On June 14, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that ELZONRISTM (tagraxofusp; SL-401) will be the subject of three clinical presentations, including an oral presentation on the pivotal BPDCN program (Press release, Stemline Therapeutics, JUN 14, 2018, View Source [SID1234527314]). Updated data from the ongoing Phase 2 trial in chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF) will also be presented. Presentations will be delivered tomorrow, Friday, June 15th at the 23rdCongress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, Sweden.

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Details on the presentations are listed below. Presentations will be available on the Stemline website (www.stemline.com), Scientific Presentations tab, after their delivery.

Results of Pivotal Phase 2 Trial of SL-401 in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm

Abstract: S116
Session: Miscellaneous Treatments in AML
Presenter: Naveen Pemmaraju, MD; MD Anderson Cancer Center
Oral Presentation: Friday, June 15; 11:45 – 12:00 CEST (5:45 AM – 6:00 AM ET)
Location: Room A4
Results from Ongoing Phase 1/2 Trial of SL-401 in Patients with Intermediate or High Risk Relapsed/Refractory Myelofibrosis

Abstract: PF618
Session: Myeloproliferative neoplasms – Clinical
Poster Presentation: Friday, June 15; 17:30 – 19:00 CEST (11:30 AM – 1 PM ET)
Location: Poster Area
Results from Ongoing Phase 1/2 Trial of SL-401 in Patients with Relapsed/Refractory CMML

Abstract: PF626
Session: Myeloproliferative neoplasms – Clinical
Poster Presentation: Friday, June 15; 17:30 – 19:00 CEST (11:30 AM – 1 PM ET)
Location: Poster Area
About ELZONRISTM (tagraxofusp; SL-401)
ELZONRISTM (tagraxofusp; SL-401) is a novel targeted therapy directed to CD123, a cell surface receptor expressed on a range of malignancies. ELZONRIS has successfully completed a pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN), an indication for which it was granted Breakthrough Therapy Designation (BTD). A rolling Biologics License Application (BLA) submission is underway. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

On June 14, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has successfully completed the comprehensive characterization of its proprietary cell clone known as 22P1G (Press release, PharmaCyte Biotech, JUN 14, 2018, View Source [SID1234527315]).

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The 22P1G cells constitute the cells in the Master Cell Bank (MCB) that were prepared and tested by PharmaCyte’s contractor, Eurofins Lancaster Laboratories. The cells from the MCB will serve as the active pharmaceutical ingredient (API) in the company’s Cell-in-a Box capsules that will be used (together with low doses of the cancer prodrug ifosfamide) for the treatment of locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) in its planned clinical trial.

The comprehensive characterization studies include long-term stability of the cells, and stability of the potency of the cells as a therapeutic. All studies performed are required by the U.S. Food and Drug Administration (FDA).

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, elaborated on the significance of the studies saying, "PharmaCyte is complying with all of the FDA guidelines and recommendations for all cell tests and other recent studies with the 22P1G cells. Successful completion of these studies was a pre-requisite for the approval by the FDA for us to conduct a clinical trial in patients with LAPC. Our treatment is primarily dependent upon genetically engineered live-human cells that produce a particularly potent cytochrome P450 enzyme that can activate the chemotherapy prodrug ifosfamide (clone 22P1G cells).

"With each individual batch, these cells must be stable for the long term, and the properties of the 22P1G cells must remain consistent from batch to batch. The newly completed studies provide evidence that both requirements have been met. Our pancreatic cancer treatment utilizes 22P1G cells that have been encapsulated using the Cell-in-a-Box technology. For treatment of LAPC patients, the capsules containing the cells are implanted near the pancreatic tumor so that a high local concentration of the cancer-killing ifosfamide metabolite is produced near the tumor."

On June 14, 2018 Apexigen, Inc., and Yale Cancer Center reported a clinical trial collaboration to evaluate Apexigen’s APX005M in combination with cabiralizumab and Opdivo in patients with advanced solid tumors (Press release, Apexigen, JUN 14, 2018, View Source [SID1234591001]). The Phase 1/2 clinical trial will evaluate the safety, tolerability, and preliminary activity of APX005M in combination with cabiralizumab and Opdivo in metastatic NSCLC, metastatic melanoma and RCC patients whose disease has progressed on prior anti-PD-1/PD-L1 therapy (www.clinicaltrials.gov: NCT03502330). In addition to providing funding, Bristol-Myers Squibb will supply Opdivo and cabiralizumab, an investigational antibody being developed in partnership with Five Prime Therapeutics.

