On February 5, 2019 Exicure, Inc. (OTCQB: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported four of the clinical trial sites for the Company’s Phase 1b/2 trial of AST-008 (Press release, Exicure, FEB 5, 2019, View Source [SID1234533074]).

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"Exicure is thrilled to announce the four initial sites for the Phase 1b/2 clinical trial to evaluate our immune system activating spherical nucleic acid in combination with Keytruda for the treatment of solid tumors," said Dr. David Giljohann, Chief Executive Officer of Exicure. "We are encouraged by the results of our Phase 1 clinical trial, which demonstrated that our drug is well-tolerated and activates key immune cells and signals. Eligible patients will be able to enroll in trial sites in the US."

The open-label Phase 1b/2 trial will begin with a dose finding Phase 1b stage in combination with the anti-PD-1 therapy pembrolizumab (KEYTRUDA), followed by a Phase 2 expansion stage. In the Phase 1b, Exicure will enroll patients with superficial injectable tumors and will prioritize those with Merkel cell carcinoma, cutaneous squamous cell carcinoma, melanoma, and squamous cell carcinoma of the head and neck. Preliminary data from the Phase 1b stage of the trial are expected in late 2019.

The clinical trial sites are as follows:

Dana Farber Cancer Institute (Boston, Massachusetts)
Holden Comprehensive Cancer Center at the University of Iowa (Iowa City, Iowa)
John Wayne Cancer Institute at Providence St. John’s Health Center (Santa Monica, California)
Sylvester Comprehensive Cancer Center at the University of Miami (Miami, Florida)
AST-008 is a toll-like receptor nine (TLR9) activator being developed by Exicure. More information on the Phase 1b/2 trial of AST-008 can be found at clinicaltrials.gov, under identifier number NCT03684785.

KEYTRUDA (Pembrolizumab; Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.) is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

On February 5, 2019 Personalis, Inc., a leader in advanced genomics for precision oncology, reported that they are scheduled to present at the upcoming Immuno-Oncology 360° Conference in New York on Thursday, February 7, 2019 at 3:50 PM, EST (Press release, Personalis, FEB 5, 2019, View Source [SID1234534800]).

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The presentation, entitled "A Universal Cancer Immunogenomics Solution for Biomarker Discovery," will discuss the current challenges facing investigators in immuno-oncology translational research and explore how the Personalis Platform overcomes the limitations associated with conventional NGS approaches that are used in translational research and the clinical development of new oncology therapeutics. The presentation will also introduce Personalis’ new ImmunoID NeXT Platform.

ImmunoID NeXT is the first and only platform to provide comprehensive analysis of both a tumor and its microenvironment from a single sample. The platform can be used to investigate the key tumor- and immune-related areas of cancer biology; consolidating multiple oncology biomarker assays into one. This maximizes the biological information that can be generated from a precious tumor specimen.

The presentation will be delivered by Kedar Hastak, PhD, Field Applications Scientist for Personalis.

On February 5, 2019 The Mundipharma network of independent associated companies reported the launch of Pelmeg (pegfilgrastim), a biosimilar of Neulasta following European Commission (EC) approval in November 2018 (Press release, Mundipharma, FEB 5, 2019, View Source [SID1234533075]).2 Pelmeg is the fourth biosimilar medicine to be commercialised by Mundipharma, expanding its portfolio and commercial footprint across Europe. It was developed by Cinfa Biotech which was acquired by Mundipharma and announced in October 2018.

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Now available in Germany, the Netherlands and Ireland, Pelmeg is indicated for the reduction of the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).1

"We are delighted that Pelmeg is now available in these countries. The launch of the treatment builds on our proven commercial excellence in biosimilars over the past four years. Pelmeg has the potential to play an important role in improving the lives of patients affected by chemotherapy induced neutropenia and febrile neutropenia," said Philippe Bastide, Head of Biosimilars, Europe. "Through our partnership with Celltrion, we estimate Remsima and Truxima have already saved European healthcare systems approximately €330m.3* If all patients currently being treated with the reference product are offered access to Pelmeg, further significant savings can be realised for the healthcare community."

The acquisition of Cinfa Biotech provides Mundipharma with global reach and expanded development capabilities. Mundipharma will continue to leverage partnerships to develop its expanding portfolio of Biosimilars, reinforcing its leadership in Europe and extending its geographical footprint.

