Niki BioSolutions, Inc. · NASDAQ:NIKI
Niki BioSolutions — Aptorum Group — Investor Presentation (September 2019)
55 slides from Niki BioSolutions's investor presentation, Sep 9, 2019
Slide 1: Aptorum: Facilitating Life Science Innovations to Serve Unmet Medical Needs
APTORUM= Facilitating Life Science Innovations to Serve Unmet Medical Needs
Slide 2: Disclaimer
DISCLAIMER Certain information included in this presentation and other statements or materials published by Aptorum Group Limited (the “Company”) are not historical facts but are forward- looking statements. These forward-looking statements refer in particular to the Company's management's business
Slide 3: Investment Highlights
INV MENT HIGHLIGHTS Aptorum Group (Nasdaq: APM) is a biopharmaceutical company focused on development of novel therapeutics for unmet medical needs, with a current market capitalization of over USD 400 million Over 15 therapeutic candidates under development in areas including infectious diseases, g
Slide 4: Leadership
Leadership MR. IAN HUEN Founder, Chief Executive Officer and Executive Director * 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)
Slide 5: Pharmaceutical Development Team
TEAM Pharmaceutical Development Team DR. KWOK CHOW MR. AUSTIN FREEDMAN DR. HERMAN LAM DR. MARY MAZUR MELNYK Canada Based Development Team 7 Canada Based Development Team Canada Based Development Team Canada Based Development Team and Advisor and Advisor and Advisor and Advisor * President of Covar P
Slide 6: Clinical Advisors, Consultants and Principal Investigators
TEAM Clinical Advisor DR. DR. NISHANT AGRAWAL HENRY CHAN LIK YUEN Senior Clinical Advisor q Senior Advisor ¢ Professor of Surgery, School of < Associate Dean, Faculty of Medicine, Medicine, University of Chicago CUHK Consultants, Advisors and Principal Investigators DR. KEITH CHAN Consultant + Adjun
Slide 7: Aptorum's core pillars of therapeutic discovery and development
APTORUM'S 3 core pillars of therapeutic discovery and development, focused on novel therapeutics for unmet medical needs Ever expanding universe of proprietary intellectual property in relation to our pipeline products approach to infectious di Anti-virulence (non-bactericidal) jseases APTORUM CLAVE
Slide 8: Lead Projects, Other Candidates, Device Candidates Projected timeline
Lead Projects > Other Candidates —> Device Candidates Projected timeline Pillar 1: Acticule (ALS series) - Infectious diseases Small molecule, anti-virulence and non-bactericidal approach ALS-4 Anti S. aureus (including MRSA) 932019 Qu/2.2020 ALS-2— Gram+ bacteria ALS-3— Gram+ bacteria > Hybrid stud
Slide 9: Project Portfolio (Others)
PROJECT PORTFOLIO (OTHERS) nulation DOI (NLS-2) Supplement Menopausal symptoms 12020 Program NLS-1 small molecule Endometriosis > vLs-2 Small molecule Alzheimer's & Parkinson's disease > SPLS-1 Small molecule Liver cancer == phantom trial ssa Robotic catheter platform for intra-operative MRI-guided
Slide 10: Overview of Our Lead Projects: ALS-4
