On November 6, 2025 Cidara Therapeutics, Inc. (Nasdaq: CDTX) (the Company or Cidara), a biotechnology company using its proprietary Cloudbreak platform to develop drug-Fc conjugate (DFC) therapeutics, reported financial results for the third quarter ended September 30, 2025, and provided recent business updates.

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"With our Phase 3 ANCHOR study now over 50 percent enrolled, we expect to achieve target enrollment of 6,000 participants by December 2025 and thereby advance CD388 as a potential universal preventative for people at increased risk of complications from influenza as well as those seeking alternatives to flu vaccines," said Jeffrey Stein, Ph.D., president and chief executive officer of Cidara. "Based on constructive feedback from the FDA, the ANCHOR study population has been expanded to include generally healthy adults over the age of 65 in addition to individuals with certain comorbidities or compromised immune status. This change more than doubles the target population potentially eligible to receive CD388. Our successful financing this summer has provided us with a strong balance sheet that we expect to be sufficient to fully fund our Phase 3 development program through completion."

Recent and Expected Corporate Highlights

Expanded and accelerated Phase 3 plan for CD388 in influenza. Cidara is proceeding with an expanded and accelerated development plan to seek biologics license application (BLA) approval based on a single Phase 3 study, following an End-of-Phase 2 (EOP2) meeting with the United States (U.S.) Food and Drug Administration (FDA). The Phase 3 study is evaluating the safety and efficacy of CD388 in populations at high-risk for complications from influenza (ANCHOR study). Based on FDA feedback, the ANCHOR study population was expanded to include adults over 65 years of age with no specific comorbidities in addition to subjects over 12 years of age with high-risk comorbidities or immune-compromised status. This increases the initial number of patients that would be potentially eligible to receive CD388 from approximately 50 million to well over 100 million people in the U.S.
Enrolled and dosed the first patients in the Phase 3 ANCHOR study. The ANCHOR study has a target enrollment of 6,000 participants. Cidara dosed the first patients in the U.S. at the end of September 2025 and enrollment is ongoing in 150 sites in the Northern Hemisphere across the U.S. and the United Kingdom (UK). The ANCHOR study will include an interim analysis in the first quarter of 2026 to assess the trial size and powering assumptions and determine the potential need for additional enrollment during the subsequent Southern Hemisphere flu season.
BARDA award for CD388. The award from Biomedical Advanced Research and Development Authority (BARDA) is valued at up to $339.2 million in total. The base period funding of $58.1 million over the initial 24 months will support the onshoring of CD388 manufacturing to the U.S. as an addition to the initial commercial supply chain. This project is being supported in whole or in part with federal funds from the U.S. Department of Health and Human Services; Administration for Strategic Preparedness and Response; BARDA, under contract number 75A50125C0017.
Breakthrough Therapy designation of CD388. The Breakthrough Therapy designation is based on positive results from the Phase 2b study (NAVIGATE study) which showed that CD388 was well-tolerated and that all primary and secondary endpoints were met in connection with preventing seasonal influenza in healthy unvaccinated adults aged 18-64. This designation comes in addition to the previously awarded Fast Track designation and is intended to expedite the review of medicines that treat a serious or life-threatening condition and have shown preliminary clinical evidence indicating the potential for substantial improvement over available therapies.
Highlighted CD388 in presentations at various medical conferences. During September 2025 and October 2025, Cidara gave presentations at various medical conferences including the International Society for Respiratory Viruses 8th Antiviral Group Meeting and 3rd International Meeting on Respiratory Pathogens in Singapore, ID Week 2025 in Atlanta, Georgia, and the European Scientific Working Group on Influenza’s 10th Influenza Conference in Valencia, Spain. These oral presentations included late-breaking Phase 2b clinical data from Cidara’s successful NAVIGATE study that showed CD388 to be well-tolerated with all primary and secondary endpoints met in connection with preventing influenza illness in healthy adults.
Third Quarter 2025 Financial Results

