Niki BioSolutions, Inc. · NASDAQ:NIKI
Niki BioSolutions — Aptorum Group — Investor Presentation (January 2022)
27 slides from Niki BioSolutions's investor presentation, Jan 10, 2022
Slide 1: Corporate Presentation
Facilitating Life Science Innovations to Serve Unmet Medical Needs NASDAQ GLOBAL MARKET: APM CORPORATE PRESENTATION
Slide 2: 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 n
Slide 3: About Aptorum Group: Company Information
Company Information • Established in 2010, Aptorum is a clinical stage biopharmaceutical company dedicated to the discovery, development and commercialization of therapeutic assets to treat diseases with unmet medical needs, particularly in oncology, auto - immune and infectious diseases. • Business
Slide 4: Directors, Management and Significant Employees
Directors, Management and Significant Employees Leadership Mr. Ian HUEN Founder, Chief Executive Officer and Executive Director 4 For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Over 18 years
Slide 5: Consultants and Advisors to Aptorum Group and Subsidiaries
Aptorum Team Consultants and Advisors to Aptorum Group and Subsidiaries MR. William WEISS Consultant 5 For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Currently Director of Preclinical Service
Slide 6: Clinical Stage Programs
Clinical Stage Programs Project Candidate / Modality Indication Development Stage Ac ೦ cule's Series Target id e n t i f i ca t io n & Selection Lead D i sc o v er y Lead Op t i m i z a t io n I N D ( O r I N D e qui v a l e n t Enabling) C lini ca l T r i a l Application S ub m i ss io n Phase I P
Slide 7: Discovery Stage Programs
Discovery Stage Programs Project Candidate / Modality Indication Development Stage SACT’s Series C o m pu t a t ion a l Discovery I n V i t r o Validation E x i s t in g P hI/I I C lini ca l S a f e ty Data 1 I n V i v o Validation I N D P re p ara t io n & Submission Phase I P h as e I b / II a SAC
Slide 8: ALS-4 anti-virulent drug candidate overview
• Additional novel anti - virulent, non - bactericidal approach therapeutics targeting Gram - positive bacteria 1 • In discovery/lead optimization stage and generating good traction towards doing IND - enabling studies 1 • Aptorum's lead program ALS - 4 is an anti - virulent, non - bactericidal drug
Slide 9: ALS-4: Stand Alone or as Combination Therapy with Antibiotics
• Generic antibiotic that is the most frequently prescribed for MRSA - suspected infections 1,2 • After >60 years 3 of clinical use, its use against S. aureus is becoming limited. Vancomycin has been shown to have slow bactericidal activity, poor anti - staphylococcal activity, poor tissue penetrati
Slide 10: Mechanism of Action: ALS-4 on Staphyloxanthin Synthesis
The above diagram summarizes our findings about how ALS - 4 inhibits Staphyloxathin synthesis: • ALS - 4 inhibits a key enzyme in the biosynthesis of Staphyloxanthin with an IC 50 = 20nM. • In the absence of Staphyloxanthin, the bacteria become susceptible to damage by ROS, triggering the usual seri
Slide 11: ALS-4: Oral Formulation Treatment in an MRSA Survival Study
• A lethal dose (10 9 CFU) of MRSA was introduced through the tail vein • ALS - 4 was administered orally 30 minutes after infection for twice a day thereafter The combination of ALS - 4 ’s anti - virulence properties together with host immune system, efficacy is still superior. The in vivo data inc
Slide 12: 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 administered orally twice a day at 10mg/kg per animal ALS - 4 is shown to greatly
Slide 13: ALS-4: Survival Study in Combination with Vancomycin
N = 10, CFU per mouse is 6 x 10 7 . All of the treatments were administrated through i.p. 15 hours after infection; (a) Vehicle (b) ALS - 4: 4.5mg/kg (c) Vancomycin: 4.5mg/kg (d) Combo: 4.5mg/kg LM32+4.5mg/kg Vancomycin IMMEDIATE TREATMENT POST LETHAL DOSE N = 10, CFU per mouse is 6x10 7 ALS - 4 at
Slide 14: ALS-4: Summary of Clinical Study
ALS - 4: Summary of Clinical Study 14 For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • ALS - 4’s first - in - human Phase I trial is a randomized, double - blinded, placebo - controlled, single
Slide 15: SACT-1 targets neuroblastoma
• ~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 ~15% of all cancer - related deaths in the pediatric population 4 • Neuroblastoma is a rare disease and drugs usually qualify fo
Slide 16: SACT-1: In vivo Study and Synergistic Effect with Chemotherapy
SACT - 1 when combined with standard of care chemotherapy showed a statistically significant reduction in tumor volume in a xenograft mouse model. ** Unpaired Student’s T - test, p<0.01, n=8 (based on data observed over initial 22 day period of the study, with SOC applied from day 1 to day 15 and SA
Slide 17: SACT-1: Summary of Clinical Study
• A repurposed small molecule drug discovered from our SMART - ACT® platform, which has the potential to help develop drugs with well - established safety profiles in a time - and cost - effective manner • SACT - 1 targeted indication is neuroblastoma, an orphan oncology disease predominantly in chi
Slide 18: Infectious diseases of unknown causes remain high
INFECTIOUS DISEASES OF UNKNOWN CAUSES REMAIN HIGH Although hospitals have extensive laboratory testing for infectious diseases, it is estimated that aetiology in over 30% of infectious disease cases remained unknown 1 . Current most common clinical diagnostics for infectious disease: Blood Culture C
Slide 19: RPIDD Executive Summary
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 turnaround time + cost effective • Blood sample and adaptable to others (includin
Slide 20: Why Molecular Rapid Diagnostic Testing Is Not First-Line
But Why Is Molecular Rapid Diagnostic Testing (mRDT) Currently Not 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 21: 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 22: Analytical Performance: 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 Based on internal results, RPIDD device detected organisms ranging from bacteria, RNA viruses and fungi in ONE TEST • 8 species of bacte
Slide 23: RPIDD: Summary of Clinical Validation
• Sensitivity and specificity targeted to improve with further v alidation - Sensitivity : Achieved 99.99% - Specificity : Achieved 90%, targeted to exceed 95% (subjected to ongoing clinical validation) • Clinical Validation in progress in Singapore - 12 patients have been enrolled to the scheme - 5
Slide 24: NativusWell menopause supplement
• Global menopause supplement market is projected to exceed US$50 billion by 2025 1 • NativusWell® is a novel nutraceutical supplement targeting women who are between 45 and 65 years old and experiencing menopausal, perimenopausal and postmenopausal syndromes • Commercialization in the UK, Hong Kong
Slide 25: New Ambition in 2022: Oncology and Autoimmune Focus
NEW AMBITION IN 2022 - Resistance develops within months or years 3 (varied by the type of cancer and the targeted therapy used) - Some cancer is not responsive to targeted treatments (e.g. RAS - mutated lunch cancer) 4 Next - Generation gene mutation inhibitors Proteolysis - Targeting Chimera (PROT
Slide 26: Aptorum's Focus: Next-generation inhibitors and protein degradation
(3) Immunomodulator: Targets inflammatory pathways which have been implicated in the tumorigenesis, angiogenesis, and metastasis of many cancer phenotypes. Can be used for auto - immune and infectious disease, including Lupus and Rheumatoid Arthritis 1st G ene r a tion E r lotinib G efitinib Icotini
Slide 27: Aptorum Group
APTORUM GROUP [email protected] T +852 3953 7700 F +852 3953 7711