On November 4, 2020 Genmab A/S (Nasdaq: GMAB) reported that more than 40 abstracts related to Genmab owned and partnered programs were accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place virtually December 5-8 (Press release, Genmab, NOV 4, 2020, View Source [SID1234569837]). Abstracts accepted for presentation include data from the ongoing Phase I/II trial of epcoritamab in B-cell non-Hodgkin lymphomas, which will be presented during an oral session of the conference. Accepted abstracts also include pre-clinical data from Genmab’s next generation CD38 antibody, HexaBody-CD38 and updates on multiple daratumumab clinical trials. In addition, data for teclistamab and talquetamab, two of Janssen’s bispecific antibodies created with Genmab’s DuoBody technology platform, were accepted for oral presentations at the conference.

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All abstracts are available on the ASH (Free ASH Whitepaper) website at www.hematology.org. Details regarding the key abstracts to be presented are included below.

"2020 has been another strong year for Genmab with our proprietary pipeline progressing rapidly. We are very excited to be sharing additional data from our epcoritamab program as an oral presentation at the prestigious ASH (Free ASH Whitepaper) conference as well as data from our pre- clinical HexaBody-CD38 program," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. "We are also very pleased to see that, once again, a significant number of daratumumab abstracts were accepted for presentation, as this confirms our confidence in the broad potential of daratumumab."

Late breaking abstracts are not yet available.

On December 8 at 12:30 PM EST (6:30 PM CET / 5:30 PM GMT) Genmab will hold its virtual 2020 ASH (Free ASH Whitepaper) Data Review and present its 2021 Key Priorities. The event will be webcast live on the following link: View Source Details, including the webcast link, can also be found on Genmab’s website, www.genmab.com.

This meeting is not an official program of the ASH (Free ASH Whitepaper) Annual Meeting.

Genmab Abstracts:

Subcutaneous Epcoritamab Induces Complete Responses With an Encouraging Safety Profile Across Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma Subtypes, Including Patients With Prior CAR-T Therapy: Updated Dose-Escalation Data– Oral presentation, Sunday, December 6 12.30 PST

Novel semi-mechanistic model leveraging preclinical and clinical data to inform the recommended phase 2 dose (RP2D) selection for epcoritamab (DuoBody CD3×CD20)– Poster presentation, Monday December 7, 7.00 AM – 3.30 PM PST.

Preclinical Anti-Tumor Activity of Hexabody-CD38 in Patient-Derived B Cell Lymphoma and Acute Myeloid Leukemia Xenograft Models– Poster presentation, Sunday, December 6, 7.00 AM – 3.30 PM PST.

Key Abstracts Sponsored by Janssen Biotech, Inc. include:
APOLLO: Phase 3 Randomized Study of Subcutaneous Daratumumab Plus Pomalidomide and Dexamethasone (D-Pd) Versus Pomalidomide and Dexamethasone (Pd) Alone in Patients (Pts) with Relapsed/Refractory Multiple Myeloma (RRMM)" – Oral Presentation Sunday, December 6, 2020, 12:00 PM PST.

Daratumumab (DARA) Plus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Patients with Transplant-eligible Newly Diagnosed Multiple Myeloma (NDMM): Updated Analysis of GRIFFIN After 12-months of Maintenance Therapy" – Oral Presentation Monday, December 7, 2020, 7:15 AM PST.

Reduction in Absolute Involved Free Light Chain and Difference Between Involved and Uninvolved Free Light Chain Is Associated with Prolonged Major Organ Deterioration Progression-Free Survival in Patients with Newly Diagnosed AL Amyloidosis Receiving Bortezomib, Cyclophosphamide, and Dexamethasone With or Without Daratumumab: Results From ANDROMEDA" – Oral Presentation Monday, December 7, 2020, 8:00 AM PST.

Updated Analysis of Daratumumab Plus Lenalidomide and Dexamethasone (D-Rd) Versus Lenalidomide and Dexamethasone (Rd) in Patients with Transplant-ineligible Newly Diagnosed Multiple Myeloma (NDMM): the Phase 3 MAIA Study – Poster Presentation Sunday, December 6, 2020, 7.00 AM – 3.30 PM PST.

