Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (April 2025)
44 slides from Moleculin Biotech's investor presentation, Apr 21, 2025
Slide 1: Moleculin
0 dt o<}MOLECULIN O96
Slide 2: Disclaimer
Disclaimer \ All statements contained herein other than statements of historical fact, including statements regarding our futur results of operations and financial position, and our objectives for future operations, are forward-looking statements. The words “believe,” “may” “will” “estimate” “contin
Slide 3: Recent Developments
Recent Developments Y Preparing to launch MIRACLE Phase 3 Pivotal Trial for Annamycin in combination with cytarabine (AnnAraC) for the treatment of R/R/ AML Y 1site activated in Europe with multiple subjects in screening; Egypt & Georgia ethics approval received with regulatory expected Q2 2025 _Fir
Slide 4: Annamycin Performance in R/R AML
Annamycin Performance in R/R AML CR 50% CRe 60% OS _~11 months+ Durability ~11 months+ MRD- 78% BMT 44% OS ~10 months+ 1—Median; 21 subjects (n=10) 2~Median; 1-31 responders (n=9) It is widely recognized that the majority of 2L AML patients are underserved by available approved therapies and that be
Slide 5: Opportunity for Market Cap Breakout
Opportunity for Market Cap Breakout Annamycin: Disruptive Technology — Unique, patented, non-cardiotoxic, overcomes multidrug resistance; addresses huge unmet need; other potential cancer targets; potential for multiple cycles Annamycin: Clinically Advanced — Phase 2 efficacy significantly better th
Slide 6: Annamycin: A New Disruptive Tool for Second Line AML and Beyond
Annamycin: A New Disruptive Tool for ’ Second Line AML and Beyond
Slide 7: Annamycin Attributes
/ Novesicant activity (safer to handle and administer, no need for Annamycin Attributes Developed in collaboration with Composition ind licensed fre i and esed rom of Matter Avoids Cross Patents thru Resistance a ‘Significantly lower No with currently prescribed incidence (10%) of Cardiotoxic ricco
Slide 8: Approved Therapies Leave an Unmet Need in ~60% of AML Patients
Approved Therapies Leave an Unmet Need in ~60% of AMLPatients? Daunorubicin Venetoclax Targeted Therapy Regimen [i733 Ven-Aza _ Gene-Targeted FitPatients UnfitElderly §5%-65% Sub-population (60%) (50%) Eligible Durable CR% [NBC 37% ~21% AIAML benefit? 14% 18% 7% Criticar Cardiotox;de Stillleaves Lim
Slide 9: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) Subjects Evaluable t 1 1 ana Ons 2 @ 1 10 ! 14 Subjects Evaluable Not ' Dosed per Protocol 2 o ' 1 1 1 Median Age - Years Rewe) 67.5 (19-78) 56.5 (19-69) ‘ 71 (53 - 78) 4 69.5 (53-78) ‘Complete Remission 1 (CR) 8 (36%) 2 (60%) H 5(50%) | 6 (43%) Complete Re
Slide 10: MB-106: Durability, MRD, Prior Therapies
MB-106: Durability, MRD, Prior Therapies - CR/CRi 1 2 3 ~9 mos+ median durability / 7 ~14 mos median OS+ for CRe’s 5 e 70% MRO negative é e 50% of CRs bridged to BMT Pos [I= durability of Remission > = Continuing @ sudject relapsed - Continues with F/U @ subject died fom infection/PO pesca
Slide 11: MB-106: AnnAraC 2L Efficacy in Venetoclax R/R Patients
MB-106: AnnAraC 2L Efficacy in Venetoclax R/R Patients Venetoclax regimen failure | dismal options AnnAraC 60% CRc rate and 40% CR rate in Venetoclax R/R AML al Mnctactsry | SO 2 1 R 29 Recent study? of Ven-HMA failures 4 Relapsed cR 2 2 crit 32 inat '* Dismal outcomes upon failure - 74 pubiesractor
Slide 12: MB-106 Response by Genotype and Mutation
MB-106 Response by Genotype and Mutation Q31 CHRM Complex Karyotypes 89% (n=9) of CRc responses ee came from subjects with RUNXWITI “generally poor prognosis SEE ” cytogenetics and mutations’ EC: Annamycin appears to be cos “genotype/mutation agnostic” pers fr oH ‘TPS3 NoneiNot tested 8 6 4 2 Note —
Slide 13: AnnAraC Should Increase 2nd Line AML CRs 2-Fold or More
AnndAraC Should Increase 2nd Line AML CRs 2-Fold or More 60.0% ~13% of 2L AML Pa’ 500% Achieve CR 40.0% A 1 1 1 i 30.0% 1 20.0% { 10.0% I See 2.3% [ 17% 2.4% 13%! i 0.0% a mn. . a. ant : Gilteritinib Enasidenib —_Ivosidenib Olutasidenib Gemtuzumat!_ AnnAraC | (Xsopata) —(Idhifa}_—(Tibsovo)__(Rezlidh
Slide 14: AnnAraC Demonstrated Superior Complete Remission Rate Compared to Standard of Care
AnnAraC Demonstrated Superior Complete Remission (CR) Rate Compared to Current Standard of Care Complete Remission (CR) “on Ea ioe oes Annamycin NDA -_ to be based on CR rate in 2" line 7s subjects at ~1 10% month om Hoact ES Aanarac? Aenaac3 ——Annrac? Mires Teal Clase ral ——lComers «Onde SrdUne—2nd
