Full Press Release Details
Karyopharm Reports First Quarter 2020 Financial Results and
Highlights Recent Company Progress
Pivotal Phase 3 BOSTON Study Meets Primary Endpoint with Significant Increase in Progression-Free Survival; Oral Presentation at ASCO
2020 Virtual Scientific Program and sNDA To Be Submitted by End of May 2020
First Quarter 2020 XPOVIO Net Product
Sales of $16.1 Million and Total Revenues of $18.1 Million
To Receive $12.0 Million from Antengene for Expanded
sNDA for Selinexor in DLBCL Accepted by FDA and Granted Priority Review; Assigned PDUFA Target Action Date
Initiated Randomized Study Evaluating Low Dose Selinexor in Patients with Severe COVID-19
Conference Call Scheduled for Today at 8:30 a.m. ET
NEWTON, Mass. May 5, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an innovation-driven pharmaceutical company, today reported
financial results for the quarter ended March 31, 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.
am extremely proud of the significant progress we have made thus far in 2020. Following our recent announcement of the positive top-line results from the pivotal Phase 3 BOSTON study, we are actively preparing
to share the dataset at the American Society of Clinical Oncology 2020 (ASCO20) Virtual Scientific Program and expect to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) by the end of May 2020,
requesting an expansion of the currently approved indication for XPOVIO to include second line treatment for patients with relapsed or refractory multiple myeloma, said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm.
Additionally, despite industry-wide challenges created by the current COVID-19 pandemic, we remain pleased by the ongoing commercialization of XPOVIO in the U.S., as well as our ability to significantly
strengthen our balance sheet in the first quarter. Finally, I want to assure our many stakeholders that the entire Karyopharm team has risen to the challenge of navigating the Company through these difficult times and we are working day and night to
provide critical medicines to the patients and physicians who need them most. In addition to the applications in oncology, we remain highly encouraged by the potential anti-viral and anti-inflammatory activity of XPO1 inhibition with selinexor and
look forward to working, as quickly as possible, with the medical community of regulators, treating physicians and patients on advancing our new clinical study of low dose selinexor to treat patients with severe
First Quarter 2020 and Recent Highlights
XPOVIO in Multiple Myeloma
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)
Low Dose Selinexor in COVID-19
Corporate and Financial Updates
Certain countries in the expanded territory with
Antengene became available following Karyopharm s reacquisition of the exclusive development and commercial rights from Ono which were transferred to Karyopharm in April 2020 at no cost to Karyopharm. Karyopharm has chosen to retain the rights
to selinexor and eltanexor in Japan while granting Antengene the exclusive rights to develop and commercialize selinexor and eltanexor in South Korea, Taiwan, Hong Kong and the ASEAN countries.
Outside of the territories licensed by Antengene, and the market for selinexor in Israel, Karyopharm maintains complete development and
commercial rights to selinexor and eltanexor throughout the world, including in the U.S., Canada, Europe, Japan, and Latin America.
Net product revenue: Net product revenue for the first quarter of 2020 was $16.1 million. Karyopharm commenced sales of
XPOVIO in the U.S. during the third quarter of 2019 and therefore did not have net product revenue during the first quarter of 2019.
revenue: License and other revenue for the first quarter of 2020 was $2.1 million, compared to $0.2 million for the first quarter of 2019. The increase was driven in part by the recognition of $1.1 million pursuant to our license
agreement with Antengene.
Cost of sales: Cost of sales were $0.8 million for the first quarter of 2020. Cost of sales reflects the costs of
XPOVIO units sold and third-party royalties on net product revenue.
Research and development expenses (R&D): R&D expenses for the first
quarter of 2020 were $34.0 million, compared to $38.0 million for the first quarter of 2019.
Selling, general and administrative expenses
(SG&A): For the first quarter of 2020, SG&A expenses were $30.7 million, compared to $27.1 million for the first quarter of 2019. The increase in SG&A expenses compared to the prior year was due primarily to activities to
support the U.S. commercialization of XPOVIO and in preparation for the potential launch of additional indications in 2020.
Interest expense for the first quarter of 2020 was $6.5 million, compared to $3.0 million for the first quarter 2019. The increase in interest expense is attributable to the imputed interest on the deferred royalty obligation
Karyopharm has with HealthCare Royalty Partners.
Net loss: Karyopharm reported a net loss of $52.9 million, or $0.78 per share, for the first
quarter of 2020, compared to a net loss of $66.2 million, or $1.09 per share, for the first quarter of 2019. Net loss includes non-cash stock-based compensation expense of $5.2 million and
$3.9 million for the 2020 and 2019 quarters, respectively.
Cash position: Cash, cash equivalents, restricted cash and investments as of March 31, 2020
totaled $385.2 million, compared to $265.8 million as of December 31, 2019.