Cash, cash equivalents, restricted cash and available-for-sale investments totaled $476.5 million as of September 30, 2025, compared with $196.2 million as of December 31, 2024.
Collaboration revenue was zero for each of the three and nine months ended September 30, 2025, compared to zero and $1.3 million for the same periods in 2024, respectively. Collaboration revenue related to research and development (R&D) and clinical supply services provided to J&J Innovative Medicine, previously Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), under our license and collaboration agreement with Janssen (the Janssen Collaboration Agreement), which was terminated upon the effectiveness of our license and technology transfer agreement with Janssen (the Janssen License Agreement) on April 24, 2024.
Acquired in-process research and development (IPR&D) expenses were $45.0 million for each of the three and nine months ended September 30, 2025, compared to zero and $84.9 million for the same periods in 2024, respectively. Acquired IPR&D in 2025 related to a $45.0 million milestone incurred under the Janssen License Agreement, upon dosing the first five subjects in our ANCHOR study, which will be paid to Janssen in the fourth quarter of 2025. Acquired IPR&D in 2024 related to an upfront payment of $85.0 million paid to Janssen under the Janssen License Agreement on April 24, 2024, in connection with the re-acquisition of CD388, plus $0.4 million in direct transaction costs, offset by a gain of $0.5 million to settle the preexisting Janssen Collaboration Agreement relationship.
R&D expenses were $35.5 million and $84.9 million for the three and nine months ended September 30, 2025, respectively, compared to $12.4 million and $25.0 million for the same periods in 2024, respectively. The increase in R&D expenses for the three months ended September 30, 2025 is primarily due to higher expenses associated with CD388 manufacturing related costs and the preparation and initiation of our ANCHOR study. The increase in R&D expenses for the nine months ended September 30, 2025 is primarily due to higher expenses associated with our NAVIGATE study, CD388 manufacturing related costs, and the preparation and initiation of our ANCHOR study, offset by lower nonclinical expenses associated with our Cloudbreak platform.
General and administrative (G&A) expenses were $8.1 million and $20.8 million for the three and nine months ended September 30, 2025, respectively, compared to $5.0 million and $13.3 million for the same periods in 2024, respectively. The increase in G&A expenses is primarily due to higher personnel costs, driven by higher stock-based compensation, offset by lower audit fees.
During the nine months ended September 30, 2025, the Company determined that accrued indirect taxes relating to shipments of our former rezafungin assets totaling $9.4 million were not due and payable upon voluntary disclosure and full compliance in certain jurisdictions and the associated liabilities and operating expenses were reversed as part of continuing operations. No indirect tax reversals were recorded during the three months ended September 30, 2025, or during the three and nine months ended September 30, 2024.
Other income, net was $5.4 million and $8.9 million for the three and nine months ended September 30, 2025, respectively, compared to $1.9 million and $4.0 million for the same periods in 2024, respectively. Other income, net related primarily to interest income generated from cash held in interest-bearing accounts and available-for-sale investments.
Income/loss from discontinued operations for each of the three and nine months ended September 30, 2025 was zero, compared to a loss of $0.5 million and income of $0.4 million for the same periods in 2024, respectively. On April 24, 2024, the Company entered into an asset purchase agreement with Napp Pharmaceutical Group Limited (Napp), an affiliate of Mundipharma Medical Company, pursuant to which all rezafungin assets and related contracts were sold to Napp. All conditions of the sale were completed on April 24, 2024, and the financial results of rezafungin have been reported separately as discontinued operations.
Net loss for the three and nine months ended September 30, 2025 was $83.2 million and $132.4 million, respectively, compared to a net loss of $16.0 million and $117.5 million for the same periods in 2024, respectively.
Third Quarter 2025 Conference Call and Webcast Details

Cidara Therapeutics management will host a conference call and webcast beginning at 5:00 pm ET / 2:00 pm PT today, November 6, 2025. A live webcast may be accessed here. The conference call can be accessed by dialing toll-free 1-844-825-9789 or 1-412-317-5180 (international). The passcode for the conference call is 10203589.

A replay of the webcast will be archived on www.cidara.com for one year under the "Events & Presentations" tab in the Investors section of the company’s website.

(Press release, Cidara Therapeutics, NOV 6, 2025, View Source [SID1234659604])

On November 6, 2025 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported financial results for the third quarter of 2025, along with recent product highlights and corporate updates.

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"Zai Lab is entering the next phase of our growth, powered by the rapid advancement of our global pipeline and supported by a commercially profitable and scalable business in China," said Dr. Samantha Du, Founder, Chairperson, and CEO of Zai Lab. "With zoci moving into pivotal development less than two years after IND and multiple differentiated global programs progressing in parallel, we are demonstrating the speed, scientific rigor, and global ambition of our R&D engine. At the same time, our commercial platform in China remains strong, with new products and indications – including KarXT, povetacicept and VYVGART – broadening our long-term growth profile. Together, we are building a company that will make a lasting difference for patients and create long-term value for our shareholders."

"This quarter, we continued to deepen the foundations of the VYVGART launch," said Josh Smiley, President and COO of Zai Lab. "In gMG, we are seeing steady new patient starts and increasing treatment duration, supported by updated treatment guidelines and real-world experience. While adoption is building gradually, physician confidence continues to grow and reinforces the long-term potential of VYVGART as a new standard of care in the treatment of this chronic disease. Looking ahead, we are preparing for our next expected launch for KarXT in schizophrenia. We remain focused on disciplined execution as we position for the significant opportunities ahead, supported by a growing regional business and a rapidly progressing, global pipeline."

Recent Pipeline Highlights

Below are key product updates since our last earnings release:

Oncology Pipeline

•Zocilurtatug Pelitecan (zoci, DLL3 ADC) (formerly ZL-1310):

–In October 2025, Zai Lab initiated a global registrational study of zocilurtatug pelitecan monotherapy in second-line+ extensive-stage small cell lung cancer (ES-SCLC).

