On May 12, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the first quarter ended March 31, 2025, and provided an overview of recent developments (Press release, UroGen Pharma, MAY 12, 2025, View Source [SID1234652889]).

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"We are entering a pivotal and exciting period for UroGen as we approach the anticipated FDA approval of our lead pipeline product, UGN-102, in June for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer," said Liz Barrett, President and Chief Executive Officer of UroGen. "This milestone has the potential to mark the first major advancement in treatment for this patient population in decades, delivering a much-needed novel and innovative treatment option that may offer meaningful disease and treatment-free intervals. With momentum building across the organization, we are entering the final phase of launch readiness. If approved, UGN-102 represents a significant commercial opportunity, with a total addressable market of over $5 billion. Backed by a strong balance sheet and a growing pipeline, we are well-positioned to build a long-term, sustainable growth company. We remain steadfast in our mission to transform the treatment paradigm in uro-oncology and see a great opportunity to advance patient care and deliver value for shareholders."

Q1 2025 and Recent Business Highlights:

UGN-102 (mitomycin) for intravesical solution

UroGen’s New Drug Application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution as a treatment for recurrent low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC), is currently under review at the U.S. Food and Drug Administration (FDA). The FDA granted a Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025. The FDA has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting on May 21, 2025 to discuss the UGN-102 NDA.
Updated 18-month Duration of Response (DOR) data from the Phase 3 ENVISION trial evaluating UGN-102 in patients with recurrent LG-IR-NMIBC were featured in a podium presentation by Dr. Sandip Prasad at the American Urological Association (AUA) 2025 Annual Meeting on April 26 in Las Vegas. The data are consistent with prior Kaplan-Meier estimates, with the probability of remaining in complete response (CR) of 80.6% (95% CI: 74.0, 85.7) at 18-months after achieving CR at 3 months (N=101) compared to 82.5% (95% CI: 76.1, 87.3) at 12-months after 3-month CR (N=146). Median follow-up time at 18 months was 18.7 months after the three-month CR.
The results of a patient reported outcomes analysis from three UGN-102 studies (OPTIMA II, ATLAS and ENVISION) were presented in a poster at the AUA meeting. The results showed that UGN-102 did not have a negative impact on symptom burden, patient function, or quality of life.
Long-term outcomes of the OPTIMA II LT study of UGN-102 were also presented at the meeting and demonstrated median DOR of 24.2 months (95% CI 9.72, 47.18) with a median follow-up time of 33.6 months (95% CI 10.78, 42.94) in the 41 patients who achieved a CR in the parent study.
Results from a post-hoc sub-analysis of the ENVISION trial, showing that tumor burden and the number of tumors did not significantly affect the CR rate or durability of response for patients treated with UGN-102, were presented at the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO-GU 2025) in February 2025.
Results from the ENVISION trial were published in the February 2025 issue of The Journal of Urology.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC)

Generated net product revenue of $20.3 million in the quarter ended March 31, 2025, an increase of 8% over the $18.8 million reported for the same quarter in 2024. Underlying demand grew by 12% year-over-year.
A poster featuring long-term outcomes of patients with recurrent and new-onset LG-UTUC who achieved CR in the Phase 3 trial of JELMYTO was presented at AUA 2025. As previously reported, the median DOR of all patients achieving CR in the JELMYTO Phase 3 study was 47.8 months, irrespective of whether their cancer was new onset or recurrent.
A long-term study on JELMYTO titled, "Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study," was published online in Urologic Oncology: Seminars and Investigations in January 2025. The results showed 68% recurrence-free survival rate at three years across a broad patient population with LG-UTUC.
UGN-301 (zalifrelimab) intravesical solution, an anti-CTLA4 antibody for use in high-grade non-muscle invasive bladder cancer (NMIBC)

Enrollment is complete in the multi-arm dose escalation Phase 1 clinical study of UGN-301 in patients with high-grade NMIBC, alone and in combination with fixed dose UGN-201 or gemcitabine. The investigational treatments demonstrated an acceptable safety profile and were generally well tolerated across dose levels. Responses were observed in both monotherapy and combination therapy arms, and patient follow-up is ongoing to further assess the durability of these responses.
Next-generation investigational oncolytic virus UGN-501 (ICVB-1042)

In February 2025, UroGen expanded its nonclinical oncology portfolio with the purchase of product candidate UGN-501 from IconOVir Bio, Inc (IconOVir). UGN-501 is a potent and fast-replicating investigational next generation oncolytic virus therapy, being developed as a locally administered treatment for bladder cancer and other specialty cancers. UroGen hosted a webinar to discuss UGN-501 (ICVB-1042) on February 20, 2025, and a replay can be accessed on the Company’s website here.
Next-generation novel mitomycin-based formulation for urothelial cancers