*Figures relate to data from 2015-2017

Notes to editors

About the clinical data

A comprehensive analytical, biofunctional, preclinical and clinical comparability programme has demonstrated a high degree of similarity between Pelmeg and Neulasta.4 Its biosimilarity has been studied in healthy volunteers who have no comorbidities, require no co-medication and are immunocompetent.5,6,7

The data:

Confirmed biosimilarity to Neulasta in sensitive clinical study settings
Demonstrated pharmacokinetic comparability to Neulasta at the clinical dose of 6 mg
Demonstrated pharmacodynamic comparability to Neulasta at the clinical dose of 6 mg and at the reduced dose of 3 mg
Did not show any clinically meaningful differences in the safety and immunogenicity profile compared to Neulasta
About Pelmeg

It is a pegfilgrastim biosimilar.1 Pegfilgrastim is a pegylated version of granulocyte-colony stimulating factor (G-CSF) that works by stimulating the bone marrow to produce more neutrophils, thereby reducing the duration of neutropenia and the incidence of febrile neutropenia. It is administered as a subcutaneous injection once per chemotherapy cycle, at least 24 hours after cytotoxic chemotherapy.1

The approval of Pelmeg was based on a robust regulatory submission of rigorous analytical, biofunctional, preclinical and clinical studies to demonstrate biosimilarity in terms of its quality, safety and efficacy profile compared with the reference pegfilgrastim.4 As such, it is indicated in the exact same way as subcutaneous (pre-filled syringe) Neulasta.

Most standard-dose chemotherapy regimens are associated with 6–8 days of neutropenia, and febrile neutropenia is observed in approximately 8 cases per 1000 patients receiving cancer chemotherapy. People with febrile neutropenia caused by chemotherapy treatment for cancer are at increased risk of severe infection and death.8

About neutropenia and febrile neutropenia

People taking chemotherapy for cancer are at risk of dangerously low levels of a type of white blood cell called a neutrophil. Neutrophils play an important role in the immune system guarding against infection. Febrile neutropenia is a low level of neutrophils in the blood accompanied by a fever.9

On February 4, 2019 Five Prime Therapeutics, Inc. (NASDAQ:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics reported that Aron Knickerbocker, Chief Executive Officer, will present at the Guggenheim Healthcare Talks Idea Forum on Thursday, February 14, 2019 at 2:00 pm PT (Press release, Five Prime Therapeutics, FEB 4, 2019, View Source [SID1234533041]).

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The presentation will be webcast and may be accessed at the "Events & Presentations" section of the Company’s website at: View Source Five Prime will maintain an archived replay of the webcast on its website for 30 days after the conference.

On February 4, 2019 BioLineRx Ltd. (NASDAQ/TASE:BLRX), a clinical-stage biopharmaceutical company focused on oncology, reported that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead oncology candidate, BL-8040, for the treatment of pancreatic cancer (Press release, BioLineRx, FEB 4, 2019, View Source [SID1234533026]).

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"Orphan Drug Designation in pancreatic cancer is a very important milestone in the development plan of BL-8040, and joins previously approved orphan designations by the FDA for BL-8040 in AML and stem-cell mobilization," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Despite advances in the treatment of various cancers with immune checkpoint inhibitors, pancreatic cancer is refractory to these treatment options, and remains an area of significant unmet medical need. We have previously reported encouraging clinical data supporting the potential of BL-8040 as part of an immunotherapy combination treatment in pancreatic cancer, and we look forward to top-line results from our ongoing pancreatic clinical studies later this year."

BL-8040 is currently being investigated in clinical studies for the treatment of pancreatic cancer under two separate immuno-oncology collaborations – one with Merck & Co., Inc., Kenilworth, N.J., USA (known as MSD outside the United States and Canada), and a second collaboration with Genentech, a member of the Roche Group.

Orphan Drug Designation by the FDA entitles BioLineRx to seven years of market exclusivity for the use of BL-8040 for the treatment of pancreatic cancer, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

About BL-8040
BL-8040 is a short synthetic peptide for the treatment of hematological malignancies, solid tumors, and stem cell mobilization. It functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in many human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells and immune-cells, sensitization of cancer cells to chemo- and bio-based anti-cancer therapies, and direct anti-cancer effect by inducing programmed cell death (apoptosis). BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Pancreatic Cancer
Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. Its incidence rate in the US is estimated at 3.2% of new cancer cases. Each year, about 185,000 individuals globally are diagnosed with this condition, and an estimated 55,000 individuals were diagnosed with pancreatic cancer in the US during 2018. Symptoms are usually non-specific and as a result, pancreatic cancer is often not diagnosed until it reaches an advanced stage. Surgical resection does not offer adequate treatment since only 20% of patients have resectable tumors at the time of diagnosis. Even among patients who undergo resection for pancreatic cancer and have tumor-free margins, the five-year survival rate is only 10%-25%. The overall five-year survival rate among pancreatic cancer patients is 7-8%, which constitutes the highest mortality rate among solid tumor malignancies. The overall median survival is less than one year from diagnosis, highlighting the need for the development of new therapeutic options.

Despite advances in chemotherapeutics and immunotherapy, increases in median and overall survival rates in pancreatic cancer have been modest. Pancreatic cancer remains an area of unmet medical need, with no new approved therapies since the approval of nab-paclitaxel in combination with gemcitabine (Abraxane) for first-line treatment in 2013 and Onivyde in combination with fluorouracil and leucovorin for second-line treatment in 2015. The limited clinical benefits demonstrated by these existing standard treatment options reinforce the need for additional approaches.