On April 14, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that data on investigational drug UGN-102 (mitomycin) for intravesical solution, JELMYTO (mitomycin) for pyelocalyceal solution and UGN-301 (zalifrelimab) will be presented at the American Urological Association (AUA) 2025 Annual Meeting being held in Las Vegas, Nevada from April 26-29 (Press release, UroGen Pharma, APR 14, 2025, View Source [SID1234651908]).

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"We are thrilled to present the 18-month DOR data on UGN-102, along with additional data on JELMYTO and our investigational drug UGN-301 (zalifrelimab) an anti-CTL4 antibody in development for the treatment of recurrent non-muscle invasive bladder cancer, at the AUA Annual Meeting," said Mark Schoenberg, Chief Medical Officer, UroGen. "These data highlight the potential of our portfolio to offer significant advancements in the treatment of urothelial cancers."

Key details of UGN-102, JELMYTO and UGN-301 abstracts accepted by AUA:

UGN-102

Abstract Title

Presentation Details

Presenter

Treatment of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer with UGN-102: ongoing results from a single-arm, open-label, phase 3 trial (ENVISION)

Podium Oral Presentation:

Abstract ID: PD12 – Galileo 1001

Saturday, April 26, 2025

3:30 PM – 5:30 PM

Dr. Sandip Prasad

Patient-reported side-effect burden for patients with low-grade intermediate-risk non-muscle invasive bladder cancer receiving treatment with UGN-102 (UGN-102 Integrated PROs)

Moderated Poster – MP15

Marco Polo 703

Sunday, April 27, 2025

9:30 AM – 11:30 AM

Dr. Charles Peyton

Treatment of low-grade intermediate-risk non-muscle invasive bladder cancer with UGN-102: long-term outcomes of the (OPTIMA II LT study)

Moderated Poster – MP15 –

Marco Polo 703

Sunday, April 27, 2025

9:30 AM – 11:30 AM

Dr. Neal Shore

JELMYTO

Long-term outcomes of treatment of recurrent or new-onset low-grade upper tract urothelial carcinoma with UGN-101, a mitomycin reverse thermal gel (OLYMPUS LT)

Interactive poster – IP12 – Casanova 501

Sunday, April 27, 2025

1:00 PM – 3:00 PM (IP12)

Dr. Brian Hu

UGN-301

Treatment of recurrent non-muscle invasive bladder cancer with UGN-301 (zalifrelimab): results of a phase 1 dose-escalation study (UGN-301-MONO)

Interactive Poster – IP02 – Marco Polo 701

Saturday, April 26, 2025

7:00 AM – 9:00 AM

Dr. Jay Raman

UroGen is a Founders’ Circle Sponsor of the AUA Innovation Nexus Conference
UroGen’s President and Chief Executive Officer, Liz Barrett, will participate in a Showcase Panel discussion on April 25, alongside exciting urology startups that span the globe and are developing products—devices, artificial intelligence platforms, diagnostic tests, etc.—covering a variety of urologic issues such as prostate and bladder cancer, kidney injuries, fertility testing, nocturnal enuresis, overactive bladder, and interstitial cystitis. Liz will also take part in the Founders’ Circle Awards Presentation.

The AUA Innovation Nexus is a powerful forum to advance urologic discovery to solutions that improve patient care and save lives. Register here: View Source

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 recurrent 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 submission of the rolling new drug application (NDA) for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a Prescription Drug User Free Act (PDUFA) goal date of June 13, 2025.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
In the U.S., bladder cancer is the second most common urologic cancer in men. LG-IR-NMIBC represents approximately 23,000 newly diagnosed bladder cancer patients each year and an estimated 59,000 recurrences annually among patients diagnosed in previous years. 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 trans-urethral 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.

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.

About UGN-301
UGN-301 is our investigational, in-licensed, anti-CTLA-4 monoclonal antibody (zalifrelimab), prepared with reverse-thermal hydrogel for intravesical administration into the bladder. Intravesical administration of UGN-301 is designed to increase drug concentrations in the bladder without significant systemic exposure, potentially diminishing the systemic toxicity associated with CTLA-4 blockade.

UroGen is evaluating UGN-301 as a monotherapy and as combination therapy for the intravesical treatment of high-grade NMIBC. UroGen is evaluating UGN-301, in a multi-arm Phase 1 study of UGN-301 as monotherapy and in combination with other agents.

On April 13, 2025 AdvanCell, a clinical-stage radiopharmaceutical company developing innovative cancer therapeutics, reported $18M in Australian Federal Government Funding from the Medical Research Future Fund (MRFF) Frontiers Initiative for ‘Defeating Prostate Cancer with Targeted Alpha Therapy’ (Press release, Advancell, APR 13, 2025, View Source [SID1234651896]).

