On November 6, 2019 IMV Inc. (Nasdaq: IMV; TSX: IMV), a clinical-stage biopharmaceutical company pioneering a novel class of immunotherapies, reported that updated results from SPiRel, a Phase 2 study of the Company’s lead program, DPX-Survivac, as a combination therapy in patients with recurrent/refractory diffuse large B-cell lymphoma (DLBCL), will be featured in a poster session at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 7-10, 2019 in Orlando, FL (Press release, IMV, NOV 6, 2019, View Source [SID1234550536]).

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Poster Presentation Details:

Poster Title: Combination of DPX-Survivac, Low Dose Cyclophosphamide, and Pembrolizumab in Recurrent/Refractory DLBCL: The SPiReL Study

Presenter: Neil Berinstein, MD, FRCPC, ABIM, Haematologist at the Sunnybrook Health Sciences Centre, Toronto, ON.

Publication Number: 3236

Session Name: 704. Immunotherapies: poster II

Date and Time: December 8, 2019, 6:00 p.m. – 8:00 p.m. EDT

Location: Orange County Convention Center, Hall B

The American Society of Hematology (ASH) (Free ASH Whitepaper) has published the official abstracts on its meeting website in advance of the ASH (Free ASH Whitepaper) Annual Meeting.

The final conference poster presentation will include additional data collected between the abstract submission on June 27, 2019 and the presentation itself. The poster will be available under Events, Webcasts and Presentations in the investors section of IMV’s website on the day of presentation.

About the SPiReL study

"SPiReL" is a Phase 2 non-randomized, open label, efficacy and safety study. Eligible subjects have recurrent/refractory DLBCL, confirmed expression of survivin are eligible for curative therapy. Study treatment includes administering two doses of 0.5 mL of DPX-Survivac 3 weeks apart followed by up to six 0.1 mL doses every 8 weeks. Intermittent low dose cyclophosphamide is administered orally at 50 mg twice daily for 7 days followed by 7 days off. Pembrolizumab 200 mg is administered every 3 weeks. Study participants continue active therapy for up to one year or until disease progression, whichever occurs first.

The primary objective of this study is to document the response rate to this treatment combination using modified Cheson criteria. Secondary objectives include duration of response and safety. Exploratory endpoints include T cell response, tumor immune cell infiltration, and gene expression analysis. Enrollment is ongoing with a goal of up to 25 subjects in this multi-center study.

At the time of data cut-off for the abstract on June 27, 2019, 23 subjects have been screened and 12 have been enrolled.

About DPX-Survivac

DPX-Survivac is the lead candidate in IMV’s new class of immunotherapies that programs targeted T cells in vivo. It has demonstrated the potential for industry-leading targeted, persistent, and durable CD8+ T cell generation. IMV believes this mechanism of action (MOA) is key to generating durable solid tumor regressions. DPX-Survivac consists of survivin-based peptides formulated in IMV’s proprietary DPX drug delivery platform. DPX-Survivac is designed to work by eliciting a cytotoxic T cell immune response against cancer cells presenting survivin peptides on their surface.

Survivin, recognized by the National Cancer Institute (NCI) as a promising tumor-associated antigen, is broadly over-expressed in most cancer types, and plays an essential role in antagonizing cell death, supporting tumor-associated angiogenesis, and promoting resistance to chemotherapies. IMV has identified over 20 cancer indications in which survivin can be targeted by DPX-Survivac.

DPX-Survivac has received Fast Track designation from the U.S. Food and Drug Administration (FDA) as maintenance therapy in advanced ovarian cancer, as well as orphan drug designation status from the U.S. FDA and the European Medicines Agency (EMA) in the ovarian cancer indication.

On November 6, 2019 Kite, a Gilead Company (Nasdaq: GILD), reported the acceptance of eight abstracts, including five oral presentations, for Yescarta (axicabtagene ciloleucel) and other ongoing research from the company’s chimeric antigen receptor (CAR) T cell therapy development program, at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, in Orlando from December 7–10, 2019 (Press release, Kite Pharma, NOV 6, 2019, View Source [SID1234550535]).

