#None #Bethesda #Rigel Pharmaceuticals #sickle cell disease #Fostamatinib

Rigel Pharmaceuticals' Fostamatinib Trials

Rigel Pharmaceuticals, Inc. has recently made headlines with the commencement of its first patient enrollment in a Phase 1 clinical trial for treating sickle cell disease (SCD) with fostamatinib, an innovative oral SYK inhibitor. This groundbreaking study is sponsored by the National Heart, Lung, and Blood Institute (NHLBI), a prominent branch of the National Institute of Health (NIH). The study aims to evaluate the safety and tolerability of fostamatinib, which is already known as TAVALISSE—an approved treatment in the United States for chronic immune thrombocytopenia (ITP).

The Significance of the Study

Fostamatinib's introduction into the realm of sickle cell disease treatment is significant given the high prevalence of this genetic disorder, affecting over 100,000 individuals in the United States alone. Patients with SCD often experience acute pain crises and face numerous life-threatening complications. This Phase 1 trial explores fostamatinib's potential to mitigate these challenges by inhibiting the spleen tyrosine kinase (SYK), which plays a key role in the pathophysiology of sickle cell disease.

Richard Childs, M.D., the scientific director at NHLBI, expressed optimism about the study, stating, "This is an opportunity to explore a potential new treatment option for a disease characterized by recurrent acute pain and various other complications. Previous research suggests that SYK inhibition could offer substantial benefits in terms of reducing red cell sickling and thrombo-inflammation, which are pivotal to SCD pathology."

Study Details

This open-label, dose-escalation study aims to enroll around 20 patients diagnosed with SCD (NCT05904093). Participants will receive fostamatinib starting at a dose of 100 mg, administered twice daily for two weeks, followed by a potential increase to 150 mg for an additional four weeks based on tolerability. The primary aim is to assess the safety and tolerability of the drug, while secondary objectives will look at the drug's mechanism of action, along with various markers of blood cell integrity and functionality.

The trial will occur at the NIH Clinical Center located in Bethesda, Maryland, and is funded by NIH/NHLBI with Rigel providing the necessary study materials.

Understanding Sickle Cell Disease

Sickle cell disease is genetically derived from abnormal hemoglobin, leading to red blood cells becoming rigid and crescent-shaped. This alteration causes blockages in blood vessels, leading to significant health issues, including strokes, infections, and serious pain episodes known as vaso-occlusive crises. With millions affected worldwide, the need for effective treatments is more pressing than ever.

In preclinical stages, fostamatinib has demonstrated its potential in inhibiting neutrophil extracellular traps (NETs). Studies have shown that it decreases platelet activation and aggregation, suggesting it may reduce the prothrombotic state associated with SCD without elevating bleeding risks—an important consideration in treating such a delicate patient population.

Rigel Pharmaceuticals' Commitment

Raul Rodriguez, Rigel's President and CEO, emphasized the importance of this trial, noting Rigel's dedication to improving the quality of life for patients with severe blood disorders and cancer. "This study is focused on addressing a critical unmet medical need and aligns with our mission to develop innovative treatments for debilitating conditions like sickle cell disease," he remarked.

The phase 1 trial of fostamatinib promises hope for individuals suffering from sickle cell disease, as Rigel Pharmaceuticals, along with the NHLBI, take significant steps towards potentially revolutionizing treatment protocols for this chronic condition. Stakeholders will be eagerly awaiting results that may unveil new avenues to improve the lives of patients grappling with the daily realities of SCD.