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One out of eight men develops prostate cancer. With the support from the Medical Research Future Fund (MRFF) Frontiers Initiative, the goal of the multidisciplinary multi-institutional investigator team is to transform the clinical management of prostate cancer by leveraging leading therapeutic radiopharmaceutical technology paired innovative clinical development and a deep understanding of tumour biology to improve the lives of patients with prostate cancer.

The research program is enabled by AdvanCell’s proprietary (Lead-212) 212Pb alpha isotope production technology along with the delivery of a first-in-field clinical platform trial to accelerate the translation of 212Pb-based targeted alpha therapies, one of the most exciting breakthroughs in cancer treatment.

Dr. Anna Karmann MD PhD, AdvanCell Chief Medical Officer said: "On behalf of all Co-Investigators, I would like to thank the Australian Government and MRFF Frontiers Initiative committee for this prestigious award. Targeted alpha therapies are among the most promising in oncology. We believe this MRFF-funded research can be practice changing and have a lasting positive impact on the lives of patients with prostate cancer. We highly value the support and opportunity this funding provides to fast-track translation and accelerate the development of novel combination therapies in an industry-academic partnership."

AdvanCell is collaborating with world-leading experts in Australia and globally, underpinning the transformative nature of the important clinical research. The clinical PIs include internationally renowned physician scientists Prof. Louise Emmett (St Vincent’s Hospital, Sydney and University of New South Wales) and Prof. Shahneen Sandhu (Peter MacCallum Cancer Centre, Melbourne).

Prof. Louise Emmett: "This collaborative Frontiers grant gives us the tools to deeply evaluate optimal combinations with targeted alpha therapy in prostate cancer – aiming for longer better lives using great technology in a smart way."

Prof. Shahneen Sandhu: "Our MRFF grant will accelerate the development of innovative targeted alpha therapy combinations designed to enhance patient care and clinical outcomes."

Scientific investigators include Prof. Richard Payne (The University of Sydney), Prof. Matt Trau, Dr. Alain Wuethrich, Dr. Kevin Koo (The University of Queensland), Dr. Scott Lovell (University of Bath), A/Prof. Serigne Lo (Melanoma Institute Australia) and Dr. Thomas Kryza and Dr. Simon Puttick (AdvanCell).

Prof. Stephen Rose, Head of Translational Medicine and Clinical Science at AdvanCell, highlighted the importance of the MRFF funding. "The MRFF funding supports Australian innovation to drive the establishment of sovereign manufacturing capabilities to accelerate clinical translation of 212Pb-targeted alpha therapy."

On April 12, 2025 Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (the "Company") reported that results from a Phase 3 registrational clinical study evaluating the novel TROP2 antibody drug conjugate (ADC) sacituzumab tirumotecan (sac-TMT) for the treatment of adult patients with advanced triple-negative breast cancer (TNBC) and results from an early Phase I/II clinical study of sac-TMT for the treatment of adult patients with advanced non-small-cell lung cancer (NSCLC) were published in the top international medical journal Nature Medicine (Impact Factor (IF)= 58.7) (Press release, Kelun, APR 12, 2025, View Source [SID1234651895]).

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Results

TNBC: Based on the results of the multicenter, randomized, controlled Phase III OptiTROP-Breast01 (NCT05347134) clinical study, the current publication reports on the efficacy and safety of sac-TMT versus investigator’s choice chemotherapy for the treatment of patients with locally advanced, recurrent or metastatic TNBC. The results of the study showed that sac-TMT demonstrated statistically and clinically significant improvements in both progression-free survival (PFS) and overall survival (OS) compared to investigator’s choice chemotherapy. Based on this study, sac-TMT was approved for marketing for the treatment of adult patients with unresectable locally advanced or metastatic TNBC who have received at least two prior systemic therapies (at least one of them for advanced or metastatic setting). The data from this study were presented in an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

NSCLC: Based on the results of two early clinical studies of sac-TMT, the article reports on the efficacy and safety of sac-TMT in previously treated advanced NSCLC with or without epidermal growth factor receptor (EGFR) mutations. The article also explored in vitro experiments on the potential mechanisms of sac-TMT treatment for NSCLC, suggesting that EGFR mutations can increase the endocytosis and anti-tumor activity of TROP2 ADCs.

Dr. Michael Ge, CEO of Kelun-Biotech said, "These successful publications in Nature Medicine mark the international academic community’s recognition of the clinical efficacy and application value of sac-TMT in the treatment of advanced TNBC and NSCLC. The Company’s novel product, sac-TMT, has been marketed in China for two indications. Meanwhile, the Company is also actively promoting the registrational clinical studies of sac-TMT in multiple indications, including breast cancer and lung cancer. 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 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).

