On November 4, 2020 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported six clinical and preclinical abstracts related to acute myeloid leukemia (AML) and flotetuzumab, an investigational bispecific CD123 × CD3 DART molecule, and one abstract related to tebotelimab, an investigational bispecific PD-1 × LAG-3 DART molecule, to be presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, December 5-8, 2020 (Press release, MacroGenics, NOV 4, 2020, View Source [SID1234569852]).

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"We look forward to presenting clinical and biomarker results for flotetuzumab in patients with relapsed or refractory acute myeloid leukemia at the 2020 ASH (Free ASH Whitepaper) annual meeting," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "The first of our two oral presentations will address results of the role of immune senescence and exhaustion-related RNA profiles in predicting outcomes in AML. The second oral presentation will provide the results on the use of flotetuzumab as salvage therapy in AML patients who failed induction therapy or experienced an early relapse. Together with our four posters, these presentations add to the growing medical and scientific interest in the potential of flotetuzumab in AML. Finally, we are very pleased to present data from the cohort of diffuse large B-cell lymphoma (DLBCL) patients who were treated in the dose expansion study of tebotelimab."

Oral Presentations

An Immune Senescence and Exhaustion-related RNA Profile Predicts Clinical Outcomes in Acute Myeloid Leukemia
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: Single Cell Profiling and Novel molecular Markers
Session Date: Saturday, December 5, 2020
Session Time: 7:30 AM – 9:00 AM
Presentation Time: 8:15 AM
Flotetuzumab as Salvage Therapy for Primary Induction Failure and Early Relapse Acute Myeloid Leukemia
Session Name: 613. Acute Myeloid Leukemia: Novel Therapies and Treatment Approaches
Session Date: Sunday, December 6, 2020
Session Time: 9:30 AM – 11:00 AM
Presentation Time: 9:45 AM

Poster Presentations

Prophylactic Ruxolitinib for Cytokine Release Syndrome (CRS) in Relapse/Refractory (R/R) AML Patients Treated with Flotetuzumab
Session Name: 613. Acute Myeloid Leukemia: Clinical Studies: Poster III
Date: Monday, December 7, 2020
Tp53 Abnormalities Correlate with Immune Infiltration and Associate with Response to Flotetuzumab Immunotherapy in Acute Myeloid Leukemia
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: Poster II
Date: Sunday, December 6, 2020
Immune Senescence and Exhaustion Correlate with Response to Flotetuzumab, An Investigational CD123 × CD3 Bispecific DART Molecule, In Acute Myeloid Leukemia
Session Name: 617. Acute Myeloid Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: Poster III
Date: Monday, December 7, 2020
Flotetuzumab and other Cellular Immunotherapies Upregulate MHC Class II Expression on Acute Myeloid Leukemia Cells In Vitro and In Vivo
Session Name: 704. Immunotherapies: Poster III
Date: Monday, December 7, 2020
A Phase 1, Open-Label Study of MGD013, a Bispecific DART Molecule Binding PD-1 and LAG-3 in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma
Session Name: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster III
Date: Monday, December 7, 2020
The above abstracts were published today on the ASH (Free ASH Whitepaper) website at View Source

About Flotetuzumab

Flotetuzumab (also known as MGD006) is a clinical-stage bispecific, investigational DART molecule that recognizes both CD123 and CD3. CD123, the interleukin-3 receptor alpha chain, has been reported to be over-expressed on malignant cells in AML and other hematologic malignancies. The primary mechanism of action of flotetuzumab is believed to be its ability to redirect T lymphocytes to kill CD123-expressing cells. To achieve this, the DART molecule combines a portion of an antibody recognizing CD3, an activating molecule expressed by T cells, with an arm that recognizes CD123 on the target cells. MacroGenics is conducting a single-arm, registration-enabling clinical study to evaluate flotetuzumab in up to 200 patients with primary induction failure / early relapse (PIF/ER) AML, with complete remission (CR) and CR with partial hematological recovery (CRh) as the primary endpoint. The study will be conducted as a continuation of the ongoing Phase 1/2 study (NCT02152956). The FDA has granted orphan drug designation to flotetuzumab for the treatment of AML.

