Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (September 2025)
41 slides from Moleculin Biotech's investor presentation, Sep 8, 2025
Slide 1: Moleculin Biotech
-<S/MOLECULIN We is
Slide 2: Disclaimer
Disclaimer \ All statements contained herein other than statements of historical fact, including statements regarding our future results of operations and financial position, ‘our business strategy and plans, and our objectives for future operations, are forward-looking statements. The words “believ
Slide 3: Recent Developments
Recent Developments ¥ Enrollment and dosing underway in Phase 3 clinical trial, MIRACLE, evaluating Annamycin (naxtarubicin) in combination with, cytarabine (AnnAraC) for the treatment of R/R AML Y — Multination trial with active sites in the United States, Europe, Ukraine and Georgia First patient
Slide 4: Technology Portfolio
Technology Portfolio Program Indication IND/CTA Phase 3 2H 2025 R/RAML Data readout nat) Annamycin efficacy/safery reve Next-Gen STS Lung Mets Anthracycline Pediatric R/R AML we1066! Adult GBM STAT3 Inhibitor Pediatric Brain Tumors wp1122 Anti- GBN; Virology Metabolite
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 bette
Slide 6: Our Team
Our Team Water\xeme Founder resin, CEO ond Chan zo BRS Denald Pick PhO Chef Scientific Offexr a Executive VP & Chet Financial Officer cm Oe FE See) petonte. De John Paul Waymack Senior Chef Magical OFicar Wotram ¢.M. Dar European Medal ranconei sana Tee Strep Adisor . & FDA Approvals Big Pharma Exit
Slide 7: Annamycin: A New Disruptive Second Line AML
Annamycin: A New Disruptive 7 Second Line AML
Slide 8: Annamycin Delivered Superior Efficacy in R/R AML
Annamycin Delivered Superior Efficacy in R/ “AML The primary endpoint for the MIRACLE CR! 50% trial is CR. Efficacy in our MB-106 single CRc: 60% arm trial with Annamycin plus Cytarabine for for the treatment of AML is shown at OS for CR? 15 months+ left. Itis widely recognized that the Fi majority
Slide 9: Annamycin is the First Meaningful Advance in Anthracycline Therapy in Decades
Annamycin is the First Meaningful Advance in Anthracycline Therapy in Decades Non- Cardiotoxic Composition of Matter Patents thru 2040 in US & EU Zero cardiotoxicity per independent expert (84 subjects reviewed to date) Patie eated up to 5x FDA life * More potent than Dox in most max for Dox ‘tumor
Slide 10: Approved Therapies Leave an Unmet Need in ~60% of AML Patients
Approved Therapies Leave an Unmet Need in ~60% of AML Patients? Sub-population Durable CR% All AML benefit? characteristics Daunorubicin | Venetoclax Targeted (+ Azacitidine) Therapy 73 Ven-Aza Gene-Targeted Fit Patients Unfit Elderly 55%-65% (50%) (50%) Eligible 36%? 37% ~21% 14% 15% 7% Cardiotox;d
Slide 11: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) Gatun z 4 ! 10 1 rm 1 Subjects valuable Not i asad po reacel 2 a I 8 H : Mada Ae ven (pee 6755 (19-78) 56.5 (19-69) yj 71 (53 - 78) 4 69.5 (53.78) 1 Complete Remission (CR) 8 (36%) 2 (50%) 1 5 (50%) 6 (43%) 1 Complete Remission Composite (eRe) OFS) EE) 1 6
Slide 12: Annamycin Provided CRs in 2L AML 2-Fold or Higher than Targeted Therapies
Annamycin Provided CRs in 2L AML CRs 2-Fold or Higher than Targeted Therapies? 60% ~13% of 2L AML Patients Expected to Achieve CR w/ EERE therapies Gilteritinib (%sopata) (rua) Enasideniy (idhifa) (1DH2)2 Ivosidenib Olutasidenib Gemtuzumab —Ziftomenib (Tibsovo) (Rezlidhia) _— (Mylotarg) ——(NPMa/ (1H
Slide 13: AML Key Opinion Leaders' Support for Annamycin
AML Key Opinion Leaders’ Support for Annamycin “The preliminary clinical activity of Annamycin in heavily pretreated, rolapsed/refractory AML patiants who had progressive disease following Ara-C and VEN & very exctng and would provide a much-needed treatment option fr ether pationts who otherwise ha
