On June 2, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, reported the presentation of the uTRACT Registry study design at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL (Press release, UroGen Pharma, JUN 2, 2025, View Source [SID1234653627]). The uTRACT Registry is a single-arm, multicenter, prospective and retrospective study evaluating the real-world use of JELMYTO (mitomycin) for pyelocalyceal solution for the treatment of adult patients with low-grade upper tract urothelial carcinoma (LG-UTUC) across the United States.

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"By capturing these real-world insights, the uTRACT Registry will inform best practices in the management of LG-UTUC," said Mark Schoenberg, Chief Medical Officer, UroGen. "This robust data set aims to support the ongoing clinical utility of JELMYTO in managing LG-UTUC, offering new insights into treatment patterns, long-term outcomes, and patient safety in real-world settings."

As of May 2025, the uTRACT Registry (NCT05874921) has enrolled 274 participants across 22 clinical sites nationwide, with a target enrollment of approximately 400 patients. Of these, about 340 are expected to have LG-UTUC, the population for which JELMYTO is FDA-approved. The study is designed to collect comprehensive data over a three-year period. This includes patient history and baseline disease characteristics prior to initiating treatment with JELMYTO, as well as details on the mode of administration, dosing regimens, surveillance endoscopy findings, and imaging results. Investigators will assess key clinical outcomes such as the presence of no evidence of disease at three months post-treatment, duration of response, recurrence-free survival, time to recurrence or progression, and adverse events.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel approved for the treatment of adult patients with LG-UTUC. 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 a 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 Upper Tract Urothelial Cancer

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 LG-UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often have multiple comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). Treatment with endoscopic surgery can be associated with a high rate of recurrence and relapse.

On June 2, 2025 Eli Lilly and Company (NYSE: LLY) reported new Phase 1 data showing that its folate receptor alpha (FRα) antibody-drug conjugate (ADC) (LY4170156) demonstrated an encouraging safety profile and anti-tumor activity across dose and FRα expression levels in women with heavily pre-treated platinum-resistant ovarian cancer, including patients previously treated with mirvetuximab soravtansine (Press release, Eli Lilly, JUN 2, 2025, View Source [SID1234653643]). A preliminary overall objective response rate (ORR) of 55% was observed at the potential recommended Phase 2 dose of 4 mg/kg. Lilly’s FRα targeting ADC is composed of an Fc-silent, FRα specific humanized monoclonal antibody linked to exatecan, a topoisomerase I inhibitor, via a proprietary cleavable polysarcosine linker. These data will be presented today in a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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"ADCs have begun to change the treatment paradigm for some women with ovarian cancer, but a large proportion of patients still have a significant need for new therapies that improve outcomes regardless of FRα expression level," said Isabelle Ray-Coquard, M.D., Ph.D., president of the ENGOT (European Network of Gynecological Oncology Trial) group, medical oncologist at the Centre Léon Bérard Lyon France and principal investigator for the trial. "These initial data show activity across all doses and levels of FRα expression, including in patients previously treated with a FRα targeting treatment. Taken together with the emerging safety and tolerability profile, these data demonstrate early potential to meaningfully improve outcomes for women living with advanced ovarian cancer."

As of the March 9, 2025 data cutoff, the study enrolled 95 participants with high-grade serous ovarian cancer across four dose levels (2 – 6 mg/kg). Patients received a median of five prior systemic regimens (range 1-10), and 15% were previously treated with mirvetuximab soravtansine. Among the 95 patients, 51% had tumors with FRα expression less than 75%, 34% had FRα expression of 75% or higher, and 16% had expression results pending. Key endpoints were safety, pharmacokinetics, and anti-tumor activity per RECIST v1.1.

Efficacy results demonstrate responses at all dose levels, across all FRα expression levels, including in patients who progressed on prior mirvetuximab soravtansine. In the 58 efficacy-evaluable patients (37 patients remain ongoing prior to first response assessment and were therefore not yet efficacy-evaluable at the time of the data cutoff), the ORR was 45% (26/58 patients), and the disease control rate was 74% (43/58). At the potential recommended Phase 2 dose of 4 mg/kg, the ORR was 55% (11/20 patients). The most common treatment-emergent adverse events across all dose levels included nausea (64%), anemia (40%), fatigue (32%), vomiting (32%), diarrhea (28%), and neutropenia (27%). Treatment-emergent neuropathy and ocular toxicity has not been observed to date. No maximum tolerated dose has been established.

