-- Second Quarter 2020 XPOVIO Net Product Sales of $18.6 Million; Strongest Quarterly Sales Since July 2019 Launch --
-- XPOVIO Approved by FDA as a New Treatment for Patients with Relapsed or Refractory DLBCL; Commercial Rollout Began Immediately Upon Approval --
-- Supplemental New Drug Application for XPOVIO as a Treatment for Patients with Multiple Myeloma After At Least One Prior Line of Therapy Accepted by FDA and Assigned a Target PDUFA Date of March 19, 2021 --
-- Conference Call Scheduled for Today at 8:30 a.m. ET --
NEWTON, Mass., Aug. 4, 2020 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today reported financial results for the quarter ended June 30, 2020. In addition, Karyopharm highlighted select corporate milestones, including details regarding the ongoing U.S. commercialization of XPOVIO® (selinexor), and provided an overview of its key clinical development programs.
"Despite the ongoing global COVID-19 pandemic, Karyopharm was able to achieve record quarterly XPOVIO sales as well as execute on several important initiatives, including receiving approval of XPOVIO for its second cancer indication from the U.S. Food and Drug Administration (FDA) to treat patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). XPOVIO is now the only single-agent oral therapy approved in this indication, including DLBCL arising from follicular lymphoma, and now approved in both multiple myeloma and DLBCL," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "Other recent highlights include the reporting of positive clinical results from the Phase 3 BOSTON study in a late breaking oral presentation at the American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program. Additionally, a supplemental New Drug Application (sNDA) was recently accepted by the FDA based on these positive data. Finally, our planned interim analysis of the randomized Phase 2 study of low dose selinexor in patients with severe COVID-19 indicated that while the agent is unlikely to demonstrate a statistically significant efficacy benefit across the entire patient population studied, it appears to confer clinical benefit in a clearly defined subpopulation of patients. Based on these results, we expect that future clinical development of low dose selinexor for patients with COVID-19 will focus on this subpopulation."
Second Quarter 2020 and Recent Highlights
XPOVIO in Multiple Myeloma
- XPOVIO U.S. Commercialization. Oral XPOVIO became commercially available to patients with penta-refractory multiple myeloma in the U.S. in July 2019 and generated net product sales of $18.6 million in the second quarter of 2020, representing an approximate 16% increase in net product sales compared to the first quarter of 2020. Despite COVID-19-related challenges, which included the diversion of physician and other healthcare resources to manage the ongoing COVID-19 pandemic and the significant reduction in Karyopharm's field force in-person activities, XPOVIO sales grew as a result of an increasing demand from both academic and community-based physicians. As of June 30, 2020, nearly 3,200 XPOVIO prescriptions have been filled since launch, and approximately 170 new physician prescribing accounts were added in the second quarter. Approximately 950 prescriptions were filled in the second quarter of 2020, representing the highest quarterly demand since launch and a 12% increase over the first quarter of 2020 and a 10% increase over the fourth quarter of 2019. This growth was particularly noteworthy as sales in the U.S. for a number of competing multiple myeloma drugs declined in the second quarter primarily as a result of the ongoing COVID-19 pandemic. Finally, based on data from specialty pharmacies, prescription refill rates for XPOVIO also continued to grow and reached a new high in the quarter with the average number of prescriptions per patient reaching 2.7 by the end of June 2020 as compared to 2.0 at the end of December 2019.
- Pivotal Phase 3 BOSTON Data Reported at ASCO 2020. In May 2020, Karyopharm presented positive detailed results from the BOSTON study as part of the ASCO 2020 Virtual Scientific Program. The BOSTON study evaluated once-weekly XPOVIO 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 with a significant increase in median progression-free survival (PFS) in patients with multiple myeloma following one to three prior lines of therapy. 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=0.0075). There were no new safety signals on the SVd arm, and deaths were numerically lower on the SVd arm (N=47) as compared with the Vd arm (N=62). Additionally, peripheral neuropathy rates were significantly lower on the SVd arm compared to the Vd arm (32.3% vs. 47.1%; p=0.0010). Karyopharm's sNDA, based on these data, was accepted by the FDA in July 2020 and the FDA has assigned an action date of March 19, 2021 under the Prescription Drug User-Fee Act (PDUFA).
- Regulatory Strategy Update in Europe. In January 2019, Karyopharm submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) requesting conditional approval for selinexor in combination with dexamethasone as a treatment for patients with heavily pretreated multiple myeloma based on the results of the Phase 2b STORM study. In January 2020, Karyopharm was granted a three-month extension from the EMA's Committee for Medicinal Products for Human Use (CHMP) to provide additional time to respond to the outstanding questions from the MAA, primarily re-monitoring certain clinical data. Due to the COVID-19 pandemic and the resulting disruption at many clinical sites, re-monitoring activities requested by CHMP have delayed the review timelines in Europe. We expect to submit the requested re-monitoring data in the third quarter of 2020, and we expect to receive an opinion from CHMP with respect to our MAA before the end of 2020. In addition, we expect to submit an MAA based on data from the BOSTON study before the end of 2020.
XPOVIO in Diffuse Large B-Cell Lymphoma (DLBCL)
- XPOVIO Receives Accelerated Approval from the FDA. On June 22, 2020, the FDA approved oral XPOVIO for the treatment of adult patients with relapsed or refractory DLBCL, not otherwise specified, including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. This indication was approved based on response rate under the FDA's Accelerated Approval Program, which was developed to allow for expedited approval of drugs that treat serious conditions and that fill an unmet medical need. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). XPOVIO became commercially available in the U.S. for patients with relapsed or refractory DLBCL immediately following approval of this new indication and Karyopharm is leveraging its existing commercial infrastructure to market this second oncology indication.
- Phase 2b SADAL Data Published in The Lancet Haematology. In June 2020, the results of the Phase 2b SADAL study, which served as the basis for the FDA's approval of the DLBCL indication, were published in The Lancet Haematology. The SADAL study evaluated single-agent oral XPOVIO for the treatment of adult patients with relapsed or refractory DLBCL, not otherwise specified, who have received at least two prior therapies. In this population, XPOVIO demonstrated an overall response rate of 28%, including a complete response rate of 12%. Responses were seen in all subgroups regardless of age, gender, prior therapy, DLBCL subtype or prior stem cell transplant therapy. Patient responses were durable with a median duration of response of 9.3 months (23.0 months for patients who achieved a complete response). Importantly, responses were associated with longer survival, underscoring the potential of oral XPO1 inhibition as an oral, non-chemotherapeutic option for patients with relapsed or refractory DLBCL.
Low Dose Selinexor in COVID-19
- Future Clinical Development to Focus on Specific Patient Subpopulation. In April 2020, Karyopharm began enrollment in a placebo-controlled, randomized Phase 2 study to evaluate low dose oral selinexor in hospitalized patients with severe COVID-19 (XPORT-CoV-1001 / NCT04349098). In May 2020 the protocol was amended to allow enrollment of patients with more severe disease. Following a planned interim analysis (115 patients included in the efficacy analysis and 113 patients included in the safety analysis), the Data Safety Monitoring Board (DSMB) for the study recommended that the Company discontinue the trial as it is unlikely to demonstrate a statistically significant efficacy benefit across the entire heterogenous patient population studied. However, the DSMB concluded that the trial was likely to show a benefit in a subpopulation of patients