Akari Therapeutics, Plc (NASDAQ: AKTX) is a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases. Akari’s lead asset, investigational nomacopan, is a bispecific recominant inhibitor of complement C5 and leukotriene B4 (LTB4) activity.
Novel C5+LTB4 inhibitor
Nomacopan is a unique asset inhibiting 2 codependent, proinflammatory targets; complement C5 and leukotriene B4 (LTB4)
Broad potential
Potential for use in several diseases; commercial flexibility due to multiple routes of administration (subcutaneous, topical, intravitreal, IV)
Robust clinical dataset
Extensive clinical and safety data from multiple clinical trials
Akari’s pipeline includes Phase 3 clinical trials investigating nomacopan for both pediatric and adult hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA).
HSCT-TMA pediatric & adult Phase 3 clinical trials
No approved therapies for pediatric or adult HSCT-TMA and an ~80% mortality in high-risk (severe) patients; registrational Phase 3 clinical trial in pediatric HSCT-TMA is on track to begin enrollment by end of 2023; FDA Orphan, Fast Track, and Rare Pediatric Disease (eligible at approval for a Priority Review Voucher) designations and European Commission orphan drug designation; Akari is moving forward into a Phase 3 double-blind placebo-controlled clinical trial of nomacopan in adult HSCT-TMA and will begin enrollment in 2024; FDA Orphan Drug designation granted for adult HSCT-TMA
Akari’s pipeline includes a pre-clinical program developing long-acting PAS-nomacopan as a potential treatment for geographic atrophy (GA).
GA development advancing into clinical trials
Bispecific (complement C5+LTB4 inhibition) PAS-nomacopan is being developed with the potential to address three areas of significant need in GA: complement inhibition, fewer needle injections into the eye, and reduction of the risk of patients developing sight-threatening choroidal neovascularization (CNV), a safety risk associated with complement-only inhibitors approved for GA treatment; pre-clinical program investigating PAS-nomacopan in GA with target dose interval of 3 months or longer without increased risk of CNV; the PAS-nomacopan candidate that will move forward into clinical trials for treatment of GA has been selected (fully active drug potency, planned small (<100μL) injection volume, viscosity enabling intravitreal injection with a fine needle, pre-clinical half-life that supports a potential clinical dose interval of 3 months or longer); Akari is on track for the start of clinical trials in 2024
