Soquelitinib Receives FDA Orphan Drug Designation for T-Cell Lymphomas


There are currently no FDA-approved agents for relapsed peripheral T-cell lymphoma, and soquelitinib has the potential to fill an unmet need.

  • Soquelitinib (formerly known as CPI-818) is an interleukin-2-inducible T cell kinase (ITK) inhibitor that has shown promise in treating patients with T-cell lymphomas.

  • A phase 3 clinical trial is planned to evaluate soquelitinib in patients with relapsed peripheral T cell lymphoma (PTCL).

  • Soquelitinib was well-tolerated in a phase 1/1b trial (NCT03952078) with no dose-limiting toxicities observed.

The FDA has granted an orphan drug designation to soquelitinib for the treatment of patients with T-cell lymphoma, according to Corvus Pharmaceuticals, Inc.1

Soquelitinib is a lead ITK inhibitor candidate. The oral, investigational small molecule drug is designed to selectively inhibit ITK, which is expressed in T cells. ITK also mediates T-cell and natural killer cell immune function.

A phase 3 registrational clinical trial is expected to launch in the second quarter of 2024 to evaluate soquelitinib in patients with relapsed PTCL.

“Peripheral T-cell lymphoma is an aggressive subset of non-Hodgkin lymphoma typically associated with a poor prognosis,” said Richard A. Miller, MD, co-founder, president, and chief executive officer of Corvus, in a news release. “For these patients, there is significant need for new therapies given that existing drugs provide limited efficacy and are associated with significant toxicity.

blood cells : © Dr_Microbe -

blood cells : © Dr_Microbe -

At the 2022 American Society of Hematology Annual Meeting (ASH), interim data from a phase 1/1b trial were presented, showing soquelitinib to produce tumor responses among patients with advanced, refractory T-cell lymphomas.2 Eleven patients with difficult-to-treat T-cell malignancies were included and among them, 1 patient achieved a complete response (CR), 1 patient had a nodal CR, and 2 patients had partial responses when treated with the agent twice daily at a dose of 200 mg. This dose was then identified as the optimal dose.

Further, safety findings showed soquelitinib to be well tolerated at doses up to 600 mg twice a day. There were no dose-limiting toxicities observed in the study.

Then at the 2023 ASH Annual Meeting, an analysis of the agent’s immune regulatory mechanism was presented from the ongoing, international phase 1/1b trial. Results showed soquelitinib to induce normal CD4-positive Th1 cells and CD8-positive TEMRA cells. The agent also reduced CD4-positive T regulatory cells in the tumor microenvironment in responding patients.4 Three of the 8 evaluable patients had disease progression and 4 patients achieved stable disease.

Adverse effects (AEs) that were observed in less than 1% of patients consisted of anemia, bilirubin increase, neutrophil count decrease, white blood cell decrease, and pruritus. AEs that were grade 3 or greater included 1 patient with neutrophil count decrease. With these findings, investigators believe that targeting specific molecular pathways in T cells with soquelitinib may hold potential across a variety of cancers, particularly solid tumors.1

Now, the planned phase 3 trial of soquelitinib aims to enroll 150 patients with relapsed PTCL. Patients are required to have received no more than 3 prior therapies and they will be randomly assigned in a 1:1 fashion to receive either soquelitinib at the optimal 200 mg dose or standard-of-care chemotherapy.3

The primary end point of the study is progression-free survival, and key secondary end points are objective response rate and overall survival.

“There are no FDA-approved agents for relapsed PTCL,” added Miller in the press release.1 “The orphan drug designation is an important milestone in the development of soquelitinib that reinforces the unmet need for patients with T-cell lymphoma.”

  1. Corvus Pharmaceuticals announces orphan drug designation granted to soquelitinib for the treatment of T cell lymphoma. News release. Corvus Pharmaceuticals. February 8, 2023. Accessed February 12, 2024.
  2. Song Y, Ding N, Yoon DH, et al. ITK inhibitor induces dose-dependent Th1 skewing in normal T cells and is active in refractory T cell lymphomas. Blood. 2022;140(suppl 1):8857-8859. doi:10.1182/blood-2022-159177
  3. Corvus Pharmaceuticals confirms planned initiation of soquelitinib (CPI-818) phase 3 registrational clinical trial in peripheral T cell lymphoma following meeting with FDA. News release. Corvus Pharmaceuticals, Inc. September 6, 2023. Accessed February 12, 2024.
  4. Ding N, Xie Yan, Guo Y, et al. Dynamic single-cell profiling reveals novel immune regulatory mechanism of ITK inhibitor soquelitinib in refractory T cell lymphoma. Blood. 2023;142(suppl 1):1442. doi:10.1182/blood-2023-174421
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