Hemogenyx Pharmaceuticals PLC Announces Clearance to Initiate Pediatric Enrolment

Rhea-AI Summary

Hemogenyx Pharmaceuticals (HOPHF) announced on October 6, 2025 that the Institutional Review Board at MD Anderson Cancer Center approved an amendment to its Phase I HG-CT-1 clinical protocol to include children and adolescents with relapsed or refractory acute myeloid leukemia (R/R AML).

The PR states this expansion builds on safety and early efficacy signals observed in adult participants and allows the company to begin pediatric enrolment in the HG-CT-1 trial; the company says it will provide further updates as adult and pediatric enrolment progresses.

Positive

• MD Anderson IRB approval to expand HG-CT-1 eligibility to pediatric patients
• Pediatric enrolment enabled for Phase I HG-CT-1 trial as of Oct 6, 2025
• Company cites safety and early efficacy signals in adult participants supporting expansion

Negative

• IRB clearance permits pediatric enrolment but is not regulatory marketing approval
• HG-CT-1 remains in Phase I, so clinical efficacy and commercial outcome remain unproven

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MD Anderson Clearance to Initiate Pediatric Enrolment in HG-CT-1 Clinical Trial

LONDON, GB / ACCESS Newswire / October 6, 2025 / Hemogenyx Pharmaceuticals plc (LSE:HEMO) is pleased to announce that the Institutional Review Board ("IRB") at MD Anderson Cancer Center ("MD Anderson") has approved an amendment to the clinical protocol of the Company's ongoing Phase I trial of HG-CT-1, its proprietary CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia ("R/R AML").

The approved amendment expands the eligibility criteria for the trial to include children and adolescents with R/R AML, allowing the Company to begin enrolling pediatric patients into the study. This important step builds on the safety and early efficacy signals already observed in adult participants and significantly broadens the potential patient population who may benefit from HG-CT-1.

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

"Securing IRB clearance to proceed with pediatric enrolment in our HG-CT-1 clinical trial is a major milestone. It marks the transition of HG-CT-1 into the pediatric setting, where treatment options remain limited, and demonstrates the continued momentum of our development program. This expansion not only increases the potential reach and impact of HG-CT-1 but also underscores the value of the Company's strong intellectual property portfolio, which underpins our pipeline and supports long-term growth."

The Company will provide further updates as both adult and pediatric patient enrolment progresses.

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.

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.

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SOURCE: Hemogenyx Pharmaceuticals PLC

View the original press release on ACCESS Newswire

FAQ

What did Hemogenyx (HOPHF) announce on October 6, 2025 about HG-CT-1?

The company announced MD Anderson IRB approval to amend the Phase I protocol to include children and adolescents with R/R AML, enabling pediatric enrolment.

Does the MD Anderson IRB approval mean HG-CT-1 has regulatory approval for pediatric use?

No. According to the PR, IRB approval allows pediatric enrolment in the clinical trial but is not a regulatory marketing approval.

When can Hemogenyx (HOPHF) start enrolling pediatric patients into the HG-CT-1 trial?

Per the PR dated October 6, 2025, the company may begin enrolling pediatric patients following the IRB-approved protocol amendment.

How does the protocol amendment affect Hemogenyx's clinical development timeline?

The PR states the amendment expands the eligible patient population and the company will provide further updates as adult and pediatric enrolment progresses.

What evidence did Hemogenyx cite to support pediatric expansion of HG-CT-1?

The PR cites observed safety and early efficacy signals in adult participants as the basis for expanding enrolment to pediatrics.