On March 10, 2025 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and genetic testing, reported the enrollment of the first patients in the HEROES clinical trial. HEROES is a multi-center, phase II trial that explores the discontinuation or de-escalation of anti-HER2 targeted therapy among patients with metastatic HER2+ breast cancer (Press release, Natera, MAR 10, 2025, View Source [SID1234651050]). The trial is supported by funding from the French Ministry of Health through the Hospital Clinical Research Program (PHRC) and is being sponsored by Unicancer. It is being developed within Unicancer’s French Breast Cancer Intergroup (UCBG) network.

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Human epidermal growth factor receptor 2 (HER2) is a type of protein found in cancer cells that can cause rapid cancer growth when higher than normal levels are present. Between 15%-20% of breast tumors are HER2+.¹ Current standard-of-care (SOC) recommendations for maintenance treatment include the use of anti-HER2 therapies, which for many patients means remaining on the drug for life.

The HEROES (NCT06450314) trial will enroll approximately 170 metastatic HER2+ breast cancer patients who have discontinued anti-HER2 maintenance therapy. The primary endpoint of the study is 1-year progression-free survival in the Signatera-negative cohort. It will also assess ctDNA dynamics and quality-of-life measures to gain deeper insights into the potential for ctDNA-guided de-escalation of treatment in patients with no detectable molecular disease burden.

Highlights from the study protocol include:

At baseline, all patients are currently being treated with SOC anti-HER2 targeted therapies and have been on therapy for two or more years.
Signatera-negative patients at baseline will stop treatment and will be monitored with serial Signatera testing and diagnostic imaging. If at any point during the trial radiological progression is confirmed or a patient becomes Signatera-positive, prior drug therapy will resume or a new treatment will begin.
Signatera-positive patients at baseline will continue maintenance therapy and will not move forward in the trial.
"The HEROES study could significantly reshape the way oncologists treat patients with metastatic HER2+ breast cancer," said Thibault de la Motte Rouge, M.D., Ph.D., principal investigator of the trial and medical oncologist at the Comprehensive Cancer Centre Eugène Marquis (Rennes, France), where he currently holds the position of research director. "This could also pave the way for future research into ctDNA-guided treatment de-escalation in breast cancer."

"We are excited to see the first patients enrolled in the HEROES clinical trial," said Angel Rodriguez, M.D., senior medical director of oncology at Natera. "Safely discontinuing treatment has been a long-lasting dilemma in HER2 metastatic breast cancer. With Signatera monitoring, we hope oncologists can identify the patients without detectable disease who may be able to avoid additional therapy that can be costly and potentially harmful to their care."

About Signatera

Signatera is a personalized, tumor-informed, molecular residual disease test for patients previously diagnosed with cancer. Custom-built for each individual, Signatera uses circulating tumor DNA to detect and quantify cancer left in the body, identify recurrence earlier than standard-of-care tools, and help optimize treatment decisions. The test is available for clinical and research use and is covered by Medicare for patients with colorectal cancer, breast cancer, ovarian cancer, and muscle-invasive bladder cancer, as well as for immunotherapy monitoring of any solid tumor. Signatera has been clinically validated across multiple cancer types and indications, with published evidence in over 100 peer-reviewed papers.

On March 10, 2025 NiKang Therapeutics Inc. ("NiKang"), a clinical stage biotech company focused on developing innovative small molecule oncology medicines to bring transformative therapies to patients in need, reported the successful completion of dosing the first cohort of patients in its Phase 1 dose escalation study of NKT3964 as a single agent. NKT3964 is a first-in-class, orally bioavailable small molecule that selectively degrades CDK2 (Press release, NiKang Therapeutics, MAR 10, 2025, View Source [SID1234651049]). NKT3964, with high potency, selectivity and sustained inhibition of the CDK2 pathway without cyclin E accumulation, has the potential to provide therapeutic benefits for patients with aberrant CDK2/cyclin E pathway activation, such as ovarian, endometrial, gastric and HR+HER2- breast cancers.

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The Phase 1 trial (NCT06586957) is an open-label, dose escalation study designed to evaluate safety, tolerability, PK, PD and preliminary anti-tumor activity to determine the recommended dose(s) for expansion of NKT3964 monotherapy in adults with advanced or metastatic solid tumors.

"We are thrilled to reach this milestone in the clinical development of NKT3964," said Zhenhai Gao, Ph.D., co-founder, president, and CEO of NiKang. "Completing dosing in our first cohort brings us one step closer to understanding the potential of this groundbreaking CDK2 degrader, one of several molecules in our portfolio targeting the cell cycle. Initial PK data from the first cohort demonstrated good oral exposure that aligns with human PK projections. Additionally, NKT3964 has achieved CDK2 degradation levels in patients that are consistent with those observed in preclinical in vivo studies. These early observations are particularly encouraging as they address the considerable challenges of achieving oral bioavailability with a PROTAC degrader. These findings will help guide dose optimization as the trial advances. Our pipeline focused on cell cycle inhibition via CDK2 degradation or CDK2/CDK4 dual degradation enables us to have multiple opportunities for success. We are excited by this progress and remain committed to advancing transformative therapies to help patients fight cancer and live better lives."