"We are excited to share these first clinical data for our FRα targeting ADC, demonstrating a promising tolerability and efficacy profile across all FRα expression levels," said David Hyman, M.D., Chief Medical Officer, Lilly. "Based on these results, we believe there is the potential to significantly expand the number of ovarian cancer patients who could benefit from a FRα ADC. We are now focused on rapidly advancing this potential new medicine into registrational Phase 3 clinical trials."

For more information on Lilly’s oncology pipeline click here.

About LY4170156
LY4170156 is an investigational, next-generation antibody-drug conjugate (ADC) targeting folate receptor alpha (FRα). FRα is a cell-surface glycoprotein encoded by the gene FOLR1 that binds to the essential nutrients folic acid and reduced folates, bringing them into cells to facilitate cell division and growth.1,2 FRα is overexpressed in many solid tumors such as ovarian, non-small cell lung, and colorectal cancers.1,3,4

LY4170156 was designed to target FRα across expression levels with an improved therapeutic index. LY4170156 is composed of an Fc-silent, FRα specific humanized monoclonal antibody linked to exatecan, a topoisomerase I inhibitor, via a proprietary cleavable polysarcosine linker (PSARlink). PSARlink’s unique structure "masks" the cytotoxic molecules enabling them to stay in the body longer, providing the potential to broaden the therapeutic index of ADCs. LY4170156 is currently being studied in patients with ovarian cancer as well as other FRα-expressing solid tumors, NCT06400472.

On June 2, 2025 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported that the results of the Phase III SERENA-6 trial – sponsored by AstraZeneca – demonstrate the clinical value of the Guardant360 CDx test in a circulating tumor DNA-guided approach to detect and treat emerging resistance in 1st-line therapy ahead of radiological disease progression in breast cancer (Press release, Guardant Health, JUN 2, 2025, View Source [SID1234653659]). Study results were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago and were published in The New England Journal of Medicine.

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SERENA-6 is the first global, double-blind, registrational Phase III trial to use a ctDNA-guided approach to detect the emergence of endocrine resistance and inform a switch in therapy before disease progression is detected in imaging scans. The novel trial design used ctDNA monitoring with the Guardant360 liquid biopsy test at the time of routine tumor scans to identify patients for early signs of endocrine resistance and the emergence of ESR1 mutations.

"SERENA-6 is a landmark study that is creating a new paradigm using liquid biopsy to enable a switch to a new treatment as soon as you see the cancer showing signs of resistance," said Helmy Eltoukhy, Guardant Health chairman and co-CEO. "This use of the Guardant360 CDx test highlights how we are pushing the boundaries of what can be done with liquid biopsy in characterizing disease and potential drug efficacy, providing insights that could potentially change clinical practice and improve outcomes in patients with advanced breast cancer."

Following detection of an ESR1 mutation without radiological disease progression, the endocrine therapy of patients was switched to AstraZeneca’s camizestrant from ongoing treatment with an aromatase inhibitor (AI), while continuing combination with the same cyclin-dependent kinase (CDK) 4/6 inhibitor. The trial demonstrated a highly statistically significant and clinically meaningful improvement in progression-free survival (PFS) in the 1st-line treatment of patients with hormone receptor (HR)-positive, HER2-negative advanced breast cancer whose tumors had an emergent ESR1 mutation as detected by Guardant360 CDx.

About Guardant360 CDx

The first FDA-approved blood test for complete genomic testing, Guardant360 CDx is approved as a companion diagnostic fur multiple therapies in non-small cell lung cancer. It is also the only FDA-approved companion diagnostic for targeted therapy in advanced breast cancer patients with ESR1 mutations. The test is broadly covered by Medicare and commercial insurers, representing over 300 million lives. For more information, visit the Guardant360 CDx website.

