On August 7, 2025 Whitehawk Therapeutics, Inc. (Nasdaq: WHWK), an oncology therapeutics company applying advanced technologies to established tumor biology to efficiently deliver improved ADC cancer treatments, reported financial results for the second quarter ended June 30, 2025, and provided recent corporate progress (Press release, Whitehawk Therapeutics, AUG 7, 2025, View Source [SID1234655008]).

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"We’re pleased with the progress made in Q2 to advance our ADC portfolio and remain on track to file INDs for our first two programs – HWK-007 and HWK-016 – by year-end 2025, with the third program, HWK-206, to follow in mid-2026," said Dave Lennon, PhD, President and CEO of Whitehawk Therapeutics. "We believe we are well-positioned to advance our pipeline and generate key clinical data across the portfolio with our existing cash position."

Recent Operational Highlights:

On track to bring all three assets to IND by mid-2026. IND submissions are planned by year-end 2025 for HWK-007 and HWK-016. An IND for HWK-206 is expected by mid-2026.

Focused execution and capital efficiency support anticipated runway into 2028. Based on current plans, cash position enables initial clinical data readouts across the portfolio.
Second Quarter 2025 Financial Results:

Cash, cash equivalents and short-term investments as of June 30, 2025, were $177.2 million as compared to $47.2 million as of December 31, 2024. Cash is anticipated to fund operations into 2028 based on current plans.

Net loss for the three months ended June 30, 2025, was $52.6 million as compared to $14.6 million for the three months ended June 30, 2024. This includes the remaining portion of the upfront payment of $38.0 million under the Wuxi ADC agreement.

On August 7, 2025 Helix, a leader in precision health, reported a partnership with leading cancer diagnostics company Veracyte that will make it easier for urologists to add comprehensive germline testing for patients with high-risk localized and metastatic prostate cancer (Press release, Veracyte, AUG 7, 2025, View Source [SID1234655007]). Through this collaboration, Helix’s whole-exome based hereditary cancer test will be available alongside Veracyte’s Decipher Prostate test, enabling a more complete view of each patient’s cancer biology and inherited risk.

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The partnership was also highlighted during Veracyte’s earnings call, reinforcing the shared commitment to expanding access to clinically actionable genomic insights in prostate cancer care.

The National Comprehensive Cancer Network (NCCN) guidelines recommend germline genetic testing for patients with metastatic and high-risk localized prostate cancer. Germline findings can influence therapeutic decision-making, including the use of targeted therapies like PARP inhibitors. The integration of Helix’s hereditary cancer test into the Decipher ordering workflow gives physicians a convenient way to access this important information and deliver more personalized care.

"Patients diagnosed with cancer deserve seamless access to genomic insights that can improve their care – not just in academic centers, but wherever they’re treated," said James Lu, M.D., Ph.D., CEO and co-founder of Helix. "Through our work with Veracyte, we’re helping expand access to both germline and transcriptomic insights in a single clinical workflow, so physicians can more easily align care decisions with the latest clinical guidelines."

Understanding a patient’s inherited predisposition to cancer offers a number of benefits. It can inform treatment choices, identify risk for additional cancers, and provide actionable information to at-risk family members. When paired with pharmacogenomic (PGx) testing, providers can also better match treatments to each patient’s genetic profile to improve safety and efficacy.

This collaboration is part of Helix’s broader mission to make precision medicine more accessible and impactful for all patients. Helix currently runs the Helix Research Network, the largest and fastest growing precision clinical research network in the world, where several of their partners have begun launching universal cancer screening programs for patients diagnosed with cancer. With its proprietary Exome+ assay and Sequence Once, Query Often model, Helix delivers high-quality genomic insights at scale, enabling ongoing clinical value that may reduce the need for repeat testing.

On August 7, 2025 aTyr Pharma, Inc. (Nasdaq: ATYR) ("aTyr" or the "Company"), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported second quarter 2025 results and provided a corporate update (Press release, aTyr Pharma, AUG 7, 2025, View Source [SID1234655006]).

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"With the recent completion of the last patient visit in our Phase 3 EFZO-FIT study of efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease (ILD), we are on track to report topline data in mid-September," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "This upcoming readout represents a major inflection point for aTyr, our clinical program for efzofitimod in ILD, and the broader sarcoidosis community, and we look forward to sharing the results."