About Tebotelimab

Tebotelimab (also known as MGD013) is a clinical-stage bispecific, investigational DART molecule designed to independently or coordinately block PD-1 and LAG-3 checkpoint molecules to sustain or restore the function of exhausted T cells for the treatment of cancer. Data from the Phase 1 dose escalation study in cohorts of patients with advanced solid tumors at escalating flat doses of 1-1200 mg every two weeks were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program. A maximum tolerated dose was not identified. At the same meeting, it was reported that LAG-3 expression on immune effector cells was shown to be enhanced by margetuximab, MacroGenics’ investigational Fc-engineered monoclonal antibody targeting HER2. Given the early signal of activity and acceptable safety profile observed in an initial, small expansion cohort of patients, the Company is evaluating the combination of tebotelimab and margetuximab in patients with HER2-positive tumors. The Company believes that combining Fc-engineering and checkpoint blockade has the potential to engage both innate and adaptive immune responses against a broad range of tumors with varied tumor microenvironments. For more information about the study design, please visit ClinicalTrials.gov (NCT03219268).

On November 4, 2020 NanOlogy LLC, a clinical-stage oncology company advancing intratumoral therapy for solid tumors,reported that has enrolled the first patient in a Phase 2 clinical trial of NanoPac (sterile nanoparticulate paclitaxel) for suspension via intratumoral injection for local prostate cancer (Press release, NanOlogy, NOV 4, 2020, View Source [SID1234569851]). The single arm trial is evaluating up to 3 monthly injections of the investigational drug in patients scheduled for prostatectomy approximately 90 days after the first injection. Patients will be followed for safety and tumor response. Immune effects will be evaluated via multiplex immunohistochemistry and flow cytometry.

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Craig G. Rogers, MD (Henry Ford Cancer Institute), a clinical investigator for the Phase 2 trial, commented: "NanoPac completed a first-in-human clinical safety trial in 2019. In this innovative second trial, we will be evaluating both safety and efficacy of multiple intratumoral injections of NanoPac in patients with prostate cancer. We will also be analyzing tissue and blood for immune response and what therapeutic potential NanoPac may offer patients suffering from local disease. This trial aligns with our vision of offering precision medicine approaches along with cutting edge clinical trials at Henry Ford Cancer Institute."

The multicenter Phase 2 trial expects to enroll up to 18 patients with localized prostate cancer (tumors classified as

This second clinical trial follows completion of a first-in-human phase 2a dose-rising trial (NCT03077659) at a single institution to establish the safety of a single focal injection of NanoPac into the tumor-bearing lobe of patients scheduled for prostatectomy. Selected findings from the first trial:

No drug related SAEs including no prostatitis reported
Highest dose up to 75mg of NanoPac well tolerated
Mean reductions in tumor volume, PSA-density, and percent adenocarcinoma in biopsy
Peak paclitaxel plasma concentration was well below toxicity threshold at all timepoints, while drug was detected in all prostatectomy tissue specimens following prostatectomy.
A summary of study findings submitted to FDA can be found under NCT03077659 in the clinicaltrials.gov database.

Prostate cancer affects about 3 million men in the USA with 191,930 new cases and 33,330 deaths estimated for 2020 by the American Cancer Society. Patients at higher risk for disease progression may face surgical removal of the prostate or radiation therapy. Unfortunately, these patients may suffer incontinence or impotence, which significantly decreases quality of life. If the cancer becomes metastatic, patients have an estimated 5-year survival rate of only 31%.

In addition to prostate cancer, NanOlogy clinical programs are advancing in other genitourinary cancers, as well as gastrointestinal, peritoneal, lung, and dermal cancers. Data from preclinical and clinical studies in a variety of solid tumors have shown persistent tumor kill, antitumoral immune response, and minimal local or systemic toxicity.

