Investing.com — stock rose 3.1% in premarket trading Wednesday after the company reported encouraging preliminary data from its ongoing pivotal Phase 2B/3 “MIRACLE” trial for its AML treatment candidate.
The clinical-stage pharmaceutical company announced that its Annamycin drug, in combination with cytarabine, has shown a preliminary blinded complete remission (CR) rate that is 67% higher than historical cytarabine response rates in treating acute myeloid leukemia (AML). The blinded data from the first 30 subjects revealed a composite complete remission rate of 40%, consisting of a 30% complete remission rate and 10% complete remission with partial hematological recovery.
Moleculin has treated 35 subjects to date with another 11 identified or in screening. The company expects to reach its first milestone of treating 45 subjects in Q1 2026, with data unblinding to follow shortly thereafter.
“The blinded efficacy rates we’re seeing in MIRACLE are exceptionally encouraging,” said Walter Klemp, Chairman and CEO of Moleculin. “Even with the control arm included, these preliminary, blinded results substantially outperform historical outcomes for CR with cytarabine alone.”
Notably, approximately 35% of the treated subjects represent patients who failed venetoclax regimens, a population considered particularly difficult to treat with second-line therapies. The company also highlighted the continued absence of cardiotoxicity in Annamycin, a significant advantage over traditional anthracyclines used in cancer treatment.
The MIRACLE trial is a randomized, double-blind, placebo-controlled study evaluating Annamycin at two dosing levels (190 mg/m² and 230 mg/m²) in combination with cytarabine, compared to cytarabine plus placebo in patients who have had a single prior induction therapy.
The company expects to complete the unblinding of the first 45 subjects in late Q2 2026, with recruitment for the second group of 45 subjects continuing uninterrupted.
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