Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Day (October 2024)
30 slides from Moleculin Biotech's investor day presentation, Oct 17, 2024
Slide 1: Virtual Acute Myeloid Leukemia KOL Day
7 “S:MOLECULIN “Virtual Acute Myeloid Leukemia KOL Day 43 , October 14, 2024 = < 3 FT a os
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: Opening and Introductions
Opening and Introductions Annamycin Overview Anthracyclines: Powerful Treatment Tools Annamycin: Potential to Revolutionize the AML Treatment Landscape Annamycin: First Non-Cardiotoxic Anthracycline MIRACLE: Phase 3 Pivotal Clinical Trial
Slide 4: Walter V. Klemp and John Paul Waymack, MD
od MOLECULIN Walter V. Klemp. John Paul Waymack, MD CEO and Chairman Senior Chief Medical Officer
Slide 5: Michael Andreeff, MD, PhD
Michael Andreeff, MD, PhD Professor of Medicine, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center + Professor of Medicine, Department of Stem Cell Transplantation, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center + R
Slide 6: Dr. Giovanni Martinelli
Dr. Giovanni Martinelli Associate Professor; Department of Medical and Surgical Sciences, Bologna University *+ Lead of the Clinical and Research Leukemia Program at institute of Hematology "Le A. Seragnoli” Bologna ‘Specialized in general hematology and medical genetics and primarily cares for pati
Slide 7: Mohamad Cherry, MD
Mohamad Cherry, MD Medical Director of Hematology, Atlantic Health System Principal Investigator of multiple cinical trials, performing groundbreaking research in niche areas that include epigenetics and development of new therapeutics in relapsed and refractory blood Clinical Associate Professor of
Slide 8: Walter V. Klemp
@ Walter V. Klemp CEO and Chairman
Slide 9: The Annamycin Opportunity in AML
The Annamycin Opportunity in AML OF Annamycin in combination with Cytarabine (AnnAraC) has potential to fill unmet need in AML; 60% CRc (50% CR) in 2nd line patients reported in latest Phase 18/2 study (MB-106) AnnAraC has the potential to more than double 2nd line AML Complete Remissions AIL 84 Ann
Slide 10: Driving Towards Important Data Readouts and NDA Submission
Driving Towards Important Data Readouts ()\— and NDA Submission \ -_. Begin contracting with MIRACLE trial sites 2H 2024 First subject treated in MIRACLE trial 10.2025 Recruitment Update (n=~45) 40.2025 Annamycin Data Readout (n=90) unblinded and Optimum Dose set for nai zoae AML MIRACLE trial Begin
Slide 11: The Disconnect in The Annamycin Opportunity
The Disconnect in The Annamycin Opportunity Market distracted by targeted therapies * Anthracyclines haven’t changed in 50 years, not on radar Breast Cancers = * Nearly half of all cancers are treated with anthracyclines AML Patients = 50% * Asafer and more effective anthracycline changes the game L
Slide 12: Topic Round Table: Anthracyclines: Powerful Treatment Tools
Topic Round Table Anthracyclines: Powerful Treatment Tools Dr. Giovanni Martinelli Michael Andreeff, MD, PhD Mohamad Cherry, MD
Slide 13: Topic Q&A Session
opic Q&A Session Anthracyclines: werful Treatment Tools
Slide 14: John Paul Waymack, MD
John Paul Waymack, MD Senior Chief Medical Office
Slide 15: AML Clinical History
AML Clinical History Phase 1: MB-104 MONOT# Phase 1/2: MB-105 IMONOTHE Phase 1/2: MB-106. ATION THER N 17% CRI (at suboptimal dosing) Dosing limited by FDA Lifetime ‘Anthracycline Dose (\TMAD) Tal location — US \Well-toleratedin the study population. Limited to low doses Morphologic leukemia fre sta