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Data from Kite’s CAR T cell therapy development program to be presented at the meeting include survival data at three years from the pivotal ZUMA-1 trial of Yescarta in patients with refractory large B-cell lymphoma to be highlighted in the ZUMA-1 mechanism of relapse presentation below (Abstract #203), as well as primary results from a cohort investigating the effect of earlier steroid use with Yescarta on the rates of cytokine release syndrome and neurologic events (Abstract #243). Real-world data on Yescarta, including an analysis of post-marketing outcomes for Yescarta in large B-cell lymphoma (Abstract #764), as well as the primary analysis for the ZUMA-2 study evaluating investigational KTE-X19 in relapsed or refractory mantle cell lymphoma (Abstract #754), will also be presented.

"As we continue to lead the field in cell therapy, this year’s ASH (Free ASH Whitepaper) Annual Meeting will be a seminal one for Kite," said Christi Shaw, Chief Executive Officer of Kite. "We are excited to share three-year survival data, insights into potential adverse event management and findings from other studies that help further the understanding of Yescarta and cell therapy as we aim to bring this potentially life-saving treatment approach to as many appropriate people with blood cancers as possible."

Yescarta was the first CAR T cell therapy to be approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, and high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma. The Yescarta U.S. Prescribing Information has a BOXED WARNING for the risks of cytokine release syndrome and neurologic toxicities; see below for Important Safety Information.

Dates and times for presentations are as follows:

Area of Focus, Presentation Number and Date/Time (ET)

Abstract Title

Presentations

Large B-cell Lymphoma
Abstract #203 (Oral)
Saturday, Dec 7 (1:00 pm)

CD19-Loss with Preservation of Other B-Cell Lineage Features with Large B-Cell Lymphoma Who Relapsed Post-Axi-Cel

Large B-cell Lymphoma
Abstract #243 (Oral)
Saturday, Dec 7 (2:30 pm)

Earlier Steroid Use with Axicabtagene Ciloleucel (Axi-Cel) in Patients with Relapsed/Refractory Large B-Cell Lymphoma

Large B-cell Lymphoma
Abstract #793 (Oral)
Monday, Dec 9 (2:45 pm)

Medicare Patients Receiving Chimeric Antigen Receptor T Cell Therapy for Non-Hodgkin Lymphoma: A First Real-world Look at Patient Characteristics, Healthcare Utilization and Costs

Large B-cell Lymphoma
Abstract #764 (Oral)
Monday, Dec 9 (3:00 pm)

Outcomes of Post-marketing Use of an Anti-CD19 CAR T Cell Therapy, Axicabtagene Ciloleucel (Axi-Cel), for the Treatment of Large B-Cell Lymphoma in the United States

Mantle Cell Lymphoma
Abstract #754 (Oral)
Monday, Dec 9 (3:30 pm)

KTE-X19, an Anti-CD19 CAR T Cell Therapy, in Patients with Relapsed/Refractory Mantle Cell Lymphoma: Results of the Phase 2 ZUMA-2 Study

Large B-cell Lymphoma
Abstract #4095 (Poster)
Monday, Dec 9 (6:00-8:00 pm)

A Comparison of Two-year Outcomes in ZUMA-1 (Axicabtagene Ciloleucel) and SCHOLAR-1 in Patients with Refractory Large B-Cell Lymphoma

Trials-In-Progress

Large B-cell Lymphoma
Abstract #4084 (Poster)
Monday, Dec 9 (6:00-8:00 pm)

ZUMA-11: A Phase 1/2 Multicenter Study of Axicabtagene Ciloleucel (Axi-Cel) + Utomilumab in Patients with Refractory Large B-Cell Lymphoma

Large B-cell Lymphoma
Abstract #4093 (Poster)
Monday, Dec 9 (6:00-8:00 pm)

A Phase 2, Open-label, Multicenter Study Evaluating the Safety and Efficacy of Axicabtagene Ciloleucel in Combination with Either Rituximab or Lenalidomide in Patients with Refractory Large B-Cell Lymphoma (ZUMA-14)

For more information, including a complete list of abstract titles at the meeting, please visit: View Source

The use of Yescarta with utomilumab, rituximab, or lenalidomide is investigational and not approved globally. Efficacy and safety of these potential combinations have not been established. KTE-X19 is investigational and not approved anywhere globally. Its efficacy and safety have not been established. More information about clinical trials with KTE-X19 is available at www.clinicaltrials.gov.

