On November 2, 2023 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported that a broad set of clinical and translational data from its programs will be presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, to be held Dec. 9-12, 2023, in San Diego (Press release, Agios Pharmaceuticals, NOV 2, 2023, View Source [SID1234636721]). The presentations will focus on rare blood disorders, including PK deficiency, thalassemia, sickle cell disease and anemia associated with lower-risk myelodysplastic syndromes (LR-MDS).

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In total, 23 abstracts led by Agios and external collaborators will be presented or published. 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 (mitapivat) in adults with PK deficiency is associated with sustained clinical benefits, including improvements in hemoglobin, iron overload, and decreased burden of disease on work and school activities.

Poster Presentations:

Title: Mitapivat Treatment Reduces Levels of Interference in Work/School Activity for Adult Patients with Pyruvate Kinase Deficiency
Poster Session: 904. Outcomes Research – Non-Malignant Conditions: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 2365
Lead Author: Jennifer A. Rothman, MD, Duke University Medical Center, Durham, NC

Title: Understanding the Physical and Psychosocial Impacts of Pyruvate Kinase Deficiency: Patient-Led Development of the Pyruvate Kinase Deficiency Life Phase Model
Poster Session: 901. Health Services and Quality Improvement – Non-Malignant Conditions: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 3691
Lead Author: Rachael F. Grace, MD, MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, Harvard Medical School, Boston, MA

Publication Only:

Title: Improvements in Markers of Hemolysis and Liver Iron Concentration in Mitapivat-Treated Adult Patients with a Delayed Hemoglobin Response
Abstract: 5266
Lead Author: Eduard J. van Beers, MD, PhD; Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands

Title: Regional Genetic Heterogeneity Among Patients with Pyruvate Kinase Deficiency
Abstract: 5203
Lead Author: Paola Bianchi, BSc, PhD; Hematology Unit, Pathophysiology of Anemia Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy

Thalassemia
Data for PYRUKYND in adults with beta-thalassemia show evidence of reduced oxidative stress in red blood cells, as well as enhanced PK activity and metabolic reprogramming. Additionally, qualitative data demonstrate the equally common burden of disease across alpha- and beta-thalassemia patients, regardless of transfusion dependency.

Poster Presentations:

Title: Mitapivat Treatment Increases β-thalassemic Erythroblasts Energy Production and Responsiveness to Oxidative Stress
Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster III
Session Date and Time: Monday, Dec. 11, 2023, 6:00-8:00 p.m. PT
Abstract: 3850
Lead Author: Alessandro Matte, PhD; Department of Medicine, University of Verona and AOUI Verona, Verona, Italy

Publication Only:

Title: Association of Hemoglobin Levels With Healthcare Resource Utilization and Costs in Non–Transfusion-Dependent α- and β-Thalassemia: A Retrospective Observational Study Using Real-World Data
Abstract: 5244
Lead Author: Arielle L. Langer, MD; MPH, Division of Hematology, Brigham & Women’s Hospital, Harvard Medical School, Boston MA

Title: Burden of Illness of Alpha- and Beta-Thalassemia: A Qualitative Study
Abstract: 7329
Lead Author: Sujit Sheth, MD; Joan and Sanford I Weill Medical College of Cornell University, New York, NY

Title: Investigating Health Literacy in Thalassemia: Founding a Patient-Led Research Approach
Abstract: 5251
Lead Author: Sujit Sheth, MD; Joan and Sanford I Weill Medical College of Cornell University, New York, NY

Sickle Cell Disease
Results from the Phase 2 portion of Agios’ RISE UP study will highlight improvements in hemoglobin response rates, markers of hemolysis and erythropoiesis and reductions in annualized rates of pain crises for mitapivat compared to placebo, supporting advancement into the Phase 3 portion of RISE UP. Agios will showcase its patient-centric approach to sickle cell disease clinic trials, including the incorporation of patient insights and decision making into the clinical trial design and campaign.

