Moleculin Biotech, Inc. · NASDAQ:MBRX
Moleculin Biotech — Investor Presentation (May 2019)
37 slides from Moleculin Biotech's investor presentation, May 31, 2019
Slide 1: Developing breakthrough therapies for highly resistant cancers
Developing breakthrough therapies for highly resistant cancers May 2019 Corporate Presentation (Nasdaq: MBRX) 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: Moleculin Biotech is a clinical stage pharmaceutical company
Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on developing breakthrough treatments for highly resistant cancers. These highly resistant cancers include: • deadly glioblastoma brain tumors • acute myeloid leukemia • pancreatic cancer • cutaneous T-cell lymphoma (a deadly
Slide 4: Core Technologies
3 Core Technologies 1 2 3 Next Generation Immune / Transcription Metabolism / Anthracycline Modulator Glycosylation Inhibitor • Avoids multidrug resistance • Enables immune response • Prodrug enables drug-like properties in glucose decoy • Little to no cardiotoxicity • Inhibits p-STAT3, c-Myc and HI
Slide 5: Focus on Highly Resistant Tumors
Focus on Highly Resistant Tumors Example Common Regulatory Indications Characteristics Advantage • R/R AML • Multidrug resistance • Significant unmet need • Glioblastoma • Immune evasion • Modest gains = new • Pancreatic • Upregulation of oncogenic drug approval transcription factors • Met melanoma
Slide 6: Partnerships & Collaborations
Partnerships & Collaborations Our world-leading partnerships & collaborations significantly expand our research capabilities and increase our knowledgebase 6
Slide 7: 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 8: Key Developments
Key Developments Moleculin has three drug candidates in four clinical trials. Next Generation Anthracycline - Annamycin Immune/Transcription Modulators - WP1066 Portfolio • Positive interim safety and efficacy data from Annamycin trial in • Emory University to conduct pediatric brain tumor trial. Po
Slide 9: Milestones & Accomplishments
Milestones & Accomplishments Anticipated Milestones Potential Timeframe Next Generation Anthracycline - Annamycin Initial IRB (Institutional Review Board) approvals and site initiations of various clinical sites participating in our Phase 1/2 clinical trial of Annamycin Accomplished and ongoing 2019
Slide 10: Technology Review
Technology Review 10
Slide 11: Annamycin (Next Generation Anthracycline)
Annamycin (Next Generation Anthracycline) Critical Advantages Leading AML induction therapy drugs are cardiotoxic and lose efficacy due to Annamycin Process over Leading Drug multidrug resistance Annamycin has little to no cardiotoxicity, avoids multidrug resistance, has been shown to be more potent
Slide 12: Annamycin Delivers Remarkable Performance in Relapsed/Refractory Acute Leukemia
Annamycin Delivers Relapsed/Refractory Acute Leukemia Remarkable In a proof-of-concept Phase I/II clinical trial, Annamycin was Patients Performance given to patients who had failed an average of five previous induction therapy attempts 63% Efficacy 37% of those patients cleared enough of their leuk
Slide 13: Annamycin Recap & Status
Annamycin Recap & 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
Slide 14: WP1066 (Immune / Transcription Modulators)
WP1066 (Immune / Transcription Modulators) Based On Natural Built from chemical backbone of propolis (caffeic acid benzyl STAT3 is Activated by Multiple Upstream Compound ester) Effectors Unique Dual Action First-in-class drug to both directly inhibit tumor signaling (p-STAT3, HIF-1⍺, c-Myc) while a
Slide 15: STAT3 is a Master Regulator of Tumor Progression
STAT3 is a Master Regulator of Tumor Progression Triggering oncogenic transcription activity: • Survival Activated STAT3 • Proliferation Resulting in tumor (p-STAT3) binds to • Inflammation growth and DNA • Invasion metastasis • Metastasis • Angiogenesis • Immunosuppression 15
Slide 16: WP1066 Affects Tumors Directly & Indirectly
WP1066 Affects Tumors Directly & 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 & Status
WP1066 Recap & 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 met
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 Tumors are hyper-consumers survive as a healthy brain cell of glucose and starve to death without it Starving a Tumor to This eventually led to the theory that, if we fee
Slide 20: Many Tumors are Highly Glycolytic
Many Tumors are Highly Glycolytic • Tumors rely preferentially on glycolysis even in the presence of abundant oxygen • For example: PET diagnostic imaging relies on a modified MRI glucose with a radio-tracer (F18DG) • Tumors over-consume F18DG because of their dependence on glycolysis • Cutting off
Slide 21: Chemistry
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 th
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 1050. diacetate WP1122 • CNS distribution and retention of 2-DG is dramatically higher when generated from 700. WP1122 • No observed systemic toxic
Slide 23: WP1122 is Effective In Vivo against Gliomas
WP1122 is Effective In Vivo against Gliomas July 2017 WP1122 used alone has at least the same or greater activity than temozolomide (Temodar®), a current standard of care in patients diagnosed with glioblastoma Control Temodar WP1122 10 • Human brain tumors 8 injected into mice • WP1122 performed as
Slide 24: WP1122 Shows In Vivo Activity Against Pancreatic Cancer
WP1122 Shows In Vivo Activity Against Pancreatic Cancer January 2018 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 & Status
WP1122 Recap & 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 ve
Slide 27: American Journal of Cancer Research
American Journal of Cancer Research 27
Slide 28: Financial Review
Financial Review
Slide 29: Financial Statement Summary
Financial Statement Summary For the Quarter Ended For the Year Ended In thousands, except for share and per share data and shares outstanding 03/31/19 12/31/18 (Unaudited) (Unaudited) Statement of Operations Data Revenue - - Research and development $2,932 $9,728 General and administrative and depre
Slide 30: Highly Experienced Leadership
Highly Experienced Leadership
Slide 31: Executive Management Team
Executive 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 31
Slide 32: Science Advisory Board
Science Advisory Board Waldemar Elihu Estey, Priebe, PhD MD John Paul Waymack, Marty MD, SCD Tallman, MD Jorge Cortes, James Abbruzzese, MD MD 32
Slide 33: 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 34: Partnerships & Collaborations
Partnerships & Collaborations The University of Texas MD Anderson Cancer Center Moleculin actively sponsors ongoing research at The University of Texas MD Anderson Cancer Center. MD Anderson is the largest cancer research center in the world and one of the country’s largest recipients of NIH medical
Slide 35: Partnerships & Collaborations
Partnerships & Collaborations Medical University of Gdansk The Medical University of Gdańsk (MUG) is the largest medical university in northern Poland, located in one of the most beautiful cities in Europe with an old town and beautiful sandy beaches. The MUG educates nearly 6,000 undergraduate and
Slide 36: Key Take-aways
3 6 Key Take-aways 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 t
Slide 37: Developing breakthrough therapies for highly resistant cancers
Developing breakthrough therapies for highly resistant cancers Walter Klemp, CEO / Jonathan Foster, CFO info@moleculin.com (713) 300-5160 (Nasdaq: MBRX) 3 7