Akari Therapeutics Receives Positive and Constructive Pre-IND Feedback from US FDA for PAS-nomacopan in Treatment of Geographic Atrophy
Akari Therapeutics (AKTX) has received positive Pre-IND feedback from the FDA for PAS-nomacopan, a long-acting bispecific inhibitor of C5 and LTB4 for treating geographic atrophy (GA). The feedback provides clarity on preclinical plans, drug manufacturing, and Phase 1 clinical strategy. PAS-nomacopan potentially offers longer dose intervals between intravitreal injections and reduced choroidal neovascularization (CNV) risk compared to current complement inhibitors. A GMP-compliant batch of drug substance has been manufactured by Wacker Biotech GmbH for IND-enabling studies and initial clinical development. Akari plans to submit the IND in 2025, paving the way for Phase 1 clinical studies in GA treatment.
Akari Therapeutics (AKTX) ha ricevuto un feedback positivo Pre-IND dalla FDA per PAS-nomacopan, un inibitore bispecifico a lunga durata d'azione di C5 e LTB4 per il trattamento dell'atrofia geografica (GA). Il feedback fornisce chiarezza sui piani preclinici, la produzione del farmaco e la strategia clinica della Fase 1. PAS-nomacopan offre potenzialmente intervalli di dosaggio più lunghi tra le iniezioni intravitreali e un rischio ridotto di neovascolarizzazione coroidale (CNV) rispetto agli attuali inibitori del complemento. Un lotto conforme GMP della sostanza medicinale è stato prodotto da Wacker Biotech GmbH per studi abilitanti l'IND e lo sviluppo clinico iniziale. Akari prevede di presentare l'IND nel 2025, aprendo la strada per studi clinici di fase 1 nel trattamento della GA.
Akari Therapeutics (AKTX) ha recibido una retroalimentación positiva Pre-IND de la FDA para PAS-nomacopan, un inhibidor bispecífico de acción prolongada de C5 y LTB4 para tratar la atrofia geográfica (GA). La retroalimentación proporciona claridad sobre los planes preclínicos, la fabricación del medicamento y la estrategia clínica de la Fase 1. PAS-nomacopan ofrece potencialmente intervalos de dosis más largos entre inyecciones intravítreas y un riesgo reducido de neovascularización coroidea (CNV) en comparación con los actuales inhibidores del complemento. Un lote conforme a GMP de la sustancia farmacéutica ha sido fabricado por Wacker Biotech GmbH para estudios que habilitan el IND y el desarrollo clínico inicial. Akari planea presentar el IND en 2025, allanando el camino para estudios clínicos de Fase 1 en el tratamiento de la GA.
Akari Therapeutics (AKTX)는 FDA로부터 PAS-nomacopan에 대한 긍정적인 Pre-IND 피드백을 받았습니다. PAS-nomacopan은 C5 및 LTB4의 작용을 길게 하는 이중 특이적 억제제로, 지리적 위축(GA) 치료를 위해 개발되었습니다. 이 피드백은 전임상 계획, 약물 제조와 1상 임상 전략에 대한 명확성을 제공합니다. PAS-nomacopan은 현재의 보Complement 억제제에 비해 더 긴 용량 간격과 티증식혈관(Choroidal Neovascularization, CNV) 위험 감소를 제공합니다. Wacker Biotech GmbH가 GMP 규정에 맞는 약물 배치를 제조하여 IND 승인 연구와 초기 임상 개발을 위한 기초로 삼았습니다. Akari는 2025년에 IND를 제출할 계획이며, GA 치료를 위한 1상 임상 연구를 위한 길을 엽니다.
Akari Therapeutics (AKTX) a reçu un retour positif Pre-IND de la FDA concernant PAS-nomacopan, un inhibiteur bispéculatif à action prolongée de C5 et LTB4 destiné à traiter l'atrophie géographique (GA). Ce retour apporte des précisions sur les plans précliniques, la fabrication du médicament et la stratégie clinique de phase 1. PAS-nomacopan offre potentiellement des intervalles de dose plus longs entre les injections intravitréennes et réduit le risque de néovascularisation choroïdienne (CNV) par rapport aux inhibiteurs de complément actuels. Un lot conforme aux GMP de la substance médicamenteuse a été fabriqué par Wacker Biotech GmbH pour des études d'habilitation IND et le développement clinique initial. Akari prévoit de soumettre l'IND en 2025, ouvrant ainsi la voie aux études cliniques de phase 1 dans le traitement de la GA.
Akari Therapeutics (AKTX) hat von der FDA ein positives Pre-IND-Feedback für PAS-nomacopan erhalten, einen langwirksamen bispezifischen Inhibitor von C5 und LTB4 zur Behandlung der geografischen Atrophie (GA). Das Feedback bietet Klarheit zu den präklinischen Plänen, der Arzneimittelherstellung und der klinischen Strategie der Phase 1. PAS-nomacopan bietet potenziell längere Dosisintervalle zwischen intravitrealen Injektionen und ein reduziertes Risiko für choroidale Neovaskularisation (CNV) im Vergleich zu aktuellen Komplementinhibitoren. Ein GMP-konformes Batch des Arzneimittels wurde von der Wacker Biotech GmbH für IND-fähige Studien und die initiale klinische Entwicklung hergestellt. Akari plant, das IND im Jahr 2025 einzureichen, um den Weg für klinische Studien der Phase 1 zur Behandlung von GA zu ebnen.