–In October 2025, Zai Lab presented updated Phase 1 results at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference, demonstrating a best overall response rate of 68% at the 1.6 mg/kg dose in second-line extensive-stage small cell lung cancer. The estimated median duration of response (DoR) of 6.1 months across all doses and all lines of therapy is highly encouraging for a monotherapy in this heavily pre-treated and difficult-to-treat patient population. Meaningful activity in patients with brain metastases was also observed, including an 80% response rate in patients with untreated brain metastases. The data also demonstrated a well-tolerated safety profile at 1.6 mg/kg, with Grade ≥ 3 treatment-related adverse events of 13%, no Grade ≥2 interstitial lung disease, and no drug discontinuations. Together, these results reinforce zoci’s best-in-class potential and support the initiation of the global registrational study.

•Tumor Treating Fields (TTFields): In August 2025, Zai Lab announced that the China National Medical Products Administration (NMPA) has granted Innovative Medical Device Designation for TTFields for patients with pancreatic cancer based on the positive results from the Phase 3 PANOVA-3 trial. We plan to submit for regulatory approval in China in the fourth quarter of 2025.

•Bemarituzumab (FGFR2b): In November 2025, Zai Lab partner Amgen announced FORTITUDE-102, a Phase 1b/3 study of bemarituzumab plus chemotherapy and nivolumab in patients with first-line gastric cancer, was stopped.

Immunology Pipeline

•ZL-1503 (IL-13/IL-31R): In November 2025, Zai Lab initiated a global Phase 1/1b study to evaluate safety, tolerability and pharmacokinetics of ZL-1503 in healthy volunteers and participants with moderate to severe atopic dermatitis.

•Efgartigimod (FcRn):

–Sjogren’s disease: In September 2025, Zai Lab joined the registrational UNITY study of efgartigimod subcutaneous given by prefilled syringe in Sjogren’s disease in Greater China (mainland China, Hong Kong, Macau and Taiwan, collectively).

–Seronegative gMG: In August 2025, Zai Lab partner argenx announced topline data from the pivotal ADAPT SERON study of VYVGART in patients with AChR-Ab seronegative gMG. The study met its primary endpoint (p-value=0.0068) and it is the first global phase 3 study to demonstrate clinically meaningful improvements in disease activity across all three subtypes – MuSK+, LRP4+, triple seronegative. Zai Lab participated in the global Phase 3 study in Greater China, enabling a potential China regulatory submission.

•Xanomeline-Trospium (or KarXT) (M1/M4-agonist): In September 2025, the "China Schizophrenia Prevention and Treatment Guidelines (2025 Edition)" were officially released, and KarXT was included for the first time, marking the first national-level guideline globally to include KarXT. The guidelines emphasize KarXT’s broad efficacy across all three symptom domains (positive, negative, and cognitive symptoms) and its unique safety profile, supporting long-term adherence and functional recovery. China’s NMPA accepted the New Drug Application (NDA) for the treatment of schizophrenia in January 2025.

•Povetacicept: In September 2025, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to povetacicept for the treatment of IgA nephropathy (IgAN). Subsequently, the FDA also granted a rolling review of the Biologics License Application (BLA) submission for povetacicept for this indication. Vertex has completed full enrollment of the Phase 3 study, including the interim analysis cohort for potential accelerated approval in the U.S. Zai Lab participated in the global Phase 3 RAINIER study in patients with IgAN in Greater China.

Third Quarter 2025 Financial Results

•Product revenue, net was $115.4 million in the third quarter of 2025, compared to $101.8 million for the same period in 2024, representing 13% y-o-y growth, 14% y-o-y growth at constant exchange rate (CER). This increase was primarily driven by increased sales for NUZYRA and XACDURO, partially offset by softer sales for ZEJULA:

–VYVGART and VYVGART Hytrulo were $27.7 million in the third quarter of 2025 which includes a $2.4 million reduction following a voluntary price adjustment on Hytrulo ahead of National Reimbursement Drug List (NRDL) negotiation, compared to $26.5 million in the second quarter of 2025. Sales grew 4.6% quarter over quarter driven by an extension of duration of therapy and increased market penetration.

–ZEJULA was $42.4 million in the third quarter of 2025, compared to $48.2 million for the same period in 2024. Sales were softer due to evolving competitive dynamics within the PARPi class.

–XACDURO, which was launched in the fourth quarter of 2024, was $6.4 million in the third quarter of 2025.

–NUZYRA was $15.4 million in the third quarter of 2025, compared to $10.0 million for the same period in 2024. This growth was supported by increased market coverage and penetration.