Oral Presentations:

Title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study of Mitapivat in Patients With Sickle Cell Disease: RISE UP Phase 2 Results
Presentation Time: Saturday, Dec. 9, 2023, at 4:00 p.m. PT
Oral Abstract Session: 114. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Building on Momentum in Disease-Modifying Therapeutics for Sickle Cell Disease
Abstract: 271
Presenter: Modupe Idowu, MD; McGovern Medical School, UT Health, Houston, TX

Title: Long-term safety and Efficacy of Mitapivat, an Oral Pyruvate Kinase Activator, in Adults with Sickle Cell Disease: Extension of a Phase 1 Dose Escalation Study
Presentation Time: Saturday, Dec. 9, 2023, at 4:30 p.m. PT
Oral Abstract Session: 114. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Building on Momentum in Disease-Modifying Therapeutics for Sickle Cell Disease
Abstract: 273
Presenter: Swee Lay Thein, MBBS, DSc, FRCP, FRCPath, MRCP, MRCPath; Sickle Cell Branch, National Heart, Lung & Blood Institute, NIH, Bethesda, MD

Title: Pyruvate Kinase Thermostability Is Associated with Red Blood Cell Adhesion, Deformability and Oxygen Affinity in Patients with Sickle Cell Disease
Presentation Time: Sunday, Dec. 10, 2023, at 5:00 p.m. PT
Oral Abstract Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Pathophysiology of Sickle Hemoglobinopathies: From Mice to Humans
Abstract: 561
Lead Author: Marissa J.M. Traets, PhD Candidate; Department of Central Diagnostic Laboratory – Research, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands

Poster Presentations:

Title: A Patient-Centric Approach to Sickle Cell Disease Clinical Trials: Integrating Patient Perspectives in the RISE UP Phase 2/3 Trial of Mitapivat for Informed Protocol Design and Associated Patient Community Benefit
Poster Session: 904. Outcomes Research – Non-Malignant Conditions: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 2376
Lead Author: Charles Jonassaint, PhD, MHS; School of Medicine, University of Pittsburgh, Pittsburgh, PA

Title: One-Year Safety and Efficacy of Mitapivat in Sickle Cell Disease: Follow-Up Results of a Phase 2, Open-Label Study
Poster Session: 114. Sickle cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 2515
Lead Author: Myrthe J. van Dijk, PhD; Division Laboratories, Pharmacy and Biomedical Genetics, CDL en Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands

Title: Functional and Multi-omics Signatures of Mitapivat Efficacy Upon Activation of Pyruvate Kinase in Red Blood Cells from Patients with Sickle Cell Disease
Poster Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 2485
Lead Author: Angelo D’Alessandro, PhD; Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO

Title: Longitudinal Characterization of Hemodynamic Changes with Multimodal Optical Techniques in Patients with Sickle Cell Disease Treated with Mitapivat
Poster Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 2492
Lead Author: Timothy Quang, PhD; Section on Biomedical Optics, National Institute of Child Health and Human Development, NIH, Bethesda, MD

Publication Only:

Title: Outpatient Costs of Patients with Sickle Cell Disease With or Without Hydroxyurea at an Institution in Rio de Janeiro, Brazil
Abstract: 7325
Lead Author: Tarun Aurora, MD; Department of Global Pediatric Medicine, St Jude Children’s Research Hospital, Memphis, TN

AG-946
New data from preclinical studies of Agios’ AG-946 continue to support its novel mechanism of action and PK activation as a promising potential treatment option for anemia associated with lower-risk myelodysplastic syndromes.

Poster Presentations:

Title: AG-946, An Activator of Pyruvate Kinase, Improves Ineffective Erythropoiesis in the Bone Marrow of Mouse Models of Myelodysplastic Syndromes
Poster Session: 636. Myelodysplastic Syndromes – Basic and Translational: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 1854
Lead Author: Megan Wind-Rotolo, PhD, Agios Pharmaceuticals, Cambridge, MA

Title: The Pyruvate Kinase (PK) Activator AG-946 Improves PK Properties and Red Blood Cell (RBC) Characteristics upon Ex Vivo Treatment of RBCs from Patients with Myelodysplastic Syndromes
Poster Session: 636. Myelodysplastic Syndromes—Basic and Translational: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 3222
Lead Author: Jonathan R.A. de Wilde, PhD Candidate; Red Blood Cell Research Group, Central Diagnostic Laboratory-Research, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands

Publication Only:

Title: Biochemical and Metabolomic Analysis of Glycolytic Activity in Red Blood Cells (RBC) from Low-risk Myelodysplastic Syndromes (LR-MDS) Patients and In-vitro Effect of the Pyruvate Kinase (PK) Activator AG-946
Abstract: 6456
Lead Author: Bruno Fattizzo, MD; Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy

Patient Advocacy
Agios is fueled by connections, with patient and KOL engagement at the core of our mission. Information presented provides an overview of the formation and vision of the Red Cell Revolution – a multi-stakeholder, patient-advocacy data collection approach to understanding the unmet needs of the patients, caregivers and healthcare professionals for PK deficiency, sickle cell disease and thalassemia.

