Karyopharm Announces Presentation of Interim Data from Phase 2 Study of Single-Agent Eltanexor in Relapsed/Refractory (R/R) Higher-Risk Myelodysplastic Neoplasms (MDS) at 17th International Congress on MDS
- Positive interim data from the Phase 2 study of eltanexor in patients with relapsed/refractory myelodysplastic neoplasms. Promising single-agent efficacy with a 27% overall response rate and a median overall survival of 8.7 months.
- None.
– Relapsed/Refractory MDS Patients Achieved Median Overall Survival of 8.7 months –
– Historically, Median Overall Survival in this Hard to Treat Patient Population is Only 4-6 Months1,2 –
– Results Indicate Potential Single Agent Activity of Eltanexor in Patients with R/R MDS –
As of the February 8, 2023 data cut-off date, 30 patients had been treated with 10mg eltanexor, oral, on Days 1-5 of each week. Eltanexor demonstrated a
Eltanexor was generally well-tolerated and manageable. The most common adverse events (AEs) were asthenia (
"Existing first-line treatments for higher risk MDS are not typically curative; approximately half of these patients do not respond. Upon progression, median overall survival for these higher risk, relapsed or refractory MDS patients is approximately four to six months. There is a critical need for novel and more effective treatment options for this patient population," said Reshma Rangwala, MD, PhD, Chief Medical Officer of Karyopharm. "We are encouraged by the improved overall survival observed to date with eltanexor in this higher risk patient population. These preliminary results are promising and reinforce the potential of XPO1 inhibition to provide meaningful clinical benefit to patients with relapsed/refractory myelodysplastic neoplasms."
Approximately 12,000 to 20,000 people in
About Eltanexor
Eltanexor (KPT-8602) is an investigational novel SINE compound that functions by binding with, and inhibiting, the nuclear export protein, XPO1, leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells.
In preclinical models, eltanexor has a broad therapeutic window with minimal penetration of the blood brain barrier and, therefore, has the potential to serve as another SINE compound for cancer indications. Following oral administration, animals treated with eltanexor show lower percentage of body weight loss and improved food consumption than animals similarly treated with selinexor. This allows more frequent dosing of eltanexor, enabling a longer period of exposure than is possible with selinexor.
Eltanexor is an investigational medicine and has not been approved by the United States Food and Drug Administration or any other regulatory agency.
About Karyopharm Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company pioneering novel cancer therapies. Since its founding, Karyopharm has been an industry leader in oral Selective Inhibitor of Nuclear Export (SINE) compound technology, which was developed to address a fundamental mechanism of oncogenesis: nuclear export dysregulation. Karyopharm's lead SINE compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the
Forward-Looking Statements
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 ability of eltanexor to treat patients with MDS; and expectations related to the clinical development of eltanexor and potential regulatory submissions of eltanexor. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond Karyopharm's control, that may cause actual events or results to differ materially from Karyopharm's current expectations. For example, there can be no guarantee that Karyopharm will successfully commercialize XPOVIO or that any of Karyopharm's drug candidates, including selinexor and eltanexor, will successfully complete necessary clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in the development or commercialization of 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: the risk that the COVID-19 pandemic could disrupt Karyopharm's business more severely than it currently anticipates, including by negatively impacting sales of XPOVIO, interrupting or delaying research and development efforts, impacting the ability to procure sufficient supply for the development and commercialization of selinexor or other product candidates, delaying ongoing or planned clinical trials, impeding the execution of business plans, planned regulatory milestones and timelines, or inconveniencing patients; the adoption of XPOVIO in the commercial marketplace, the timing and costs involved in commercializing XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; the ability to obtain and retain regulatory approval of XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; 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
XPOVIO® and NEXPOVIO® are registered trademarks of Karyopharm Therapeutics Inc. Any other trademarks referred to in this release are the property of their respective owners.
References:
1 Jabbour E, Cancer. 2010;116(16):3830-4
2 Prébet T. J Clin Oncol. 2011;29:3322-7
3 What is MDS?," MDS Foundation, accessed April 21, 2021, https://www.mds-foundation.org/what-is-mds/
4 Gil-Perez A. Ther Adv Hematol. 2019 doi:10.1177/2040620719847059
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