Eric Falcand, VP, Global Head of Business Development & Licensing at Servier. "We are continuing to strengthen our oncology portfolio through our R&D efforts and strategic alliances."

#ENDS#

About PIXUVRI (pixantrone)

PIXUVRI is a cytotoxic medicine that works by interfering with the DNA within cells and preventing them from making more copies of DNA. This means that the cancer cells cannot divide and eventually die.3 PIXUVRI is indicated in the European Union as monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphoma.3 PIXUVRI is also approved in Iceland, Israel, Liechtenstein, Myanmar, Norway, Pakistan, Russia and Ukraine.

PIXUVRI is mentioned in the ESMO (Free ESMO Whitepaper) guidelines as an anthracycline-like drug with reduced cardiotoxicity, which demonstrated some efficacy in heavily treated patients.4

More detail is available in the summary of the European public assessment report (EPAR) on the EMA website at www.ema.europa.eu.