Latest news with #CTLA-4
Time of India
22-07-2025
- Health
- Time of India
Glioblastoma breakthrough: 40-year-old dad is brain cancer-free after groundbreaking immunotherapy trial
In a surprising and hopeful turn of events, a 40-year-old father is living his best life in his second innings, after he was diagnosed with terminal glioblastoma in 2023. In fact, three years later, he is not only healthy and thriving, but his brain cancer has completely 'vanished'! How? Read on to know more. What happened? In October 2022, 40-year-old Ben Trotman was diagnosed with terminal glioblastoma, the most common and deadliest form of brain cancer that typically carries a grim prognosis with a median survival of just 12–18 months. Yet, over two years after receiving a single dose of ipilimumab before standard care, Trotman reportedly remains cancer-free and healthy as ever. Under the care of consultant oncologist Dr. Paul Mulholland at UCLH (University College London Hospital), Trotman became part of an NHS-funded trial that administers ipilimumab, a checkpoint inhibitor designed to boost the immune system, before traditional surgery, radiotherapy, and chemotherapy. Two years later, his scans show no signs of recurrence, an unprecedented outcome in glioblastoma treatment. The medical marvel The new clinical trial for the most deadly type of brain cancer, glioblastoma, which helped Ben with another shot at life, is looking for patients to join after an incredible success story. Organized by the NHS, this study aims to enroll 16 people over 18 months and is dedicated to Baroness Margaret McDonagh, who lost her battle with the disease. by Taboola by Taboola Sponsored Links Sponsored Links Promoted Links Promoted Links You May Like Villas For Sale in Dubai Might Surprise You Dubai villas | search ads Get Deals Undo Patients diagnosed with glioblastoma will receive an immunotherapy drug called ipilimumab before starting standard treatments, making the most of their immune systems when they are at their strongest. The trial is being led by experts at University College London Hospital's National Hospital for Neurology and Neurosurgery. It follows a previous study with the same drug, which had to close because not enough patients joined. The trial honors the late Baroness Margaret McDonagh's memory and, like Trotman, participants receive ipilimumab to prime their immune defenses, with hopes of replicating his reassuring response. Ipilimumab: The key behind the breakthrough Ipilimumab is a monoclonal antibody that blocks CTLA-4, an immune checkpoint. In cancers like melanoma, it enhances T-cell activity, enabling the body to attack tumors. When used before standard glioblastoma therapy, it primes the immune system to better target cancer cells. In Trotman's case, it appears to have either eradicated or dramatically controlled his tumor, an outcome never before reported in this context. This 'window-of-opportunity' pre-treatment approach is groundbreaking. By delivering immunotherapy before surgery, it maximizes immune activation and potentially aids post-operative cancer control, a novel strategy in glioblastoma care. What is glioblastoma? Glioblastoma, previously known as glioblastoma multiforme or GBM, is the most common and aggressive type of primary brain cancer in adults. Glioblastoma originates from astrocytes, a type of glial cell that supports nerve cells in the brain and spinal cord. Glioblastoma is classified as a Grade IV tumor, meaning it's highly aggressive, grows rapidly, and infiltrates surrounding brain tissue. The exact cause of most glioblastomas is unknown. However, it is understood that the disease develops when brain cells undergo DNA mutations that cause them to grow and multiply uncontrollably. The prognosis for glioblastoma is generally poor, with a median survival of about 12-18 months after diagnosis. The five-year survival rate is around 5%, meaning about 5% of patients are still alive five years after their diagnosis. Glioblastoma