Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (January 2019)
31 slides from Moleculin Biotech's investor presentation, Jan 15, 2019
Slide 1: Moleculin Biotech Investor Presentation January 14, 2019
NASDAQ: MBRX Non - Confidential Information January 14, 201 9 1
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: Robust pipeline
3 Robust pipeline 3 distinctly different technologies, all with blockbuster potential World - leading collaboration MD Anderson Cancer Center/Mayo Clinic/Emory Breakthrough disruptive technologies Annamycin for AML: designed to be non - cardiotoxic, avoids MDR1 WP1066: STAT3 inhibitor that also stim
Slide 4: Experienced Management Team
Experienced Management Team 4 Selected Prior Experience Walter Klemp , Chairman, President & CEO Don Picker, PhD Chief Science Officer Jonathan P. Foster , CPA, CGMA EVP & CFO Robert Shepard, MD, FACP Chief Medical Officer Sandra Silberman, MD & PhD Chief Medical Officer – New Products
Slide 5: Science Advisory Board Members
Science Advisory Board Members 5 Waldemar Priebe, PhD Elihu Estey, MD John Paul Waymack, MD, SCD Jorge Cortes, MD James Abbruzzese, MD Marty Tallman, MD
Slide 6: Board Of Directors
Board Of Directors Michael D. Cannon - Chair of Compensation Committee Between 1997 and 2004, Mr. Cannon was the Chief Science Officer, EVP and a Director of SICOR, Inc. until its acquisition by Teva Pharmaceutical Industries, Inc. SICOR focused on generic finished dosage injectable pharmaceuticals,
Slide 7: Focus on Highly Resistant Tumors
Focus on Highly Resistant Tumors Example Indications Common Characteristics Regulatory Advantage • R/R AML • Glioblastoma • Pancreatic • Met melanoma • Other • Multidrug resistance • Immune evasion • Upregulation of oncogenic transcription factors • Increase in dependence on glycolysis • Significant
Slide 8: Core Technologies
3 Core Technologies Next Generation Anthracycline Immune/Transcription Modulator Metabolism/ Glycosylation Inhibitor • Avoids multidrug resistance • Little to no cardiotoxicity • Enables immune response • Inhibits p - STAT3, c - Myc and HIF - 1 α • Prodrug enables drug - like properties in glucose d
Slide 9: Development Pipeline
9 Development Pipeline Preclinical Clinical Preparation Phase 1/2 Collaboration Annamycin Annamycin + WP1066 WP1066 - Glioblastoma, melanoma metastasized to the brain WP1066 - Pediatric brain tumors WP1122 - Glioblastoma WP1122 + Avastin - Glioblastoma WP1066/WP1732 WP1122/WP1234 WP1220 - Cutaneous
Slide 10: Technology Review
Technology Review 10
Slide 11: Annamycin (Next Generation Anthracycline) Critical Advantages over Leading Drug
Annamycin ( Next Gen eration Anthracycline) Critical Advantages over Leading Drug Leading AML induction therapy drugs are cardiotoxic and lose efficacy due to multidrug resistance Annamycin has little to no cardiotoxicity, avoids multidrug resistance, has been shown to be more potent in AML cell lin
Slide 12: Annamycin Delivers Remarkable Performance
Annamycin Delivers Remarkable Performance In a proof - of - concept Phase I/II clinical trial, Annamycin was given to patients who had failed an average of five previous induction therapy attempts 37% of those patients cleared enough of their leukemic cells to qualify for a bone marrow transplant We
Slide 13: Annamycin Recap and Status
Annamycin Recap and Status Annamycin is a “ Next Generation” anthracycline designed to be non - cardiotoxic and avoid multidrug resistance Prior developer failed financially and lost rights to license Although impressive activity was shown in prior acute leukemia trials, developer did not properly c
Slide 14: WP1066 Immune/Transcription Modulators
WP1066 Immune/Transcription Modulators Based On Natural Compound Built from chemical backbone of propolis (caffeic acid benzyl ester) Unique Dual Action First - in - class drug to both directly inhibit tumor signaling (p - STAT3, HIF - 1 ⍺ , c - Myc) while also stimulating patient immune response (T
Slide 15: STAT3 is a Master Regulator of Tumor Progression
STAT3 is a Master Regulator of Tumor Progression 15 Activated STAT3 (p - STAT3) binds to DNA Triggering oncogenic transcription activity: Resulting in tumor growth and metastasis Survival Proliferation Inflammation Invasion Metastasis Angiogenesis Immunosuppression
Slide 16: WP1066 Affects Tumors Directly and Indirectly
WP1066 Affects Tumors Directly and Indirectly 16 Tumor WP1066 Immune Modulation Transcription Modulation WP1066 modulates transcription factors resulting in direct tumor cytotoxicity while also stimulating a natural immune response by reducing Regulatory T - c ells (T r egs)
