Niki BioSolutions, Inc. · NASDAQ:NIKI
Niki BioSolutions — Aptorum Group — Investor Presentation (October 2020)
28 slides from Niki BioSolutions's investor presentation, Oct 20, 2020
Slide 1: Next-Generation Rapid Pathogen Molecular Liquid Biopsy Diagnostics
Facilitating Life Science Innovations to Serve Unmet Medical Needs NASDAQ GLOBAL MARKET: APM NEXT - GENERATION RAPID PATHOGEN MOLECULAR LIQUID BIOPSY DIAGNOSTICS
Slide 2: Table of Contents
4 Executive Summary 5 One Page Summary of RPIDD 6 Technology Origins 7 Challenges Faced from Infectious Diseases 10 Economic Evaluation of RPIDD 12 RPIDD Market Overview 16 RPIDD Workflow Overview and Unique Features 21 RPIDD Prototype Clinical Sample Testing So Far 23 RPIDD AI and Software Analytic
Slide 3: Disclaimer
Disclaimer 3 © Copyright 2020 Aptorum Group Limited This document includes statements concerning Aptorum Group Limited and its future expectations, plans and prospects that constitute “forward - looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. For this
Slide 4: Infectious diseases have had a devastating global impact
Infectious diseases have experienced major development in the past two decades and have had a devastating global impact on humanity and our economy . Within the past 20 years, the world has been affected by outbreaks such as the SARS ( 2004 ), Avian flu ( 2008 ), Swine flu ( 2010 ), MERS ( 2012 ), E
Slide 5: Summary Overview of RPIDD
Summary OVERVIEW • RPIDD : Next - generation molecular based diagnostics for “unbiased" detection of any foreign pathogens (virus, bacteria, fungus, parasites) in infected patients using DNA/RNA • <24hours turn around time (improve further with NGS platforms) + cost effective • Blood sample based •
Slide 6: Technology Highlights
TECHNOLOGY HIGHLIGHTS * • Since its operation in 2009, the Molecular Engineering Lab has attracted researchers from all over the world (e.g. Stanford University, University of Oxford, Imperial College London, etc) to develop technologies that have novel applications in areas such as nucleic acid amp
Slide 7: Significant Financial Burden to Society
Significant F inancial B urden to S ociety • 7% of deaths, 8% of hospital bed days ¹ • Global infectious disease diagnostics market valued between USD16bn – 26bn in 2019, with CAGR 6.2% ⁷ Pathogens in B lood • 1 in 4 hospital patients have an infection in their blood stream and at least 1 in 2 infec
Slide 8: Diagnostic Challenges Faced from Infectious Diseases
Diagnostic Challenges Faced f rom Infectious Diseases INFECTIOUS DISEASES OF UNKNOWN CAUSES REMAIN HIGH Although hospitals have extensive laboratory testing for infectious diseases, it is estimated that etiology between 16 - 55 % of infectious disease cases remained unknown 1 Current most common cli
Slide 9: Why Are Timely Precision Medicine Tests Important For Infectious Diseases?
Why Are Timely Precision Medicine Tests Important For Infectious Diseases? Question To Provide Crucial Information For Clinicians To Initiate The Appropriate Antibiotic Therapy Answer • RPIDD is a form of Precision Medicine • Between 2008 - 2011, it is estimated that in the U.S. alone, over 70% of c
Slide 10: Improved Clinical Outcomes and Healthcare Cost Savings
Improved Clinical Outcomes i n Bloodstream Infections Substantial Healthcare Cost Savings ASP Antimicrobial Stewardship Programs COST SAVINGS Estimated to save > US$20,000 per infected patient per treatment 2 Diagnostic S trategy Average Cost (USD) ² 1 . Conventiona l method without ASP 55,932.02 2.
Slide 11: Why Is Molecular Rapid Diagnostic Testing Currently Not First-line?