Second Quarter 2025 and Subsequent Period Highlights

Completed the last patient visit in the global pivotal Phase 3 EFZO-FIT study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. Topline data from the study are expected in mid-September 2025. This is a randomized, double-blind, placebo-controlled, 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo administered intravenously monthly for a total of 12 doses. The study enrolled 268 patients with pulmonary sarcoidosis across 85 centers in nine countries. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction measured as the absolute change from baseline to week 48. Secondary endpoints include measures of sarcoidosis symptoms and lung function. Patients who complete the study and wish to receive treatment with efzofitimod outside of the clinical trial are eligible to participate in an Individual Patient Expanded Access Program.

Announced interim data from the ongoing Phase 2 EFZO-CONNECT study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with limited or diffuse systemic sclerosis (SSc, or scleroderma)-related ILD (SSc-ILD). This proof-of-concept study is a randomized, double-blind, placebo-controlled, 28-week study consisting of three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo administered intravenously monthly for a total of six doses. Enrollment in the study is ongoing, and the study intends to enroll up to 25 patients at multiple centers in the United States. The interim analysis evaluated skin assessments and serum biomarkers at baseline and week 12 for efzofitimod and placebo patients. Eight patients were evaluated, including five with diffuse and three with limited SSc-ILD. Key findings for efzofitimod-treated patients to date included:

Stable or improved modified Rodnan Skin Score (mRSS), a key measure of skin fibrosis, for all patients and an improvement of 4 points or greater for three out of four efzofitimod-treated patients with diffuse SSc-ILD, where the minimal clinically important difference (MCID) is a 4 to 6 point improvement at 12 months

Preliminary signals of improvement for inflammatory biomarkers including interferon gamma (IFN-γ) and monocyte chemoattractant protein-1 (MCP-1) and disease biomarkers Krebs von den Lungen-6 (KL-6) and surfactant protein-D (SP-D)

Efzofitimod was generally safe and well tolerated at all doses, with no treatment related serious adverse events

Advanced ATYR0101 to investigational new drug (IND) candidate stage for pulmonary fibrosis. ATYR0101 is a fusion protein derived from a proprietary extracellular domain of aspartyl-tRNA synthetase (DARS) that binds to latent transforming growth factor beta binding protein 1 (LTBP-1) to induce cell death of myofibroblasts, which are key cells responsible for driving the progression of fibrosis. The Company anticipates filing an IND application in the second half of 2026.

Preclinical data generated to date demonstrating ATYR0101’s unique anti-fibrotic mechanism through LTBP-1 were presented in an oral presentation at the American Thoracic Society 2025 Respiratory Innovation Summit

Announced that the Company was added to the Russell 2000 Index and broad market Russell 3000 Index. These additions were a part of the 2025 Russell U.S. Indexes annual reconstitution.

Second Quarter 2025 Financial Highlights and Cash Position

Cash & Investment Position: Cash, cash equivalents, restricted cash and available-for-sale investments as of June 30, 2025, were $83.2 million. Subsequent to the end of the second quarter 2025, the Company raised approximately $30.7 million in gross proceeds from its at-the-market (ATM) offering with Jefferies LLC. The Company believes its cash runway will be sufficient to fund its operations for a period of one year following the Phase 3 EFZO-FIT readout.

R&D Expenses: Research and development expenses were $15.4 million for the second quarter 2025, which consisted primarily of clinical trial costs for the Phase 3 EFZO-FIT and Phase 2 EFZO-CONNECT studies, manufacturing costs for the efzofitimod program and research and development costs for the efzofitimod and discovery programs.

G&A Expenses: General and administrative expenses were $4.9 million for the second quarter 2025.

About Efzofitimod

Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. aTyr is currently investigating efzofitimod in the global Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD, and in the Phase 2 EFZO-CONNECT study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.

On August 7, 2025 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported financial results for the second quarter of 2025, along with recent product highlights and corporate updates (Press release, Zai Laboratory, AUG 7, 2025, View Source [SID1234655002]).