Slide 17: Transcriptional Control of Cancer
Transcriptional Control of Cancer 17 HIF - 1 α STAT3 c - Myc Immunosuppressive Oncogenic Axis Implications for: • GBM • Pancreatic cancer • Other cancers
Slide 18: WP1066 Recap and Status
WP1066 Recap and Status WP1066 is a small molecule that inhibits p - STAT3 by accelerating proteasomal degradation (not by blocking phosphorylation) without respect to upstream signaling WP1066 also inhibits HIF1 - α, c - Myc and T r egs Currently in Phase 1 trial at MD Anderson for GBM and melanoma
Slide 19: WP1122 Metabolism/Glycosylation Inhibitor
WP1122 Metabolism/Glycosylation Inhibitor Addicted to Sugar A brain tumor requires as much as 18 to 37 times as much glucose to survive as a healthy brain cell Starving a Tumor to Death This eventually led to the theory that, if we feed tumor cells a glucose decoy (one that can’t convert into energy
Slide 20: Many Tumors are Highly Glycolytic
Many Tumors are Highly Glycolytic 20 F 18 DG PET MRI Tumors rely preferentially on glycolysis even in the presence of abundant oxygen For example: PET diagnostic imaging relies on a modified glucose with a radio - tracer (F 18 DG) Tumors over - consume F 18 DG because of their dependence on glycolys
Slide 21: Chemistry – Improving the Drug-Like Properties of 2-DG
Chemistry – Improving the Drug - Like Properties of 2 - DG and Targeting Brain Cancers 21 Example of heroin Heroin is the diacetyl ester of morphine that increases by 100 - fold levels of morphine in the brain WP1122 is the diester of 2 - DG of our design, which greatly enhances CNS uptake and level
Slide 22: Pharmacology (PK/PD)
Pharmacology (PK/PD) 22 0. 350. 700. 1050. 1400. 2-DG WP1122 Mean CNS Concentration (μg/gm) Peak CNS Concentration (μg/gm) T last (minutes) AUC (μg/gm x hr) 2 - DG CNS distribution and retention was measured after oral administration of equimolar amounts of 2 - DG and its diacetate WP1122 CNS distri
Slide 23: Orthotopic Glioblastoma Model in Mice
23 Orthotopic Glioblastoma Model in Mice WP1122 used alone has at least the same or greater activity than temozolomide (Temodar ® ), a current standard of care in patients diagnosed with glioblastoma 0 2 4 6 8 10 20 21 22 23 24 25 26 27 28 29 30 31 32 33 34 # of Animals Days Control Temodar WP1122 H
Slide 24: WP1122 Shows In Vivo Activity Against Pancreatic Cancer
WP1122 Shows In Vivo Activity Against Pancreatic Cancer - January 2018 24 Days after first administration Treatment period
Slide 25: Inhibitors of Sugar Metabolism in Cancer and Immunotherapeutic Implications
Inhibitors of Sugar Metabolism in Cancer and Immunotherapeutic Implications 25 Glycolysis Glycosylation 2 - DG 2 - Deoxyglucose or 2 - Deoxymannose?
Slide 26: WP1122 Recap and Status
WP1122 Recap and Status WP1122 is a prodrug of 2 - DG that increases circulation time and ability to cross BBB and provides other drug - like properties Utilizing Warburg principle, WP1122 converts to 2 - DG in highly glycolytic tumor cells causing autophagy due to energy starvation Evaluating oral
Slide 27: Deglycosylation of PD-L1 by 2-deoxyglucose publication abstract
‘am J Concet Res 2018 849) 1837-1846 aus /ISSN 2156 6976,/9r0083903 Original Article Deglycosylation of PD-L1 by 2-deoxyglucose reverses PARP inhibitor-induced immunosuppression in triple-negative breast cancer ‘Bin Shao, Chin Wet Li. Seung Oe Lin, inti Sun. Yun La June Hout, Chunioo Lut. Caung Wen
Slide 28: Milestones and Financials Review
Milestones and Financials Review 28
Slide 29: Anticipated Milestones
Anticipated Milestones Potential Timeframe NextGen – Anthracycline - Annamycin Initial IRB (Institutional Review Board) approvals and site initiations of various clinical sites participating in our Phase I/I I clinical trial of Annamycin Accomplished and ongoing 2019 Complete cohort of 150 mg/m2 - p
Slide 30: Financial Statement Summary
Financial Statement Summary The above financial information is summarized from the Company’s MOST CURRENT FORMS 10 - Q & 10 - K filed with the SEC ( www.sec.gov ) 30 In thousands $ - except for loss per share and shares outstanding For the Nine Months Ended 9 /30/18 For the Year Ended 12/31/17 (Unau
Slide 31: Robust pipeline
31 Robust pipeline 3 distinctly different technologies, all with blockbuster potential World - leading collaboration MD Anderson Cancer Center/Mayo Clinic/Emory Breakthrough disruptive technologies Annamycin for AML: designed to be non - cardiotoxic, avoids MDR1 WP1066: STAT3 inhibitor that also sti
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