Moleculin Biotech (MBRX) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Key product and clinical advancements

• Annamycin is a next-generation anthracycline designed to avoid multidrug resistance and eliminate cardiotoxicity, representing the first major breakthrough in this drug class in 50 years.

• Clinical trials show Annamycin delivers a 60% CRC rate in second-line AML patients, with a 50% CR rate and 10% CRi, outperforming all approved drugs for relapsed/refractory AML.

• Over 80 patients treated with no cardiotoxicity observed, enabling regulatory discussions for higher dosing and accelerated approval pathways.

• Annamycin is genotype and mutation agnostic, with 89% of complete remissions in patients with poor-prognosis mutations.

• 78% of responders achieved MRD-negative status, indicating strong long-term outcomes.

Market opportunity and competitive landscape

• Annamycin addresses an unmet need for over half of AML patients, especially those ineligible for targeted therapies.

• Targeted therapies benefit only about 13% of second-line AML patients, while Annamycin's trial data suggest more than double that rate.

• Recent industry deals valued less effective drugs at $2 billion, highlighting significant market potential.

• Market caps and exits in this space have been 10 to 1,000 times higher than current valuation.

Regulatory and clinical trial strategy

• FDA has agreed to allow U.S. AML patients to be treated above the current anthracycline dose limit due to Annamycin's lack of cardiotoxicity.

• The pivotal phase 3 MIRACLE Trial will use an adaptive design, randomizing patients to HiDAC plus placebo or two Annamycin doses, with dose optimization after interim analysis.

• Primary endpoint is complete remission at one month, with secondary endpoints including durability of remission.

• MIRACLE 2 trial will follow for third-line patients, further supporting regulatory approval.