Core Insights - Karyopharm Therapeutics is nearing the completion of patient enrollment for its Phase 3 SENTRY trial in myelofibrosis, with top-line results expected in March 2026 [2][9] - The company reported total revenue of $37.9 million for the second quarter of 2025, with U.S. XPOVIO (selinexor) net product revenue at $29.7 million, reflecting a 6% increase compared to the same quarter in 2024 [1][15] - Karyopharm has reaffirmed its full-year 2025 total revenue guidance of $140 million to $155 million and updated its U.S. XPOVIO net product revenue guidance to $110 million to $120 million [1][13] Financial Performance - Total revenue for Q2 2025 was $37.9 million, down from $42.8 million in Q2 2024 [15] - U.S. net product revenue for Q2 2025 was $29.7 million, compared to $28.0 million in Q2 2024 [5][15] - License and other revenue decreased to $8.2 million in Q2 2025 from $14.8 million in Q2 2024, primarily due to non-recurring license-related revenue recognized in the previous year [16] - R&D expenses for Q2 2025 were $32.8 million, down from $38.4 million in Q2 2024, attributed to reduced personnel and clinical trial costs [18] - SG&A expenses decreased to $28.5 million in Q2 2025 from $31.1 million in Q2 2024, reflecting cost reduction initiatives [19] - The company reported a net loss of $37.3 million for Q2 2025, compared to a net income of $23.8 million in Q2 2024 [22] Clinical Development - The Phase 3 SENTRY trial is targeting 350 patients and is evaluating the efficacy of 60 mg once-weekly selinexor in combination with ruxolitinib [5][9] - Preliminary safety data from the first 61 patients in the SENTRY trial suggest potential improvements in treatment-emergent adverse events compared to previous studies [5] - Karyopharm is also enrolling patients in the Phase 3 XPORT-EC-042 trial for endometrial cancer and has completed enrollment in the Phase 3 XPORT-MM-031 trial for multiple myeloma [7][8][11] Strategic Initiatives - The company is exploring financing transactions and strategic alternatives to extend its cash runway and enhance liquidity [1][14] - Karyopharm's cash position as of June 30, 2025, was $52.0 million, down from $109.1 million at the end of 2024 [23]

Financial Data and Key Metrics Changes - In Q2 2025, Coherus Oncology reported net revenue of $10 million, representing a 36% increase quarter-over-quarter and a 65% increase year-over-year [29][37] - The company ended Q2 with $238 million in cash and investments, projecting sufficient cash to provide runway through 2026 [37][38] - Cost savings are expected to reach approximately $30 million on an annualized basis due to headcount reductions [38] Business Line Data and Key Metrics Changes - Lactorsi's net revenue grew 36% over Q1 2025, driven by strong demand from new patients and a positive impact from wholesaler inventory rebuild [29][30] - The number of new purchasing accounts for Lactorsi increased by 20%, indicating broader adoption among physicians [30] Market Data and Key Metrics Changes - The nasopharyngeal cancer market opportunity for Lactorsi is estimated to be between $150 million and $200 million [29][34] - The U.S. market opportunity for casdozoketog in hepatocellular carcinoma is approximately $4 billion, while the potential for CHS-114 in second-line head and neck cancer is about $4.5 billion [34] Company Strategy and Development Direction - Coherus Oncology aims to establish Lactorsi as the standard of care for nasopharyngeal cancer, focusing on educating physicians and expanding market presence [28][30] - The company is pursuing strategic partnerships to enhance its pipeline and expand indications cost-effectively [9][20] - The development strategy includes advancing combination studies across prioritized indications and leveraging collaborations for indication expansion [9][20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving revenue goals of $40 million to $50 million for 2025, with a focus on maximizing the nasopharyngeal cancer indication [28][34] - The company anticipates key data readouts