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"Zai Lab is entering a pivotal period – defined by innovation, scale and strong execution," said Dr. Samantha Du, Founder, Chairperson, and CEO of Zai Lab. "We are making meaningful progress throughout our business – expanding patient impact, accelerating global innovation, and operating with financial discipline. Updated ASCO (Free ASCO Whitepaper) data for ZL-1310 (DLL3 ADC) reaffirm its best-in-class potential in second-line SCLC, and we are moving swiftly into pivotal development while exploring opportunities in first-line SCLC and other neuroendocrine carcinomas. We were also encouraged by positive data for bemarituzumab in first-line gastric cancer and by emerging preclinical results for our IL-13/IL-31 bispecific antibody in atopic dermatitis, reinforcing both our near-term commercial opportunities and the potential of our global pipeline, respectively. With multiple launches ahead, a robust pipeline, and profitability1 within reach, Zai Lab is executing on its vision to become a leading global biopharma company."

"VYVGART continues to lead our commercial momentum, with record patient utilization driven by longer treatment durations and growing adoption in the maintenance setting," said Josh Smiley, President and COO of Zai Lab. "The July update to national MG guidelines further strengthens VYVGART’s role in both acute and chronic care, and we expect momentum to accelerate in the second half. We are also preparing for several high-impact launches – including KarXT and bemarituzumab – that, together with pipeline-in-a-product assets like VYVGART and povetacicept, will fuel our next wave of growth. With a 28% year-over-year reduction in operating loss and a 37% improvement on an adjusted basis, we are on track to achieve profitability1 in the fourth quarter. Backed by a strong cash position2, a growing commercial business, and an advancing global pipeline, Zai Lab is well equipped to deliver long-term shareholder value."

Second Quarter 2025 Financial Results

•Product revenue, net was $109.1 million in the second quarter of 2025, compared to $100.1 million for the same period in 2024, representing 9% y-o-y growth, 10% y-o-y growth at constant exchange rate (CER). This increase was primarily driven by increased sales for VYVGART, XACDURO, and NUZYRA, partially offset by softer sales for ZEJULA:

–VYVGART and VYVGART Hytrulo were $26.5 million in the second quarter of 2025, compared to $18.1 million in the first quarter of 2025. Sales grew 46% quarter over quarter driven by an extension of duration of therapy and increasing market penetration.

–ZEJULA was $41.0 million in the second quarter of 2025, compared to $45.0 million for the same period in 2024. Sales were softer due to evolving competitive dynamics within the PARPi class.

–XACDURO, which was launched since the fourth quarter of 2024, was $4.6 million in the second quarter of 2025.

–NUZYRA was $14.3 million in the second quarter of 2025, compared to $12.3 million for the same period in 2024. This growth was supported by increasing market coverage and penetration.

•Research and Development (R&D) expenses were $50.6 million in the second quarter of 2025, compared to $61.6 million for the same period in 2024. The decrease reflects reduced personnel and clinical trial costs, driven by ongoing resource prioritization and efficiency efforts.

•Selling, General and Administrative expenses were $71.0 million in the second quarter of 2025, compared to $79.7 million for the same period in 2024. The decrease was primarily driven by reduced personnel costs because of resource prioritization and efficiency efforts.

•Loss from operations was $54.9 million in the second quarter of 2025, $34.2 million when adjusted to exclude certain non-cash expenses including depreciation, amortization, and share-based compensation. A reconciliation of loss from operations (GAAP) to adjusted loss from operations (non-GAAP) is included at the end of this release.

•Net loss was $40.7 million in the second quarter of 2025, or a loss per ordinary share attributable to common stockholders of $0.04 (or loss per American Depositary Share (ADS) of $0.37), compared to a net loss of $80.3 million for the same period in 2024, or a loss per ordinary share of $0.08 (or loss per ADS of $0.82). These decreases in net loss were primarily due to product revenue growing faster than net operating expenses.

•Cash and cash equivalents, short-term investments, and current restricted cash totaled $832.3 million as of June 30, 2025, compared to $857.3 million as of March 31, 2025.

Recent Pipeline Highlights

Below are key product updates since our last earnings release:

Oncology Pipeline

•ZL-1310 (DLL3 ADC):

–In June 2025, Zai Lab presented positive data from an ongoing, global Phase 1a/1b clinical trial of ZL-1310 for the treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. In second-line (2L) SCLC, objective response rate (ORR) was 67% across all dose levels (n=33) and 79% at 1.6 mg/kg dose (n=14). ZL-1310 demonstrated a well-tolerated safety profile at target doses of less than 2.0 mg/kg, with Grade ≥3 treatment-related adverse events (TRAEs) of 6%, no Grade ≥2 interstitial lung disease, and no drug discontinuations.