Slide 14: FDA Established Pathway to Approval
li | Qf 1e i = FDA Established Pathway to Approval Moleculin R/R AML AnnAraC Clinical Evaluation 3 Arm w/ CR= AnnAraC 2 Early Data Primary SoC Post R/R v. HiDACt Readouts Endpoint Either Arm Compare Adaptive trial design | Primary Endpoint is | Standard of Care ‘Annamycin per Project Optimus? | Comp
Slide 15: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation Annamycin 2 Unblinded Data Readouts HiDAC ene Optimum Establish thetic Optimum Dose* Annamycin ‘Adult R/R AML 230 mg/m? + paies HIDAC “Criteria 0) Efficacy randomize 1:1 Control Arm Safety HiDAC Tolerability Control Arm HIDA
Slide 16: Potential Accelerated Timeline
Potential Accelerated Timeline 2025 2026 2027 Rolling NDA fety/DEI only Drug approval process could start as early as 2026
Slide 17: MIRACLE Sites Open
@ Sites Open (sept 2025) MIRACLE ParttA&B © Completed Regulatory Molec RR AML AnnAvaC Gincal Evaluation ns A @ Additional Targeted Total ~45 sites targeted Updated: 8/5/35 ar ee Site Selection is sortie Moving Quickly ee “10sites Consistent feedback from face-to-face meetings with over 30+ potential
Slide 18: MB-107 STS Lung Mets
MB-107 STS Lung Mets w/ Annamycin Summary* *Per final clinical study report
Slide 19: MB-107 Overview
MB-107 Overview LIPOSOMAL ANNAMYCIN (L-ANNAMYCIN}?, MB-107 — PHASE 18/2 STUDY OF LIPOSOMAL ANNAMYCIN (L- ANNAMYCIN) IN SUBJECTS WITH PREVIOUSLY TREATED SOFT TISSUE SARCOMAS WITH PULMONARY METASTASES A multi-center, open-label, single-arm study that in Phase 1B determined the Maximum Tolerable Dose a
Slide 20: Median OS Outperforming in STS Lung Mets
Median OS Outperforming in STS Lung Mets Meta Analysis of 10 Studies and 2,267 Subjects* (Range of Salvage Therapies) 13.4 Months MB-107 (n=36) (Annamycin) 13.5 Months = Ea L | = | _— 24 Line 7 Line Annamycin delivering better performance 7" line than would be expected even in 2"4 line for monothera
Slide 21: PFS and OS Improved in Dose Dependent Manner
PFS and OS Improved in Dose Dependent Manner Responders (SD or PR) after 2 cycles showed improvement in OS and PFS Median PFS (pays) 122 127 Median OS (months) 19.7 19.9 13.5 63 210-390 mene, $330 210-390 meme, < 330 Median 6 prior lines mes Median 6 prior lines me/n $330 mg/m2 and 2 prior lines of
Slide 22: The Full Annamycin Opportunity Extends to Multiple Solid Tumor Indications
The Full Annamycin Opportunity Extends to Multiple Solid Tumor Indications? al & Pr ted by Existing Clin How Big Could the Market Be for Annamycin? Cancer cases per Garner year Treated with anthracyclines
Slide 23: Slide 23
Slide 24: Financials: Runway into the 1st Qtr. 2026
=, ~Runway into the 1st Qtr. 20267 [2] with pro forma $13.6M Q2-25 ) Cash Balance? Nasdaq: MBRX Financials \ ~$18.9M Market Cap? / 48.5M Shares 0/S* ~6.5M — Daily Trading Vol.° institutions for IIT programs for preclinical and clinical activity for non-Annamycin programs which reduces projected cash
Slide 25: Upcoming Milestones
Upcoming Milestones Annamycin AM Updates on rectuhenent; retin. [ impact of data readout Begin enrolment of 5 ine USand EU (n=45) on regulatory ‘Subjects in MIRACLE2 Begin contracting way, Recnitment ‘with MIRACLE ¥ Medical conference [ts ! Update Enrollment ends for 2° trial sites Posters/presentat
Slide 26: Comparable AML Therapy Data and Valuations
N 153 286 199/174 20 57 7 10 CR 38% 37% 19% /25% 35% 18% 20% 50% | CRe 48% 64% 23%/33% 40% 25% 47% 60% AML 50% 50% 15-23% 30%? Population Los half ts value a a Revenue? $128M $28 ~$150M license deal less than an $1.58 $28 Valuation Exit N/A Exité (tcqtion ot (equion ot eon, 206) foo 200)
Slide 27: Appendix
Appendix
Slide 28: AML Clinical History
