Following the November 2021 acquisition of AbFero Pharmaceuticals Inc including the drug candidate SP-420, Pharmacosmos A/S has initiated a Phase II, proof-of-concept, dose-finding trial in patients with transfusion-dependent β-thalassemia.
Transfusional iron overload (TIO) is a common complication in patients that receive frequent blood transfusions. Living with iron overload puts patients at risk for serious complications such as liver disease, heart failure, and diabetes mellitus. Despite current treatment options, many patients remain insufficiently controlled with 20-30% of patients still exhibiting moderate or severe iron overload of the heart or liveri. The development of SP-420, a novel oral iron chelator, may offer a new favorable treatment alternative in a convenient thrice-weekly dosing regimen.
This week, the first patient has been enrolled into the trial which is planned to include patients with transfusion-dependent β-thalassemia from countries in North America, Europe, Middle East, Asia and Australia.
“We are very pleased to advance SP-420 into this Phase II trial, which is an important step towards the development of needed improved treatment options for patients with transfusional iron overload”, said Lars Lykke Thomsen, Chief Medical Officer at Pharmacosmos.
The primary objective of the Phase II trial is to assess the efficacy of SP-420 in removing iron from the body at three different dose levels. The main efficacy outcome will be assessed on the basis of the amount of iron removed from the liver as measured by Magnetic Resonance Imaging (MRI). Assessment of safety and tolerability and a range of other efficacy outcome measures are included as secondary outcomes.
The trial is designed as a three-arm trial with an initial dose-escalation phase, and a total dosing-period of 48 weeks. The trial is planned to enroll a total of 90 patients across participating centers spanning countries in North America, Europe, Middle East, Asia, and Australia. For more information about the trial please visit clinicaltrials.govii
Transfusional iron overload is a common clinical challenge in many hematological patients that receive frequent blood transfusions, most commonly observed in diseases such as β-thalassemia, sickle cell disease and myelodysplastic syndrome.
With frequent transfusions, the iron from red blood cells in blood products, accumulates in the body and is deposited in different organs such as the heart, liver and endocrine glands. iii Each unit of blood contains between 200-250 mg iron, and since the body has no natural mechanism for excretion of iron (apart from minor epithelial losses) iron will begin to accumulate in the body. These patients need close monitoring and usually treatment for iron overload upon 10-20 blood transfusions.
Control of iron overload is an important treatment objective in these patients. If poorly controlled, poorly compliant or left untreated, long-term iron overload can cause serious organ damage and elevated iron levels correlate with worse long-term prognosis. iv v vi vii
β-thalassemia is a rare inherited disorder characterized by reduced or no production of hemoglobin and chronic anemia, due to ineffective erythropoiesis and peripheral hemolysis. Consequently, patients have a lifelong need for blood transfusions.
The disease is associated with a number of clinical complications stemming both from the underlying disorder and therapies provided. Severity of symptoms is dependent on underlying genetics, as well as consequences and extent of control achieved by blood transfusions and iron chelation therapy. Ineffective erythropoiesis leads to bone marrow expansion and associated bone changes, pain and characteristic bone deformities. If poorly controlled, patients are at increased risk of infections and vascular events, and may exhibit hepatosplenomegaly, liver fibrosis and cirrhosis, as well as endocrinopathies and growth and pubertal delays.
Iron overload remains one of the most common and severe complications in patients with transfusion-dependent β-thalassemia. Complications associated with iron overload include endocrine dysfunction, hepatic pathology, and cardiac disease. In the absence of a well-controlled treatment patients are at increased risk of cardiac arrhythmias, heart failure, and early mortality.
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i Shah et al. (2021) eJHaem. 2:738-749
ii ClinicalTrials.gov Identifier: NCT05693909
iii Saliba & Taher (2015) Hematology, 20:5, 311-312
iv Moukalled et al. (2018) Cancer 124:3979-3989
v Delea et al. (2007) Transfusion. 47(10):1919-1929.
vi Gabutti & Piga (1996) Acta Haematol. 95(1):26-36.
vii Porter et al. (2011) Int J Hematol 94:453-460
Pharmacosmos Group, headquartered in Holbaek, Denmark, and founded in 1965, is a highly specialised company focused on carbohydrate chemistry and a global leader in the development of innovative treatments for patients suffering from iron deficiency and iron deficiency anaemia. With companies in the UK, Nordics, Germany, the USA, and China, as well as through partners, Pharmacosmos markets its products around the world. With a strong and ongoing commitment to R&D, Pharmacosmos is able to leverage a unique carbohydrate production platform along with deep expertise in the synthesis of iron-carbohydrate complexes. The Pharmacosmos Group has more than 500 employees.
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