Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (December 2024)
46 slides from Moleculin Biotech's investor presentation, Dec 18, 2024
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 ¥ Announced new findings showing Annamycin overcomes resistance to Venetoclax in AML Y 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: 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 every approved for use
Slide 5: Opportunity for Market Cap Breakout
Opportunity for Market Cap Breakout
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 iz \ No vesicant activity Developed in \, (safer to handle and / collaboration with administer) and licensed from / Composition ( of Matter y \ \ Patents thru Al Wel(e Oa @ roxy Ls \ Non- ‘iiew Resistance Cardiotoxic pe Was anthracyclines, Ara-C and Venetodaxin preclinical model
Slide 8: Eliminating Cardiotoxicity
Eliminating Cardiotoxicity Zero Cardiotoxicity in ~100 Patients 100% Cardiac Event Treated Level >8% Heart Failure FDA Lifetime Limit 65% Cardiac Event Level Cardiac Events 550 rom | | Currently Prescribed Anthracyclines Annamycin
Slide 9: Approved Therapies leave ~60% Unmet Need of AML Patients
Approved Therapies leave “60% Unmet Need of AML Patients’ Daunorubicin| Venetoclax Targeted Therapy Regimen [i733 Ven-Aza _ Gene-Targeted FitPatients UnfitElderly §5%-65% Sub-population (60%) (50%) Eligible Durable CR% (NNNBER2 37% ~21% All AML benefit? 14% 15% 7% criticar Catdiotox; de Stillleaves
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% 50.0% 40.0% 30.0% 20.0% 10.0% 0.0% ~13% of 2L AML Patients Expected to Ji Achieve CR 1 rn i) 1 \ 1 1 i) 1 | a I 2.3% 1.7% 2.4% 1.3% 1 | - - a =| Gilteritinib (Xsopata) (FtT3) Enasidenib Ivosidenib Olutasidenib Gemtuzumab AnnAraC (Idhifa)_
Slide 11: Opportunity for Market Cap Breakout
Opportunity for Market Cap Breakout proved I Line | Abbvie | Servier Revumenib N 153 286 199/174 20 37 7 10 CR (38% 37% 19%/25% 35% 18% 20% 50% | CRe 48% 64% 23%6/33% 40% 25% 47% 60% Poni 50% 50% 15-23% 24962 Revenue? $128M $28 ~$150M $1.58 $28 Valuation Exit! N/A Exits (equston of egustion of elr,
Slide 12: The Bar for Approval is Low
The Bar for Approval is Low Complete Remission (CR) HIDAC CR rates are well documented Annamycin NDA to be based on CR rate in 2"¢ line subjects at ~1 ae month Hoact wioac? onarac? Aonanc3 ——Annrac? Mires ral Clase ral ——lComars nde 2rd Une Ondine 41~Mirros Trial, 81% 2°° line patients; 17% CR, with
Slide 13: Next-Generation Anthracycline
Next-Generation Anthracycline — Redefining how anthracyclines are used Anthracyclines remain the bedrock treatment for many cancers — because they work Annem appears safer and more effective than current ly prescribed anthracyclines Lack of cardiotoxicity enables increase in dosage and number of cyc
Slide 14: The Full Annamycin Opportunity
The Full Annamycin Opportunity? Potential for Significant Upside — 60K 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 ~33.5K
Slide 15: Slide 15
Slide 16: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) Line of Therapy All Lines (1-7) Subjects Evaluable To E : ate 22 4 ! 10 1 4 Subjects Evaluable Not ' Dosed Per Protocal 2 u H d ! ; Leaky 67.5 (19-78) s65(1962) | 74(53-78) 1 69.5(53-78) ace) 1 I ‘Complete Remission 1 <a 8 (36%) 2 (60%) 1 5(50%) | 6 (43%) r
Slide 17: Durability, MRD, Prior Therapies
Durability, MRD, Prior Therapies ~8 months median durability and climbing 78% MRD negative 50% of CRs bridged to BMT [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 infec
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 apsedor e and 40% CR rate in Venetoclax R/R AML Recent study? of Ven-HMA failures in 1L patients: + Dismal outcomes upon failure - 05 median for R/R AML
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 ome 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 ‘TPS3 NoneiNot tested Note~n=20; Some su
Slide 20: AML Clinical History
AML Clinical History Phase 1: MB-104 MONOTHERAPY 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 free state was actievedin one subject inthe 120me/m? cohort D
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 prelit jata suggest that Annamycin could re atment for AML. | am excited to work alongside the N er explore ‘Martin Tallman, MD, Internationally Renowned Clinical Investigator whose Discoveries have Fueled the Progress of Leukemia-targeting
