Karyopharm Presents Hematologic Cancer Data on Lead Drug Candidate Selinexor at International Conference on Malignant Lymphoma

Karyopharm Therapeutics Inc.

, a clinical-stage pharmaceutical company, today announced the presentation of positive clinical and preclinical data for its lead product candidate, selinexor (KPT-330), a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE™ compound, at the 13th International Conference on Malignant Lymphoma (ICML) 2015 held June 17-20, 2015 in Lugano, Switzerland. In an ongoing Phase 1 clinical trial which included 14 evaluable, relapsed, refractory DLBCL patients with triple, double or single hit MYC, BCL2 and/or BCL6 translocations, selinexor demonstrated clinically meaningful activity with a 43% overall response rate (partial response or better). Responses included two complete responses (CR) and four partial responses (PR), while two additional patients achieved stable disease (SD). In preclinical models, selinexor demonstrated potency in double hit (DH)-DLBCL cell lines in vitro and in an aggressive patient-derived xenograft (PDX) model of triple hit (TH) DLBCL, with 84% tumor growth inhibition. DLBCL with MYC, BCL2 and/or BCL6 translocations is an area of significant unmet medical need associated with poor prognosis and no standard-of-care treatment options. "These results, along with DLBCL clinical data recently presented at the European Hematology Association (EHA) 2015 Annual Meeting, further demonstrate the potential of selinexor to serve significant unmet needs including high-risk DLBCL," said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. "These data demonstrate selinexor's potent activity and encouraging disease control in double-hit and other high-risk DLBCL, an area of significant unmet medical need given the poor prognosis and limited treatment options associated with these disease subtypes." Selinexor data in DH-DLBCL were described during an oral presentation by Dr. Ramiro Garzon of Ohio State University on Saturday, June 20, entitled "Selinexor Shows Marked Activity in Double-Hit Diffuse Large B Cell Lymphoma (DLBCL) in Pre-Clinical Models and in Patients with Heavily Pre-Treated Relapsed / Refractory Double-Hit DLBCL". These data from an ongoing Phase 1 clinical study of single-agent selinexor in patients with diffuse large B-cell lymphoma were as of June 1, 2015, including the following highlights: Among 14 evaluable, heavily pre-treated, patients with relapsed, refractory DLBCL with TH, DH or single-hit (SH) MYC, BCL2 and/or BCL6 translocations treated with selinexor, the overall response rate (ORR) was 43%, including two CRs and four PRs, while two patients achieved SD. Responses included three out of five patients with TH or DH DLBCL and three out of nine SH DLBCL, for a total of six out of 14 patients achieving objective responses. Toxicities were similar to the other patients in the study and no clinically significant organ dysfunction or cumulative toxicities were observed. In preclinical models, selinexor was potently cytotoxic and reduced Myc, Bcl2 and Bcl6 protein levels in DH-DLBCL cell lines in vitro and selinexor was highly active in a PDX model of TH DLBCL with 84% tumor growth inhibition.