PR Newswire
NEWTON, Mass., Aug. 4, 2020
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 in Diffuse Large B-Cell Lymphoma (DLBCL)
Low Dose Selinexor in COVID-19
Selinexor in Solid Tumors
Selinexor in Other Cancer Indications
KPT-9274
Corporate Updates
Second Quarter 2020 Financial Results
Net product revenue: Net product revenue for the second quarter of 2020 was $18.6 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 second quarter of 2019.
License and other revenue: License and other revenue for the second quarter of 2020 was $14.9 million, compared to $9.5 million for the second quarter of 2019. License and other revenue for the second quarter of 2020 included $12.7 million in revenue recognized as a result of a May 2020 amendment to our license agreement with Antengene Therapeutics Limited (Antengene), coupled with $2.2 million in revenue recognized upon the April 2020 termination of our license agreement with Ono Pharmaceutical Co., Ltd. Karyopharm recognized $9.4 million in revenue during the second quarter of 2019 pursuant to the terms of the original agreement with Antengene and $0.1 million related to clinical supply provided to various partners, as well as grant revenue pursuant to a government grant arrangement.
Cost of sales: Cost of sales were $0.4 million for the second 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 second quarter of 2020 were $42.6 million, compared to $26.5 million for the second quarter of 2019. The increase in R&D expenses compared to the second quarter of last year was primarily attributable to COVID-19 trial activity and continued activity in our other ongoing clinical trials.
Selling, general and administrative expenses (SG&A): For the second quarter of 2020, SG&A expenses were $30.8 million, compared to $24.7 million for the second quarter of 2019. The increase in SG&A expenses compared to the second quarter of last year was due primarily to activities to support the U.S. commercialization of XPOVIO and preparations for the launch of XPOVIO as a treatment for patients with relapsed or refractory DLBCL.
Interest expense: Interest expense for the second quarter of 2020 was $6.8 million, compared to $3.1 million for the second quarter of 2019. The increase in interest expense was attributable to the imputed interest on the deferred royalty obligation Karyopharm has with HealthCare Royalty Partners.
Net loss: Karyopharm reported a net loss of $46.4 million, or $0.63 per share, for the second quarter of 2020, compared to a net loss of $43.4 million, or $0.71 per share, for the second quarter of 2019. Net loss includes non-cash stock-based compensation expense of $6.4 million and $4.1 million for the second quarters of 2020 and 2019, respectively.
Cash position: Cash, cash equivalents, restricted cash and investments as of June 30, 2020 totaled $348.2 million, compared to $265.8 million as of December 31, 2019.
2020 Financial Outlook
Based on its current operating plans, Karyopharm continues to expect its non-GAAP R&D and SG&A expenses, which excludes stock-based compensation expense, for the full year 2020 to be in the range of $240.0 million to $260.0 million. 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, August 4, 2020, at 8:30 a.m. Eastern Time, to discuss the second quarter 2020 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (877) 870-4263 (local) or (412) 317-0790 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. 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 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 in this same RRMM indication. Karyopharm's supplemental New Drug Application (sNDA) requesting an expansion of its current indication to include the treatment for patients with multiple myeloma after at least one prior line of therapy has been accepted for filing by the FDA. In June 2020, Karyopharm received accelerated FDA approval of XPOVIO for its second indication in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including as a potential backbone therapy in combination with approved myeloma 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.
For more information about Karyopharm's products or clinical trials, please contact the Medical Information department at:
Tel: +1 (888) 209-9326
Email: [email protected]
IMPORTANT SAFETY INFORMATION
Thrombocytopenia: XPOVIO can cause life-threatening thrombocytopenia, potentially leading to hemorrhage. Thrombocytopenia was reported in patients with multiple myeloma (MM) and developed or worsened in patients with DLBCL.
Thrombocytopenia is the leading cause of dosage modifications. Monitor platelet counts at baseline and throughout treatment. Monitor more frequently during the first 3 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, reduce dose, or permanently discontinue based on severity of adverse reaction.
Neutropenia: XPOVIO can cause life-threatening neutropenia, potentially increasing the risk of infection. Neutropenia and febrile neutropenia occurred in patients with MM and in patients with DLBCL.
Obtain white blood cell counts with differential at baseline and throughout treatment. Monitor more frequently during the first 3 months of treatment. Monitor patients for signs and symptoms of concomitant infection and evaluate promptly. Consider supportive measures, including antimicrobials and growth factors (e.g., G-CSF). Interrupt, reduce dose, or permanently discontinue based on severity of adverse reaction (AR).
Gastrointestinal Toxicity: XPOVIO can cause severe gastrointestinal toxicities in patients with MM and DLBCL.
Nausea/Vomiting: Provide prophylactic antiemetics. Administer 5-HT3 receptor antagonists and other anti-nausea agents prior to and during treatment with XPOVIO. Interrupt, reduce dose, or permanently discontinue based on severity of ARs. Administer intravenous fluids to prevent dehydration and replace electrolytes as clinically indicated.
