Annual IND Report with FDA

In brief · summary, not quotable

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.

Full announcement

Select text to share a quote on X · sign in to keep highlights & notes in your HEMO notes

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:

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.

Quality Summary Information:

The report includes data from investigations conducted during the reporting period relating to the stability of the HG-CT-1 drug product and its lentiviral vector manufacturing process, supporting continued clinical development.

Update to the General Investigational Plan:

The Company plans to continue patient enrollment and dose escalation in the upcoming year to further evaluate the safety profile and potential anti-leukemic activity of HG-CT-1.

This filing marks another important step in the Company's clinical development of HG-CT-1 and reaffirms its commitment to regulatory compliance and transparent communication.

Further updates will be provided as the trial progresses.

Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented:

"Submitting our second annual IND report to the FDA represents continued progress in the development of HG-CT-1. While early-stage data remain preliminary, we are encouraged by the observed biological activity and manageable safety profile. We are focused on advancing the study through additional dose levels to further evaluate the therapeutic potential of HG-CT-1 for patients with relapsed or refractory AML."

Cleaned text: letterheads, contacts and legal notices removed. View the original announcement ↗ · Company filings. Not investment advice.