Updated Phase 1 Results of Teclistamab, a B-cell Maturation Antigen (BCMA) x CD3 Bispecific Antibody, in Relapsed and/or Refractory Multiple Myeloma (RRMM) – Oral Presentation, Saturday, December 5, 2020, 12:45 PM PST.

A Phase 1, First-in-Human Study of Talquetamab, a G Protein-Coupled Receptor Family C Group 5 Member D (GPRC5D) x CD3 Bispecific Antibody, in Patients with Relapsed and/or Refractory Multiple Myeloma (RRMM) – Oral Presentation, Saturday, December 5, 2020, 2:00 PM PST.

On November 4, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it will present new clinical data from its fully enrolled, ongoing Phase 2 trial of SY-1425, its first-in-class oral selective retinoic acid receptor alpha (RARα) agonist, at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, taking place virtually December 5-8, 2020 (Press release, Syros Pharmaceuticals, NOV 4, 2020, View Source [SID1234569836]). The oral presentations will include data on SY-1425 in combination with azacitidine from a cohort of RARA-positive patients with relapsed or refractory acute myeloid leukemia (AML) and mature data on the combination in newly diagnosed unfit AML patients who are not suitable candidates for standard chemotherapy.

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In a separate poster presentation, Syros will present new data showing that the majority of RARA-positive patients have a disease phenotype associated with resistance to upfront treatment with venetoclax, further underscoring the potential of SY-1425 in combination to address an ongoing unmet need in newly diagnosed unfit AML patients.

The abstracts are now available online on the ASH (Free ASH Whitepaper) conference website at View Source

Details of the oral presentations are as follows:

Presentation Title: SY-1425, a Potent and Selective RARα Agonist, in Combination with Azacitidine Demonstrates a High Complete Response Rate and a Rapid Onset of Response in RARA-Positive Newly Diagnosed Unfit Acute Myeloid Leukemia
Session Date & Time: Saturday, December 5, 12:30 p.m. – 2 p.m. ET/9:30 a.m. – 11:00 a.m. PT
Presentation Time: 12:45 p.m. ET/9:45 a.m. PT
Session Title: Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Novel promising therapies for relapsed/refractory AML
Presenter: Stéphane de Botton, M.D., Institut Gustave Roussy
Abstract Number: 134600

Presentation Title: Initial Results from a Biomarker-Directed Phase 2 Trial of SY-1425, a Potent and Selective RARα Agonist, in Combination with Azacitidine in Relapsed/Refractory Acute Myeloid Leukemia
Session Date & Time: Saturday, December 5, 12:30 p.m. – 2 p.m. ET/9:30 a.m. – 11:00 a.m. PT
Presentation Time: 1:15 p.m. ET/10:15 a.m. PT
Session Title: Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Novel promising therapies for relapsed/refractory AML
Presenter: Eytan M. Stein, M.D., Memorial Sloan Kettering Cancer Center
Abstract Number: 134602

Details of the poster presentation are as follows:

Presentation Title: Selection of RARA-Positive Newly Diagnosed Unfit AML Patients with Elevated RARA Gene Expression Enriches for Features Associated with Primary Resistance to Venetoclax and Clinical Response to SY‑1425, a Potent and Selective RARα Agonist, Plus Azacitidine
Session Date & Time: Monday, December 7, 10 a.m. – 6 p.m. ET/7 a.m. – 3 p.m. PT
Session Title: Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III
Presenter: Graeme Hodgson, Ph.D., Syros
Abstract Number: 137323

On November 4, 2020 Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported the release of two abstracts at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, one oral presentation of initial data for its BALLI-01 clinical trial and one Trials in Progress poster presentation of its AMELI-01 clinical trial (Press release, Cellectis, NOV 4, 2020, View Source [SID1234569835]). This will be the first publicly released data from Cellectis’ Phase 1 dose-escalation study of UCART22 product candidate in adult patients with Relapsed/Refractory CD22+ B-ALL.

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"We are pleased with the encouraging preliminary results from patients administered UCART22 cells in our lower dose cohorts with fludarabine and cyclophosphamide lymphodepletion regimen, and are eagerly awaiting additional data from our currently enrolling cohorts that include alemtuzumab in the lymphodepletion regimen," said Carrie Brownstein, MD, Chief Medical Officer, Cellectis. "We strongly believe in the potential of our innovative product candidates and are looking forward to presenting more data in the near future."

