Niki BioSolutions, Inc. · NASDAQ:NIKI
Niki BioSolutions — Aptorum Group — Investor Presentation (January 2021)
24 slides from Niki BioSolutions's investor presentation, Jan 22, 2021
Slide 1: Corporate Presentation
APTORUM= Facilitating Life Science Innovations to Serve Unmet Medical Needs CORPORATE PRESENTATION NASDAQ GLOBAL MARKET: APM
Slide 2: Forward-looking statements disclaimer
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 purpose, any statements contained herein that are not
Slide 3: About Aptorum
About Aptorum up Company Information ¢ Established in 2010, Aptorum focuses on current unmet medical needs, including orphan diseases, infectious diseases, metabolic diseases and women’s health, with over 15 therapeutic candidates * Business Strategy: from Discovery to Phase II Proof-of-Concept (PoC
Slide 4: Directors, Management and Significant Employees
Directors, Management and Significant Employees Leadership MR. IAN HUEN, MR. DARREN LUI DR-ICLARIC CHENG! Founder, Chief Executive Officer and Executive President and Executive Director cher Medical Officer and Executive Director » & < + Over 15 years in global asset management; + Over 13 years in g
Slide 5: Aptorum Team: Consultants and Advisors
Aptorum Team Consultants and Advisors to Aptorum Group and Subsidiaries DR. KEITH CHAN Consultant a Adjunct professor and advisor at the Research Center for Drug Discovery, National Yang Ming University in Taipei; Former Division Director of Office of Generic Drugs, US FDA; Co-founder of Globomax LL
Slide 6: Pipeline Programs and Discovery
ine Programs and Discovery Lead Projects + Other Candidates > Non-therapeutics Candidates Therapeutics Development Stage IND (orIND | Clinical Trial Indication Target 4) Identification & | Lead Discovery) urvat equivalent) | Application Phase l/l ction ptimization | “Enabling Submission Projects Can
Slide 7: Executive Summary: ALS-4 Project and Market Size
ALS-4 is an ti-virul Executive Summary: Acticule Projects Market Size 30” Mortality rate of patients with S. aureus bacteremia? ALS-4 i US$2.9bn Value of the global RSA drugs market 20167 Jal drug candidate for Staphylococcus aureus infections including MRSA 2 % e CAGR global MRSA rugs market (2017-
Slide 8: ALS-4: Addressing the Shortfall of Vancomycin
ALS-4: Addressing the Shortfall of Vancomycin * Generic antibiotic that is the most frequently prescribed for MRSA-suspected infections? * After >60 years? of clinical use, its use against S. aureus is becoming limited. Vancomycin has been shown to have slow bactericidal activity, poor anti-staphylo
Slide 9: Mechanism of Action: Staphyloxanthin Inhibition
Mechanism of Actio Staphyloxat DMSO control ALS-4 200nM 100nM 50nM 25nM The above diagram summarizes our findings about how ALS-4 inhibits Staphyloxathin synthesis: 12.5nM + ALS-4 inhibits a key enzyme in the biosynthesis of Staphyloxanthin with an IC., = 20nM. 6.3nM * — Inthe absence of Staphyloxan
Slide 10: 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. 100 =@® Vehicle (N=9)
Slide 11: ALS-4: Oral Formulation Treatment in a Non-Lethal Bacteremia Model
ALS-4: Oral Formulation Treatment in a Non-Lethal Bacteremia Model ALS-4 is shown to greatly reduce organ bacterial count in a bacteremia animal model. 8 Py 1048 = 63,096 CFU/g Kidney bacterial count (CFU/g) (log,, scale) 4 99.5%reduction 1025 = 316 CFU/g Control ALS-4 (N=8) (10mg/kg) (N=8) * Rats w
Slide 12: Efficacy of ALS-4 in Combination with Vancomycin in Mouse Model
ly of ALS-4 in Combination of Vancomycin in a Mouse Model Infected with MRSA USA 300 Immediate Treatment Post Lethal Dose Delayed Treatment 20% bodyweight loss as HEP ; > Vehicle 100. -e Vehicle 100 = LM32 3 -—® LM32 § V = ~ § ~~ Van B an + 5 * se Lv324Van 3 oie 7) ¥ 50 * + 50. c c o o ° * Statistic
Slide 13: ALS-4: Oral Administration in MRSA Mouse Skin Wound Infection Model
ALS-4: Oral Administration in a MRSA Mouse Skin Wound Infection Model ALS-4 (Compound IM032) shows a statistically significant improvement in skin wound closure / healing. _~ 80 * g = £ —_———_=—_4 2 3 g 60 oS ao < 3 g 40 x} o =) = 20 8 oD a 0 Non-infection Mupirocin Linezolid IM032 control 2% Top, 1
Slide 14: SMART-ACT Drug Discovery Platform: Orphan Disease Focus and Selection
SMART-ACT® Drug Discovery Platform: Orphan Disease Focus and Selection Disease selection criteria High Life threatening disease priority High unmet need 7000+ Orphan Diseases IP protection SMART-ACT ® Patient lation definition: 7 a anent pope wm en Market size High Priority * US: <200,000 patients .
