Corvus Pharmaceuticals Announces Initiation of Phase 2 Clinical Trial of Soquelitinib for Patients with Autoimmune Lymphoproliferative Syndrome (ALPS)

Trial for this rare genetic disease to be conducted by NIH/NIAID in partnership with Corvus

ITK inhibition intended to address dysregulation of T cells that causes ALPS and has been shown to be effective at treating the disease in preclinical models

BURLINGAME, Calif., March 12, 2025 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), has initiated a Phase 2 clinical trial of soquelitinib for the treatment of patients with autoimmune lymphoproliferative syndrome (ALPS), a rare genetic disease. The lead investigator of the trial is V. Koneti Rao, MD, FRCPA, Senior Research Physician, Primary Immune Deficiency Clinic (ALPS Clinic) at NIH Clinical Center. Dr. Rao previously presented preclinical data supporting the potential of soquelitinib in patients with ALPS. Other trial sites include Children’s Hospital of Philadelphia and Texas Children’s Cancer and Hematology Center.

“Developing safe and effective targeted treatments to address ALPS and related disorders is a priority at the NIAID since this condition was first discovered in NIAID in the early 1990s,” said Dr. Rao. “ALPS typically begins to manifest by age two and most patients live into adulthood with burdensome symptoms often related to refractory autoimmune cytopenias (low blood counts). This requires ongoing surveillance and the risk for potentially fatal conditions such as infection, hemorrhage and malignant lymphoma. We look forward to investigating the potential of ITK inhibition with soquelitinib to improve immune system balance and reduce the buildup of dysfunctional T cells in lymph nodes and spleens and autoantibodies that drive the disease.”

ALPS is most often caused by a mutation in the gene for the Fas protein, which spans the cell membrane and helps facilitate apoptosis, or programmed cell death. When this protein is missing or defective, T cells can build up and disrupt the immune system, leading to potentially debilitating symptoms and an increased risk for developing serious health conditions, including autoimmune diseases and lymphoma. There currently is no cure for ALPS and the disorder is managed by a variety of empirical methods aimed at addressing pathological changes caused by the disease, such low blood-cell counts (cytopenias), excessive lymphocyte production (lymphoproliferation), enlarged spleen or lymph nodes, and lymphoma. Treatments may include corticosteroids such as prednisone and other immunosuppressive medications to modulate the immune system, or cancer therapies to address lymphoma. 

“We are excited to expand the potential of ITK inhibition to address people with ALPS, which is a serious genetic disease that begins in childhood and gets worse due to progression during young adulthood and lacks good treatment options,” said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. “There is compelling preclinical evidence that soquelitinib may address the immune dysregulation in T cells that is a hallmark of the disease. It also bridges the biology of T cell lymphomas and autoimmunity, highlighting the role that ITK inhibition may play in restoring immune balance in lymphoma and immune disease. This adds to our conviction for the potential of soquelitinib as we continue to enroll patients in our registrational Phase 3 trial in PTCL and Phase 1 trial in atopic dermatitis.”

The Phase 2 clinical trial () is designed to enroll up to 30 patients aged 16 or older with confirmed ALPS-FAS based on genetic testing. Two dosing cohorts will be studied. The patients will receive soquelitinib doses of 200 mg or 400 mg twice per day for a period of up to 360 days. The primary endpoint of the trial is efficacy determined by reductions in splenomegaly and lymph node volumes as measured by computed tomography (CT) or positron emission tomography/computed tomography (PET/CT). Improvements in cytopenias will be assessed by complete blood count (CBC). Cytopenias are caused by autoantibodies as well as splenic sequestration that may lead to destruction of red blood cells, platelets and/or neutrophils leading to anemia, thrombocytopenia or neutropenia. Improvements in cytopenias can improve quality of life and overall health, and they also serve as a valuable biomarker associated with ALPS disease activity. Secondary endpoints include safety and tolerability.

This is the third indication under clinical development for soquelitinib, which is also in a registrational Phase 3 trial for peripheral T cell lymphoma (PTCL) and a Phase 1 trial for atopic dermatitis (AD). Corvus also plans to initiate a Phase 1 clinical trial of soquelitinib in patients with solid tumors in the second quarter 2025.

