Akari Therapeutics Plc · NASDAQ:AKTX
Akari Therapeutics — Investor Presentation (October 2018)
25 slides from Akari Therapeutics's investor presentation, Oct 1, 2018
Slide 1: October 2018 Investor Presentation
October 2018 Investor Presentation Copyright Akari Therapeutics, Plc - 2018
Slide 2: Disclaimers
2 Disclaimers Certain statements in this presentation constitute contains “forward - looking” statements within the meaning of Section 27A of th e Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. These forward - looking statements reflect our current views about our pla
Slide 3: Akari Overview
3 Akari Overview ● Creating drugs for acute and chronic orphan inflammatory diseases by modulating complement/C5 and leukotriene/LTB4 pathways ● Lead drug – Coversin has unique dual MOA in both C5 & LTB4 ● Diversified portfolio : four target orphan inflammatory diseases ‒ Bullous pemphigoid (BP) ‒ A
Slide 4: Coversin: Synchronous C5 and LTB4 Inhibition
4 Coversin: Synchronous C5 and LTB4 Inhibition C5a MAC Activation Granulocyte population at inflammatory site LTB4 Activation Synthesis Granulocytes at distal sites Recruitment to local site & amplification Direct damage Cell lysis / damage PNH C5 TMA AKC BP LTB4 Direct damage - MAC C5 & LTB4 granul
Slide 5: Growing Clinical Data Set Supports Coversin Development for Range of Inflammatory Conditions
5 Positive safety profile – Eleven patients on ongoing or completed treatment for between 2 and 32 months; cumulative total of over 13 patient years of treatment – No SAEs related to Coversin and no neutralizing antibodies Proven C5 binding across disease categories – Met PNH Phase II primary effica
Slide 6: Focused on Orphan Inflammatory Indications with High Unmet Need
6 Focused on Orphan Inflammatory Indications with High Unmet Need • Four lead programs : Pemphigoids ( incl BP), Surface of the eye ( incl AKC), TMAs, and PNH all with peak sale potentials of $500m+ • Most disease target conditions have no approved treatment • Smaller diseases such as HSCT – TMA pro
Slide 7: Portfolio Builds on Coversin PNH Clinical Results
7 Portfolio Builds on Coversin PNH Clinical Results Focus on Maximizing Return on Capital 1. PNH: Paroxysmal nocturnal hemoglobinuria; 2. TMA: Thrombotic Microangiopathy; HSCT Hema topoietic stem cell transplant 3 aHUS: Atypical hemolytic - uremic syndrome; 4. BP: Bullous Pemphigoid; 5. AKC: Atopic
Slide 8: Clinical Data
8 Clinical Data
Slide 9: Patients Being Treated With Coversin Across Four Primary/Lead Indications
9 Patients Being Treated With Coversin Across Four Primary/Lead Indications Trial Enrollment Completed PNH naïve (chronic) Phase II and long - term safety safety 4 and 32 Months PNH Resistant (chronic) Phase II 11 - 21 Months 2 patients Ongoing 6 patients Ongoing 18 Months 1 patient Complete TMA (HS
Slide 10: C5 Inhibition Demonstrated in PNH patients treated with Coversin
10 C5 Inhibition Demonstrated in PNH patients treated with Coversin 0 1 2 3 4 5 0 10 20 30 40 50 60 70 80 90 Days Weeks LDH (IU/ml) 0 1 2 3 4 5 100 0 25 50 75 PNH COBALT Phase II Resistant PNH Patients Median and range LDH x ULN Original maintenance dosing: 30 mg daily (N=5, N=4 after day 43*) Revis
Slide 11: Phase II PNH Clinical Endpoint: 67% Decline in Transfusion Dependence
11 Phase II PNH Clinical Endpoint 67% Decline in Transfusion Dependence • 6 patients transfused in 6 months prior to entering Phase II • 3 became transfusion independent during Phase II • 4 transfusion independent during long - term safety study RECEIVING COVERSIN 0 10 20 30 40 50 60 70 80 90 100 6
Slide 12: Pediatric TMA: Post-Bone Marrow Transplant Complement Inhibition demonstrated with Coversin
12 0 50 100 150 200 250 300 350 400 Pre 3 h 6 h 12 h 24 h Day 3 Day 7 Day 14 Day 21 Day 28 Week 8 CH50 Elisa (U Eq /mL) 0 100 200 300 400 500 600 700 800 Pre Day 14 Day 21 Day 28 sC5b - 9 levels ( Hng /mL) ULN ULN Complete and rapid C5 inhibition as seen in PNH patients Pediatric dosing regime effec
Slide 13: Coversin Activity Demonstrated in HSCT-TMA Across a Range of Outcome Measures