To date, two indications for sac-TMT have been approved and marketed in China for the treatment of adult patients with unresectable locally advanced or metastatic TNBC who have received at least two prior systemic therapies (at least one of them for advanced or metastatic setting) and EGFR mutation-positive locally advanced or metastatic non-squamous NSCLC following progression on EGFR-TKI therapy and platinum-based chemotherapy. Sac-TMT became the first domestic ADC with global intellectual property rights to be fully approved for marketing. It is also the world’s first TROP2 ADC to be approved for marketing in a lung cancer indication. In addition, the NDA application for sac-TMT for the treatment of adult patients with EGFR-mutant locally advanced or metastatic NSCLC who progressed after treatment with EGFR-TKI therapy was accepted by the NMPA, and was included in the priority review and approval process. As of today, Kelun-Biotech has initiated 8 registrational clinical studies in China. MSD has initiated 12 ongoing Phase 3 global clinical studies of sac-TMT as a monotherapy or with pembrolizumab or other agents for several types of cancer. These studies are sponsored and led by MSD.

On April 11, 2025 Sermonix Pharmaceuticals Inc., a privately held biopharmaceutical company developing innovative therapeutics to specifically treat metastatic breast cancers (mBC), and Quantum Leap Healthcare Collaborative reported that in the Phase 2 clinical trial evaluating lasofoxifene as a neoadjuvant endocrine therapy in molecularly selected HR+/HER2-, locally advanced breast cancer, the investigational drug was well tolerated and demonstrated promising early activity in suppressing the Ki67 protein in both premenopausal and postmenopausal patients (Press release, Sermonix Pharmaceuticals, APR 11, 2025, View Source [SID1234651888]).

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Results from the study, an arm of the Endocrine Optimization Pilot Protocol (EOP), a sub-study of Quantum Leap’s ongoing I-SPY 2 TRIAL (Investigation of Serial studies to Predict Your Therapeutic Response with Imaging And moLecular analysis 2), were initially shared yesterday in a poster presentation at RISE UP for Breast Cancer.

Twenty patients (10 pre- and nine postmenopausal women, and one male) were enrolled in the lasofoxifene arm of the study, during which early Ki67 suppression in premenopausal patients was seen in the absence of ovarian function suppression (OFS). Ki67 is a marker that measures how fast cancer cells are dividing.

"Lasofoxifene continued to demonstrate a strong tolerability profile while showing promising Ki67 suppression in this Phase 2 study, supporting our plans to further explore its potential in the neoadjuvant setting," said Dr. David Portman, Sermonix founder and chief executive officer. "We are currently studying lasofoxifene in the ELAINE-3 Phase 3 combination study with abemaciclib in the ESR1-mutated metastatic breast cancer setting. We are excited to see it continue to demonstrate broad potential while offering unique quality of life benefits for people confronted with breast cancer."

Patients were enrolled in the I-SPY 2 arm from March 2023 to May 2024. Median total days of treatment was 154 days. At the time of data analysis, three patients were still on treatment and two discontinued treatment due to physician or patient preference.

Median Ki67 expression went from 10% at baseline (range 1.0-40%) to 4% (1.0-18.0%) at Week 3. At three weeks, Ki67 expression was suppressed to <10% in 14 out of 16 (88%) patients, and to <2.7% in 6 out of 16 (38%) patients. Adverse events (AEs) were all grade 1-2. Most common AEs include hot flushes (65%), constipation (40%), fatigue (40%) and nausea (25%).

"The I-SPY 2 EOP provides a biomarker-rich platform to test novel endocrine agents in the neoadjuvant setting in a molecularly selected group of patients with tumors that are predicted to have little to no benefit from chemotherapy," said Dr. Jo Chien, principal investigator of the sub-study. "Lasofoxifene has demonstrated significant activity based on early Ki67 suppression, not only in postmenopausal women, but also in 10 premenopausal women without concomitant ovarian function suppression, with baseline Ki67 of 12.5% (1.0-40.0%) going to 3.0% (1.0-15%) at Week 3. It is exciting to see that lasofoxifene is well-tolerated even in our youngest patients who are often most impacted by the debilitating side effects of current standard endocrine therapy options."

Quantum Leap partners with a consortium that includes the U.S. Food and Drug Administration (FDA), industry, patient advocates, philanthropic sponsors and clinicians from 41 major U.S. cancer research centers.