Diarrhea: Interrupt, reduce dose, or permanently discontinue based on severity of ARs. Provide standard anti-diarrheal agents, administer intravenous fluids to prevent dehydration, and replace electrolytes as clinically indicated.
Anorexia/Weight Loss: Monitor weight, nutritional status, and volume status at baseline and throughout treatment. Monitor more frequently during the first 3 months of treatment. Interrupt, reduce dose, or permanently discontinue based on severity of ARs. Provide nutritional support, fluids, and electrolyte repletion as clinically indicated.
Hyponatremia: XPOVIO can cause severe or life-threatening hyponatremia. Hyponatremia developed in patients with MM and in patients with DLBCL.
Monitor sodium level at baseline and throughout treatment. Monitor more frequently during the first 2 months of treatment. Correct sodium levels for concurrent hyperglycemia (serum glucose >150 mg/dL) and high serum paraprotein levels. Assess hydration status and manage hyponatremia per clinical guidelines, including intravenous saline and/or salt tablets as appropriate and dietary review. Interrupt, reduce dose, or permanently discontinue based on severity of the AR.
Serious Infection: XPOVIO can cause serious and fatal infections. Most infections were not associated with Grade 3 or higher neutropenia. Atypical infections reported after taking XPOVIO include, but are not limited to, fungal pneumonia and herpesvirus infection.
Monitor for signs and symptoms of infection, and evaluate and treat promptly.
Neurological Toxicity: XPOVIO can cause life-threatening neurological toxicities.
Coadministration of XPOVIO with other products that cause dizziness or mental status changes may increase the risk of neurological toxicity.
Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, until the neurological toxicity fully resolves. Optimize hydration status, hemoglobin level, and concomitant medications to avoid exacerbating dizziness or mental status changes. Institute fall precautions as appropriate.
Embryo-Fetal Toxicity: XPOVIO can cause fetal harm when administered to a pregnant woman.
Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with a female partner of reproductive potential to use effective contraception during treatment with XPOVIO and for 1 week after the last dose.
ADVERSE REACTIONS
The most common adverse reactions (ARs) in ≥20% of patients with MM are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea, and upper respiratory tract infection.
The most common ARs, excluding laboratory abnormalities, in ≥20% of patients with DLBCL are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3-4 laboratory abnormalities in ≥15% of patients included thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. Grade 4 laboratory abnormalities in ≥5% were thrombocytopenia, lymphopenia, and neutropenia.
In patients with MM, fatal ARs occurred in 9% of patients. Serious ARs occurred in 58% of patients. Treatment discontinuation rate due to ARs was 27%. The most frequent ARs requiring permanent discontinuation in ≥4% of patients included fatigue, nausea, and thrombocytopenia.
In patients with DLBCL, fatal ARs occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal AR was infection (4.5% of patients). Serious ARs occurred in 46% of patients; the most frequent serious AR was infection. Discontinuation due to ARs occurred in 17% of patients.
USE IN SPECIFIC POPULATIONS
In MM, no overall difference in effectiveness of XPOVIO was observed in patients >65 years old when compared with younger patients. Patients ≥75 years old had a higher incidence of discontinuation due to an AR than younger patients, a higher incidence of serious ARs, and a higher incidence of fatal ARs.
Clinical studies in patients with relapsed or refractory DLBCL did not include sufficient numbers of patients aged 65 and over to determine whether they respond differently from younger patients.
The effect of end-stage renal disease (CLCR <15 mL/min) or hemodialysis on XPOVIO pharmacokinetics is unknown.
To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1-888-209-9326 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see XPOVIO Full Prescribing Information available at www.XPOVIO.com.
About Karyopharm Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company pioneering novel cancer therapies and 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 Selective Inhibitor of Nuclear Export (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 U.S. Food and Drug Administration (FDA) in July 2019 in combination with dexamethasone as a treatment for patients with heavily pretreated multiple myeloma. In June 2020, XPOVIO was approved by the FDA as a treatment for patients with relapsed or refractory diffuse large B-cell lymphoma. A Marketing Authorization Application for selinexor for patients with heavily pretreated multiple myeloma is also currently under review by the European Medicines Agency. 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.
