Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (November 2024)
38 slides from Moleculin Biotech's investor presentation, Nov 18, 2024
Slide 1: Moleculin Biotech
pmouscuum We:
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, four business strategy and plans, and our objectives for future operations, are forwardlocking statements. The words “believe,”
Slide 3: Recent Developments
Recent Developments * Announced new findings showing Annamycin overcomes resistance to Venetoclax in AML * Released median overall survival in MB-106 trial for Annamycin in combination with cytarabine (AnnAraC) treating AML was 9.1 months for 0-6 prior lines of therapies (n=22) and 11.6 months for 2
Slide 4: AnnAraC Performance in R/R AML
AnnAraC 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 every approved for use in
Slide 5: Management and Chairman
1 Prose, CEO and airman a mk = 200 FDA Approvals Big Pharma Exits Moleculin Clinical Trials Million in Recent Management Investment Years of Drug Development Experience
Slide 6: Technology Portfolio
Technology Portfolio Program Indication Phase 3 paanven R/R AML Next-Gen Anthracycline sts tung Mets we1066 Adult GBM STAT Inhibitor Pediatric Brain Tumors we1122 Anti-metabolte CPM: Virology
Slide 7: Current Market Cap Creates Opportunity
7 “oe _ <X{MOLECULIN | | 2) Current Market Cap Creates Opportunity aN tom * Investors focused on targeted therapies, yet Venetoclax (a Anthracyclines Used In: chemotherapy) created far more value over last 5 years Breast ancars 256) * For 50+ years anthracyclines have remained the bedrock treatment
Slide 8: Annamycin Attributes
Annamycin Attributes No vesicant activity Developed in (safer to handle and collaboration with administer) and licensed from Composition (ore of Matter focus Avoids Cross Pla mie) wet le 2040 Resistance ieee oo Cardiotoxic wah curently escibed ema ie Setanta ice Sicoacgas : Omer BE Silents Fee wet 1
Slide 9: Approved Therapies are Successful for Only ~40% of AML Patients
Approved Therapies are Successful for Only ~40% of AML Patients? Daunorubicin| Venetoclax Targeted Therapy Regimen 143 Ven-Aza Gene-Targeted FitPatients UnfitElderly §5%-65% Sub-population (60%) (50%) Eligible Durable CR% (NBC 37% ~21% AIl AML benefit? 14% 15% 7% criticar Catdiotox; de Stillleaves L
Slide 10: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) nbined Subjects Evaluable Tc 1 1 gerbe rel? 22 4 ! 10 1 14 Subjects Evaluable Not ' Saapeteod 2 rn Median Age - Years euce 67.5 (19-78) 56.5 (19-69) H 71 (53 - 78) Hl 69.5 (53-78) Complete Remission i (CR) 8 (36%) 2 (50%) H 5 (50%) H 6 (43%) Complete Remiss
Slide 11: Durability, MRD, Prior Therapies
Durability, MRD, Prior Therapies Prior 600 | Therapy 78% MRD negative 50% of CRs bridged to BMT Prior Lines of Therapy e e e e e e Fi Subject relapsed, likely due to cessation of treatment due to infection, Continues with F/U Subject died from infection PD © Progressive disease (PD) 1) Mesa nb fo dr
Slide 12: AnnAraC 2L Efficacy in Venetoclax R/R Patients
AnnAraC 2L Efficacy in Venetoclax R/R Patients Venetoclax regimen failure leaves patients with dismal options Durability AnnAraC 60% CRc rate Relapsed or and 40% CR rate in biti Venetoclax R/R AML 78 Relapsed PD 7 3 R 20 64 — Refractory SD 2 1 R 2s 64 elapsed aR a 2 crit 33 75 Refractory PR 6 1 PD 7
Slide 13: MB-106 Response by Genotype and Mutation
MB-106 Response by Genotype and Mutation 031 CHRM Complex Karyotypes 89% (n=9) of CRc responses ore ee came from subjects with RUNXWITI “generally poor prognosis rs} ” cytogenetics and mutations’ co Annamycin appears to be cos “genotype/mutation agnostic” Fer) oH DH ‘TPS3 NoneiNot tested Note —n=20;
Slide 14: AML Clinical History
AML Clinical History Phase 1: MB-104 + 17% CRi at suboptimal dosing) : * Dosing limited by FDA Ufetime Anthracycline + Dose (UIMAD) + Tal lecation— US ¢ ‘+ Well:oleratedin the study population . + Uinrted to low doses + Morphologic leukeria free state was actievedin one subject inthe 120me/m? cohort
Slide 15: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE ‘Moleculin R/R AML AnnAraC Clinical Evaluation 2 unblinded Data Readouts HIDAC (0-30) Annamycin Optimurr Establish >se + HiD, Optimum Dose* Annamycin 230 me/m? + HIDAC *Criteria Efficacy i Control Arm Safety DAC + placeb« Tolerability Control Arm Doses and adaptive desi
Slide 16: FDA provided clear written guidance on dose comparison
FDA provided clear written guidance by recommending the comparison of 190 mg/m? vs. 230 mg/m? Project (e) ptim US Clinical experience to date shows no significant safety or efficacy difference between 190 and 230 Gu id ance / Initial PK analysis shows no correlation between AUC oF Crnae y, and chang
Slide 17: Estimated Regulatory Timeline
Estimated Regulatory Timeline {3 line trial and secondary ipoints (OS, DoR) for .¢ label expansion pescn
Slide 18: Part A Acceleration
