By: IPP Bureau
Last updated : August 04, 2026 2:48 pm
Blinded interim analysis shows 37% composite remission rate for drug that aims to treat relapsed or refractory acute myeloid leukaemia
Texas-based Moleculin Biotech, Inc., a Phase 3 clinical-stage pharmaceutical company, has reported encouraging preliminary blinded data from Part A of its ongoing pivotal Phase II/III MIRACLE trial evaluating Annamycin in combination with cytarabine (AnnAraC) for the treatment of adults with relapsed or refractory acute myeloid leukaemia (r/r AML).
The interim analysis included 62 evaluable patients and demonstrated a complete remission (CR) rate of 24% and a composite complete remission (CRc) rate of 37%.
Notably, 30 patients in the analysis had previously failed first-line venetoclax-based therapy, a difficult-to-treat population where published salvage remission rates are approximately 13% and median survival is estimated at 2.4 months.
The company has enrolled 74 of the planned 90 patients for Part A and expects treatment completion by September 2026, with comprehensive unblinded results anticipated between December 2026 and February 2027.
Under the MIRACLE study protocol, Part A compares two dosing regimens of Annamycin plus cytarabine against cytarabine plus placebo, with all participants receiving a single treatment cycle. The multinational trial is being conducted across sites in the United States, the European Union, and other European countries, enrolling AML patients who have received one prior induction therapy.
Walter Klemp, Chairman and CEO of Moleculin Biotech, said: “Because this analysis is still blinded and includes control-arm subjects, we would expect it to sit below the unblinded Annamycin-arm results we reported in June. That it has held in a narrow band while the population became measurably harder to treat is what gives us added confidence as we approach the completion of Part A.”
He added: “Just as importantly, based on reported ejection fractions and adverse events, we continue to observe no evidence of cardiotoxicity, one of the defining characteristics that differentiates Annamycin from conventional anthracyclines.”