- Positive FDA feedback on PAS-nomacopan development plans
- Successful manufacturing of GMP-compliant drug substance batch
- Potential for longer dose intervals between intravitreal injections
- Possible reduction in choroidal neovascularization (CNV) risk
- Clear path forward for IND submission in 2025
- IND submission not expected until 2025, indicating a lengthy development timeline
The FDA's positive feedback on Akari's PAS-nomacopan for Geographic Atrophy (GA) treatment is a significant milestone. The bispecific inhibitor of C5 and LTB4 shows promise in two key areas: potential for longer dose intervals and reduced risk of choroidal neovascularization (CNV). These advantages could address major pain points in current GA treatments.
The successful GMP manufacturing of a full-scale batch is crucial, as it will be used for both IND-enabling studies and initial clinical development. This streamlines the path to clinical trials, potentially accelerating the drug's development timeline. However, with the IND submission expected in 2025, it's important to note that market entry is still years away, assuming successful trials.
While promising, investors should remain cautious. The road from pre-IND to market is long and fraught with potential setbacks. The true test of PAS-nomacopan's efficacy and safety will come in clinical trials.
PAS-nomacopan's potential to offer longer intervals between intravitreal injections is a game-changer for GA treatment. Current therapies often require monthly injections, which is burdensome for patients and healthcare systems. If PAS-nomacopan can deliver on this promise, it could significantly improve patient compliance and quality of life.
The dual inhibition of C5 and LTB4 is particularly intriguing. While C5 inhibition is a known approach in GA treatment, the addition of LTB4 inhibition could provide added benefits in reducing inflammation and potentially slowing disease progression. The possible reduction in CNV risk is another critical advantage, as CNV is a serious complication in GA patients.
However, it's important to see how these theoretical benefits translate into clinical outcomes. The upcoming Phase 1 trials will be pivotal in establishing the drug's safety profile and initial efficacy signals.
Akari's progress with PAS-nomacopan positions the company well in the competitive landscape of GA treatments. The positive FDA feedback and successful GMP manufacturing are significant de-risking events, potentially making Akari more attractive to investors and potential partners.
However, the GA market is becoming increasingly crowded, with several large pharma companies already having approved treatments or late-stage candidates. Akari will need to demonstrate clear differentiation and superior efficacy to capture market share.
The company's focus on addressing key unmet needs - longer dosing intervals and reduced CNV risk - is strategically sound. If successful, PAS-nomacopan could command premium pricing and potentially become a preferred treatment option. Investors should monitor upcoming milestones, particularly the IND submission in 2025 and subsequent clinical trial results, as these will be critical valuation inflection points for Akari.
Pre-IND meeting with FDA completed with positive feedback on clinical and nonclinical development plans for PAS-nomacopan in treatment of geographic atrophy
BOSTON and LONDON, Aug. 19, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics announced today that the company has received positive and constructive Pre-IND (PIND) feedback from the FDA on July 29, 2024, which provides additional clarity on Akari’s final Investigational New Drug Application (IND)- enabling preclinical plans, drug manufacturing and Phase 1 clinical strategy for long-acting PAS-nomacopan for intravitreal treatment of geographic atrophy (GA).
PAS-nomacopan is a long-acting bispecific inhibitor of C5 and leukotriene B4 (LTB4) product candidate that Akari believes has the potential for longer dose intervals between intravitreous injections than currently approved complement only inhibitors, as well as potential reduction of the choroidal neovascularization (CNV) risk that is associated with approved inhibitors. CNV is a sight-threatening over development of blood vessels within the retina, which is typically treated with additional anti-vascular endothelial growth factor (VEGF) intravitreal injections.
A full-scale batch of drug substance was recently successfully manufactured and released under applicable Good Manufacturing Practices (GMP) conditions by our manufacturing partner, Wacker Biotech GmbH, and is planned to be used for both the final IND-enabling studies and initial clinical development.
The regulatory feedback from the FDA and production of GMP material enables a clear path forward to submitting an IND, with the IND expected to be submitted in 2025.
Dr. Miles Nunn, Chief Scientific Officer of Akari Therapeutics said, “We thank the FDA for its positive and constructive feedback,” and added, “The PIND feedback enables us to align our final IND-enabling non-clinical plans with the FDA’s advice, strengthening PAS-nomacopan’s progress towards Phase 1 clinical studies for treatment of GA.”
Samir R Patel, MD, Interim CEO of Akari said, “I’m very pleased with the progress that is being made in bringing PAS-nomacopan closer to the clinic as we believe it has the potential to offer an improved therapy for treatment of GA via longer dose intervals and reduction in CNV risk which could offer clinically meaningful benefits to patients.” He added, “We look forward to filing an IND application in 2025 for our first clinical studies of PAS-nomacopan for treatment of this disease.”
About Akari Therapeutics
Akari Therapeutics, plc (Nasdaq: AKTX) is a biotechnology company developing advanced therapies for autoimmune and inflammatory diseases. Akari’s lead asset, investigational nomacopan, is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity. The company is conducting pre-clinical research for its lead product candidate, long-acting PAS-nomacopan in geographic atrophy (GA). For more information about Akari, please visit akaritx.com.
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Investor Contact:
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FAQ
What is the purpose of PAS-nomacopan in Akari Therapeutics' (AKTX) development pipeline?
When does Akari Therapeutics (AKTX) plan to submit the IND for PAS-nomacopan?
What advantages does PAS-nomacopan offer over current geographic atrophy treatments?
Has Akari Therapeutics (AKTX) completed manufacturing of PAS-nomacopan for clinical trials?