The NanOlogy large surface area particle therapeutic platform is based on a proprietary supercritical precipitation technology that converts taxane API crystals into stable particles of pure drug with disproportionate size to surface area ratio. The particles are covered by two composition of matter patents (US 9,814,685) and (10,507,195) both valid until 2036 in the US and pending globally, forming the foundation of an extensive intellectual property portfolio protecting the investigational drugs and technology.

On November 4, 2020 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that three abstracts for CA-4948, a small molecule IRAK4 inhibitor, have been accepted for oral and poster presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper) which will be held virtually from December 5-8, 2020 (Press release, Curis, NOV 4, 2020, View Source [SID1234569850]).

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"We are pleased with the progress to date for our first-in-class IRAK4 kinase inhibitor, CA-4948 and are on track to report data by year-end for both our Phase 1 study in patients with non-Hodgkin’s lymphoma and our Phase 1 study in patients with acute myeloid leukemia and myelodysplastic syndromes," said James Dentzer, President and Chief Executive Officer of Curis. "In addition to the data published in the abstracts this morning, we look forward to providing updated safety and efficacy data at ASH (Free ASH Whitepaper) from both studies."

Details of the presentations are as follows:
Oral Presentation:

Title: Safety, Pharmacokinetics and Activity of CA-4948, an IRAK4 Inhibitor, for Treatment of Patients with Relapsed or Refractory Hematologic Malignancies: Results from the Phase 1 Study
Author: Grzegorz S. Nowakowski, MD, Mayo Clinic
Session Name: 623. Mantle Cell and Indolent B-Cell Lymphoma – CAR-T and immunotherapy clinical studies
Session Date & Time: Monday, December 7, 2020, 1:30 pm – 3:00 pm ET
Presentation Time: 2:15 pm
Poster Presentation

Title: A Phase 1, Open Label Dose Escalation Trial Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of Orally Administered CA-4948 in Patients with Acute Myelogenous Leukemia or Myelodysplastic Syndrome
Author: Guillermo Garcia-Manero, MD, MD Anderson Cancer Center
Session Name: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III
Session Date & Time: Monday, December 7, 2020, 10:00 a.m. – 6:30 p.m. ET
Poster Presentation

Title: A Multi-Center, Dose-Finding Study to Assess Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of a novel IRAK4 inhibitor CA-4948 in combination with ibrutinib, in Patients with Relapsed or Refractory Hematologic Malignancies
Author: Grzegorz S. Nowakowski, MD, Mayo Clinic
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma —Clinical Studies: Poster III
Session Date & Time: Monday, December 7, 2020, 10:00 a.m. – 6:30 p.m. ET
Additional meeting information can be found on the ASH (Free ASH Whitepaper) website at www.hematology.org/Annual-Meeting/. Each presentation will also be available under "Events and Presentations" in the Investors section of the Company’s website at www.curis.com

On November 4, 2020 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage biotechnology company focused on discovering and developing novel small-molecule drugs for the treatment of cancer and other life-threatening diseases, reported the expansion of the company’s ongoing clinical trial evaluating telaglenastat in combination with Pfizer’s CDK 4/6 inhibitor palbociclib (IBRANCE) (Press release, Calithera Biosciences, NOV 4, 2020, View Source [SID1234569849]). This Phase 1/2 study, which is being conducted by Calithera, will be expanded to include an additional cohort of patients with pancreatic ductal adenocarcinoma (PDAC) whose tumors harbor mutations in both KRAS and CDKN2A.

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"There is a strong rationale to target KRAS and CDKN2A mutated tumors with the combination of palbociclib and telaglenastat," said Susan Molineaux, PhD, president and chief executive officer of Calithera. "Mutations in CDKN2A lead to upregulation of both CDK4/6 activity and glutamine utilization in cancer cells. By inhibiting these activities simultaneously with the telaglenastat-palbociclib combination, we hope to have a measurable impact on pancreatic cancer, which still has very few viable treatment options."

