Moleculin Biotech, Inc.

“Shown in its clinical trials to be non-cardiotoxic (N=84) with some patients being safely dosed at five times the typical lifetime maximum allowed anthracycline dose, which potentially enables repeated dosing and consolidation;”

“During 2023, we started our MB-106 clinical trial with Annamycin in combination with Cytarabine (AnnAraC) for the treatment of AML in an all-comers trial, accepting subjects with a wide range of prior therapies in the Phase 1 portion with a limit of two prior therapies in the Phase 2 portion. The total recruited to date is 20 where one subject just began treatment and another subject has not been evaluated for efficacy. To date we have a composite complete response (CRc) rate of 39% in all subjects (n= 18). This is comprised of a CR rate of 33% and CRi of 6%. The 19th subject, being treated as a 3rd line therapy, had two BMAs tested where they have been inconclusive, and we are awaiting further testing. This subject will move the CRc to either 42% or 37% (n=19). Durability data are developing with one CR and one CRi having relapsed to date. Durability of CRs is confirmed by repeat BMAs. The first CR subject was treated in February 2023 and remains durable to date. The median age of all subjects recruited is 69, ranging from 19 to 78. This trial may enroll up to 28 subjects, although recruitment for subjects as 2nd line therapy ended in January 2024. The trial continues recruitment for treatment as 1st line and 3rd line therapy. Since we intend to position AnnAraC for approval as a 2nd line therapy, we believe that the most important data from this trial are the results in 2nd line subjects (excluding subjects who are either 1st line or 3rd line and beyond). When stratified for that population (n=10), the CRc rate is 60%, being comprised of a CR rate of 50% and a CRi of 10%.”

“The following advantages over early-generation anthracyclines which in their totality support Annamycin’s classification as a “next-generation” anthracycline: 1) demonstrated across five clinical trials to be non-cardiotoxic (n=90) with some patients being safely dosed at five times the typical lifetime maximum allowable anthracycline dose, (which potentially enables repeated dosing, consolidation and even use as a maintenance therapy); 2) exhibits no vesicant activity as well as greater tolerability, making it safer to handle and administer and easier on patients; 3) shown in preclinical models to circumvent multidrug resistance by avoiding MDR1 mechanisms and also to avoid cross-resistance with some of the most commonly used drugs for the treatment of AML (as well as many other cancers), including currently approved early-generation anthracyclines, cytarabine and venetoclax and, 4) exhibited in preclinical models substantially improved tissue/organ distribution;”

“Durability data are developing with one CR having relapsed to date with 4.5 months of durability. The first CR subject was treated in February 2023 and remains durable to date. Durability of CRc's is confirmed by repeat bone marrow aspirates (BMAs). The median age of all subjects recruited is 69, ranging from 19 to 78. This trial may enroll up to 28 subjects with recruitment for subjects as 2nd line therapy having ended in January 2024. The trial continues recruitment for treatment as 1st line and 3rd line therapy.”