Moleculin Biotech Inc (NASDAQ:MBRX) saw its shares rise 3.1% in premarket trading Wednesday after the company disclosed encouraging preliminary, blinded results from the ongoing pivotal Phase 2B/3 MIRACLE trial of its AML therapy candidate.
According to the company, the Annamycin regimen administered with cytarabine has produced a composite blinded complete remission (CR) rate of 40% among the first 30 subjects. That composite rate comprises a 30% complete remission rate and an additional 10% classified as complete remission with partial hematological recovery. Moleculin said this preliminary composite CR rate is 67% higher than historical response rates observed with cytarabine alone.
Moleculin reported that it has treated 35 subjects to date, with another 11 individuals identified or currently in screening. The company reiterated its expectation to reach the trial milestone of treating 45 subjects in Q1 2026, at which point it plans to proceed with unblinding the data shortly thereafter.
Management highlighted the make-up of the treated population, noting that roughly 35% of subjects so far had previously failed venetoclax regimens - a subgroup that the company described as particularly challenging to treat with second-line options. Moleculin also emphasized the continued absence of observed cardiotoxicity with Annamycin, presenting it as a differentiator relative to conventional anthracycline chemotherapy.
The company reiterated a comment from its chairman and chief executive on the early data: "The blinded efficacy rates we’re seeing in MIRACLE are exceptionally encouraging," said Walter Klemp. "Even with the control arm included, these preliminary, blinded results substantially outperform historical outcomes for CR with cytarabine alone."
Trial design details confirm MIRACLE is a randomized, double-blind, placebo-controlled study. It compares two dosing levels of Annamycin - 190 mg/m2 and 230 mg/m2 - each given with cytarabine, against cytarabine plus placebo in patients who have undergone a single prior induction therapy.
The company said it anticipates completing the unblinding of the first 45-subject cohort in late Q2 2026. Recruitment for the subsequent 45-subject cohort is expected to continue without interruption.
Context for investors and markets
- The initial blinded efficacy signal has coincided with a modest premarket uptick in the company’s stock price.
- Enrollment remains ongoing and key readouts are tied to upcoming unblinding milestones in Q1 2026 and late Q2 2026.