Poster Presentation:

Title: Cross-community Collaboration and Data Collection to Optimize Patient Care in Hemolytic Anemias
Poster Session: 901. Health Services and Quality Improvement – Non-Malignant Conditions: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 3692
Lead Author: Biree Andemariam, MD; New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT

Publication Only:

Title: Setting Industry Standards for Patient Engagement, Partnership, Allyship and Care: The Patient Vision Project
Abstract: 7233
Lead Author: Biree Andemariam, MD; New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT

Other
Agios’ collaborators present new data evaluating mitapivat as a potential treatment for other rare blood diseases.

Poster Presentation:

Title: Ex Vivo Treatment by Mitapivat, an Allosteric Pyruvate Kinase Activator, Reduced Oxidative Stress and Promoted Terminal Erythropoiesis in a Severe Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 Mutations
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 1071
Lead Author: Thidarat Suksangpleng, PhD.; Siriraj-Thalassemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand

Title: Safety and Efficacy of Mitapivat Sulfate in Adult Patients with Erythrocyte Membranopathies (SATISFY)
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 1085
Lead Author: Andreas Glenthøj, MD, PhD; Department of Haematology, Copenhagen University Hospital – Rigshospitalet, Copenhagen, Denmark

Conference Call Information
Agios will host a live investor event on Dec. 11, 2023, at 7:00 a.m. PT in San Diego 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 2, 2023 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of targeted radiotherapies, reported that it will be attending Bio-Europe, the largest biopharmaceutical industry partnering event in Europe, taking place November 6 – 8, 2023 in Munich, Germany (Press release, Actinium Pharmaceuticals, NOV 2, 2023, View Source [SID1234636720]).

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Actinium is advancing a pipeline of differentiated, clinical-stage targeted radiotherapeutics including Iomab-B for bone marrow transplant conditioning, which met the primary endpoint in the Phase 3 SIERRA trial and is being prepared for a BLA filing, Iomab-ACT for conditioning prior to cell and gene therapy and Actimab-A as a therapeutic for relapsed or refractory acute myeloid leukemia that has shown backbone therapy potential in combinations. Actinium is also advancing novel solid tumor programs leveraging its industry leading experience with the alpha-particle payload Actinium-225 and intellectual property including linker technology and Actinium-225 manufacturing.

Members of Actinium’s senior leadership team will be available for one-on-one meetings during the in-person meeting as well as the digital partnering taking place November 14-15, 2023. To request a meeting with Actinium through partneringONE https://informaconnect.com/bioeurope/. Interested parties may also contact the Company directly to schedule in person or virtual meetings by emailing Actinium.

On November 2, 2023 Imugene Limited (ASX: IMU), a clinical stage immuno-oncology company, reported that its Phase 1 MAST (metastatic advanced solid tumours) trial evaluating the safety of novel cancer-killing virus CF33-hNIS (VAXINIA) has now cleared cohort 4 of the intravenous (IV) arm of the monotherapy dose escalation study, as well as IV cohort 2 of the combination study where VAXINIA is administered with blockbuster checkpoint inhibitor drug pembrolizumab (KEYTRUDA) (Press release, Imugene, NOV 2, 2023, https://mcusercontent.com/e38c43331936a9627acb6427c/files/17bdc1de-a079-186b-11e8-6ef4ab6ae92a/Next_cohort_reached_in_IV_arms_of_CF33_hNIS_VAXINIA_study.pdf [SID1234636670]).

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Cohort 5 of the IV arm for the monotherapy dose escalation is now open as is IV cohort 3 of the combination study.

Imugene MD & CEO Leslie Chong said: "As we near closer to opening and completing the final cohorts that were planned at the beginning of the trial, we have an opportunity to expand the trial by enrolling patients in additional cohorts for the monotherapy dose escalation component. This will provide us with a far more robust data set to analyse and speak to at the conclusion of the MAST study, and provide us with a stronger platform as we further the clinical development of CF33 and VAXINIA."

