FDA written feedback supports: - a single-arm, open-label registrational trial in 2nd-line refractory high-grade NMIBC with CIS - a randomized vs observation single trial in intermediate-risk NMIBC in the adjuvant setting Phase 3 program expected to initiate in 1H 2026 CORAL GABLES, Fla., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Relmada Therapeutics, Inc. (Nasdaq: RLMD, “Relmada” or the “Company”), a clinical-stage biotechnology company advancing innovative therapies for oncology and central nervous system disorde ...

Core Insights - Astellas Pharma and Pfizer announced positive topline results from the Phase 3 EV-304 clinical trial for PADCEV in combination with Keytruda, showing significant improvements in event-free survival and overall survival for muscle-invasive bladder cancer patients [1][5][7] Company Insights - Astellas Pharma is focused on advancing treatment options for muscle-invasive bladder cancer, with recent findings reinforcing the potential of PADCEV plus pembrolizumab to improve survival outcomes for a broad patient population [4][5] - Pfizer emphasizes the significance of the EV-304 results, indicating a potential shift in the standard of care for muscle-invasive bladder cancer patients, particularly those ineligible for platinum-based chemotherapy [5][6] Industry Insights - Muscle-invasive bladder cancer is a significant health concern, with nearly half of patients progressing to metastatic disease within three years of diagnosis, highlighting the need for effective treatment options [3][6] - The EV-304 trial results may lead to a new standard of care in bladder cancer treatment, moving away from conventional platinum-based chemotherapy [5][7]

Core Viewpoint - The European Medicines Agency (EMA) has recommended conditional marketing authorization for ImmunityBio's Anktiva in combination with BCG for treating BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) [1][4]. Group 1: Clinical Trial Results - A single-arm clinical trial involving 100 adults showed a complete response rate of 71%, with responses lasting up to 54+ months and an average duration of approximately 27 months [2]. - The complete response rates at 12 months and 24 months were 66% and 42%, respectively [3]. Group 2: Regulatory and Market Context - The conditional marketing authorization is part of an EU regulatory mechanism aimed at providing early access to medicines for unmet medical needs [4]. - Bladder cancer is the fifth-most common cancer and the seventh most frequently diagnosed cancer in men, with an estimated 200,000 new cases expected in 2025 [4]. - Approximately 75% of these patients (150,000) will have NMIBC, which is the most common form of bladder cancer [5]. Group 3: Next Steps and Market Reaction - ImmunityBio will continue to follow up with trial participants and submit long-term safety and efficacy results to the EMA post-marketing [3]. - Following the EMA's recommendation, the opinion will be forwarded to the European Commission for final approval of EU-wide marketing authorization [6]. - ImmunityBio's shares increased by 2.74% to $2.25 at the time of publication [6].

Core Insights - New data from the Phase 2b SunRISe-1 study indicate that over 95% of patients with high-risk, non-muscle invasive bladder cancer remained progression-free at one year, with more than 92% avoiding bladder removal surgery [1][2][3] Company Overview - Johnson & Johnson is committed to developing innovative treatments for patients with high-risk non-muscle invasive bladder cancer (NMIBC) who have limited options beyond radical cystectomy [2] - The company is focusing on the gemcitabine intravesical system, which has shown promising results in clinical trials for patients unresponsive to Bacillus Calmette-Guérin (BCG) therapy [1][5] Study Details - Cohort 4 of the SunRISe-1 study involved 52 patients with papillary-only, high-risk NMIBC who did not respond to BCG therapy and were ineligible for or declined radical cystectomy [2][5] - The therapy was administered every three weeks for six months, followed by every 12 weeks for up to an additional 18 months [2] Efficacy Results - At a median follow-up of 15.9 months, the one-year disease-free survival (DFS) rate was 74.3%, with progression-free survival (PFS) at 95.6% and overall survival (OS) at 98% [3] - Notably, 92.3% of patients did not undergo radical cystectomy, indicating the treatment's potential to preserve bladder function [3] Safety Profile - The therapy was generally well-tolerated, with 80.8% of patients experiencing low-grade treatment-related side effects, primarily mild urinary symptoms [4] - Serious side effects were uncommon, occurring in 13.5% of patients, and no treatment-related deaths were reported [4] Industry Context - High-risk non-muscle invasive bladder cancer represents a significant challenge in oncology, with traditional treatment options often leading to radical cystectomy, which can severely impact patients' quality of life [2][7] - The ongoing need for effective bladder preservation therapies highlights the importance of studies like SunRISe-1 in addressing unmet medical needs in this patient population [2][7]

