On November 3, 2022 Orum Therapeutics, a private biotechnology company pioneering the development of tumor-directed targeted protein degraders (TPDs), reported the presentation of preclinical data for ORM-6151, a first-in-class, anti-CD33 antibody-enabled GSPT1 degrader for acute myeloid leukemia (AML) (Press release, Orum Therapeutics, NOV 3, 2022, View Source [SID1234622906]). The data will be presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) (ASH 2022) Annual Meeting & Exposition taking place December 10-13, in New Orleans.

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Details of the presentation, which will be available in the poster hall and via the virtual meeting platform, are as follows:

The accepted abstract is available online through the ASH (Free ASH Whitepaper) conference website: www.hematology.org/Annual-Meeting/Abstracts/.

About Orum’s GSPT1 Platform Using the TPD² Approach

Orum’s GSPT1 platform uses the Company’s unique Dual-Precision Targeted Protein Degradation (TPD²) approach to build novel targeted protein degraders combined with the precise tumor cell delivery mechanisms of antibodies to generate innovative, first-in-class, cell-specific TPD for the treatment of cancer. The company has developed new molecular glue degrader payloads to specifically degrade an intracellular target protein within cancer cells via the E3 ubiquitin ligase pathway. Conjugated to antibodies, the payloads are designed to be delivered specifically to cancer cells and degrade the intracellular target protein GSPT1 and cause tumor cell death.

On November 3, 2022 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that new data across its hematology pipeline will be highlighted in 17 presentations at the 2022 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting from December 10-13 in New Orleans, LA (Press release, Regeneron, NOV 3, 2022, View Source [SID1234622905]).

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"We’ve made significant strides toward developing a comprehensive hematology portfolio that has the potential to address diverse and difficult-to-treat blood cancers and blood disorders," said L. Andres Sirulnik, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Hematology at Regeneron. "Our ASH (Free ASH Whitepaper) presentations not only showcase some of the many modalities we’re exploring – which includes monoclonal antibodies, bispecific antibodies, gene modifying technologies, and siRNA inhibition – but also the depth of research we’re conducting in support of our pipeline."

Notable Regeneron blood cancer presentations at ASH (Free ASH Whitepaper) include the first interim data from the Phase 2 ELM-2 study of odronextamab (CD20xCD3) in relapsed/refractory (R/R) follicular lymphoma (FL) and R/R diffuse large B-cell lymphoma (DLBCL), which will be shared in two oral sessions. Updated Phase 1/2 data will also be presented for linvoseltamab (REGN5458; BCMAxCD3 bispecific antibody) in patients with heavily pre-treated multiple myeloma. Based on these findings, a recommended dose was selected for the Phase 2 portion of the linvoseltamab trial.

Additionally, first clinical data from two Phase 2 studies evaluating pozelimab (C5 antibody) in combination with Alnylam Pharmaceuticals, Inc.’s cemdisiran (siRNA C5 inhibitor) in patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder, will be shared.

Investor Webcast Information
Regeneron will host a conference call and simultaneous webcast to share updates on the company’s hematology portfolio on Wednesday, December 14 at 8:30 AM ET. A link to the webcast may be accessed from the ‘Investors and Media’ page of Regeneron’s website at View Source To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. A replay of the conference call and webcast will be archived on the company’s website for at least 30 days.

Odronextamab in patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL): Results from a prespecified analysis of the pivotal Phase II study ELM-2

Odronextamab in patients with relapsed/refractory (R/R) follicular lymphoma (FL) Grade 1–3a: Results from a prespecified analysis of the pivotal Phase II study ELM-2

Trial in Progress: Follicular lymphoma outcomes in relapsed/refractory patients treated with systemic therapy in a real-world assessment (FLORA)

Trial in Progress: Outcomes in patients with relapsed/refractory DLBCL treated with systemic therapy from real-world experience (ORCHID)

Selection of odronextamab pediatric dosing regimens for aggressive non-Hodgkin lymphoma via a modeling and simulation approach

