On April 27, 2025 Oncoinvent ASA, a clinical-stage radiopharmaceutical company developing innovative treatments for solid cancers, reported the 18-month follow-up results from its Phase 1 clinical trial (RAD-18-001) evaluating Radspherin in patients with platinum-sensitive recurrent ovarian cancer and peritoneal carcinomatosis (Press release, Oncoinvent, APR 27, 2025, https://www.oncoinvent.com/press-release/oncoinvent-reports-updated-18-month-data-from-phase-1-ovarian-cancer-trial-with-radspherin-no-new-recurrences-demonstrate-continued-promise-of-preventing-disease-progression/?utm_source=mailpoet&utm_medium=email&utm_source_platform=mailpoet [SID1234652202]). The trial was closed for recruitment at the end of 2023 and patients are currently in long-term follow-up.

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In this trial, 10 patients received a single intraperitoneal dose of 7 MBq Radspherin. Oncoinvent reported 12-month data in November 2024, where only 1 out of the 10 receiving the selected dose had peritoneal recurrence. At the 18-month follow-up, no further patient had experienced recurrence. i.e., only 10% of patients treated have so far had a recurrence. In similar populations, receiving best standard of care, it is expected that approximately 40% of patients would have had disease recurrence at this time point , , .

"Ovarian cancer’s characteristic peritoneal metastasis lead to frequent recurrences despite a comprehensive treatment approach," commented Dr. Luis Chiva, Principal Investigator and Director of Department of Obstetrics and Gynecology, Clinica Universidad de Navarra, Spain. "I am proud to be part of a study program exploring whether Radspherin may become a novel therapy that can prevent disease progression, offering hope for a better and longer life for my patients."

"These compelling results underscore Radspherin’s potential to significantly reduce peritoneal recurrence in ovarian cancer patients," said Øystein Soug, CEO of Oncoinvent. "The data not only reinforce our confidence in Radspherin but also support its advancement in Phase 2 trials, including the ongoing randomized controlled Phase 2 trial assessing progression-free survival in patients with peritoneal metastasis from ovarian cancer."

To discuss these promising clinical results, provide an update on operations, and discuss the ongoing Phase 2 trial, Oncoinvent has decided to move up its Quarterly Update from the previously announced May 27 to April 30 at 11:00 CEST.

On April 27, 2025 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics ("Molecular Partners" or the "Company"), reported the presentation of three posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, taking place April 25–30 in Chicago, IL (Press release, Molecular Partners, APR 27, 2025, View Source [SID1234652200]). The first poster includes positive Investigational New Drug (IND)-enabling data for MP0712, a Radio-DARPin targeting DLL3 and labeled with 212Pb for small cell lung cancer patients. The second poster presents initial preclinical data on the second 212Pb-based Radio-DARPin, targeting mesothelin (MSLN) in solid tumors – both programs are co-developed with Orano Med. The third poster includes additional preclinical proof-of-concept data on the logic-gated CD3 Switch-DARPin T cell engager with CD2 co-stimulation in solid tumors.

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"Our presentations at AACR (Free AACR Whitepaper) highlight the breadth of Molecular Partners’ DARPin innovation and the progress in our strategic Radio-DARPin partnership with Orano Med. Our first Radio-DARPin program, MP0712, targeting DLL3, is well advanced and on track to provide initial clinical data in the second half of 2025. Additionally, we are proud to present the first preclinical data from our second program with Orano Med, targeting MSLN. The results show substantial uptake into MSLN-positive tumors, with limited accumulation in other organs, justifying continued investment in our RDT x MSLN program for solid tumors," said Patrick Amstutz, Ph.D., CEO of Molecular Partners.

"Furthermore, we continue to advance our wholly-owned logic-gated and co-stimulated T cell engager program. Our SWITCH approach activates CD3/T-cells only when binding to tumor-associated antigens, while remaining inactive in circulation. This gating allows us to add a CD2 DARPin for co-stimulation without the risk of fratricide. This project demonstrates the value of our platform, which can be used for any target combination."

Preclinical data presented on MP0712, a DLL3-targeting and 212Pb-labeled Radio-DARPin, show a high tumor uptake and a favorable safety profile for MP0712, with good efficacy and tumor reduction in mouse models matching clinically relevant DLL3 expression levels. With these data, the IND-enabling package is complete; IND filing and initial first-in-human clinical data are expected in 2025. DLL3 is a promising target for radioligand therapy as it is highly upregulated in small cell lung cancer and other high-grade neuroendocrine tumors, while not expressed in healthy tissues.

The MSLN x 212Pb Radio-DARPin poster outlines how MSLN may be a promising target for ovarian cancer due to its differentiated expression profiles – high in tumor, and lower in healthy tissues. High levels of shed MSLN, however, can act as a decoy receptor and have historically hampered the development of MSLN-targeted therapeutics. Molecular Partners has leveraged the unique properties of DARPins to develop a Radio-DARPin able to selectively bind membrane-bound MSLN without being impacted by shed MSLN. In vivo results show a favorable biodistribution with strong tumor accumulation of the Radio-DARPin in a MSLN-overexpressing model in mice.