AML Clinical History Phase 1: MB ‘+ 17% CRi (at suboptimal dosing) + Dosing limited by FDA lifetime Anthracycine Dose (ITMAD) ‘+ Trial location — US, + Well-tolerated in the study population + Limited to low doses + Morphologic leukemia free state was achieved in one subject in the 120 mg/m cohort ‘
Slide 29: MB-106: Durability, MRD, Prior Therapies - CR/CRi
MB-106: Durability, MRD, Prior Therapies - CR/CRi Preliminary HH - Durability of Remission [i @ = Se Notes Below 28305 eat ae relia and sje to change url a ln Sy Resor is ube. 2| MAD testing not standard between ste. 8) ropesson based on SOSEBNT and ded of in fs id net recehe SOC for infcton and suc
Slide 30: MB-106 Response by Genotype and Mutation
MB-106 Response by Genotype and Mutation men 31 cHRM KMT2A, is | [e20; RUNG | sort] ———ae] 89% (n=9) of CRc responses came from subjects with “generally poor prognosis cytogenetics and mutations” Annamycin appears to be “genotype/mutation agnostic”
Slide 31: 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 Aza/ | Venza Ven-Dec | Ven-Dec eral ‘AnnAraC 40% CR rate and 60% CRe Rolapsedor | Best yes | AnntraC | Annv® | Survival rate in Venetoclax R/R AML Refractory | Efficacy | (months
Slide 32: Notes (see Slide 12)
Notes (see Slide 12) 1, This chart compares Complete Response (CR) rates as submitted for FDA approval for existing 2nd line therapies (as single agents) to preliminary CR data for AnnArac (the combination of Annamycin and Ara-C); Note: these data are from separate clinical trials with differing pro
Slide 33: Annamycin: A Next-Generation Anthracycline
Annamycin: A Next-Generation Anthracycline enon oH 8 3 een Annamycin on, Son To ron wm Dt Tas 3 ma A “h Epirubicin Idarubicin Doxorubicin
Slide 34: Eliminating Cardiotoxicity
Eliminating Cardiotoxicity 3000 -0n Zero Cardiotoxicity in ~100 100% Cardiac Event Patients Treated FDA Lifetime Limit Level 65% Cardiac Event >8% Heart Failure | Level B50 mone 550 nome Currently Prescribed Anthracyclines Annamycin pean
Slide 35: 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 amycin is effectively shows Ani jotoxic when compared icin. non-ca with doxor Free Annamycin (API only) is bstantially le diotoxic t
Slide 36: Annamycin Synergizes with Ara-C in Increasing Survival
Annamycin Synergizes with Ara-C in Increasing Survival in Highly Aggressive p53-null, FLT3 mutated AML Model Analysis of Minimal Residual Disease (MRD) LANN LANN# ARAC Day 36 Day 36 100 Bone marrow : spleen was Sian Senos o> LANN+ARAG 0 10 20 30 40 60 60 70 80 90 100 110 120 130 50: Percent survival
Slide 37: Performance of AML Therapies in Line
Performance of AML Therapies in 24 Line CLAVELA: International Randomized Phase III Study of Elacytarabine Versus Investigator Choice in Patients with Relapsed/Refractory Acute Myeloid Leukemia 381 Elacytarabine Therapies compared: + high-dose + low-dose cytarabine R/R AML (compared with) ie ‘ cytar
Slide 38: 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) oom at ~1 month Annamycin NDA to som PME HIDAC CR rates are well documented 7 ~ be based on CR rate no = in 2"4 line subjects paman
Slide 39: WP1066 Update
WP1066 Update Clinical Trial with WP1066 at Northwestern University + Externally funded Phase 2 trial of oral WP1066 in combination with radiation treating glioblastoma (GBM), a form of brain cancer + Northwestern is actively enrolling subjects with 7 recruited dosed to date + Minimal investment fro
Slide 40: Science Advisors
Science Advisors Waldemar Priebe, PhD MO Anderson Cancer Center Founding Scientist & SAB Chair Hematology Oncology Or. Martin Tallman Northwestern University Dr. Daniel Von Hoff Mayo Clinic Dr. Jorge Cortes ‘Augusta University Dr. Michael Andreef Dr. Giovanni Martinelli MD Anderson Cancer Center Bol
Slide 41: Global Network
Global Network 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 tria
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