NIH and Corvus test drug for rare genetic disorder

Published 12/03/2025, 13:06
NIH and Corvus test drug for rare genetic disorder

BURLINGAME, Calif. - The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), has launched a Phase 2 clinical trial in collaboration with Corvus Pharmaceuticals (market cap: $280.8M) to evaluate the effectiveness of soquelitinib in treating autoimmune lymphoproliferative syndrome (ALPS), a rare genetic condition. According to InvestingPro data, Corvus maintains a strong financial position with more cash than debt on its balance sheet, positioning it well for clinical development programs. The trial, led by Dr. V. Koneti Rao at the NIH Clinical Center, will also take place at Children’s Hospital of Philadelphia and Texas Children’s Cancer and Hematology Center.

ALPS typically emerges by age two and can lead to severe health issues, including autoimmune diseases and lymphoma. The disease is characterized by mutations in the Fas gene, which disrupts the programmed cell death of T cells, causing them to accumulate and impair the immune system.

Soquelitinib, an oral medication designed to selectively inhibit ITK, a kinase involved in T cell and natural killer cell function, has shown promise in preclinical studies for addressing the immune dysregulation in ALPS. The Phase 2 trial aims to enroll up to 30 patients aged 16 or older with genetically confirmed ALPS-FAS. Participants will receive daily doses of soquelitinib, with the primary goal of the study being to reduce spleen and lymph node enlargement and improve blood count-related symptoms.

This is the third indication for soquelitinib, which is also undergoing a Phase 3 trial for peripheral T cell lymphoma (PTCL) and a Phase 1 trial for atopic dermatitis (AD). Corvus Pharmaceuticals plans to initiate a Phase 1 trial for solid tumors in the second quarter of 2025. The company’s stock has shown remarkable strength with a 116% return over the past year, though InvestingPro analysis indicates current valuation metrics suggest the stock is slightly overvalued.

The current management of ALPS involves various empirical treatments, including corticosteroids and other immunosuppressive medications. The new trial seeks to offer a more targeted therapeutic approach, potentially improving the quality of life for patients with ALPS. Analysts maintain a bullish outlook on Corvus, with price targets significantly above current levels. For deeper insights into Corvus’s financial health and growth potential, including 8 additional ProTips and comprehensive valuation metrics, check out the full research report available on InvestingPro.

The information for this article is based on a press release statement from Corvus Pharmaceuticals, Inc.

In other recent news, Corvus Pharmaceuticals has reported interim results from its Phase 1 trial of soquelitinib, targeting moderate to severe atopic dermatitis. The trial, which is randomized and placebo-controlled, showed promising safety and efficacy results, with significant improvements in patients receiving the drug compared to the placebo group. Notably, 26% of patients in the treatment group achieved an Investigator Global Assessment score of 0 or 1, and 37% reached an Eczema Area and Severity Index score of 75 after 28 days. Mizuho Securities and Oppenheimer have both maintained their Outperform ratings for Corvus Pharmaceuticals, with price targets of $12.00 and $14.00, respectively, highlighting the drug’s competitive edge over current treatments. The company also announced that Samlyn Capital exercised common stock warrants early, providing approximately $12.7 million in cash proceeds. Furthermore, Corvus is preparing to share more data on soquelitinib’s potential at a conference call and webcast. The company’s ongoing research includes a Phase 3 trial for relapsed peripheral T cell lymphoma, indicating a broader scope of application for soquelitinib. These developments underscore the potential of soquelitinib as a treatment for various immune diseases.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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