Hemogenyx Pharmaceuticals PLC Announces Clearance to Proceed with Pediatric Expansion

Rhea-AI Summary

Hemogenyx Pharmaceuticals (HOPHF) has received FDA clearance to expand its Phase I clinical trial of HG-CT-1, a proprietary CAR-T therapy for relapsed/refractory acute myeloid leukemia (R/R AML), to include pediatric and adolescent patients. The FDA's 30-day review period concluded without a clinical hold, allowing the company to proceed with Institutional Review Board submissions and site activation procedures for pediatric cohorts. This expansion broadens the potential therapeutic reach of HG-CT-1 and represents a significant milestone in the company's development program, particularly in addressing the needs of younger patients with this aggressive form of leukemia.

Positive

• FDA clearance received for pediatric expansion of HG-CT-1 clinical trial, expanding the potential market and therapeutic applications
• Successful completion of FDA review period without clinical hold indicates positive safety profile
• Expansion to pediatric patients strengthens the company's intellectual property portfolio and market position

Negative

• None.

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.

Regulatory Clearance to Proceed with Pediatric Expansion of HG-CT-1 Clinical Trial

LONDON, UK / ACCESS Newswire / June 17, 2025 / Hemogenyx Pharmaceuticals plc (LSE:HEMO) is pleased to announce that the 30-day review period by the U.S. Food and Drug Administration (FDA) for the Company's previously submitted amendment to the clinical protocol of its ongoing Phase I trial of HG-CT-1 has concluded without a clinical hold. As a result, the Company is now cleared to proceed with the next steps required to initiate pediatric enrolment in the trial.

The protocol amendment expands the eligibility criteria for the Phase I trial of HG-CT-1, Hemogenyx's proprietary CAR-T therapy for relapsed/refractory acute myeloid leukemia (R/R AML), to include children and adolescents with this aggressive and hard-to-treat disease.

The Company will now move forward with Institutional Review Board (IRB) submissions and associated site activation procedures to enable the opening of pediatric cohorts.

Dr. Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented:
"Regulatory clearance to proceed with the pediatric expansion of our HG-CT-1 clinical trial is a significant milestone. It reflects continued momentum in our development program and underscores our commitment to delivering innovative therapies to patients across age groups. This expansion broadens the potential impact and value of HG-CT-1 as we continue to advance toward key inflection points in the clinic. Importantly, it also reinforces the value of Company's robust intellectual property portfolio, which underpins our pipeline and supports long-term strategic growth."

The Company will update the market as further progress is made in pediatric site activation and patient enrolment.

Market Abuse Regulation (MAR) Disclosure

Certain information contained in this announcement would have been inside information for the purposes of Article 7 of Regulation No 596/2014 (as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018) until the release of this announcement. The person responsible for arranging for the release of this announcement on behalf of Hemogenyx Pharmaceuticals plc is Dr Vladislav Sandler, Chief Executive Officer & Co-Founder.

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 diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as platform technologies that it uses as engines for novel product development.

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit www.rns.com.

SOURCE: Hemogenyx Pharmaceuticals PLC

View the original press release on ACCESS Newswire

FAQ

What is the significance of FDA clearance for Hemogenyx's (HOPHF) pediatric trial expansion?

The FDA clearance allows Hemogenyx to include children and adolescents in its Phase I trial of HG-CT-1, expanding the potential therapeutic reach for treating relapsed/refractory acute myeloid leukemia (R/R AML).

What is HG-CT-1 and what condition does it treat?

HG-CT-1 is Hemogenyx's proprietary CAR-T therapy designed to treat relapsed/refractory acute myeloid leukemia (R/R AML), an aggressive and hard-to-treat blood cancer.

What are the next steps for Hemogenyx (HOPHF) following FDA clearance?

The company will proceed with Institutional Review Board (IRB) submissions and site activation procedures to begin enrolling pediatric patients in the clinical trial.

How does the pediatric expansion affect Hemogenyx's (HOPHF) business potential?

The expansion broadens HG-CT-1's potential market impact, strengthens the company's intellectual property portfolio, and supports long-term strategic growth by addressing a wider patient population.