Protokinetix, Inc. · OTC:PKTX
Protokinetix — Investor Presentation (September 2020)
26 slides from Protokinetix's investor presentation, September 2020
Slide 1: PKX-001: Novel Treatment for Dry Eye Disease (DED)
orotoKinetix PKX-O01 + Novel Treatment for Dry Eye Disease (DED)
Slide 2: Overview
Slide 3: Dry eye disease is a multifactorial disease of the ocular surface
• Dry eye disease (DED) is a multifactorial disease of the ocular surface which results in a spectrum of symptoms and/or signs that Consensus roadmap 2019- affect 5–30% of the population. 2025 developed by European Vision Institute • Dry eye symptoms include discomfort and visual disturbances that s
Slide 4: Dry Eye Disease market value and growth
Studies of the Dry Eye Disease market1,2 indicated a value of ~USD 4.5 billion in 2018, and ~USD 6.2 billion by 2024, with an anticipated CAGR of 5.23%, during the forecast period (2019-2024). The growth of DED market include several factors: • Population aging • Decrease in the supportive hormone,
Slide 5: PKX-001
PKX-OOl PKX-001: Novel Glycopeptide in clinical development as cytoprotective agent YY | © i O HII UO.
Slide 6: Novel Glycopeptide in clinical development as cytoprotective agent
Novel Glycopeptide in clinical development as cytoprotective agent © Small, synthetic analog of the family of anti-freeze glycoproteins. © Cytoprotective and anti-inflammatory activity in various cellular and animal models. © Currently under investigation in patients with type 1 diabetes (effect on
Slide 7: Test Results
Test Results seener lon
Slide 8: PKX-001 demonstrates strong cytoprotective activity in vitro and in vivo
Test Results / PKX-OOl demonstrates strong cytoprotective activity in in vitro and in vivo models
Slide 9: In Vitro & In Vivo Models
In Vitro & In Vivo Models © PKX-001 increased survival of various cells [human and mouse fibroblasts, human peripheral blood mononuclear cells, Jurkat cells, mouse embryonic stem cells, human and mouse islets, and rat cardiomyocytes] incubated under - conditions of: = * Oxidative stress * Low temper
Slide 10: In Vitro & In Vivo Models
In Vitro & In Vivo Models © PKX-001 increased in vivo survival and functional activity of transplanted cells (human islets and photoreceptor precursor cells) following their in vitro pre-treatment with PKX-001. © Invitrotests suggest that PKX has anti-oxidative Activity and anti-Inflammatory activit
Slide 11: PKX-001 Shows Efficacy in Murine DED Model: Proof-of-Concept Study
Test Results / PKX-O01 Shows Efficacy in Murine DED Model: Proof-of-Concept Study
Slide 12: Murine DED Model: Proof of Concept Study
Murine DED Model Proof of Concept Study o Murine model of desiccating stress (induced by low humidity, constant airflow and injections of scopolamine). Oo 5% PKX-001 formulated in BSS (Ocular sterile irrigating solution) and delivered topically QID. YT ~ | oO Results for CD4 + T-cell infiltration up
Slide 13: Murine DED Model: Corneal Permeability
Murine DED Model Proof of Concept Study eee ns, p=0.0537 900 | ***')=0.003 | | | 800 T Corneal Permeability - * 5% PKX-OO1 improved corneal 3500 permeability to similar extent #400 as Positive control oe. (Cyclosporin CsA-MiDROPS™). 2 100 Oo © xs Ss a ra » io a & ce £0 Q FS Study conducted by EyeCRO
Slide 14: Murine DED Model: Conjunctival Goblet Cell Density
Murine DED Model Proof of Concept Study Conjunctival Goblet Cell Density * PKX-OO1 treatment normalized conjunctival goblet cell density. Goblet cells/ mm Study conducted by EyeCRO LLC, Oklahoma City, OK ,USA. Additional information and study report available under CDA
Slide 15: PKX-001 Shows Efficacy in Murine DED Model: Confirmatory Study
Test Results / PKX-O01 Shows Efficacy in Murine DED Model: Confirmatory Study
Slide 16: Murine DED Model: Confirmatory Study
Murine DED Model Confirmatory Study © Murine model of desiccating stress (induced by low humidity, constant airflow and injections of Study Study 2 scopolamine). Treatment % reduction of elevated © 2-5% PKX-001 formulated in BSS and delivered corneal permeability topically under different treatment
Slide 17: PKX-001 Is Not Genotoxic and Has Beneficial Safety Profile in vivo
Test Results / PKX-OO1 is Not Genotoxic and Has Beneficial Safety Profile in vivo Study conducted by EyeCRO LLC, Oklahoma City, OK ,USA. Additional information and study report available under CDA
Slide 18: Beneficial Safety Profile in vivo
Beneficial Safety Profile in vivo o Rabbit eye irritation study (2-10% PKX-00I, single ocular administration): not considered an eye irritant. © Mouse eye irritation study (5% PKX-001, QID ocular administration, 5 days): well tolerated and not considered an eye irritant. Studies conducted by ITR Lab
Slide 19: Rabbit ocular tolerance, genotoxicity and acute toxicity
© Rabbit ocular tolerance and PK study (5% PKX-001, BID or QID, 14 days): well tolerated at all dose levels. + No difference between Control and PKX-001 groups for clinical signs, body weight, food consumption, gross ocular evaluations for local ocular irritation, or ophthalmology examinations. Ocul
Slide 20: PKX-001 Blood and Ocular Tissue Concentrations
Test Results / PKX-OO1 Blood and Ocular Tissue Concentrations Study conducted by EyeCRO LLC, Oklahoma City, OK ,USA. Additional information and study report available under CDA
Slide 21: Rabbit ocular tolerance and PK study
Rabbit ocular tolerance and PK study (5% PKX-001, BID or QID, 14 days). Blood samples collected at several time points post PKX-001 dosing. Ocular tissue samples collected after last PKX-001 dosing (conjunctiva, cornea, aqueous humor, vitreous humor, retina and RPE/choroid/sclera complex) and flash
Slide 22: Secured CMO for cGMP Manufacturing
Test Results / Secured CMO for CGMP Manufacturing
Slide 23: Secured CMO for cGMP Manufacturing
Test Results / e secured CMO for cGMP Manufacturing © Process development, analytical and manufacturing of PKX-001 lyophilized powder for clinical studies provided by experienced US-based Contract Manufacturing Organization © PKX-001 powder (API) manufactured using proprietary synthetic manufacturin
Slide 24: PKX-001 has strong IP protection status
Test Results / PKX-OOl has strong IP protection status Study conducted by EyeCRO LLC, Oklahoma City, OK ,USA. Additional information and study report available under CDA
Slide 25: Patent coverage and market exclusivity
Secured CMO for cGMP Manufacturing © ProtoKinetix has patent coverage for the composition of matter (for the family of AAGP compounds including PKX-001). © Method of use patent applications are pending for the use of PKX-001 in ocular setting. Specifically, for enhancing neurosensory precursor cells
Slide 26: Additional information and contact
• Additional information on efficacy, safety and manufacturing of PKX-001 is available under CDA For information please contact: • ProtoKinetix Inc. is seeking collaboration and licensing L. Grant Young partners to further the development of PKX-001 for the Co-founder, treatment of dry eye disease.
ProtoKinetix — Investor Presentation (September 2020) on 6ix