− Combination of Once-Weekly XPOVIO® (selinexor), Once-Weekly Velcade® (bortezomib) plus Dexamethasone (SVd) Results in Statistically Significant Reduction in the Risk of Disease Progression or Death Compared to Standard Twice-Weekly Velcade® plus Dexamethasone (Vd) Regimen −
− 47% Increase in Median PFS on SVd versus Vd –
− Regulatory Submission Planned in 2Q 2020; Data to be Submitted for Presentation at Upcoming Medical Meetings –
NEWTON, MA, USA I March 02, 2020 I Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, today announced positive top-line results from the randomized Phase 3 BOSTON study evaluating once-weekly XPOVIO® (selinexor) in combination with once-weekly Velcade® (bortezomib) and low-dose dexamethasone (SVd) compared to standard twice-weekly Velcade plus low-dose dexamethasone (Vd) in patients with multiple myeloma who have received one to three prior lines of therapy. The BOSTON study met its primary endpoint of a statistically significant increase in progression-free survival (PFS). The median PFS in the SVd arm was 13.93 months compared to 9.46 months in the Vd arm, representing a 4.47 month (47%) increase in median PFS (hazard ratio=0.70; p=0.0066). There were no new safety signals on the SVd arm and there was no imbalance in deaths between the two arms in the study. The full top-line data will be submitted for presentation at upcoming medical meetings.
“We are thrilled to report these highly significant top-line results from the BOSTON study, the first randomized Phase 3 trial to demonstrate clinically and statistically significant activity of once-weekly XPOVIO in combination with a current standard of care treatment in patients with myeloma after one to three prior therapies,” said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. “In the study, patients on the SVd regimen lived 47% longer without their disease worsening, which we believe represents an important improvement in the treatment of patients with relapsed or refractory multiple myeloma. We plan to submit the full data set for presentations at upcoming medical meetings to share the results with the medical community. We also intend to submit these data as quickly as possible to the U.S. Food and Drug Administration (FDA) as part of a supplemental New Drug Application seeking to expand the approved indication for XPOVIO into second line treatment for patients with relapsed or refractory multiple myeloma. If approved, the SVd regimen would be the first and only FDA-approved combination drug regimen that includes once-weekly Velcade therapy for relapsed myeloma.”
XPOVIO received accelerated approval from the FDA on July 3, 2019 for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Karyopharm expects to submit data from the Phase 3 BOSTON study to serve as this confirmatory trial. The full Prescribing Information for XPOVIO is available at www.XPOVIO.com.
About the BOSTON Study
BOSTON is a Phase 3 randomized, active comparator-controlled, open-label, multicenter study that is designed to compare the efficacy, safety and certain health-related quality of life (HR-QoL) parameters of once-weekly XPOVIO (selinexor) in combination with once-weekly Velcade® (bortezomib) plus low-dose dexamethasone (SVd) versus twice-weekly Velcade plus low-dose dexamethasone (Vd) in adult patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy. The BOSTON study enrolled approximately 402 patients. The primary endpoint of the study is progression-free survival (PFS) and key secondary endpoints include overall response rate (ORR), among others. Additionally, the BOSTON study allows for patients on the Vd control arm to crossover to the SVd arm following objective (quantitative) progression of disease. The BOSTON study is being conducted at over 150 clinical sites internationally.
Vd is a standard therapy for previously treated patients with multiple myeloma that is given by injection twice-weekly. Unlike other drugs used to treat multiple myeloma, selinexor is taken orally. Patients randomized to the SVd arm received selinexor (100mg once-weekly), Velcade (1.3 mg/m2 once-weekly given subcutaneously) and dexamethasone (40mg weekly). Patients randomized to the Vd arm received Velcade® (twice-weekly) plus low-dose dexamethasone (standard therapy given on the recommended schedule).
Conference Call Information
Karyopharm will host a conference call today, Monday, March 2, 2020, at 8:30 a.m. Eastern Time, to discuss the top-line results from the BOSTON study. To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference 3515858. A live audio webcast of the call will be available under “Events & Presentations” in the Investor section of the Company’s website, http://investors.karyopharm.com/events-presentations. An archived webcast will be available on the Company’s website approximately two hours after the event.
About Multiple Myeloma
According to the National Cancer Institute (NCI), multiple myeloma is the second most common blood cancer in the U.S. with more than 32,000 new cases each year and over 130,000 patients living with the disease. Despite recent therapeutic advances, there is currently no cure and most patients’ disease will typically progress following treatment with currently available therapies. According to the NCI, nearly 13,000 deaths due to multiple myeloma were expected in the U.S. in 2019.
About XPOVIO® (selinexor)
XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. Karyopharm received accelerated U.S. Food and Drug Administration (FDA) approval of XPOVIO in July 2019 in combination with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. Karyopharm has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for conditional approval of selinexor. A supplemental New Drug Application was recently submitted to the FDA seeking accelerated approval for selinexor as a new treatment for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), and selinexor has received Fast Track and Orphan designation from the FDA for this patient population. 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 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 an oncology-focused pharmaceutical company dedicated to the discovery, development, and commercialization of novel first-in-class drugs directed against nuclear export 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). Karyopharm’s lead compound, XPOVIO® (selinexor), received accelerated approval from the FDA in July 2019 in combination with dexamethasone as a treatment for patients with heavily pretreated multiple myeloma. An MAA for selinexor is also currently under review by the EMA for the same indication. The Company recently submitted a New Drug Application to the FDA seeking approval for XPOVIO in patients with DLBCL. 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 has several investigational programs in clinical or preclinical development. For more information, please visit www.karyopharm.com.