almost always recurs despite maximal treatment. Hence, Trotman's cancer-free scans more than two years post-treatment represent a hopeful breakthrough. It suggests that bolstering immune readiness before conventional treatment might overcome glioblastoma's defenses. The success story The success story comes from Ben Trotman, who was diagnosed in October 2022 at the age of 40. Now, at 43, two years and eight months after his treatment, his scans are clear, and there are no traces of the tumor. Dr. Paul Mulholland, the consultant medical oncologist who is leading this new trial and treated Ben, as reported by The Independent, shared, 'It is very unusual to have a clear scan with glioblastoma, especially when he didn't have the follow-up surgery that had been planned to remove all of the tumor that was initially visible on scans. We hope that the immunotherapy and follow-up treatment Ben has had will hold his tumor at bay – and it has so far, which we are delighted to see. ' Ben's life has changed significantly since his diagnosis; he got married to Emily just two months after receiving the immunotherapy treatment, and in April, they welcomed their daughter, Mabel. Emily expressed the emotional toll of Ben's diagnosis, saying, 'Getting this diagnosis was the most traumatic experience. We were grappling with the fact that Ben had gone from being apparently perfectly healthy to having months to live. Had we not met Dr. Mulholland, that would have been it for us. We felt we had a lucky break in an otherwise devastating situation.' After the immunotherapy, Ben went on to have the standard treatments of radiotherapy and chemotherapy. He continues to have quarterly scans, which remain clear. Ben reflected on their journey, stating, 'We obviously don't know what the future holds, but having had the immunotherapy treatment and getting these encouraging scan results has given Emily and me a bit of hope. We are focused on rebuilding the life we thought we had lost and enjoying being parents. ' Dame Siobhain McDonagh MP led a successful fundraising campaign that gathered over £1 million to support this new trial. Her sister, Baroness McDonagh, sadly passed away from glioblastoma in 2023. Dame Siobhain shared, 'My beloved sister Margaret was appalled to discover that there had been no advances in brain cancer treatment for decades when she was diagnosed with glioblastoma. Changing this was Margaret's final campaign and one that I have continued in her memory. I am so grateful to the many people who knew and respected Margaret, who have come together and helped to raise funds and campaign for this new trial that we are calling Margaret's Trial.' Dr. Mulholland is also thankful for Margaret and Siobhain's dedication, stating, 'When I met Margaret, she said to me, 'What can I do to support you to cure this disease?'. I am incredibly grateful to her and to Siobhain, whose campaigning and fundraising in her sister's memory have led to this new clinical trial opening for patients with this most aggressive form of brain cancer that has such a poor prognosis, with most patients surviving just nine months after diagnosis. The crucial element of this trial is that patients will have their immune system boosted by the drug before they have any other treatment, when they are fit and well enough to tolerate the immunotherapy. We're taking everything we have learned from previous trials into this new study, and we are already planning follow-on trials. My aim is to find a cure for glioblastoma.' What's ahead? Led by Dr. Mulholland, the trial is expanding enrollment, aiming to confirm both safety and effectiveness. Currently, the National Brain Appeal is funding two positions to support Dr. Mulholland's vital research. The treatment will take place at the NIHR UCLH's Clinical Research Facility and the National Hospital for Neurology and Neurosurgery. Patients who are interested in participating in the new Win-Glio trial should discuss it with their consultant.
