Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (March 2019)
31 slides from Moleculin Biotech's investor presentation, Mar 18, 2019
Slide 1: Moleculin Biotech Investor Presentation, March 18, 2019
Exhibit 99.1 NASDAQ: MBRX Non-Confidential Information March 18, 2019 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 “believe,”
Slide 3: Robust pipeline
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 stimulates
Slide 4: Experienced Management Team
Experienced Management Team 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 4
Slide 5: Science Advisory Board Members
Science Advisory Board Members Waldemar Elihu Estey, Priebe, PhD MD John Paul Marty Tallman, Waymack, MD MD, SCD James Jorge Abbruzzese, Cortes, MD MD 5
Slide 6: Board Of Directors
Board Of Directors Robert E. George - Chair of Audit and Nominating & Governance Committees Mr. George joined our board of directors upon our IPO. He was a partner with the international accounting firm of PricewaterhouseCoopers (PWC) for 27 years until 2010. Mr. George currently serves as Chairman
Slide 7: Focus on Highly Resistant Tumors
Focus on Highly Resistant Tumors Example Regulatory Common Characteristics Indications Advantage • R/R AML • Multidrug resistance • Significant unmet need • Glioblastoma • Immune evasion • Modest gains = • Pancreatic • Upregulation of oncogenic new drug approval transcription factors • Met melanoma
Slide 8: Core Technologies
3 Core Technologies Next Generation Immune/Transcription Metabolism/ Anthracycline Modulator Glycosylation Inhibitor • Avoids multidrug • Enables immune • Prodrug enables resistance response drug-like properties in glucose decoy • Little to no • Inhibits p-STAT3, cardiotoxicity c-Myc and HIF-1α • Al
Slide 9: Development Pipeline
Development Pipeline Preclinical Clinical Preparation Phase 1/2 Collaboration Annamycin AML Annamycin + WP1066 WP1066 - Glioblastoma, melanoma metastasized to the brain WP1066 - Pediatric brain tumors WP1122 - Glioblastoma Brain Tumors WP1122 + Avastin - Glioblastoma WP1066/WP1732 Cancer Pancreatic
Slide 10: Technology Review
Technology Review 10
Slide 11: Annamycin (Next Generation Anthracycline)
Annamycin (Next Generation Anthracycline) Critical Leading AML induction therapy drugs are cardiotoxic and lose Advantages efficacy due to multidrug resistance over Leading Annamycin has little to no cardiotoxicity, avoids multidrug Annamycin Process Drug resistance, has been shown to be more potent
Slide 12: Annamycin Delivers Remarkable Performance in Relapsed/Refractory Acute Leukemia Patients
Annamycin Delivers Relapsed/Refractory Acute Leukemia Patients Remarkable In a proof-of-concept Phase I/II clinical trial, Performance Annamycin was given to patients who had 63% failed an average of five previous induction Efficacy therapy attempts Signal 37% of those patients cleared enough of the
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 clos
Slide 14: WP1066 Immune/Transcription Modulators
WP1066 Immune/Transcription Modulators Based On Natural Built from chemical backbone of propolis (caffeic acid STAT3 is Activated by Compound benzyl ester) Multiple Upstream Effectors Unique Dual Action First-in-class drug to both directly inhibit tumor signaling (p-STAT3, HIF-1⍺, c-Myc) while also
Slide 15: STAT3 is a Master Regulator of Tumor Progression
STAT3 is a Master Regulator of Tumor Progression Triggering oncogenic transcription activity: Activated Survival Resulting in STAT3 Proliferation Inflammation tumor growth (p-STAT3) binds Invasion and metastasis to DNA Metastasis Angiogenesis Immunosuppression 15
Slide 16: WP1066 Affects Tumors Directly and Indirectly
WP1066 Affects Tumors Directly and Indirectly WP1066 WP1066 modulates transcription factors resulting in direct tumor cytotoxicity while also Immune Transcription stimulating a natural Modulation Modulation immune response by reducing Regulatory T-cells (Tregs) Tumor 16