The multicenter Phase 1 MAST trial commenced by delivering a low dose of VAXINIA to patients with metastatic or advanced solid tumours who have had at least two prior lines of standard of care treatment. The City of Hope-developed oncolytic virus has been shown to shrink colon, lung, breast, ovarian and pancreatic cancer tumours in preclinical laboratory and animal models. Overall the study aims to recruit up to 100 patients across approximately 10 trial sites in the United States and Australia.

The clinical trial is titled "A Phase I, Dose Escalation Safety and Tolerability Study of VAXINIA (CF33- hNIS), Administered Intratumorally or Intravenously as a Monotherapy or in Combination with Pembrolizumab in Adult Patients with Metastatic or Advanced Solid Tumours (MAST)." The trial commenced in May 2022 and is anticipated to run for approximately 24 months while being funded from existing budgets and resources.

Full study details can also be found on clinicaltrials.gov under study ID: NCT05346484.

On November 2, 2023 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported that it will present preliminary data from its ongoing Phase 1/2a trial of MP0533, a novel tetra-specific T cell engager at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held from December 9–12 in San Diego, California (Press release, Molecular Partners, NOV 2, 2023, View Source [SID1234636664]). MP0533 is in development for the treatment of patients with relapsed/refractory acute myeloid leukemia (r/r AML) and myelodysplastic syndrome (AML/MDS).

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As of data cut-off (20 July 2023) of the abstract published today, five patients across three dosing regimens had been treated. The preliminary data reported indicate an acceptable safety profile, with no dose-limiting toxicity or Grade ≥3 adverse reactions. Grade 1/2 events considered related to MP0533 included infusion-related reactions and cytokine release syndromes. One of the two patients evaluable for MP0533 antitumor activity in the third treatment cohort achieved a response. The study is currently enrolling its fifth cohort with up to seven dose-escalating cohorts planned and a total enrollment of up to 45 patients. The Company anticipates to present data including from the fourth dose cohort at the ASH (Free ASH Whitepaper) Annual Meeting and Exposition in December this year.

"The data from the ASH (Free ASH Whitepaper) abstract represent the beginning of an exciting and encouraging clinical journey for the MP0533 program. We are now able to show initial clinical activity of the first tetra-specific, non-antibody-based T cell engager, MP0533, in patients with r/r AML and MDS/AML," said Patrick Amstutz, Ph.D., Molecular Partners’ CEO. "We see both an acceptable tolerability profile at initial doses, as well as the emergence of single-agent anti-tumor activity at relatively low dose levels and we look forward to presenting additional data on MP0533’s potential to treat this particularly intractable blood cancer at the ASH (Free ASH Whitepaper) Annual Meeting in December."

The clonal heterogeneity and lack of single AML-specific target antigens represent major challenges for the development of targeted immune therapies for AML. To overcome these hurdles, Molecular Partners designed MP0533, a novel tetra-specific T cell-engaging, half-life extended DARPin, which simultaneously targets CD33, CD123 and CD70, as well as CD3 on T cells. This unique mode of action is designed to enable avidity-driven, T cell-mediated killing of leukemic stem cells and malignant blast cells, which commonly co-express at least two of the three target antigens, while preserving a therapeutic window that minimizes damage to healthy cells.

The ongoing single-arm, open-label, multicenter Phase 1/2a study of MP0533 is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics, as well as assess preliminary antileukemic activity of MP0533 as a monotherapy for patients with r/r AML and AML/MDS.

The presentation details are as follows:

Session Name: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster 2
Publication Number: 2921
Title: MP0533, a CD3-Engaging DARPin Targeting CD33, CD123, and CD70 in Patients with Relapsed/Refractory AML or MDS/AML: Preliminary Results of a Phase 1/2a Study
Session Location & Date: San Diego Convention Center, Halls G-H; Sunday, December 10, 2023
Presentation Time: 6:00–8:00 pm PT

The abstract will become available today on the ASH (Free ASH Whitepaper) website at 9:00 am ET.

On November 1, 2023 Oblique Therapeutics reported its Interim report 1 July to 30 September 2023 (Press release, Oblique Therapeutics, NOV 1, 2023, https://obliquet.com/wp-content/uploads/2023/11/delarsrapport-q3-2023-oblique-therapeutics-ab.pdf [SID1234640429]).

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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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