Akari Therapeutics PLC · NASDAQ:AKTX
Akari Therapeutics — Investor Presentation (July 2017)
33 slides from Akari Therapeutics's investor presentation, July 2017
Slide 1: Akari Therapeutics Corporate Presentation July 2017
NASDAQ: AKTX July 2017 Copyright Akari Therapeutics, Plc 2017
Slide 2: Cautionary Note Regarding Forward-Looking Statements
Cautionary Note Regarding Forward-Looking Statements Certain statements in this presentation constitute "forward-looking candidates and support existing products; the approval by the FDA and statements" within the meaning of Section 27A of the Securities Act EMA and any other similar foreign regulat
Slide 3: Akari Mission Statement
Akari Mission Statement Developing the next generation of rare & orphan anti-inflammatory therapies Ticks have undergone 300 million years of natural selection to produce inhibitors that bind to key inflammatory mediators and are well tolerated in humans. Our unique molecules are derived from these
Slide 4: Exploiting Evolutionary Benefits of Tick-Derived Proteins
Exploiting Evolutionary Benefits of Tick-Derived Proteins Tick salivary proteins work by inhibiting host immune responses, enabling tick to repeatedly feed without damage from host inflammatory response EFFICIENT Target early inflammatory mediators TARGETED Specific binding avoids off-target effects
Slide 5: Akari Highlights
Akari Highlights Developing tick-derived proteins to inhibit early inflammation Three separate development programs, each focused on distinct inflammatory pathways 1) Complement program • PNH* - projected Phase III start – 4Q 2017 • aHUS* - projected Phase II start – 4Q 2017 • Once-weekly dosing mol
Slide 6: Strategic Positioning
Strategic Positioning Leukotriene Complement inhibition + Complement inhibition Expected to be Developing ~$5 billion peak market orphan market Sub Q delivery Differentiated differentiated against Mechanism of Action current IV standard of care Active AKC, BP, Systemic programs Active PNH & upcoming
Slide 7: Akari Portfolio Builds On Complement Experience
Akari Portfolio Builds On Complement Experience Discovery Preclinical Phase I Phase II Phase III Complement System (C5) Coversin™ Coversin LA™ Tissue Targeted (NMJ) LTB4 Coversin - LTB4 + C5 LTB4 (L-Coversin™) LTB4 LA Bioamine System Histamine binding Serotonin binding 6
Slide 8: Complement Program
COMPLEMENT PROGRAM 7
Slide 9: Akari - Complement Pathway Positioning
Akari - Complement Pathway Positioning • Two ongoing Phase II PNH programs – CONSENT and COBALT; and a long-term safety study - CONSERVE • Resistant PNH trial (CONSENT) – 1 patient ‒ Currently recruiting ‒ Patient has been on Coversin since February 2016 • Naïve PNH trial (COBALT) - 5 patients ‒ All
Slide 10: Positive Response For Months In Eculizumab-Resistant PNH Patient Treated With Coversin
Positive Response For 15 Months* In Eculizumab- Resistant PNH Patient Treated With Coversin 1,400 1,200 1,000 LDH (IU/ml) 800 LDH 600 400 200 1.5 ULN 0 0 8 16 24 32 40 48 56 64 Weeks CH50 <8.0 U Eq/mL (below level of detection) No hemolytic episodes; no dose changes; symptom free; twice daily self i
Slide 11: Phase 2 PNH Trials Update
Phase 2 PNH Trials Update All five continuing patients (in CONSENT and COBALT) on ongoing treatment have experienced: • Daily (COBALT) or twice daily (CONSENT) subQ self-administration • No neutralizing antibodies • No SAEs related to Coversin • AEs (including mild/moderate injection site reactions)