About NKT3964

NKT3964 is a first-in-class, highly potent and selective, orally bioavailable CDK2 degrader, causing prolonged CDK2 pathway inhibition without cyclin E accumulation. It has the potential to maximally and selectively suppress the CDK2 pathway, thereby harnessing the full therapeutic benefits of CDK2 inhibition. NKT3964 is currently under evaluation in a Phase 1 clinical study in advanced or metastatic solid tumors as a single agent (NCT06586957).

On March 10, 2025 Hoth therapeutics reported that HT-001 is being developed to alleviate the adverse dermatological effects experienced by cancer patients undergoing epidermal growth factor receptor (EGFR) inhibitor treatments (Press release, Hoth Therapeutics, MAR 10, 2025, View Source [SID1234651048]). With positive progress in ongoing clinical studies, Hoth Therapeutics aims to provide patients access to HT-001 outside of traditional clinical trials through the Expanded Access Program (EAP), commonly known as "compassionate use."

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NEW YORK, March 10, 2025 /PRNewswire/ — Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical company dedicated to developing innovative therapies, reported its intention to submit an Expanded Access application for HT-001, its novel therapeutic candidate for the treatment of dermatological conditions associated with cancer therapy.

HT-001 is being developed to alleviate the adverse dermatological effects experienced by cancer patients undergoing epidermal growth factor receptor (EGFR) inhibitor treatments. With positive progress in ongoing clinical studies, Hoth Therapeutics aims to provide patients access to HT-001 outside of traditional clinical trials through the Expanded Access Program (EAP), commonly known as "compassionate use."

"Submitting an Expanded Access application is an important step in our commitment to ensuring patients who may benefit from HT-001 have the opportunity to receive this promising treatment," said Robb Knie, CEO of Hoth Therapeutics. "We remain dedicated to advancing solutions that address critical unmet medical needs and improve quality of life for patients undergoing cancer treatment."

The Expanded Access Program is designed to provide investigational treatments to patients with serious or life-threatening conditions who lack comparable treatment options. Through this program, Hoth Therapeutics seeks to offer HT-001 to eligible patients while continuing to gather valuable data on its safety and efficacy.

For more information about HT-001, ongoing clinical trials, or the Expanded Access Program, please visit www.hoththerapeutics.com

On March 10, 2025 Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (the "Company") reported that the Company’s trophoblast cell-surface antigen 2 (TROP2)-directed antibody-drug conjugate (ADC) sacituzumab tirumotecan (sac-TMT, formerly SKB264/MK-2870) was approved for marketing by the National Medical Products Administration (NMPA) for the second indication which is for the treatment of adult patients with epidermal growth factor receptor (EGFR) mutant-positive locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) following progression on EGFR-tyrosine kinase inhibitor (TKI) therapy and platinum-based chemotherapy (Press release, Kelun, MAR 10, 2025, View Source [SID1234651047]). This is the first TROP2 ADC drug approved for marketing in lung cancer globally. Compared with current standard of care, sac-TMT significantly extents the overall survival benefits of these patients.

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The approval is based on a multi-center, randomized, controlled, pivotal study (OptiTROP-Lung03) that evaluates the efficacy and safety profile of sac-TMT monotherapy 5mg/kg every other week (Q2W) as an intravenous injection versus docetaxel for the treatment of patients with locally advanced or metastatic EGFR-mutant NSCLC who have failed after treatment with an EGFR-TKI and platinum-based chemotherapy (used sequentially or in combination). As demonstrated in a pre-specified interim analysis, sac-TMT monotherapy demonstrated a statistically significant and clinically meaningful improvement in objective response rate (ORR), progression-free survival (PFS) and overall survival (OS) compared with docetaxel.

Sac-TMT is also the first ADC drug to demonstrate a statistically and clinically significant improvement in overall survival benefit versus standard of care in patients with EGFR-mutant NSCLC after failure of EGFR-TKI and platinum-based chemotherapy.

Previously, the NMPA has approved the marketing of sac-TMT in China for adult patients with unresectable locally advanced or metastatic triple negative breast cancer (TNBC) who have received at least two prior systemic therapies (at least one of them for advanced or metastatic setting) and accepted a supplemental new drug application (sNDA) seeking the approval of sac-TMT monotherapy for patients with EGFR-mutant NSCLC who have failed after EGFR-TKI therapy.

