Annamycin:

Next Generation Non-Cardiotoxic and Organotropic Anthracycline

Annamycin is a next-generation anthracycline being evaluated in the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial, a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia (AML). If successful, Annamycin has the potential to transform the standard of care for 2^nd line AML. Annamycin is also in development for the treatment of soft tissue sarcoma (STS).

We believe anthracyclines are among the most important treatments for AML and advanced STS. However, cardiotoxicity issues limit their effectiveness with life-time maximum dosing set by the US FDA.

Annamycin has demonstrated in multiple clinical trials to have zero cardiotoxicity, allowing us to exceed the currently set maximum lifetime dosing for an anthracycline and potentially realizing the full therapeutic potential of anthracyclines to treat serious, hard-to-treat cancers.

In addition to showing zero cardiotoxicity in patients treated in Moleculin trials to date, Annamycin is demonstrating to have superior efficacy in 2^nd line AML compared to currently available treatments.

Key Highlights:

Active Clinical Studies:

AnnAraC Performance in R/R AML

It is widely recognized that the majority of 2L AML patients are underserved by available approved therapies and that better options are needed for this unmet need.

We believe the performance of AnnAraC is significantly better than any therapy ever approved for use in 2L AML.

Annamycin Attributes:

Notes: 1) Current Cardiology Review, Anthracycline Cardiotoxicity: Prevalence, Pathogenesis and Treatment,  Maria Volkova and Raymond Russel III. Referenced from Cancer. 2003 Jun 1;97(11):2869-79. “Congestive heart failure in patients treated with doxorubicin: a retrospective analysis of three trials”. Swain SM, Whaley FS, Ewer MS., PMID: 12767102; 2) Preliminary clinical studies from Moleculin; data subject to change; 3) Refer to  Form 10K for FYE 2023 for discussion on latest subject with an increase in troponins and our Expert’s opinion.

Annamycin Has Demonstrated Substantially Greater Cardiac Safety Compared to Approved Anthracyclines

Source Current Cardiology Review, Anthracycline Cardiotoxicity: Prevalence, Pathogenesis and Treatment,  Maria Volkova and Raymond Russel III. Referenced from Cancer. 2003 Jun 1;97(11):2869-79. “Congestive heart failure in patients treated with doxorubicin: a retrospective analysis of three trials”. Swain SM, Whaley FS, Ewer MS., PMID: 12767102; Preliminary clinical studies from Moleculin.

MB-106 (Annamycin + Ara-C (AnnAraC); n=22)

Notes: 1) Data from MB-106 are for intent to treat subjects who had efficacy determined (n=22); 2) Data from MB-106 are preliminary and subject to change; 3) Durability is developing; and 3) Relapses include 1 death due to pneumonia (unrelated to drug).

Durability, MRD, Prior Therapies

1) MB106 data are preliminary and subject to change until a Clinical Study Report is published. 2) MRD testing is not standard between sites

MB-106 Response by Genotype and Mutation

Note – n=20; Some subjects had multiple mutations or abnormalities, hence totals of treatment failures (TF), partial remissions (PR) or composite complete remissions (CRc) do not equal totals for each response category – TF’s/PR’s, or CRc’s; Data are anecdotal only and not intended to indicate statistical significance. Not all mutations/subjects were tested.