–In May 2025, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to ZL-1310 for treatment of ES-SCLC.

•Bemarituzumab (FGFR2b): In June 2025, Zai Lab announced the Phase 3 FORTITUDE-101 clinical trial evaluating first-line bemarituzumab plus chemotherapy (mFOLFOX6) met its primary endpoint of overall survival (OS) at a pre-specified interim analysis. At the interim analysis, bemarituzumab plus chemotherapy significantly improved OS in patients with FGFR2b overexpression compared to chemotherapy alone. The most common treatment-emergent adverse events (>25%) in patients treated with bemarituzumab plus chemotherapy were reduced visual acuity, punctate keratitis, anemia, neutropenia, nausea, corneal epithelium defect and dry eye. While ocular events were consistent with the Phase 2 experience and observed in both arms, they occurred with greater frequency and severity in the Phase 3 bemarituzumab arm. Detailed results from FORTITUDE 101 will be shared at a future medical meeting. Zai Lab plans to move rapidly toward regulatory submission in China in the second half of 2025.

•Tumor Treating Fields (TTFields): In May 2025, Zai Lab partner Novocure presented results from the Phase 3 PANOVA-3 trial for pancreatic cancer as a late-breaking oral presentation at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting. The data demonstrated that TTFields therapy, when added to standard of care, achieved an OS benefit supported by significantly improved quality of life and extended pain-free survival, a key outcome for patients with pancreatic cancer. Zai Lab participated in the study in Greater China (mainland China, Hong Kong, Macau and Taiwan, collectively) and plans to file for regulatory approval in China in the second half of 2025.

Immunology Pipeline

•Efgartigimod (FcRn): In July 2025, the China Guidelines for the Diagnosis and Treatment of Myasthenia Gravis (MG) (2025) was published, emphasizing the importance of Minimal Symptom Expression (MSE) as the primary treatment goal in MG. The guidelines have highlighted VYVGART’s ability to achieve MSE rapidly and provide durable benefit. VYVGART is now recommended for early use in mild-to-moderate and highly active patients and for sustained long-term treatment to maximize potential benefit.

•ZL-1503 (IL-13/IL-31R): In June 2025, Zai Lab announced new preclinical data highlighting the potential of ZL-1503 as a promising treatment for moderate-to-severe atopic dermatitis and other IL-13 and IL-31-driven diseases. Its favorable preclinical safety profile, prolonged half-life and durable suppression of both inflammatory and pruritogenic pathways support the continued advancement of ZL-1503 toward clinical development.

Anticipated Major Milestones in 2025 and the First Half of 2026

Upcoming Potential NMPA Submissions

•Bemarituzumab (FGFR2b) in first-line gastric cancer in the second half of 2025

•Tumor Treating Fields (TTFields) in first-line pancreatic cancer in the second half of 2025

•Efgartigimod (FcRn) for prefilled syringe in generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP) in the second half of 2025

Upcoming Potential NMPA Approvals

•Xanomeline-Trospium (or KarXT) (M1/M4-agonist) in schizophrenia

•Tisotumab Vedotin (Tissue Factor ADC) in recurrent or metastatic cervical cancer following progression on or after chemotherapy

•Repotrectinib (ROS1/TRK) in NTRK+ solid tumors

Expected Clinical Developments and Data Readouts

Global Pipeline

ZL-1310 or Zocilurtatug pelitecan (DLL3 ADC)

•Second-Line ES-SCLC: Zai Lab to provide data update and to initiate a global registrational study of ZL-1310 monotherapy in the second half of 2025.

•First-Line ES-SCLC: Zai Lab to provide data readout for dose escalation of ZL-1310 doublet in combination with atezolizumab.

•Other neuroendocrine carcinomas: Zai Lab to provide data readout from the global Phase 1/2 study in patients with selected solid tumors.

ZL-1503 (IL-13/IL-31R)

•Zai Lab to advance into a global Phase 1 study in moderate-to-severe atopic dermatitis in the second half of 2025.