Enrollment is ongoing in the Phase 3 UTOPIA clinical trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with LG-IR-NMIBC and enrollment is expected to be completed by mid-2025. UGN-103 is a next generation product that combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH. UGN-103 is planned to follow the potential FDA approval and launch of UGN-102 for recurrent LG-IR-NMIBC. To learn more about the UTOPIA trial, refer to clinicaltrials.gov/NCT06331299.
UroGen plans to initiate a Phase 3 trial to explore the safety and efficacy of UGN-104 by mid-2025. UGN-104 is a next generation investigational product for LG-UTUC.
First quarter 2025 Financial Results

JELMYTO Revenue: JELMYTO net product revenues were $20.3 million for the three months ended March 31, 2025, compared with $18.8 million for the same quarter of 2024. Year-over-year revenue growth of 8% was driven by underlying demand growth of 12%, partially offset by higher 340B chargebacks.

R&D Expenses: R&D expenses for the first quarter of 2025 were $19.9 million, including non-cash share-based compensation expense of $0.6 million as compared to $15.5 million, including non-cash share-based compensation expense of $0.5 million, for the same period in 2024. The increase in R&D expenses of $4.4 million was primarily driven by the equity consideration issued to IconOVir for the acquisition of UGN-501 (ICVB-1042) which was expensed in the first quarter of 2025, higher manufacturing costs, and costs associated with the Phase 3 UTOPIA trial for UGN-103, partially offset by lower clinical trial costs and regulatory expenses in connection with UGN-102.

SG&A Expenses: SG&A expenses for the first quarter of 2025 were $35.0 million, including non-cash share-based compensation expense of $2.5 million. This compares to $27.3 million, including non-cash share-based compensation expense of $2.2 million, for the same period in 2024. The increase in SG&A expenses of $7.7 million was primarily driven by UGN-102 commercial preparation activities.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $4.6 million in the first quarter of 2025, compared to $5.7 million in the same period in 2024. The decrease was primarily driven by changes in underlying assumptions for remeasuring the effective rate.

Interest Expense on Long-Term Debt: Interest expense related to the $125 million term loan facility with funds managed by Pharmakon Advisors was $4.1 million in the first quarter of 2025, compared to $2.4 million in the same period in 2024. The increase was primarily attributable to the interest expense on the third tranche of the loan that was funded in September 2024.

Net Loss: UroGen reported a net loss of $43.8 million or ($0.92) per basic and diluted share in the first quarter of 2025 compared with a net loss of $32.3 million or ($0.87) per basic and diluted share in the same period in 2024.

Cash and Equivalents: As of March 31, 2025, cash, cash equivalents and marketable securities totaled $200.4 million.

For further details on the Company’s financials, refer to Form 10-Q, filed with the SEC.

2025 JELMYTO Revenue and Company Operating Expense Guidance: Guidance for full-year 2025 net product revenues for JELMYTO remains unchanged and is expected to be in the range of $94 to $98 million. This implies a year-over-year growth rate of approximately 8% to 12% over the $87.4 million in demand driven JELMYTO sales in 2024, which excludes the $3.0 million in CREATES Act sales reported in 2024. Continue to expect full-year 2025 operating expenses to be in the range of $215 to $225 million, including non-cash share-based compensation expense of $11 million to $14 million.

Conference Call & Webcast Information: Members of UroGen’s management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review UroGen’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

On May 12, 2025 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported that it will webcast its presentation at the BofA Securities 2025 Healthcare Conference at 9:20 a.m. PT on Tuesday, May 13, 2025 (Press release, Sana Biotechnology, MAY 12, 2025, View Source [SID1234652888]). The presentation will feature a business overview and update by Steve Harr, Sana’s President and Chief Executive Officer.

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The webcast will be accessible on the Investor Relations page of Sana’s website at View Source A replay of the presentation will be available at the same location for 30 days following the conference.

On May 12, 2025 Nusano, a physics company transforming the production of radioisotopes, reported its Chief Product Officer, Kevin Haehl, will participate in a radioisotope supply chain panel discussion at the 21st Annual PEGS (Protein & Antibody Engineering) Summit on Wednesday, May 14, 2025, in Boston (Press release, Nusano, MAY 12, 2025, View Source [SID1234652887]).

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Haehl is a 35-year veteran of the biotech and pharmaceutical manufacturing industries, with extensive experience in operations. During the panel, he will describe existing radiopharmaceutical supply chain constraints and explain how Nusano’s first-of-its-kind radioisotope production platform will deliver high volume, high purity radioisotopes to address existing needs and enable radiopharmaceutical innovation.

