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BIA-Brukerstyrt innovasjonsarena

Development of a monoclonal antibody to treat systemic sclerosis

Alternativ tittel: Utvikling av et monoklonalt antistoff for å behandle systemisk sklerose

Tildelt: kr 16,0 mill.

Prosjektets mål var å utvikle et monoklonalt antistoff til en klinisk legemiddelkandidat mot sjeldne fibrosesykdommer. I løpet av prosjektperioden har vi etablert produksjon av antistoffet i tråd med regulatoriske krav for kliniske studier og gjennomført sikkerhetsstudier (toxikologi) i dyr. I 2023 i tråd med tidsplanen initierte vi en klinisk fase-1 studie. Vi har fullført doseringen på friske frivillige og fulgt alle deltakerne opp i 100 dager etter doseringen. Preliminære resultater viser at legemiddelet er trygt og tilsynelatende ikke hadde noen alvorlige bivirkninger i deltakerne i studien. Vi har også gjennomført prekliniske studier for å karakterisere mekanismen ytterligere. En av disse studiene ble i år publisert i Annals of The Rheumatic Diseases, som er det vitenskapelige tidsskriftet innenfor revmatologi som har høyest Impact Factor. Prosjektet har fullt ut forløpt på tidslinjene som ble lagt i 2019.

Outcomes: The project has enabled the development of a monoclonal antibody for the treatment of rare fibrotic diseases including systemic sclerosis and pulmonary fibrosis. The project has contributed to the successful completion of preclinical development of our drug candidate and the initiation of a clinical phase 1 study. This progress the company has made over the project period recently resulted in a large series A fundraising (75 million EUR) and the merger with a Dutch biotech company to form a new company called Calluna Pharma. The lead candidate of Calluna Pharma is the monoclonal antibody developed in part with the support of the grant. Impact: Over the course of the project period, the company has added three additional employees in Norway and is now and established Norwegian biotech company with plans for further hires in the near term.

Arxx Therapeutics is a Norwegian start-up biotechnology company that aims to develop a disease-modifying therapeutic for systemic sclerosis and other rare fibrotic diseases by targeting a novel biological mechanism with a monoclonal antibody developed by the founding scientists of the company. Systemic sclerosis is a rare, progressive and life-threatening disease that afflicts mostly women (80%) between the ages of 30 and 60, causes extensive fibrosis of multiple organs (eg skin, lungs, heart, gastro-intestinal system) and leads to premature morbidity and mortality. There are currently no approved disease-modifying agents available and the unmet need is extremely high. The therapeutic target is a pro-fibrotic and pro-inflammatory mediator that has been demonstrated to play a key role in the development and progression of fibrosis. Arxx has a patent-protected high affinity, selective monoclonal antibody that effectively neutralizes the target both in vitro and in vivo and confers anti-inflammatory and anti-fibrotic effects. The aim of the project is to conduct studies that will enable the monoclonal antibody to be ready for clinical testing in patients. The activities planned include: 1) Additional pre-clinical studies (in vivo and in vitro) to further characterize the mechanism of action; 2) Cell-line development, bioanalytical work and toxicology studies in animals to establish the safety of the drug prior to testing in humans; 3) A first-in-human study to establish the safety and pharmacokinetic properties of the drug. The activities will be performed at the Oslo University Hospital (Professor Finn Olav Levy) and University College London (Professor Christopher Denton) and by a world-leading contract research organization.

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BIA-Brukerstyrt innovasjonsarena