Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (October 2025)
29 slides from Moleculin Biotech's investor presentation, Oct 30, 2025
Slide 1: Moleculin R/R AML AnnAraC Clinical Evaluation
64 MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation % October 25, 2025 % mek
Slide 2: Annamycin: A Next-Generation Anthracycline
Annamycin: A Next-Generation Anthracycline ° On ° Zeno “ou enon enon Son on if if mo 8 on 8 oom 6 : ee ” ti rE to Epirubicin = Idarubicin 8 Doxorubicin
Slide 3: Annamycin Has Demonstrated Substantially Greater Cardiac Safety
Current Anthracyclines Annamycin Has Demonstrated Substantially Greater Cardiac Safety Compared to Approved L Anthracyclines = Limit Risk of Heart Failure Pwocecuum
Slide 4: FDA Recommended Model Shows Annamycin's Lack of Cardiotoxicity
FDA Recommended Model Shows Annamycin’s Lack of Cardiotoxicity % of Mice with Heart Lesions The gold standard preclinical Bertozolli model for measuring cardiotoxicity shows Annamycin is effectively non-cardiotoxic when compared with doxorubicin. Free Annamycin (API only) is substantially less cardi
Slide 5: Comparison of Cardiotoxic Effects of Annamycin Vs. Doxorubicin
Comparison of Cardiotoxic Effects of Annamycin Vs. Doxorubicin on Human Cardiomyocytes Treatment Affects on Viability and Contractility of cardiomyocytes Vehicle Doxorubicin exposure leads to a dose dependent reduction of amplitude and an increase in frequency of contractions Bworecuum
Slide 6: Comparison of Cardiotoxic Effects of Annamycin Vs. Doxorubicin
Comparison of Cardiotoxic Effects of Annamycin Vs. Doxorubicin on Human Cardiomyocytes Affects on the Electrical Potential of Cardiomyocytes Electrical Activity Signals Were Significantly Reduced by exposure to Doxorubicin. Annamycin cells remain comparable to vehicle Pwocecuum
Slide 7: Solid Tumors Are An Important Opportunity
Solid Tumors Are An Important Opportunity Approximately 100 Sarcoma and AML Patients Treated With L-Annamycin Patients monitored with serial echocardiograms and troponin concentrations Patients treated with up to 5 times the maximal recommended dose for an anthracycline No change in mean ejection fr
Slide 8: L-Annamycin for the Treatment of AML
L-Annamycin for the Treatment of AML In vitro data In vivo Clinical data animal data
Slide 9: Annamycin Shown Effective Against both Cytarabine and Venetoclax Resistant Cell Lines
Annamycin Shown Effective Against both Cytarabine and Venetoclax Resistant Cell Lines MOLM-13 MOLM-13 Cytarabine resistant Venetoclax resistant wd eptivyrere om 100 ite 2 My ® 3 \ eam, sm ean, ara 50. ee aK, tem 0. Se VER, 21000 ui 3 cON aac, som 2 + MAC, Tea Ae a 1 40 100» 100010000 1 40 100» 1000-
Slide 10: Annamycin Synergizes with ARA-C in Increasing Survival
Annamycin Synergizes with ARA-C in Increasing Survival in p53-null, FLT3 Mutated AML Model (AML-mTurqoise2) 100] —7----~ 7 50: Percent survival . : 0 10 20°30 40 50 60 70 80 90 100 110 120 130 Time, days Biworecuum
Slide 11: MB-105: Phase 1b Dose Escalation, Monotherapy Trial in R/R AML Patients
MB-105: Phase 1b Dose Escalation, Monotherapy Trial in R/R AML Patients * R/RAML patients received L-Annamycin as monotherapy * Patients received L-Annamycin by infusion for 3 days * Dose escalation in cohorts of three until DLT or efficacy seen * Dose expansion after DLT or efficacy seen * No effic
Slide 12: MB-105 Phase 1b: 80% ORR in 240mg/m2 Cohort
MB-105 Phase 1b: 80% ORR in 240mg/m2 Cohort Patient Population Per Protocol Subjects in 240mg/m2 Cohort * 8 subjects enrolled * Scompleted per protocol »? Ll * 80% (4 of 5) patients “ responded to drug * 60% (3 of 5) CRi 75 PR CRi CRI CRi * 20% (1 of 5) PR
Slide 13: MB-106 Trial (EHA 2024, P537)
MB-106 Trial (EHA 2024, P537) L-Annamycin plus cytarabine in AML subjects Phase 1b/2 multi-center, open-label dose-escalation/dose expansion study Treatment: L-Annamycin (190 - 230 mg/m?, days 1-3); cytarabine (2,000 mg/m/?, days 1-5) 22 subjects were enrolled (ITT population) with 20 subjects of wh
Slide 14: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) All Lines (1° -7") (mos) Subjects Erato a 4 ‘ 10 H 14 i] Subjects Evaluable Not { Dosed per Protocol 2 7 ' q H ' Median ee 67.5 (19-78) 56.5 (19-69) y 71 (53 - 78) 1 69.5 (53-78) Carell Resin 8 (36%) 2 (60%) 1 5 (50%) H 6 (43%) 1 Ciba 9 (41%) 2 (60%) lH 6 (
Slide 15: MB-106: Durability, MRD, Prior Therapies - CR/CRi
MB-106: Durability, MRD, Prior Therapies - CR/CRi. Preliminary urability of Remission [f= Overall Survival = Continuing JJ = Prior Lines of Therapy @ = see Notes Below 1) M106 data are oreliminay and subject to chan untl a lina Study Report is publihed 2) MRD testing i nt standard betneen ses 8) rog