About KTE-X19

KTE-X19 is an investigational, autologous, anti-CD19 CAR T cell therapy. KTE-X19 uses the XLP manufacturing process that includes T-cell selection and lymphocyte enrichment. Lymphocyte enrichment is a necessary step in certain B-cell malignancies with evidence of circulating lymphoblasts. KTE-X19 is currently in Phase 1/2 trials in acute lymphoblastic leukemia (ALL), mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL).

U.S. Important Safety Information for Yescarta

BOXED WARNING: CYTOKINE RELEASE SYNDROME AND NEUROLOGIC TOXICITIES

Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving Yescarta. Do not administer Yescarta to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab or tocilizumab and corticosteroids.
Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving Yescarta, including concurrently with CRS or after CRS resolution. Monitor for neurologic toxicities after treatment with Yescarta. Provide supportive care and/or corticosteroids as needed.
Yescarta is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Yescarta REMS.
CYTOKINE RELEASE SYNDROME (CRS): CRS occurred in 94% of patients, including 13% with ≥ Grade 3. Among patients who died after receiving Yescarta, 4 had ongoing CRS at death. The median time to onset was 2 days (range: 1-12 days) and median duration was 7 days (range: 2-58 days). Key manifestations include fever (78%), hypotension (41%), tachycardia (28%), hypoxia (22%), and chills (20%). Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), cardiac arrest, cardiac failure, renal insufficiency, capillary leak syndrome, hypotension, hypoxia, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome. Ensure that 2 doses of tocilizumab are available prior to infusion of Yescarta. Monitor patients at least daily for 7 days at the certified healthcare facility following infusion for signs and symptoms of CRS. Monitor patients for signs or symptoms of CRS for 4 weeks after infusion. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab or tocilizumab and corticosteroids as indicated.

NEUROLOGIC TOXICITIES: Neurologic toxicities occurred in 87% of patients. Ninety-eight percent of all neurologic toxicities occurred within the first 8 weeks, with a median time to onset of 4 days (range: 1-43 days) and a median duration of 17 days. Grade 3 or higher occurred in 31% of patients. The most common neurologic toxicities included encephalopathy (57%), headache (44%), tremor (31%), dizziness (21%), aphasia (18%), delirium (17%), insomnia (9%) and anxiety (9%). Prolonged encephalopathy lasting up to 173 days was noted. Serious events including leukoencephalopathy and seizures occurred with Yescarta. Fatal and serious cases of cerebral edema have occurred in patients treated with Yescarta. Monitor patients at least daily for 7 days at the certified healthcare facility following infusion for signs and symptoms of neurologic toxicities. Monitor patients for signs or symptoms of neurologic toxicities for 4 weeks after infusion and treat promptly.

YESCARTA REMS: Because of the risk of CRS and neurologic toxicities, Yescarta is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Yescarta REMS. The required components of the Yescarta REMS are: Healthcare facilities that dispense and administer Yescarta must be enrolled and comply with the REMS requirements. Certified healthcare facilities must have on-site, immediate access to tocilizumab, and ensure that a minimum of 2 doses of tocilizumab are available for each patient for infusion within 2 hours after Yescarta infusion, if needed for treatment of CRS. Certified healthcare facilities must ensure that healthcare providers who prescribe, dispense or administer Yescarta are trained about the management of CRS and neurologic toxicities. Further information is available at www.YESCARTAREMS.com or 1-844-454-KITE (5483).

HYPERSENSITIVITY REACTIONS: Allergic reactions may occur. Serious hypersensitivity reactions including anaphylaxis may be due to dimethyl sulfoxide (DMSO) or residual gentamicin in Yescarta.

SERIOUS INFECTIONS: Severe or life-threatening infections occurred. Infections (all grades) occurred in 38% of patients, and in 23% with ≥ Grade 3. Grade 3 or higher infections with an unspecified pathogen occurred in 16% of patients, bacterial infections in 9%, and viral infections in 4%. Yescarta should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after Yescarta infusion and treat appropriately. Administer prophylactic anti-microbials according to local guidelines. Febrile neutropenia was observed in 36% of patients and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad spectrum antibiotics, fluids and other supportive care as medically indicated. Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure and death, can occur in patients treated with drugs directed against B cells. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

PROLONGED CYTOPENIAS: Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and Yescarta infusion. Grade 3 or higher cytopenias not resolved by Day 30 following Yescarta infusion occurred in 28% of patients and included thrombocytopenia (18%), neutropenia (15%), and anemia (3%). Monitor blood counts after Yescarta infusion.