Part A Acceleration 2024 2025 Enrollment Begins finial unblinding result inan optimum dose selection, 3° arm may be dropped Unblinded Data Readout 45 Breakthrough! Designation 2026 Unblinded Data Readout +30-45 DMC review to confirm dose for Part Band ethics of control arm
Slide 19: Change to Protocol Explained
Change to Protocol Explained * Allows unblinding at 45 subjects for evaluation of primary endpoint, safety and tolerability * If at that point, it is apparent that one or the other of the two Annamycin doses (190 mg/m2 or 230 mg/m2) is considered “optimum,” we will discontinue the non-optimum dosage
Slide 20: The Bar for Approval is Low
The Bar for Approval is Low Complete Remission (CR) 43% HIDAC CR rates are well documented sell Hoact wioac? anarac? ponarac3 Mires Teal Clase Tra Comars 2nd 3d ne 50% Annamycin NDA to be based on CR rate in 2"¢ line subjects at ~1 month 41 —Mirros Trial, 81% 2° ine patients; 17% CR, within 56 days,
Slide 21: Potential Accelerated Regulatory Timeline
Potential Accelerated Regulatory Timeline 2024 | | | | Enrollment] Accelerated Approval Ends on Primary Endpoint Enrollment Begins wr 2025 | 2028 | Breakthrough Designation
Slide 22: Site Selection is Moving Quickly
© Interested/visited 60 sites interested . © Additional 17 more sites targeted Updated: 11, Site Selection is Moving Quickly G orcs
Slide 23: 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 ~ ~13% of 2L AML Patients Expected to JAMaaag Achieve CR 1 } 40.0% 1 C , ‘0H ' 30.0% 1 i 1 20.0% 1 | 1 10.0% 5.8% 2.3% 1.7% 2.4% 1.3% 1 ' i 1 0.0% | - - - =; 1 GilteritnibEnasidenib_Ivosidenib Olutasidenib Gemtuzumab| AnnArac | (Xso
Slide 24: Potential Asset Value
Potential Asset Value ————— Dprov 1 Line line vier Kura mda’ Idhifa/Tib iftomenib rumenib N 153 286 199/174 20 57 17 10 CR 38% 37% 19%/25% 35% 18% 20% | 50% ‘| cRe | 48% 64% 23%6/33% 40% 25% 47% 60% Pouca 50% 15-23% 3032 2a 300 Revenue? $128M $28 ~$150M ton of in of eon 6) eo 20). Cap? Cant $1.5B $2
Slide 25: The Full Annamycin Opportunity
The Full Annamycin Opportunity? Potential for Significant Upside — 60K | ; Pancreatic ver 13,500 PFS and OS rivaling om BOWE 1° line in 4-5" line are 67K STS subjects? Endometrial Lung : _ — 20,000 104K Initial Annamycin pelea Colorectal Target Indications Leukemia Leacmnsranerae) iver ‘TOTAL INCIDE
Slide 26: Financials
Financials — Nasdaq: MBRX \ [8] ~$9.4M Cash Balance ~$17.3M Market Cap? 3.0M Shares O/S and 6.1M Shares Fully ‘ Diluted Outstanding: =f 32K — 65-day Avg. Daily Trading Vol.5
Slide 27: Upcoming Milestones
Upcoming Milestones’ Begin contracting with MIRACLE trial sites 2H 2024 First subject treated in MIRACLE trial 1Q,2025 Data Readout (n=45) unblinded efficacy/safety review 2H 2025 Annamycin __ Data Readout (n=~75-90) unblinded and Optimum Dose 5 2H 2026 AML set for MIRACLE trial Begin enrollment of
Slide 28: MIRACLE
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 29: Appendix
Appendix .
Slide 30: Annamycin: A Next-Generation Anthracycline
Annamycin: A Next-Generation Anthracycline Zeenon ae Gu = ene Annamycin aon, [ Son <on | | | mo 8 on 6 om} dn, em 8 (om) I SENT Oye Seon 7 i" < he Epirvbicin NN Idarbicin Doxorubicin
Slide 31: Annamycin Has Demonstrated Substantially Greater Cardiac Safety
Annamycin Has Demonstrated Substantially Greater Cardiac Safety Compared to Approved Anthracyclines Current Anthracyclines Annamycin diac Peworecuum
Slide 32: 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 00 measuring cardiotoxicity shows Annamycin is effectively non-cardiotoxic when compared with doxorubicin. Free Annamycin (API only) is substantially less ca
Slide 33: Annamycin Synergizes with ARA-C in p53-null, FLT3 mutated AML
Bone marrow Spleen Lungs Annamycin Synergizes with ARA-C in Increasing Survival in p53-null, FLT3 mutated AML Analysis of Minimal Residual Disease (MRD) LAN LANN# ARA-C Day 36 Day 36 Percent survival Model (AML-mTurqoise2) 100) a a © Vonicie g 8 Soot smone Shacoane aint finns 0 10 20 30 40 50 60 70
Slide 34: 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 cytarabin
Slide 35: 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 stay 4(28)a(aa) team) 202) 28a 17 asf gaz ae ae aa Preliminary and C
Slide 36: 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 * Preliminary results, subject to final clinical study report which is expected to be issued in
Slide 37: Science Advisors
Science Advisors Waldemar Priebe, PhO MO 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 Cancer C
Slide 38: All technology licensed from MD Anderson Cancer Center
All technology licensed from MD Anderson Cancer Center (MDACC) Supports continuing preclinical research on our technology at MDACC close to $1M per year Active contractors in US, EU and Asia for drug production and distribution as well as for clinical trial management Past & current externally funde
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