ZL-6201 (LRRC15 ADC)

•Zai Lab to advance into global Phase 1 development for patients with sarcoma and potentially other LRRC15-positive solid tumors, such as breast cancer and other malignancies.

Regional Pipeline

Bemarituzumab (FGFR2b)

•Zai Lab partner Amgen to provide detailed results from the Phase 3 FORTITUDE-101 study of bemarituzumab combined with chemotherapy versus chemotherapy alone in first-line gastric cancer at an upcoming medical meeting. Zai Lab participated in the study in Greater China.

•Zai Lab partner Amgen to provide potential data readout from the Phase 1b/3 FORTITUDE-102 study of bemarituzumab plus chemotherapy and nivolumab versus chemotherapy and nivolumab in first-line gastric cancer. Zai Lab participated in the study in Greater China.

Xanomeline-Trospium (or KarXT) (M1/M4-agonist)

•Zai Lab partner Bristol Myers Squibb to provide data readout from the Phase 3 ADEPT-2 study of KarXT in Alzheimer’s Disease Psychosis in the second half of 2025. Zai Lab participated in the study in Greater China.

Efgartigimod (FcRn)

•Lupus Nephritis (LN): Zai Lab and partner argenx to provide topline results from the Phase 2 study in LN in the fourth quarter of 2025.

•Seronegative gMG: Zai Lab partner argenx to provide topline results from the global Phase 3 ADAPT-SERON study in seronegative gMG in the second half of 2025. Zai Lab participated in the study in Greater China.

•Ocular myasthenia gravis: Zai Lab partner argenx to provide topline results from the global Phase 3 ADAPT-OCULUS study in the first half of 2026. Zai Lab participated in the study in Greater China.

•Sjogren’s disease: Zai Lab to join the registrational UNITY study of efgartigimod subcutaneous given by prefilled syringe in Sjogren’s disease in Greater China in the third quarter of 2025.

Povetacicept (APRIL/BAFF)

•Primary Membranous Nephropathy (pMN): Zai Lab plans to partner with Vertex to execute the global pivotal Phase 2/3 study of povetacicept in pMN in Greater China. The study is expected to start in the second half of 2025.

•IgA Nephropathy (IgAN): Zai Lab partner Vertex will conduct an interim analysis of the global Phase 3 RAINIER study following 36 weeks of treatment with the potential to file for U.S. accelerated approval in the first half of 2026.

VRDN-003 (IGF-1R, subcutaneous)

•Zai Lab to initiate a registrational study in thyroid eye disease in Greater China in the second half of 2025.

•Zai Lab partner Viridian to provide topline results from the global registrational REVEAL-1 and REVEAL-2 studies in the first half of 2026.

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast today, August 7, 2025, at 8:00 a.m. ET (8:00 p.m. HKT). Listeners may access the live webcast by visiting the Company’s website at View Source Participants must register in advance of the conference call.

Details of registration links are as follows:

•For webcast: View Source

•For dial-in: View Source

All participants must use the link provided above to complete the online registration process in advance of the conference call. Dial-in details will be in the confirmation email which the participant will receive upon registering.

A replay will be available shortly after the call and can be accessed by visiting the Company’s website.

On August 7, 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 second quarter ended June 30, 2025, and provided an overview of recent developments (Press release, UroGen Pharma, AUG 7, 2025, View Source [SID1234655001]).

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"The recent FDA approval of ZUSDURI for the treatment of adults with recurrent LG-IR-NMIBC represents a truly transformative milestone for patients and for UroGen, marking our evolution into a multi-product uro-oncology company and our leadership in the field," said Liz Barrett, President and Chief Executive Officer of UroGen. "Our commercial team is enthusiastically executing the ZUSDURI launch plan as we scale the organization to address the estimated $5 billion+ market opportunity. JELMYTO (mitomycin) for pyelocalyceal solution, our treatment for low-grade upper tract urothelial carcinoma (LG-UTUC), continues to grow with strong underlying demand in the second quarter reflecting continued interest in this important therapy for patients. We are equally excited about the continued progress across our pipeline, including our next-generation mitomycin formulations and immuno-oncology candidates. With a solid balance sheet, we are well positioned to fully support the launch of ZUSDURI while advancing strategic business initiatives to drive sustained growth and innovation."

