Efficacy

Ninlaro (Ixazomib) Efficacy in Multiple Myeloma

Ninlaro (ixazomib) is an oral proteasome inhibitor used in the treatment of multiple myeloma, a type of blood cancer that affects plasma cells. It is specifically indicated for use in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy. Clinical trials have demonstrated that ixazomib, when added to lenalidomide and dexamethasone, extends progression-free survival (PFS) compared to the two drugs alone. The TOURMALINE-MM1 study, a pivotal phase 3 clinical trial, showed that the median PFS for patients treated with the ixazomib regimen was significantly longer than for those who received placebo plus lenalidomide and dexamethasone.

While the overall survival (OS) benefit with ixazomib has not been definitively established, the improvement in PFS indicates a delay in disease progression, which is a valuable outcome for patients with multiple myeloma. The response rates, including complete response and very good partial response, were also higher in the ixazomib group. The safety profile of ixazomib is generally manageable, with common adverse events including gastrointestinal symptoms, thrombocytopenia, and peripheral neuropathy.

Xpovio (Selinexor) Efficacy in Multiple Myeloma

Xpovio (selinexor) is a first-in-class, oral selective inhibitor of nuclear export (SINE) compound that blocks the exportin 1 (XPO1) protein. It is approved for use in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. The approval of selinexor was based on results from the STORM study, a phase 2b clinical trial, which demonstrated a meaningful overall response rate (ORR) in heavily pretreated patients.

In the STORM study, selinexor, in combination with dexamethasone, showed an ORR of approximately 25% in patients with RRMM. The median duration of response was also notable, considering the heavily pretreated population. While the efficacy of selinexor has been established in this difficult-to-treat population, its use is associated with a unique set of side effects, including nausea, vomiting, fatigue, and anorexia, which require careful management to optimize patient outcomes. Further research and ongoing clinical trials continue to evaluate the role of selinexor in earlier lines of treatment and in combination with other myeloma therapies.