Petadeferitrin

Pharmacosmos is developing petadeferitrin, a novel iron chelator, as a new treatment for patients with transfusional iron overload.

Patients who receive frequent blood transfusions often accumulate significant amounts of iron in their bodies. Petadeferitrin addresses this issue by binding to iron in a 2:1 ratio, allowing the body to excrete the resulting complex and thereby eliminate excess iron.

A chelator with potential in tissue penetration and efficacy

Petadeferitrin, a small molecule chelator, features a unique chemical structure distinct from existing chelators. In preclinical models, it has demonstrated significant tissue penetration in key target organs such as the liver and heart, and it has also shown potential for enhanced safety and efficacy. Moreover, it offers a more convenient treatment option with a dosing regimen of three times a week.

Need for new iron chelation therapy

There is a substantial unmet medical need for new iron chelation therapies. Up to 40% of patients have been reported to exhibit persistently high liver iron levels above treatment targets recommended by current guidelines and at levels associated with increased risk of iron overload-induced complications1.

Pharmacosmos is seeking partners in markets where we do not currently have affiliates, including Japan, Southeast Asia, Latin America, Southern Europe, and the Middle East.

  1. ShahF,TelferP,VelangiM,PanchamS,WynnR,PollardS, et al.Routine management,healthcare resource use and patient and carer - reported outcomes of patients with transfusion - dependent β-thalassaemia in the United Kingdom: A mixed methods observational study. eJHaem.2021;2:738–749.https://doi.org/10.1002/jha2.282

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