Yahoo
06-05-2025
- Business
- Yahoo
CStone Presents the Latest Preclinical Findings of CS2009 (PD-1/VEGF/CTLA-4 trispecific antibody) at 2025 AACR
SUZHOU, China, May 5, 2025 /PRNewswire/ -- CStone Pharmaceuticals ("CStone", HKEX: 2616), an innovation-driven biopharmaceutical company focused on anti-cancer therapies, announced today that a poster presentation of preclinical data of CS2009 (PD-1/VEGF/CTLA-4 trispecific antibody), a key asset in CStone Pipeline 2.0, has been delivered at the 2025 American Association for Cancer Research (AACR) Annual Meeting. CS2009 global phase I trial is ongoing in Australia with first patient dosed in this March and will soon be expanded to China and the US. PD-1, VEGFA, and CTLA-4 are clinically validated targets for immunotherapies, which can exert complementary, multi-dimensional anti-tumor effects through synergistic mechanisms. Blockade of PD-1 and CTLA-4 has demonstrated synergistic effects, extending overall survival benefits as well as progress-free survival benefits in many tumor types including NSCLC, which could be further strengthened by the blockade of VEGFA signaling. By integrating these three mechanisms of actions in one molecule, CS2009 holds great potential to deliver superior clinical benefits over PD-(L)1 antibodies or PD-(L)1/VEGF and PD-(L)1/CTLA4 bispecific antibodies. CS2009 is a promising trispecific antibody targeting PD-1, VEGFA, and CTLA-4, which demonstrates broad clinical application prospects for solid tumors with the great potential to become the next-generation immune-oncology backbone to replace current anti-PD-(L)1-based therapies. Key Highlights: CS2009 exhibited enhanced affinity upon simultaneous PD-1/CTLA-4 engagement. CS2009 enhances anti-tumor efficacy by preferentially targeting PD-1/CTLA-4 double positive T cells in TME. CS2009 exhibited an approximately 150-fold enhancement in checkpoint inhibitory activity on PD-1/CTLA-4 dual-reporter assay through crosslinking with VEGFA dimers. In PD-1 reporter assay, the CS2009/VEGFA combination demonstrated approximately 300-fold greater immune checkpoint activity compared to CS2009 alone. Mixed lymphocyte reaction (MLR) assays evaluating primary T cell activation revealed that crosslinking with VEGFA dimer significantly increases the activity of CS2009. These findings indicate enhanced activity of CS2009 in VEGFA-enriched TME and minimized peripheral overactivation-induced immune-related toxicity, thereby expanding its therapeutic window. CS2009 demonstrated dose-dependent T-cell activation during GLP-toxicity study in cynomolgus monkeys. CS2009 exhibited comparable pharmacokinetic (PK) profiles to those of monoclonal antibodies. CS2009 demonstrates superb tolerability with the highest non-severely toxic dose (HNSTD)/ the no observed adverse effect level (NOAEL) identified as 100 mg/kg. In summary, CS2009 is a trispecific antibody targeting PD-1, VEGFA, and CTLA-4, with the potential to be first- or best-in-class for major tumor types. Its differentiated molecular design combines three clinically validated targets, preferentially invigorating exhausted tumor infiltrating lymphocytes (TILs) while demonstrating VEGF neutralization comparable to existing anti-VEGF antibodies. CS2009 covers a wide range of cancers, including but not limited to non-small cell lung cancer, hepatocellular carcinoma, gastric adenocarcinoma, endometrial cancer, ovarian cancer, renal cell carcinoma, and cervical cancer. Our results show that CS2009 exhibits superior anti-tumor activity compared to potential competitors, including PD-(L)1/CTLA-4 bispecific antibodies, PD-(L)1/VEGF bispecific antibodies, and PD-(L)1/CTLA-4 combination therapies. Poster Information: Title: CS2009: A first-in-class trispecific antibody targeting PD-1, CTLA-4, and VEGFA with potential to be a next-generation backbone therapy with combined checkpoint inhibition and anti-angiogenesisSession Title: Overcoming Checkpoint Inhibition and Tumor SuppressionAbstract Number: 7299Date & Time: Wednesday, April 30, 2025, 9:00 AM - 12:00 PM ETLocation: Poster Section 39, Board #14 About CStone CStone (HKEX: 2616), established in late 2015, is an innovation-driven biopharmaceutical company focused on the research and development of anti-cancer therapies. Dedicated to addressing patients' unmet medical needs in China and globally, the Company has made significant strides since its inception. To date, the Company has successfully launched 4 innovative drugs and secured approvals for 16 new drug applications covering 9 indications. The company's pipeline is balanced by 16 promising candidates, featuring potentially first-in-class or best-in-class antibody-drug conjugates (ADCs), multispecific antibodies, immunotherapies and precision medicines. CStone also prides itself on a management team with comprehensive experiences and capabilities that span the entire drug development