About Autoimmune Lymphoproliferative Syndrome (ALPS)

ALPS is a rare genetic disease inherited as a germline autosomal dominant pattern or due to somatic mosaicism affecting children that manifests with lymphadenopathy, splenomegaly, cytopenias (low blood counts) and autoimmunity. The disease is caused by a mutation in the Fas gene, which provides instructions for making a signaling protein involved in the induction of apoptosis to get rid of redundant lymphocytes. The mutation results in immune dysregulation due to accumulation abnormally high levels of “double negative” T cells (CD4 and CD8 double negative), which infiltrate the blood, spleen and lymphoid tissues. Fas signaling is regulated by ITK and T cell receptor signaling and patients with ALPS have an imbalance in this regulation resulting in a failure of T cells to undergo apoptosis and an accumulation of abnormal T cells.

About Soquelitinib

Soquelitinib (formerly CPI-818) is an investigational small molecule drug given orally designed to selectively inhibit ITK (interleukin-2-inducible T cell kinase), an enzyme that is expressed predominantly in T cells and plays a role in T cell and natural killer (NK) cell immune function. Soquelitinib has been shown to affect T cell differentiation and induce the generation of Th1 helper cells while blocking the development of both Th2 and Th17 cells and production of their secreted cytokines. Th1 T cells are required for immunity to tumors, viral infections and other infectious diseases. Th2 and Th17 helper T cells are involved in the pathogenesis of many autoimmune and allergic diseases. The Company believes the inhibition of specific molecular targets in T cells may be of therapeutic benefit for patients with cancers, including solid tumors, and in patients with autoimmune and allergic diseases. Recent studies have demonstrated that ITK controls a switch between the differentiation of Th17 proinflammatory cells and T regulatory suppressor cells. Inhibition of ITK leads to a shift toward T regulatory cell differentiation which has the potential to suppress autoimmune and inflammatory reactions. Based on interim results from a Phase 1/1b clinical trial in patients with refractory T cell lymphomas, which demonstrated tumor responses in very advanced, refractory, difficult to treat T cell malignancies, the Company has initiated a registrational Phase 3 clinical trial () of soquelitinib in patients with relapsed PTCL. Soquelitinib is also being investigated in a randomized placebo-controlled phase 1 clinical trial in patients with atopic dermatitis. A recent publication describing the chemistry, enzymology and biology of soquelitinib appeared in npj Drug Discovery in December 2024 and is available online at the website and on the page of the Corvus website.

About Corvus Pharmaceuticals

Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company pioneering the development of ITK inhibition as a new approach to immunotherapy for a broad range of cancer and immune diseases. The Company’s lead product candidate is soquelitinib, an investigational, oral, small molecule drug that selectively inhibits ITK. Its other clinical-stage candidates are being developed for a variety of cancer indications. For more information, visit .

Forward-Looking Statements

This press release contains forward-looking statements, including statements related to the potential of the Company’s product candidates including soquelitinib; the outlook for the Phase 2 clinical trial of soquelitinib; the potential use of soquelitinib to treat autoimmune lymphoproliferative syndrome and other immune diseases; the Company’s conduct of, enrollment in and timing of clinical trials and results, including the Company’s Phase 3 clinical trial in PTCL and Phase 1 clinical trial in atopic dermatitis; and the potential of ITK inhibition as a new approach to immunotherapy. All statements other than statements of historical fact contained in this press release are forward-looking statements. These statements often include words such as “believe,” “expect,” “anticipate,” “intend,” “plan,” “estimate,” “seek,” “will,” “may” or similar expressions. Forward-looking statements are subject to a number of risks and uncertainties, many of which involve factors or circumstances that are beyond the Company’s control. The Company’s actual results could differ materially from those stated or implied in forward-looking statements due to a number of factors, including but not limited to, risks detailed in the Company’s Quarterly Report on Form 10-Q for the three months ended September 30, 2024, filed with the Securities and Exchange Commission on November 12, 2024, as well as other documents that may be filed by the Company from time to time with the Securities and Exchange Commission. In particular, the following factors, among others, could cause results to differ materially from those expressed or implied by such forward-looking statements: the Company’s ability to demonstrate sufficient evidence of efficacy and safety in its clinical trials of its product candidates; the accuracy of the Company’s estimates relating to its ability to initiate and/or complete preclinical studies and clinical trials and release data from such studies and clinical trials; the results of preclinical studies and interim data from clinical trials not being predictive of future results; the Company’s ability to enroll sufficient numbers of patients in its clinical trials; the unpredictability of the regulatory process; regulatory developments in the United States and foreign countries; the costs of clinical trials may exceed expectations; and the Company’s ability to raise additional capital. Although the Company believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the events and circumstances reflected in the forward-looking statements will be achieved or occur, and the timing of events and circumstances and actual results could differ materially from those projected in the forward-looking statements. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and the Company undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Corvus Pharmaceuticals, Inc.

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