On June 2, 2025 BioNTech SE (Nasdaq: BNTX, "BioNTech") and Bristol Myers Squibb (NYSE: BMY, "BMS") reported that the companies have entered into an agreement for the global co-development and co-commercialization of BioNTech’s investigational bispecific antibody BNT327 across numerous solid tumor types (Press release, BioNTech, JUN 2, 2025, View Source [SID1234653596]). Under the agreement, BioNTech and BMS will work jointly to broaden and accelerate the development of this clinical candidate.

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BioNTech’s BNT327, a next-generation bispecific antibody candidate targeting PD-L1 and VEGF-A, is currently being evaluated in multiple ongoing trials with more than 1,000 patients treated to date, including global Phase 3 trials with registrational potential evaluating BNT327 as first-line treatment in extensive stage small cell lung cancer ("ES-SCLC") and non-small cell lung cancer ("NSCLC"). A global Phase 3 trial evaluating the candidate in triple negative breast cancer ("TNBC") is planned to start by the end of 2025. Preliminary data from ongoing trials underscore the potential for combining anti-PD-L1 and anti-VEGF-A – two well-established therapeutic targets – into a single molecule to deliver synergistic clinical benefits for patients across multiple tumor types.

Under the terms of the agreement, the companies will jointly develop and commercialize BNT327, including the development of BNT327 as monotherapy and in combination with other products. Both companies have the right to independently develop BNT327 in further indications and combinations, including combinations of BNT327 with proprietary pipeline assets.

"We believe BNT327 has the potential to become a foundational immuno-oncology backbone, moving beyond single-mechanism checkpoint inhibitors and expanding into multiple solid-tumor indications. Our collaboration with BMS, a pioneering leader in immuno-oncology, aims to accelerate and broadly expand BNT327’s development to fully realize its potential," said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech. "Our focus remains on advancing high-impact, pan-tumor programs and combination strategies in oncology, with BNT327 complementing our antibody-drug conjugate programs and mRNA-based immunotherapies. We are dedicated to delivering truly transformative options for patients in need."

"Our deep experience and expertise in advancing and delivering groundbreaking immuno-oncology medicines positions BMS well to collaboratively realize the potential of BNT327, an asset with significant potential for transforming the standard of care for patients with solid tumors," said Christopher Boerner, Ph.D., Board Chair and CEO of Bristol Myers Squibb. "The science behind BNT327 and its leading clinical position in multiple hard-to-treat tumor types, further bolsters our pursuit of novel mechanisms and multiple modalities in oncology, and enhances our growth trajectory. We are impressed by the innovation that BioNTech has achieved to date, and we look forward to partnering to accelerate existing clinical trials and time to market, while expanding the number of potential indications."

BMS will pay BioNTech $1.5 billion in an upfront payment and $2 billion total in non-contingent anniversary payments through 2028. These tax-deductible charges will be recorded as Acquired IPR&D Expense when incurred, with the $1.5 billion being incurred in Q2. In addition, BioNTech will be eligible to receive up to $7.6 billion in additional development, regulatory and commercial milestones. BioNTech and BMS will share joint development and manufacturing costs on a 50:50 basis, subject to certain exceptions. Global profits/losses will be equally shared between BioNTech and BMS.

About BNT327
BNT327 is a novel investigational bispecific antibody combining two complementary, validated mechanisms in oncology into one single molecule. BNT327 combines PD-L1 checkpoint inhibition aimed at restoring T cells’ ability to recognize and destroy tumor cells with the neutralization of VEGF-A. The blocking of VEGF-A is aimed at reversing the tumor’s immuno-suppressive effect in its microenvironment and cutting off the blood and oxygen supply that feeds tumor cells (anti-angiogenesis effect), with the intention of preventing the tumor from growing and proliferating. BNT327 may be differentiated via its mechanism of action of targeting PD-L1 on tumor cells to localize anti-VEGF activity within the tumor microenvironment, aiming to enhance therapeutic precision and minimize systemic exposure. A treatment with BNT327 is intended to help normalize blood vessels at the tumor site, improving delivery and potential effectiveness of combination therapies. This targeted vascular remodeling positions BNT327 as a potential backbone therapy across a wide range of solid tumors.1,2

