– Achieves Second Quarter 2024 Total Revenue of $42.8 Million and U.S. XPOVIO® (selinexor) Net Product Revenue of $28.0 Million; Positive Momentum ex-US with Continued Regulatory and Reimbursement Approvals – – Updated Clinical Results at ASCO Annual Meeting Showed Median Progression-Free Survival (PFS) of 28.4 Months in the TP53 Wild-Type Exploratory Subgroup and 39.5 Months in the Proficient Mismatched Repair Status (pMMR) TP53 Wild-Type Exploratory Subgroup from Phase 3 SIENDO Study of Selinexor Maintena ...

-- Conference Call Scheduled for Tuesday, August 6, 2024, at 8:00 a.m. ET -- NEWTON, Mass., July 31, 2024 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced it will report second quarter 2024 financial results on Tuesday, August 6, 2024. Karyopharm's management team will host a conference call and audio webcast at 8:00 a.m. ET on Tuesday, August 6, 2024, to discuss the financial results and other company u ...

- Additional reimbursements of XPOVIO® in APAC markets are expected. MM, the second most common hematologic malignancy, is caused by the dysregulated proliferation of plasma cells. Patients with MM face a range of challenges including a high relapse rate, short survival, and limited treatment options. According to market data, the global MM market has exceeded US$17 billion in 2023 and is expected to reach US$ 26 billion by 2028, with a compound annual growth rate (CAGR) of 8.7%[1]. Antengene has already su ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-36167 Karyopharm Therapeutics Inc. (Exact name of registrant as specified in its charter) (State or other jur ...

Exhibit 99.1 Targeting Disease at the Nuclear Pore "We have taken a significant step that improves our capital structure, strengthening our opportunity to realize the full value of three potential new indications for selinexor. We are strongly positioned for our next stage of growth, driven by our focused and rapidly advancing late-stage pipeline with expected data readouts from our three ongoing Phase 3 trials next year," said Richard Paulson, President and Chief Executive Officer of Karyopharm. First Quar ...

– $148.0 Million (86%) of Existing Convertible Notes due in 2025 to be Exchanged for $111.0 Million of New Convertible Notes due in 2029 and Warrants – – Issues New $100.0 Million Senior Secured Term Loan due in 2028 – – Repays Principal Portion and Amends Royalty Agreement with HealthCare Royalty – NEWTON, Mass., May 8, 2024 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, announced today that it has entered into a ser ...

Thomas Barwick Topline Summary and Update Karyopharm Therapeutics Inc. (NASDAQ:KPTI) is a long-struggling commercial-stage biotech working on developing the market for their nuclear exportin 1 inhibitor selinexor. While it is currently approved to treat multiple myeloma in later lines of therapy, KPTI is on track to potentially expand the label into endometrial cancer, with a phase 3 trial eagerly anticipated (though still a ways away). Now that we know the abstracts being presented at the American Society ...

Financial Data and Key Metrics Changes - Total revenue for Q4 2023 was $33.7 million, consistent with Q4 2022, while total revenue for the full year 2023 was $146 million, down from $157.1 million in 2022 [55][56] - Net product revenue from US commercial sales of XPOVIO for Q4 2023 was $25.1 million, compared to $31.1 million in Q4 2022, and for the full year 2023, it was $112 million, down from $120.4 million in 2022 [56][60] - The gross-to-net discount for XPOVIO in Q4 2023 was 23.5%, and for the full year 2023, it was 22%, with expectations for 2024 to be in the 25% to 30% range [68] Business Line Data and Key Metrics Changes - XPOVIO's new patient share in the community setting approached 70% in the second to fourth line, representing double-digit growth year-over-year [44] - The community setting now accounts for two-thirds of the business, with a 20% year-over-year increase in prescribing sites [51][52] - The company expects US XPOVIO net product revenue for 2024 to be in the range of $100 million to $120 million, reflecting a balance of growth conviction and competitive market recognition [60][71] Market Data and Key Metrics Changes - The competitive landscape in the multiple myeloma market has intensified, particularly with the introduction of bispecific therapies in the second half of 2023, impacting demand in the academic setting [12][44] - Despite competition, the company achieved total demand growth year-on-year in the community setting in 2023, which is a key growth driver for 2024 [84] Company Strategy and Development Direction - The company is focused on advancing its late-stage pipeline, particularly in myelofibrosis and endometrial cancer, with three pivotal Phase 3 trials expected to read out results in 2025 [5][17] - The strategy includes shifting the use of selinexor into earlier lines of therapy, where patients tend to stay on treatment longer, thus increasing duration of therapy [95] - The company aims to leverage its existing commercial infrastructure to support rapid launches of selinexor in new indications, with a potential peak annual revenue of approximately $2 billion in the US alone [13][60] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing Phase 3 studies and the potential for selinexor to address high unmet needs in cancer treatment [67][73] - The company anticipates a cash runway into late 2025, supported by expected revenues from XPOVIO and partnerships [66][71] - Management highlighted the importance of evolving the regulatory landscape and potential changes in study design to enhance the value proposition of selinexor [105] Other Important Information - The company reported a decrease in R&D expenses due to reduced personnel costs, while SG&A expenses also decreased, reflecting cost optimization efforts [64][69] - The existing cash and investments as of December 31, 2023, totaled $192.4 million, down from $279.7 million in 2022 [70] Q&A Session Summary Question: Can you quantify the community versus academic setting growth in 2023? - The company achieved total demand growth year-on-year in the community setting, with a 20% increase in prescriber base, which is a significant growth driver for 2024 [84][85] Question: What is the impact of BCMA therapies moving into 2024? - The introduction of bispecific therapies posed a significant competitive threat in the academic setting, but the community setting has not seen a major impact yet [78] Question: Can you comment on the enrollment experience of the Phase 3 SPD triplet study in multiple myeloma? - Enrollment is progressing well, with significant interest in the study both in the US and EU, and completion of enrollment is expected later this year [115] Question: What is the company's cash position and runway? - The company finished 2023 with $192 million in cash, with a projected cash runway into late 2025, considering expected revenues and expenditures [119]