Optimization of intravenous Odronextamab step-up dosing regimen for reducing the risk of high-grade cytokine release syndrome

#5245

Publication Number

Online publication

Modeling and simulation in support of odronextamab subcutaneous dose selection for adult patients with indolent or aggressive non-Hodgkin lymphoma

#5257

Publication Number

Online publication

Evaluate dynamics of IL-6 release during step-up dosing of subcutaneous administration of odronextamab via a quantitative systems pharmacology modelling approach

#5424

Publication Number

Online publication

A quantitative systems pharmacology modelling framework for evaluation of cytokine release syndrome mediated by intravenous odronextamab monotherapy in patients with B-cell non-Hodgkin lymphoma

#5433

Publication Number

Online publication

Linvoseltamab (formerly REGN5458)

Updated safety and efficacy of REGN5458, a BCMAxCD3 bispecific antibody, treatment for relapsed/refractory multiple myeloma: A Phase 1/2 first-in-human study

Trial in Progress: REGN5458, a BCMAxCD3 bispecific antibody, in a Phase Ib multi-cohort study of combination regimens for patients with relapsed/refractory multiple myeloma

Trial in Progress: A Phase II window of opportunity study of the BCMAxCD3 bispecific antibody REGN5458 in previously untreated patients with symptomatic multiple myeloma

Real-world study of patients with triple-class exposed relapsed or refractory multiple myeloma: Analysis across a spectrum of advanced disease stage patients in the U.S.

Prevalence of ocular comorbidities in elderly patients with multiple myeloma in the U.S.: An Analysis of 100% Medicare sample data during 2007-2020

Incidence of second primary malignancies (SPMs) in patients in the U.S. with triple-class-exposed (TCE) relapsed or refractory multiple myeloma (RRMM)

A Phase 2, randomized trial evaluating the safety and efficacy of pozelimab and cemdisiran in patients with paroxysmal nocturnal hemoglobinuria

Patient-reported outcomes from a Phase 2, randomized trial evaluating the safety and efficacy of pozelimab and cemdisiran in patients with paroxysmal nocturnal hemoglobinuria

A Phase 2, open-label study evaluating the safety and efficacy of combination pozelimab and cemdisiran therapy in patients with paroxysmal nocturnal hemoglobinuria who switch from eculizumab

Blockade of common gamma chain cytokine signaling with REGN7257, an interleukin 2 receptor gamma (IL2RG) monoclonal antibody, protected mice from inflammatory and autoimmune diseases

Evaluation of common gamma chain cytokine signaling blockade with REGN7257, an interleukin 2 receptor gamma (IL2RG) monoclonal antibody, on immune cell populations in monkey and human

The potential uses of odronextamab, linvoseltamab, pozelimab, cemdisiran and REGN7257 described above are investigational, and their safety and efficacy have not been fully evaluated by any regulatory authority.

About Regeneron in Hematology
At Regeneron, we’re applying more than three decades of biology expertise with our proprietary VelociSuite technologies to develop medicines for patients with diverse blood cancers and rare blood disorders.

Our blood cancer research is focused on bispecific antibodies that are being investigated both as monotherapies and in combination with each other and emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop customized and potentially synergistic cancer treatments.

Our research and collaborations to develop potential treatments for rare blood disorders include explorations in antibody medicine, gene editing and gene-knockout technologies, and investigational RNA-approaches focused on depleting abnormal proteins or blocking disease-causing cellular signaling.

If you are interested in learning more about our clinical trials, please contact us ([email protected] or 844-734-6643) or visit our clinical trials website.

About Regeneron’s VelocImmune Technology
Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt. in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create approximately one in five of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes REGEN-COV (casirivimab and imdevimab), Dupixent (dupilumab), Libtayo (cemiplimab-rwlc), Praluent (alirocumab), Kevzara (sarilumab), Evkeeza (evinacumab-dgnb) and Inmazeb (atoltivimab, maftivimab and odesivimab-ebgn).