Core Insights - The article expresses optimism about enGene Holdings Inc. (ENGN) in the context of bladder cancer research and investment potential [1] Company Analysis - enGene Holdings Inc. is focused on developing therapies for bladder cancer, indicating a specialized niche within the biotech sector [1] - The author has a PhD in biochemistry and extensive experience in analyzing clinical trials and biotech companies, which adds credibility to the insights provided [1] Investment Thesis - The investment thesis for enGene Holdings Inc. is based on its market valuation relative to its potential in the bladder cancer treatment space, suggesting that the company may be undervalued [1]

Core Viewpoint - Relmada Therapeutics has appointed Dr. Raj S. Pruthi as Chief Medical Officer-Urology to advance the development of NDV-01, a novel therapy for non-muscle invasive bladder cancer (NMIBC), with a Phase 3 trial expected to begin in the first half of 2026 [2][4]. Company Overview - Relmada Therapeutics is a clinical-stage biotechnology company focused on innovative therapies for oncology and central nervous system conditions [11]. - The lead program, NDV-01, is designed for the treatment of NMIBC and aims to address significant unmet medical needs in this area [11]. Appointment of Dr. Raj S. Pruthi - Dr. Pruthi brings over 25 years of experience in urologic oncology, clinical trials, and robotic surgery, having previously served as Chief Medical Officer at enGene Holdings Inc. and Global Medical Affairs leader at Johnson and Johnson [3][5]. - His expertise includes developing practice guidelines for NMIBC and executing global clinical studies for bladder cancer treatments [3][6]. NDV-01 Program - NDV-01 is a sustained-release formulation of gemcitabine and docetaxel, designed for intravesical administration, allowing for gradual drug release over 10 days [9]. - The formulation aims to enhance local exposure while minimizing systemic toxicity and is designed for in-office administration in under 10 minutes without the need for anesthesia [9]. - Positive initial Phase 2 data presented at the AUA 2025 indicated impressive response rates and favorable tolerability for NDV-01 [4][10]. Market Opportunity - NMIBC accounts for approximately 75% of all bladder cancer cases, with a high recurrence rate of 50-75% over seven years, indicating a significant market opportunity for effective treatments [10]. - With over 600,000 prevalent cases in the U.S. and limited treatment options, NDV-01 has the potential to serve as a frontline or salvage therapy across multiple NMIBC subtypes [10].

Core Insights - CG Oncology, Inc. announced positive results from the Phase 3 BOND-003 study of cretostimogene grenadenorepvec for high-risk non-muscle invasive bladder cancer (NMIBC) unresponsive to BCG treatment, presented at the 2025 American Urological Association Annual Meeting [1] Study Results - The study reported a 75.5% complete response (CR) rate at any time, with 34 confirmed CRs at 24 months and nine patients pending assessment as of March 14, 2025 [2] - The 12-month and 24-month CR rates were estimated at 50.7% and 42.3%, respectively, with a median duration of response (DOR) of 28 months [3] - 97.3% of patients were free from progression to muscle-invasive disease at 24 months [3] - In Cohort P, patients with BCG-unresponsive Ta/T1 disease without CIS showed an estimated 90.5% high-grade recurrence-free survival at 3 and 9 months [3] Safety Profile - A well-tolerated safety profile was observed, with no Grade 3 or greater treatment-related adverse events (TRAEs) or deaths reported among 110 highly pretreated patients [4] - Patients who experienced TRAEs had a median resolution time of one day, and 97.3% completed all expected treatments, indicating favorable adherence [4][5] Market Reaction - Following the announcement, CGON stock increased by 39.9%, reaching $31.30 [6]