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 Karyopharm's expectations and plans relating to XPOVIO for the treatment of patients with relapsed or refractory multiple myeloma or relapsed or refractory diffuse large B-cell lymphoma; commercialization of XPOVIO or any of its drug candidates and the commercial performance of XPOVIO; submissions to, and the review and potential approval of selinexor by, regulatory authorities, including the Company's regulatory strategy, the anticipated availability of data to support such submissions, timing of such submissions and actions by regulatory authorities and the potential availability of accelerated approval pathways; the expected design of the Company's clinical trials; the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, especially selinexor; Karyopharm's collaboration efforts with third-parties, including the National Cancer Institute; 2020 financial expectations, including forecasted non-GAAP R&D and SG&A expenses; and expectations of the sufficiency of Karyopharm's existing cash and investments. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond Karyopharm's control, that may cause actual events or results to differ materially from Karyopharm's current expectations. For example, there can be no guarantee that Karyopharm will successfully commercialize XPOVIO; that regulators will agree that selinexor qualifies for conditional approval in the E.U. as a result of data from the STORM study or confirmatory approval in the U.S. or EU based on the BOSTON study in patients with relapsed or refractory multiple myeloma; or that any of Karyopharm's drug candidates, including selinexor, will successfully complete necessary clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in the development or commercialization of 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: the risk that the COVID-19 pandemic could disrupt Karyopharm's business more severely than it currently anticipates, including by reducing sales of XPOVIO, interrupting or delaying research and development efforts, impacting the ability to procure sufficient supply for the development and commercialization of selinexor or other product candidates, delaying ongoing or planned clinical trials, impeding the execution of business plans, planned regulatory milestones and timelines, or inconveniencing patients; the adoption of XPOVIO in the commercial marketplace, the timing and costs involved in commercializing XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; the ability to retain regulatory approval of XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; 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; the ability of Karyopharm or its third party collaborators or successors in interest to fully perform their respective obligations under the applicable agreement and the potential future financial implications of such agreement; 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 Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, which was filed with the Securities and Exchange Commission (SEC) on May 5, 2020, 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.
KARYOPHARM THERAPEUTICS INC. | ||||||||||||||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||||||
(unaudited) | ||||||||||||||||
(in thousands, except per share amounts) | ||||||||||||||||
Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
2020 | 2019 | 2020 | 2019 | |||||||||||||
Revenues: | ||||||||||||||||
Product revenue, net | $ | 18,601 | $ | — | $ | 34,662 | $ | — | ||||||||
License and other revenue | 14,913 | 9,493 | 16,990 | 9,648 | ||||||||||||
Total revenues | 33,514 | 9,493 | 51,652 | 9,648 | ||||||||||||
Operating expenses: | ||||||||||||||||
Cost of sales | 396 | — | 1,215 | — | ||||||||||||
Research and development | 42,594 | 26,517 | 76,591 | 64,491 | ||||||||||||
Selling, general and administrative | 30,843 | 24,662 | 61,521 | 51,765 | ||||||||||||
Total operating expenses | 73,833 | 51,179 | 139,327 | 116,256 | ||||||||||||
Loss from operations | (40,319) | (41,686) | (87,675) | (106,608) | ||||||||||||
Other income (expense): | ||||||||||||||||
Interest income | 849 | 1,412 | 1,824 | 3,183 | ||||||||||||
Interest expense | (6,758) | (3,089) | (13,267) | (6,087) | ||||||||||||
Other expense, net | (61) | (44) | (36) | (46) | ||||||||||||
Total other expense, net | (5,970) | (1,721) | (11,479) | (2,950) | ||||||||||||
Loss before income taxes | (46,289) | (43,407) | (99,154) | (109,558) | ||||||||||||
Income tax provision | (137) | (8) | (203) | (18) | ||||||||||||
Net loss | $ | (46,426) | $ | (43,415) | $ | (99,357) | $ | (109,576) | ||||||||
Net loss per share—basic and diluted | $ | (0.63) | $ | (0.71) | $ | (1.41) | $ | (1.80) | ||||||||
Weighted-average number of common shares | 73,237 | 60,929 | 70,475 | 60,893 |
Karyopharm Therapeutics Inc. | ||||||||
Condensed Consolidated Balance Sheets | ||||||||
(in thousands) | ||||||||
(unaudited) | ||||||||
June 30, 2020 | December 31, 2019 | |||||||
Assets | ||||||||
Cash, cash equivalents and investments | $ | 345,573 | $ | 263,972 | ||||
Restricted cash | 2,629 | 1,831 | ||||||
Accounts receivable | 9,581 | 7,862 | ||||||
Property and equipment, net | 2,592 | 3,046 | ||||||
Other assets | 19,636 | 18,252 | ||||||
Total assets | $ | 380,011 | $ | 294,963 | ||||
Liabilities and stockholders' equity | ||||||||
Deferred revenue | $ | 297 | $ | 4,533 | ||||
Convertible senior notes | 113,776 | 109,857 | ||||||
Deferred royalty obligation | 73,588 | 73,588 | ||||||
Other liabilities | 62,551 | 57,211 | ||||||
Total liabilities | 250,212 | 245,189 | ||||||
Total stockholders' equity | 129,799 | 49,774 | ||||||
Total liabilities and stockholders' equity; 73,366 and 65,370 shares | $ | 380,011 | $ | 294,963 |
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SOURCE Karyopharm Therapeutics Inc.
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