Preclinical proof-of-concept data on Molecular Partners’ conditionally activated CD3 Switch-DARPin shows it activates T cells specifically in the presence of cells co-expressing MSLN and epithelial cell adhesion molecule (EpCAM), increasing tumor specificity. Concurrent CD2 co-engagement leads to sustained T cell activation and cytotoxic capacity, preventing T cell dysfunction. The Switch-DARPin effectively induces significant tumor regression in mice engrafted subcutaneously with MSLN- and EpCAM-expressing cells, without signs of T cell activation in the periphery, indicating a favorable safety profile.

Click here to access the poster presentations, which will be made available on Molecular Partners’ website.

Details of the presentations:

MP0712, the first anti-DLL3 212Pb Radio-DARPin (RDT) candidate for targeted radiotherapy of small cell lung cancer (SCLC)
Session Category: Experimental and Molecular Therapeutics
Session Title: Biochemical Modulators of Cancer / Differentiation Therapeutic Strategies
Session Timing: Sunday April 27 at 2:00pm – 5:00pm CST
Location: Poster Section 16, Poster Board Number: 13
Published Abstract Number: 346

Development of 212Pb-based Radio-DARPin therapy (RDT) for the treatment of mesothelin (MSLN)-positive solid tumors
Session Category: Experimental and Molecular Therapeutics
Session Title: Biochemical Modulators of Cancer / Differentiation Therapeutic Strategies
Session Timing: Sunday April 27 at 2:00 – 5:00pm CST
Location: Poster Section 16, Poster Board Number: 6
Published Abstract Number: 339

Next-generation multi-specific and conditionally activated CD3 Switch-DARPins with CD2 co-stimulation to tackle the current limitations of T cell engagers in solid tumors
Session Category: Experimental and Molecular Therapeutics
Session Title: Therapeutic Approaches to Attack the Tumor Microenvironment
Session Timing: Monday April 28 at 2:00pm – 5:00pm CST
Location: Poster Section 24, Poster Board Number: 3
Published Abstract Number: 3119

On April 27, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, reported a duration of response of nearly four years from a long-term follow-up study with JELMYTO (mitomycin) for pyelocalyceal solution, which is FDA-approved for the treatment of low-grade upper tract urothelial cancer (LG-UTUC) in adult patients (Press release, UroGen Pharma, APR 27, 2025, View Source [SID1234652198]).

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Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41), the median duration of response was 47.8 months, irrespective of whether their cancer was new-onset or recurrent (median follow-up 28.1 months [95% CI 13.1, 57.5]). Of these patients, 21 had new-onset UTUC, and 20 had recurrent UTUC at baseline; there were no significant differences in durability between groups, with 8 patients in each group experiencing recurrence or death not due to treatment. Twenty patients entered long-term follow-up with a median follow-up of 53.3 months (95% CI 27.9, 65.3); median duration of response was not estimable (95% CI 43.5, not estimable) due to the low event rate.

"This sub-analysis highlights new evidence for the impressive long-term benefits of JELMYTO, in both new-onset and recurrent low-grade upper tract urothelial cancer patients," said Brian Hu, MD, Associate Professor of Urology at Loma Linda University Health, CA. "These findings further establish JELMYTO as an effective treatment, providing patients with durable responses and a significant reduction in recurrence rates."

LG-UTUC is a challenging disease often managed through endoscopically guided ablation, but recurrence is common, and patients face a lifetime of surveillance and potential complications. The OLYMPUS Phase 3 trial evaluated JELMYTO, a reverse thermal gel containing mitomycin (4 mg/mL), as a primary treatment for adults with LG-UTUC. While the overall trial demonstrated the potential of JELMYTO to significantly eradicate disease, this sub-analysis focuses specifically on patients with new-onset and recurrent LG-UTUC who achieved a complete response, providing new insights into the durability and long-term effectiveness of the treatment. Limitations of this study include the post-hoc nature of the analysis and potential selection bias regarding entry of patients into long-term follow-up.

"We are encouraged by the long-term outcomes evidence observed in both new-onset and recurrent LG-UTUC patients," said Mark Schoenberg, MD, Chief Medical Officer, UroGen. "The sub-analysis provides further compelling evidence that JELMYTO offers durable disease control and clinically meaningful responses, which could significantly reduce the need for repeated interventions. We look forward to expanding our understanding of JELMYTO through the ongoing uTRACT Registry and further evaluating its potential in real-world settings."

To further explore the potential of JELMYTO in treating patients with LG-UTUC, investigators are currently enrolling participants in the JELMYTO uTRACT Registry (NCT05874921) to gather longitudinal real-world usage data. As of April 2025, 22 sites have been activated with 251 patients enrolled.