Slide 15: AML Key Opinion Leaders' Support for Annamycin
AML Key Opinion Leaders’ Support for Annamycin “The preliminary clinical activity of Annamycin in heavily pretreated, relapsed/refractory AM patients who had progressive disease following Ara-C and VEN is very exciting and would provide a much-needed treatment option for other patients who otherwise
Slide 16: AML Key Opinion Leaders' Support for Annamycin
AML Key Opinion Leaders’ Support for Annamycin ‘Martin Tallman, MD, Internationally Renowned Cl “The latest preliminary AML data suggest that Annamycin could result in a promising new treatment| for AML. | am excited to work alongside the Moleculin team to continue advancing its development. and fur
Slide 17: End of P1B/2 Meeting with FDA Establishes Pathway to Approval
End of P1B/2 Meeting with FDA Establishes Pathway to Approval 3 Arm w/ CR= AnnAraC 2 Early Data Primary SoC Post R/R v. HiDAC? Readouts Endpoint Either Arm Compare Primary Endpoint is | Standard of Care Adaptive trial design ‘Annamycin per Project Optimus? | Complete response available to all + HIDA
Slide 18: MIRACLE Phase 3 Pivotal Clinical Trial
MIRACLE , Moleculin R/R AML AnnAraC Clinical Evaluation Phase 3 Pivotal Clinical Trial Designed to seek accelerated new drug approval for Annamycin in the treatment of relapsed or refractory acute myeloid leukemia. Targeted to begin recruiting in the first quarter of 2025
Slide 19: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation Annamycin 2 Unblinded Data Readouts Annamycin Optimum *Criteria: Efficacy 0 Control Arm Safety HIDAC + placet Tolerability Control Arm HIDAC + placebo Wee Doses and adaptive design recommended by FDAin end of Phase 18/2 meet
Slide 20: Potential Accelerated Timeline
Potential Accelerated Timeline Data Readout @n=45 20 Note NOArolingprocessengh depender pen leet undngof CMC and supporting NOA alts Part A meets high end of expectations, we may be able to shorten Part B to single arm safety/DE! only Drug approval process could start as early as 2026 Bworecuum
Slide 21: MIRACLE Sites Selected to Date
@ intrestedisted MIRACLE 25 sites selected to date @ Interested ioe NRA eee rl enon © Additional Targeted Updated: 3/20/25 Total ~70 sites targeted Targeted Sites Site Selection is Moving Quickly 1Dskes “10 sites Consistent feedback from face-to-face meetings with over 30 potential investigators: “A
Slide 22: The Full Annamycin Opportunity
The Full Annamycin Opportunity? Potential for Significant Upside —— 0K | Pancreatic Liner 13,500 Os rivaling 1* line in ox Bette median 7 line STS ne : ork subjects? Endometrial Lung 20,000 Initial Annamycin Yeas Coca Target Indications Leukemia tential Anaya iar TOTAL INCIDENCE K TOTAL INCIDENCE Sa
Slide 23: Corporate
Slide 24: Management Experience
= Oo. FS ee) detente, Wolfram C. M. Dempke, MD, PhO, MBA European Medical Advsor nanamecl? Seige Gxovnrs Grown 200 FDA Approvals Big Pharma Exits Moleculin Clinical Trials Million in Recent Management Investment Years of Combined Drug Development Experience
Slide 25: Financials – Runway into the Qtr. 2025
Financials _— Runway into the 3 Qtr. 2025 Nasdaq: MBRX {5 with $13.6M Pro Forma Cash Balance as of 12/31/2412 ~$11M Market Cap? x 14.0M Shares O/S* -f —~5.8M — Daily Trading Vol.> Mat has reduced overhead, pushed CMC projects into 2026, and engaged with preclinical and clinical, tions for iT program
Slide 26: Upcoming Milestones
Upcoming Milestones \\ Annamycin AM Tinpact of data readour Bogin enrollment of 3 ine (0-85) on regulatory subjects in MIRACLE? Begin contracting pathway; Recruitment I with MIRACLE Medical conference Update Enrolment ends for 2"! trial sites Dosters/presentations, fine subjects. | | \ aime — 10205
Slide 27: Recent Developments
Recent Developments Y Preparing to launch MIRACLE Phase 3 Pivotal Trial for Annamycin in combination with cytarabine (AnnAraC) for the treatment of R/R/ AML Y 1site activated in Europe with multiple subjects in screening; Egypt & Georgia ethics approval received with regulatory expected Q2 2025 Y- F
Slide 28: Appendix
— ~ VN Appendix pe Pr #
Slide 29: Annamycin: Outperforming Billion-Dollar Assets in AML; Phase 3 Data Readouts in 2025 & 2026
Slide 30: FDA provided clear written guidance on 190 mg/m² vs. 230 mg/m²
