On April 28, 2025 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for precision oncology, reported new interim analysis results of the VICTORI study showing strong performance of its ultra-sensitive NeXT Personal assay in detecting early signs of residual or recurrent colorectal cancer (CRC) (Press release, Personalis, APR 28, 2025, View Source [SID1234652279]).

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The study, led by Dr. Jonathan Loree’s team at BC Cancer in Vancouver, Canada, utilized NeXT Personal to look for small traces of circulating tumor DNA (ctDNA) in blood samples from a cohort of 71 patients with resectable Stage I-IV CRC. The data was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Chicago, Illinois by Emma Titmuss at BC Cancer, in an oral presentation titled Detection of post-surgical minimal residual disease (MRD) in colorectal cancer; preliminary results from the VICTORI study.

"After surgery, ctDNA-based liquid biopsies may help identify patients who would benefit most from additional treatment," said Jonathan Loree, MD, MS, a medical oncologist at BC Cancer and the senior investigator of the study. "Alternatively, this may help patients with good prognosis avoid toxicities from unnecessary chemotherapy. By monitoring patients for recurrences, liquid biopsies can continue to support clinical care and allow more patients to undergo second curative intent surgeries to remove early recurrences."

Key findings presented from the interim analysis for VICTORI:

100% of patients who have recurred were detected as ctDNA positive by NeXT Personal prior to detection on imaging.
100% of patients who have been ctDNA negative throughout the study remain disease-free.
87% of clinical recurrences were detectable within the early "landmark window" 2 to 8 weeks after surgery, with 85% detectable by 4 weeks.
64% of detections within the landmark window were in the ultrasensitive range (under 100 ppm).
100% of distant metastatic recurrences were detected prior to imaging, including lung metastasis, an area which has traditionally been more challenging to detect by ctDNA testing.
70% of the first ctDNA detections were in the ultrasensitive range, with a median of 24.4PPM and as low as 2.45 PPM.
Median patient follow-up at the time of the interim analysis was 15.75 months, with the patients continuing to be followed clinically.

"The initial results from our study show the importance of using a highly sensitive MRD assay like NeXT Personal in colorectal cancer," said Dr. Loree.

"We are encouraged by the preliminary results from the VICTORI study, which show the ability of our ultrasensitive ctDNA assay NeXT Personal to detect residual and recurrent colorectal cancer at high rates in the early landmark window after surgery," said Dr. Richard Chen, Chief Medical Officer and Executive Vice President, R&D at Personalis. "We continue to expand this prospective study as we strive towards helping patients with colorectal cancer detect and treat recurrence earlier."

On April 27, 2025 Zoldonrasib, a RAS(ON) G12D-selective inhibitor, demonstrated acceptable tolerability and encouraging initial antitumor activity in patients with previously treated KRAS G12D mutant non-small cell lung cancer
REDWOOD CITY, Calif., April 27, 2025 (GLOBE NEWSWIRE) — Revolution Medicines, Inc. (Nasdaq: RVMD), a late-stage clinical oncology company developing targeted therapies for patients with RAS-addicted cancers, reported new clinical data for zoldonrasib (RMC-9805), a RAS(ON) G12D-selective inhibitor, as monotherapy in patients with KRAS G12D mutant non-small cell lung cancer (NSCLC) (Press release, Revolution Medicines, APR 27, 2025, View Source [SID1234652192]). Results were highlighted in the official press program at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Chicago, Illinois, and will be featured in a late-breaking oral presentation on April 27, 2025, at 5:00 p.m. Central Time.

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"We are pleased to share new clinical data for zoldonrasib, our innovative, oral RAS(ON) G12D-selective inhibitor, which demonstrates acceptable safety and tolerability and encouraging initial antitumor activity in patients with non-small cell lung cancer. These data reinforce the clinical potential of zoldonrasib following the initial tolerability and antitumor activity reported late last year in patients with pancreatic ductal adenocarcinoma," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines. "Together these results support further evaluation of zoldonrasib as monotherapy and in combination as we continue efforts to advance innovative targeted medicines for patients living with these hard-to-treat cancers."

RMC-9805-001 is a multicenter, open-label, dose escalation and dose-expansion Phase 1 study designed to evaluate zoldonrasib in patients with advanced solid tumors harboring a KRAS G12D mutation.

As of a December 2, 2024 data cutoff date, 90 solid tumor patients were treated with 1200 mg once daily (QD), the candidate recommended Phase 2 dose. In these patients, zoldonrasib demonstrated an acceptable safety profile, that was generally consistent with previously reported data for this compound in pancreatic cancer, and was generally well tolerated. The most common treatment-related adverse events (TRAEs) occurring in at least 10% of patients were nausea (39%), diarrhea (24%), vomiting (18%), and rash (12%). TRAEs were primarily Grade 1 or 2 in severity, with two patients (2%) experiencing Grade 3 events that resolved following dose interruption. Zoldonrasib had a favorable mean dose intensity of 98% and no dose limiting toxicities were observed.

Preliminary antitumor activity was assessed in 18 efficacy-evaluable patients with NSCLC at the 1200 mg QD dose. The objective response rate (confirmed or pending confirmation) was 61% (n=11) and the disease control rate was 89% (n=16).

"There is a high unmet need for new treatments within this patient population as there are currently no targeted therapies approved for any RAS G12D mutant cancer," said Kathryn Arbour, M.D., thoracic medical oncologist at Memorial Sloan Kettering Cancer Center and principal investigator and lead author for the RMC-9805-001 presentation. "While the data are from an early, small subset of patients, it is encouraging to see this level of tolerability and antitumor activity in patients with NSCLC carrying this RAS mutation."

On April 27, 2025 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported that the Company anticipates the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will adopt an opinion on the marketing authorization application (MAA) for nirogacestat, an oral gamma secretase inhibitor, for the treatment of adults with desmoid tumors in the second quarter of 2025 (Press release, SpringWorks Therapeutics, APR 27, 2025, View Source [SID1234652193]).

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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).

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).