BALLI-01 investigating UCART22 product candidate in R/R B-ALL

BALLI-01 is a Phase 1 open-label dose-escalation study designed to assess the safety, the maximum tolerated dose (MTD), and preliminary anti-leukemia activity of UCART22 in patients with R/R B-ALL. Additional endpoints include characterization of the expansion, trafficking, and persistence of UCART22 cells.

As of July 2020, seven patients were enrolled. One patient failed screening and one patient was discontinued prior to the administration of UCART22 cells due to an adverse event related to the lymphodepletion.

The abstract includes preliminary data from the first five patients who received escalating doses of UCART22 cells after fludarabine/cyclophosphamide (FC) lymphodepletion. Enrolled patients were predominantly male [n=4], younger (median age 24 [range 22-52]), and heavily pretreated with a median of 3 prior lines of therapy [range 2-4]. The median baseline bone marrow blasts percentage prior to lymphodepletion was 35% [5-78.4%].

Adverse events were mainly mild to moderate in intensity and manageable. Four patients experienced treatment-related, treatment-emergent, adverse events which primarily consisted of abnormalities in liver function tests (i.e. increased alkaline phosphatase, increased bilirubin, and transaminitis), hypotension, fever, and other constitutional symptoms. Cytokine release syndrome was reported in three patients (one patient with Grade 1 and two patients with Grade 2). Two patients experienced serious treatment-emergent adverse events: one patient had Grade 3 febrile neutropenia and Grade 3 hepatic hematoma; one patient had Grade 4 bleeding and Grade 5 sepsis in the context of progressive disease. Importantly, no patients experienced treatment-related serious TEAE, GvHD, ICANS, protocol-defined Dose Limiting Toxicity nor AESI1.

Two of three patients in Dose Level 1 achieved an objective response, one patient with best response of CR, and a second patient with CR with incomplete hematologic recovery (CRi). One patient in Dose Level 2 with refractory disease did achieve a noteworthy reduction in bone marrow blasts [40% (Day -1) to 13% (Day 28)] after treatment with UCART22 product candidate.

Host lymphocyte reconstitution was observed in all patients within the DLT period (range Day 17-Day 28). Correlative analysis of UCART cell expansion and persistence is ongoing.

UCART22 demonstrated preliminary signs of activity at low dose levels with fludarabine/cyclophosphamide (FC) lymphodepletion regimen, without unexpected nor significant treatment-related toxicities. CRS was observed in three patients and was mild to moderate and manageable. No patients reported DLT, GvHD nor ICANS1. One patient achieved a CR and another a CRi. Host immune recovery was observed early, supporting the addition of alemtuzumab to the FC lymphodepletion regimen which is expected to result in a deeper and more sustained T-cell depletion and thereby promote expansion and persistence of UCART22 cells. Enrollment into the Dose Level 2 cohorts with alemtuzumab is ongoing.

Oral Abstract Session:

Abstract: #163
Title: Preliminary results of BALLI-01: A Phase I study of UCART22 (allogeneic engineered T-cells expressing anti-CD22 Chimeric Antigen Receptor) in adult patients with relapsed or refractory (R/R) CD22+ B-cell Acute Lymphoblastic Leukemia (B-ALL).
Presenter: Jain Nitin, MD, The University of Texas MD Anderson Cancer Center, Department of Leukemia, Houston, TX
Session Name: 614. Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Chimeric Antigen Receptor T Cell Therapy
Session Release Date & Time: Saturday, December 5, 2020 at 1:00 PM Pacific Time

AMELI-01 investigating UCART123 product candidate in R/R AML

This abstract is a Trials in Progress presentation. AMELI-01 is a Phase 1, multi-center clinical trial of Cellectis’ UCART123 product candidate that employs a modified toxicity probability interval (mTPI) design to evaluate the safety, tolerability and preliminary anti-leukemia activity of UCART123 cells in patients with R/R AML. Additional objectives include the determination of the maximum tolerated dose or suitable lower dose for expansion; characterization of the expansion, trafficking and persistence of UCART123 cells; assessment of cytokine, chemokine and C-reactive protein expression after UCART123 cell infusion; and assessment of immune cell depletion, reconstitution and immune response.