Slide 16: MB-106 (Annamycin + Ara-C (AnnAraC); n=22)
MB-106 (Annamycin + Ara-C (AnnAraC); n=22) : ; Line otTherapy | AlLines (1# 7") 1 Line 2" Line Line Combined Subjects Evaluable To: 1 me 22 4 1 10 ; 14 Subjects Evaluable Not 1 Dosed Per Protocol 2 Z H 1 4 2 PENSE CS) GAGE) ses(ie6s) 1 74 (53-78) | 69.5(63-78) ' 1 Complete bees 8 (36%) 2 (50%) { 5 (
Slide 17: Durability, MRD, Prior Therapies
Durability, MRD, Prior Therapies Prior ‘ 1 Therapy MRO, i |e e 1 |e) e i H e i 1 e : i ~7 months median H e durability and climbing u i e Hi 78% MRD negative iH e i i e t H e i Prior Lines of Therapy 1 gale i glee 1 Sag @ sudject relapsed, likely due cessation of treatment due to infection, Continue
Slide 18: Clinical History Supporting Activity in Leukemia
Clinical History Supporting Activity in Leukemia Trial to! Subjects at RP2D 8 5 22 RR AML/ALL ALL AML AML ae Total of 36 rior Lines of PUR AMLALL 15 (2 2-8 (A) 0-5 (1 Therapy (median) @) @) 0) subjects treated at over 10 unique sites Regimen Single Agent Single Agent Combination ee #of Sites 4 (Us)
Slide 19: 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% Achieve CR d f 1 [.- i 23% | 1.7% |. | ia a - _ - wale 1 GilteritinibEnasidenib —_Ivosidenib_Olutasidenib. Gemtuzumab, AnnAraC (Xsopata) (Idhifa}?_(Tibsovo) —(Rezlidhia)__ (Mylotarg)) (Anna+AraC)? (FLT3)
Slide 20: Topic Round Table: Annamycin: Potential to Impact the AML Treatment Landscape
Topic Round Table Annamycin: Potential to Impact the AML Treatment Landscape Dr. Giovanni Martinelli Michael Andreeff, MD, PhD Mohamad Cherry, MD
Slide 21: Topic Q&A Session
opic Q&A Session ycin: Potential to Impact the ML Treatment Landscape
Slide 22: Annamycin: First Non-Cardiotoxic Anthracycline
Annamycin: First Non-Cardiotoxic Anthracycline 3: John Paull Waymack, MD Senior Chief Medical Of
Slide 23: Annamycin: Overcoming Limitations of Current Anthracyclines
Annamycin: Overcoming Limitations of Current Anthracyclines Unique new structure: + Incorporates key structural elements of 3 different clinically used anthracyclines, plus “GY * The 3/-deamination and introduction of os He es 6 iodine at C-2 =k f Annamycin ~ 7 v7 Ken J Result: + Elimination of card
Slide 24: Topic Round Table: Annamycin: First Non-Cardiotoxic Anthracycline
Topic Round Table Annamycin: First Non-Cardiotoxic Anthracycline Dr. Giovanni Martinelli Michael Andreeff, MD, PhD Mohamad Cherry, MD
Slide 25: Topic Q&A Session
opic Q&A Session Annamycin: Von-Cardiotoxic Anthracycline
Slide 26: 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 John Paul Waymack, MD ws Se
Slide 27: Adaptive Trial Design MIRACLE
Adaptive Trial Design MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation Annamycin Unblinded 0 maf? + Data Readout HIDAC (0-30) Annamycin Optimum ‘Annamycin 230 maf? + HiDAC “Criteria: (0=30) Efficacy ome Control Arm Safety JiDAC + placebc Tolerability 150) Control Arm HIDAC + placebo eae Primary
Slide 28: The Bar for Approval is Low
The Bar for Approval is Low Complete Remission (CR) HIDAC CR rates are wel documented Annamycin NDA to be based on CR 2% rate in 2"4 —— 1% 1 e subjects - ss at ~1 month % Hoact Hioac2 Anarac Mires Tral Clase Teal and 3d Une 41.—Mitros Trial, 81% 2° ine patients, Konopleva et al, Blood Advances, 26 J
Slide 29: Topic Round Table: MIRACLE Phase 3 Pivotal Clinical Trial in AML
Topic Round Table MIRACLE Phase 3 Pivotal Clinical Trial in AML Dr. Giovanni Martinelli Michael Andreeff, MD, PhD Mohamad Cherry, MD
Slide 30: Q&A Session
“RA Session MIRACLE Moleculin R/R AML AnnAraC Clinical Evaluation