Hemogenyx Pharmaceuticals (HEMO) , a biopharma company developing therapies for blood diseases, said it has received IRB approval for a Phase 1 clinical trial of its lead asset HEMO-CAR-T for the treatment of relapsed/refractory acute myeloid leukemia (R/R AML) in adults. Following approval by the International Review Board, HEMO-CAR-T was given the formal designation HG-CT-1.
Hemogenyx's Phase 1 trial for HG-CT-1 will be a dose escalation study, with its primary objective to assess the safety of the treatment in adult patients with R/R AML. Secondary objectives include estimating the efficacy of the treatment, the overall survival, progression-free survival, and duration of response in evaluable subjects.
Vladislav Sandler, CEO of Hemogenyx Pharmaceuticals, commenting: "We are excited to receive IRB approval to proceed with our Phase I clinical trial of HEMO-CAR-T. This trial is a critical step in the development of our CAR-T therapy for AML patients who have exhausted other treatment options. With this study, we aim to establish the safety profile of HG-CT-1 and gather preliminary efficacy data that could pave the way for future therapeutic development."
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Hemogenyx (mktcap £22.6m) clears a regulatory hurdle in the development of its flagship treatment for R/R AML, now known as HG-CT-1. The asset has been cleared by the IRB for a Phase 1 study that will evaluate its safety as well as number of key secondary characteristics. The secondary objectives are crucial for assessing the broader impact of HG-CT-1 and include efficacy, survival, and duration of response metrics.
The study will pave the way for commercialisation of HG-CT-1, a promising treatment for patients with R/R AML who currently have few therapeutic options. AML is the most common type of acute leukemia in adults. It has poor survival rates, with a 5-year rate of less than 30% in adults. It is currently treated with chemotherapy rather than potentially more benign and effective forms of therapy such as HEMO's HG-CT-1.
The successful development of a new therapy for AML would have a major impact on treatment and survival rates for the disease, and would be a major value inflection point for HEMO. Overall, IRB approval marks a significant milestone for the now clinical-stage company, enabling it to advance its disruptive lead therapy into clinical testing at one of the world's top cancer research institutions. Markets welcomed the news, driving HEMO shares 14% higher in early Friday trade.
HEMO recently raised £600k from an institutional investor for the Phase 1 clinical trial of HG-CT-1, adding to £3.32m raised in 1H24 for the asset and leaving sufficient runway to continue development. In 1H24, HEMO narrowed its pre-tax loss to £2.82m from £4.32m in 1H23.
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