Q2 2025 and Recent Business Highlights:

ZUSDURI (mitomycin) for intravesical solution:

On June 12, 2025, the U.S. Food and Drug Administration (FDA) approved ZUSDURI (formerly UGN-102), the first and only FDA-approved medication for adults with recurrent LG-IR-NMIBC.
Updated Duration of Response (DOR) data from the Phase 3 ENVISION trial evaluating ZUSDURI in patients with recurrent LG-IR-NMIBC demonstrate a probability of remaining in complete response (CR) of 72.2% (95% CI: 64.1%, 78.8%) by Kaplan-Meier estimate, at 24-months after achieving a CR at three-months. Median follow-up time at 24-months was 23.7 months after the three-month CR.
Five-year results from the long-term extension study of the Phase 2b OPTIMA II trial evaluating ZUSDURI in patients with newly diagnosed or recurrent LG-IR-NMIBC were published in the Journal of Clinical Genitourinary Cancer. Among the 17 patients who achieved a CR and entered the long-term extension study, the median DOR was approximately 3.5 years by Kaplan-Meier estimate. A copy of the paper can be found here.
JELMYTO (mitomycin) for pyelocalyceal solution in LG-UTUC:

Generated net product revenue of $24.2 million in the quarter ended June 30, 2025, an increase of 11% compared to the same quarter in 2024, driven by underlying demand growth of 7% and year-over-year price favorability.
JELMYTO is being evaluated in the uTRACT Registry, details of which were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June 2025. This single-arm, multicenter, prospective and retrospective study is looking at real-world use of JELMYTO for adult patients with UTUC across the United States. Comprehensive data over a three-year period will be collected, with the goal of providing insights into treatment patterns, long-term outcomes, and patient safety in real-world settings.
Next-generation novel mitomycin-based formulations for urothelial cancers

Enrollment is now complete in the ongoing Phase 3 UTOPIA clinical trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with recurrent LG-IR-NMIBC. UGN-103 is a next-generation product designed to offer improvements over ZUSDURI (mitomycin) for intravesical solution, including a shorter manufacturing process and simplified reconstitution procedure. It combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH. To learn more about the UTOPIA trial, refer to clinicaltrials.gov/NCT06331299.
A Phase 3 study to explore the safety and efficacy of UGN-104 has been initiated. UGN-104 is a next generation product for LG-UTUC. To learn more about this trial, refer to View Source
UGN-301 (zalifrelimab), an anti-CTLA4 antibody for use in high-grade non-muscle invasive bladder cancer

Enrollment is complete in the dose escalation phase of UGN-301, alone and in combination with fixed dose UGN-201 or gemcitabine. The 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.
Second Quarter 2025 Financial Results

JELMYTO Revenue: JELMYTO net product revenues were $24.2 million for the three months ended June 30, 2025, compared with $21.8 million for the same quarter of 2024. Year-over-year revenue growth of 11% was driven by underlying demand growth of 7% and price favorability.

Research & Development Expenses (R&D): R&D expenses for the second quarter of 2025 were $18.9 million, including non-cash share-based compensation expense of $0.4 million as compared to $15.4 million, including non-cash share-based compensation expense of $0.6 million, for the same period in 2024. The increase in R&D expenses of $3.5 million was primarily driven by 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 ZUSDURI.

Selling, General and Administrative Expenses (SG&A): SG&A expenses for the second quarter of 2025 were $43.2 million, including non-cash share-based compensation expense of $2.3 million. This compares to $30.1 million, including non-cash share-based compensation expense of $3.0 million, for the same period in 2024. The increase in SG&A expenses of $13.1 million was primarily driven by ZUSDURI commercial preparation activities as well as an increase in overall commercial operation costs.

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 second quarter of 2025, compared to $5.8 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 second quarter of 2025, compared to $3.5 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 $49.9 million or ($1.05) per basic and diluted share in the second quarter of 2025 compared with a net loss of $33.4 million or ($0.82) per basic and diluted share in the same period in 2024.

Cash and Equivalents: As of June 30, 2025, cash, cash equivalents and marketable securities totaled $161.6 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.