OVERVIEW OF OUR LEAD PROJECTS Asmall molecule anti-virulence (non-bactericidal) drug for the treatment of infections caused by Staphylococcus aureus including MRSA. It disarms the bacteria by inhibition of the production of staphyloxanthin, the golden pigment covering the bacteria, which makes it re
Slide 11: Overview of Our Lead Projects: ALS-1
OVERVIEW OF OUR LEAD PROJECTS the Asmall molecule that targets nucleoproteins for the treatment of infections caused by Influenza A. Influenza A nucleoprotein is an essential protein for the proliferation of the influenza virus. ALS-1 causes the aggregation of nucleoprotein and this prevents the agg
Slide 12: Aptorum Focuses on Unmet Medical Needs and Orphan Diseases
APTORUM FOCUSES ON UNMET MEDICAL NEEDS AND ORPHAN DISEASES Po Unmet medical needs Orphan (rare) diseases “defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200
Slide 13: Acticule Projects - Infectious Disease
APTORUM= Facilitating Life Science Innovations to Serve Unmet Medical Needs ACTICULE PROJECTS - INFECTIOUS DISEASE q
Slide 14: Executive Summary: Acticule Projects
EXECUTIVE SUMMARY: ACTICULE PROJ e Aptorum's lead program ALS-4 is an anti-virulent, non-bactericidal drug candidate for Staphylococcus 4 A jac cil " aureus infections including MRSA? ¢ Unlike all major treatments on the market’, ALS-4 relies on an anti-virulent non-bactericidal approach’, potential
Slide 15: Market Overview
MARKET OVERVIEW MARKETSIZE (MMTV (FTEDYAR | (RS A Mortality rate of patients Value of the global CAGR global MRSA drugs with S. aureus MRSA drugs market market (2017-2025)? bacteremia’ in 20167 RECENT DEALS IN INFECTIOUS DISEASE e In 2014, Merck's acquisition of Cubist Pharmaceuticals, a large devel
Slide 16: ALS-4: Approved Drugs for MRSA Infections
ALS-4: APPROVED DRUGS FOR MRSA INFECTIONS Frequently prescribed antibiotics for MRSA infections! Product ibiotic Indication(s Cost of Treat! (Company) Class (dura Vancomycin Severe infections caused Generic Glycopeptide by MRSA Daptomycin ABSSSI, S. aureus erck Bppepepude bacteremia Linezolid ABSSSI
Slide 17: ALS-4: Addressing the Shortfall of Vancomycin
S-4: ADDRESSING THE SHORTFALL OF VANCOMYCIN VAN YCIN e Generic antibiotic that is the most frequently prescribed for MRSA-suspected infections!” e After >60 years? of clinical use, its use against S. aureus is becoming limited. Vancomycin has been shown to have slow bactericidal activity, poor antis
Slide 18: ALS-4: Value Proposition
ALS-4: VALUE PROPOSITION ALS-4 is uniquely poised to tackle multidrug-resistant S. aureus Brilacidin Lefamulin Afabicin oti (Phase tl) (Phase I) Symbiotics© Treatment (Gene therapy for bacteria to GS 74 GS inactivate antibiotic sensitivity) Delafloxacin Omadacycline Exebacase (Phase Il) APTORUM ALS-
Slide 19: Value Proposition
VALUE PROPOSITION ANTIBIOTIC ANTI-VIRULENCE (ALS-4) = Antibiotic resistance in S. aureus has been discovered v in most prescribed antibiotics for MRSA* Not bactericidal, potentially less selective pressure and much less likely for bacteria to develop resistance*> = Broad spectrum and indiscriminate
Slide 20: ALS-4: Mechanism of Action