•Research and Development (R&D) expenses were $47.9 million in the third quarter of 2025, compared to $66.0 million for the same period in 2024, primarily due to a decrease in licensing fees in connection with upfront and milestone payments.

•Selling, General and Administrative expenses were $70.1 million in the third quarter of 2025, compared to $67.2 million for the same period in 2024. This increase was primarily driven by higher general selling expenses to support the growth of NUZYRA and VYVGART, partially offset by decreases in selling expenses related to ZEJULA.

•Loss from operations was $48.8 million in the third quarter of 2025, $28.0 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 $36.0 million in the third quarter of 2025, or a loss per ordinary share attributable to stockholders of $0.03 (or loss per American Depositary Share (ADS) of $0.33), compared to a net loss of $41.7 million for the same period in 2024, or a loss per ordinary share of $0.04 (or loss per ADS of $0.42). These decreases in net loss were primarily due to increased product revenue and decreased operating expenses.

•Cash and cash equivalents, short-term investments, and current restricted cash totaled $817.2 million as of September 30, 2025, compared to $832.3 million as of June 30, 2025.

Anticipated Major Milestones in the Fourth Quarter of 2025 and Full Year 2026

Expected Clinical Developments and Data Readouts

Global Pipeline

Zocilurtatug Pelitecan (zoci, DLL3 ADC) (formerly ZL-1310)

•Second-Line+ ES-SCLC: Zai Lab to present updated data on intracranial activity from the ongoing Phase 1 study in the first half of 2026.

•First-Line ES-SCLC: Zai Lab to provide data readout from the Phase 1 study evaluating zoci combination therapy (with atezolizumab and/or chemotherapy) in the first half of 2026 and advance into a registrational study in 2026 based on emerging data. Zai Lab also plans to initiate a Phase 1 study to explore zoci in a novel combination in 2026.

•Other neuroendocrine carcinomas: Zai Lab to provide data readout from the global Phase 1/2 study in patients with selected solid tumors in the first half of 2026 and advance into a registrational-enabling cohort in 2026.

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

•Zai Lab to provide the initial data readout from the global Phase 1/1b study in healthy volunteers and participants with moderate to severe atopic dermatitis in 2026.

ZL-6201 (LRRC15 ADC)

•Zai Lab to submit an Investigational New Drug application to the FDA for a global Phase 1 study for patients with sarcoma and potentially other LRRC15-positive solid tumors in the fourth quarter of 2025.

Upcoming Potential NMPA Submissions

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

•Efgartigimod (FcRn) for prefilled syringe in generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP) in the fourth quarter 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

Regional Pipeline

Efgartigimod (FcRn)

•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.

•Myositis: Zai Lab partner argenx to provide topline results from the global Phase 2/3 ALKIVIA study evaluating three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS) and dermatomyositis (DM)) in the second half of 2026. Zai Lab participated in the study in Greater China.

•Thyroid eye disease (TED): Zai Lab partner argenx to provide topline results from two registrational UplighTED studies in the second half of 2026. Zai Lab participated in the studies in Greater China.

Povetacicept (APRIL/BAFF)

•Primary membranous nephropathy (pMN): Zai Lab to join the global pivotal Phase 2/3 OLYMPUS study of povetacicept in pMN in Greater China in the fourth quarter of 2025.

•IgAN: Zai Lab partner Vertex will conduct an interim analysis of the global Phase 3 RAINIER study following 36 weeks of treatment. Vertex expects to submit the first module of the IgAN BLA to the FDA before the end of 2025 and plans to complete the full BLA submission in the first half of 2026 for potential accelerated approval in the U.S.

VRDN-003 (anti-IGF-1R, subcutaneous)

•Zai Lab to initiate a registrational study in TED in Greater China in the fourth quarter of 2025.

•Viridian to provide topline results from global registrational REVEAL-1 study in active TED patients in the first quarter of 2026 and global registrational REVEAL-2 study in chronic TED in the second quarter of 2026. Zai Lab, through its license agreement with Zenas, obtained a sublicense to the Viridian anti-IGF-1R antibody and is proceeding with clinical development.

Full-Year 2025 Outlook

Zai Lab is revising total revenue guidance for the full year 2025 to at least $460 million.

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast today, November 6, 2025, at 8:00 a.m. ET (9:00 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 (preferred): 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.

(Press release, Zai Laboratory, NOV 6, 2025, View Source [SID1234659593])

On November 6, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported robust preliminary results from its ongoing Phase 3 UTOPIA trial, demonstrating a 77.8% three-month complete response (CR) rate (95% CI, 68.3%, 85.5%) with UGN-103 (mitomycin) for intravesical solution in patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). This result is consistent with the 79.6% three-month CR rate (95% CI, 73.9%, 84.5%) observed following treatment with ZUSDURI in the pivotal ENVISION trial. In addition, the U.S. Food and Drug Administration (FDA) agreed that CR and durability results from the UTOPIA trial can support the submission of a New Drug Application (NDA) for UGN-103 for recurrent LG-IR-NMIBC.