Financial Performance & Guidance - Karyopharm reported XPOVIO Net Product Revenue of $112 million for FY 2023 and $25 million for Q4 2023[118] - The company anticipates U S XPOVIO Net Product Revenue between $100 million and $120 million in 2024[119] - Karyopharm expects its cash runway to be sufficient to fund planned operations into late 2025[122] - Total revenue was $33.7 million for 4Q 2023 and $146 million for FY 2023[138] Clinical Development Programs - Karyopharm is conducting a Phase 3 trial of selinexor + ruxolitinib in treatment-naïve myelofibrosis (MF)[18] - Preliminary analysis from the Phase 3 SIENDO trial showed encouraging overall survival in the TP53 wild-type exploratory subgroup[29] - The company is planning to report top-line results from the pivotal EC-042 Phase 3 trial in TP53 wildtype endometrial cancer in 1H 2025[141] - Karyopharm is also expecting to report top-line results from the Phase 3 trial of selinexor + ruxolitinib in treatment-naïve MF in 2H 2025[141] Market Opportunity - Karyopharm estimates a potential for ~$2 billion in annual peak U S revenues from selinexor across multiple myeloma, endometrial cancer, and myelofibrosis[16, 20] - Approximately 20,000 people in the U S live with myelofibrosis[25] - In the U S each year, approximately 16,000 patients are diagnosed with advanced and recurrent endometrial cancer[100]

Financial Performance - As of December 31, 2023, the company reported an accumulated deficit of $1.5 billion and net losses of $143.1 million for the year, compared to $165.3 million in 2022 and $124.1 million in 2021[574]. - Total revenue for 2023 was $146.0 million, which included $112.0 million from XPOVIO net product revenue and $34.0 million from license revenue[574]. - Net product revenue from U.S. commercial sales of XPOVIO decreased by 7% in 2023 compared to 2022, primarily due to the closure of myeloma foundations that support Medicare Part D patients[585]. - License and other revenue decreased by $2.6 million in 2023 compared to 2022, mainly due to a decline in milestone-related revenue and royalty revenue from Antengene, partially offset by increased revenue from Menarini[587]. - Interest income rose significantly by $8.58 million (364%) in 2023 compared to 2022, contributing to a total other expense net decrease of $9.5 million (42%)[595]. - Net cash used in operating activities decreased by $56.83 million (38%) in 2023 compared to 2022, primarily due to reduced expenses and milestone payments received[602]. - The company received $32.0 million in milestone and upfront payments in 2023, with additional potential payments tied to development goals and sales milestones[607]. Expenses and Cost Management - Total operating expenses decreased by $23.7 million (8%) in 2023 compared to 2022, with research and development expenses down by $9.9 million (7%) and selling, general and administrative expenses down by $13.5 million (9%)[589][593]. - Research and development expenses for 2023 were $138.75 million, a decrease from $148.66 million in 2022, primarily due to a reduction in personnel costs and stock-based compensation[591]. - Clinical trial costs increased by $9.2 million (16%) in 2023, driven by advancements in three pivotal Phase 3 trials[591][592]. - Selling, general and administrative expenses are expected to decrease slightly in 2024 due to cost optimization efforts[594]. - The company anticipates research and development expenses to increase slightly in 2024 as it continues to advance its pivotal Phase 3 trials[592]. Cash and Liquidity - As of December 31, 2023, the company had $191.4 million in cash, cash equivalents, and investments[574]. - As of December 31, 2023, the company had cash, cash equivalents, and investments totaling $191.4 million, which are expected to fund operations for at least twelve months[619]. - The company has provided a security deposit of $0.3 million for its operating lease, classified as long-term restricted cash[612]. - The company does not believe its cash and investments have significant risk of default or illiquidity, but acknowledges potential instability in financial institutions[620]. Clinical Development and Commercialization - The company plans to continue clinical trials and seek additional approvals for selinexor in multiple high unmet need cancer indications, including endometrial cancer and multiple myeloma[573]. - The commercialization of XPOVIO is supported by a comprehensive patient and healthcare provider support program, KaryForward™, and a dedicated network of specialty pharmacy providers[571]. - The company announced in January 2024 that further clinical development of the eltanexor program is on hold to focus resources on prioritized late-stage programs[573]. - XPOVIO has received regulatory approval in over 40 countries outside the U.S. and is commercially available in a growing number of countries[572]. - The company expects changes in Medicare Part D under the Inflation Reduction Act to reduce the need for patient assistance programs in 2024 compared to 2023[586]. - License and other revenue is expected to increase in 2024 compared to 2023 due to anticipated milestone achievements[588]. - The company expects to continue incurring costs related to commercialization expenses for its products, depending on the responsibilities of collaborators[615]. Market and Risk Factors - The company is exposed to market risks related to interest rates, with an immediate 100 basis point shift in rates not expected to materially affect the fair market value of its investment portfolio[619]. - The company does not currently hedge its foreign currency exchange rate risk, which could impact costs related to contracts with organizations in Canada, the UK, and Europe[621]. - The company is subject to fluctuations in foreign currency rates for clinical trial budgets frequently denominated in foreign currencies[621]. - The company has incurred significant losses since inception and may not achieve profitability for several years, necessitating reliance on additional financing[616].