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.

On November 3, 2022 Loxo@Lilly, the oncology unit of Eli Lilly and Company (NYSE: LLY), reported that study investigators will present data from the BRUIN Phase 1/2 trial of pirtobrutinib at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting to be held December 10-13, 2022, in New Orleans, Louisiana, and virtually (Press release, Eli Lilly, NOV 3, 2022, View Source [SID1234622901]). Pirtobrutinib is an investigational, highly selective, potent, reversible inhibitor of the Bruton’s tyrosine kinase (BTK).

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The pirtobrutinib oral and poster presentations will provide updated clinical data from the ongoing BRUIN Phase 1/2 study in previously treated chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL), Richter transformation, and Waldenström macroglobulinemia. In addition, an analysis of the safety and tolerability of pirtobrutinib monotherapy in patients with relapsed or refractory B-cell malignancies who were intolerant to a prior covalent BTK inhibitor will be presented in a poster presentation. Submitted abstracts on CLL/SLL, Richter transformation, Waldenström macroglobulinemia, and intolerance to prior covalent BTK therapy utilized a January 2022 data cut-off date, and the presentations will utilize a July 2022 data cut-off date.

About Pirtobrutinib (LOXO-305)
Pirtobrutinib is an investigational, highly selective, reversible (non-covalent) Bruton’s tyrosine kinase (BTK) inhibitor. BTK plays a key role in the B-cell antigen receptor signaling pathway, which is required for the development, activation and survival of normal white blood cells, known as B-cells, and malignant B-cells. BTK is a validated molecular target found across numerous B-cell leukemias and lymphomas including chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), and Waldenström macroglobulinemia. Pirtobrutinib was developed to reversibly bind BTK, deliver consistently high target coverage regardless of BTK turnover rate, and preserve activity in the presence of the C481 acquired resistance mutations. Interested patients and physicians can contact the Loxo@Lilly Physician and Patient BTK Clinical Trial Hotline at 1-855-LOXO-305 or email [email protected].

About the BRUIN Phase 1/2 Trial
The BRUIN Phase 1/2 clinical trial is the ongoing first-in-human, global, multi-center evaluation of pirtobrutinib in patients previously treated for mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), or other non-Hodgkin lymphomas (NHL).

The trial includes a Phase 1 dose-escalation phase, a Phase 1b combination arm, and a Phase 2 dose-expansion phase. The primary endpoint of the Phase 1/1b study is safety, and secondary endpoints include pharmacokinetics and preliminary efficacy of the drug combinations. The primary endpoint for Phase 2 is overall response rate. Secondary endpoints include duration of response, overall survival, safety, and pharmacokinetics.

On November 3, 2022 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases reported that it will have a poster presentation for ALPN-303’s phase 1 study (RUBY-1) at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, December 10-13, 2022 (Press release, Alpine Immune Sciences, NOV 3, 2022, View Source [SID1234622900]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Date/Time: December 12, 2022, at 6:00pm – 8:00pm EST
Poster Title: A Randomized, Placebo-Controlled, Phase 1 Study of Healthy Adult Volunteers of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALPN-303, a Potent Dual BAFF/APRIL Antagonist for the Treatment of Autoimmune Cytopenias
Poster Number: 3763
Session Name: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster III
Location: Ernest N. Morial Convention Center, Hall D

About ALPN-303 and the RUBY-1 Phase 1 Study

ALPN-303 is a dual antagonist of the BAFF (B cell activating factor) and APRIL (proliferation inducing ligand) cytokines, which play key roles in the survival and activation of B cells. Based upon an engineered TACI (transmembrane activator and CAML interactor) domain, ALPN-303 exhibits greater potency in preclinical studies versus comparators based on wild-type TACI, as well as other inhibitors of BAFF and/or APRIL alone. ALPN-303 is in development for multiple B cell and/or autoantibody-related diseases, such as systemic lupus erythematosus, glomerulonephritides, and autoimmune cytopenias.

RUBY-1 (NCT05034484) is a phase 1, randomized, placebo-controlled study in healthy adult volunteers designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single doses of intravenously and subcutaneously administered ALPN-303. Initial data show ALPN-303 to be well tolerated up to 960 mg with dose-dependent pharmacokinetics and reductions in circulating immunoglobulins and antibody-secreting cells, supporting the use of a once every four-week dose regimen for subsequent studies.