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"The robust 77.8% three-month CR rate observed in the UTOPIA trial is highly encouraging and reinforces the potential of UGN-103 to deliver meaningful benefits to patients," said Liz Barrett, President and Chief Executive Officer of UroGen. "In addition, the FDA’s agreement that the UTOPIA trial can support the submission of an NDA for UGN-103 represents a significant regulatory milestone and a strong validation of our clinical strategy. Together, these achievements give us significant momentum and a clear path toward potential approval, positioning UGN-103 as a key growth driver and marking an important advancement in expanding our leadership in uro-oncology. We look forward to working closely with the FDA as we complete the UTOPIA trial and prepare for an NDA submission in 2026."

UGN-103 is designed to offer key improvements over ZUSDURI (mitomycin) for intravesical solution, the first and only FDA-approved treatment for adults with recurrent LG-IR-NMIBC. These include a shorter manufacturing process and a simplified reconstitution procedure, while maintaining UroGen’s established approach that enables prolonged drug exposure within the bladder. UroGen has two U.S. patents with claims relating to the combination of the Company’s RTGel technology combined with medac’s licensed proprietary lyophilized mitomycin formulation. These patents cover UGN-103 as well as the use of UGN-103 for the treatment of LG-IR-NMIBC and expire in December 2041.

About UTOPIA

The UTOPIA trial is a single-arm, multicenter study evaluating the efficacy and safety of UGN-103 in 99 patients across global sites. Enrolled patients received 75 mg of UGN-103 via intravesical instillation once weekly for six weeks. The primary endpoint is complete response rate at three months, with responders entering a follow-up phase of up to 12 months to assess durability of response. For more information on the UTOPIA study, please visit View Source

About UGN-103
In January 2024, UroGen entered into a licensing and supply agreement with medac to develop UGN-103 for LG-IR-NMIBC. UGN-103 is an innovative mitomycin formulation that aims to streamline manufacturing and reconstitution processes while providing intellectual property coverage until December 2041. It utilizes UroGen’s RTGel technology for prolonged mitomycin exposure.

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.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include transurethral resection of bladder tumor (TURBT) as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence, and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.

(Press release, UroGen Pharma, NOV 6, 2025, View Source [SID1234659592])

On November 6, 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 third quarter ended September 30, 2025, and provided an overview of recent developments.

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"Our launch of ZUSDURI, the first and only FDA-approved medicine for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer, continues to gain momentum," said Liz Barrett, President and Chief Executive Officer of UroGen. "Despite slower than anticipated new patient starts, we are encouraged by the patient demand reflected in our patient enrollment forms. Strong enthusiasm and engagement from urologists, growing physician awareness, and broad reimbursement coverage are expanding patient access. Early launch indicators reflect robust interest and confidence in ZUSDURI’s clinical value, reinforcing our belief in the significant commercial opportunity ahead and our ability to fully capitalize on it. The strong complete-response rate for UGN-103 and the FDA’s agreement with our NDA submission plan supports our strategy for the next-generation medicines that are expected to enhance supply, improve manufacturing and preparation efficiencies and provide opportunity for lifecycle extensions. With a strong financial position, we are committed to driving a successful launch of ZUSDURI and advancing our pipeline in ways that deliver lasting impact for patients and long-term value for shareholders."

Q3 2025 and Recent Business Highlights:

ZUSDURI (mitomycin) for intravesical solution:

Following U.S. Food and Drug Administration (FDA) approval on June 12, 2025, UroGen launched ZUSDURI (formerly UGN-102), the first and only FDA-approved medicine for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC), marking a major milestone in bladder cancer care.
ZUSDURI was assigned a unique, permanent Healthcare Common Procedure Coding System J-code (J9282) by the Centers for Medicare & Medicaid Services. The J-code is expected to be effective January 1, 2026.
ZUSDURI is now broadly accessible to patients through Commercial, Medicare, and Medicaid insurance programs, with open access for more than 95% of covered lives and approximately 296 million eligible patients.
ZUSDURI achieved net product revenue of $1.8 million in Q3 2025; October 2025 preliminary demand revenue estimate of $4.5 million demonstrates accelerating commercial uptake and growing physician adoption.
From launch on July 1, 2025 through October 31, 2025, UroGen reports:
592 activated sites of care
54 unique ZUSDURI prescribers
16 repeat ZUSDURI prescribers
The article "Review of UGN-102: A Reverse Thermal Gel Containing Mitomycin for the Treatment of Recurrent, Low-Grade, Intermediate-Risk Non-Muscle Invasive Bladder Cancer" was published in Reviews in Urology and highlights durable efficacy and manageable safety profile of ZUSDURI in patients with recurrent LG-IR-NMIBC.

JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC)

Generated net product revenue of $25.7 million in the quarter ended September 30, 2025, compared to $25.2 million (which included CREATES Act sales of $2.6 million) reported for same quarter in 2024. Underlying demand revenue grew by approximately 13% year-over-year.

Next-generation novel mitomycin-based formulation for urothelial cancers

Enrollment is complete in the ongoing Phase 3 UTOPIA clinical trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with LG-IR-NMIBC. UroGen reported a three-month complete response rate (CRR) of 77.8% (95% CI, 68.3% to 85.5%), consistent with results from the ENVISION clinical trial.
The FDA agreed with the regulatory plan to submit an NDA based on the data from the single-arm Phase 3 UTOPIA trial to support potential approval of UGN-103. UroGen anticipates submitting an NDA for UGN-103 in the second half of 2026 with potential approval anticipated in 2027.
UGN-103 is a next generation product designed to offer certain improvements over ZUSDURI (mitomycin) for intravesical solution, including a shorter manufacturing process and simplified reconstitution procedure. UGN-103 combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH. For more information on the UTOPIA trial, refer to clinicaltrials.gov/NCT06331299.
The Phase 3 clinical trial to explore the safety and efficacy of UGN-104 is ongoing. UGN-104 is an investigational next-generation mitomycin product for LG-UTUC. Like UGN-103, UGN-104 combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH.

UGN-301 (zalifrelimab), an anti-CTLA4 antibody for use in high-grade non-muscle invasive bladder cancer

UroGen has made the strategic decision to discontinue development of UGN-301 (zalifrelimab) following completion of its Phase 1 dose escalation study. While the study confirmed proof of concept for RTGel as a viable platform for local delivery of complex immunotherapies, UGN-301’s overall clinical profile did not meet UroGen’s internal benchmarks for advancement to Phase 2. The program achieved key proof of concept objectives, including sustained bladder exposure with minimal systemic absorption and was generally well tolerated, demonstrating the ability to mitigate CTLA-4–related toxicities, and encouraging efficacy signals. These findings further reinforce the versatility and potential of RTGel technology to enable localized delivery of immunotherapy candidates.
UroGen provided notice to Agenus Inc. of termination for convenience of the License Agreement between the parties. In accordance with the terms of the agreement, termination will become effective upon the later of (i) expiration of the 180-day notice period, or (ii) completion of all required wind-down activities, including the delivery of any Agenus Improvements as defined in the License Agreement.

UGN-501 (investigational next-gen oncolytic virus) for use in high-grade non-muscle invasive bladder cancer (

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. Investigational New Drug (IND)-enabling studies are currently ongoing, with the goal of submitting an IND and initiating a Phase 1 trial in 2026.

Third quarter 2025 Financial Results

Revenue: Total revenues for the third quarter ended September 30, 2025 were $27.5 million. JELMYTO generated net product revenue of $25.7 million for the quarter ended September 30, 2025, compared to $25.2 million (which included $2.6 million of CREATES Act sales) in the same period of 2024, reflecting approximately 13% underlying year-over-year demand driven revenue growth. ZUSDURI achieved net product revenue of $1.8 million in its first quarter on the market, with October 2025 preliminary demand revenue estimate of $4.5 million demonstrating growing early commercial momentum.

R&D Expenses: Research and development (R&D) expenses for the third quarter of 2025 were $14.0 million, including non-cash share-based compensation expense of $0.7 million as compared to $11.4 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 $2.6 million was primarily driven by costs associated with the Phase 3 UTOPIA trial for UGN-103, partially offset by lower clinical trial costs, manufacturing costs, and regulatory expenses in connection with ZUSDURI.

SG&A Expenses: Selling, general and administrative expenses (SG&A) for the third quarter of 2025 were $37.6 million, including non-cash share-based compensation expense of $2.3 million. This compares to $28.9 million, including non-cash share-based compensation expense of $2.9 million, for the same period in 2024. The increase in SG&A expenses of $8.7 million was primarily driven by ZUSDURI commercial launch 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 third quarter of 2025, compared to $5.9 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 $3.4 million in the third quarter of 2025, compared to $2.7 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 $33.3 million or ($0.69) per basic and diluted share in the third quarter of 2025 compared with a net loss of $23.7 million or ($0.51) per basic and diluted share in the same period in 2024.

Cash and Equivalents: As of September 30, 2025, cash, cash equivalents and marketable securities totaled $127.4 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.

(Press release, UroGen Pharma, NOV 6, 2025, View Source [SID1234659591])

On November 6, 2025 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the third quarter 2025.

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Sana announced it will prioritize future development activity for SC451, a HIP-modified stem cell-derived pancreatic islet cell therapy for type 1 diabetes, and for SG293, an in vivo CAR T with CD8-targeted fusogen delivery of a CD19-directed CAR. The company continues to see meaningful progress in its type 1 diabetes program, particularly in advancing SC451 toward an IND. In addition, Sana improved the potency and manufacturability of its next-generation in vivo CAR T product platform, and the company is incorporating these updates into SG293 and expects to file an IND for this program as early as 2027. The company has suspended development of its two allogeneic cell therapy CAR T programs – SC291 in B-cell mediated autoimmune diseases and SC262 in oncology.

