Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Corporate Update (November 2024)
40 slides from Moleculin Biotech's corporate update, Nov 14, 2024
Slide 1: Moleculin Corporate Presentation
Slide 2: Disclaimer
Disclaimer All statements contained herein other than statements of historical fact, including statements regarding our future results of operations an financial position, ‘our business strategy and plans, and our objectives for future operations, are forward-looking statements. The words “belive,”
Slide 3: Recent Developments
Recent Developments * Accelerates Planned Unblinding Data Readout for MB-108 MIRACLE Phase 3 Pivotal Trial for Annamycin in Combination with Cytarabine (AnnAraC) for the Treatment of R/R/ AML to H2 2025 * Received Institutional Review Board Approval for MB-108 MIRACLE Phase 3 Pivotal Trial * Announc
Slide 4: AnnAraC Performance in 2nd Line AML
AnnAraC Performance in 2"4 Line 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. These performance numbers are significantly better than any therapy every approved for use in 2L AM
Slide 5: FDA Approvals
7 FDA Approvals 2. Big Pharma Exits Moleculin 14 Clinical Trials $ GC Million in Recent *9 Management Investment Years of Drug 200 Development Experience
Slide 6: Technology Portfolio
Technology Portfolio Program Annamycin Next-Gen. Anthracycline we1066 STAT Inhibitor we1122 Anti-metabolite Indication R/RAML STS Lung Mets Adult GBM Pediatric Brain Tumors. GBN; Virology Phase 3
Slide 7: Current Market Cap Creates Opportunity
- MOLECULIN | - Current Market Cap Creates Opportunity aN emm * Investors focused on targeted therapies, yet Venetoclax (a Anthracyclines Used In: chemotherapy) created far more value over last 5 years i pranse cancarss 32%. * For 50+ years anthracyclines have remained the bedrock treatment for many
Slide 8: Annamycin Attributes
Annamycin Attributes No vesicant activity Developed in (safer to handle and ‘collaboration with oe administer) and licensed from Composition é MD Anderson: of Matter fac” Avoids Cross ey eae Non- ae) Resistance ee Cardiotoxic wth curently prescribed Cemphalylts ) preclinical models \. ax for Dox NCE
Slide 9: Estimated Regulatory Timeline
Estimated Regulatory Timeline 43 line trial and secondary "endpoints (05, DoR) for MilestMilestone #0 expansion
Slide 10: Part A Acceleration
Part A Acceleration 2024 2025 Enrollment Begins finial unblinding results in an optimum dose lection, 3° arm may be dropped Unblinded Data Readout 45 Designation 2026 Unblinded Data Readou +30-45 t
Slide 11: 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 dosag
Slide 12: Approved Therapies are Successful for Only ~40% of AML Patients
Approved Therapies are Successful for Only ~40% of AML Patients? Daunor Venetoclax Targeted Therapy Regimen 743, Ven-Aza Gene-Targeted FitPatients UnfitElderly §5%-65% Sub-population (60%) (50%) Eligible Durable CR% [iS6%2 37% ~21% AIAML benefit? 14% 15% 7% Criticar Catdiotox; de —Stillleaves Limite
Slide 13: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) Subjects Evaiuable To 1 ashi 22 4 1 10 H 14 Subjects Evaluable Not 1 ‘Dosed Per Protocol 2 ° ‘ 1 4 i bela gaat 67.5 (19-78) 56.5 (19-69) ' 71 (53 - 78) 4 62.5 62-78) srission 1 Cleon Bir asad 8 (36%) 2(60%) t 560%) 1 6 (43%) 1 Complete Remission spell 9 (41
Slide 14: Durability, MRD, Prior Therapies
Durability, MRD, Prior Therapies Bao} £ Therapy MRD 78% MRD negative 50% of CRs bridged to BMT urabilty of Remission continuing f= Prior Lines of Therapy Subject relapsed, likely due to cessation of treatment due to infection, Continues with F/U Subject died from infection PD © Progressive disease (
Slide 15: MB-106 Response by Genotype and Mutation
MB-106 Response by Genotype and Mutation Q31 CHRM Complex Karyotypes 89% (n=9) of CRc responses anes peed came from subjects with arenas “generally poor prognosis a cytogenetics and mutations” Es Annamycin appears to be os “genotype/mutation agnostic” Pers: He DH P53 NoneiNot tested Note —n=20; Some
Slide 16: AML Clinical History
AML Clinical History Phase 1/2: MB-106. oN + N=20 ‘+ N=22alllines (0-6), N= 10 2" line) + 17% CRi (at suboptimal dosing) + Median fines of prior therapy = 4 1+ All subjects (N22) 41% CRe (177) + Dosing limited by FDA tifetime + Median age of 240 me/r?(RPD2) + 2nd tine N=10, 60% CRe ‘auhracycline Dos
Slide 17: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation Annamyci 2 Unblinded 4190 mg/m? + Data Readouts HIDAC 0) _ Annamycin Optimurr Optimum Dose* Annamycin 230 mest HIDAC *Criteria: Efficacy mize 1: Control Arm Safety DAC + placeb Tolerability Control Arm HIDAC + acebo Lee Dose