Slide 17: Transcriptional Control of Cancer
Transcriptional Control of Cancer Immunosuppressive Oncogenic Axis HIF-1α STAT3 c-Myc Implications for: • GBM • Pancreatic cancer • Other cancers 17
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 Tregs Currently in Phase 1 trial at MD Anderson for GBM and melanoma metasta
Slide 19: WP1122 Metabolism/Glycosylation Inhibitor
WP1122 Metabolism/Glycosylation Inhibitor Addicted to A brain tumor requires as much as 18 to 37 times as Tumors are hyper-consumers of Sugar much glucose to survive as a healthy brain cell glucose and starve to death without it Starving a Tumor This eventually led to the theory that, if we feed tum
Slide 20: Many Tumors are Highly Glycolytic
Many Tumors are Highly Glycolytic Tumors rely preferentially on glycolysis even in the presence of abundant oxygen MRI For example: PET diagnostic imaging relies on a modified glucose with a radio-tracer (F18DG) Tumors over-consume F18DG because of their dependence on glycolysis Cutting off this “fu
Slide 21: Chemistry – Improving the Drug-Like Properties of 2-DG
Chemistry – Improving the Drug-Like Properties of 2-DG and Targeting Brain Cancers 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 levels 2-DG in t
Slide 22: Pharmacology (PK/PD)
Pharmacology (PK/PD) 1400. 2-DG CNS distribution and retention was 2-DG WP1122 measured after oral administration of equimolar amounts of 2-DG and its diacetate WP1122 1050. CNS distribution and retention of 2-DG is dramatically higher when generated from WP1122 700. No observed systemic toxicity 35
Slide 23: WP1122 is Effective In Vivo against Gliomas
WP1122 is Effective In Vivo against Gliomas - July 2017 Control Temodar WP1122 10 Human brain tumors 8 injected into mice WP1122 performed as 6 well or better than 4 temazolomide # of Animals 2 Not shown is that a Orthotopic combination of both 0 performed even better 20 21 22 23 24 25 26 27 28 29 3
Slide 24: WP1122 Shows In Vivo Activity Against Pancreatic Cancer
WP1122 Shows In Vivo Activity Against Pancreatic Cancer - January 2018 Treatment period Days after first administration 24
Slide 25: Inhibitors of Sugar Metabolism in Cancer and Immunotherapeutic Implications
Inhibitors of Sugar Metabolism in Cancer and Immunotherapeutic Implications Glycolysis Glycosylation 2-DG 2-Deoxyglucose or 2-Deoxymannose? 25
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 versus
Slide 27: Deglycosylation of PD-L1 by 2-deoxyglucose (Am J Cancer Res 2018)
Am J Cancer Res 2018,8(9) 1837-1846 wnw.ajcr us /ISSN:2156-6976/ajcr0083303 Original Article Deglycosylation of PD-L1 by 2-deoxyglucose reverses PARP inhibitor-induced immunosuppression in triple-negative breast cancer Bin Shao**, Chia Wei Lit, Seung.Oe Lim**, Linlin Sun‘, Yun-Ju Lai*, Junwei Hout,
Slide 28: Milestones and Financials Review
Milestones and Financials Review 28
Slide 29: Milestones and Potential Timeframe
Milestones Potential Timeframe Next Generation Anthracyctine - Annamycin inital 1RB (Instituional Review Board) approvals and site initiations of various clinical sites participating in our Phase Il clinical tial of Annamycin ‘Accomplished and ongoing 2019 (Complete cohort of 150 mgim2 - prior tial
Slide 30: Financial Statement Summary
Financial Statement Summary In thousands $ - except for loss per share and shares For the Year Ended For the Year Ended outstanding 12/31/18 12/31/17 (Unaudited) (Audited) Statement of Operations Data Revenue - - Research and development 9,728 4,545 General and administrative and depreciation 5,297
Slide 31: Robust pipeline
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 stimulates
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