On November 3, 2022 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat rare and genetically defined diseases, reported that a broad set of clinical and translational data from its programs in hemolytic anemias, including PK deficiency, thalassemia and sickle cell disease, will be presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, to be held Dec. 10-13, 2022, in New Orleans (Press release, Agios Pharmaceuticals, NOV 3, 2022, View Source [SID1234622904]).

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In total, 22 abstracts led by Agios and external collaborators will be presented. The accepted abstracts are listed below and are available online on the ASH (Free ASH Whitepaper) conference website at View Source

PK Deficiency
Data to be presented highlight that long-term treatment with PYRUKYND in adults with PK deficiency is associated with sustained clinical benefits, including improvements in hemoglobin, iron overload, transfusion burden and patient-reported outcomes. In addition, Agios is presenting data supporting its pediatric PK deficiency program, including characterization of disease complications and co-morbidities in pediatric patients.

Oral Presentation:

Title: Long-term Improvements in Patient-reported Outcomes in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat
Presentation Time: Sunday, Dec. 11, 2022, at 9:45 a.m. CT
Oral Abstract Session: 904. Outcomes Research—Non-malignant Conditions: Classical Hematology: From Horses to Zebras
Abstract: 506
Presenter: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada

Poster Presentations:

Title: Next Generation Sequencing for the Diagnosis of Hereditary Hemolytic Anemias Including Pyruvate Kinase Deficiency: Report from a No-cost Diagnostic Program
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1010
Lead Author: Jorune Balciuniene, PerkinElmer Genomics

Title: Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency
Poster Session: 102. Iron Homeostasis and Biology: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1021
Lead Author: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada

Title: Long-term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2328
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center

Title: Comorbidities and Complications in Pediatric Patients with Pyruvate Kinase Deficiency Enrolled in the Peak Registry / Peak Pediatric Comorbidities
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2329
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center

Title: ACTIVATE-KidsT: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Regularly Transfused
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2330
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center

Title: Age of Onset of Complications in Patients with Pyruvate Kinase Deficiency: Analysis from the Peak Registry
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2332
Lead Author: Andreas Glenthøj, M.D., Ph.D., Department of Haematology, Copenhagen University Hospital – Rigshospitalet, Copenhagen, Denmark

Title: ACTIVATE-Kids: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2335
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center

Title: Mitapivat Improves Markers of Hemolysis and Erythropoiesis in Patients with Pyruvate Kinase Deficiency Irrespective of Hemoglobin Response
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster III
Session Date and Time: Monday, Dec. 12, 2022, 6-8 p.m. CT
Abstract: 3644
Lead Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States

Publication Only:

Title: The Launch of Two Sub-studies of the Peak Registry, A Global, Longitudinal Study of Pyruvate Kinase Deficiency
Abstract: 4960
Lead Author: Carl Lander, Thrive with Pyruvate Kinase Deficiency Patient Organization

Thalassemia
Long-term Phase 2 data for PYRUKYND in adults with alpha- or beta-thalassemia treated for up to 72 weeks show sustained hemoglobin response and improvements in hemolysis and ineffective erythropoiesis. Additional data underscore the significant disease burden across both alpha- and beta-thalassemia.

Poster Presentations:

Title: Mitapivat Improves Markers of Erythropoietic Activity in Long-term Study of Adults with Alpha- or Beta-non-transfusion-dependent Thalassemia
Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1030
Lead Author: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada

Title: Characterizing the Clinical, Health-related Quality of Life and Economic Burden of Alpha-thalassemia: A Systematic Literature Review and Evidence Gaps Assessment
Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1036
Lead Author: Khaled M. Musallam, M.D., Ph.D., Thalassemia Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates

Title: Clinical Burden of Alpha- and Beta-thalassemia Compared to Matched Controls in the Real-world Setting
Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2351
Lead Author: Arielle L. Langer, M.D., MPH, Division of Hematology, Brigham & Women’s Hospital

Sickle Cell Disease
Data to be presented continue to highlight that PK activation may be a promising therapeutic approach for patients with sickle cell disease.

