Niki BioSolutions, Inc. · NASDAQ:NIKI

Niki BioSolutions — Aptorum Group — Technical Presentation (June 2022)

18 slides from Niki BioSolutions's technical presentation, Jun 9, 2022

  1. 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 SACT - 1 Nonconfidential Overv

  2. Slide 2: Forward-looking statements

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited This document includes statements concerning Aptorum Group Limited and its future expectations, plans and prospects that constitute “forward - loo

  3. Slide 3: Executive summary

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • SACT - 1 is a repurposed oral suspension in development a s an adjunctive therapy to standard of care in relapsed or refractory high - risk neur

  4. Slide 4: Executive summary (cont'd)

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • Our completed Phase 1 trial showed the safe use of SACT - 1. The distinctive PK between SACT - 1 and Edurant ® further strengthen the advantage

  5. Slide 5: MEK5 and ERK5 as potential therapeutic targets for cancers

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited So far no approved products are available MEK5 and ERK5 as potential therapeutic targets for cancers in recent years

  6. Slide 6: Proposed mode of action

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • MEK5 - ERK5 pathway is reported to regulate MYCN oncogene expression (Kang et al., 2006; Umapathy et al., 2014; van Hoang et al., 2017) which is

  7. Slide 7: Targeted product profile

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 7 Targeted product profile Preclinical Toxicology Not relevant as this is a repurposed drug PK/PD Model To be determined in Phase 1b/2a trial CM&C

  8. Slide 8: Neuroblastoma - market overview

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • ~ 700 cases of high - risk neuroblastoma patients each year in the US 3 and we estimated EU has 1.5x this number of cases, c. 1050 high risk neu

  9. Slide 9: Standard of care for high-risk neuroblastoma

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited Standard of care for high - risk neuroblastoma • Surgery • Chemotherapy • Radiotherapy • Immunotherapy (anti - GD2 monoclonal antibodies) o Dinutu

  10. Slide 10: In vitro and in vivo efficacy

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 10 In vitro and in vivo efficacy In vitro • SACT - 1 demonstrated inhibition of all tested neuroblastoma cell lines (IMR - 32, SK - N - BE(2), SK

  11. Slide 11: In vivo efficacy - neuroblastoma xenograft studies in mice

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 11 In vivo efficacy - neuroblastoma xenograft studies in mice Cont’d SACT - 1 (RPV) dosed at 60 and 160 mg/kg (PO; daily x 21) potentiates the ant

  12. Slide 12: Mechanistic studies - decrease of MYCN expression

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • MYCN is involved in regulating various biological activities, such as apoptosis, proliferation, and angiogenesis in neuroblastoma (Huang and Wei

  13. Slide 13: Mechanistic studies - ERK5 phosphorylation

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited • SACT - 1 was found to have a binding constant (K D ) of 150 nM against MEK 5 in a previous study by employing KdELECT Kinase Assay Panel • Expec

  14. Slide 14: Phase I study on bioavailability/food effect

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 14 Phase I study on bioavailability/ food effect Relative Bioavailability • Comparative bioavailability analysis between administration of SACT -

  15. Slide 15: Clinical Development Plan

    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: Open - label, Randomized, 3 period, 3 - sequence, Single - dose Crossover Bioavailability and Food Effect Study of SACT - 1 and Edurant

  16. Slide 16: Phase 1b/2a trial: Proposed Primary and Secondary Objectives

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited Phase 1b/2a trial: Proposed Primary and Secondary Objectives Phase 1b Primary Objective • Evaluate the safety and tolerability of SACT - 1 in comb

  17. Slide 17: Intellectual property

    For illustrative purposes only. There is no guarantee of any project being completed or having a specific outcome. © Copyright 2022 Aptorum Group Limited 17 Intellectual property Exclusive IP​ Title​ Country​ Application Date​ Expiration date​ Status​ Composition Including SACT - 1 and Method for Tr

  18. Slide 18: 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 18

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