2020 Financial Outlook
Karyopharm expects XPOVIO net product sales to be slightly higher in the second quarter of 2020 as compared to the first quarter of 2020. In addition, total
revenues are expected to be higher due to an increase in collaboration revenue from the expanded territory agreement with Antengene. The Company will not be issuing XPOVIO revenue guidance for the full year 2020 as it continues to monitor the
ongoing commercial impact from the COVID-19 pandemic as well as the timing of key expected regulatory actions in 2020. These regulatory events include the potential approval of XPOVIO for patients with
relapsed or refractory DLBCL as well as Karyopharm s planned sNDA submission, and subsequent FDA-review period, requesting expansion of the approved indication for XPOVIO to include second line treatment
for patients with relapsed or refractory multiple myeloma.
Based on its current operating plans, including the reduction of some R&D costs as a
result of trial delays due to the ongoing COVID-19 pandemic, Karyopharm expects its non-GAAP R&D and SG&A expenses, which excludes stock-based compensation
expense, for the full year 2020 to be at the lower end of the previously projected range of $240 million to $260 million. This estimate includes the additional costs associated with our new selinexor clinical trial in patients with severe COVID-19. Karyopharm has not reconciled the full year 2020 outlook for non-GAAP R&D and SG&A expenses to full year 2020 outlook for GAAP R&D and SG&A expenses
because Karyopharm cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the full year 2020 outlook for non-GAAP R&D and SG&A expenses.
The Company expects that its existing cash, cash equivalents and investments,
and the revenue it expects to generate from XPOVIO product sales, will be sufficient to fund its planned operations into the middle of 2022.
Non-GAAP Financial Information
Karyopharm uses a non-GAAP financial measure,
including R&D and SG&A expenses, to provide operating expense guidance. Non-GAAP R&D and SG&A expenses exclude stock-based compensation expense. Karyopharm believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Karyopharm s operating performance as it excludes non-cash stock
compensation expense. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP R&D and SG&A expenses and should not be considered a measure of Karyopharm s
liquidity. Instead, non-GAAP R&D and SG&A expenses should only be used to supplement an understanding of Karyopharm s operating results as reported under GAAP.
Conference Call Information
Karyopharm will host a
conference call today, Tuesday, May 5, 2020, at 8:30 a.m. Eastern Time, to discuss the first quarter 2020 financial results, recent accomplishments, clinical developments and business plans. 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 ID 9936655. 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.
IMPORTANT SAFETY INFORMATION
XPOVIO can cause thrombocytopenia,
leading to potentially fatal hemorrhage. Thrombocytopenia was reported as an adverse reaction in 74% of patients, and severe (Grade 3-4) thrombocytopenia occurred in 61% of patients treated with XPOVIO. The
median time to onset of the first event was 22 days. Bleeding occurred in 23% of patients with thrombocytopenia, clinically significant bleeding occurred in 5% of patients with thrombocytopenia and fatal hemorrhage occurred in <1% of patients.
Monitor platelet counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment.
Institute platelet transfusion and/or other treatments as clinically indicated. Monitor patients for signs and symptoms of bleeding and evaluate promptly. Interrupt and/or reduce dose, or permanently discontinue based on severity of adverse
XPOVIO can cause neutropenia,
potentially increasing the risk of infection. Neutropenia was reported as an adverse reaction in 34% of patients, and severe (Grade 3-4) neutropenia occurred in 21% of patients treated with XPOVIO. The median
time to onset of the first event was 25 days. Febrile neutropenia was reported in 3% of patients.
Obtain neutrophil counts at baseline, during treatment,
and as clinically indicated. Monitor more frequently during the first two months of treatment. Monitor patients for signs and symptoms of concomitant infection and evaluate promptly. Consider supportive measures including antimicrobials for signs of
infection and use of growth factors (e.g., G-CSF). Interrupt and/or reduce dose, or permanently discontinue based on severity of adverse reaction.
Gastrointestinal Toxicity
Gastrointestinal toxicities
occurred in patients treated with XPOVIO.
Nausea was reported as an adverse reaction in 72% of patients, and Grade 3 nausea occurred in 9% of patients treated with XPOVIO. The median time to onset of
the first nausea event was 3 days.
Vomiting was reported in 41% of patients, and Grade 3 vomiting occurred in 4% of patients treated with XPOVIO. The
median time to onset of the first vomiting event was 5 days.
Provide prophylactic 5-HT3 antagonists and/or other
anti-nausea agents, prior to and during treatment with XPOVIO. Manage nausea/vomiting by dose interruption, reduction, and/or discontinuation. Administer intravenous fluids and replace electrolytes to prevent dehydration in patients at risk. Use
additional anti-nausea medications as clinically indicated.
Diarrhea was reported as an adverse reaction in 44% of patients, and Grade 3 diarrhea occurred in 6% of patients treated with XPOVIO. The median time to onset
of diarrhea was 15 days.
Manage diarrhea by dose modifications and/or standard anti-diarrheal agents; administer intravenous fluids to prevent
dehydration in patients at risk.
Anorexia/Weight Loss
Anorexia was reported as an adverse reaction in 53% of patients, and Grade 3 anorexia occurred in 5% of patients treated with XPOVIO. The median time to onset
of anorexia was 8 days.
Weight loss was reported as an adverse reaction in 47% of patients, and Grade 3 weight loss occurred in 1% of patients treated