Moleculin Posts Encouraging Phase 2/3 MIRACLE Trial Results; Shares Down Despite Data
(RTTNews) - Moleculin Biotech, Inc. (MBRX) announced positive interim efficacy results from Part A of its pivotal Phase 2/3 MIRACLE trial evaluating Annamycin in patients with relapsed or refractory acute myeloid leukemia. The analysis, based on the first 45 patients enrolled, showed that both Annamycin dose arms significantly outperformed the control arm on the primary endpoint of complete remission.
Complete remission (CR) rates reached 43% and 35% in the respective Annamycin cohorts compared with 12% for the control arm, while composite complete remission (CRc) rates were 50% and 57% versus 29% for control.
The trial population included a high proportion of older patients, with 75.6% over 60 years of age, and prior exposure to intensive regimens such as 7+3 and (55.6%) and venetoclax-based therapies (31.1%). Moleculin noted that remission rates in this interim analysis were expected to be lower than historical multi-cycle datasets but emphasized that the most meaningful comparison is the performance of Annamycin relative to the concurrent randomized control arm.
The Independent Data Monitoring Committee (iDMC) reviewed the interim data and recommended that the trial continue without modification, supporting the safety and efficacy profile observed to date.
Walter Klemp, Chairman and CEO of Moleculin, described the results as "a defining moment" for the company, noting that both dose arms meaningfully outperformed control across primary and composite remission endpoints. He added that the findings provide compelling evidence of Annamycin's differentiated profile and strengthen confidence as the MIRACLE trial advances toward completion.
Enrollment in Part A of the MIRACLE trial is ongoing, with 67 of 90 subjects already enrolled, as the company moves toward optimal dose selection.
MBRX has traded between $1.79 and $24.00 over the past year. The stock is currently trading at $2.56, down 28.25%.
