Moleculin Biotech, Inc. announced 2023 year-end Annamycin clinical trials preliminary data and 2024 expectations for multiple data readouts and a transition to pivotal Phase 2B/3 clinical studies. The Company also updated its upcoming milestones. Preliminary 2023 Year-End Annamycin Clinical Trials Data: AML: The Company is currently conducting its Phase 1B/2 clinical trial evaluating Annamycin in combination with Cytarabine (also known as Ara-C and for which the combination of Annamycin and Ara-C is referred to as AnnAraC) for the treatment of subjects with AML as both first line therapy and for subjects who are refractory to or relapsed after induction therapy (MB-106).

The Company has also begun recruiting 1st line subjects into this trial. The Company continues to treat subjects in the Phase 2 portion of the study. Below is a summary of the data to date on this trial which is preliminary and subject to change. Among subjects who had an evaluable post treatment bone marrow biopsy, or who dropped out due to an adverse event (AE), there have been 6 complete responses (CR's) and 1 complete response with incomplete recovery of platelets and/or neutrophils or 40% CR and 47% CR/CRi's of the intent to treat (ITT) subjects (N=15) and 46% CR's and 54% CR/CRi's of the subjects treated (dosed with Annamycin) (N=13).

Two subjects experienced adverse events and were not dosed per protocol with one having an allergic reaction to Annamycin, the first the Company has seen in over 70 subjects dosed in the Company's multiple Annamycin clinical trials; the second adverse event was due to an allergic reaction to cytarabine. The CR/CRi's have been spread across 3 different sites in two different countries (Poland and Italy) and 7 out of 9 sites participating in the study have recruited subjects to date. The Company has expanded the study protocol to include a cohort of 1st line subject which should provide data to enable the designing of a potential confirmatory Phase 3 post-approval study, however it does not expect the addition of this cohort to delay its End of Phase 2 (EOP2) Meeting with the FDA, which will focus primarily on securing an accelerated approval pathway for the treatment of 2nd line patients (those who were relapsed from or refractory to a 1st line AML therapy).

The first 1st line therapy AML subject for Annamycin was recruited, treated, and upon evaluation it was determined that the treatment resulted in a CR. The first CR subject was treated in February of 2023. The Company has not been notified of any relapses to date, indicating significant durability of the CRs being generated in this study.

There continues to be no evidence of cardiotoxicity as reported by an independent expert's reports following assessments of ejection fractions, strain analyses, ECGs, and cardiac biomarkers including Troponin-I and T cross all trials (N=62). Most subjects have experienced myelosuppression, febrile neutropenia and/or thrombocytopenia, which are common adverse events with other anthracyclines. Currently, the median age of the 15 intent to treat subjects in MB-106 is 69 years (range of age is 32 to 78 years) with a median number of prior therapies for AML of 1 (range of 0 to 6).

The Company has recruited 19 subjects to date with 2 subjects withdrawing from the trial due to adverse events and 4 other subjects not yet having a bone marrow aspirate fully evaluable. The Company may recruit up to 28 subjects in the Phase 1B/2 clinical trial. At the Company's current rate of recruitment for the study, Moleculin expects to complete recruitment and report topline data in 1H 2024, if not by the end of the first quarter.

STS Lung Mets: On September 21, 2023, Moleculin announced the completion of enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases (MB-107) clinicaltrials.gov: NCT04887298. All subjects had pulmonary metastasis from soft tissue sarcoma (STS Lung Mets or STS) and at least one prior therapy. There was no limit on how many prior therapies a subject could have prior to entering this study.

Most subjects were heavily treated with other therapies prior to entering our trial with our treatment representing the 4th median therapy for all subjects in the Phase 1B and Phase 2 portion of the trial (range of 2 to 12). Below is a summary of the data to date on this trial which is preliminary and subject to change. The Company previously announced the preliminary data from the Phase 1B portion of the study showing median progression free survival (PFS) for 67% of the Phase 1B subjects of over 2 months (N=15) and median overall survival (OS) of 11.3 months.

Not including the one subject that withdrew from OS follow-up the median OS increased to 13 months. Additionally, the Company noted that Phase 1B subjects with less than or equal to 2 prior treatments and treated at the recommended Phase 2 dose or lower demonstrated a higher percentage of subjects with PFS of 2 months or greater at 78% (N=9). Of the 17 subjects in the Phase 2 portion of the study, all treatments have ceased, and subjects are being followed for PFS and OS.

A majority of subjects remain alive at this time. The Company believes that the data will be similar to or better than the Phase 1B portion of the study and will announce the results once the study has substantively concluded. 2024 Expected Upcoming Milestones: The Company announces the updated expected upcoming milestones for 2024.

Annamycin: AML: 1H 2024: In-depth data review and presentation of topline data on MB-106 clinical trial. 1H 2024: MB-106 End of Phase 2 (EOP2) Meeting with the FDA and/or its European equivalent. Mid 2024: Identify next phase of development/pivotal program.

Late 2024 into 1H 2025: Initiate pivotal program. STS Lung Mets: 2H 2024: Final MB-107 data readout. 2024: Identify next phase of development/pivotal program.

Late 2024 into 1H 2025: Initiate 1st line STS study with an investigator-initiated trial. Other Drug Candidates: Moleculin is in ongoing discussions with multiple academic institutions in separate programs evaluating WP1066 for the treatment of glioblastomas and/or pediatric brain tumors. The Company expects to finalize agreements with Northwestern University for an investigator-initiated and funded trial in early 2024.