in the first half of 2026, which are expected to validate their pipeline and support future growth [41][44] Other Important Information - Coherus Oncology completed a strategic repositioning and renamed the company to better reflect its mission in oncology [5] - The company is the only independent U.S. biotech developing a CCR8 Treg depleter, which is expected to have significant market potential [14] Q&A Session Summary Question: Importance of identifying a partner for the CCRA program - Management indicated that they are actively seeking partners to accelerate progress and offset development costs, with data readouts expected in the first half of next year [46][47][49] Question: Confidence in reaching revenue goals for Lactorsi - Management expressed confidence in achieving peak revenues by 2028, supported by strong uptake following NCCN guideline updates [52][54] Question: Competitive landscape for anti CCR8 and anti IL-27 programs - Management noted that there are currently no known competitors in clinical development for anti IL-27, while the anti CCR8 program is seeing robust development activity [58][60][61] Question: Community engagement strategy - The company is focusing on a multi-pronged approach to engage community oncologists, anticipating that peak sales will take three to four years due to the dispersed nature of patient management [66][67] Question: Enrollment progress for casdozoketog trials - Management confirmed that recruitment is going well in both the U.S. and Asia Pacific regions, with data expected in the first half of next year [74][75] Question: Impact of guidelines on community adoption - Management highlighted that while academic centers have adopted the guidelines quickly, community oncologists require ongoing education to integrate the guidelines into practice [96][98]

Core Insights - Syndax Pharmaceuticals reported strong financial results for Q2 2025, with significant growth in net revenues from its products Revuforj and Niktimvo, indicating a positive trajectory towards profitability [2][3][4]. Financial Performance - Revuforj (revumenib) generated $28.6 million in net revenue for Q2 2025, reflecting a 43% increase compared to Q1 2025 [4][8]. - Niktimvo (axatilimab-csfr) achieved $36.2 million in net revenue in its first full quarter post-launch, with Syndax recording $9.4 million in collaboration revenue from this product [3][11]. - Total revenue for Q2 2025 was reported at $38.0 million, combining Revuforj and Niktimvo revenues [8]. - The company had cash, cash equivalents, and investments totaling $517.9 million as of June 30, 2025, which is expected to support operations until profitability [7][14]. Product Updates - Revuforj is under Priority Review by the FDA for the treatment of relapsed or refractory mutant NPM1 acute myeloid leukemia (mNPM1 AML), with a PDUFA action date set for October 25, 2025 [4][17]. - Niktimvo is positioned as a treatment for chronic graft-versus-host disease (GVHD) and has shown rapid sales growth since its launch [11][21]. Clinical Trials and Research - Multiple ongoing clinical trials for revumenib are evaluating its efficacy in various acute leukemia settings, including pivotal Phase 3 trials [5][18]. - Recent data from the AUGMENT-101 trial indicated an overall response rate of 48% for patients with R/R mNPM1 AML, with a complete remission rate of 26% [4][5]. Expense Management - Research and development expenses increased to $62.2 million in Q2 2025, up from $48.7 million in the same period last year, primarily due to costs associated with revumenib trials [9]. - Selling, general, and administrative expenses rose to $43.8 million from $29.1 million year-over-year, reflecting increased marketing efforts for Revuforj [10]. Future Outlook - The company anticipates stable operating expenses over the next few years, which, combined with product revenues, is expected to lead to profitability [14]. - For Q3 2025, total expenses are projected to be between $95 million and $100 million, excluding non-cash stock compensation [13].

