Business
Annual IND Report with FDA
Hemogenyx Pharmaceuticals plc has filed its annual Investigational New Drug (IND) report with the FDA for HG-CT-1, a CAR-T cell therapy for relapsed or refractory acute myeloid leukemia. The report covers the second year of the Phase 1 clinical trial, which aims to enroll up to 36 patients. So far, three adult patients treated at the initial lowest dose level of 7 × 10⁷ CAR⁺ cells have shown CAR-T cell expansion and persistence, blast burden reductions, and no reported immune effector cell-associated neurotoxicity syndrome or dose-limiting toxicities, with generally low-grade and manageable adverse events. The company plans to continue patient enrollment and dose escalation in the upcoming year. Disclaimer*
About this update from Hemogenyx Pharmaceuticals Plc
THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION AS STIPULATED UNDER THE UK VERSION OF THE MARKET ABUSE REGULATION NO 596/2014 WHICH IS PART OF ENGLISH LAW BY VIRTUE OF THE EUROPEAN (WITHDRAWAL) ACT 2018, AS AMENDED. ON PUBLICATION OF THIS ANNOUNCEMENT VIA A REGULATORY INFORMATION SERVICE, THIS INFORMATION IS CONSIDERED TO BE IN THE PUBLIC DOMAIN. 14 April 2026 Hemogenyx Pharmaceuticals plc ("Hemogenyx Pharmaceuticals" or the "Company") Hemogenyx Pharmaceuticals Files Annual IND Report with FDA for HG-CT-1 CAR-T Therapy for AML Hemogenyx Pharmaceuticals plc (LSE: HEMO) is pleased to announce that it has submitted its Annual Report to the U.S. Food and Drug Administration (FDA) under the active Investigational New Drug (IND) application for HG-CT-1, the Company's proprietary CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML). The annual report provides a comprehensive update on the Company's activities under the IND during the second year of the clinical trial of HG-CT-1 and includes the following key elements: 1. Individual Study Information: The ongoing Phase 1 study is designed to evaluate the safety and preliminary efficacy of HG-CT-1 in adult and pediatric patients with R/R AML. The study plans to enroll up to 36 evaluable subjects (18 adults and 18 pediatric patients), with a primary objective of assessing safety based on dose-limiting toxicities. As of the IND anniversary date, three adult patients have been enrolled and treated at the initial lowest dose level (7 × 10⁷ CAR+ cells). Across these patients: · CAR-T cell expansion and persistence were observed in all subjects, with peak levels typically occurring between 14 and 28 days post-infusion · Reductions in blast burden were observed in peripheral blood and/or bone marrow · No immune effector cell-associated neurotoxicity syndrome (ICANS) or dose-limiting toxicities (DLTs) were reported · Adverse events were generally low grade and manageable These findings remain preliminary due to the limited number of patients and evaluation at a single dose level. 2. Quality Summary Information: The report includes data from investigations conducted during the reporting period relating to th...
View stock analysis, news, and events for Hemogenyx Pharmaceuticals Plc