WHO:
Nusano Chief Product Officer, Kevin Haehl

WHAT:
Panel Discussion, Isotope Selection, Acquisition, and Production—Challenges and Opportunities

WHEN:
Wednesday, May 14, 2025
11:25 AM Eastern

WHERE:
21st Annual PEGS Summit, Omni Boston Hotel at the Seaport, Boston, MA

On May 12, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, reported that the European Medicines Agency (EMA) has approved its Clinical Trial Application (CTA) to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") (Press release, Moleculin, MAY 12, 2025, View Source [SID1234652886]). The study is for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML) and is approved in all nine countries submitted in the European Union (EU). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East.

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"EMA approval of the MIRACLE trial protocol is a huge milestone for us. Although we’re already seeing recruitment in our first non-EU country, we believe that this expansion into the EU really supercharges our recruitment potential," said Walter Klemp, Chairman and CEO of Moleculin. "Importantly, when combined with the sites we are opening in the US, this approval from the EMA, along with the individual country committee and/or ethics approvals, for Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain positions us to remain on track with our expected enrollment and data milestones."

Mr. Klemp continued: "Being accepted in all nine of the countries for which we submitted, we believe indicates the magnitude of the need for a better answer for R/R AML patients, especially venetoclax regimen failures where the outcomes from currently available therapies are considered dismal in published studies. While there are minor differences between the US and EU protocols with the FDA and EMA, respectively, we do not view these as a barrier to conducting the study and are working to harmonize the protocols as appropriate. We are grateful for the international collaboration and believe it underscores the significant unmet need in R/R AML and the potential of Annamycin to provide a much needed second line treatment option. We remain focused on driving enrollment and patient dosing and look forward to reporting initial data on the first 45 subjects in the second half of 2025."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.

The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) and HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

The clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the study. Results will be submitted as a substantial modification to the existing approved protocol.

For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on clinicaltrials.eu, and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

On May 12, 2025 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported financial results for the first quarter ended March 31, 2025 and highlighted progress on key clinical development programs (Press release, Karyopharm, MAY 12, 2025, View Source,-Symptom-Improvement,-Hemoglobin-Stabilization-and-Disease-Modificati [SID1234652885]).

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"We are pleased that our Phase 3 SENTRY trial in patients with JAKi-naïve myelofibrosis has passed its pre-specified futility analysis and continues as planned without modifications. Our conviction in this trial is further strengthened by our new data in hard-to-treat myelofibrosis patients where we observed spleen volume reduction, symptom improvement, hemoglobin stabilization and evidence of disease modification with selinexor monotherapy, addressing all four hallmarks of the disease," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "The outcomes that we have observed from our multiple pre-clinical and clinical trials continue to suggest that selinexor may be additive, if not synergistic, when combined with ruxolitinib in JAKi-naïve myelofibrosis patients. This combination holds the potential to be a much needed, new standard of care for patients with myelofibrosis."

First Quarter 2025 Highlights

XPOVIO Commercial Performance

Demand for XPOVIO increased 5% in the first quarter of 2025 compared to the first quarter of 2024, with demand growth in both the community and academic settings.
U.S. net product revenue was $21.1 million in the first quarter of 2025 compared to $26.0 million in the first quarter of 2024. Net product revenue in the first quarter of 2025 was impacted by a $5.0 million increase in the product return reserve due to atypical returns of expired 80 mg and 100 mg units from clinics and hospitals that had purchased these units following the 2020 approval by the U.S. Food and Drug Administration of XPOVIO 100 mg in combination with bortezomib and dexamethasone. The majority of XPOVIO that is prescribed today are 40 mg and 60 mg doses, and the Company expects product returns will revert to historical levels in future quarters.
Expanded global patient access for selinexor is translating into growth in royalty revenue from Menarini, Antengene and other international partners. Royalty revenue increased 57% to $1.7 million in the first quarter of 2025 compared to the first quarter of 2024.
Research and Development (R&D) Highlights