HYPOGAMMAGLOBULINEMIA: B-cell aplasia and hypogammaglobulinemia can occur. Hypogammaglobulinemia occurred in 15% of patients. Monitor immunoglobulin levels after treatment and manage using infection precautions, antibiotic prophylaxis and immunoglobulin replacement. The safety of immunization with live viral vaccines during or following Yescarta treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during Yescarta treatment, and until immune recovery following treatment.

SECONDARY MALIGNANCIES: Patients may develop secondary malignancies. Monitor life-long for secondary malignancies. In the event that a secondary malignancy occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

EFFECTS ON ABILITY TO DRIVE AND USE MACHINES: Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following Yescarta infusion. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, during this initial period.

ADVERSE REACTIONS: The most common adverse reactions (incidence ≥ 20%) include CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, decreased appetite, chills, diarrhea, febrile neutropenia, infections-pathogen unspecified, nausea, hypoxia, tremor, cough, vomiting, dizziness, constipation, and cardiac arrhythmias.

On November 6, 2019 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that it will present new data for several investigational therapies in its AML/blood cancer portfolio at the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), December 7-10, 2019, in Orlando, Florida (Press release, Daiichi Sankyo, NOV 6, 2019, https://www.businesswire.com/news/home/20191106005138/en/Daiichi-Sankyo-Data-ASH-Showcases-Scientific-Clinical [SID1234550534]).

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Highlights include three oral presentations featuring post-hoc analyses from the global, pivotal phase 3 QuANTUM-R study including post-transplant survival, genetic/biomarker-related responses and outcomes, and quality-adjusted time without symptoms or toxicity in patients with relapsed/refractory FLT3-ITD AML receiving quizartinib versus salvage chemotherapy.

"The additional findings from the phase 3 study will help inform clinical use as well as further development of quizartinib, which is currently being evaluated in newly-diagnosed FLT3-ITD AML in the QuANTUM-First trial," said Arnaud Lesegretain, Vice President, Oncology Research and Development and Head, AML Franchise, Daiichi Sankyo. "Other ASH (Free ASH Whitepaper) presentations reflect clinical development progress for several of our investigational therapies, including valemetostat, which is an important asset in our R&D program for patients with AML and other blood cancers."

Updated phase 1 study results will be reported for valemetostat, a potential first-in-class EZH1/2 dual inhibitor, in relapsed/refractory non-Hodgkin lymphomas (NHLs), including a subgroup analysis in patients with adult T-cell leukemia/lymphoma (ATL/L). A pivotal phase 2 study in patients with ATL/L is planned in Japan.

Following is a full list of abstracts from the Daiichi Sankyo oncology portfolio accepted at ASH (Free ASH Whitepaper):

ASH Abstract Title

Presentation Details

Quizartinib / QuANTUM-R

Clinical Outcomes and Characteristics of Patients with FLT3-ITD-Mutated Relapsed/Refractory (R/R) AML Undergoing Hematopoietic Stem Cell Transplantation (HSCT) after Quizartinib or Salvage Chemotherapy in the QuANTUM-R Trial

Abstract #736; Oral Presentation (Ganguly S)
Monday, December 9, 2019
Session 613: 2:45 – 4:15 PM (3:30 PM)
Tangerine 3, WF3-4

Effect of Co-Mutations and FLT3-ITD Variant Allele Frequency (VAF) on Response to Quizartinib or Salvage Chemotherapy (SC) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML)

Abstract #737; Oral Presentation (Perl A)
Monday, December 9, 2019
Session 613: 2:45 – 4:15 PM (3:45 PM)
Tangerine 3, WF3-4

Quality-Adjusted Time without Symptoms or Toxicity (Q-TWiST) Analysis of Quizartinib Vs. Salvage Chemotherapy in Patients with Relapsed/Refractory (R/R) FLT3-ITD AML

