Kaleido Biosciences, Inc. · OTC:KLDO
Kaleido Biosciences — Investor Presentation (April 2019)
31 slides from Kaleido Biosciences's investor presentation, Apr 8, 2019
Slide 1: Company Overview April 2019
APRIL 2019 Company Overview Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the anticipated use of our existing cash resources, including proceeds from our
Slide 3: The 1st Company to Use a Chemistry-Driven Approach to Drive Microbiome Function
The 1st Company to Use a Chemistry-Driven Approach Intended to Systematically Drive FUNCTIONAL OUTPUTS OF THE MICROBIOME ORGAN Translating the Promise of the Microbiome Into Solutions for Patients
Slide 4: The Kaleido Opportunity
The Kaleido Opportunity Proprietary Product Platform Broad Pipeline with Large Market Opportunity Faster, More Cost Efficient Model Significant Computational Biology and Data Science Capabilities Enable Kaleido to Learn MORE, and Learn FASTER Human-Centric Discovery & Development World-Class Leaders
Slide 5: Built A World-Class Leadership Team
Built A World-Class Leadership Team Alison Lawton President & Chief Executive Officer Former GM, Genzyme Biosurgery, COO Aura & OvaScience Josh Brumm Chief Operating Officer, Chief Financial Officer Former COO & CFO, Versartis, EVP Finance, Pharmacyclics & CFO, ZELTIQ Kate Knobil, M.D. Chief Medical
Slide 6: Founded Company in 2015 to Create a New Category of Drugs and Health Products
Founded Company in 2015 to Create a New Category of Drugs and Health Products Significant Accomplishments Since Founding 1,000+ MMT candidates in our library today IND Cleared, Phase 2 clinical trial initiation planned for 1H 2019 7 non-IND human clinical studies conducted with our Microbiome Metabo
Slide 7: Pipeline Programs
PROGRAM Target Mechanism of Action Ex Vivo Screening Ex Vivo Testing Healthy Volunteers Patients Potential IND Filing2 Phase 13 Phase 2 Phase 3 Hyperammonemia: Urea Cycle Disorders (KB195) Ammonia N/A4 1H’19 Hyperammonemia: Hepatic Encephalopathy (KB174 or KB195) Ammonia 1H’20 2H’20 Infections Cause
Slide 8: The Microbiome Organ Has the Potential to Address a Broad Range of Diseases
The Microbiome Organ Has the Potential to Address a Broad Range of Diseases The Microbiome is Implicated in Numerous Conditions Obesity Type 2 Diabetes Parkinson’s Disease Short-Chain Fatty Acids (SCFAs) Urea Cycle Disorders Hepatic Encephalopathy Nitrogen Metabolism Chronic Kidney Disease Organic A
Slide 9: We're Leveraging Years of Scientific Insights and Knowledge
We’re Leveraging 10 Years of Scientific Insights and Knowledge Computational Capabilities Automation & Miniaturization Rapid, human-relevant screening Powerful Enabling Tools Are Available Now Is the Time to Develop Targeted Therapies for the Microbiome Organ Shallow Shotgun Sequencing 16S RNA Seque
Slide 10: We Have a Unique and More Evolved Approach
FOCUS KNOWLEDGE OVER TIME Functions of the Microbiome Composition of the Microbiome Subtract Bacteria “Kill the bugs” (e.g. Antibiotics) Add Bacteria “Bugs as drugs” (e.g. fecal microbiota transplants, bacterial therapy) Drive Existing Bacteria Microbiome Metabolic Therapies, or MMTs (i.e. targeted
Slide 11: Our Microbiome Metabolic Therapies (MMT) Are Proprietary, Novel Compounds
Our Microbiome Metabolic Therapies (MMT™) Are Proprietary, Novel Compounds CHARACTERISTICS ADVANTAGES Initial MMTs are targeted glycans Drivers of gut metabolism, long history of safe human consumption Related to a class of compounds that is Generally Recognized As Safe (GRAS) Ability to advance rap