Myelofibrosis

The Phase 3 SENTRY trial (XPORT-MF-034; NCT04562389) continues as planned without any modifications following a pre-specified futility analysis conducted by the independent Data Safety and Monitoring Board. The trial is now approximately 80% enrolled. SENTRY is evaluating 60 mg once-weekly selinexor in combination with ruxolitinib compared to ruxolitinib plus placebo and is targeting 350 patients for enrollment.
Data from the XPORT-MF-035 (NCT04562870) Phase 2, randomized, open-label trial of selinexor versus physician’s-choice (PC) in hard-to-treat patients with heavily pretreated myelofibrosis indicated signs of single-agent clinical activity with selinexor, including spleen volume reduction, hemoglobin stabilization, symptom improvement and evidence of disease modification. Patients were randomized 1:1 to either selinexor monotherapy or PC. Selinexor was administered at 80 mg weekly for the first two cycles and then decreased to 60 mg weekly from cycle 3 onwards. An optional crossover was available for PC treated patients if they met predefined spleen progression criteria. In total, 12 patients were randomized to the selinexor arm and 12 patients to the PC arm; 6 patients crossed over from PC to selinexor. Inclusive of crossover, spleen volume reduction of 25% or more (SVR25) at anytime was achieved in 8/12 (67%) efficacy evaluable selinexor treated patients versus 3/8 (38%) efficacy evaluable patients treated with PC. Spleen volume reduction of 35% or more (SVR35) at anytime was observed in 4/12 (33%) efficacy evaluable patients treated with selinexor versus 1/8 (13%) efficacy evaluable patients treated with PC. Patients treated with selinexor had higher mean hemoglobin levels throughout the study duration and lower rates of red blood cell transfusions than those treated with PC. In addition, data on key cytokines that are relevant to myelofibrosis pathogenesis, symptom development, and anemia including IL-6, IL-8, TNFa, and hepcidin, demonstrated greater reductions at week 4 compared to baseline in patients treated with selinexor than patients treated with PC. Most common (≥25% overall) treatment emergent adverse events (TEAEs) in the randomized arms were decreased weight (selinexor: 50%; PC: 50%), anemia (25%; 58%), asthenia (42%; 25%), nausea (33%; 25%), thrombocytopenia (33%; 25%) and upper respiratory tract infection (25%; 33%). Most common (≥15% in any arm) Grade 3+ TEAEs were anemia (17%; 58%), cardiac failure (0%; 17%), decreased appetite (17%; 0%) and nausea (17%; 8%). No treatment discontinuations due to TEAEs were observed in the selinexor arm. In 2023, Karyopharm decided to stop enrollment at 24 patients in this trial to focus resources on the Phase 3 SENTRY trial.
The Company continues to enroll patients into the 60 mg cohort of the Phase 2 SENTRY-2 trial of selinexor monotherapy in JAKi-naïve patients with moderate thrombocytopenia (XPORT-MF-044; NCT05980806).
Endometrial Cancer

Enrollment continues in the Phase 3 XPORT-EC-042 (NCT05611931) trial evaluating selinexor as a maintenance-only therapy following systemic therapy versus placebo in patients with TP53 wild-type advanced or recurrent endometrial cancer. The Company expects to report top-line data from this event-driven trial in mid-2026.
Multiple Myeloma

Enrollment of approximately 120 patients in the Phase 3 XPORT-MM-031 trial (EMN29; NCT05028348) was completed in the fourth quarter of 2024. The trial is being conducted in collaboration with the European Myeloma Network and is evaluating the all-oral combination of selinexor 40 mg, pomalidomide and dexamethasone (SPd40) in patients with previously treated multiple myeloma who received an anti-CD38 in their immediate prior line of therapy. The Company expects to report top-line data from this event-driven trial in the first half of 2026.
Anticipated Catalysts and Operational Objectives in 2025

Myelofibrosis

Announce completion of target enrollment (N=350) of the Phase 3 SENTRY trial evaluating selinexor in combination with ruxolitinib in JAKi-naive myelofibrosis patients in June/July 2025.
Report preliminary data on a subset of participants in the 60 mg cohort from the Phase 2 SENTRY-2 trial evaluating selinexor as a monotherapy in patients with JAKi-naïve myelofibrosis with moderate thrombocytopenia in 1H 2025.
Report top-line results from the Phase 3 SENTRY trial in late 2025/early 2026.
Multiple Myeloma

Maintain the Company’s commercial foundation in the increasingly competitive multiple myeloma marketplace and drive increased XPOVIO revenues.
Continue global launches and regulatory and reimbursement approvals for selinexor by partners in ex-U.S. territories.
Continue to follow patients that are enrolled in the Phase 3 XPORT-MM-031 (EMN29) trial.
Endometrial Cancer

Continue to enroll patients into the Phase 3 XPORT-EC-042 trial of selinexor as a maintenance monotherapy for patients with TP53 wild-type advanced or recurrent endometrial cancer.
2025 Financial Outlook

Based on its current operating plans, Karyopharm expects the following for full year 2025:

