Newton, MA (Press Release) – Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clin­i­cal-stage pharma­ceu­tical com­pany, today announced that the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Admin­istra­tion (FDA) is scheduled to review data sup­porting the Company’s New Drug Application (NDA) requesting accelerated approval for selinexor, a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) com­pound, at a meeting on February 26, 2019 at 12:30 p.m. ET. The proposed indi­ca­tion to be discussed at this upcoming ODAC meeting is for selinexor in com­bi­na­tion with dexa­meth­a­sone for the treat­ment of patients with refractory multiple myeloma who have received at least three prior ther­a­pies and whose disease is refractory to at least one pro­te­a­some inhibitor (PI), one immuno­modu­la­tory agent (IMiD), and one anti-CD38 mono­clonal anti­body, and to their most recent treat­ment regi­men.

The ODAC is an independent panel of experts that eval­u­ates data con­cern­ing the efficacy and safety of mar­keted and inves­ti­ga­tional prod­ucts for use in the treat­ment of cancer and makes appro­pri­ate recom­men­da­tions to the FDA. Although the FDA will con­sider the recom­men­da­tion of the panel, the final de­ci­sion re­gard­ing the approval of the prod­uct is made by the FDA solely, and the recom­men­da­tions by the panel are non-binding.

Karyopharm’s NDA seeking accelerated approval for oral selinexor in com­bi­na­tion with dexa­meth­a­sone as a treat­ment for patients with triple class refractory multiple myeloma who have received at least three prior ther­a­pies is under Priority Review by FDA with an action date of April 6, 2019, under the Prescription Drug User-Fee Act (PDUFA).

The Company has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for selinexor requesting con­di­tional approval for the treat­ment of patients with re­lapsed or refractory multiple myeloma who have received at least three prior lines of ther­apy and whose disease is refractory to at least one PI, one IMiD, and one anti-CD38 mono­clonal anti­body, and to their most recent treat­ment regi­men. The selinexor MAA has been granted accelerated assess­ment by the EMA’s Committee for Medicinal Products for Human Use.

About Selinexor

Selinexor is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) com­pound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), 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 2018, Karyopharm reported pos­i­tive data from the Phase 2b STORM study eval­u­ating selinexor in com­bi­na­tion with low-dose dexa­meth­a­sone in patients with penta-refractory multiple myeloma. Selinexor has been granted Orphan Drug Desig­na­tion in multiple myeloma and Fast Track desig­na­tion for the patient pop­u­la­tion eval­u­ated in the STORM study. Karyopharm’s New Drug Application (NDA) has been accepted for filing and granted Priority Review by the FDA, and oral selinexor is cur­rently under review by the FDA as a possible new treat­ment for patients with penta-refractory multiple myeloma. The Company has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for con­di­tional approval and was granted accelerated assess­ment. Selinexor is also being studied in patients with re­lapsed or refractory diffuse large B-cell lym­phoma (DLBCL). In 2018, Karyopharm reported pos­i­tive top-line results from the Phase 2b SADAL study eval­u­ating selinexor in patients with re­lapsed or refractory DLBCL after at least two prior multi-agent ther­a­pies and who are in­eli­gible for trans­plan­ta­tion, in­­clud­ing high dose chemo­ther­apy with stem cell rescue. Selinexor has received Fast Track desig­na­tion from the FDA for the patient pop­u­la­tion eval­u­ated in the SADAL study. Selinexor is also being eval­u­ated in several other mid-and later-phase clin­i­cal trials across multiple cancer indi­ca­tions, in­­clud­ing in multiple myeloma in a pivotal, ran­dom­ized Phase 3 study in com­bi­na­tion with Velcade® (bor­tez­o­mib) and low-dose dexa­meth­a­sone (BOSTON), as a poten­tial back­bone ther­apy in com­bi­na­tion with approved ther­a­pies (STOMP), in liposarcoma (SEAL), and an investigator-sponsored study in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or cur­rently planned, in­­clud­ing multiple studies in com­bi­na­tion with approved ther­a­pies in a variety of tumor types to further inform Karyopharm's clin­i­cal devel­op­ment priorities for selinexor. Additional clin­i­cal trial in­for­ma­tion for selinexor is avail­able at www.clinicaltrials.gov.

About Karyopharm Therapeutics

Karyopharm Therapeutics Inc. (Nasdaq:KPTI) is a clin­i­cal-stage pharma­ceu­tical com­pany focused on the discovery and devel­op­ment of novel first-in-class drugs directed against nuclear transport and related targets for the treat­ment of cancer and other major diseases. Karyopharm's SINE com­pounds function by binding with and inhibiting the nuclear export protein XPO1 (or CRM1). In addi­tion to single-agent and com­bi­na­tion activity against a variety of human cancers, SINE com­pounds have also shown biological activity in models of neurodegeneration, inflammation, auto­immune disease, certain viruses and wound-healing. Karyopharm, which was founded by Dr. Sharon Shacham, cur­rently has several inves­ti­ga­tional pro­grams in clin­i­cal or pre­clin­i­cal devel­op­ment. For more in­for­ma­tion, please visit www.karyopharm.com.

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 our ex­pec­ta­tions relating to sub­missions to and the review and poten­tial approval of selinexor by regu­la­tory author­i­ties, in­­clud­ing the antic­i­pated timing of such sub­missions and actions, and the poten­tial avail­a­bil­ity of accelerated approval path­ways, the thera­peutic poten­tial of and poten­tial clin­i­cal devel­op­ment plans for Karyopharm's drug can­di­dates, especially selinexor, and the plans for com­mer­cial­iza­tion. 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 regulators will agree that selinexor qualifies for accelerated approval in the U.S. or con­di­tional approval in the E.U. as a result of the data from the STORM study in patients with penta-refractory myeloma or the SADAL study in patients with re­lapsed or refractory DLBCL or that any of Karyopharm's drug can­di­dates, in­­clud­ing selinexor, 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 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: 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 U.S. Food and Drug Admin­istra­tion and other regu­la­tory author­i­ties, inves­ti­ga­tional review boards at clin­i­cal trial sites and publication review bodies, in­­clud­ing with respect to the need for addi­tional clin­i­cal studies; Karyopharm's ability to obtain and main­tain requisite regu­la­tory approvals and to enroll patients in its clin­i­cal trials; unplanned cash require­ments and ex­pen­di­tures; devel­op­ment of drug can­di­dates by Karyopharm's com­pet­i­tors for diseases in which Karyopharm is cur­rently devel­op­ing its drug can­di­dates; and Karyopharm's ability to obtain, main­tain and enforce patent and other intellectual property protection for any drug can­di­dates it is devel­op­ing. These and other risks are described under the caption "Risk Factors" in Karyopharm's Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, which was filed with the Se­cu­ri­ties and Exchange Com­mis­sion (SEC) on November 8, 2018, and in other filings that Karyopharm may make with the SEC in the future. Any for­ward-looking state­ments con­tained in this press release speak only as of the date hereof, and, except as required by law, Karyopharm expressly disclaims any obli­ga­tion to update any for­ward-looking state­ments, whether as a result of new in­for­ma­tion, future events or other­wise.

Velcade® is a registered trademark of Takeda Pharma­ceu­tical Company Limited.

Source: Karyopharm Therapeutics.