UROGEN PHARMA LTD.
SELECTED CONSOLIDATED BALANCE SHEETS
(U.S. dollars in thousands)
(Unaudited)


June 30, 2025 December 31, 2024
Cash and cash equivalents and marketable securities $ 161,586 $ 241,707
Total assets $ 208,717 $ 285,711
Total liabilities $ 302,093 $ 294,514
Total shareholders’ deficit $ (93,376 ) $ (8,803 )

UROGEN PHARMA LTD.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(U.S. dollars in thousands, except share and per share data)
(Unaudited)

Three months ended June 30, Six months ended June 30,
2025 2024 2025 2024

Revenue $ 24,215 $ 21,848 $ 44,469 $ 40,629
Cost of revenue 3,550 2,229 5,880 3,957
Gross profit 20,665 19,619 38,589 36,672
Operating expenses:
Research and development expenses 18,914 15,402 38,785 30,896
Selling, general and administrative expenses 43,199 30,056 78,166 57,355
Total operating expenses 62,113 45,458 116,951 88,251
Operating loss (41,448 ) (25,839 ) (78,362 ) (51,579 )
Financing on prepaid forward obligation (4,644 ) (5,773 ) (9,227 ) (11,433 )
Interest expense on long-term debt (4,132 ) (3,461 ) (8,200 ) (5,908 )
Interest and other income, net 1,299 1,708 3,413 3,323
Loss before income taxes $ (48,925 ) $ (33,365 ) $ (92,376 ) $ (65,597 )
Income tax expense (1,015 ) (38 ) (1,407 ) (92 )
Net loss $ (49,940 ) $ (33,403 ) $ (93,783 ) $ (65,689 )
Net loss per ordinary share basic and diluted $ (1.05 ) $ (0.82 ) $ (1.97 ) $ (1.69 )
Weighted average shares outstanding, basic and diluted 47,739,816 40,501,315 47,582,610 38,785,924

About ZUSDURI

ZUSDURI (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, approved for the treatment of adults with recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology (a sustained release, hydrogel-based formulation) ZUSDURI is delivered directly into the bladder by a trained healthcare professional using a urinary catheter in an outpatient setting, thereby enabling the treatment of tumors by non-surgical means.

APPROVED USE FOR ZUSDURI

ZUSDURI (mitomycin) for intravesical solution is a prescription medicine used to treat adults with a type of cancer of the lining of the bladder called low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC) after previously receiving bladder surgery to remove tumor that did not work or is no longer working.

IMPORTANT SAFETY INFORMATION

You should not receive ZUSDURI if you have a hole or tear (perforation) of your bladder or if you have had an allergic reaction to mitomycin or to any of the ingredients in ZUSDURI.

Before receiving ZUSDURI, tell your healthcare provider about all of your medical conditions, including if you:

have kidney problems
are pregnant or plan to become pregnant. ZUSDURI can harm your unborn baby. You should not become pregnant during treatment with ZUSDURI. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with ZUSDURI.

Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with ZUSDURI and for 6 months after the last dose.

Males being treated with ZUSDURI: You should use effective birth control (contraception) during treatment with ZUSDURI and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if ZUSDURI passes into your breast milk. Do not breastfeed during treatment with ZUSDURI and for 1 week after the last dose.
How will I receive ZUSDURI?

You will receive your ZUSDURI dose from your healthcare provider 1 time a week for 6 weeks into your bladder through a tube called a urinary catheter. It is important that you receive all 6 doses of ZUSDURI according to your healthcare provider’s instructions.
If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment.
During treatment with ZUSDURI, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving ZUSDURI:

ZUSDURI may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 24 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
The most common side effects of ZUSDURI include: increased blood creatinine levels, increased blood potassium levels, trouble with urination, decreased red blood cell counts, increase in certain blood liver tests, increased or decreased white blood cell counts, urinary tract infection, and blood in your urine.

You are encouraged to report negative side effects of prescription drugs to the FDA.

Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see ZUSDURI Full Prescribing Information, including the Patient Information, for additional information.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for the treatment of adult patients with LG-UTUC. It is recommended for primary treatment of biopsy-proven LG-UTUC in patients deemed appropriate candidates for renal-sparing therapy. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

APPROVED USE FOR JELMYTO
JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO. Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose. Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.