Karyopharm Announces Positive Top-Line Data from Phase 2b STORM Study Evaluating Selinexor in Patients with Penta-Refractory Multiple Myeloma
- Oral Selinexor Achieves 25.4% Overall Response Rate and Median Duration of Response of 4.4 Months in Patients with Penta-Refractory Myeloma -
- Company Plans to Submit a New Drug Application to the
- Selinexor Continues to Demonstrate a Predictable and Manageable Tolerability Profile -
- Management to Host Conference Call Tomorrow,
The data reported today are from Part 2 of the international, multi-center, single-arm Phase 2b STORM (Selinexor Treatment of Refractory Myeloma) study, which enrolled 122 heavily pretreated patients with penta-refractory myeloma. Each patient received 80mg oral selinexor twice weekly in combination with low-dose dexamethasone (20mg twice weekly). In this study, penta-refractory is defined as patients who have previously received at least one alkylating agent, glucocorticoids, two immunomodulatory drugs (IMiDs) (Revlimid® (lenalidomide) and Pomalyst® (pomalidomide)), two proteasome inhibitors (PIs) (Velcade® (bortezomib) and Kyprolis® (carfilzomib)), and Darzalex® (daratumumab), an anti-CD38 monoclonal antibody, and whose disease is refractory to glucocorticoids, at least one PI, at least one IMiD, and Darzalex, and whose disease has progressed following their most recent therapy.
Oral selinexor demonstrated a predictable and manageable tolerability profile, with safety results that were consistent with those previously reported from Part 1 of this study (Vogl et al., J Clin Oncol, 2018) and from other selinexor studies. As anticipated, the most common adverse events (AEs) were nausea, vomiting, fatigue and reduced appetite and were primarily low grade and manageable with standard supportive care and/or dose modification. The most common hematologic AEs were Grade ≥3 cytopenias and were generally not associated with clinical sequelae. Karyopharm plans to submit detailed STORM study results for presentation at an upcoming medical oncology meeting.
“The 25.4% response rate and 4.4 month duration of response observed in the STORM study are highly compelling,” stated Sundar Jagannath, MD, Director of the Multiple Myeloma Program and Professor of Medicine (Hematology and Medical Oncology) at
Selinexor has been granted Orphan Drug Designation in multiple myeloma and Fast Track designation for the patient population evaluated in the STORM study. Karyopharm plans to submit a New Drug Application (NDA) to the
“We are extremely grateful to, and thank, the patients, their families and the investigators for their important contributions to the STORM study,” said
Further Information About Potential Accelerated Approval for Selinexor in Multiple Myeloma
Conference Call Information
Karyopharm will host a conference call tomorrow,
Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), thus leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies the tumor suppressor functions of these proteins and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 2,400 patients have been treated with selinexor, and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade® (bortezomib) and low-dose dexamethasone (
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, especially selinexor. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Karyopharm's current expectations. For example, there can be no guarantee that any of Karyopharm's drug candidates, including selinexor, will successfully complete necessary clinical development phases, that development of any of Karyopharm's drug candidates will continue or that any feedback from regulatory authorities will ultimately lead to the approval of selinexor or any of Karyopharm’s other drug candidates. Further, there can be no guarantee that any positive developments in Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: Karyopharm's results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the
Velcade® is a registered trademark of
Revlimid® and Pomalyst® are registered trademarks of
Kyprolis® is a registered trademark of
Darzalex® is a registered trademark of
Source: Karyopharm Therapeutics Inc.