Telaglenastat blocks glutamine consumption in tumor cells, which, due to specific genetic alterations such as mutations in KRAS and CDKN2A, often become dependent on increased metabolism of glutamine. Approximately 50 percent of PDAC patients harbor mutations in both KRAS and CDKN2A. In preclinical studies with KRAS-mutated cancer models, telaglenastat showed synergistic anti-tumor effects when used in combination with CDK4/6 inhibitors, such as palbociclib, enhancing cell cycle arrest and blocking cancer cell proliferation. In the ongoing Phase 1/2 clinical trial (NCT03965845), encouraging efficacy and safety of the combination was observed in PDAC patients treated in the dose escalation phase of the trial.

The new cohort of the Phase 1/2 clinical trial will be evaluating the safety and anti-tumor activity of telaglenastat in combination with palbociclib in patients with advanced, metastatic PDAC whose tumors harbor mutations in both KRAS and CDKN2A. The ongoing Phase 1/2 trial is currently enrolling patients with colorectal cancer and non-small cell lung cancer whose tumors harbor mutations in KRAS.

On November 4, 2020 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that three abstracts including data from the Company’s clinical and research portfolio have been accepted for oral presentations and two abstracts have been accepted for poster presentations at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held virtually December 5-8, 2020 (Press release, GlycoMimetics, NOV 4, 2020, View Source [SID1234569848]).

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Of particular note are primary and key secondary endpoint data from additional analysis of the Phase 3 RESET study evaluating the efficacy of rivipansel in VOC. These findings point to the potential benefits, clinical improvements and improved outcomes associated with early treatment with GlycoMimetics’ wholly-owned product candidate, which include shorter hospital stays for patients and reduced need for IV opioids to treat pain.

A second presentation includes data from two different preclinical models of VOC using GlycoMimetics’ highly potent and specific E-selectin antagonist, GMI-1687. This presentation will highlight the product candidate’s potential for intravenous and subcutaneous administration to treat VOC by inhibiting occlusions and restoring blood flow.

A third presentation discloses how targeting E-selectin with uproleselan may help patients with AML overcome resistance to venetoclax combined with hypomethylating agent (HMA) based therapy.

Poster presentations convey how GMI-1359, a rationally-designed small molecule that inhibits E-selectin and CXCR4 (a chemokine receptor), enhances sorafenib’s anti-leukemia effect in pre-clinical AML models; and how GMI-1359 can uniquely generate motility-enhancing signals in AML cells and deplete AML cells from protective vascular niches in the bone marrow.

"GlycoMimetics is honored to have five abstracts from across our product candidate portfolio selected for presentations at this year’s ASH (Free ASH Whitepaper) meeting. The data convey new insights into the critical role of E-selectin in both malignant and inflammatory, adhesion-mediated conditions, suggesting novel treatment options for unmet need in sickle cell disease and AML," said Helen Thackray, MD, FAAP, GlycoMimetics’ Chief Medical Officer.

Details on GlycoMimetics presentations at the ASH (Free ASH Whitepaper) Meeting are as follows:

Title: Restoration of Normal Blood Flow in Mouse Models of Sickle Cell Vaso-occlusion Following Intravenous or Subcutaneous Administration of a Highly Potent E-Selectin Specific Inhibitor.
Presenters: Madhan Thamilarasan, William E. Fogler, John L. Magnani and Rahima Zennadi.
Session: 113 Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: New Insights Into Sickle Cell Disease Pathophysiology.
Date and Time: December 5, 2020. 2:00 – 3:30 p.m. EST
Presentation Time: 2:45 p.m.

Title: Targeting E-selectin with GMI-1271 Overcomes Microenvironment-mediated Resistance to Venetoclax/HMA Therapy.
Presenters: Kyung Hee Chang, Muharrem Muftuoglu, Weiguo Zhang, Mahesh Basyal, Lauren Ostermann, William E. Fogler, John L. Magnani and Michael Andreeff.
Session: 604 Molecular Pharmacology and Drug Resistance in Myeloid Diseases.
Date and Time: Saturday, December 5, 2020. 2:00 – 3:30 p.m.
Presentation Time: 3:15 p.m.

