• Akari’s priority pipeline programs remain on track to begin enrollment in the registrational nomacopan Phase 3 clinical trials in pediatric and adult HSCT-TMA and start PAS-nomacopan clinical trials in geographic atrophy (GA)
• Granted orphan drug designation from the European Commission for treatment in hematopoietic stem cell transplantation, adding to existing FDA Orphan Drug, Fast Track and Rare Pediatric Disease designations
• The company established a Boston U.S. headquarters office to support expanding operations and the start of registrational Phase 3 clinical trials
• Strengthened capabilities with appointment of Beth-Anne Lang, an experienced executive with a long track record of successful regulatory approvals, as Senior Vice President, Regulatory Affairs, and industry veterans Wa’el Hashad as independent director and Wendy DiCicco as interim CFO

BOSTON and LONDON, Sept. 29, 2023 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases today announced financial results and highlights for the first half of 2023, ended June 30th. Akari’s lead asset, investigational nomacopan, is a bispecific recombinant inhibitor of complement C5 and leukotriene B4 (LTB4). Nomacopan is in Phase 3 development for pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA) and is entering Phase 3 development in adult HSCT-TMA. Long-acting PAS-nomacopan is in final pre-clinical development and is advancing toward clinical trials in geographic atrophy (GA).

“I’m delighted our Akari team continues to make steady progress on our ambitions for investors and the patients who are waiting with urgent unmet needs,” said Rachelle Jacques, Akari President and CEO. “We believe we are building strong momentum as we prepare for the start of enrollment in the registrational portion of our nomacopan pediatric and adult Phase 3 clinical trials in HSCT-TMA and move our PAS-nomacopan geographic atrophy development program toward clinical trials.”

HSCT-TMA

Akari remains on track to start enrollment by the end of 2023 in the registrational Phase 3 study of nomacopan in pediatric HSCT-TMA, which is expected to generate safety and efficacy data that may support U.S. and European regulatory filings for potential marketing authorization in these regions.

Consensus guidelines were published in early 2023 by an international panel of experts that harmonize diagnostic criteria and support earlier screening and diagnosis in the care of patients. The design of Akari’s HSCT-TMA Phase 3 clinical trials has been significantly informed by these consensus criteria for earlier diagnosis of high-risk (severe) patients. Adult study design will be an important topic of discussion during a Type C meeting with the U.S. Food and Drug Administration (FDA) scheduled for November 15, 2023.

A case study, Clinical Response to Nomacopan in the Paediatric HSCT-TMA Setting, was presented as a late-breaker at The Transplantation & Cellular Therapy Tandem Meetings in February 2023 and as a poster in April 2023 at The European Society for Blood and Marrow Transplantation (EBMT). A 6-year-old male HSCT-TMA patient at Royal Manchester Children’s Hospital, Manchester University NHS Foundation Trust in Manchester, United Kingdom was enrolled in the nomacopan Phase 3 Part A clinical trial, treated with nomacopan and discharged home from the hospital.  

Akari was granted orphan drug designation from the European Commission for treatment in hematopoietic stem cell transplantation. This designation is an important addition to the FDA Orphan Drug, Fast Track and Rare Pediatric Disease designations for nomacopan for the treatment of pediatric HSCT-TMA. With the Rare Pediatric Disease Designation, Akari is eligible to receive a Priority Review Voucher (PRV) upon approval that it can either redeem for priority review of a subsequent marketing application for a different product or sell to a third party.

Akari is also moving forward into a registrational Phase 3 double-blind placebo-controlled clinical trial of nomacopan in adult HSCT-TMA with enrollment expected to begin in 2024 (subject to funding).

Regulatory authorities in Poland (Office for Registration of Medicinal Products, Medical Devices and Biocidal Products/URPL) and the U.K. (Medicines & Healthcare products Regulatory Agency/MHRA) approved amendments to the company’s Investigational Medicinal Product Dossier (IMPD) and Clinical Trial Authorization (CTA), respectively, for clinical use of the third-generation drug substance manufacturing process that increases the final yield of nomacopan by at least 5-fold. 

Geographic Atrophy (GA)

Akari completed evaluation of long-acting PAS-nomacopan candidates and based on extensive pre-clinical work selected a single drug candidate to move forward into clinical trials for treatment of GA. The selected version has a product profile with characteristics important for a GA therapy, including fully active drug potency, planned small (