But Why I s Molecular Rapid Diagnostic Testing (mRDT) Currently N ot First - line? Therefore, A Technology For A Rapid, Cost Effective, Sensitive And Unbiased Detection For ALL Types Of Pathogens Is Urgently Needed: RPIDD Emerging pathogens and known pathogens with new mutations may not be detected
Slide 12: RPIDD targets to become first-line diagnosis
RPIDD targets to become first - line diagnosis, disrupting existing pathways for the Infectious Disease Diagnostic Market PRECISION MEDICINE • Trend for precision medicine globally. Market for molecular diagnostics technique (e.g. NGS) will likely continue to expand KEY TARGET MARKETS South East Asi
Slide 13: RPIDD is integrated with NGS
RPIDD is integrated with NGS which is poised for widespread clinical adoption and will revolutionize precision medicine Drastic Drop in cost and time anticipated to create commercialization opportunities * • Single site reads • Slow, costly • Massively parallel reads • Fast, cheap DNA SEQUENCING REF
Slide 14: Blood Culture vs Next-Generation Sequencing
Blood cultures are time - consuming, labor intensive and cannot detect all infectious organisms Bacteria Virus Fungus BLOOD CULTURE STREN G THS WEAKNESSES NGS 1 Cannot detect all infectious pathogens - NOT ready for the next pandemic Takes up to 5 days for 1 culture A trial - and - error appro
Slide 15: RPIDD Device Targets to Improve Sensitivity
RPIDD device can efficiently and selectively deplete human background DNA (subject to ongoing clinical validation)* Improved Sensitivity = Lower Sequencing Cost Less reads will be required to give detectable signal Target to achieve >99.99% specificity and >95% sensitivity , subject to ongoing clini
Slide 16: RPIDD Device Workflow Overview
Proprietary method in DNA/RNA extraction P R OPRI E T A R Y DIAGNOSIS WORKF L OW ( 24 HOURS ) Biofluid collection • ~0.5ml of blood is used in the current generation of method 1 • One - pot DNA & RNA library preparation • Both targeted and untargeted workflows Sample extraction • Host DNA depletion
Slide 17: RPIDD Device: Untargeted Workflow Overview
Untargeted sequencing enables the detection of , in principle, majority DNA & RNA - based organisms * Biofluid collection DNA Sequencing DNA Library preparation & barcoding DNA Fragmentation One - pot Unbiased Amplification Nucleic Acid Purification Host Depletion: Both host DNA and RNA will be dele
Slide 18: Unique Features: Host DNA/RNA Depletion
HIGH SPIKE - IN LOW SPIKE - IN Controls : Zymo microbial standards, lentivirus and HCV in human plasma • Left untreated or treated with Zymo Host - Zero* or our proprietary method host depletion • DNA / RNA sequenced in similar fashion and reads mapped to microbes / viruses • Remainder mapped to hum
Slide 19: Unique Features: One-pot DNA & RNA Library Preparation
A DNA library prepared by our proprietary fragmentation method. The distribution sizes become progressively clustered around the 300 - 500nt size suitable for Illumina sequencing with increasing amount of different amount of methylated C (mC) incorporated * DNA FRAGMENTATION METHOD 1. Our Proprietar
Slide 20: Analytical Sensitivity and Specificity
1.25 copies of DNA/RNA per µl plasma Sensitivity Controls: ZymoBIOMICS Microbial Community Standard, Lentivirus and Seracare AccuSpan recombinant virus Specificity RPIDD device detected organisms ranging from bacteria, RNA virus and fungi in ONE TEST 1 • 8 species of bacteria, • 2 species of RNA vir
Slide 21: Patient Clinical Sample #1 (Singapore)
Additional mic r oo r ganisms were also identified Patient Clinical Sample #1 (Singapore) SAMPLE PROCESSED AT NUH WITH OUR PROTOCOLS AND REAGENTS 1 • Banked sample with known Hepatitis B infection • Hepatitis B, a DNA virus was successfully identified 21 © Copyright 2020 Aptorum Group Limited 1. The
Slide 22: Patient Clinical Sample #2 (Singapore)
• Undergoing chemotherapy with severe lung infection • Refractory to first - line antibiotic • Eventually responded to a combination of 2nd line antibiotics and antifungal medication • Leuconostoc, a Gram+ bacteria was identified by RPIDD device , which was not previously considered by clinicians •
Slide 23: Future Development for Report Generation
• Part of Clinical validation with hospitals is also to profile healthy or background samples so that we can identify the background microbial sequences in samples from specific hospitals or are part of the “kitnome” that we can filter out from our report • Public databases for different pathogens w
Slide 24: RPIDD Software Analytics and AI Architecture
24 © Copyright 2020 Aptorum Group Limited RPIDD Software Analytics and Artificial Intelligence Architecture Input read QC step Human Depletion Step Custom QC metrics derivation Pathogen read assignment step Non - human read assemblies Antibiotic Resistance step Machine Learning level Feedback Layer
Slide 25: Projected RPIDD Summary Business Plan
• Laboratory Device Test model: Providing RPIDD device and workflow as a private pathological laboratory service integrated inside private hospitals and clinics, targeting majority in - patient market • Build out of self - controlled diagnostics laboratory (ISO standard) to provide accredited diagno
Slide 26: Conclusion
C o n c l u s i on • When dealing with an individual patient with an infectious disease or responding to a worldwide pandemic (such as SARS, COVID19, MERS etc), it is fundamental to provide quality care and treatment through the rapid and accurate identification of microbial s . • Despite advances i
Slide 27: Appendix: Bloodstream and Non-Bloodstream Infections Impact
Appendix: Bloodstream and Non - Bloodstream Infections Impact 27 © Copyright 2020 Aptorum Group Limited Can be Bloodstream or Non - Bloodstream Infections Respiratory Diseases Sexually Transmitted and Blood - borne Infections Sepsis Influenza Tuberculosis (“TB”) 8,9 Hepatitis B and C Description / R
Slide 28: Aptorum Group Contact
APTORUM GROUP [email protected] T +44 20 80929299 F +44 20 39288277