Financial Data and Key Metrics Changes - For the six months ended June 30, 2025, the company reported revenues of $2.5 million compared to $301,000 in 2024, primarily due to the BARDA Project NextGen contract that began in June 2024 [16] - Research and development expenses increased to $10 million in 2025 from $8.7 million in 2024, representing a 16% increase, mainly associated with the BARDA contract and other programs [17] - General and administrative expenses rose to $3.2 million in 2025 from $2.5 million in 2024, a 27% increase attributed to higher investor relations consulting costs and stock-based compensation [18] - The net loss for the six-month period in 2025 was approximately $10.7 million or $0.79 per share, compared to a net loss of $10.9 million or $4.68 per share in 2024 [18] Business Line Data and Key Metrics Changes - The company is focused on advancing its product candidates, including GEO MVA, GEO CM04S1, and Gideptin, which address significant unmet healthcare needs [5][14] - The advanced MVA manufacturing process is expected to provide a competitive advantage in the production of MVA-based vaccines and therapies [6] Market Data and Key Metrics Changes - The company anticipates significant governmental interest in U.S.-based supply chains, particularly in light of the current overdependence on non-U.S. suppliers [9] - The company is engaged in discussions with various stakeholders, including the White House and WHO, regarding the progress of GEO MVA [9] Company Strategy and Development Direction - The company aims to develop innovative cancer therapies and infectious disease vaccines, focusing on expedited registration pathways and establishing partnerships for worldwide development and commercialization [14] - The strategic focus for 2025 includes advancing GEO MVA to clinical evaluation readiness, progressing GEO CM04S1 for immunocompromised patients, and enhancing the advanced MVA manufacturing process [14] Management's Comments on Operating Environment and Future Outlook - Management remains confident in the progress and outlook for its product portfolio, particularly in light of recent guidance from the European Medicines Agency for GEO MVA [5][6] - The company is committed to addressing the medical needs of immunocompromised patients with its COVID-19 vaccine candidate, GEO CM04S1, which is expected to provide a more robust immune response [10][11] Other Important Information - The company completed CGMP production and quality release of the clinical batch of GEO MVA vaccine material, with plans to produce additional product for potential emergency use distribution [8] - The company has initiated discussions for potential collaborations in the long-term development and commercialization of Gideptin [14] Q&A Session Summary Question: Regarding the new patch method for the MDA vaccine - The company does not intend to use the patch for the clinical program and plans to utilize the standard vaccine delivery method [24] Question: Is the making of enough vaccine product a limiting step for the trial start? - The vaccine is largely manufactured, and various components need to come together before the trial can start [26] Question: What is the expected start date for the Gideptin trial? - The target start date for the Gideptin trial is the second half of 2026 [51] Question: Can you elaborate on the major pathological response endpoint for Gideptin? - The major pathological response is defined by the extent of response in the resected tumor tissue, with a primary endpoint focused on pathological response [45] Question: What is the status of the BARDA manufacturing proposal? - The company was selected for funding but is currently in a holding pattern pending funding availability [65]

Core Insights - Revumenib, in combination with venetoclax and azacitidine, shows promising clinical activity in older patients with newly diagnosed mNPM1 and KMT2Ar AML, achieving a 67% complete remission (CR) rate and an 88% overall response rate (ORR) [1][2][6] - The ongoing Phase 3 EVOLVE-2 trial is evaluating the efficacy of this combination in newly diagnosed mNPM1 AML patients who are unfit for intensive chemotherapy [1][2] Group 1: Clinical Trial Results - The BEAT AML trial reported a 67% CR rate (29 out of 43 patients) and an 88% ORR (38 out of 43 patients) among newly diagnosed older adults with mNPM1 or KMT2Ar AML [1][6] - Among 37 patients assessed for measurable residual disease (MRD), 100% were found to be MRD negative [6] - The median duration of composite complete remission (CRc) was 12.0 months [6] Group 2: Patient Demographics and Safety - As of September 2024, 43 patients were enrolled, with 79% having mNPM1 AML and 21% KMT2Ar AML; the median