rebecsinib (AT44216) / Aspera Biomedicines - LARVOL DELTA

A Study of Rebecsinib for Patients With Relapsed/Refractory Secondary Acute Myeloid Leukemia or High Risk Myelofibrosis (clinicaltrials.gov) - P1 | N=28 | Recruiting | Sponsor: Aspera Biomedicines, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Oncology • FLT3

Predicting and preventing cancer stemness in low Earth orbit (AACR 2026) - "Moreover, in Axiom 2 and 3 missions with ADAR1-reporter expressing MBC organoids and in humanized MBC mouse models, an ADAR1p150 splicing modulator, rebecsinib (IND 153126), prevented tumor propagation. Thus, cancer studies in LEO may accelerate the development of innovative cancer therapeutics and countermeasures for long-term spaceflight."

Breast Cancer • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • ADAR

Therapeutic targeting of ADAR1 p150 splicing activity impairs CD44+ TNBC cell populations in preclinical studies (AACR 2026) - "Rebecsinib selectively reduces CD44+ and ADAR1+ TNBC cell populations and inhibits tumor progression in humanized preclinical models. These findings support further evaluation of Rebecsinib alone and in combination, as a targeted therapeutic approach for TNBC.Keywords: TNBC, ADAR1, CD44, Rebecsinib, targeted therapy, preclinical models."

Preclinical • Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • ADAR

RNA Editing ADAR1 Deaminase Activation Responsive (READAR) platforms for dynamic detection of clonal myeloproliferative neoplasm stem cell fitness (AACR 2024) - "In total, 44.2% (N=57) of patients in our cohort received Inrebic, with 56% reaching the maximum dose of 400mg. A significant reduction in ADAR1-GFP reporter activity was observed in MPN CD34+ cells following Rebecsinib treatment of stromal co-cultures. Also, humanized ADAR1-GFP-luciferase reporter AML mouse models showed a significant reduction in ADAR1 activity and leukemia stem cell self-renewal following Rebecsinib treatment.Conclusion These READAR platform results suggest that ADAR1 activity can be utilized to predict clonal MPN stem cell fitness and progression."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myeloproliferative Neoplasm • Oncology • ADAR • ASXL1 • CALR • CD34 • JAK2

A Study of Rebecsinib for Patients With Relapsed/Refractory Secondary Acute Myeloid Leukemia or High Risk Myelofibrosis (clinicaltrials.gov) - P1 | N=28 | Not yet recruiting | Sponsor: Aspera Biomedicines, Inc.

New P1 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Oncology