Abstract #382; Oral Presentation (Cortes J)
Sunday, December 8, 2019
Session 903: 7:30 – 9:30 AM (8:15 AM). W307

Pooled Safety Analysis of Quizartinib Monotherapy in Patients with Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML)

Abstract #1372; Poster Presentation (Cortes J)
Saturday, December 7, 2019
Session 616: Poster I, 5:30 – 7:30 PM. Hall B

Characterization of Response and Transfusion Independence in Patients with FLT3-ITD-Mutated Relapsed/Refractory AML Treated with Quizartinib or Salvage Chemotherapy in the QuANTUM-R Trial

Abstract #2599; Poster Presentation (Levis M)
Sunday, December 8, 2019
Session 613: Poster II, 6:00 – 8:00 PM. Hall B

Exposure-Response of Quizartinib Efficacy in Patients with Relapsed/Refractory AML

Abstract #1263; Poster Presentation (Kang D)
Saturday, December 7, 2019
Session 604: Poster I, 5:30 – 7:30 PM. Hall B

A Phase 1/2 Study of Quizartinib (Q) in Combination with Re-Induction Chemotherapy and As Single-Agent Continuation Therapy in Pediatric and Young Adult Patients with Relapsed/Refractory (R/R) FLT3-ITD AML

Abstract #3937; Poster Presentation (Zwaan M)
Monday, December 9, 2019
Session 616: TiP Poster III, 6:00 – 8:00 PM. Hall B

An Evaluation of Major Comorbidities and Treatment Patterns of Newly Diagnosed Acute Myeloid Leukemia Patients: A Retrospective Analysis of Electronic Medical Records from US

Abstract #5106; Publication Only (Tu N)

Valemetostat (DS-3201)

First-in-Human Study of the EZH1/2 Dual Inhibitor DS-3201b in Relapsed or Refractory Non-Hodgkin Lymphoma (NHL)— Interim Results Focusing on Adult T-Cell Leukemia-Lymphoma (ATL)

Abstract #4025; Poster Presentation (Morishima S)
Monday, December 9, 2019
Session 624: Poster III, 6:00 – 8:00 PM. Hall B

Anti-tumor Effect of the EZH1/2 Inhibitor Valemetostat Against Diffuse Large B-Cell Lymphoma via Modulation of B-cell Receptor Signaling and c-Myc Signaling Pathways (preclinical data)

Abstract #4642; Poster Presentation (Hama Y)
Monday, December 9, 2019
Session 802: Poster III, 6:00 – 8:00 PM. Hall B

Milademetan (DS-3032)

A Phase 1 Dose Escalation Study of Milademetan in Combination with 5-Azacitidine (AZA) in Patients with Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS)

Abstract #3932; Poster Presentation (DiNardo C)
Monday, December 9, 2019
Session 616: Poster III, 6:00 – 8:00 PM. Hall B

A Phase I Study of Milademetan in Combination with Quizartinib in Patients with Newly Diagnosed (ND) or Relapsed/Refractory FLT3-ITD Acute Myeloid Leukemia (AML)

Abstract #1389; Poster Presentation (Daver N)
Saturday, December 7, 2019
Session 616: Poster I, 5:30 – 7:30 PM. Hall B

Dual Inhibition of MDM2 and XPO1 Induces Synergistic Apoptosis in Acute Myeloid Leukemia with Wild-type TP53 through Nuclear Accumulation of p53 and Suppression of c-Myc (preclinical data)

Poster Presentation (Nishida Y)
Sunday, December 8, 2019
Session 604: Poster II, 6:00 – 8:00 PM. Hall B

PLX2853

Dose Escalation Study of BET Inhibitor PLX2853 in Patients with Relapsed or Refractory Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

Abstract #1391; Poster Presentation (Pemmaraju N)
Saturday, December 7, 2019
Session 616: Poster I, 5:30 – 7:30 PM. Hall B

A Novel Combination Regimen of BET and FLT3 Inhibition for FLT3-ITD Acute Myeloid Leukemia (preclinical)

Abstract #1373; Poster Presentation (Lee L)
Saturday, December 7, 2019
Session 616: Poster I, 5:30 – 7:30 PM. Hall B

Bromodomain and Extraterminal (BET) Domain Inhibition with PLX51107 and PLX2853 Improves Survival and Decreases Acute GVHD Severity in Murine Models (preclinical)

Abstract #4429; Poster Presentation (Choe H)
Monday, December 9, 2019
Session 701: Poster III, 6:00 – 8:00 PM. Hall B

About Quizartinib

Quizartinib, an oral FLT3 inhibitor, is the lead product in the AML Franchise of Daiichi Sankyo. Quizartinib currently is approved only for use in Japan under the brand name VANFLYTA for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, as detected by an approved test. It was launched in Japan on October 10, 2019.

