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Report

Teaser, summary, work performed and final results

Periodic Reporting for period 2 - ANTHEM (ANTHEM: A Novel Tolerogenic vaccine for the treatment of antibodies in HaEMophilia)

Teaser

Summary

This project will develop and validate IN-3012, a novel tolerogenic vaccine that utilizes a personâ€™s own cells to treat neutralizing antibodies (NA) in Haemophilia A patients who have failed immune tolerance induction (ITI).
Haemophilia A patients are given the blood clotting protein, factor VIII as standard of care. However, a serious consequence is the development of NA towards factor VIII leading to therapy resistance. This leads to crippling arthropathy, other morbidities and high mortality. ITI is the most used treatment of this condition but 35% of patients do not respond and are in a vulnerable situation.
Haematologists and KOLs emphasise the pressing need for safe and effective technologies to overcome NA in Haemophilia A, which is not possible today. The estimated mean annual cost of a Haemophilia patient in Europe is over EUR 117,000 EUR with the mean lifetime cost reaching EUR 7.8 million. This is substantially higher in patients who have failed ITI. Therefore, while this condition has low prevalence, it exerts heavy burden on healthcare budgets and is thus a top EU health policy priority.

Work performed

Idogenâ€™s technology platform allows cells from the patientâ€™s blood to be reprogrammed outside the body to dendritic cells with the capacity to specifically counteract adverse immune reactions. The reprogrammed cells are transferred back to the patient. IN-3012 uses this mechanism to create tolerance to factor VIII.
The research performed to date strongly supports the vaccineâ€™s potential to eradicate NA. The proposed project will bring IN-3012 through a Phase I/II clinical trial in humans. The safety and preliminary efficacy data will constitute the basis for further clinical development and commercialisation of the product.
Idogenâ€™s strategic focus lies in establishing IN-3012 as a novel treatment in Haemophilia A patients having failed ITI. This will constitute the cornerstone of Idogenâ€™s accelerated growth. This project is the key step to realize the strategy.

Final results

The proposed IN-3012 treatment is designed to eradicate NA and allow factor VIII replacement therapy to work effectively. All end users ultimately demand a safe, tolerable and clinically validated vaccine for the treatment of NA in haemophilia A patients that have failed ITI. Patients require a vaccine that can overcome NA long-term and allow current treatments to enable normal blood clotting. This essentially means recognition of factor VIII to reduce mortality and morbidity rates, reduce the length of time patients spend receiving treatment and drastically reduce the current extremely high healthcare costs. These characteristics of the therapy are highly sought after by medical professionals and patients alike. The project is positioned to satisfy this need.