Periodic Reporting for period 4 - ANTHEM (ANTHEM: A Novel Tolerogenic vaccine for the treatment of antibodies in HaEMophilia)
Reporting period: 2021-02-01 to 2022-05-31
This project has significantly advanced the development of IN-3012, a novel tolerogenic vaccine that utilizes a person’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.
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.
Idogen’s technology platform allows cells from the patient’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 vaccine’s potential to eradicate NA. The project has brought Idogen to the stage where we can initiate the clinical trial in humans.
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.