Enrollment into QuANTUM-First, a global, pivotal phase 3 study evaluating quizartinib in combination with standard chemotherapy in newly diagnosed FLT3-ITD AML was recently completed. Quizartinib is also in phase 1/2 development for pediatric and young adult relapsed/refractory FLT3-ITD AML in North America and Europe, and phase 1 development in combination with milademetan, an investigational MDM2 inhibitor, for relapsed/refractory FLT3-ITD AML and newly-diagnosed FLT3-ITD AML unfit for intensive chemotherapy in the U.S.

About Valemetostat

Valemetostat (DS-3201) is an investigational and potential first-in-class EZH1/2 dual inhibitor in phase 1 clinical development for hematologic cancers including acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and non-Hodgkin lymphomas (NHLs) including adult T-cell leukemia/lymphoma (ATL/L), peripheral T-cell lymphoma (PTCL) and B-cell lymphomas. Valemetostat has received SAKIGAKE Designation for the treatment of adult patients with relapsed/refractory peripheral T-cell lymphoma (PTCL) by the Ministry of Health, Labour and Welfare (MHLW) in Japan.

About Milademetan

Milademetan (DS-3032) is an oral selective MDM2 inhibitor currently in phase 1 development for solid and hematologic malignancies, including a combination study with quizartinib in patients with relapsed/ refractory FLT3-ITD AML or newly-diagnosed FLT3-ITD AML unfit for intensive chemotherapy in the U.S., EU and Japan; a single agent and combination study with 5-azacitidine, an inhibitor of DNA methylation, in patients with newly-diagnosed AML unfit for intensive chemotherapy, relapsed/refractory AML or high-risk MDS in the U.S.; and two single agent studies in lymphomas and solid tumors in the U.S. and Japan.

About PLX2853

PLX2853 is an investigational oral small molecule BET inhibitor currently in phase 1 development for AML and high-risk myelodysplastic syndrome (MDS) and phase 1/2 development for advanced solid tumors in the U.S. PLX2853 was discovered by Plexxikon Inc.

Valemetostat, milademetan and PLX2853 are investigational agents that have not been approved for any indication in any country. Safety and efficacy have not been established.

About Daiichi Sankyo Cancer Enterprise

The mission of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by three pillars including our investigational Antibody Drug Conjugate Franchise, Acute Myeloid Leukemia Franchise and Breakthrough Science pipeline, we aim to deliver seven distinct new molecular entities over eight years during 2018 to 2025. Our powerful research engines include two laboratories for biologic/ immuno-oncology and small molecules in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. For more information, please visit: www.DSCancerEnterprise.com.

On November 6, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported presentations at the upcoming 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 7-10, 2019 in Orlando, Florida (Press release, Rocket Pharmaceuticals, NOV 6, 2019, View Source [SID1234550533]). The two poster presentations will highlight clinical data from the Phase 1 study of RP-L102 utilizing "Process B" for the treatment of Fanconi Anemia (FA), as well as long-term follow-up data from the Phase 1/2 EUROFANCOLEN trial.

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Details for Rocket’s poster presentations are as follows:
Title: Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells
Session Title: Gene Therapy and Transfer: Poster II
Presenter: Sandeep Soni, M.D.
Session Date: Sunday, December 8, 2019
Session Time: 6:00 p.m. – 8:00 p.m. EST
Location: Orange County Convention Center, Hall B

Title: Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy
Session Title: Gene Therapy and Transfer: Poster III
Presenter: Paula Río, Ph.D.
Session Date: Monday, December 9, 2019
Session Time: 6:00 p.m. – 8:00 p.m. EST
Location: Orange County Convention Center, Hall B

