Akari Therapeutics Plc · NASDAQ:AKTX
Akari Therapeutics — Investor Presentation (December 2022)
20 slides from Akari Therapeutics's investor presentation, Dec 7, 2022
Slide 1: Akari Therapeutics
Akari Therapeutics December 2022
Slide 2: Forward-Looking Statements
Forward-Looking Statements Certain statements in this presentation constitute “forward-looking” statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. These forward-looking statements reflect our current views about our plan
Slide 3: Nomacopan Is a Novel Bispecific Recombinant Protein
Nomacopan Is a Novel Bispecific Recombinant Protein High resolution structure of nomacopan (cyan) bound to the CUB, C5d, and C345C domains of C5 1 Two Modes of Action • Inhibits complement C5 activation similar to eculizumab, but binds a different conserved region of C5 Ticks modulate host responses
Slide 4: Nomacopan Inhibits Damaging Cell Lysis and Inflammation, Preserving Opsonization and Clearance
Nomacopan Inhibits Damaging Cell Lysis and Inflammation, Preserving Opsonization and Clearance of Immune Complexes • Specifically prevents activation of C5 and binds LTB4, preventing Complement Leukotriene proinflammatory and potentially tissue-damaging effects mediated by C5a, the MAC and LTB41 • O
Slide 5: Near-Term Potential, Promising Pre-Clinical Program
Near-Term Potential, Promising Pre-Clinical Program Two Current Focus Areas Indication Candidate/ Phase 3 Phase 3 Designations Pre-Clinical Phase 1&2 Part A Part B Formulation FDA Orphan FDA Orphan, Pediatric hematopoietic Fast Track, and Drug, Fast Rare Pediatric stem cell transplant– Nomacopan/ Tr
Slide 6: Previous Areas of Clinical Development, Including PNH and BP, Have Supported Current Development Pathways
Previous Areas of Clinical Development, Including PNH and BP, Have Supported Current Development Pathways • In addition to current areas of focus, Akari has conducted clinical research in several other areas, including Phase 3 clinical trials of subcutaneous nomacopan for treatment of bullous pemphi
Slide 7: Leadership Team
Leadership Team Rachelle Jacques John Neylan, MD Melissa Bradford-Klug Miles Nunn, DPhil Torsten Hombeck, PhD President & CEO Chief Medical Officer Chief Operating Officer Chief Scientific Officer Chief Financial Officer Discovered nomacopan Volution 7
Slide 8: Thrombotic Microangiopathies (TMAs)
THROMBOTIC MICROANGIOPATHIES (TMAs) 8
Slide 9: Nomacopan May Be the First Treatment for Pediatric HSCT-TMA, a Condition with Mortality Up to 80%
Nomacopan May Be the First Treatment for Pediatric HSCT-TMA, a Condition with Mortality Up to 80% 1. Efficacy Efficacy of complement C5 inhibition is established in HSCT- TMA2 and aHUS 3,4, another TMA (eculizumab); efficacy of nomacopan C5 inhibition supported by clinical PNH research5 • TMA follow
Slide 10: Impact of C5 Inhibition on Survival in HSCT-TMA
Impact of C5 Inhibition on Survival in HSCT-TMA Survival curves for patients with high-risk TMA who were treated with the terminal complement blocker eculizumab (“Treated”, n=18) and historical controls from prospective observational TMA study with the same high risk TMA features who did not receive
Slide 11: Program Acceleration in Severe Pediatric HSCT-TMA
Program Acceleration in Severe Pediatric HSCT-TMA MIDD Predictive Rich Participation PK/PD Model Data Set Akari was selected to Akari’s robust, predictive PK/PD model Akari clinical data from participate in the FDA simulated 10,000 virtual patients 38 subjects (in previous Model-Informed Drug inform
Slide 12: Pediatric HSCT-TMA Phase 3 Open-Label Study Now Underway
Pediatric HSCT-TMA Phase 3 Open-Label Study Now Underway Nomacopan in pediatric HSCT-TMA has been granted FDA Orphan, Fast Track, and Rare Pediatric Disease Designations Part A Part B • Dose confirmation by age cohort • Efficacy Purpose • Safety • Safety Age cohorts Study • 7 total patients 0.5 to <
Slide 13: HSCT-TMA U.S. Market Opportunity
HSCT-TMA U.S. Market Opportunity HSCT-TMA U.S. Population (incidence in adults and children in the U.S. 2022) 10,000 Children: ~200-300 ~22,000 HSCTs occur annually in the U.S. PATIENT POPULATION IN THOUSANDS 7,500 ~8,400 (38%) lead to TMAs Moderate to severe complement ~3,400 mediated ~3,400 TMAs a
Slide 14: Geographic Atrophy (GA)
GEOGRAPHIC ATROPHY (GA) 14
Slide 15: PAS-Nomacopan May Provide Key Benefits: Complement Inhibition, Fewer Doses and LTB4 Inhibition
PAS-Nomacopan May Provide 3 Key Benefits: Complement Inhibition, Fewer Doses and LTB4 Inhibition to Address CNV Risk • Long-acting PAS-nomacopan is being developed with a profile that has the potential to deliver efficacy 1. Efficacy benefits of complement inhibition with a fraction of the number of
Slide 16: Patients Receiving Frequent IVI Treatments for GA and CNV Face Significant Burdens
Patients Receiving Frequent IVI Treatments for GA and CNV Face Significant Burdens That May Lead to Discontinuation • Between monthly or every other month (EOM) treatment for GA (late-stage candidates) and EOM anti-VEGF rescue for CNV, patients could face 18 injections a year1 • If bilateral, the nu
Slide 17: In CNV Treatment, Clinicians Are Compromising on Frequency of Injections with Treat & Extend
In CNV Treatment, Clinicians Are Compromising on Frequency of Injections with “Treat & Extend” • Patients typically need chronic 3 PRN (as needed) & Treat and Extend in CNV intravitreal injection (IVI) treatments such as anti-VEGF injections for sight- threatening conditions like CNV • They suffer f
Slide 18: LTB4 Inhibition May Prevent Choroidal Neovascularization
LTB4 Inhibition May Prevent Choroidal Neovascularization 2 LTB4 retina (macular) activation can lead to 1 CNV starts with inflammation in the choroid and retinal pigment epithelium (RPE) photoreceptor cells over expression of VEGF-A retinal pigment epithelium choroid • In a pre-clinical model of las
Slide 19: PAS-Nomacopan Decreased VEGF Levels As Effectively As An On-Market Anti-VEGF
PAS-Nomacopan Decreased VEGF Levels As Effectively As An On-Market Anti-VEGF In a Pre-Clinical CNV Model • PAS-nomacopan is a bispecific inhibitor of LTB4 and complement (C5) Effect PAS-nomacopan on VEGF levels in a standard pre-clinical model o Complement inhibition has shown efficacy in slowing pr
Slide 20: Thank You
THANK YOU 20
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