Core Insights - The first results from Cohort 4 of the Phase 2b SunRISe-1 study indicate that TAR-200 shows strong disease-free survival rates and bladder preservation potential in patients with high-grade papillary bladder cancer [1][2][3] - Over 95% of patients demonstrated a progression-free survival rate at 9 months, highlighting the effectiveness of TAR-200 in a high-risk patient population [1][2] Company Overview - Janssen-Cilag International NV, a subsidiary of Johnson & Johnson, is focused on innovative treatments for bladder cancer, particularly targeting patients with BCG-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC) [1][5] - The company aims to provide alternatives to radical cystectomy, which has been the standard treatment for HR-NMIBC patients for over 40 years [1][4] Product Details - TAR-200 is an investigational intravesical gemcitabine releasing system designed for sustained local release of gemcitabine into the bladder, offering a new approach to treating early-stage bladder cancer [3][4] - The product is administered in an outpatient setting without the need for anesthesia, allowing for a more convenient treatment option for patients [2][3] Clinical Study Insights - The interim analysis from Cohort 4 of the SunRISe-1 study reported disease-free survival rates of 85.3% at six months and 81.1% at nine months for patients treated with TAR-200 monotherapy [1][2] - A significant 94.2% of patients avoided radical cystectomy at a median follow-up of 12.8 months, indicating the potential of TAR-200 to provide durable disease control [1][2] Safety Profile - The safety profile of TAR-200 was consistent with previous studies, with most treatment-related adverse events being low grade and resolving quickly [2][3] - Common treatment-related adverse events included dysuria (40.4%), pollakiuria (30.8%), and urinary urgency (26.9%), with no treatment-related deaths reported [2][3]

Core Insights - The Phase 2b SunRISe-1 study results indicate that over 82% of patients with high-risk non-muscle invasive bladder cancer achieved a complete response (CR), with more than half remaining cancer-free for at least one year after treatment [1][2][3] - TAR-200, an intravesical gemcitabine releasing system, shows promise in transforming treatment outcomes for patients who are BCG-unresponsive and ineligible for radical cystectomy [1][2][3] Study Results - As of March 2025, 82.4% of the 85 enrolled patients achieved CR, with a 95% confidence interval of 72.6-89.8 [1] - 52.9% of responders maintained CR at one year, and the median duration of response was 25.8 months [1] - At 12 months, 86.6% of responders remained cystectomy-free [1] Treatment Tolerability - The treatment was well-tolerated, with 83.5% of patients experiencing mild treatment-related adverse events, primarily low-grade urinary symptoms [1] - Only 3.5% of patients discontinued treatment due to adverse events, and there were no treatment-related deaths [1] Clinical Significance - The results were presented at the 2025 American Urological Association Annual Meeting, highlighting the potential of TAR-200 to address significant unmet needs in bladder cancer treatment [1][2] - The study emphasizes the importance of patient quality of life, as TAR-200 is designed to fit into patients' lives without interrupting their daily activities [1][2] Background on Bladder Cancer - Bladder cancer is among the ten most common cancers globally, with limited treatment options available for patients who do not respond to initial BCG therapy [1][4] - High-risk non-muscle invasive bladder cancer (HR-NMIBC) accounts for 15-44% of NMIBC cases and is characterized by a high likelihood of recurrence and progression [4] About TAR-200 - TAR-200 is an investigational system designed for sustained local release of gemcitabine into the bladder, currently being evaluated in multiple clinical trials [2][3] - The ongoing Phase 2b SunRISe-1 study specifically targets patients with BCG-unresponsive HR-NMIBC [3]

Core Insights - Johnson & Johnson announced promising 12-month data from the Phase 2b SunRISe-1 study, highlighting the effectiveness of TAR-200 monotherapy in treating high-risk non-muscle invasive bladder cancer (HR-NMIBC) [1][2] - The study results will be presented at the AUA 2025 Annual Meeting, emphasizing the potential of TAR-200 to provide a new treatment option for patients unresponsive to standard BCG therapy [1][3] Company Overview - TAR-200 is an investigational intravesical gemcitabine releasing system, with over 10,000 placements in clinical programs to date [5] - The FDA granted Breakthrough Therapy Designation to TAR-200 for treating adult patients with BCG-unresponsive HR-NMIBC, indicating its potential significance in the oncology pipeline [5] Industry Context - Bladder cancer is one of the top ten most common cancers globally, affecting nearly one million people annually, with limited treatment options available for those who do not respond to initial BCG therapy [2][7] - High-risk non-muscle invasive bladder cancer (HR-NMIBC) represents a significant challenge in urology, with radical cystectomy often being the standard treatment for patients who fail BCG therapy [7][8]