Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (January 2025)
44 slides from Moleculin Biotech's investor presentation, Jan 8, 2025
Slide 1: Slide 1
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, fur business strategy and plans, and our objectives for future operations, are forward-looking statements. The words “believe,
Slide 3: Recent Developments
Recent Developments Y 2025 Year Outlook ~ engaged a CRO for MIRACLE; focus on Annamycin in pipeline; focus of MIRACLE trial in Europe, Asia & MENA; 45 subject readout 2H 2025; 75-90 subject readout 1H 2026; and reduced cash needs in 2025 v Announced new findings showing Annamycin overcomes resistanc
Slide 4: Annamycin Performance in R/R AML
Annamycin 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 i
Slide 5: Opportunity for Market Cap Breakout
Opportunity for Market Cap Breakout S MOLECULIN
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
Annamycin Attributes / Novesicant activity \ (safer tohandle and administer, no need for a central venous catheter) Developedin \\ / collaboration with / andlicensed from ‘MD Anderson / Composition of Matter Avoids Cross Resistance von No Manton eee Meet aaed Cardiotoxic ste ec 100%) preclinical mod
Slide 8: Eliminating Cardiotoxicity
Eliminating Cardiotoxicity Zero Cardiotoxicity in ~100 Patients Treated 100% Cardiac Event Level >8% Heart Failure FDA Lifetime Limit 65% Cardiac Event Level Cardiac B50 none Events 550 rom | | Currently Prescribed Anthracyclines Annamycin Bworecuum
Slide 9: Approved Therapies leave ~60% Unmet Need
Approved Therapies leave ~60% Unmet Need of AML Patients? Venetoclax | Targeted Therapy Regimen 143 Ven-Aza _Gene-Targeted Sub-population repaliente Unit Edel ag Durable CR% 36%? 37% ~21% AILAML benefit? 14% 15% 7% Cardiotox;de Stillleaves Limited to novo/acquired significant _certain gene resistanc
Slide 10: AnnAraC Should Increase 2nd Line AML CRs 2-Fold or More
AnnAraC Should Increase 2nd Line AML CRs 2-Fold or More’ 60.0% : 1 s0.0% ~13% of 2L AML Pa s Expected to : 50.0% 4 1 Achieve CR ' I 40.0% 1 C , ian 1 30.0% 1 i 1 20.0% 1 | 1 10.0% 5p 2.3% 1.7% 2.4% 13% 1 ' ; 1 0.0% | - Bo - =; 1 GilteritinibEnasidenib_Ivosidenib Olutasidenib Gemtuzumab| AnnArac | (X
Slide 11: Opportunity for Market Cap Breakout
Opportunity for Market Cap Breakout q — Approved Phase 2 Complete Venza | hit N 199/174 CR 38% 19%/25% Re 48% 23%/33% AML 50% 50% 15-23% Population Revenue? $128M $2B ~$150M $1.58 $28 Valuation Exit! N/A Exits estat tetenn terns) poet
Slide 12: The Bar for Approval is Low
The Bar for Approval is Low Complete Remission (CR) som joe gaia wel 7 Annamycin NDA to be based on CR rate in 2"¢ line subjects at ~1 month Hoact wioac? onarac? Aonanc3 ——Annrac? Mires ral Clase ral ——lComars nde 2rd Une Ondine
Slide 13: Next-Generation Anthracycline
Next-Generation Anthracycline — Redefining how anthracyclines are used Anthracyclines remain the bedrock treatment for many cancers — because they work Annamycin appears safer and more effective than currently prescribed anthracyclines Lack of cardiotoxicity enables increase in dosage and number of
Slide 14: The Full Annamycin Opportunity
The Full Annamycin Opportunity? Potential for Significant Upside _— alk — 6x Pancreatic Liner 13,500 PFS and OS rivaling on STSturg Metasases PEC oa foc ure 67K STS subjects? Endometrial Lung 20,000 Initial Annamycin PR rene conc ‘Target Indications Leukemia Potential Annarycin ime TOTAL INCIDENCE ~
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Slide 16: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) ine of Therapy All Lines (1*-7") Subjects Evaluable To ! 1 ee 22 4 ! 10 I 14 Subjects Evaluable Not ' re 2 a Median Age - Years (Range) 67.5 (19-78) 56.5 (19-69) Hl 71 (53 - 78) 4 69.5 (53-78) Complete Remission 1 reel 8 (36%) 2 (60%) 1 5(50%) | 6 (43%) Com
Slide 17: Durability, MRD, Prior Therapies
Durability, MRD, Prior Therapies ~8 months median durability and climbing 78% MRD negative ty ccs a > our [B= Durability of Remission = Continuing Prior Lines of Therapy © svrject relapse, likely due to cessation of treatment due to infection, Continues with F/U @ ssurject died from infection/PO © P
Slide 18: AnnAraC 2L Efficacy in Venetoclax R/R Patients
AnnAraC 2L Efficacy in Venetoclax R/R Patients Venetoclax regimen failure leaves patients with dismal options Ver-Ava/ | Ven: AnnAraC 60% CRc rate and 40% CR rate in Venetoclax R/R AML Recent study? of Ven-HMA failures 4 Relapsed cR 2 ing bauer: '* Dismal outcomes upon failure - 2A Reseciory gy MRCS
Slide 19: MB-106 Response by Genotype and Mutation
MB-106 Response by Genotype and Mutation Q31 CHRM Complex Karyotypes 89% (n=9) of CRc responses oe ee came from subjects with a “generally poor prognosis Sa cytogenetics and mutations” ERs! Annamycin appears to be coe3, “genotype/mutation agnostic” res He oH TP53 NoneiNot tested Note~n=20; Some subj
