Moleculin Biotech (MBRX) Status Update summary

Key clinical insights and unmet needs

• Significant unmet need remains in relapsed/refractory AML, with survival often less than three months and limited approved therapies available, especially in Europe.

• Current targeted therapies address only a small subset of AML patients, leaving many without effective options after first-line treatment failure.

• Annamycin, a novel anthracycline, shows promise due to its lack of cardiotoxicity and potential to overcome cross-resistance seen with other AML drugs.

Efficacy and safety data

• Annamycin demonstrated a 50% complete remission (CR) rate in second-line AML patients, significantly higher than the 21% average for approved second-line therapies.

• Responses were observed in both fit and unfit patients, including those previously treated with intensive chemotherapy or venetoclax.

• No evidence of cardiotoxicity was observed in 84 patients, even at doses exceeding traditional lifetime anthracycline limits.

• The drug also shows activity in preclinical models against AML cells resistant to other therapies.

Phase III Miracle trial design and expectations

• The pivotal Miracle trial uses an adaptive design, comparing two Annamycin dosing regimens plus HiDAC to HiDAC plus placebo, with early unblinding after 90 patients to select the optimal dose.

• Historical HiDAC CR rates are well established at 17-18%, providing a reliable control for comparison.

• The trial is open to both fit and unfit patients, aiming to accelerate recruitment and broaden applicability.

• First patient is expected in Q1 2025, with midpoint blinded data anticipated by late 2025 and unblinded data in mid-2026.