Abfero pharmaceuticals

transfusional iron overload

There is a critical need for a safe, well-tolerated oral iron chelator

ABFERO ANNOUNCES INITIATION OF
PHASE 1 STUDY FOR LEAD IRON CHELATOR

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SP-420 has the potential to provide those receiving chronic blood transfusions with a safe and tolerable treatment for maintaining iron balance.

Iron is essential to life, and our bodies maintain it in exquisite balance. Approximately 4 grams of total body iron is controlled through the absorption and excretion of about only 1–2 milligrams/day. Because each unit of blood contains 0.25 grams of iron and a red blood cell contains over a billion atoms of iron, chronic blood transfusion can lead to iron overload, a significant cause of morbidity and mortality.

Excess iron saturates the body’s storage capacity, leading to high levels of non-transferrin bound iron (NTBI)—or “free iron”—which in turn can trigger oxidative stress and cell death. Excess iron accumulation in the heart, liver, pancreas and the brain can result in heart failure, cirrhosis, liver cancer, diabetes mellitus, and growth impairment in children.

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Transfusional iron Overload (TIO)

Red blood transfusion is a life-saving treatment for a range of conditions, but because our bodies can’t excrete excess iron, people requiring chronic blood transfusion are prone to developing systemic transfusional iron overload (TIO). The majority of the TIO patient population are those living with β-thalassemia, sickle cell disease (SCD), myelodysplastic syndromes (MDS), myelofibrosis (MFS) and other rare anemias.

Transferrin Saturation Due
to Frequent Blood Transfusions

Subsequent Formation of
NTBI in Plasma

Uncontrolled Iron Loading
of Organs

Transferrin Saturation Due
to Frequent Blood Transfusions

Subsequent Formation of NTBI in Plasma

Uncontrolled Iron Loading of Organs

Current Therapies

There are currently only three FDA-approved iron chelators, but their accompanying prescription warnings underscore the critical need for a safer, more efficient therapeutic option.

The need: A Safe,
well-tolerated
oral iron chelator

There’s a real need for a safe and well tolerated oral iron chelator for patients with myelodysplastic syndrome or hemoglobinopathies dependent on red blood cell transfusions. The benefits could be enormous, both in terms of survival and quality of life.
Prof. Rob Hromas, Chairman of Abfero’s Scientific Advisory Board

SP-420 and TIO

AbFero has completed three Phase 1 clinical safety trials. One of the few orally active iron chelators, SP-420 is well-absorbed with high tissue exposure to the heart, liver, pancreas, and brain – key targets for iron removal.

With a novel dosing regimen and potentially fewer adverse effects than currently available chelators, SP-420 has the potential to provide TIO patients with a much-needed safe and tolerable treatment for maintaining iron balance.

Our technology