Slide 12: Declines in Blood Markers of Hemolysis – All Four Continuing COBALT Phase II Patients
Declines in Blood Markers of Hemolysis – All Four Continuing COBALT Phase II Patients LDH plotted as a multiple of ULN Aspartate aminotransferase (AST) 4 patients who remained in COBALT 4 patients who remained in COBALT 8 140 Mean Mean 7 Patient A 120 Patient A 6 Patient B 100 Patient B LDH x ULN 5
Slide 13: Packed Red Blood Cells Transfused Prior to and During 90 Day COBALT Trial
Packed Red Blood Cells (PRBC) Transfused Prior to and During 90 Day COBALT Trial 12 Patient A 10 Patient B Patient C 8 Patient D Patient E 6 4 2 0 12-9 9-6 6-3 3-0 0-3 Months prior to start of Trial Trial Patients A and E had no transfusions in the 12 months preceding the COBALT trial or while in th
Slide 14: Treatment Duration, Safety, and Tolerability Of Patients Treated with Coversin
Treatment Duration, Safety, and Tolerability Of Patients Treated with Coversin Eculizumab-resistant PNH patient (CONSENT) 17 months 17 Months • Drug well tolerated by patients Continuing Phase 2 PNH patients (COBALT)* • No SAEs related to Coversin • CONSENT patient and the 4 7 Months continuing COBA
Slide 15: Planned PNH Phase III Trials CAPSTONE and ASSET
Planned PNH Phase III Trials CAPSTONE and ASSET • Two PNH Phase III clinical trials: ‒ Naïve patients (CAPSTONE) ‒ Switch (ASSET) • CAPSTONE: Naïve patients randomized to Coversin plus standard of care vs. standard of care • ASSET: Eculizumab-treated patients randomized to remain on eculizumab or sw
Slide 16: Coversin Targeting Atypical Hemolytic Uremic Syndrome (aHUS)
Coversin Targeting Atypical Hemolytic Uremic Syndrome (aHUS) • Chronic and life-threatening genetic disease characterized by microangio-pathic hemolytic anemia, thrombocytopenia, and kidney injury • Efficacy of C5 inhibition in aHUS demonstrated by the approval of eculizumab for this indication • aH
Slide 17: Dual C5 and Leukotriene B4 (LTB4) Program
DUAL C5 AND LEUKOTRIENE B4 (LTB4) PROGRAM 16
Slide 18: Dual Leukotriene and Complement Inhibition Has Potential In Wide Range of Diseases
Dual Leukotriene and Complement Inhibition by Coversin Has Potential In Wide Range of Diseases with Unmet Need Complement Only PNH aHUS MG NMO Leukotrine & Complement Bullous Sjögren’s Pemphigoid Mucous Membrane Pemphigoid AKC Bronchiolitis obliterans Goodpasture’s AATD PAH Rheumatoid Arthritis Dry
Slide 19: Two Phase II Leukotriene/Complement Trials Projected to Start in 1Q 2018
Two Phase II Leukotriene/Complement Trials Projected to Start in 1Q 2018 Dual action (Coversin) more effective than C5-only (saturated Coversin) or • Phase II programs Zileuton alone in mouse model ‒ AKC (atopic keratoconjunctivitis) - eye (topical drops) ‒ BP (bullous pemphigoid) – skin (sub Q) • S
Slide 20: Coversin & L-Coversin Potentially Superior Strategy to Target LTB4
Coversin & L-Coversin Potentially Superior Strategy to Target LTB4 Previous strategies to target LTB4 suffered from a lack of selectivity; benefits of reducing LTB4 were offset by off-target effects Coversin and L-Coversin capture LTB4 within an internal binding site • Directly and specifically “mop
Slide 21: LTB4 Intervention Strategies
LTB4 Intervention Strategies Limitations of Other Arachidonic acid LTB4 Inhibitors Zileuton MK-0633 5-LO 5-LO / FLAP inhibitors TA-270 FLAP GSK2190915 Reduces anti-inflammatory LTA4 DG-031 lipoxins Lipoxygenases BLT1/BLT2 antagonists LTA4H Realization that anti- LTC4S inflammatory mediators also sig