More than 1,000 patients have been treated with BNT327 in clinical trials to date. More than 20 clinical trials are currently ongoing or planned to evaluate BNT327 either as a monotherapy or in combination with other treatment modalities targeting different oncogenic pathways in more than 10 solid tumor indications. Multiple global trials are ongoing or planned to start in 2025, including three global clinical trials with registrational potential in first-line small cell lung cancer ("SCLC"), first-line non-small cell lung cancer ("NSCLC") and first-line triple-negative breast cancer ("TNBC"). Additional trials will explore combining BNT327 and BioNTech’s proprietary antibody-drug conjugate candidates ("ADCs"). If successfully developed and approved, BioNTech aims to use this bispecific antibody candidate as a next-generation immuno-oncology ("IO") backbone in combination with other treatment modalities targeting a broad range of cancer indications.

On June 2, 2025 Sapience Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery and development of peptide therapeutics to address oncogenic and immune dysregulation that drive cancer, reported updated positive clinical and biomarker data from its Phase 2 clinical trial of lucicebtide (formerly known as ST101), a first-in-class antagonist of C/EBPβ, in patients with glioblastoma (GBM) (Press release, Sapience Therapeutics, JUN 2, 2025, View Source [SID1234653644]). The data were featured during an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 30-June 3, 2025, in Chicago and online.

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"GBM represents a devastating disease carrying a poor prognosis and high mortality rate, and there is a significant need for new, efficacious treatment options," said Fabio Iwamoto, MD, Division of Neuro-Oncology, New York-Presbyterian/Columbia University Irving Medical Center. "The growing body of data from this Phase 2 study, which demonstrate a marked progression free and overall survival benefit, continue to support that lucicebtide could serve as a well-tolerated, meaningful addition to the GBM treatment paradigm. I look forward to deepening our understanding of the potential for lucicebtide to improve outcomes and offer hope to patients for whom limited alternatives exist."

Sapience Chief Medical Officer, Dr. Abi Vainstein-Haras, added, "These updated results reinforce lucicebtide’s compelling clinical and safety profile, and support its potential to serve as a standalone therapy or in combination with existing treatments. The opportunity to collect biopsies in this patient population gave us a unique opportunity for biomarker analysis to demonstrate the meaningful effects of lucicebtide."

Oral Presentation Highlights Include:

Lucicebtide was well-tolerated as a monotherapy and in combination with standard-of-care (SOC) agents.
Lucicebtide combination with SOC Window-of-Opportunity study in newly diagnosed GBM cohort (n=9), data cut May 6, 2025:
5/9 patients yet to experience disease progression (10-24+ months)
6/9 patients are alive as of the data cutoff date (10-26+ months)
Lucicebtide Window-of-Opportunity study in recurrent GBM cohort (n=9), data cut May 6, 2025:
4/9 patients had disease control, with two partial responses
3/9 patients remain alive as of data cutoff
Window-of-Opportunity biomarker results demonstrate:
Lucicebtide crossed the blood-brain barrier, demonstrated uptake into the tumor and target engagement
Immune activation in the tumor microenvironment (TME) evidenced by increased macrophage M1/M2 ratio and CD8 T cell infiltration
Meaningful reductions in mesenchymal gene signature in tumor cells as shown by spacial transcriptomics analysis
Data support lucicebtide and C/EBPβ antagonism as a differentiated and promising therapeutic approach for patients with GBM.
A copy of the presentation is available under the Presentations section of the Sapience Therapeutics website.

About Lucicebtide (formerly known as ST101)

Lucicebtide, a first-in-class antagonist of C/EBPβ, has completed the main portion of a Phase 2 dose expansion study in recurrent glioblastoma (rGBM) (NCT04478279). An ongoing Window-of-Opportunity sub-study is evaluating lucicebtide in combination with radiation and temozolomide in patients with newly diagnosed GBM (ndGBM) and as a monotherapy in patients with rGBM, with patients receiving lucicebtide before and after surgical resection in both cohorts. Lucicebtide has been granted Fast Track designation for rGBM from the U.S. Food and Drug Administration (U.S. FDA) and orphan designations for glioma from the U.S. FDA and the European Commission.