Niki BioSolutions, Inc. · NASDAQ:NIKI
Niki BioSolutions — Aptorum Group — Investor Presentation (June 2022)
30 slides from Niki BioSolutions's investor presentation, Jun 9, 2022
Slide 1: Facilitating Life Science Innovations to Serve Unmet Medical Needs
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited Facilitating Life Science Innovations to Serve Unmet Medical Needs SMART - ACT Ρ - ORPHAN DRUG DEPURPOSING PLATFORM ALS - 4 Non - confidential Ove
Slide 2: Disclaimer
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 2 Disclaimer This document includes statements concerning Aptorum Group Limited and its future expectations, plans and prospects that constitute “
Slide 3: Antibiotic-resistant Staphylococcus aureus infections represent a significant unmet need
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Staphylococcus aureus are gram - positive bacteria and the leading cause of skin and soft tissue infections, but can cause serious infections su
Slide 4: The Aptorum approach: Combating emerging antibiotic resistance
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Developing non - antibiotics (non - bactericidal and non - bacteriostatic ). • Targeting virulence factors to disarm bacteria and thereby reduci
Slide 5: Antibiotics ALS program: Value proposition
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited Antibiotics 5 ALS program: Value proposition 1. Refer to “ALS - 4: Approved Drugs for MRSA Infections” for complete set of sources; 2. P T. 2016 F
Slide 6: ALS program: Value proposition
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Bacterial infections are mediated by pathogenic or opportunistic bacteria • Successful infections depends on host immunity and the pathogen’s vi
Slide 7: ALS-4 first-in-class oral therapeutic complementing vancomycin
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Novel mechanism: Anti - virulence, non - bactericidal approach as it targets virulence properties of S. aureus • Oral form : An orally administe
Slide 8: The role of staphyloxanthin in Staphylococcus aureus
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Neutrophils kill bacteria including Staphylococcus aureus intracellularly or extracellularly via Reactive Oxygen Species: ROS - oxygen radicals
Slide 9: Mechanism of action: Targeting staphyloxanthin synthesis
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 inhibits a key enzyme in the biosynthesis of staphyloxanthin. 1 In the absence of staphyloxanthin , the bacteria become susceptible to d
Slide 10: Inhibition of staphyloxanthin synthesis
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 • Inhibits S. aureus pigment production ( staphyloxanthin ) with an IC 50 = 20nM. • This is visibly confirmed by the decolorization of the
Slide 11: Inhibits staphyloxanthin production in 11 common strains of S. aureus
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 inhibits the production of staphyloxanthin in 11 common strains of S. aureus in vitro 11 ALS - 4 effectively inhibits staphyloxanthin form
Slide 12: Does not directly inhibit bacterial growth in vitro
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Lack of direct selection pressure significantly decreases the risk of emergence of drug resistance. • In the absence of neutrophils, ALS - 4 doe
Slide 13: Increases sensitivity of S. aureus to oxidative damage
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 reduces bacteria number by an additional 10 - fold in the presence of hydrogen peroxide (mimicking ROS production by neutrophils), as de
Slide 14: Enhances neutrophil killing of MRSA
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 14 ALS - 4 enhances neutrophil killing of MRSA Bacterial density following treatment with human neutrophils in ALS - 4 or vehicle treated MRSA (st
Slide 15: Rescues rats infected with a lethal dose of MRSA
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • 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 d
Slide 16: Greatly reduces organ bacterial count in a bacteremia model
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • 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
Slide 17: of any 2022 Aptorum Group Limited Vehicle Van ALS + Van Vehicle Van ALS
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 17 Vehicle ALS - 4 Van ALS + Van Vehicle ALS - 4 Van ALS + Van The description of ALS - 4 and related conclusory statements on ALS - 4 on this sli
Slide 18: Does not interfere with the action of vancomycin in vitro
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • No effect on the MIC of vancomycin was observed in vitro when the concentration of ALS - 4 was below 25µM • ALS - 4 is targeted to be efficaciou
Slide 19: Does not trigger antibiotic resistance in MRSA
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Clindamycin resistance (MIC from 0.12 μg /ml to >5 μg /ml) appeared rapidly after a 10 - day intermittent treatment • The use of Ery was to ensu
Slide 20: Does not trigger its own resistance
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited No bacterial resistance to ALS - 4 detected after continuous incubation of the bacteria in the presence of 1μM ALS - 4 for 11 days. 20 ALS - 4 doe
Slide 21: Chemistry, manufacturing and controls (CMC)
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Only 1 physical form identified from polymorph screening • Physically and chemically stable • Not hygroscopic 21 ALS - 4: chemistry, manufacturi
Slide 22: Phase 1 trial in Canada (Completed)
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited Phase 1 trial in Canada (Completed) A randomized, double - blind, placebo - controlled, dose - escalation study to assess the safety, tolerability
Slide 23: Targeted clinical development
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 23 ALS - 4: Targeted Clinical development Phase 1 (Canada; completed) Phase 2 (US) (under planning) Registrational study (future) Filing An unmet,
Slide 24: Market opportunities
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • The global methicillin - resistant Staphylococcus aureus drugs market was valued at approximately US$ 2.9 Bn in 2016 and projected to each over
Slide 25: IP status
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • The patent and patent applications cover the composition of small molecule compound and the method of treating microbial infection using same me
Slide 26: Appendix
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited APPENDIX
Slide 27: Approved drugs for MRSA infections
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Approved drugs for MRSA infections Frequently prescribed antibiotics for MRSA infections 1 27 Approved drugs for MRSA Infections Product (Compan
Slide 28: Oral administration in a MRSA non-lethal bacteraemia mouse model
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Dose - dependent efficacy of ALS - 4 ( compound IM032) shows a statistically significant reduction in bacteria count across major organs relativ
Slide 29: Protocol: resistance testing
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 1. Inoculum preparation: USA300 - 3 (LAC) was cultured overnight in BHI broth at 37˚C, 250 rpm. 2. Subculture preparation: 60 µl overnight culture
Slide 30: of any 2022 Aptorum Group Limited
For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 30