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 April 27, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported patient-reported outcomes following treatment of patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) that showed investigational drug UGN-102 (mitomycin) for intravesical solution achieved robust and durable complete response (CR) rates without negatively impacting quality of life (Press release, UroGen Pharma, APR 27, 2025, View Source [SID1234652197]). The data (Moderated Poster – MP15) were presented at the AUA 2025 Annual Meeting in Las Vegas, Nevada.

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"Patient reported quality of life outcomes are critical in evaluating the usefulness of investigational drugs, particularly when delivered in the bladder which frequently causes urinary symptoms," said Charles Peyton, M.D., Assistant Professor, University of Alabama, Department of Urology, Heersink School of Medicine and study investigator. "Aggregated quality of life data across three robust clinical trials suggests that UGN-102 is quite tolerable without negatively impacting symptom burden, patient function or quality of life compared to baseline. UGN-102, if approved, is a promising alternative intravesical treatment for patients with LG-IR-NMIBC."

In the OPTIMA II, ATLAS, and ENVISION late-phase studies, most patients (≥91% in ATLAS, ≥94% in ENVISION) completed the questionnaires at baseline, three months, and 12 months or study end. Baseline scores indicated high levels of functioning and low symptom burden prior to treatment. UGN-102 did not cause sustained declines in functioning or symptom burden, and no measured domains or items exceeded the threshold for clinically significant worsening at three or 12 months, suggesting no negative impact on quality of life.

"The patient-reported outcomes data showed that UGN-102 did not adversely impact quality of life while achieving high response rates in LG-IR-NMIBC patients," said Mark Schoenberg M.D., Chief Medical Officer, UroGen. "We’re pleased to hear directly from patients, as their experiences highlight the importance of advancing treatment options that have the potential to reduce the burden of this challenging disease. These findings reinforce our commitment to developing solutions that make a meaningful difference in patients’ lives."

In the Phase 2b OPTIMA II and Phase 3 ENVISION studies, patients received UGN-102, while in the Phase 3 ATLAS trial, patients were randomized to UGN-102 or trans-urethral resection of bladder tumor (TURBT); this analysis includes only data from UGN-102-treated patients. Each study featured a six-week UGN-102 treatment period, a CR assessment at three months, and follow-up ranging from nine to 63 months after three-month CR. Patient-reported symptoms and health status were evaluated using the EORTC-QLQ-NMIBC24 at baseline, three months, and 12 months or study end. Scores, ranging from one (not at all) to four (very much), were transformed to a 0-100 scale, with higher scores indicating greater symptom burden. Descriptive statistics were used to summarize baseline scores and changes, with positive changes reflecting symptom worsening.

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)

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 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 ENVISION

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as a chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

On April 27, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported new data from the OPTIMA II Phase 2b study of UGN‑102 (mitomycin) for intravesical solution demonstrate clinically meaningful two-year duration of response (24.2 months) by Kaplan-Meier analysis (Press release, UroGen Pharma, APR 27, 2025, View Source [SID1234652196]). UGN-102 is UroGen’s sustained-release formulation of mitomycin being developed for the treatment of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

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"The median duration of response of two years highlights UGN-102’s durability, even in patients with recurrent disease and multiple prior TURBT procedures," said Neal Shore, MD, Medical Director, Carolina Urologic Research Center, Myrtle Beach, South Carolina. "These long-term data provide encouraging evidence of UGN-102’s sustained impact."

The majority of patients included in OPTIMA II had recurrent disease at baseline (77.8%), with multiple prior transurethral resection of bladder tumor (TURBT) procedures. Among the 41 patients achieving a complete response (CR) at three months, 25 remained in CR at 12 months, and 17 of these patients entered long-term follow-up. The median Kaplan–Meier estimate of duration of response for the 41 patients that achieved CR was 24.2 months (95% CI 9.72, 47.18), with a median follow-up time of 33.6 months (95% CI 10.78, 42.94). Twenty patients (48.8%) experienced recurrence of low-grade disease. One patient progressed to high-grade disease and one patient died due to a cardiac disorder. Five patients remained disease-free at the time of the four-year data analysis.

"As the burden of LG-IR-NMIBC persists, the need for long-lasting treatment options becomes increasingly urgent," said Mark Schoenberg, Chief Medical Officer, UroGen. "The growing body of evidence supporting UGN-102 underscores its potential to address this unmet need. These results emphasize UGN-102’s potential to deliver meaningful and sustained responses, offering hope to patients who have long struggled with recurrence and limited treatment options."

UroGen completed the submission of the UGN-102 rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 with a Prescription Drug User Free Act (PDUFA) goal date of June 13, 2025.

The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. The TEAEs were typically mild-to-moderate in severity and either resolved or were resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN‑102.

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 NDA for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.

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 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 OPTIMA II

OPTIMA II (OPTimized Instillation of Mitomycin for Bladder Cancer Treatment) was an open-label, single-arm, multi-center Phase 2b clinical trial of investigational drug UGN-102 to evaluate its safety and efficacy in patients with LG-IR-NMIBC.

Learn more about the Phase 2b OPTIMA II trial at www.clinicaltrials.gov (NCT03558503).