Niki BioSolutions, Inc. · NASDAQ:NIKI
Niki BioSolutions — Aptorum Group — Investor Presentation (June 2020)
33 slides from Niki BioSolutions's investor presentation, Jun 29, 2020
Slide 1: Corporate Presentation
Facilitating Life Science Innovations to Serve Unmet Medical Needs CORPORATE PRESENTATION NASDAQ GLOBAL MARKET: APM
Slide 2: Disclaimer
Disclaimer 2 For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome © Copyright 2020 Aptorum Group Limited This document includes statements concerning Aptorum Group Limited and its future expectations, plans and prospects that constitute “f
Slide 3: About Aptorum Group
About Aptorum Group • Established in 2010, focused on current unmet medical needs, including orphan diseases, infectious diseases, metabolic diseases and women’s health, over 15 therapeutic candidates • Business Strategy: From Discovery to Ph2 Proof - of - Concept (PoC) • Markets and Regulatory: Tar
Slide 4: Aptorum Management and Directors
Aptorum Management and Directors • Over 15 years in global asset management; • US healthcare equity research analyst at Janus Henderson Group; • Trustee board member of Dr. Stanley Ho Medical Development Foundation; • CFA, Princeton University, U.S. (Econ) Leadership MR. IAN HUEN Founder, Chief Exec
Slide 5: Aptorum Team
Aptorum Team Clinical Advisor DR. NISHANT AGRAWAL Senior Clinical Advisor - ATL DR. HENRY CHAN LIK YUEN Senior Clinical Advisor - ATL DR. PHILIP W.Y. CHIU Senior Advisor - ATL DR. VINCENT MOK CHUN TONG Senior Advisor - ATL • Professor of Surgery, School of Medicine, University • Associate Dean, Facu
Slide 6: Current progress of leading pipeline programs and discovery
Current progress of leading pipeline programs and discovery 1. Refers to the drug’s existing Phase I/II safety data previously conducted by a third party. Does not refer to clinical trials conducted by Aptorum 2. Subject to FDA’s approval 6 For illustrative purposes only. There is no guarantee of an
Slide 7: Current progress of leading pipeline programs and discovery
Current progress of leading pipeline programs and discovery 7 For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome © Copyright 2020 Aptorum Group Limited
Slide 8: SMART-ACT Drug Discovery Platform: Orphan Disease Focus and Selection
SMART - ACT ™ Drug Discovery Platform: Orphan Disease Focus and Selection Life threatening disease High unmet need IP protection Market size Competitive landscape Clinical trial design Paediatric disease By region Target selection Disease knowledge Disease selection criteria High p ri o rity • US: <
Slide 9: SMART-ACT: Pipeline Workflow
SMART - ACT ™: Pipeline Workflow 2,600 FDA - approved small molecule drugs New drug indication • Life threatening disease • Lack of effective treatment • Large market size 1 Drug target selection • Computational mining from literature • Up to 5 disease drug targets selected 2 3 In vitro validation •
Slide 10: SACT-1 (neuroblastoma): market overview
SACT - 1 (neuroblastoma): market overview SACT - 1 targets neuroblastoma, a cancer that develops from nerve cells Prevalence • ~700 cases of high risk neuroblastoma (NB) patients each year in the US 3 and we estimated EU has 1.5x those cases, c. 1050 high risk NB patients per year. • Accounts for ~1
Slide 11: In vitro drug activity against neuroblastoma cell lines
In vitro drug activity against neuroblastoma cell lines • SACT - 1’s potential action against neuroblastoma might be patentable • We find that its action against neuroblastoma could be patentable The results shown in this slide are based on Aptorum's internal (in vitro/in vivo) tests/experiments tha
Slide 12: Synergistic effect of SACT-1 in combination with standard treatment
Synergistic effect of SACT - 1 in combination with standard treatment • Synergistic effect observed for SACT - 1 in combination with standard treatment in 2 different neuroblastoma cell lines, as seen in the isobologram (left) and the Excess over Bliss (right) 14 13 1 1 18 21 2 0 0.37 10 0.2 0.4 Sta
Slide 13: SACT-1: safety & tolerability
SACT - 1: safety & tolerability 13 For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome © Copyright 2020 Aptorum Group Limited Well - established Safety profile based on a FDA approved product • Did not show genotoxic potential even at the
Slide 14: Executive summary: Acticule projects
Executive summary: Acticule projects • Aptorum's lead program ALS - 4 is an anti - virulent, non - bactericidal drug candidate for Staphylococcus aureus infections including MRSA 1 • Unlike all major treatments on the market, ALS - 4 is an orally administered anti - virulent molecule using a non - b
Slide 15: ALS-4: Market Overview
ALS - 4: Market Overview ALS - 4 is an anti - virulent, non - bactericidal drug candidate for Staphylococcus aureus infections including MRSA 1. Clin Microbiol Rev. 2012 Apr;25(2):362 - 86; 2. "Methicillin - resistant Staphylococcus Aureus (MRSA) Drugs Market - Global Industry Analysis, Size, Share,
Slide 16: Market Approved Drugs for MRSA Infections
Market Approved Drugs for MRSA Infections 16 For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome © Copyright 2020 Aptorum Group Limited Frequently prescribed antibiotics for MRSA infections 1 Product (Compa n y ) Antibiotic Class Indicati
Slide 17: ALS-4: Addressing the Shortfall of Vancomycin