FDA provided clear written guidance by recommending the comparison of 190 mg/m? vs. 230 mg/m? Clinical experience to date shows no significant safety or efficacy difference between 190 and 230 Project Optimus Guidance Initial PK analysis shows no correlation between AUC OF Cinae and change in dosage
Slide 31: Next-Generation Anthracycline
Next-Generation Anthracycline — Redefining how anthracyclines are used Anthracyclines remain the bedrock treatment for lanthracycines Used ine many cancers — because they work Annamycin appears safer and more effective than currently prescribed anthracyclines Breast cancers = 32% AML patients = 50%
Slide 32: Annamycin: A Next-Generation Anthracycline
Annamycin: A Next-Generation Anthracycline Feonon Zenon Annamycin ncn mn em 8 b co enon - Pm hs, Epircbicin Idarbicin 2 Doxorubicin
Slide 33: Eliminating Cardiotoxicity
Eliminating Cardiotoxicity >3000 mine Zero Cardiotoxicity in ~100 Patients 100% Cardiac Event Treated FDA Lifetime Limit ee jece J 3 65% Cardiac Event. |< leart Failure |, Level Cardiac Events B50 moire = L | Currently Prescribed Anthracyclines Annamycin "i pemnsccnsn
Slide 34: FDA Recommended Model Shows Annamycin's Lack of Cardiotoxicity
FDA Recommended Model Shows Annamycin’s Lack of Cardiotoxicity 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 cardiotoxic than doxorubicin and L
Slide 35: Annamycin Synergizes with Ara-C in Increasing Survival
Bone marrow Annamycin Synergizes with Ara-C in Increasing Survival in Highly Aggressive p53-null, FLT3 mutated AML Model Analysis of Minimal Residual Disease (MRD) 100: ANN —LANN+ ARA-C Day 36 Day 36 S maaonail Tiana mona eS aarseke 50: Percent survival 0 10 20 30 40 50 60 70 80 90 100 110 120 130 T
Slide 36: AML Clinical History
AML Clinical History B-104, n=7 117%CRi at suboptimal dosing) Dosing limited by FDA tifetime Anthracycline Dose (UIMAD) Trial lecation— US Well-oleratedin the study population Limited to low doses Morphologic leukemia free state was achieved in one subject inthe 120me/m? cohort Demonstrated safe dos
Slide 37: AML Therapy Comparison and Valuation
N 153 286 199/174 CR 38% 37% 19%/25% CRe (48% 23%/33% AML 50% 50% 15-23% Population Revenue? $128M $28 ~$150M $1.58 $28 Valuation eit N/A Exit (equstion of egsin of er, 2036) gon),
Slide 38: Performance of AML Therapies in 2nd Line
Performance of AML Therapies in 2"! Line CLAVELA: International Randomized Phase I! Study of Elacytarabine Versus Investigator Choice in Patients with Relapsed/Refractory Acute Myeloid Leukemia 381 Elacytarabine Therapies compared: R/R AML (compared with) + high-dose + low-dose cytarabine subjects 7
Slide 39: Pipeline: Beyond Annamycin, IIT-Funded Focus
Slide 40: Technology Portfolio
Technology Portfolio Program Indication IND/CTA Phase 1 Phase 2 Phase 3 R/RAML Miracle: Adaptive Phase 3 (SIV‘s under way) Annamycin Next-Gen Anthracycline sts Lung Mets 2 Trials Active; Alt. Dosing; P Investigator wp1066 ‘Adult GBM NU IIT with Radiation Active; P GUUS Fanse STAT3 development Inhibi
Slide 41: Annamycin Demonstrates Efficacy in STS Lung Metastases
Annamycin Demonstrates Efficacy in STS Lung Metastases (MB-107) - As Reported in Feb 2024 Demonstrated Stable Disease After Demonstrated Improvement with Dose < 330 mg/m? Two Treatment Cycles and Fewer Prior Therapies All Subjects Preliminary MB-107 Summary as of Sep 23, 2024
Slide 42: Soft Tissue Sarcoma Mets to the Lungs
Soft Tissue Sarcoma Mets to the Lungs “We don’t expect to see these kinds of responses in STS patients with lung metastases who have stopped responding to 1* line therapy...let alone in 4" line!” STS KOL
Slide 43: Science Advisors
Science Advisors Waldemar Priebe, PhO Dr. Daniel Von Hoff MD Anderson Cancer Center Mayo Clinic Founding Scientist & SAB Chair Hematology Oncology ——————______——_ Dr. Martin Tallman Dr. Jorge Cortes Northwestern University ‘Augusta University Dr. Michael Andreet Dr. Giovanni Martinelli MD Anderson C
Slide 44: All technology licensed from MD Anderson Cancer Center (MDACC)
7 L / All technology licensed from MD Anderson Cancer Center (MDACC) Supports continuing preclinical research on our technology at MDACC ‘Active contractors in US, EU and Asia for drug production and distribution as well as for clinical trial management Past & current externally funded trials ~ MD A
Moleculin Biotech — Investor Presentation (April 2025) on 6ix