Company Plans to Complete Submission During the Second Half of 2018

Newtown, MA (Press Release) – Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clin­i­cal-stage pharma­ceu­tical com­pany, today announced that the Company has ini­ti­ated a rolling sub­mission of a New Drug Application (NDA) to the U.S. Food and Drug Admin­istra­tion (FDA) seeking accelerated approval for selinexor, its novel, oral SINE com­­pound, as a new treat­ment for patients with penta-refractory multiple myeloma. Patients with penta-refractory myeloma have pre­vi­ously received the two pro­te­a­some inhibitors (PIs), Velcade® (bor­tez­o­mib) and Kyprolis® (car­filz­o­mib), the two immuno­modu­la­tory drugs (IMiDs), Revlimid® (lena­lido­mide) and Pomalyst® (poma­lido­mide), and the anti-CD38 mono­clonal anti­body Darzalex® (dara­tu­mu­mab), …

• Kyprolis, lena­lido­mide, and dexa­meth­a­sone extended median over­all survival in re­lapsed or refractory multiple myeloma patients to 48 months
• Kyprolis is the first and only treat­ment to dem­onstrate over­all survival benefits in two Phase 3 studies in re­lapsed and refractory multiple myeloma

Thousand Oaks, CA (Press Release) – Amgen (NASDAQ: AMGN) today announced that the U.S. Food and Drug Admin­istra­tion (FDA) has approved the supple­mental New Drug Application (sNDA) to add the positive over­all survival (OS) data from the Phase 3 ASPIRE trial to the U.S. Prescribing Information for KYPROLIS® (car­filz­o­mib). Data added to the label showed that KYPROLIS, lena­lido­mide and dexa­meth­a­sone (KRd) sig­nif­i­cantly reduced the risk of death by 21 per­cent and extended over­all survival by 7.9 months versus lena­lido­mide and dexa­meth­a­sone alone …

• Oral Selinexor Achieves 25.4% Overall Response Rate and Median Duration of Response of 4.4 Months in Patients with Penta-Refractory Myeloma
• Company Plans to Submit a New Drug Application to the FDA in the Second Half of 2018
• Selinexor Continues to Demonstrate a Predictable and Manageable Tolerability Profile
• Management to Host Conference Call Tomorrow, May 1, 2018 at 8:00 a.m. ET

Newton, MA (Press Release) – Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clin­i­cal-stage pharma­ceutical com­pany, today reported pos­i­tive top-line results from the Phase 2b STORM study eval­u­ating the Company’s lead, oral Selective Inhibitor of Nuclear Export (SINE) com­­pound selinexor in heavily pre­treated patients with refractory multiple myeloma. Regarding the STORM study’s pri­mary objective, oral selinexor achieved a 25.4% over­all response rate (ORR), which in­cluded two com­plete responses (CRs) and 29 partial (PRs) or very good …

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), …

San Diego, CA (Press Release) – Sorrento Therapeutics, Inc. (NASDAQ:SRNE) and Celularity Inc announced today that the com­pa­nies have started screen­ing patients for its leading CD38 chi­meric an­ti­gen re­cep­tor (CAR) T cell ther­apy drug devel­op­ment pro­gram, fol­low­ing FDA review allowing clin­i­cal trial initiation.

The com­pa­nies’ CD38 CAR-T pro­gram is their most ad­vanced pro­gram targeting this dif­fi­cult-to-treat con­di­tion. This trial is cur­rently the only active US-based clin­i­cal trial targeting CD38 using a CAR-T cell ther­apy.

The first inves­ti­ga­tional site at Roger Williams Medical Center, RI, is actively engaged in the clin­i­cal study execution, with …

New York, NY (Press Release) – Phosplatin Thera­peutics LLC, a clin­i­cal stage pharma­ceu­tical com­pany focused on on­col­ogy drug devel­op­ment, an­nounced to­day it has en­rolled the first cohort of patients into its Phase I / II study of PT-112 as a single agent in re­lapsed or re­frac­tory mul­ti­ple myeloma. The Com­pany fur­ther an­nounced having re­ceived FDA Orphan Drug Desig­na­tion for PT-112 in the treat­ment of mul­ti­ple myeloma.

PT-112 is cur­rently under ad­vanced Phase I devel­op­ment in solid tumors. As re­ported at the ASCO 2017 Annual Meeting, PT-112 dis­plays an attractive tol­er­a­bil­ity profile and signals …

Designations based on pre­lim­i­nary clin­i­cal data from on­go­ing phase I study of bb2121 in heavily pre-treated mul­ti­ple myeloma

Summit, NJ and Cambridge, MA (Press Release) – Celgene Corpo­ra­tion (NASDAQ:CELG) and bluebird bio, Inc. (NASDAQ:BLUE) to­day an­nounced that bb2121, a chi­meric an­ti­gen re­cep­tor T-cell (CAR-T) ther­apy targeting b-cell maturation an­ti­gen (BCMA) in pre­vi­ously treated patients with mul­ti­ple myeloma, has been granted Break­through Therapy Desig­na­tion (BTD) by the U.S. Food and Drug Admin­istra­tion (FDA) and PRIority MEdicines (PRIME) eligibility by the Euro­pean Medicines Agency (EMA).

BTD desig­na­tion and PRIME eligibility for bb2121 were based on pre­lim­i­nary clin­i­cal data from the on­go­ing phase 1 study CRB-401. Updated data from CRB-401 is scheduled to be …