ALS - 4: Addressing the Shortfall of Vancomycin 1. “Companies Take Aim at MRSA Infections” P T. 2016 Feb; 41(2): 126 – 128; 2. Clin Infect Dis. 2011 Feb 1;52(3):e18 - 55; 3. Clin Infect Dis. 2006 Jan 1;42 Suppl 1:S5 - 12; 4. Antimicrob Agents Chemother. 2008 Jan;52(1):192 - 7; 5. Clin Infect Dis. 20
Slide 18: ALS-4: Value Proposition
Antibiotics ALS - 4: Value Proposition • Antibiotic resistance in S. aureus has been discovered in most prescribed antibiotics for MRSA 1 • Broad spectrum and indiscriminate 2 • Commonly affect normal flora, may lead to superinfection in case of drug resistance 3 Indiscriminate clearance 1. Refer to
Slide 19: Mechanism of Action: Staphyloxanthin of Staphylococcus aureus
Mechanism of Action: Staphyloxanthin of Staphylococcus aureus The above diagram summarizes the mechanism of action by Staphyloxanthin of Staphylococcus aureus: • Neutrophils kill bacteria including Staphylococcus aureus intracellularly or extracellularly via Reactive Oxygen Species “ROS - oxygen rad
Slide 20: Mechanism of Action - ALS-4 on Staphyloxanthin Synthesis
Mechanism of Action - ALS - 4 on Staphyloxathin Synthesis The description of ALS - 4 and related conclusory statements on ALS - 4 on this slide are based on Aptorum’s internal tests/experimentation and has not yet been verified by clinical trials or third party testing. The above diagram summarizes
Slide 21: ALS-4: oral formulation treatment in an MRSA survival study
ALS - 4: oral formulation treatment in an MRSA survival study The combination of ALS - 4’s anti - virulence properties together with host immune system, efficacy is still superior. The below in - vivo data includes rats infected with a lethal dose of MRSA USA300 in a bacteremia model. Survival (%) 2
Slide 22: ALS-4: oral formulation treatment in a non-lethal bacteremia model
ALS - 4: oral formulation treatment in a non - lethal bacteremia model • Rats were challenged with a non - lethal dose (10 7 CFU) of MRSA through the tail vein • In order to simulate a more realistic clinical scenario, treatment was introduced 14 - days after infection, where ALS - 4 was administere
Slide 23: ALS-1: Targeting a Novel Druggable Target for Influenza A
ALS - 1: Targeting a Novel Druggable Target for Influenza A ALS - 1 inhibits influenza A nucleoprotein (NP) • NP is the most abundantly expressed protein during the course of an infection 1 . Its primary function is to encapsidate the virus genome for RNA transcription, replication and packaging. It
Slide 24: Claves Executive Summary
Claves Executive Summary Human Microbiota • We live in constant symbiosis with our gut bacteria, and dysbiosis can be the cause to numerous diseases 1 Claves Technology • The Claves technology is designed to physically modulate the chemical signaling of diseases - causing microbiota 2 • Highly scala
Slide 25: Claves Platform and CLS-1: Value Proposition
Claves Platform and CLS - 1: Value Proposition C L S - 1 • Identified specific microbiota metabolite linked to obesity • Novel therapeutic that physically modulates microbiota metabolite • Acts locally in the gut with high affinity and specificity • Non - absorbable and is expected to be free from a
Slide 26: Mechanism of Action
Mechanism of Action Metabolite 1 produced by the microbiome and its link to obesity • High - fat diet in rats increased levels of metabolite 1 in the GI tract • High metabolite 1 turnover promotes obesity and metabolic syndrome • Metabolite 1 drives via a number of pathways to cause increased insuli
Slide 27: CLS-1: Market Overview
CLS - 1: Market Overview Recent Deals in Obesity Treatment • Boehringer Ingelheim committed up to USD 300m to work with Gubra on obesity treatments Competing Drugs • CLS - 1 is a drug candidate for obesity treatment that achieves its effect by modulating the chemical signaling of gut microbiota. The
Slide 28: CLS-1: Efficacy in a Mouse Model
CLS - 1: Efficacy in a Mouse Model CLS - 1 treatment significantly reduces body weight in mice The above data are based on Aptorum's internal tests and has not yet been verified by clinical trials or third party testing. 28 For illustrative purposes only. There is no guarantee of any project being c
Slide 29: NLS-2: Executive Summary
NLS - 2: Executive Summary NLS - 2 1 • NLS - 2 is a dietary supplement for the relief of menopausal symptoms. • The bioactive component of NLS - 2 is DOI, a novel non - hormonal compound extracted from Chinese Yam • DOI significantly increased estradiol biosynthesis and aromatase expression in granu
Slide 30: Appendix
APPENDIX
Slide 31: Income Statement Summary (U.S. GAAP)
Income Statement Summary (U.S. GAAP) 1 Notes: 1. The following slide contains selected information for the Company's income statement. Please see the Company's most recently file d F orm 20 - F for the Company's complete financial statements. 2. During the year ended December 31, 2019 and 2018, the
Slide 32: Selected Balance Sheet Items (U.S. GAAP)
Selected Balance Sheet Items (U.S. GAAP) 1 Notes: 1. The following slide contains selected information for the Company's balance sheets. Please see the Company's most recently filed form 20 - F for the Company's complete financial statements. 2. Current assets less current liabilities. 3. Since Sept
Slide 33: T +852 3953 7700 F +852 3953 7711
T +852 3953 7700 F +852 3953 7711