Are Investors Non-Believers in Moleculin (MBRX) MIRACLE Phase 3 Trail Plans? Stock Down Over 20% on Report

Moleculin Biotech, Inc. (MBRX), a clinical-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced a positive discussion and outcome in its End of Phase 1B/2 (EOP1B/2) meeting with the US Food and Drug Administration (FDA). This outcome supports the advancement of Annamycin in combination with Cytarabine to a Phase 3 pivotal trial for treating AML patients who are refractory to or have relapsed after induction therapy (R/R AML). This Phase 3 “MIRACLE” trial (Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US.

“We thank the FDA’s Divisions of Hematologic Malignancies I and Cardiology and Nephrology, as well as related divisions, for a very constructive EOP1B/2 meeting and their valuable feedback. Armed with this, we are now able to finalize plans for a pivotal approval pathway in AML,” commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. “Importantly, consistent with the FDA’s recommendations, the adaptive Phase 3 trial will rely solely on CR (complete remission) at day 30 as the primary endpoint versus placebo, a standard we are confident Annamycin will meet and that provides an opportunity for accelerated approval.”

Moleculin Planned Significant Milestones

The Company has established plans for the following milestones:

• 2H 2024 – Begin contracting with MIRACLE trial sites
• Q1 2025 – First subject treated in MIRACLE trial
• Mid 2026 – Interim data (n=75) unblinded and Optimum Dose set for MIRACLE trial
• 2026 – Begin enrollment of 3rd line subjects in MIRACLE2
• 2027 – Enrollment ends in 2nd line subjects
• 2028 – Final Data for 2nd line subjects in MIRACLE
• 2H 2028 – Begin submission of a new drug application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE

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