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APX005M is an investigational compound that is designed to activate CD40, a key immune co-stimulatory receptor essential to regulating the activation of both innate and adaptive immune responses against cancer. Cabiralizumab (FPA008) is an antibody that inhibits colony stimulating factor-1 receptor (CSF1R) and depletes immunosuppressive tumor associated macrophages (TAMs). Preclinical data from Yale researchers and others have demonstrated that treatment with a combination of CD40 activation and inhibition of CSF-1R modifies tumor-associated macrophages and activates T cells in tumors. This results in converting a "cold" into an "inflamed" tumor microenvironment capable of eliciting protective T cell responses in tumors that are either unresponsive or insensitive to immune checkpoint blockade.

"There is an urgent need to find effective therapies for the growing number of patients who have not responded to checkpoint inhibitors," said Xiaodong Yang, M.D., Ph.D., President and Chief Executive Officer of Apexigen. "CD40 has a fundamental role in the activation of both innate and adaptive immunity, and we believe that CD40 activation by APX005M will become a key component of a number of promising new I-O therapeutic regimens for treating cancer patients."

"This most exciting collaboration between Apexigen and Yale is a result of studies with tumor bearing mice that are poorly responsive to inhibitors of PD-1/PD-L1. Based on these studies, we believe that activation of the innate immune system by APX005M in combination with cabiralizumab will enhance the activity of nivolumab, leading to a novel therapeutic approach for the increasing population of cancer patients who progress on currently approved immune checkpoint inhibitors. This is the first time this combination of drugs has been given to patients and we are eager to initiate this new clinical trial," said Harriet Kluger, M.D., Professor of Medicine at Yale Cancer Center and Principal Investigator of the trial.

On June 13, 2018 Sun BioPharma, Inc. (OTCQB:SNBP) and its wholly owned subsidiary, Sun BioPharma Australia Pty Ltd, a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with pancreatic diseases, reported that on June 4, 2018 the first patients were enrolled in a Phase 1a/1b study of SBP-101 in combination with gemcitabine and nabpaclitaxel for front-line treatment of patients with metastatic pancreatic ductal adenocarcinoma (PDA) (Press release, Sun BioPharma, JUN 13, 2018, View Source [SID1234527299]). Sun BioPharma Australia Pty Ltd, is the sponsor of this study. The Phase 1a portion of this study will treat up to 18 PDA patients in three cohorts in order to determine a recommended dose of SBP-101 to be given in combination with standard treatment. The Phase 1b portion will be an expansion at the recommended dose of SBP-101, and will guide SBP-101’s subsequent development for patients with PDA. This multi-center, front-line study has 3 sites in Australia, The Austin Health Cancer Trials Centre in Melbourne, The Adelaide Cancer Centre in Adelaide, The Blacktown Cancer and Haematology Centre in Sydney and one site in the United States, The University of Florida Health Cancer Center in Gainesville, Florida. The first patients have been enrolled at the Adelaide Cancer Centre in Adelaide, Australia under the direction of Associate Professor Dusan Kotasek and at the University of Florida Health Cancer Center in Gainesville, Florida under the direction of Thomas J. George, MD, F.A.C.P.

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Dr. Kotasek received his medical degree at the University of Adelaide and specialty training in Haemotology and Oncology at The Queen Elizabeth Hospital followed by a Fellowship at the University of Minnesota, in Minneapolis, MN, USA. A Co-Founder of the Adelaide Cancer Centre, Dr. Kotasek has published over 60 scientific papers in oncology, and he is one of Australia’s leading cancer clinical research experts. Dr. Kotasek, the Principal Investigator for this study at the Adelaide Cancer Centre commented, "Pancreatic cancer is a challenging disease with few significant options available with a meaningful impact on response rates and progression free survival. We are excited to participate in this clinical study, and to continue our evaluation of SBP-101 as front-line combination treatment for previously untreated patients with metastatic PDA."

Dr. George is a medical oncologist having graduated with honors from the University of Florida College of Medicine. He is the Director of the GI Oncology Program at the University of Florida and Associate Director of Clincial Investigation at the UF Health Cancer Center. He is the Principal Investigator for this study at the University of Florida Health Cancer Center and he commented, "We are extremely honored to be participating in this important study that was born from the discoveries made by Dr. Raymond Bergeron here at the University of Florida. Pancreatic cancer
requires us to think outside the box which is exactly what this treatment has the potential to offer our patients with PDA."