S-4; ME NISM OF ACTION Acer ea Ahh | ovat ALS-4 4-Diape-cearotene | Gray inhibits a key enzyme ° in the biosynthesis poe 4A'Diaponeurosporen-al | aycypy of staphyloxanthin! Rianne anal 4,4-Diaponeurosporenoic acid) oq “e Glucosy!-4,4’-Diaponeurosporenoic acid | «0 Figure adapted from MBio. 2017 Sep
Slide 21: ALS-4: Mechanism of Action
ALS-4: MECHANISM OF ACTION DMSO Control ALS-4 & «& & 4 200 nM & & S ~ 5 _ 100 nM Geer oe a Owen 25M 5) oo 12.5 nM i > 6.3nM ALS-4 inhibits S. aureus pigment production with an IC,, = 20nM 3.1nM Based on Aptorum’s internal tests/experimentation and has not yet been verified by clinical trials or thir
Slide 22: ALS-4: In Vivo Efficacy
4: IN VIVO EFFICAC ALS-4 inhibits S. aureus pigment production with an IC,, = 20nM ACUTE TREATMENT DELAYED TREATMENT ¥ Vehicle im ALS-4 CFU/Kidney in log scale ; < < CFU/Kidney in log scale * » 7 » v rs ¥ Compound concentration: 1mM Compound concentration: 1mM Inoculum: 5X 10° per mouse Inoculum: 2X
Slide 23: Our Leading Candidate ALS-4 Presents a Unique Opportunity
OUR LEADING CANDIDATE ALS-4 PRESENTS A UNIQUE OPPORTUNITY TO TACKLE INFECTIOUS DISEASES REVOLUTIONARY ANTI-VIRULENCE APPROACH EXCELLENT TRACTION TOWARDS IND FILING Award-winning approach to tackle a huge global unmet need: bacterial infections caused by Staphylococcus aureus including MRSA Good prog
Slide 24: ALS-2 & ALS-3
ALS-2 & ALS-3 Additional anti-virulence, non-bactericidal therapeutics for the treatment of infections caused by Gram Positive bacteria Anti-virulence compound that suppresses multiple unrelated virulence factors in S. aureus! ALS-2 rescues mice ALS-2 reduces virulence ALS-2 reduces from MRSA infect
Slide 25: ALS-1: Targeting a Novel Druggable Target
ALS-1: TARGETING A NOVEL DRUGGABLE TARGET ALS-1 INHIBITS INFLUENZA A NUCLEOPROTEIN (NP) ¢ NP is the most abundantly expressed protein during the course of an infection‘. Its primary function is to encapsidate the virus genome for RNA transcription, replication and packaging. It is also a key adapter
Slide 26: ALS-1: A Unique Antiviral Therapeutic Against Influenza A
ALS-1: A UNIQUE ANTIVIRAL THERAPEUTIC AGAINST INFLUENZAA INFLUENZAA « WHO estimates ~1 billion people are infected and up to 500,000 people die from influenza each year* e There are four types of influenza viruses: influenza A, B, C, D. Influenza A causes the most serious disease in humans and is th
Slide 27: Aptorum: Facilitating Life Science Innovations
PTORUM Facilitating Life Science Innovations to Serve Unmet Medical Needs = — = AV — Ga aa pl i ae W- ~ we. //
Slide 28: Executive Summary: Claves Projects
EXECUTIVE SUMMARY AVES PROJ HUMAN MICROBIOTA ¢ We live in constant symbiosis with our gut bacteria, and dysbiosis can be the cause to numerous diseases! CLAVES TECHNOLOGY e The Claves technology is designed to physically modulate the chemical signaling of diseases-causing microbiota? e Highly scalab
Slide 29: Landscape Overview: The Human Gut Microbiota
LANDSCAPE OVERVIEW: THE HUMAN GUT MICROBIOTA e Contains 100s of species of microbes HUMAN GUT MICROBIOTA e Constantly producing 1000s of active metabolites e Some metabolites provides immunological and metabolic benefits e Dysbiosis (microbial imbalance) is a significant factor in disease Source: 1.
