Newton, MA (Press Release) – Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clin­i­cal-stage pharma­ceu­tical com­pany, today announced that the U.S. Food and Drug Admin­istra­tion (FDA) has granted Fast Track desig­na­tion to the Company’s lead, oral Selective Inhibitor of Nuclear Export (SINE) com­­pound selinexor for the treat­ment of patients with multiple myeloma who have received at least three prior lines of ther­apy. The FDA’s state­ment, con­sis­tent with the design of Karyopharm’s Phase 2b STORM study, noted that the three prior lines of ther­apy in­clude regi­mens com­prised of an alkylating agent, a gluco­corticoid, Velcade® (bor­tezo­mib), Kyprolis® (car­filz­o­mib), Revlimid® (lena­lido­mide), Pomalyst® (poma­lido­mide) and Darzalex® (dara­tu­mu­mab). In addi­tion, the patient’s disease must be refractory to at least one pro­te­a­some inhibitor (Velcade or Kyprolis), one immuno­modu­la­tory agent (Revlimid or Pomalyst), glucocorticoids and to Darzalex, as well as to the most recent ther­apy. The Company ex­pec­ts to report top-line data from the STORM study at the end of April 2018.

The FDA's Fast Track pro­gram facilitates the devel­op­ment of drugs in­tended to treat serious con­di­tions and that have the poten­tial to address unmet medical needs. A drug pro­gram with Fast Track status is afforded greater access to the FDA for the pur­pose of expediting the drug's devel­op­ment, review and poten­tial ap­proval. In addi­tion, the Fast Track pro­gram allows for eligibility for Accelerated Approval and Priority Review, if relevant criteria are met, as well as for Rolling Review, which means that a drug com­pany can submit com­pleted sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is com­pleted before the entire appli­ca­tion can be submitted for review.

“The desig­na­tion of Fast Track for selinexor rep­re­sents im­por­tant recognition by the FDA of the poten­tial of this anti-cancer agent to address the sig­nif­i­cant unmet need in the treat­ment of patients with penta-refractory myeloma that has con­tinued to progress despite avail­able ther­a­pies,” said Sharon Shacham, PhD, MBA, Founder, Pres­i­dent and Chief Scientific Officer of Karyopharm. “We are fully committed to work­ing closely with the FDA as we con­tinue devel­op­ment of this poten­tial new, orally-administered treat­ment for patients who cur­rently have no other treat­ment options of proven benefit.”

About the Phase 2b STORM Study

In the multi-center, single-arm Phase 2b STORM (Selinexor Treatment of Refractory Myeloma) study, approxi­mately 122 patients with heavily pre­treated, penta-refractory myeloma receive 80mg oral selinexor twice weekly in com­bi­na­tion with 20mg low-dose dexa­meth­a­sone, also dosed orally twice weekly. Patients with penta-refractory disease are those who have pre­vi­ously received an alkylating agent, a gluco­corticoid, two immuno­modu­la­tory drugs (IMiDs) (Revlimid® (lena­lido­mide) and Pomalyst® (poma­lido­mide)), two pro­te­a­some inhibitors (PIs) (Velcade® (bor­tezo­mib) and Kyprolis® (car­filzo­mib)), and the anti-CD38 mono­clonal anti­body Darzalex® (dara­tumu­mab), and their disease is refractory to at least one PI, at least one IMiD, Darzalex, gluco­corticoids and their most recent anti-myeloma ther­apy. Overall response rate is the pri­mary end­point of the study, with duration of response and clin­i­cal benefit rate being sec­ond­ary end­points. All responses will be adjudicated by an Independent Review Committee (IRC).

About Selinexor

Selinexor (KPT-330) 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. To date, over 2,300 patients have been treated with selinexor, and it is cur­rently being eval­u­ated in several 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), in com­bi­na­tion with low-dose dexa­meth­a­sone (STORM) and as a poten­tial back­bone ther­apy in com­bi­na­tion with approved ther­a­pies (STOMP), and in diffuse large B-cell lym­phoma (SADAL), and liposarcoma (SEAL), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or cur­rently planned, in­­clud­ing multiple studies in com­bi­na­tion with one or more 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, devel­op­ment and sub­se­quent com­mer­cial­iza­tion 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 the thera­peutic poten­tial of and poten­tial clin­i­cal develop­ment plans for Karyopharm's drug can­di­dates, especially selinexor. Such state­ments are subject to numerous im­por­tant factors, risks and un­cer­tain­ties that may cause actual events or results to differ ma­teri­ally from Karyopharm's current ex­pec­ta­tions. For example, there can be no guar­an­tee that any of Karyopharm's drug can­di­dates, in­­clud­ing selinexor, will suc­cess­fully com­plete nec­essary clin­i­cal devel­op­ment phases, that devel­op­ment of any of Karyopharm's drug can­di­dates will con­tinue or that any pos­i­tive feed­back from regu­la­tory author­i­ties will ultimately lead to the approval of selinexor or any of Karyopharm’s other drug can­di­dates. 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 Annual Report on Form 10-K for the year ended December 31, 2017, which was filed with the Se­cu­ri­ties and Exchange Com­mis­sion (SEC) on March 15, 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
Revlimid® and Pomalyst® are registered trademarks of Celgene Corpo­ra­tion
Kyprolis® is a registered trademark of Onyx Pharma­ceu­ticals, Inc.
Darzalex® is a registered trademark of Janssen Biotech, Inc.

Source: Karyopharm Therapeutics Inc.