Currently, sac-TMT is fully deployed in the NSCLC field, and Merck Sharp & Dohme and the company are conducting a total of 10 registrational Phase Ⅲ clinical studies in NSCLC globally and in China, with the threads covering from the late backline to early postoperative adjuvant, including monotherapy and combination studies. Among them, a registrational Phase Ⅲ clinical study has been conducted in China in combination with osimertinib for the first-line treatment of locally advanced or metastatic NSCLC with EGFR mutations (OptiTROP-Lung07).

Dr. Michael Ge, CEO of Kelun-Biotech said, "We are very pleased that the second indication of the Company’s TROP2 ADC sacituzumab tirumotecan has been approved for marketing, which is exciting progress for us in the field of lung cancer. The successful approval of this new indication will help to address an unmet treatment need for patients with later-stage EGFRm NSCLC in China. Kelun-Biotech has always been committed to promoting innovation and leadership, and we look forward to continuing our research in the field of ADCs in partnership with MSD."

About NSCLC

Lung cancer, the second most common cancer in the world, includes two main types of cancer: non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). NSCLC is the most common pathological type, accounting for about 80 to 85% of all lung cancers. The molecular typing of NSCLC patients in China is different from that of Western populations, and EGFR mutation is a common variant gene type, accounting for about 40 to 50% of lung adenocarcinoma patients in China. According to the 2024 Chinese Journal of Cancer Research (CSCO) guidelines, EGFR-TKIs are the preferred treatment for stage IV EGFR-mutant NSCLC.1 Platinum-containing chemotherapy is the main first-line chemotherapy regimen after resistance to EGFR-TKIs; and existing treatment regimens are ineffective in those who have failed EGFR-TKIs and platinum-containing chemotherapy. New drugs are urgently needed to improve patient survival.

About sac-TMT (佳泰莱)

Sac-TMT, a core product of the Company, is a novel human TROP2 ADC in which the Company has proprietary intellectual property rights, targeting advanced solid tumors such as NSCLC, breast cancer (BC), gastric cancer (GC), gynecological tumors, among others. Sac-TMT is developed with a novel linker to conjugate the payload, a belotecan-derivative topoisomerase I inhibitor with a drug-to-antibody-ratio (DAR) of 7.4. Sac-TMT specifically recognizes TROP2 on the surface of tumor cells by recombinant anti-TROP2 humanized monoclonal antibodies, which is then endocytosed by tumor cells and releases KL610023 intracellularly. KL610023, as a topoisomerase I inhibitor, induces DNA damage to tumor cells, which in turn leads to cell-cycle arrest and apoptosis. In addition, it also releases KL610023 in the tumor microenvironment. Given that KL610023 is membrane permeable, it can enable a bystander effect, or in other words kill adjacent tumor cells.

In May 2022, the Company licensed the exclusive rights to MSD (the tradename of Merck & Co., Inc., Rahway, NJ, USA) to develop, use, manufacture and commercialize sac-TMT in all territories outside of Greater China (includes Mainland China, Hong Kong, Macau, and Taiwan).

On March 10, 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 fourth quarter and full year ended December 31, 2024, and provided an overview of recent developments (Press release, UroGen Pharma, MAR 10, 2025, View Source [SID1234651045]).

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"2024 was a pivotal year for UroGen with achievements across our commercial business and pipeline," said Liz Barrett, President and Chief Executive Officer of UroGen. "The progress with UGN-102 for low-grade intermediate-risk non-muscle invasive bladder cancer, including completion of the submission of our NDA ahead of schedule and compelling DOR data from the Phase 3 ENVISION trial, further positions us to launch a product that we believe will represent a paradigm shift in care, if approved. UGN-102 is supported by a compelling body of clinical data, and we are very pleased to report today the 18-month DOR of 80.6% from the ENVISION trial, maintaining duration of response consistent with 12-month DOR of 82.5% after a three-month CR was achieved. If approved, UGN-102 will address an estimated market opportunity of over $5 billion. We continue to advance our early-stage pipeline, including through the recent purchase of ICVB-1042, a next generation investigational oncolytic virus therapy with potential applications in bladder and other cancers."

2024 and Recent Business Highlights:

UGN-102 (mitomycin) for intravesical solution:

UroGen’s NDA for investigational drug UGN-102 (mitomycin) for intravesical solution as a treatment for low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC), is currently under review by the FDA. The FDA assigned a PDUFA target action date of June 13, 2025.
The Company reported updated 18-month DOR data from the Phase 3 ENVISION trial that evaluated UGN-102 in patients with LG-IR-NMIBC. The 18-month DOR data is consistent with prior Kaplan-Meier estimates, with a DOR rate of 80.6% (95% CI: 74.0, 85.7) at 18-months (N=101) compared to 82.5% (95% CI: 76.1, 87.3) at 12-months (N=146). Median follow-up time at 18 months was 18.7 months after the three-month CR.
Results from a subgroup analysis of the ENVISION trial were featured in a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium (ASCO-GU 2025) in February 2025. The analysis of pre-specified subgroups (presented by Sandip M Prasad, MD, MPhil et al) suggests that tumor burden and the number of tumors at baseline did not significantly affect the CR rate or DOR for patients treated with UGN-102.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC):