"Pancreatic cancer is a leading cause of cancer deaths in both Australia and the United States, currently being the 3rd most common cause of cancer deaths in the US. It represents a significant unmet medical need, with most patients having a poor prognosis and limited life expectancy," said Suzanne Gagnon, M.D., Chief Medical Officer at Sun BioPharma. "Our recently completed safety study in heavily pre-treated PDA patientssuggested SBP-101 could be a promising addition to current front-line treatment regimens. The Data Safety Monitoring Board, Principal
Investigators and our clinical team agreed to move directly to front-line for our second trial and we are excited to study SBP-101 in combination with standard of care chemotherapy for previously untreated metastatic PDA patients."

About SBP-101
SBP-101 is a first-in-class, proprietary, polyamine compound designed to exert therapeutic effects in a mechanism specific to the pancreas. Sun BioPharma originally licensed SBP-101 from the University of Florida Research Foundation in 2011. The molecule has been shown to be highly effective in preclinical studies of human pancreatic cancer models, demonstrating superior activity to existing FDA-approved chemotherapy agents. Combination therapy potential has also been shown for pancreatic cancer. SBP-101 is expected to differ from current pancreatic cancer
therapies in that it specifically targets the exocrine pancreas and has shown efficacy against primary and metastatic disease in animal models of human pancreatic cancer. Therefore management believes that SBP-101 may effectively treat both primary and metastatic pancreatic cancer, while leaving the insulin-producing islet cells and non-pancreatic tissue unharmed. The safety and metabolic profile demonstrated in our first-in-human safety study further supports
evaluation of the potential for additive or synergistic effects in combination with current standard pancreatic cancer treatment

On June 13, 2018 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, wet age-related macular degeneration and fibrotic diseases, reported that positive preclinical data from TRC105, TRACON’s endoglin antibody, in combination with a PD-1 antibody, were discussed in an oral presentation at the 2018 International Cancer Microenvironment Society meeting (Press release, Tracon Pharmaceuticals, JUN 13, 2018, View Source [SID1234527300]).

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Dr. Mark Schoonderwoerd of Leiden University presented data from multiple murine models assessing the activity of TRC105 in combination with a PD-1 antibody. The individual antibodies as well as the combination were studied in two separate models. In the first, immunocompetent mice were implanted with a syngeneic colorectal cancer subcutaneously to mimic metastatic colorectal cancer. In the second, immunocompetent mice were implanted orthotopically to mimic advanced colorectal cancer.

In both models, treatment with either TRC105 or a PD-1 antibody alone decreased tumor volume compared to IgG control antibody to a similar degree. However, combined treatment with TRC105 and the PD-1 antibody significantly reduced tumor volume compared to treatment with TRC105 or PD-1 treatment alone.

Treatment with either TRC105 or a PD-1 antibody improved survival to a similar degree compared to control antibody. However, survival was significantly improved with combination treatment versus the individual therapies, with long-term survival demonstrated in 30% to 60% of animals. Combination treatment also increased tumor specific T cells, indicating stimulation of an immune response.

The activity of TRC105 given as a single agent or combined with a PD-1 antibody was diminished following CD8+ T cell depletion, indicating that TRC105 activity was mediated through an immunologic mechanism.
In a separately reported third preclinical study, an endoglin antibody specific for mouse endoglin that mimics TRC105 activity in mice, M1043, was studied in a chemically induced colorectal cancer model of early-stage colorectal cancer.

Combined treatment with M1043 and a PD-1 antibody significantly reduced tumor volume compared to treatment with M1043 or PD-1 treatment alone. Combination treatment also significantly reduced the number of tumors that formed in response to the chemically induced carcinogenesis.
TRC105 is currently being studied in the ongoing pivotal randomized Phase 3 TAPPAS trial in angiosarcoma (NCT02979899), the randomized Phase 2 TRAXAR trial in renal cell carcinoma (NCT01806064), a Phase 1/2 trial in patients with hepatocellular carcinoma (NCT02560779), and a Phase 1 trial studying the combination of TRC105 and nivolumab (Opdivo) in patients with non-small cell lung cancer (NCT03181308).