Oral Presentations:

Title: Effects of Pyruvate Kinase Activators on Red Blood Cell Rheology, Sickling and Senescence in Sickle Cell Disease
Presentation Time: Saturday, Dec. 10, 2022, at 9:45 a.m. CT
Oral Abstract Session: 114. Hemoglobinopathies, Excluding Thalassemia: Clinical and Epidemiological: Novel Therapies
Abstract: 8
Presenter: Philippe Joly, Ph.D., Laboratory of Biochemistry and Molecular Biology, UF Biochemistry of Red Blood Cell Diseases, Est Center of Biology and Pathology, Hospices Civils de Lyon, Lyon France

Title: Untargeted Metabolomics in Dried Blood Spots of Patients with Sickle Cell Disease Treated with the Pyruvate Kinase Activator Mitapivat
Presentation Time: Saturday, Dec. 10, 2022, at 10 a.m. CT
Oral Abstract Session: 114. Hemoglobinopathies, Excluding Thalassemia: Clinical and Epidemiological: Novel Therapies
Abstract: 9
Presenter: Myrthe J. van Dijk, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands

Title: AG-946 Normalizes Glycolysis and Improves Red Cell Indices in a Humanized Sickle Cell Mouse Model
Presentation Time: Sunday, Dec. 11, 2022, at 9:30 a.m. CT
Oral Abstract Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Targeting the Red Blood Cell: Novel Therapeutic Approaches in Sickle Cell Disease
Abstract: 391
Lead Author: Rohitash Jamwal, Ph.D., Agios Pharmaceuticals

Title: PKLR Variants Associated with Acute Pain in Sickle Cell Disease Influence ATP Concentrations in Red Blood Cells
Presentation Time: Sunday, Dec. 11, 2022, at 10 a.m. CT
Oral Abstract Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Targeting the Red Blood Cell: Novel Therapeutic Approaches in Sickle Cell Disease
Abstract: 393
Presenter: Xunde Wang, Ph.D., Sickle Cell Branch, National Heart Lung and Blood Institute, National Institutes of Health

Poster Presentations:

Title: The Pyruvate Kinase Activator Mitapivat Improves Red Blood Cell Deformability and Sickling Kinetics in Adult Patients with Sickle Cell Disease
Poster Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1044
Lead Author: Maureen Lundt, NHLBI / NIH, Bethesda, Maryland, United States

Title: Feasibility of Near-infrared Spectroscopy for Monitoring Hemodynamic Changes in Sickle Cell Disease Patients Treated with Mitapivat
Poster Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1053
Lead Author: Timothy Quang, National Institute of Child Health and Human Development

Title: Activating Pyruvate Kinase Improves Red Blood Cell Integrity by Reducing Band3 Tyrosine Phosphorylation
Poster Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2367
Lead Author: Kang Le, Ph.D., National Institutes of Health

AG-946
New clinical data from the Phase 1 healthy volunteers study of Agios’ novel PK activator, AG-946, support the initiation of a Phase 2a/2b study in lower-risk myelodysplastic syndromes.

Poster Presentations:

Title: A Phase 2a/2b Multicenter Study of AG-946 in Patients with Anemia Due to Lower-risk Myelodysplastic Syndromes
Poster Session: 637. Myelodysplastic Syndromes – Clinical and Epidemiological: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1773
Lead Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States

Title: Results from the Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers
Poster Session: 114. Hemoglobinopathies, Excluding Thalassemia: Clinical and Epidemiological: Poster II
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 2383
Lead Author: Xiaoshu Dai, Ph.D., Agios Pharmaceuticals

Conference Call Information
Agios will host a live investor event on Dec. 12, 2022, at 7:00 a.m. ET in New Orleans to review the key clinical oral and poster presentations from this year’s ASH (Free ASH Whitepaper) meeting. The event will be webcast live and can be accessed under "Events & Presentations" in the Investors and Media section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On November 3, 2022 GT Biopharma, Inc. (the "Company" or "GTB") (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company’s proprietary natural killer (NK) cell engager, TriKE platform, reported the presentation of new data at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s 64th Annual Meeting (ASH 2022) taking place in New Orleans, LA, and virtually December 10-13, 2022 (Press release, GT Biopharma, NOV 3, 2022, View Source [SID1234622903]). The presentation highlights a novel dual antigen targeting approach for the treatment of acute myeloid leukemia (AML) by combining its TriKE platform and the induced pluripotent stem cell (iPSC) product platform of Fate Therapeutics, Inc.