Slide 16: MB-106 Response by Genotype and Mutation
MB-106 Response by Genotype and Mutation or PR CR Oof1 Q31 CHRM Complex Karyotypes 89% (n=9) of GRc responses SS DE came from subjects with pian “generally poor prognosis cytogenetics and mutations” Annamycin appears to be “genotype/mutation agnostic” None/Not tested 8 6 4 Note ~ n=20; Some subjects
Slide 17: MB-106: AnnAraC 2L Efficacy in Venetoclax R/R Patients
MB-106: AnnAraC 2L Efficacy in Venetoclax R/R Patients Venetoclax regimen failure leaves patients with dismal options Ven-Dec Relapsed or Best Refractory _| Efficacy 78 Relapsed PD 64 Refractory sD 64 Relapsed cR 75 Refractory PR 75 Relapsed cR Ven-Aza/ Ven-Dec Cydes months) Unknown Annet Cydes AnnA
Slide 18: Recent Regulatory Activities
Recent Regulatory Activities * In light of successful MB-106 Phase 1b/2 clinical trial, Moleculin decided to proceed to a phase 2b/3 pivotal clinical trial * Regulatory guidance was sought from FDA and EMA regarding the design of a pivotal clinical trial * Face-to-face end-of-phase 2 meeting was hel
Slide 19: Refractory/Relapsed AML Therapies
Refractory/Relapsed AML Therapies Multiple therapies are utilized, but except for targeted therapies, none have been documented to be effective in clinical trials that meet regulatory body guidelines. To date, not even the advantage of high-intensity therapies over low- intensive therapies has been
Slide 20: IMPACT AML
IMPACT AML / Will randomize R/R patients to receive high-intensity versus low intensity chemotherapy so, as to establish if high-intensity therapy yields superior results. Investigators will be allowed to choose among multiple study approved high and low intensity regimens. Among the high intensity
Slide 21: AnnAraC Demonstrated Superior Complete Remission (CR) Rate
AnnAraC Demonstrated Superior Complete Remission (CR) Rate Compared to Current Standard of Care Complete Remission (CR) 0% HiDAC CR rates are well bis 36% documented 20% Hioact Hvac? Annirac3 Annarac3 Annarac3 Mirros Trl Classe Trial alkComers 2nd + 3rd Line 2nd Line Annamycin NDA to be based on CR
Slide 22: AnnAraC Should Increase 2nd Line AML CRs 2-Fold or More
AnnAraC Should Increase 2nd Line AML CRs 2- Fold or More 60.0% Eee 2 AIVIL Patients Expected to Achieve CR 40.0% i 1 30.0% I 20.0% _ | J ii 0.0% ! Gilteritinib Enasidenib —_Ivosidenib Olutasidenib Gemtuzumab! AnnArac (Xsopata) —(Idhifa} —(Tibsovo) _(Rezlidhia) (Mylotarg) !(Anna+AraC)31 (FLT3) (IDH2)
Slide 23: High Dose AraC (HiDAC)
High Dose AraC (HiDAC) Monotherapy Efficacy Expectations * Multiple large multicenter studies document CR rates of just under 20% with HiDAC * This is equivalent to the CR rates with targeted therapies in large multicenter studies * In light of these data, ELN continues to recommend HiDAC for R/R AM
Slide 24: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE ‘Moleculin R/R AML AnnAraC Clinical Evaluation Annamycin 2 Unblinded Data Readouts Annamycin Optimurr + HIDAC *Criteria: (1-30) Efficacy randomize 4:1 Control Arm Safety \ HIDAC + placebo Tolerability Control Arm HiDAC + a plenate Primary Endpoint = CR rate at 3—7 weeks
Slide 25: Inclusion Criteria
Inclusion Criteria Y Has a pathologically confirmed diagnosis of AML per the 2022 ELN criteria ¥ Has refractory/relapsed AML after having received only one prior line of induction therapy: maintenance therapy and/or BMT allowed ¥ Between 18 and 80 years of age (inclusive) at the time of signing the
Slide 26: Exclusion Criteria
Exclusion Criteria S Has prior or current diagnosis of acute promyelocytic leukemia (APL) or MDS Received prior mediastinal radiotherapy Has central nervous system involvement Has impaired cardiac function Known hypersensitivity to anthracyclines, cytarabine, or the excipients of L-Annamycin for Inj
Slide 27: Methodology
Methodology Bone marrow obtained at screening and at Day 35 + 14 Days. If patient doing well but platelet count is below 100,000 recommend waiting until it is > 100,000 or Day 49. If patient still has blasts in peripheral blood (obvious treatment failure), the bone marrow can be done as early as Day
Slide 28: Endpoints
Endpoints Primary efficacy endpoint is CR rate (no CRi or CRh) after first cycle Secondary efficacy endpoint is OS 1 PK and multiple safety endpoints, including changes in cardiac ejection fraction
Slide 29: Timeline
Timeline — April 2025 August 2025 Bee 03 2026 4 2027 02 2028 First patient First patient, Recruitment of Estimate Part B Estimate Part B Final topline enrolled second site 45" subject w/ of study begins completes data obtained encolles) unblinding late 3 quarter enrollment shortly thereafter - Pwoue
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