SOURCE: Karyopharm Therapeutics
Post Views: 19
− Combination of Once-Weekly XPOVIO® (selinexor), Once-Weekly Velcade® (bortezomib) plus Dexamethasone (SVd) Results in Statistically Significant Reduction in the Risk of Disease Progression or Death Compared to Standard Twice-Weekly Velcade® plus Dexamethasone (Vd) Regimen −
− 47% Increase in Median PFS on SVd versus Vd –
− Regulatory Submission Planned in 2Q 2020; Data to be Submitted for Presentation at Upcoming Medical Meetings –
NEWTON, MA, USA I March 02, 2020 I Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, today announced positive top-line results from the randomized Phase 3 BOSTON study evaluating once-weekly XPOVIO® (selinexor) in combination with once-weekly Velcade® (bortezomib) and low-dose dexamethasone (SVd) compared to standard twice-weekly Velcade plus low-dose dexamethasone (Vd) in patients with multiple myeloma who have received one to three prior lines of therapy. The BOSTON study met its primary endpoint of a statistically significant increase in progression-free survival (PFS). The median PFS in the SVd arm was 13.93 months compared to 9.46 months in the Vd arm, representing a 4.47 month (47%) increase in median PFS (hazard ratio=0.70; p=0.0066). There were no new safety signals on the SVd arm and there was no imbalance in deaths between the two arms in the study. The full top-line data will be submitted for presentation at upcoming medical meetings.
“We are thrilled to report these highly significant top-line results from the BOSTON study, the first randomized Phase 3 trial to demonstrate clinically and statistically significant activity of once-weekly XPOVIO in combination with a current standard of care treatment in patients with myeloma after one to three prior therapies,” said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. “In the study, patients on the SVd regimen lived 47% longer without their disease worsening, which we believe represents an important improvement in the treatment of patients with relapsed or refractory multiple myeloma. We plan to submit the full data set for presentations at upcoming medical meetings to share the results with the medical community. We also intend to submit these data as quickly as possible to the U.S. Food and Drug Administration (FDA) as part of a supplemental New Drug Application seeking to expand the approved indication for XPOVIO into second line treatment for patients with relapsed or refractory multiple myeloma. If approved, the SVd regimen would be the first and only FDA-approved combination drug regimen that includes once-weekly Velcade therapy for relapsed myeloma.”
XPOVIO received accelerated approval from the FDA on July 3, 2019 for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Karyopharm expects to submit data from the Phase 3 BOSTON study to serve as this confirmatory trial. The full Prescribing Information for XPOVIO is available at www.XPOVIO.com.
About the BOSTON Study
BOSTON is a Phase 3 randomized, active comparator-controlled, open-label, multicenter study that is designed to compare the efficacy, safety and certain health-related quality of life (HR-QoL) parameters of once-weekly XPOVIO (selinexor) in combination with once-weekly Velcade® (bortezomib) plus low-dose dexamethasone (SVd) versus twice-weekly Velcade plus low-dose dexamethasone (Vd) in adult patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy. The BOSTON study enrolled approximately 402 patients. The primary endpoint of the study is progression-free survival (PFS) and key secondary endpoints include overall response rate (ORR), among others. Additionally, the BOSTON study allows for patients on the Vd control arm to crossover to the SVd arm following objective (quantitative) progression of disease. The BOSTON study is being conducted at over 150 clinical sites internationally.
Vd is a standard therapy for previously treated patients with multiple myeloma that is given by injection twice-weekly. Unlike other drugs used to treat multiple myeloma, selinexor is taken orally. Patients randomized to the SVd arm received selinexor (100mg once-weekly), Velcade (1.3 mg/m2 once-weekly given subcutaneously) and dexamethasone (40mg weekly). Patients randomized to the Vd arm received Velcade® (twice-weekly) plus low-dose dexamethasone (standard therapy given on the recommended schedule).
Conference Call Information
Karyopharm will host a conference call today, Monday, March 2, 2020, at 8:30 a.m. Eastern Time, to discuss the top-line results from the BOSTON study. To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference 3515858. A live audio webcast of the call will be available under “Events & Presentations” in the Investor section of the Company’s website, http://investors.karyopharm.com/events-presentations. An archived webcast will be available on the Company’s website approximately two hours after the event.
About Multiple Myeloma
According to the National Cancer Institute (NCI), multiple myeloma is the second most common blood cancer in the U.S. with more than 32,000 new cases each year and over 130,000 patients living with the disease. Despite recent therapeutic advances, there is currently no cure and most patients’ disease will typically progress following treatment with currently available therapies. According to the NCI, nearly 13,000 deaths due to multiple myeloma were expected in the U.S. in 2019.
About XPOVIO® (selinexor)
XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. Karyopharm received accelerated U.S. Food and Drug Administration (FDA) approval of XPOVIO in July 2019 in combination with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. Karyopharm has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for conditional approval of selinexor. A supplemental New Drug Application was recently submitted to the FDA seeking accelerated approval for selinexor as a new treatment for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), and selinexor has received Fast Track and Orphan designation from the FDA for this patient population. 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 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 an oncology-focused pharmaceutical company dedicated to the discovery, development, and commercialization of novel first-in-class drugs directed against nuclear export 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). Karyopharm’s lead compound, XPOVIO® (selinexor), received accelerated approval from the FDA in July 2019 in combination with dexamethasone as a treatment for patients with heavily pretreated multiple myeloma. An MAA for selinexor is also currently under review by the EMA for the same indication. The Company recently submitted a New Drug Application to the FDA seeking approval for XPOVIO in patients with DLBCL. 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 has several investigational programs in clinical or preclinical development. For more information, please visit www.karyopharm.com.
SOURCE: Karyopharm Therapeutics
Post Views: 19