Slide 18: 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 Optimus fiisrSifcenetewwens0und 20 Guidance Initial PK analysis shows no correlation between AUC OF Cinae and change in dosage from 190 to 230 Corroborates clinical findings Could reduce potential f
Slide 19: Estimated Regulatory Timeline
Estimated Regulatory Timeline 43 line trial and secondary "endpoints (05, DoR) for MilestMilestone #0 expansion
Slide 20: The Bar for Approval is Low
The Bar for Approval is Low Complete Remission (CR) HIDAC CR rates are well documented ell Hoact woac? Aanarac3 Anarac3 Mires Tral Clase Tal kComers 2nd. 3rd Une 50% Annamycin NDA to be based on CR rate in 2"¢ line subjects at ~1 month 1~Mitros Trial, 81% 2° ine patients; 17% CR, within 56 days, Kon
Slide 21: Potential Accelerated Regulatory Timeline
Potential Accelerated Regulatory Timeline Breakthrough Designation Peworecuum
Slide 22: Site Selection is Moving Quickly
60 sites interested 17 more sites targeted Site Selection is Moving Quickly 12sites
Slide 23: CR Rate and Weighted Benefit vs Approved AML Therapies
60.0% 50.0% 40.0% 30.0% 20.0% 10.0% 0.0% 4 1 1 500% | ; 1 H 1 ; 1 i ' f 1 A 1 H 1 i ' 1 1 1 5a 2.3% 1.7% 2.4% 13%} 1 1 = = = -= —! 1 1 GilteritinbEnasidenib —_IvosidenibOlutasidenib. Gemtuzumab, AnnArac _1 (Xsopata) (Idhifa)?—(Tibsovo)_—_(Rezlidhia)__(Mylotarg)? (Anna+Arac)? 1 (FLT3) (I10H2) (1oH1)*
Slide 24: Potential Asset Value
Potential Asset Value ( ‘Approved Phase 2 Complete 1 Line Idhita/ N 153 286 199/174 20 57 7 10 CR 38% 37% 19% /25% 35% 18% 20% 50% | CR 48% 64% 2396/33% 40% 25% 47% 60% Ws 50% 50% 15-23% 30%? 24962 30%? Revenue? $128M $28 ~$150M $1.58 $28 Valuation Exit? N/A Exit? (equston of eguston of er, 2016) ‘s
Slide 25: The Full Annamycin Opportunity
The Full Annamycin Opportunity? Potential for Significant Upside — 60K Pancreatic ver 13,500 PFS and OS rivaling om Stem 1° line in 4-5" line cre STS subjects? Endometrial Lung a 20,000 — 104K Initial Annamycin ‘aie ast Colorectal Target Indications Leukemia craccap eas Liver TOTAL INCIDENCE ~33.5K
Slide 26: Financials
Financials Nasdaq: MBRX ~$9.4M Cash Balance? ~$17.3M Market Cap? 3.0M O/S and 6.1M Shares Fully Diluted Outstanding* ~32K — 65-day Avg. Daily Trading Vol.>
Slide 27: Estimated Regulatory Timeline
Estimated Regulatory Timeline 2024 2025 2026 2027 2028 | 2029
Slide 28: Upcoming Milestones
Upcoming Milestones \ = Begin contracting with MIRACLE trial sites 2H 2024 First subject treated in MIRACLE trial 1Q,2025 Data Readout (n=45) (safety and overall efficacy review) 2H 2025 Annamycin __ Data Readout (n=~90) unblinded and Optimum Dose set for MIRACLE trial 242006 AML Begin enrollment of
Slide 29: 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 30: Appendix
Appendix
Slide 31: 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 025 * Preliminary results, subject to final clinical study report which is expected to be issued
Slide 32: Annamycin: A Next-Generation Anthracycline
Annamycin: A Next-Generation Anthracycline LL dan a ee AS il ‘Annamycin a Coe = . © et, mt Epirubicin Idarubicin mm Doxorubicin
Slide 33: Annamycin Has Demonstrated Substantially Greater Cardiac Safety
Annamycin Has Demonstrated Substantially Greater Cardiac Safety Compared to Approved Anthracyclines Current Anthracyclines Annamycin Zero Cardiotoxicity
Slide 34: 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 c measuring cardiotoxicity shows Annamycin is effectively " non-cardiotoxic when compared with doxorubicin. Free Annamycin (API only) is substantially less c
Slide 35: Annamycin Shown Effective Against 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 mi nitivisss: ee oof EE, ee x {Sy ¥ bh i ve tan VIX, 1000 pnt uy mS & * oa 0. ——= 7 0. ——s a a tote seo aie concentrations, att concentrations, aMt
Slide 36: Annamycin Synergizes with ARA-C in Increasing Survival
Bone marrow Spleen Lungs Annamycin Synergizes with ARA-C in Increasing Survival in p53-null, FLT3 mutated AML Model (AML-mTurqoise2) Analysis of Minimal Residual ase (MRD) ANN LANN+ ARA-C 100: a Day 36 Day 36 a CS © Vehicle 50: = LANN 4 matkg, “+ ARA-C 50 mgikg > LAN + ARA-C Percent survival 0 10 20
Slide 37: 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 cytarabine
Slide 38: 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 Ry sua ae ateay) aga 20) 208) ae 307) [sa
Slide 39: Science Advisors
Science Advisors Waldemar Priebe, PhD 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 Andreef Dr, Giovanni Martinelli MD Anderson Cancer Center
Slide 40: Global Network
Global Network 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