Slide 23: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation Annamycin 2 unblinded 190 mg/m? + Data Readouts HIDAC Annamycin Optimum Dose + HiD/ Annamycin *Criteria: Efficacy mize Control Arm. Safety HIDAC + placebc Tolerability Control Arm HIDAC + placebo Doses and adaptive design re
Slide 24: FDA provided clear written guidance on 190 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 25: Estimated Regulatory Timeline
Estimated Regulatory Timeline Readout Readout 4s +30-45 Rolling NDA Bwouecuun
Slide 26: Potential Part A Acceleration
Potential Part A Acceleration 2026 Unblinded Unblinded Enrollment Data Data Begins Readout Readout >_> 45 +30-45 finial unblinding results in an optimum dose lection, 3° arm may be dropped DMC review to confirm \ Breakthrough! Designation
Slide 27: 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 28: Potential Accelerated Regulatory Timeline
Potential Accelerated Regulatory Timeline 2024 | Enrollment Data Begins Readout 45. Breakthrough Designation 2027 I | Data Enrollment Accelerated Approval Readout Ends 90 on Primary Endpoint Part A meets high end of expectations, we may be able to shorten Part B to single arm safety/DE! only Drug ap
Slide 29: MIRACLE Site Selection is Moving Quickly
ra © Interested/visited ~70 sites interested la liiterested © Additional Targeted More sites targeted Upd fated: 12/16/24 MIRACLE Site Selection is Moving Quickly it tes Ukraine Consistent feedback from face-to-face meetings with over 30 potential investigators: “Annamycin is desperately needed to f
Slide 30: Slide 30
Slide 31: Technology Portfolio
Technology Portfolio Program Indication Phase 1 Phase 2 Phase 3 Amana R/RAML Miracle: Adaptive Phase 3 Pivotal Initiating Next-Gen Ce Antbracyclipe STS Lung Mets 2 Trials Active; Alt. Dosing; P1 we1066 ‘Adult GEM NU IIT with Radiation Active; P2 STAT3 Inhibitor Pediatric Brain Emory IIT P2 Planning
Slide 32: Slide 32
Slide 33: Our Team
Our Team Founders Present CEO and haan 7 eo FDA Approvals 2 Big Pharma Exits Moleculin 14 Clinical Trials $ 5 Million in Recent +9 Management Investment Years of Drug 200 Development Experience Wolfram C.M. Dempke, MD, PhO, MBA European Medal Advsor panamecl> autem Exovnns Grown
Slide 34: Slide 34
Slide 35: Financials
Financials Nasdaq: MBRX ~$9.4M Cash Balance? ~$17.3M Market Cap? 3.0M Shares O/S and 6.1M Shares Fully Diluted Outstanding: ~77.2K —1 yr Avg. Daily Trading Vol.>
Slide 36: Upcoming Milestones
Upcoming Milestones Annamycin AML Impact of data readout Begin enrolment of 3 tine Development /Phvotal Program (045) on regulatory subjects in MIRACLE? Begin contracting with pathway; Recruitment I ‘MIRACLE tral sites pesca cxetanee Update Enrolment ends for 2"! posters/pesentations Recess | | I ra
Slide 37: MIRACLE Phase 3 Trial
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 38: Appendix
—" ——— Appendix porn #
Slide 39: Annamycin: A Next-Generation Anthracycline
Annamycin: A Next-Generation Anthracycline Aone sei ‘Annamycin ‘ em, <n — Trey | rey Sy Aeon ™ Epirubicin Idarubicin Doxorubicin
Slide 40: 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 41: Annamycin Synergizes with Ara-C in AML Model
Bone Annamycin Synergizes with Ara-C in Increasing Survival in Highly Aggressive p53-null, FLT3 mutated AML Model Analysis of Minimal Residual Disease (MRD) LAN LANN# ARA-C 100: =~ pened mie wee ae 06 . 2 * een cam am z P z © Vehicle so Sam anonasl “+ ARA-C 50 mgikg > LANN + ARA.C Percent survival 0
Slide 42: Performance of AML Therapies in 2nd Line
Performance of AML Therapies in 2"4 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 43: 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 ‘Two Treatment Cycles All Subjects Demonstrated Improvement with Dose < 330 mg/m? and Fewer Prior Therapies Preliminary MB-107 Summary as of ns) wa) aa 1 1
Slide 44: 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 45: 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 46: All technology licensed from MD Anderson Cancer Center
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
Moleculin Biotech — Investor Presentation (December 2024) on 6ix