Title: Dual CXCR4 and E-selectin Inhibition (GMI-1359) Rapidly Increases AML Cellular Motility Prior to Intravasation and Vascular Niche Depletion Observed by Intravital Bone Marrow 2-Photon Microscopy.
Presenters: Tomasz Zal, M. Anna Zal, Mateusz Rytelewski, Kyung Hee Chang, Rodrigo Jacamo, William E. Fogler, John L. Magnani and Michael Andreeff.
Session: 616 Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster II.
Date: Sunday, December 6, 2020.
Virtual Poster Hall: 7 a.m. – 3:30 p.m.

Title: Combined Blockage of E-selectin and CXCR4 (GMI-1359) Enhances Anti-Leukemia Effect of FLT3 Inhibition (Sorafenib) and Protects Hematopoiesis in Pre-clinical AML Models.
Presenters: Weiguo Zhang, Kyung Hee Chang, Mahesh Basyal, Yannan Jia, Lauren Ostermann, William E. Fogler, John L. Magnani, M. Anna Zal, Tomasz Zal and Michael Andreeff.
Session: 616 Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III.
Date: Monday, December 7, 2020.
Virtual Poster Hall: 7 a.m. – 3:30 p.m.

Title: Early Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: Reset Clinical Trial Analysis
Presenter: Helen Thackray
Session Name: 114 Hemoglobinopathies, Excluding Thalassemia—Clinical: Novel Treatments for Sickle Cell Disease
Date and Time: Monday, December 7, 2020. 1:30 – 3:00 p.m.
Presentation Time: 1:45 p.m.

The accepted abstracts are available online through the ASH (Free ASH Whitepaper) meeting website, View Source

About Rivipansel

Rivipansel, the company’s wholly-owned glycomimetic drug candidate that binds to all three members of the selectin family (E-, P- and L-selectin), was GlycoMimetics’ first candidate to enter clinical development. After the Phase 3 RESET trial conducted by Pfizer, GlycoMimetics’ former collaborator, did not meet its primary or key secondary efficacy endpoints in 2019, new efficacy data from an additional analysis of rivipansel were published in June 2020 and subsequently presented at the Foundation for Sickle Cell Disease Research Meeting in September 2020. GlycoMimetics is engaging with the FDA to identify what, if any, next steps to take, with a focus on determining if there is a potential streamlined path forward for this asset in sickle cell disease.

About GMI-1687

Discovered and developed by GlycoMimetics, GMI-1687 is a highly-targeted, highly-potent E-selectin antagonist. It has been shown in preclinical studies to be bioavailable via subcutaneous administration. At the 2018 Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), data presented in a poster about GMI-1687 pointed to the potential for a life-cycle extension for GlycoMimetics’ uproleselan. The investigational drug has also been shown to represent a more highly-potent and subcutaneously bioavailable potential life-cycle extension for rivipansel.

About Uproleselan (GMI-1271)

Discovered and developed by GlycoMimetics, uproleselan is an investigational, first-in-class, targeted inhibitor of E-selectin. Uproleselan (yoo’ pro le’ sel an), currently in a comprehensive Phase 3 development program in AML, has received Breakthrough Therapy Designation from the U.S. FDA for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed or refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better-than-expected remission rates and overall survival compared to historical controls, which have been derived from results from third-party clinical trials evaluating standard chemotherapy, as well as lower-than-expected induction-related mortality rates. Treatment in these patient populations was generally well-tolerated, with fewer than expected adverse effects.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer, as well as in osteosarcoma, a rare pediatric tumor. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. The Duke University Phase 1b clinical study in breast cancer patients is designed to enable investigators to identify an effective dose of the drug candidate and to generate initial biomarker data around the drug’s activity. GMI-1359 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for the treatment of osteosarcoma, a rare cancer affecting about 900 adolescents a year in the United States.