Moleculin Biotech (MBRX) Corporate presentation summary

Clinical development and efficacy

• Annamycin, a novel anthracycline, is in pivotal Phase 3 trials for relapsed/refractory AML, showing a 39% complete remission rate and 54% composite remission rate in interim pooled analysis, outperforming control arms significantly.

• No cardiotoxicity detected in over 100 patients, even at doses exceeding traditional anthracycline limits, with independent review confirming cardiac safety.

• The MIRACLE trial uses an adaptive design with FDA guidance, targeting full Part A enrollment by Q3 2026 and a pivotal readout by early 2027.

• Annamycin demonstrates efficacy in venetoclax-pretreated and high-risk AML populations, with strong support from key opinion leaders.

• Additional efficacy observed in soft tissue sarcoma lung metastases and planned expansion into pancreatic cancer.

Market opportunity and positioning

• Peak revenue potential for AML alone is estimated at $907M, with total pipeline indications potentially reaching $9.85B.

• Market research indicates high oncologist willingness to prescribe Annamycin, especially for post-venetoclax and biomarker-negative patients.

• Payers support a premium price of ~$33K per infusion, justified by efficacy, safety, and broad eligibility.

• Annamycin's non-cardiotoxic profile expands eligibility to older and comorbid patients, addressing a major unmet need in AML.

• The product is positioned as a mutation-agnostic, all-comer salvage therapy and a potential first-line backbone in combination with targeted agents.

Technology and pipeline

• Annamycin is a patented new chemical entity with orphan drug and fast track status, designed to avoid multidrug resistance and cardiotoxicity.

• The pipeline includes additional oncology and antiviral candidates (WP1066, WP1122) advancing through investigator-initiated studies.

• All technology is licensed from MD Anderson Cancer Center, with ongoing collaborations and global clinical trial sites.

• Management team has over 200 years of drug development experience and significant personal investment.