· Karyopharm Therapeutics has announced that its lead drug candidate, Selinexor
(KPT-330), has received orphan drug designation from the FDA for the treatment of Diffuse Large B-Cell Lymphoma (DLBCL), an aggressive form of Non-Hodgkin lymphoma (NHL). DLBCL is the most common of the aggressive NHLs, accounting for up to 30% of newly-diagnosed cases of NHL in the US.
According to the American Cancer Society, about 70,800 patients will be diagnosed the disease here, and an estimated 18,990 patients will die from the disease this year alone. However, government experts state that 50% of patients under the age of 65, and who “have good organ function, may be able to be cured with high dose chemotherapy combined with transplanted stem cells.”
· "The granting of Orphan Drug Designation by the FDA for DLBCL is another significant milestone in the Selinexor development program," commented Karyopharm, founder, CSO and President Dr. Sharon Shacham. "There are limited treatment options for patients with relapsed or refractory DLBCL, with no new agents approved for this indication over the past two decades. Many patients relapse after responding to multi-agent first-line therapy. The fundamental treatment of DLBCL has changed little in the past two decades, with no new or targeted agents approved for this indication. Accordingly, we are excited about the prospects for Selinexor's novel mechanism of action to potentially treat this patient population, either alone or in combination with other therapies."
"We are encouraged by the response data in patients with DLBCL who have received Selinexor in our ongoing Phase 1 clinical trial in advanced hematological malignancies,” added. Michael G. Kauffman, MD, PhD, the company’s Chief Executive Officer. “We look forward to the commencement of additional trials in patients with DLBCL, including a
registration-directed clinical trial of Selinexor and investigator-sponsored combination studies of Selinexor. We plan to present updated clinical data for Selinexor across multiple indications, including DLBCL, at ASCO 2014."
According to Kauffman, Selinexor works by “binding to the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus, which subsequently reinitiates and amplifies their tumor suppressor function. This is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells.”