The Sunday poster session will be followed by a breakout session to give investors and analysts the opportunity to ask questions and discuss the data. The breakout session, hosted by Rocket management, will be held on Sunday, December 8th at 8:30 p.m. EST, directly after Dr. Soni’s presentation. At the event, Dr. Soni, Clinical Associate Professor of Stem Cell Transplantation and Regenerative Medicine at the Stanford University School of Medicine and principal investigator of the U.S. Phase 1 trial of RP-L102 and Paula Río, Ph.D., Senior Scientist, División de Terapias Innovadoras en el Sistema Hematopoyético, CIEMAT/CIBERER Unidad Mixta de Terapias Avanzadas CIEMAT/IIS Fundación Jiménez Díaz will be participating in a Q&A panel. For further information, please contact [email protected].

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the ‘gold standard’ test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as ‘nature’s gene therapy’ provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

On November 6, 2019 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer, autoimmune diseases, asthma and allergic diseases, reported that initial data from its ongoing Phase 1 dose-escalation study of XmAb13676, a CD20 x CD3 bispecific antibody, in patients with B-cell malignancies will be presented in a poster session during the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando, Florida on Monday, December 9, 2019 (Press release, Xencor, NOV 6, 2019, View Source [SID1234550532]).

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"Initial results from dose-escalation cohorts demonstrate encouraging clinical activity in heavily pretreated patients with several subtypes of non-Hodgkin lymphoma, as well as chronic lymphocytic leukemia. The most common treatment-related adverse event has been fever. Cytokine release syndrome, the second most common adverse event, has been generally manageable with premedication," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor.

Key Highlights from the Abstract

The abstract with accepted data from the study are available through the ASH (Free ASH Whitepaper) website. Updated results will be shared at the ASH (Free ASH Whitepaper) Annual Meeting.

At data cut off on June 28, 2019, 36 patients with relapsed/refractory non-Hodgkin’s lymphoma (r/r NHL) and 8 patients with relapsed/refractory chronic lymphocytic leukemia (r/r CLL) had received XmAb13676. The study was designed in two parts: Part A to establish an initial priming dose and Part B to escalate dosing on subsequent administration. Prophylactic treatment for cytokine release syndrome (CRS) was mandated prior to each administration of XmAb13676.
Patients with r/r NHL (n=36) had a median age of 61.5 years, a median of 3.5 prior therapies and had been diagnosed a median of 24.6 months prior to treatment. In the efficacy evaluable population and at doses ranging from 80 to 125 mcg/kg, objective responses were observed in 33% of patients (n=6/18), including 42% of DLBCL patients (n=5/12). CRS occurred in 42% of patients (n=15/36), and one patient receiving an initial dose of 125 mcg/kg experienced Grade 4 CRS. A priming dose of 45 mcg/kg was chosen for Part B.
Patients with r/r CLL (n=8) had a median age of 76 years, a median of 4.5 prior therapies and had been diagnosed a median of 76.1 months prior to treatment. There was one complete response (Richter transformation) in five patients treated at the 20 mcg/kg dose level, the highest dose administered in the ongoing Part A of the study. CRS occurred in 25% of patients (n=2/8), and one patient experienced Grade 3 CRS.
Presentation Details

Abstract: 4079
Title: Preliminary Safety and Anti-tumor Activity of XmAb13676, an Anti-CD20 x Anti-CD3 Bispecific Antibody in Patients with Relapsed/Refractory Non-Hodgkin’s Lymphoma and Chronic Lymphocytic Leukemia
Presenter: Krish Patel, M.D., Director of the Lymphoma Program at Swedish Cancer Institute
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster III
Date & Time: Monday, December 9, 2019, 6:00 p.m. – 8:00 p.m. EST
Location: Orange County Convention Center, Hall B
About XmAb13676

XmAb13676 is a tumor-targeted antibody that contains both a CD20 binding domain and a cytotoxic T-cell binding domain (CD3) in a Phase 1 clinical trial for the treatment of B-cell malignancies. An XmAb bispecific Fc domain serves as the scaffold for these two antigen binding domains and confers long circulating half-life, stability and ease of manufacture on XmAb13676. CD20 is highly expressed on B-cell tumors, including in chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma (NHL). Engagement of CD3 by XmAb13676 activates T cells for highly potent and targeted killing of CD20-expressing tumor cells.