"Given our recent progress and the potentially transformative impact with SC451 in type 1 diabetes, as well as with our in vivo CAR T platform across a range of diseases, now is the time to concentrate our efforts in these programs," said Steve Harr, President and CEO of Sana. "Our goal for SC451 in type 1 diabetes is a single treatment leading to normal blood glucose with no need for further insulin treatment or immunosuppression, and the past several quarters of clinical results, manufacturing progress, and regulatory developments have solidified our confidence that this is possible. Additionally, as we progress toward our goals of filing an IND and beginning our Phase 1 clinical trial next year, we believe now is the time to free up resources to invest in scaling this important therapy. Separately, we have increased the potency of in vivo CAR T platform, and based upon preclinical data, believe we have the opportunity to develop best-in-class therapies with a single treatment and no conditioning chemotherapy for a range of B-cell cancers and B-cell mediated autoimmune diseases. Based upon our momentum and given the resources required to fully exploit these opportunities, we have made the difficult decision to suspend our allogeneic CAR T programs, including SC291 and SC262. While these programs increased our confidence in our HIP platform, we believe the impact we can have for patients and shareholders is now greater with increased focus on SC451 and in vivo CAR T cells."

Recent Corporate Highlights

Published positive results from an investigator-sponsored, first-in-human study transplanting UP421, an allogeneic primary islet cell therapy engineered with hypoimmune platform (HIP) technology, into a patient with type 1 diabetes without the use of any immunosuppression.

UP421 is a primary human HIP-modified pancreatic islet cell therapy for patients with type 1 diabetes. The goal of this investigator-sponsored trial (IST) is to understand safety, immune evasion, islet cell survival, and beta cell function, as measured by C-peptide production, of HIP-modified pancreatic islet cells transplanted into type 1 diabetes patients without the use of any immunosuppression. The trial is being conducted under a clinical trial authorization at Uppsala University Hospital with Dr. Per-Ola Carlsson as the principal investigator.
Results of the study through 6 months after cell transplantation demonstrate the survival and function of pancreatic beta cells as measured by the presence of circulating C-peptide, a biomarker indicating that transplanted beta cells are producing insulin. C-peptide levels also increase with a mixed meal tolerance test at these timepoints, consistent with insulin secretion in response to a meal. 12-week PET-MRI scanning demonstrated islet cells at the transplant site. The study identified no safety issues, and the HIP-modified islet cells evaded immune detection.
The New England Journal of Medicine (NEJM) published a journal article titled "Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression" (DOI: 10.1056/NEJMoa2503822). The article discusses 12-week results from this study. NEJM also published an accompanying editorial that further describes both the Sana technology and progress in the field (DOI: 10.1056/NEJMe2507578).
Sana and Uppsala University Hospital expect to report additional data from the IST, including longer-term follow-up.
Advancing our focused pipeline across two platforms:

Hypoimmune Platform – Type 1 diabetes – Sana continues pre-clinical development of SC451, an O-negative, HIP-modified, iPSC-derived pancreatic islet cell therapy, which uses the same HIP technology as UP421. Sana has had multiple interactions with regulators over the past several months, including an FDA INTERACT meeting, the results of which increase confidence in moving forward with our HIP-edited master cell bank for GMP manufacturing and our nonclinical testing plan. Sana expects to file an IND and begin our Phase 1 clinical trial for SC451 as early as 2026.
Fusogen Platform – In vivo CAR T cells – SG293 is the next-generation version of our prior SG299 product candidate and was renamed to reflect an updated construct and manufacturing process. SG293 is a CD8-targeted fusosome that delivers to CD8+ T cells the genetic material to make CD19-directed CAR T cells while avoiding potentially troublesome delivery to tissues such as the liver. SG293 builds on data from SG299, showing cell-specific delivery and deep B-cell depletion – as measured by depletion in circulating and lymph node B cells as well as a phenotypic reset when B cells return – in non-human primates without the use of any lymphodepleting chemotherapy. Sana intends to explore SG293 in both B-cell cancers and B-cell mediated autoimmune diseases. The company expects to file an IND for SG293 as early as 2027.
SC291 (HIP-modified CD19-directed allogeneic CAR T) in autoimmune diseases and SC262 (HIP-modified CD22-directed allogeneic CAR T) in oncology – Sana recently published in the journal Cell Stem Cell an article titled "Hypoimmune CD19 CAR T cells evade allorejection in patients with cancer and autoimmune disease," which provides additional clinical data showing how HIP-modified cells avoid immune attack (doi.org/10.1016/j.stem.2025.07.009). Nevertheless, as the company seeks to further prioritize resources and follow promising data in the SC451 and fusogen programs, Sana has suspended enrollment and further internal investment in the respective Phase 1 trials.
Raised aggregate gross proceeds of $133.2 million from sales of common stock through Sana’s at-the-market offering facility (ATM) and equity financing in the third and fourth quarters of 2025; expected cash runway into late 2026.