Slide 12: Our MMTs Are Designed to Work Through One or More Mechanisms of Action
Our MMTs Are Designed to Work Through One or More Mechanisms of Action, Including ... DECREASE Production of Metabolites INCREASE Production of Metabolites ADVANTAGE or DISADVANTAGE Certain Species in the Microbiome Community
Slide 13: Human-Centric Discovery & Development
Human-Centric Discovery & Development EX VIVO SCREENING IN HEALTHY MICROBIOME SAMPLES 1 EX VIVO TESTING IN PATIENT MICROBIOME SAMPLES 2 RAPID ADVANCEMENT INTO NON-IND HUMAN CLINICAL STUDIES 3 Patients Healthy Volunteers & Data Collected at Every Stage to Leverage Computation Capability Human Data Dr
Slide 14: Faster, More Cost Efficient Model
Faster, More Cost Efficient Model Note(s): 1. To date with respect to KB195, which we have decided to develop as a drug candidate 2. DiMasi, Joseph, et. al: Innovation in the pharmaceutical industry: New estimates of R&D costs, Journal of Health Economics, 2016, 47, 20-23. Year 1 Year 2 Year 3 Year
Slide 15: Pipeline Programs
PROGRAM Target Mechanism of Action Ex Vivo Screening Ex Vivo Testing Healthy Volunteers Patients Potential IND Filing2 Phase 13 Phase 2 Phase 3 Hyperammonemia: Urea Cycle Disorders (KB195) Ammonia N/A4 1H’19 Hyperammonemia: Hepatic Encephalopathy (KB174 or KB195) Ammonia 1H’20 2H’20 Infections Cause
Slide 16: The Hyperammonemia Opportunity
BRAIN Main Site of Ammonia Toxicity GUT Ammonia Production LIVER Main Site of Ammonia Clearance The Hyperammonemia Opportunity Sources: Batshaw, M. L., et. al. (2014). A longitudinal study of urea cycle disorders, Molecular Genetics and Metabolism, 113(0), 127-130; Montgomery, J. Y., & Bajaj, J. S.
Slide 17: Ex Vivo Screening: KB195 Reduced Ammonia in Healthy Microbiomes
Ex Vivo Screening: KB195 Reduced Ammonia in Healthy Microbiomes Ability to Screen Many MMTs Across Our Compound Library for Ability to Reduce Ammonia Compound Ammonia Levels Normalized to Control 1 KB195 Lead MMT Reduced Ammonia by 97%, Compared to Control (water) 97% Control Ammonia Levels Normaliz
Slide 18: Ex Vivo Testing: KB195 Reduced Ammonia in Patient Microbiomes
2 KB195 in Hepatically Impaired Patients Ex Vivo Testing: KB195 Reduced Ammonia in Patient Microbiomes KB195 in UCD Patients Reduced Ammonia in All Microbiome Samples Reduced Ammonia by > 50% in 10 Out of 12 Samples Reduced Ammonia in 18 of 19 Microbiome Samples Outperformed Lactulose in Reducing Am
Slide 19: Non-IND Human Clinical Studies: KB195
3 Randomized, controlled, double-blind study in healthy subjects on a high protein diet KB195 Significantly Lowered Nitrogen 40.5% Decrease in Urinary 15N Excretion Compared to Negative Control (p=0.0126) Non-IND Human Clinical Studies: KB195 Note(s): 1. De Preter V. et al., Alimentary Pharmacology
Slide 20: Non-IND Human Clinical Studies: KB195 Fewer Subjects Reported Diarrhea
Non-IND Human Clinical Studies: KB195 Fewer Subjects Receiving KB195 Reported Diarrhea3 1 3 18g/day 1 4 36g/day 1 6 54g/day 4 10 72g/day 1 1 3 4 Negative control KB195 Comparator glycan Dosing Period Lactulose ~20% reduction1 Urine 15N Excreted; % Change from Baseline Results from 72 g/day dose KB19
Slide 21: Our Additional Programs Focus on Multiple Indications
