The European Medicines Agency (EMA) has approved Moleculin Biotech’s clinical trial application (CTA) for a pivotal, multi-centre Phase IIb/III trial of Annamycin plus cytarabine (Ara-C) for adults with acute myeloid leukaemia (AML).
The randomised, placebo-controlled, double-blind trial is intended for AML individuals who are refractory to or relapsed post-induction therapy (r/r AML).
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Moleculin noted that the trial, a global study including sites in Europe, the Middle East, and the US, has been approved in all nine European Union (EU) nations where it was submitted. This trial will combine data from the Phase IIb portion with the Phase III portion to measure its primary efficacy endpoint.
The trial’s adaptive design will see the first 75 to 90 participants randomised in Part A to obtain high-dose cytarabine (HiDAC) with either a placebo or one of two doses, 190mg/m² or 230mg/m² of Annamycin, as per the US Food and Drug Administration’s (FDA) recommendation.
According to the trial protocol, the preliminary efficacy and safety data will be unblinded after 45 subjects have been treated, with the first early unblinding expected in the second half of this year.
The EMA’s approval is contingent upon the presentation of nonclinical good laboratory practice (GLP) study results before commencing the Phase III portion of the study.
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By GlobalDataNearly 220 additional participants will be randomised in Part B to determine the optimum dose of Annamycin’s ideal dose with HiDAC or HiDAC with a placebo.
The optimal dose choice will be based on the overall balance of pharmacokinetics, efficacy, and safety, in line with the FDA’s Project Optimus initiative.
Subject dosing has started, with the initial data readout anticipated for the second half of this year.
Moleculin Biotech CEO and chairman Walter Klemp said: “EMA approval of the MIRACLE trial protocol is a huge milestone for us. Although we’re already seeing recruitment in our first non-EU country, we believe that this expansion into the EU really supercharges our recruitment potential.”
Also referred to as naxtarubicin, Annamycin has the FDA’s orphan drug designation and fast track status to treat relapsed or refractory AML and soft tissue sarcoma, as well as an orphan drug designation from the EMA for AML.
In May 2024, the company announced the start of a National Institutes of Health (NIH)-funded Phase II NU 21C06 clinical trial of WP1066 with radiation therapy for adults with glioblastoma.