Closed public offering in August 2025 of 24.3 million shares of Sana’s common stock, including 3.4 million shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, and pre-funded warrants to purchase 1.5 million shares of Sana’s common stock. The gross proceeds from this offering were $86.3 million before deducting underwriting discounts and commissions and offering expenses.
Raised gross proceeds of $29.5 million in the third quarter of 2025 and an additional $17.4 million in the fourth quarter of 2025 from sales of common stock through Sana’s ATM.
Third Quarter 2025 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of September 30, 2025 were $153.1 million compared to $152.5 million as of December 31, 2024. The increase of $0.6 million was primarily driven by net proceeds from equity financings of $109.7 million and other cash inflows of $2.4 million, partially offset by cash used in operations of $111.2 million.
Research and Development Expenses: For the three and nine months ended September 30, 2025, research and development expenses, inclusive of non-cash expenses, were $30.1 million and $97.1 million, respectively, compared to $53.2 million and $170.5 million for the same periods in 2024. The decreases of $23.1 million and $73.5 million for the three and nine months ended September 30, 2025 compared to the same periods in 2024, respectively, were primarily due to the portfolio prioritization announced in the fourth quarter of 2024, which resulted in lower research, laboratory, and clinical development costs, lower personnel-related costs, including non-cash stock-based compensation, and a decrease in facility and other allocated costs. Research and development expenses include non-cash stock-based compensation of $3.2 million and $12.0 million, respectively, for the three and nine months ended September 30, 2025, and $6.5 million and $19.5 million for the same periods in 2024.
Research and Development Related Success Payments and Contingent Consideration: For the three and nine months ended September 30, 2025, Sana recognized non-cash expenses of $3.1 million and $15.3 million, respectively, compared to a non-cash gain of $5.5 million and a non-cash expense of $4.6 million for the same periods in 2024, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate. The value of these potential liabilities fluctuates significantly with changes in Sana’s market capitalization and stock price.
General and Administrative Expenses: General and administrative expenses for the three and nine months ended September 30, 2025, inclusive of non-cash expenses, were $10.3 million and $32.1 million, respectively, compared to $14.1 million and $46.8 million for the same periods in 2024. The decreases of $3.8 million and $14.7 million for the three and nine months ended September 30, 2025, respectively, compared to the same periods in 2024 were primarily due to lower personnel-related costs, including non-cash stock-based compensation, due to a decrease in headcount in connection with the portfolio prioritization announced in the fourth quarter of 2024, and decreased legal and consulting fees. General and administrative expenses include non-cash stock-based compensation of $2.3 million and $7.1 million for the three and nine months ended September 30, 2025, respectively, compared to $4.3 million and $11.8 million for the same periods in 2024.
Impairment of Long-Lived Assets: For the nine months ended September 30, 2025, non-cash impairment of long-lived assets was $44.6 million, compared to zero for the same period in 2024. The non-cash impairment, recorded in the second quarter of 2025, was primarily related to Sana’s manufacturing facility in Bothell, Washington and certain laboratory and office space in Seattle, Washington. Because of the increased availability of manufacturing capacity at third-party contract development and manufacturing organizations (CDMOs) for cell and gene therapy products as well as progress in understanding its near-term manufacturing needs, Sana expects to use CDMOs to meet its manufacturing needs at present and has suspended further build-out of internal manufacturing capabilities.
Net Loss: Net loss for the three and nine months ended September 30, 2025 was $42.2 million, or $0.16 per share, and $185.3 million, or $0.75 per share, respectively, compared to $59.9 million, or $0.25 per share, and $217.7 million, or $0.95 per share, for the same periods in 2024.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the nine months ended September 30, 2025 was $108.0 million compared to $153.1 million for the same period in 2024. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities, excluding cash inflows from financing activities, costs related to the portfolio prioritizations in the fourth quarters of 2024 and 2023, and the purchase of property and equipment.
Non-GAAP Net Loss: Non-GAAP net loss for the three and nine months ended September 30, 2025 was $39.0 million, or $0.15 per share, and $125.4 million, or $0.51 per share, respectively, compared to $64.7 million, or $0.27 per share, and $208.3 million, or $0.91 per share, for the same periods in 2024. Non-GAAP net loss excludes non-cash expenses and gains related to the change in the estimated fair value of contingent consideration and success payment liabilities, and non-cash impairment losses recorded in 2025.

(Press release, Sana Biotechnology, NOV 6, 2025, View Source [SID1234659590])