Slide 15: SMART-ACT: Pipeline Workflow
SMART-ACT® : Pipeline Workflow New drug indication Drug target selection yar = TNE 2,600 FDA-approved small molecule drugs + — Life threatening disease + Computational mining @2XD) * Lack of effective treatment from literature NAD + Large market size + Up to 5 disease drug LP) targets selected Compu
Slide 16: SACT-1: Market Overview
stoma): Market Overview SACT-1 targets neuroblastoma, a cancer that develops from nerve cells Market Size #1 uss2.6bn 34/7 Most common Value of the global CAGR global type of solid tumor neuroblastoma neuroblastoma in children? treatment market in treatment market 2017? (2019-2023)? Prevalence * ~70
Slide 17: SACT-1: In-Vivo Study and Synergistic Effect with Chemotherapy
SACT-1: In-Vivo Study and Synergistic Effect with Chemotherapy Synergistic effect observed for SACT-1 in combination with standard SACT-1 when combined with standard of care chemotherapy showed treatment in 2 different neuroblastoma cell lines, as seen in the a statistically significant reduction in
Slide 18: PIDD: Challenges Faced By Infectious Diseases
PIDD: Challenges Faced By Infectious Diseases INFEC DISEASES OF UNKNOWN CA ES REMAIN HIGH Although hospitals have extensive laboratory testing for infectious diseases, it is estimated that aetiology in “En over 30% of infectious disease cases remained unknown!. * Cheap (average $50 per test) but ina
Slide 19: Our Solution: RPIDD
OUR SOLUTION: RPIDD (Rapid Pathogen Identification and Detection Device Technology) Executive 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 + <24 hours tu
Slide 20: RPIDD Aims to Shift mRDT Methods to First-line Diagnosis
RPIDD Aims to Shift mRDT Methods to First-line Diagnosis But why is molecular rapid diagnostic testing (mRDT) Therefore, 2 technology fora rapid, cost-effective, currently not first-line? sensitive and unbiased detection for ALL types of pathogens is urgently needed: RPIDD Current commercially avail
Slide 21: RPIDD Device Workflow Overview
RPIDD Device Workflow Overview Proprietary method in DNA/RNA extraction Biofluid collection + ~0.5ml of blood is used in the current generation of method Sample extraction : Proprietary 2 6 hrs + Host DNA depletion * One-pot DNA & RNA library preparation ? Method PROPRIETARY Dedede] * Microbial DNA/
Slide 22: Analytical Performance: Sensitivity and Specificity
lytical Performance: Sensitivity and Specificity Based on internal results, RPIDD device detected organisms ranging from bacteria, RNA viruses and fungi in ONE TEST + 8 species of bacteria, + 2 species of RNA viruses, and + 2 fungal samples were spiked into human plasma All 12 species identified in
Slide 23: NativusWell
tivusWell® Exec NativusWell® ( . Global menopause supplement market is projected to exceed USS50 billion by 20251 NativusWell® is a novel nutraceutical supplement targeting women who are between 45 and 65 years old and experiencing menopausal, perimenopausal and postmenopausal syndromes Planned to c
Slide 24: Aptorum Group Limited
APTORUM APTORUM GROUP LIMITED invector relationc@antonumsraln co