age was 70 years [4][5] - Revumenib was generally well tolerated, with common non-hematologic treatment-emergent adverse events including nausea (60%), constipation (53%), and QTc prolongation (44%) [5][6] Group 3: Comparative Outcomes - The observed median overall survival (OS) was 15.5 months, with a CRc rate of 77% in mNPM1 patients and 89% in KMT2Ar patients [7][8] - Historical data indicated lower CRc rates and median OS for patients treated with venetoclax and azacitidine alone, highlighting the potential of revumenib in improving outcomes [8] Group 4: Future Developments - Revumenib is being developed for the treatment of relapsed or refractory AML with mNPM1 mutations, with a supplemental NDA submitted in April 2025 [10] - The company is conducting multiple trials of revumenib in combination with standard-of-care agents across various treatment settings [10][25]

Core Insights - Karyopharm Therapeutics Inc. announced that its abstract on selinexor monotherapy for myelofibrosis has been selected for presentation at the 2025 European Hematology Association Annual Meeting [1][2] - The data from the Phase 2 XPORT-MF-035 trial indicates that selinexor shows clinical activity in heavily pretreated myelofibrosis patients, demonstrating spleen volume reduction, hemoglobin stabilization, and symptom improvement [2][4] Summary by Sections Company Overview - Karyopharm Therapeutics is a commercial-stage pharmaceutical company focused on developing novel cancer therapies, particularly through its lead compound, XPOVIO (selinexor), which is a first-in-class oral exportin 1 (XPO1) inhibitor [4][11] - The company has received regulatory approvals for XPOVIO in multiple oncology indications in the U.S. and various international markets, including Europe and China [5][11] Clinical Trial Insights - The XPORT-MF-035 trial involved 24 patients, randomized 1:1 to receive either selinexor monotherapy or physician's choice treatment, with selinexor administered at 80 mg weekly for the first two cycles, then reduced to 60 mg [4] - Key findings from the trial include: - 67% of selinexor-treated patients achieved a spleen volume reduction of 25% or more compared to 38% in the physician's choice group [4] - 33% of selinexor patients achieved a spleen volume reduction of 35% or more versus 13% in the physician's choice group [4] - Patients on selinexor had higher mean hemoglobin levels and lower rates of red blood cell transfusions compared to those on physician's choice [4] Safety and Adverse Events - The most common treatment-emergent adverse events (TEAEs) in the selinexor arm included decreased weight (50%), asthenia (42%), and nausea (33%) [4] - Serious adverse reactions were reported in 52% of patients in the BOSTON trial, with a treatment discontinuation rate of 19% due to adverse reactions [9]

Core Insights - Karyopharm Therapeutics reported positive developments in its Phase 3 SENTRY trial for selinexor in JAKi-naïve myelofibrosis, which has passed a planned futility analysis and is approximately 80% enrolled [2][4] - The company experienced a 5% increase in demand for XPOVIO® (selinexor) in Q1 2025 compared to Q1 2024, with total revenue of $30.0 million [5][15] - Karyopharm reaffirmed its full-year 2025 revenue guidance of $140 million to $155 million, including U.S. net product revenue guidance of $115 million to $130 million [21] Financial Performance - Total revenue for Q1 2025 was $30.0 million, down from $33.1 million in Q1 2024 [15] - U.S. net product revenue was $21.1 million in Q1 2025, compared to $26.0 million in Q1 2024, impacted by a $5.0 million increase in product return reserves [16] - License and other revenue increased to $9.0 million in Q1 2025 from $7.1 million in Q1 2024, attributed to timing of revenue recognition from Menarini [17] Research and Development Highlights - The Phase 3 SENTRY trial is evaluating the combination of selinexor and ruxolitinib, targeting 350 patients for enrollment [4] - The Phase 2 XPORT-MF-035 trial showed promising results for selinexor monotherapy in hard-to-treat myelofibrosis patients, with 67% achieving spleen volume reduction of 25% or more [10] - Enrollment continues in the Phase 3 XPORT-EC-042 trial for selinexor as maintenance therapy in advanced endometrial cancer [6] Operational Objectives - Karyopharm aims to complete target enrollment for the Phase 3 SENTRY trial by June/July 2025 and report top-line results in late 2025/early 2026 [11] - The company is exploring alternatives to extend its cash runway, with cash and investments totaling $70.3 million as of March 31, 2025 [25][14] - R&D expenses for Q1 2025 were $34.6 million, slightly down from $35.4 million in Q1 2024, reflecting reduced personnel costs [19]