Dose escalation will include up to 28 patients. The dose expansion portion follows a Simon 2-stage design and will enroll up to an additional 37 patients. Eligible patients must be ≤ 65 years of age with R/R AML, adequate organ and bone marrow function, a confirmed donor for potential back-up stem cell transplantation, and no > Grade 1 toxicity from prior treatment. Patients with acute promyelocytic leukemia, prior gene or cellular therapy, > 1 allogeneic stem cell transplants, or those with a clinically relevant central nervous system disorder (including CNS leukemia) are not eligible. Patients receive a lymphodepletion regimen of either fludarabine and cyclophosphamide (FC) or fludarabine cyclophosphamide plus alemtuzumab (FCA) starting on Day-5, followed by an infusion of UCART123 cells at one of 5 dose levels on Day 0. Patients are evaluated for the presence of dose-limiting toxicities during a 28-day observation period, which extends to 42 days in the setting of an aplastic marrow and/or persistent clinically significant cytopenia without residual AML. Dose Levels 1 and 2 with FC lymphodepletion regimen have cleared safety without dose limiting toxicity, and enrollment at the next dose levels including cohorts with fludarabine and cyclophosphamide (FC) or fludarabine, cyclophosphamide plus alemtuzumab (FCA) is proceeding.

Poster Abstract Session:

Abstract: #1039
Title: AMELI-01: Phase I, open label dose-escalation and dose-expansion study to evaluate the safety, expansion, persistence and clinical activity of UCART123 (allogeneic engineered T-cells expressing anti-CD123 chimeric antigen receptor), administered in patients with Relapsed/Refractory Acute Myeloid Leukemia
Presenter: Gail J. Roboz, MD, Professor of Medicine at Weill Cornell Medicine and New York-Presbyterian, New York, NY
Session Name: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster I
Session Release Date & Time: Date: Saturday, December 5, 2020
The Virtual Poster Hall will be open for attendees to browse a different set of posters each day. The Poster Hall hours are as follows:
Saturday, December 5: 7:00 AM – 3:30 PM Pacific Time
Sunday, December 6: 7:00 AM – 3:30 PM Pacific Time
Monday, December 7: 7:00 AM – 3:00 PM Pacific Time

Cellectis is the sponsor of these Phase 1 clinical trials which are designed to assess the safety and tolerability at increasing dose levels of UCART22 and UCART123 in patients with R/R B-Cell Acute Lymphoblastic Leukemia (B-ALL) and R/R Acute Myeloid Leukemia (AML), respectively.

About UCART22
UCART22 is one of Cellectis’ wholly owned, allogeneic, off-the-shelf gene-edited T-cell product candidates, designed for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). Like CD19, CD22 is a cell surface antigen expressed from the pre-B-cell stage of development through mature B-cells. CD22 expression occurs in more than 90% of patients with B-ALL.

About UCART123
Our wholly controlled product candidate, UCART123, is a gene-edited T-cell investigational drug that targets CD123, an antigen expressed at the surface of leukemic cells in AML.

On November 4, 2020 CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported data in seven patients from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH (Free ASH Whitepaper) Meeting and Exposition, which will take place virtually from December 5-8, 2020 (Press release, CRISPR Therapeutics, NOV 4, 2020, View Source [SID1234569828]). Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcare’s TriStar Centennial Medical Center, will deliver the presentation on behalf of all the authors on December 6, 2020.

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An abstract posted online today includes data from five patients with three months to 15 months of follow-up after CTX001 infusion in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and data from two patients with three months and 12 months of follow-up in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Additional data will be presented at ASH (Free ASH Whitepaper), including longer-duration follow-up data for the patients included in the abstract and data for additional patients with greater than three months of follow-up.

CTX001 is being investigated in these two ongoing clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.

The accepted abstract is now available on the ASH (Free ASH Whitepaper) conference website.

About CTX001

CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About CLIMB-111

The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121

The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These Trials

Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patient’s cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex Collaboration

CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

On November 4, 2020 RGENIX, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, reported that CEO Masoud Tavazoie, M.D., Ph.D., will present at the 2020 Stifel Virtual Healthcare Conference at 8:40 A.M. EST on November 16, 2020 (Press release, Rgenix, NOV 4, 2020, View Source [SID1234569827]).

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To view this virtual presentation live, register for the event here.

Links to the live and archived version of this presentation will also be available on RGENIX’s website within the News section.