Our Additional Programs Focus on Multiple Indications DECREASE Production of Metabolites TMA Atherosclerotic Cardiovascular Disease (ASCVD) No Marketed Prescription Treatments That Address Elevated Levels of TMAO in Treatment of ASCVD Uremic Toxins Chronic Kidney Disease Significant Prevalence & Cos
Slide 22: Ex Vivo Testing: MMTs Observed to Reduce Pathogens
Ex Vivo Testing Our MMTs Observed to Reduce Pathogens by a Median of 45% Relative to Control in 10 of 13 Samples Microbiome Samples from 13 ICU Patients Pathogen Reduction Relative to Control (%) Hours % Pathogen MMTs for Infections Caused by Multi-Drug Resistant Bacteria Ex Vivo Screening Our MMTs
Slide 23: Ex Vivo Screening: Top MMTs Reduced TMA in Healthy Microbiomes
Ex Vivo Screening: Top MMTs Reduced TMA in Healthy Microbiomes Our top MMTs reduced TMA by >90% in Ex Vivo Screening Compared To Control Conducted high-throughput MMT screening to identify hits Top leads will be tested ex vivo in microbiomes of patients with cardiovascular disease Following ex vivo
Slide 24: Lead Programs in Hyperammonemia Demonstrate the Power of Our Platform
Lead Programs in Hyperammonemia Demonstrate the Power of Our Proprietary Product Platform Note(s): 1. As evidenced by lowered urinary15N excretion in the non-IND clinical study of KB195 in healthy subjects Pathologies that Result in an Excess of Ammonia in the Blood Decrease Production of Ammonia Si
Slide 25: Large Market Opportunity Across Diseases
Large Market Opportunity Across Diseases UCD HE ~3,000 patients in the U.S. Current treatment (Ravicti) costs $800K/year in the U.S. ~40% of patients on Ravicti have high ammonia levels and remain at risk ~500K patients in the U.S., not all of whom have been diagnosed (100K OHE, 400K MHE) Only 1/3rd
Slide 26: High Quality, Consistent, and Scalable Manufacturing in Place
High Quality, Consistent, and Scalable Manufacturing in Place Internal Manufacturing Production for ex vivo, toxicology, and human clinical studies 3rd Party Manufacturers Production of larger quantities of our MMT candidates for clinical trials and future commercial supply (Thermo Fisher Scientific
Slide 27: Robust Global IP Portfolio
Robust Global IP Portfolio 8 U.S. Patents + 2 EPO Patents: Glycan pharmaceutical compositions (Europe) Methods to reduce ammonia (U.S.) Methods to treat diarrhea (U.S.) Methods to treat dysbiosis (U.S.) Catalyst compositions (U.S. and Europe) 100+ Non-Provisional Applications Pending, Worldwide in 2
Slide 28: Financial Overview
Financial Overview1 $76M (as of 12/31/18) $54M (for the year ended 12/31/18) $15M (as of 12/31/18) Cash and Cash Equivalents Operating Expenses (2) Long Term Debt Note(s): 1. Numbers are rounded to nearest million 2. Excludes stock-based compensation
Slide 29: Executing Towards our Key Milestones
Executing Towards our Key Milestones UCD HE Infections Caused by MDR Bacteria TMA Non-IND clinical study in patients Non-IND clinical study in patients with cirrhosis Non-IND clinical study Initiate non-IND clinical study 2019 1H 2H 2020 1H 2H Phase 2 clinical trial Data expected Non-IND clinical st
Slide 30: The Kaleido Opportunity
The Kaleido Opportunity Expanding number of proprietary MMTs, IP, computational capabilities Multiple indications and MOAs targeting unmet patient needs in significant markets; non-IND human clinical studies completed and ongoing Demonstrated speed and cost efficiency to the clinic Enables rapid adv
Slide 31: @kKaleido | ©2019 KALE
@kKaleido 31 | ©2019 KALE
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