Total revenue to be in the range of $140 million to $155 million. Total revenue consists of U.S. XPOVIO net product revenue and license, royalty and milestone revenue earned from partners.
U.S. XPOVIO net product revenue to be in the range of $115 million to $130 million.
R&D and selling, general and administrative (SG&A) expenses to be in the range of $240 million to $255 million, which includes approximately $20 million of estimated non-cash stock-based compensation expense.
The Company expects that its existing cash, cash equivalents and investments, and the revenue it expects to generate from XPOVIO net product sales, as well as revenue generated from its license agreements, will fund its planned operations into early first quarter of 2026.1
1Excluding re-payment of $24.5 million aggregate principal amount of the Company’s remaining senior convertible notes due October 2025 (the 2025 Notes) and $25.0 million minimum liquidity covenant under the Company’s senior secured term loan due 2028. Taking into account the repayment of the 2025 Notes and the minimum liquidity covenant, Karyopharm expects its cash, cash equivalents and investments will fund its operations into early fourth quarter of 2025. The Company is exploring various alternatives to extend its cash runway, which may not result in the consummation of any transaction.

First Quarter 2025 Financial Results

Please note: All share amounts and per share amounts in this press release have been adjusted to reflect a 1-for-15 reverse split of our common stock, which we effected on February 25, 2025.

Total revenue: Total revenue for the first quarter of 2025 was $30.0 million, compared to $33.1 million for the first quarter of 2024.

Net product revenue: Net product revenue for the first quarter of 2025 was $21.1 million, compared to $26.0 million for the first quarter of 2024. The decrease in net product revenue was due to an increase in the gross-to-net provision, primarily related to atypical product returns recorded in the first quarter of 2025, resulting in a $5.0 million increase in the product return reserve.

License and other revenue: License and other revenue for the first quarter of 2025 was $9.0 million, compared to $7.1 million for the first quarter of 2024. The increase was attributable to timing of revenue recognition for the reimbursement of development-related expenses from Menarini.

Cost of sales: Cost of sales for the first quarter of 2025 was $1.3 million, compared to $1.9 million for the first quarter of 2024. Cost of sales reflects the costs of XPOVIO units sold and the costs of products sold to our partners.

R&D expenses: R&D expenses for the first quarter of 2025 were $34.6 million, compared to $35.4 million for the first quarter of 2024. The decrease was due to a reduction in personnel costs, partially offset by increased clinical trial activity related to our myelofibrosis trial.

SG&A expenses: SG&A expenses for the first quarter of 2025 were $27.4 million, compared to $29.5 million for the first quarter of 2024. The decrease was primarily due to the realization of previously implemented cost reduction initiatives.

Interest income: Interest income for the first quarter of 2025 was $1.0 million, compared to $2.2 million for the first quarter of 2024. The decrease in interest income was due to a lower cash and investments balance quarter-over-quarter.

Interest expense: Interest expense for the first quarter of 2025 was $11.0 million, compared to $5.9 million for the first quarter of 2024. The increase was related to the term loan and convertible debt that were issued in the second quarter of 2024.

Other income: Other income for the first quarter of 2025 was $19.8 million due to recurring non-cash fair value remeasurements which related to the refinancing transactions that were completed in the second quarter of 2024. The Company had immaterial other income in the first quarter of 2024.

Net loss: Karyopharm reported a net loss of $23.5 million, or $2.77 per basic and diluted share, for the first quarter of 2025, compared to a net loss of $37.4 million, or $4.85 per basic and diluted share, for the first quarter of 2024. Net loss included non-cash stock-based compensation expense of $3.6 million and $5.0 million for the first quarters of 2025 and 2024, respectively.

Cash position: Cash, cash equivalents, restricted cash and investments as of March 31, 2025 totaled $70.3 million, compared to $109.1 million as of December 31, 2024.

Conference Call Information

Karyopharm will host a conference call today, May 12, 2025, at 4:30 p.m. Eastern Time, to discuss the first quarter 2025 financial results, the financial outlook for 2025 and to provide other business updates. To access the conference call, please dial (800) 836-8184 (local) or (646) 357-8785 (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, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company’s website. 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 exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with VELCADE (bortezomib) and dexamethasone (XVd) in adult patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in adult patients with heavily pre-treated multiple myeloma; and (iii) under accelerated approval in adult patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, Mainland China, Taiwan, Hong Kong, Australia, South Korea, Singapore, Israel, and Canada. XPOVIO/NEXPOVIO is marketed in these respective ex-U.S. territories by Karyopharm’s partners: Antengene, Menarini, Neopharm, and FORUS. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis.

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]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma 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 (Xd).
For the treatment of adult patients with relapsed or refractory diffuse large B‐cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3‐4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
Use In Specific Populations
Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.

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.