About Lasofoxifene
Lasofoxifene is an investigational novel endocrine therapy in clinical development which has demonstrated robust target engagement as an ESR1 antagonist in the breast, particularly in the presence of ESR1 mutations. Lasofoxifene has demonstrated anti-tumor activity as monotherapy and in combination with a CDK4/6 inhibitor in Phase 2 studies and has unique tissue selectivity distinguishing it from other current and investigational endocrine therapies, with beneficial effects seen on vagina and bone in previous clinical studies. Lasofoxifene, which Sermonix licensed globally from Ligand Pharmaceuticals Inc., has been studied in previous comprehensive Phase 1-3 non-oncology clinical trials in more than 15,000 postmenopausal women worldwide. Lasofoxifene’s bioavailability and activity in mutations of the estrogen receptor could potentially hold promise for patients who have acquired endocrine resistance due to ESR1 mutations, a common finding in the metastatic setting and an area of high unmet medical need. Lasofoxifene’s novel activity in ESR1 mutations was discovered at Duke University and Sermonix has exclusive rights to develop and commercialize the product in this area. Lasofoxifene, a novel targeted and tissue selective oral endocrine therapy, could, if approved, play a critical role in the precision medicine treatment of advanced ER+ breast cancer.

On April 11, 2025 Amgen (NASDAQ:AMGN) reported that the global Phase 3 DeLLphi-304 clinical trial evaluating IMDELLTRA (tarlatamab-dlle) as a treatment for patients with small cell lung cancer (SCLC) who progressed on or after a single line of platinum-based chemotherapy met its primary endpoint at a planned interim analysis (Press release, Amgen, APR 11, 2025, View Source [SID1234651892]). IMDELLTRA demonstrated statistically significant and clinically meaningful improvement in overall survival (OS) compared to local standard-of-care (SOC) chemotherapy.

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"Small cell lung cancer is one of the most aggressive malignancies, with a high unmet need for more effective therapies.1 The topline results from DeLLphi-304 demonstrate overwhelming clinical benefit for people living with this devastating disease and affirm IMDELLTRA as standard of care," said Jay Bradner, M.D., executive vice president, Research and Development, at Amgen. "We look forward to sharing these results with the scientific community and health authorities as we continue our efforts to bring IMDELLTRA to patients worldwide."

The safety profile for IMDELLTRA was consistent with its known profile. Detailed data from DeLLphi-304 will be presented at an upcoming medical congress.

DeLLphi-304 is a global Phase 3 randomized controlled open-label clinical trial evaluating the efficacy and safety of IMDELLTRA as a treatment for patients with SCLC who progressed on or after a single line of platinum-based chemotherapy.2 Patients were randomized to receive either IMDELLTRA or local SOC chemotherapy (topotecan in all countries except Japan; lurbinectedin in the U.S., Canada, Australia, Singapore, Korea; and amrubicin in Japan).2,3 The primary outcome measure of the trial is OS.2

About IMDELLTRA (tarlatamab-dlle)
IMDELLTRA is a first-in-class immunotherapy engineered by Amgen researchers that binds to both DLL3 on tumor cells and CD3 on T cells, activating T cells to kill DLL3-expressing SCLC cells. This results in the formation of a cytolytic synapse with lysis of the cancer cell.4,5 DLL3 is a protein that is expressed on the surface of SCLC cells in ~85-96% of patients with SCLC, but is minimally expressed on healthy cells, making it an exciting target.6,7

IMDELLTRA (tarlatamab-dlle) U.S. Indication
IMDELLTRA (tarlatamab-dlle) is indicated for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

About Small Cell Lung Cancer (SCLC)
SCLC is one of the most aggressive and devastating solid tumor malignancies, with a 5-10% five-year relative survival rate across all stages combined.1 SCLC comprises about 15% of the more than 2.4 million patients diagnosed with lung cancer worldwide each year.8-10 Despite initial high response rates to first-line platinum-based chemotherapy, most patients quickly relapse within months and require subsequent treatment options.9

About Tarlatamab Clinical Trials
Amgen’s robust tarlatamab development program includes the DeLLphi clinical trials, which evaluate tarlatamab as both a monotherapy and in combination regimens in earlier lines of SCLC.

Tarlatamab is being investigated in multiple studies including DeLLphi-303, a Phase 1b study investigating tarlatamab in combination with standard of care therapies in first-line ES-SCLC; DeLLphi-304, a randomized Phase 3 trial comparing tarlatamab monotherapy with standard of care chemotherapy in second-line treatment of SCLC; DeLLphi-305, a randomized Phase 3 trial comparing tarlatamab in combination with durvalumab versus durvalumab alone as first-line maintenance treatment in ES-SCLC; DeLLphi-306, a randomized placebo-controlled Phase 3 trial of tarlatamab following concurrent chemoradiotherapy in limited-stage SCLC; DeLLphi-308, a Phase 1b study evaluating subcutaneous tarlatamab in second line or later ES-SCLC; and DeLLphi-309, a Phase 2 study evaluating alternative intravenous dosing regimens with tarlatamab in second-line ES-SCLC.11

For more information, please visit www.tarlatamabclinicaltrials.com.