Hemogenyx Pharmaceuticals PLC Announces FDA Annual Report

Rhea-AI Summary

Hemogenyx Pharmaceuticals (LSE:HEMO) has submitted its Annual Report to the FDA under the active Investigational New Drug (IND) application for HG-CT-1, their CAR-T cell therapy targeting relapsed or refractory acute myeloid leukemia (R/R AML).

The report covers the first year of clinical trials through February 6, 2025, and includes:

• Study information detailing trial design, objectives, and confirmation of first patient enrollment
• Quality data on HG-CT-1 drug product stability and lentiviral vector manufacturing
• Forward-looking plans for continued patient enrollment

The submission represents a significant milestone in HG-CT-1's clinical development, demonstrating the company's progress in advancing a potential therapy for R/R AML patients.

Positive

• First patient successfully enrolled in HG-CT-1 clinical trial
• Active IND status maintained with FDA compliance
• Clinical development progressing as planned

Negative

• None.

Hemogenyx Pharmaceuticals Files Annual IND Report with FDA for HG-CT-1 CAR-T Therapy for AML

LONDON, UK / ACCESS Newswire / April 3, 2025 / 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 first year of the clinical trial of HG-CT-1 and includes the following key elements:

1. Individual Study Information:

A summary of the ongoing clinical study, including its title, design, purpose and objectives, patient population, the total number of planned subjects, and the number enrolled as of the IND anniversary date (February 6, 2025). The report confirms the enrollment of the first patient and includes demographic details such as age, sex, and race.

2. Quality Summary Information:

Data obtained from the past year's investigations under the IND related to the stability of the HG-CT-1 drug product and the lentiviral vector used in its manufacturing.

3. Update to the General Investigational Plan:

A forward-looking update outlining the plan for continued patient enrollment during the upcoming year of the study.

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 with stakeholders.

Further updates will be provided as the trial progresses.

Dr. Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented:
"Submitting our first annual IND report to the FDA is an important milestone that underscores the steady progress we are making in the clinical development of HG-CT-1. For patients facing relapsed or refractory AML, it brings us one step closer to delivering a potentially life-saving therapy. For our investors and partners, it demonstrates our continued execution, scientific rigor, and commitment to transparency. We are advancing with purpose and precision-guided by the urgency of patient need and the confidence of our supporters."

Enquiries:

Hemogenyx Pharmaceuticals plc	https://hemogenyx.com
Dr Vladislav Sandler, Chief Executive Officer & Co-Founder	headquarters@hemogenyx.com
Peter Redmond, Director	peter.redmond@hemogenyx.com
SP Angel Corporate Finance LLP	Tel: +44 (0)20 3470 0470
Matthew Johnson, Vadim Alexandre, Adam Cowl
Peterhouse Capital Limited	Tel: +44 (0)20 7469 0930
Lucy Williams, Duncan Vasey, Charles Goodfellow

About Hemogenyx Pharmaceuticals plc

Hemogenyx Pharmaceuticals is a publicly traded company (LSE:HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility.

The Company is a clinical-stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune disease and to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development.

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SOURCE: Hemogenyx Pharmaceuticals PLC

View the original press release on ACCESS Newswire

FAQ

What is the current development stage of Hemogenyx's HG-CT-1 CAR-T therapy for AML (HOPHF)?

HG-CT-1 is in clinical trials, with the first patient enrolled and the first annual IND report submitted to the FDA as of February 2025.

What are the key components included in Hemogenyx's FDA annual report for HG-CT-1?

The report includes study information with enrollment data, quality summary on drug stability and viral vectors, and plans for future patient enrollment.

What type of cancer does Hemogenyx's HG-CT-1 therapy target?

HG-CT-1 targets relapsed or refractory acute myeloid leukemia (R/R AML).

When did Hemogenyx's HG-CT-1 IND application anniversary date occur?

The IND application anniversary date was February 6, 2025.