13 Coversin Activity Demonstrated in HSCT – TMA Across a Range of Outcome Measures (2 Patients) TMA Marker Patient Baseline Day 7 Day 14 Day 28 Day 60 Hemolytic anaemia 1 Yes Resolved 2 No Red blood cell fragments 1 Yes Resolved 2 Yes Resolved Thrombocytopenia 1 Yes Resolved 2 Yes Resolved Increased
Slide 14: Clinical Programs
14 Clinical Programs
Slide 15: Clinical Programs focused on C5 & C5/LTB4 Targets And Both SQ and Topical Delivery
15 Clinical Programs focused on C5 & C5/LTB4 Targets And Both SQ and Topical Delivery BP AKC PNH TMAs C5 Only C5 & LTB4 Subcutaneous Delivery Topical AKC: Atopic keratoconjunctivitis; BP: Bullous Pemphigoid; TMA: Thrombotic Microangiopathy; PNH: Paroxysmal nocturnal hemoglobinuria
Slide 16: Thrombotic Microangiopathies (TMA) Several Diseases With Unmet Need
16 Thrombotic Microangiopathies (TMA) Several Diseases With Unmet Need • Thrombotic Microangiopathies ‒ Range of orphan disease, in most cases no approved therapy ‒ Numerous conditions considered complement driven with LTB4 implicated in several • Atypical hemolytic - uremic syndrome ( aHUS ) ‒ Ongo
Slide 17: Thrombotic Microangiopathy (TMA) After Transplant: Significant Unmet Need
17 Thrombotic Microangiopathy (TMA) After Transplant: Significant Unmet Need • Orphan condition in which there is evidence that both terminal complement activation and possibly LTB4 have a role in driving disease • Complications following bone marrow transplantation (BMT) in up to 30% of patients •
Slide 18: Bullous Pemphigoid (BP)
18 Bullous Pemphigoid (BP) • Orphan condition in which there is evidence that both terminal complement activation (C5) and LTB4 have a role in driving disease • TPP – moderate to severe patients, newly presenting or relapsing • Other biologicals with different MOA are also in Phase II development fo
Slide 19: Bullous Pemphigoid Phase II Trial Design in Patients with Mild-to-Moderate Disease
19 Bullous Pemphigoid Phase II Trial Design in Patients with Mild - to - Moderate Disease Study design • Phase II Open label single arm (n = 9); 42 days treatment • Test role of C5 & LTB4 dual inhibition in improving BP outcomes • Active; newly diagnosed or recurrent, mild to moderate treated with t
Slide 20: Atopic Keratoconjunctivitis (AKC)
20 Atopic Keratoconjunctivitis (AKC) AKC VKC PKC SAC SJS MMP AKC offers opportunity to expand into related conditions • Severe eye surface inflammation causing infiltration of immune cells (neutrophils & T cells) – a major cause of blindness • Topical drugs, such as steroids or cyclosporin, often no
Slide 21: Planned AKC Phase I/II Proof-of-Principle Trial Design
21 Planned AKC Phase I/II Proof - of - Principle Trial Design Study design • Phase I/II randomized, double blind, placebo - controlled, safety and dose finding study – 11 active 8 placebo Treatment • Coversin topical eye drops three times daily for 2 months – High (0.25%), medium (0.125%) and low (0
Slide 22: PNH Clinical Programs: Staged Phase III Commitment
22 PNH Clinical Programs: Staged Phase III Commitment Focus on PNH Patient Convenience • Recruiting • 2 patients currently on treatment in Holland & USA • FDA post - Phase II review meeting completed Resistant Program Phase III CAPSTONE Phase III ASSET • Naïve patients, recruiting • 6 month treatmen
Slide 23: Expanding Pipeline Focused on Indications Where C5 and LTB4 Both Involved
23 N = 5 mice in each treatment group Start drug treatment Expanding Pipeline Focused on Indications Where C5 and LTB4 Both Involved Zileuton PAS - Coversin PAS - LTB4 - Coversin Cell recruitment to Lung Induced by LPS Dual action Coversin more effective than C5 - only Coversin (or Zileuton®) alone
Slide 24: Akari Summary
24 Akari Summary ● Unique mode of action - inhibiting both C5 and LTB4 ● Diversified pipeline : ‒ AKC and BP - initial readouts Q1 2019 ‒ TMAs - ongoing readouts in aHUS and TMA post HSCT – H1 2019 ‒ PNH - staged readouts in 2019 ‒ Preclinical - ongoing programs in lung, RA, trauma ● $20 million fin
Slide 25: October 2018 Copyright Akari Therapeutics, Plc - 2018
October 2018 Investor Presentation Copyright Akari Therapeutics, Plc - 2018
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