Efficacy

Xpovio (Selinexor) Efficacy in Treating Lymphoma

Xpovio (selinexor) is an oral selective inhibitor of nuclear export (SINE) compound that has shown efficacy in the treatment of certain types of lymphoma. It works by inhibiting the nuclear export protein XPO1, leading to the accumulation of tumor suppressor proteins in the cell nucleus and inducing cancer cell death. In July 2020, the U.S. Food and Drug Administration (FDA) approved Xpovio for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. This approval was based on the SADAL study, a single-arm, open-label, multicenter trial that demonstrated a meaningful overall response rate (ORR) in patients treated with selinexor.

The efficacy of Xpovio in the SADAL study was specifically measured by the ORR, which was the proportion of patients achieving either a complete response (CR) or partial response (PR) as assessed by an independent review committee. The results showed that Xpovio achieved an ORR of 29%, with 13% of patients reaching a CR. The median duration of response (DOR) for those who achieved a response was 23 months, indicating a potential for long-term benefit in a subset of patients. These results suggest that Xpovio can be an effective treatment option for patients with relapsed or refractory DLBCL, especially for those who have limited treatment options.

Ukoniq (Umbralisib) Efficacy in Treating Lymphoma

Ukoniq (umbralisib) is a novel, oral, dual inhibitor of PI3K-delta and CK1-epsilon, which has shown efficacy in treating certain types of lymphoma. It was approved by the FDA in February 2021 for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based regimen and for adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy. The approval was based on the results from the UNITY-NHL trial, a multicenter, open-label, single-arm trial that demonstrated significant ORR in both MZL and FL patient populations.

In the UNITY-NHL trial, the efficacy of Ukoniq was evaluated by ORR, with responses assessed by an independent review committee. For MZL, Umbralisib demonstrated an ORR of 49% with 16% achieving a CR. For patients with FL, the ORR was 43%, with a CR rate of 3%. The median DOR was not reached for MZL and was 11.1 months for FL at the time of the data cutoff. These findings indicate that Ukoniq provides a new treatment option for patients with relapsed or refractory MZL and FL, particularly after other therapies have failed.