US FDA Provides Positive Pre-IND Feedback to Akari Therapeutics for PAS-nomacopan in Geographic Atrophy Treatment

On August 19, 2024, Akari Therapeutics made an important announcement regarding their progress in developing PAS-nomacopan, a long-acting bispecific inhibitor targeting C5 and leukotriene B4 (LTB4). The company reported that they had received positive and constructive feedback from the FDA on their Pre-IND (PIND) submission dated July 29, 2024. This feedback provides Akari with additional clarity and guidance on their final Investigational New Drug (IND) application, preclinical plans, drug manufacturing, and Phase 1 clinical strategy specifically for the intravitreal treatment of geographic atrophy (GA).

PAS-nomacopan is distinguished by its potential to offer longer dosing intervals between intravitreal injections compared to existing complement-only inhibitors. Additionally, it may reduce the risk of choroidal neovascularization (CNV), a serious condition involving the overdevelopment of blood vessels within the retina, which is typically managed with anti-vascular endothelial growth factor (VEGF) intravitreal injections.

Akari also reported that a full-scale batch of the drug substance had been successfully manufactured and released in compliance with Good Manufacturing Practices (GMP) by their partner, Wacker Biotech GmbH. This batch is intended for use in both the final IND-enabling studies and preliminary clinical development stages.

The positive regulatory feedback from the FDA, combined with the successful production of GMP-compliant materials, paves the way for Akari to submit their IND application, which they aim to do in 2025. Dr. Miles Nunn, Chief Scientific Officer of Akari Therapeutics, expressed gratitude for the FDA's feedback, noting that it aligns their final IND-enabling non-clinical plans with the agency's advice. This alignment is crucial for advancing PAS-nomacopan towards Phase 1 clinical trials for GA treatment.

Samir R Patel, MD, Interim CEO of Akari, shared his satisfaction with the progress, emphasizing the potential benefits of PAS-nomacopan. He highlighted its ability to offer longer intervals between doses and reduce CNV risk, which could result in meaningful clinical improvements for patients with GA. Patel also stated that the company looks forward to filing the IND application in 2025 to initiate the first clinical studies for this treatment.

Akari Therapeutics, trading on the Nasdaq under the ticker AKTX, is a biotechnology firm focused on developing advanced therapies for autoimmune and inflammatory diseases. Their lead asset, investigational nomacopan, functions as a bispecific recombinant inhibitor targeting both complement C5 activation and leukotriene B4 (LTB4) activity. The company is currently conducting pre-clinical research on their main product candidate, long-acting PAS-nomacopan, specifically for the treatment of geographic atrophy.

The announcement signifies a significant step forward for Akari Therapeutics in their mission to develop innovative treatments for challenging medical conditions. With the FDA's positive feedback and successful GMP-compliant production, Akari is well-positioned to advance PAS-nomacopan into clinical trials, bringing them closer to potentially offering improved therapeutic options for patients suffering from GA.