The National Institute of Allergy and Infectious Diseases (NIAID), part of the NIH, has launched a Phase 2 clinical trial of Corvus Pharmaceuticals’ soquelitinib for autoimmune lymphoproliferative syndrome (ALPS). This rare genetic disorder, often manifesting in early childhood, causes a buildup of T cells due to a faulty Fas protein, leading to immune system disruption and serious health risks like lymphoma and autoimmune diseases. The trial, led by Dr. V. Koneti Rao of the NIH, will explore soquelitinib’s potential to inhibit ITK, an enzyme crucial for T cell function, and restore immune balance.
This trial represents a significant advancement in ALPS treatment. Currently, ALPS management relies on addressing symptoms with corticosteroids and immunosuppressants, or even cancer therapies for lymphoma, but there is no cure. Soquelitinib’s targeted approach to correcting the underlying immune dysregulation offers a potentially transformative treatment option for patients who currently face lifelong management of a debilitating disease. The collaboration between the NIH and Corvus leverages the expertise of both institutions, increasing the likelihood of successful development and ultimately providing a much-needed targeted therapy.
The Phase 2 trial will enroll up to 30 patients aged 16 and older with genetically confirmed ALPS-FAS. Patients will receive either 200 mg or 400 mg of soquelitinib twice daily for up to 360 days. The primary endpoint focuses on reductions in spleen and lymph node sizes, key indicators of ALPS severity. Secondary endpoints assess improvements in cytopenias (low blood counts) and overall safety and tolerability. This trial adds to the ongoing investigations of soquelitinib in a Phase 3 trial for peripheral T cell lymphoma and a Phase 1 trial for atopic dermatitis, further demonstrating the drug’s potential across a range of immune-related conditions.
The initiation of this Phase 2 trial marks a crucial step toward a potential new therapeutic avenue for ALPS. Positive results could lead to the first targeted treatment for this rare disease, significantly improving the quality of life for patients. Furthermore, the findings could broaden our understanding of ITK inhibition and its applications in treating other immune disorders, paving the way for further advancements in immunotherapy.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
