Rigel Pharmaceuticals has enrolled the first subject in a Phase I trial of its oral spleen tyrosine kinase (SYK) inhibitor, fostamatinib, to treat sickle cell disease (SCD).
The trial aims to assess the tolerability and safety of the therapy’s escalating doses.
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Being sponsored by the National Institutes of Health’s (NIH) National Heart, Lung, and Blood Institute (NHLBI), the open-label trial anticipates recruiting nearly 20 SCD subjects.
Funding for the trial is also provided by NIH/NHLBI while Rigel is providing material for the study.
NHLBI’s sickle cell branch chief and senior investigator Swee Lay Thein is leading the trial, which is being conducted at the NIH Clinical Center in Bethesda, Maryland.
A 100mg dosage of the therapy will be administered to the subjects two times a day for 14 days, with a possible increase to 150mg for an extra 28 days based on tolerability.
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By GlobalDataAdditionally, the trial will explore the mechanisms by which fostamatinib, through SYK inhibition, may impact red blood cell membrane integrity, sickling kinetics rate, and other factors relevant to SCD pathology.
Rigel Pharmaceuticals president and CEO Raul Rodriguez said: “We are excited to support another important study conducted by the NIH/NHLBI for fostamatinib, as they investigate SYK inhibition and its potential to benefit patients with sickle cell disease, a devasting, lifelong condition.
“The study focuses on an area of critical unmet need and contributes to Rigel’s mission to improve the lives of patients with haematologic disorders and cancer.”
Known as Tavalisse in the US market, fostamatinib is approved for treating chronic immune thrombocytopenia (ITP) in adults who have not responded to previous treatments.
SCD leads to abnormal haemoglobin production, causing red blood cells to become rigid and sickle-shaped. This can result in serious complications such as strokes, infections, and multi-organ dysfunction.
Last September, the company announced the first subject enrolment in a Phase Ib/II trial of Rezlidhia combined with decitabine and venetoclax for individuals with mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukaemia (AML).