Slide 30: CLS-1: Unmet Medical Needs in Obesity Treatment
CLS-1: UNMET MEDICAL NEEDS IN OBESITY TREATMENT OBESITY TREATMENT First-line intervention for obesity such as dietary change and increased physical activity often fail in the long term! e No consensus regarding the optimal therapy, and existing pharmacotherapy has limited effectiveness and an imperf
Slide 31: Value Proposition: CLS-1 and Claves Platform
VALUE PROPOSITION cts-1 CLAVES PLATFORM ¢ Identified specific microbiota metabolite linked to obesity e Novel therapeutic that physically modulates microbiota metabolite e Acts locally in the gut with high affinity and specificity « Non-absorbable and is expected to be free from any systemic toxicit
Slide 32: CLS-1: Market Overview
LS-1; MARKET OVERVIEW CLS-1: the lead program in the Claves projects, targeting obesity MARKETSIZE (054) uss6.1>, 16.7” of the world’s adult Value of the global CAGR global obesity population were obesity treatment treatment market obese in 2016! market in 2018? (2018-2026)? RECENT DEALS IN OBESITY
Slide 33: Efficacy in a Mouse Model
EFFICACY IN A MOUSE MODEL CLS-1 treatment significantly reduces body weight in mice 40 CLS-1 given to the animals _ 35 Significant weight 5 . loss within 2 weeks 2 & 2 z £ 20: S) o z = 30 =] o = 25 0 20 40 60 Group 1 Group 2 Group 3 Days @ Group 1: High-fat diet +CLS-1 @ Group 2:High-fat diet + cont
Slide 34: Orphan Drug Repurposing Platform
PTORUM= Facilitating Life Science Innovations to Serve Unmet Medical Needs ORPHAN DRUG REPURPOSING PLATFORM i
Slide 35: Executive Summary: SMART-ACT
EXECUTIVE SUMMARY S SMART-ACT™ e Smart-ACT™ is a platform technology that repurposes drugs for orphan diseases with unmet clinical needs e Repurposing drugs greatly reduces time and cost of drug development and clinical trials on average from ~12 years! to ~4 years? e Orphan Designation may, if obta
Slide 36: SMART-ACT: Value Proposition
SMART: ™; VALUE PROPOSITION TRADITIONAL eax! O aw y4 3 o DRUG DISCOVERY & SUCCESS SUCCESS ‘SUCCESS DEVELOPMENT DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 APPROVED Expedited process relying on existing data of approved drug Financial incentives from Orphan Drug Designation? S ACT > ¢ 2 DISCOVERY,
Slide 37: Value Proposition: Continuous Asset Pipeline
OSITION PROBABILITY OF SUCCESS'" SMARTPHARMA CONTINUOUS ASSET PIPELINE e SMART-ACT™ is screening 2,600 approved drugs against a pool of up to 7,000 orphan diseases ¢ Targeting 5-10! candidates each year to enter clinical trials e Expecting 1st clinical asset in 2020 and 4 clinical assets in total by
Slide 38: SACT-1 Development Timeline
SAC DEVELOPMENT CURRENT Q3 2019 Computer database Screening against Candidate validation Q4 2019 2020 2024 of chemical 3 major therapeutic on in vitro . ; structures of 2,600 targets for neuroblastoma In vivo xenograft Phase 2/3 trial Regulatory approved drugs neuroblastoma model efficacy model comm
Slide 39: Dietary Supplement for Menopausal Symptoms (NLS-2)
APTORUM= Facilitating Life Science Innovations to Serve Unmet Medical Needs DIETARY SUPPLEMENT FOR MENOPAUSAL SYMPTOMS (NLS-2)
Slide 40: Executive Summary: NLS-2
EXECUTIVE SUMMARY e NLS-2is a dietary supplement for the relief of menopausal symptoms. e 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 granulosa cells in vitro and in
Slide 41: Landscape Overview and Value Proposition
Menopausal syndrome Hormone replacement therapy (HRT) Currently available dietary supplements PTORUM DOI supplement LANDSCAPE OVERVIEW AND VALUE PROPOSITION Symptoms include hot flashes, mood disorders, night sweats, depression, nervous tension, and insomnia Menopause also puts women at increased ri
Slide 42: Market Overview
MARKET OVERVIEW o*e°e YD | a 1.2: uso 2«D bn uso 7. Ton 4.2” postmenopausal women Value of the global Value of the global CAGR global projected by the year 2030+ menopause isoflavone menopause treatment treatment marketin supplement market market (2017-2023)? ¢ 85% of postmenopausal women experience
