Moleculin Biotech (MBRX) The 38th Annual Roth Conference summary

MIRACLE phase III trial progress and design

• MIRACLE phase III trial for relapsed/refractory AML is registration-enabling, with two parts and three arms in part A, including two Annamycin doses and a HiDAC control.

• Recruitment accelerated after initial delays, reaching 45 patients, with interim unblinded data expected in a few months.

• Trial design allows early unblinding for dose optimization, aligning with FDA Project Optimus and enabling efficient data collection.

• Fast track status allows rolling NDA submission; surrogate endpoint is complete remission (CR), measurable within a month per patient.

• Interim data will include CR rates for each arm, median age, and percentage of venetoclax regimen failures, a challenging patient group.

Annamycin differentiation and clinical potential

• Annamycin is a new chemical entity with patent protection through at least 2040, featuring a unique lipid-based delivery system.

• Demonstrates zero cardiotoxicity in over 100 patients, even above FDA lifetime limits, and avoids typical anthracycline side effects like alopecia and mucositis.

• Not recognized by multidrug resistance mechanisms, avoiding cross-resistance with current anthracyclines, venetoclax, and cytarabine.

• Phase II data showed higher efficacy than any approved drug for relapsed/refractory AML, with mutation-agnostic results.

• Organotropism allows Annamycin to hyperaccumulate in key organs, including lungs, liver, pancreas, and spleen, expanding its therapeutic reach.

Market landscape, demand, and future milestones

• Current gene-targeted therapies for AML benefit only about 18% of relapsed/refractory patients, leaving a large unmet need.

• Annamycin is positioned as a potential $500 million to $1 billion global market opportunity in AML alone.

• High inbound demand for investigator-sponsored trials across sarcomas, colorectal, renal cell carcinoma, and pancreatic cancer, but drug supply is prioritized for the phase III trial.

• Anticipated milestones include unblinding of 45-patient and 90-patient data, potential breakthrough therapy status, and further market cap growth.

• Diversity of trial sites across nine countries reduces bias and increases approval prospects.