Generated net product revenue of $90.4 million and $82.7 million for the years ended December 31, 2024 and 2023, respectively. Underlying demand revenue increased 12% year-over-year, partially offset by a decrease in CREATES Act sales, which totaled $3.0 million in 2024 compared to $4.4 million in 2023. "New prescribers" and "new patient" starts increased 33% and 13%, respectively, in 2024.
A long-term study on JELMYTO titled, "Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study," was published online in Urologic Oncology: Seminars and Investigations in January 2025. The results showed 68% recurrence-free survival rate at three years across a broad patient population with LG-UTUC.
A long-term follow-up study to the OLYMPUS trial evaluating JELMYTO was presented at the Society for Urologic Oncology annual meeting in December 2024. The study reported a median DOR of 47.8 months in patients who achieved a CR after primary chemoablation with JELMYTO. These results were also published online in the Journal of Urology.
Next-generation novel mitomycin-based formulation for urothelial cancers

Enrollment is ongoing in the Phase 3 UTOPIA trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with LG-IR-NMIBC. UGN-103 is a next generation investigational medicine that combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH. UGN-103 is planned to follow the potential FDA approval and launch of UGN-102 for LG-IR-NMIBC. To learn more about the UTOPIA trial, refer to clinicaltrials.gov/NCT06331299.
UroGen plans to initiate a Phase 3 trial to explore the safety and efficacy of UGN-104, its next generation product for LG-UTUC in the first half of 2025.
UGN-301 (zalifrelimab), an anti-CTLA4 antibody for use in high-grade non-muscle invasive bladder cancer

Completed enrollment of 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.
Next-generation investigational oncolytic virus ICVB-1042

In February 2025, UroGen expanded its pre-clinical oncology portfolio with the purchase of the product candidate ICVB-1042 and certain related assets from IconOVir Bio, Inc. ICVB-1042 is being developed to be a potent, fast-replicating, next-generation oncolytic virus therapy that can be administered locally to treat bladder cancer and potentially other specialty cancers. UroGen hosted a webinar to discuss ICVB-1042 on February 20, 2025 and a replay can be accessed on the Company’s website here.
Fourth Quarter and Full Year 2024 Financial Results

JELMYTO Revenue: JELMYTO net product revenue was $24.6 million in the fourth quarter of 2024, compared to $23.5 million for the same period in 2023. Underlying demand revenue increased 15% year-over-year, partially offset by a decrease in CREATES Act sales, which totaled $0.2 million in the fourth quarter of 2024, compared to $2.4 million in the same period in 2023. JELMYTO net product revenue for the full year ended December 31, 2024 was $90.4 million, compared with $82.7 million for the full year ended December 31, 2023.

R&D Expense: Research and development expenses for the fourth quarter of 2024 were $14.9 million, as compared to $11.3 million for the same period in 2023. Research and development expenses for the full year 2024 were $57.1 million compared with $45.6 million for the full year 2023.

SG&A Expense: Selling, general and administrative expenses for the fourth quarter of 2024 were $34.9 million compared to $24.6 million in the same period in 2023. Selling, general and administrative expenses were $121.2 million for the full year 2024 compared with $93.3 million for the full year 2023.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $6.1 million in the fourth quarter of 2024, compared to $5.5 million in the same period in 2023. Non-cash financing expense related to RTW Investments were $23.4 million in the full year 2024 compared with $21.6 million in the full year 2023.

Interest Expense on Long-Term Debt: Interest expense related to the outstanding $125 million term loan facility with funds managed by Pharmakon Advisors was $3.9 million and $12.5 million, respectively, for the fourth quarter and full year 2024, compared with $3.6 million and $14.7 million, respectively for the fourth quarter and full year 2023.

Net Loss: UroGen reported a net loss of $37.5 million or ($0.80) per basic and diluted share in the fourth quarter of 2024 compared with a net loss of $26.0 million or ($0.72) per basic and diluted share in the same period in 2023. Net loss was $126.9 million or ($2.96) per basic and diluted share in 2024 compared with a net loss of $102.2 million or ($3.55) per basic and diluted share in 2023.

Cash & Cash Equivalents: As of December 31, 2024, cash, cash equivalents and marketable securities totaled $241.7 million.

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

2025 JELMYTO Revenue and Company Operating Expense Guidance: The Company expects full-year 2025 net product revenues from JELMYTO 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. Full-year 2025 operating expenses are expected 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 investors.UroGen.com. 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.

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.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as UTUC. In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can be associated with a high rate of recurrence and relapse.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.