Slide 43: DOI - A Chinese Yam Extract to Address Menopausal Syndrome
DOI - A CHINESE YAM EXTRACT TO ADDRESS MENOPAUSAL SYNDROME DOI, a novel bioactive peptide with estrogen-stimulating activity! * Discovered an estrogen-stimulating activity from an extract obtained from the Chinese yam, Dioscorea opposita Thunb * Identified and isolated a novel bioactive component, D
Slide 44: DOI - A Chinese Yam Extract: Bone Density
DOI - A CHINESE YAM EXTRACT TO ADDRESS MENOPAUSAL SYNDROME DOI and bone density! ¢ DOlin old female SD rats demonstrated an increase in the apparent bone mineral density, bone volume fraction and trabecular thickness by microCT scanning (a (b) © (d) Apparent Trabecular Bone Bone Volume Fraction of T
Slide 45: Regulatory Information
REGU Y INFORMATION . 9 ay4 . oe Canada Traditional Chinese Medicine (TCM) "wp be N United States Dietary Supplement C4 GS PRC Dietary Supplement US HONG KONG Health Food Yay Europe / UK o Food Supplement
Slide 46: NLS-1: Treating Endometriosis with a Non-Hormonal Approach
LS-1: TREATME OMETRIOSIS WITH A NON-HORMONAL APPROACH OFE Fallopian tube Pr In in vivo rat models, DOI is shown to stimulate estradiol level and induce estrogen-related gene expressions! Achronic gynecological disorder affecting 10% of woman during reproductive ages with 176 million known cases worl
Slide 47: Pathophysiology
PATHOPHYSIOLOG Ectopic endometrial cells y The areas of endometriosis bleed, oc c resulting in inflammation and scarring? 50 Immune Pelsettia fe} clearance endometrial cells moO | Qin ee) Surviving 0% endometrial cells | Proliferation Angiogenesis Peritoneal linia (ovary, oviduct, ligaments, bowels,
Slide 48: Anti-Angiogenesis Agent for Endometriosis in Mice
ENT Al ESIS AGENT FOR ENDOMETRIOSIS IN MI Saline Vitamin EGCG fo-EGCG LESSEE if - il , | | u i ' saline" Vitamin” EGCG Studies utilizing mouse endometriosis models demonstrated that administration of NLS-1 resulted in greater reductions in the size and weight of lesions than Vitamin E, EGCG or the c
Slide 49: NLS-1: Efficacy in an Animal Model
NLS-1: EFFICACY IN AN ANIMAL MODEL 250% 200% Change in the Lesion Size 150% 50% 0% Vehicle / EGCG / Pro-EGCG / GnRH /PTK787 NLS-1 Weeko (Pro-EGCG at different doses and dosing schedule) weeks Weekl Week2 -- Week3.-« Week4 = WeekS-—— Week Week7 Week9 Week10.- Week11._ Week 12 Time (week) Comparison o
Slide 50: Appendix
Slide 51: Recent Business Update
RECENT BUSINESS UPDATE SEPTEMBER9, 2019 Aptorum Group has initiated IND-enabling studies for its ALS-4 small molecule candidate against S. aureus including MRSA Aptorum Group commenced the preclinical development of two novel drug candidates, CLS-1 and SACT-1, for indications in Obesity and Neurobla
Slide 52: Projected Sales
LS PROJECTED SALE Ahighly-specific, anti-virulent drug candidate’, ALS-4's pricing will be in contrast with the low pricing of generic antibiotics (e.g. vancomycin)? Invasive MRSA? US EU PRC JP RoW Global Infection rate 0.029% 0.029% 0.029% 0.029% 0.029% 0.029% Treatment penetration rate 100% 100% 8
Slide 53: Income Statement Summary (U.S. GAAP)
INCOME EMEN MMARY GAAP)! MARCH 1, 2017 taser DECEMBER, | oettnner, USD USD USD USD Revenue 239,792 26,662 383,450 6 Research and development expenses (2,714,217) (1,342,179) (3,101,432) (2,560,323) General and administrative fees (3,232,916) (2,238,025) (4,919,626) (1,480,093) Legal and professional
Slide 54: Selected Balance Sheet Items (U.S. GAAP)
LECTED BALANCE SHEET ITEMS (U.S. GAAP)! June 30 USD USD USD Cash, restricted cash and marketable securities 6,135,837 27,121,576 18,698,455 Total current assets 7,742,644 28,722,941 20,283,399 Property, plant and equipment, net 5,777,657 4,260,602 346,587 Total assets 24,730,370 45,074,640 31,559,98
Slide 55: Aptorum
APTORUM
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