Karyopharm Therapeutics Inc. (NASDAQ: KPTI) saw its shares make a handy gain on Tuesday after the firm announced results from its midstage trial. Specifically, the firm reported positive top-line results from the Phase 2b STORM study evaluating selinexor in heavily pretreated patients with refractory multiple myeloma.
Regarding the STORM study’s primary objective, oral selinexor achieved a 25.4% overall response rate, which included two complete responses and 29 partial or very good partial responses in these patients with penta-refractory myeloma.
The median duration of response, a key secondary objective, was 4.4 months. All responses were confirmed by an Independent Review Committee. Selinexor was recently granted Fast-Track designation by the U.S. Food and Drug Administration (FDA) for this indication.
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 FDA during the second half of 2018, with a request for accelerated approval for oral selinexor as a new treatment for patients with penta-refractory multiple myeloma.
The company also plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in early 2019 with a request for conditional approval.
Paul G. Richardson, MD, Director of Clinical Research, Jerome Lipper Multiple Myeloma Center at the Dana-Farber Cancer Institute, commented:
Despite numerous advances in myeloma treatment, currently available therapies are insufficient to address the increasing number of patients with highly resistant, penta-refractory myeloma, where the disease has ultimately become non-responsive to approved therapy. There is, therefore, a real urgency for new therapies with novel mechanisms of action for these patients, who have a critical unmet medical need. Selinexor’s targeted inhibition of nuclear export could potentially expand the armamentarium of treatment options significantly in this important population for which no other established treatment is readily available.
Shares of Karyopharm were last seen up about 14% at $14.88, with a consensus analyst price target of $20.27 and a 52-week range of $7.48 to $18.00.