Karyopharm secures FDA orphan drug status for Selinexor to treat DLBCL

Selinexor is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound and it functions by binding to the nuclear export protein XPO1 (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.

So far, over 300 patients have been treated with Selinexor in Phase I and Phase II clinical trials in advanced hematologic malignancies and solid tumors.

Additional Phase I and Phase II studies are ongoing or currently planned and three registration-directed clinical trials in hematological indications are expected to initiate patient enrolment during 2014.

Karyopharm founder, CSO and president Sharon Shacham said the granting of Orphan Drug Designation by the FDA for DLBCL is another significant milestone in the Selinexor development program.

"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," Shacham said.

"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."

Karyopharm chief executive officer Michael Kauffman said the company is encouraged by the response data in patients with DLBCL who have received Selinexor in its ongoing Phase I clinical trial in advanced hematological malignancies.

"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," Kauffman said.

"We plan to present updated clinical data for Selinexor across multiple indications, including DLBCL, at ASCO 2014."

Karyopharm is focused on the discovery and development of novel first-in-class drugs directed against nuclear transport targets for the treatment of cancer and other major diseases.