Slide 20: AML Clinical History
AML Clinical History Phase 1: MB-104 MONOTHERAPY Phase 1/2: MB-105 MONC Phase 1/2: MB-106 COMBINATION THERAPY 117% CRi at suboptimal dosing) DDosing limited by FDA Lifetime Anthracycline Dose (UIMAD) Tal location — US ‘Well-tolerated in the study population Limited to low doses Morphologic leukerna
Slide 21: AML Key Opinion Leaders' Support for Annamycin
AML Key Opinion Leaders’ Support for Annamycin
Slide 22: AML Key Opinion Leaders' Support for Annamycin
AML Key Opinion Leaders’ Support for Annamycin “The latest preliminary AML data su treatment for AML. | am excited to development and further explore ‘Martin Tallman, MD, Internationally Renowned Clinical Investigator whose Discoveries have Fueled the Progress of Leukemia-targeting Therapies ‘Mohama
Slide 23: 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 Adaptive trial design | Primary Endpoint is | Standard of Care Annamycin per Project Optimus? | Complete response } available to all + H
Slide 24: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation Annamycin 2 unblinded Data Readouts Annamycin Optimum *Criteria Efficacy ni Control Arm Safety HIDAC + placebc Tolerability Control Arm HIDAC + placebo Fi Doses and adaptive design recommended by FDAin end of Phase 18/2 meet
Slide 25: Potential Accelerated Timeline
Potential Accelerated Timeline 2025 2026 Part B (per prot: Data Read Out @n=45 25 Nete NOArolingpreceseng dependere gen lel of unengcFCMCandsugearing NDA aes Part A meets high end of , we may be able to Drug approval process could start as early as 2026
Slide 26: MIRACLE Site Selection
o intrestedhsted MIRACLE Me resected ean BRAM, Antec aan © Additional Targeted Updated: 12/16/24 ~70 sites interested More sites targeted Site Selection is Ukraine Georgia Moving Quickly Bates ry Consistent feedback from face-to-face meetings with fi2 sites over 30 potential investigators: “Annamyci
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Slide 28: Technology Portfolio
Technology Portfolio Program Indication Phase 1 Phase 2 Phase 3 R/RAML Miracle: Adaptive Phase 3 (SIV’s under way) Annamyci Next-Gen Anthracycline 2 Trials Active; Alt. Dosing; P1B/ STS Lung Mets Investigator winanee ‘Adult GBM NU IIT with Radiation Active; P2 Naas STAT3 development Inhibitor Pediat
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Slide 30: Our Team
Our Team Founder President, CEO and harman come 2600 BR Rl om lective VP & Chi Financial Offcar om Oc FS ee) tone, Wolfram C. M. Dempke, MD, PhO, MBA European Medical Advsor nanamecl> “Ease GxoMKTs Grow 200 FDA Approvals Big Pharma Exits Moleculin Clinical Trials Million in Recent Management Investm
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Slide 32: Financials
Financials cts Nasdaq: MBRX (3) ~$9.4M Cash Balance? ~$8.2M Market Cap? x 3.2M Shares O/S and 4.8M Shares Fully Diluted Outstanding* —f “67K — Daily Trading Vol.° educed overhead, pushed CMC projects into 2026, and engaged with institutions for IT programs for preclinical and clinical activity for n
Slide 33: Upcoming Milestones
Upcoming Milestones /\\ \__/ Annamycin Tnpat of data readout Begin eniment of line (45) on regulatory subjects in MIRACLED Begin contracting with pathway, Reenatment \ MIRACLE ta sites ted tere Unde Enrollment ends for °° | i | line subjects 1 ange —— 192005 aaa. ae sa205 oe bi me name = Update onsi
Slide 34: MIRACLE
MiRACHS 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 35: Appendix
—" ——— Appendix pels
Slide 36: FDA provided clear written guidance by recommending the comparison of 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 Crnae and change in dosage
Slide 37: Annamycin: A Next-Generation Anthracycline
Annamycin: A Next-Generation Anthracycline Zeon Zenon Annamycin Ane Sor | <n mn om 9 ) Aino * i" on ha Epircbicin Idarbicin Doxorubicin
Slide 38: 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
Slide 39: Annamycin Synergizes with Ara-C in Increasing Survival
Bone 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 a6 Day 36 Day 36 ase os 2001 z * comet once <oonet E © Vehicle so Semanal “= ARA-C 50 mgikg Percent survival 0 10 20 30
Slide 40: Performance of AML Therapies in 2nd Line
Performance of AML Therapies in 2"! Line CLAVELA: International Randomized Phase III 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
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 Brouecuum
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 MD Anderson Cancer Center Dr. Daniel Von Hoff 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
Slide 44: All technology licensed from MD Anderson Cancer Center (MDACC)
O- 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 ‘Ande
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