Moleculin Biotech (MBRX) Corporate Connect Webinar Series summary

Company overview and leadership

• Leadership team has over 200 years of drug development experience, including 7 FDA approvals and 2 Big Pharma exits.

• Recently added an ex-Roche executive as a strategic advisor to support outlicensing and partnering efforts.

• Management has invested approximately $1 million of personal funds into company stock.

• Portfolio includes three technologies discovered at MD Anderson Cancer Center, all with Phase 2 human activity.

Focus on Annamycin and clinical progress

• Nearly all capital is now focused on Annamycin, a next-generation anthracycline designed to be non-cardiotoxic.

• Over 100 patients treated with Annamycin at high doses showed no evidence of cardiotoxicity.

• Annamycin avoids cross-resistance with existing anthracyclines and is more potent with fewer side effects.

• Holds composition and matter patent protection through 2040, with recent additional patents and orphan drug/fast-track status.

Clinical data and trial design

• Phase 2 trial of Annamycin plus Ara-C showed 50% complete remission in second-line AML patients, outperforming existing therapies.

• 75% of responders tested negative for measurable residual disease; 44% proceeded to curative bone marrow transplant.

• MIRACLE Phase 3 trial uses an adaptive design with two unblindings for early efficacy and safety assessment.

• First unblinding after 45 patients, with completion of Part A expected in 2026.