Slide 22: Dual Action Eye (AKC) and Skin (BP) Program
DUAL ACTION EYE (AKC) AND SKIN (BP) PROGRAM 21
Slide 23: Demonstrated Benefit in Mouse Model Late-Phase Ocular Inflammation
Demonstrated Benefit in Mouse Model Late-Phase Ocular Inflammation Collaboration with Moorfields Hospital (Institute of Ophthalmology) EIC pre-clinical model of severe eye surface inflammation Effect of Coversin on OVA induced inflammation (n=16 per group) • Coversin - 64% reduction in inflammatory
Slide 24: Atopic Keratoconjunctivitis (AKC)
Atopic Keratoconjunctivitis (AKC) • Severe eye surface inflammation causing infiltration of immune cells such as neutrophils and T cells. A cause of blindness worldwide • Topical drugs, such as steroids or cyclosporin, often not effective or cannot be given chronically • In AKC disease, despite best
Slide 25: Coversin Demonstrated Benefit in Bullous Pemphigoid Model
Coversin Demonstrated Benefit in Bullous Pemphigoid Model • ~60% reduction in affected area on Coversin (Sub Q) compared to vehicle or steroid in mouse model • Clear dose response P=0.0023 between vehicle and 250 µg/kg Vehicle 250µg/kg Coversin Preclinical passive mouse model from Dr. Sadik in Lubec
Slide 26: Bullous Pemphigoid (BP) Significant Unmet Need
Bullous Pemphigoid (BP) Significant Unmet Need • Immune complex deposition initiates complement cascade and inflammatory process • LTB4 recruits neutrophils to dermis-epidermis junction and amplifies inflammation • Phase II open label trial against standard of care projected to start in 1Q 2018 in t
Slide 27: Scientific Development Program
SCIENTIFIC DEVELOPMENT PROGRAM 26
Slide 28: Akari Discovery Platform
Akari Discovery Platform Coversin engineered molecules targeted to Other Akari tick-derived complement pathway molecules • Extended half life (LA) • Eicosanoid pathway (LTB4 only) L-Coversin (lung) • Bioamine pathway • Tissue targeting (NMJ*) ‒ Ligand capture preventing (Myasthenia Gravis) action on
Slide 29: Coversin LA: Once Weekly Formulation
Coversin LA: Once Weekly Formulation Human PK simulation of free C5 following single dose injection Inhibition of complement alternative potential for weekly dosing pathway rabbit red blood cell lysis 120 Coversin Eculizumab 100 PAS Coversin Coversin &Eculizumab 80 % Total Lysis Coversin 60 40 5% PA
Slide 30: Financial Summary March 31, 2017
Financial Summary March 31, 2017 • ADS* outstanding 11,776,934 • ADS Fully Diluted 12,583,641 • Cash and Cash Equivalents** $35.1m (no debt) * Each ADS represents 100 ordinary shares ** Includes short-term investments 29
Slide 31: Akari Clinical Program Highlights
Akari Clinical Program Highlights Complement program • PNH - projected Phase III start – 4Q 2017 - additional Phase II patients – 4Q 2017 • aHUS - projected Phase II start – 4Q 2017 • Coversin LA - projected Phase I start – 4Q 2018 Dual C5 & leukotriene B4 (LTB4) program • AKC & BP - projected Phase
Slide 32: COBALT Phase II Dosing and Response Summary
COBALT Phase II Dosing and Response Summary • Three of the four continuing patients were updosed – Patient A and B were updosed from 30 mg to 45 mg once daily at Days 40 and 54, respectively – Patient C was updosed to 22.5 mg twice daily at Day 24 and moved to 45 mg once daily at Day 67 – Patient B,
Slide 33: Akari Therapeutics
IN AKARI THERAPEUTICS NASDAQ: AKTX July 2017
Akari Therapeutics — Investor Presentation (July 2017) on 6ix