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The study utilized a combination of GT Biopharma’s investigational GTB-3650, a CD33 targeting Tri-specific Killer Engager (TriKE), and Fate Therapeutics’ multiplexed-engineered, iPSC-derived, CAR NK cells targeting the α3 domain of MICA and MICB. Preclinical data to be presented demonstrate that iPSC-derived, MICA/B-targeted CAR NK cells induced potent activity against the AML cell line HL60, and that further enhancement of activity was achieved in combination with GTB-3650 through antibody-dependent cellular cytotoxicity.

Poster Presentation

Title: A Novel Dual-Antigen Targeting Approach Enables Off-the-Shelf CAR NK Cells to Effectively Recognize and Eliminate the Heterogenous Population Associated with AML
Abstract Number: 4623
Category: Cellular Immunotherapies: Basic and Translational
Presenter: Zachary David, PhD
Date: December 12, 2022
Location and time: Ernest N. Morial Convention Center, Hall D, New Orleans, LA from 6:00pm – 8:00pm ET

About ASH (Free ASH Whitepaper)

The American Society of Hematology (ASH) (Free ASH Whitepaper) (www.hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 60 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH (Free ASH Whitepaper)’s flagship journal, Blood (www.bloodjournal.org), is the most cited peer-reviewed publication in the field, and Blood Advances (www.bloodadvances.org) is the Society’s online, peer-reviewed open-access journal.

On November 3, 2022 CTI BioPharma Corp. (Nasdaq: CTIC) reported an oral presentation and two poster presentations from the Company’s pacritinib program at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place in New Orleans, Louisiana and virtually December 10-13, 2022 (Press release, CTI BioPharma, NOV 3, 2022, View Source [SID1234622902]).

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The details of the oral presentation are as follows:

Abstract Title: Pacritinib Is a Potent ACVR1 Inhibitor with Significant Anemia Benefit in Patients with Myelofibrosis
Abstract Number: 628
Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Towards Personalized Medicine in Myeloproliferative Neoplasms and Mastocytosis: New and Repurposed Drugs for Unmet Clinical Needs
Session Date: Sunday, December 11, 2022
Presentation Time: 5:15–5:30 p.m. CST/6:15–6:30 p.m. EST
Presenter: Dr. Stephen Oh

The details of the poster presentations are as follows:

Abstract Title: Differential Impact of Thrombocytopenia and Anemia on Myelofibrosis (MF) Symptom Burden
Abstract Number: 1712
Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I
Session Date: Saturday, December 10, 2022
Presentation Time: 5:30–7:30 p.m. CST/6:30–8:30 p.m. EST
Presenter: Dr. Jeanne Palmer
Abstract Title: PACIFICA: A Randomized, Controlled Phase 3 Study of Pacritinib Versus Physician’s Choice in Patients with Primary or Secondary Myelofibrosis and Severe Thrombocytopenia

Abstract Number: 4316
Session Name: 631. Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Poster III
Session Date: Monday, December 12, 202
Presentation Time: 6:00–8:00 p.m. CST/7:00–9:00 p.m. EST
Presenter: Dr. John Mascarenhas

About VONJO (pacritinib)
Pacritinib is an oral kinase inhibitor with activity against wild type Janus Associated Kinase 2 (JAK2), mutant JAK2V617F form, IRAK1, ACVR1 (ALK2) and FMS-like tyrosine kinase 3 (FLT3), which contribute to signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. Myelofibrosis is often associated with dysregulated JAK2 signaling. At clinically relevant concentrations, pacritinib does not inhibit JAK1.

VONJO is indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). CTI is conducting the Phase 3 PACIFICA study of VONJO in patients with myelofibrosis and severe thrombocytopenia as a post-marketing requirement.