spectrum, from preclinical and translational research to clinical development, drug manufacturing, business development, and commercialization. For more information about CStone, please visit Forward-looking statements The forward-looking statements made in this article only relate to events or information as of the date when the statements are made in this article. Except as required by law, we undertake no obligation to update or publicly revise any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. All statements in this article are made on the date of publication of this article and may change due to future developments. Disclaimer: only for communication and scientific use by medical and health professionals, it is not intended for promotional purposes. View original content: SOURCE CStone Pharmaceuticals Sign in to access your portfolio
Yahoo
30-04-2025
- Health
- Yahoo
Immuno-Oncology Results from Research Collaboration with Léon Bérard Cancer Center Presented at AACR Annual Meeting 2025
Immuno-Oncology Results from Research Collaboration with Léon Bérard Cancer Center Presented at AACR Annual Meeting 2025 New gene expression signature for cancer patients to predict clinical response to immune checkpoint. Potential of signature to enhance personalized treatment for patients, regardless of cancer type. NANTES, France – April 30, 2025, 7:30 a.m. CET - OSE Immunotherapeutics SA (ISIN: FR0012127173; Mnemo: OSE), a biotech company dedicated to developing first-in-class therapies in immuno-oncology and immuno-inflammation, today reported on an oral presentation from a research collaboration with the renowned Léon Bérard Cancer Center in Lyon at the American Association for Cancer Research (AACR) Annual Meeting held April 25 – 30, 2025, in Chicago. Immune checkpoint blockade (ICB) therapies, targeting PD-1, PD-L1, and CTLA-4, have revolutionized cancer treatment by helping the immune system recognize and attack cancer cells more effectively. These therapies offer lasting responses and significant survival benefits across various cancers. However, current biomarkers like PD-L1 expression and tumor mutational burden (TMB) are not always reliable. PD-L1 expression can vary within tumors and between patients, making predictions inconsistent. Therefore, more universally applicable biomarkers are needed to predict patient responses and guide treatment decisions. While RNA sequencing has created detailed gene expression signatures (GES) that describe the tumor environment, their use in everyday clinical practice is limited due to their specificity and lack of widespread adoption. The presentation, entitled ' reported on the development and validation of a robust and tumor-agnostic composite gene expression signature (cGES) that can predict overall survival and progression free survival in cancer patients treated with immune checkpoint blockers (e.g. Anti PD-L1, Anti PD-1 and Anti CTLA-4). This signature could be useful in clinical practice to better stratify patients on risk and potentially guide treatment decisions of multiple cancer types. Professor Pierre Saintigny of Université Claude Bernard Lyon 1, Centre Léon Bérard Comprehensive Cancer Center and Team leader Inserm/Inria at Cancer Research Centre of Lyon stated: 'Our collaborative research program validated a composite gene expression signature with a strong potential for stratifying and predicting clinical outcomes more accurately, which could significantly improve personalized treatment in clinical practice for ICB-treated patients, regardless of cancer type. We were very pleased to present the results of our translational research at the AACR conference on this cutting-edge program conducted in collaboration with OSE Immunotherapeutics' teams.' ABOUT THE LÉON BÉRARD CENTERThe Centre Léon Bérard (CLB) is part of the twenty French Comprehensive Cancer Centres in France, providing a global management of cancer patients on a unique area, from diagnosis to treatment and beyond. The Centre is a regional, national and international recognized reference cancer Centre assigned with three essential missions: Care, Research and Education and is willing to continuously improve the quality and accessibility of care for cancer patients. ABOUT OSE IMMUNOTHERAPEUTICS OSE Immunotherapeutics is a biotech company dedicated to developing first-in-class assets in immuno-oncology (IO) and immuno-inflammation (I&I) that address the unmet patient needs of today and tomorrow. We partner with leading academic institutions and biopharmaceutical companies in our efforts to develop and bring to the market transformative medicines for people with serious diseases. OSE Immunotherapeutics is based between Nantes and Paris and is quoted on Euronext. Additional information about OSE Immunotherapeutics assets is available on the Company's website: Follow us on LinkedIn. Contacts Fiona Dé French Media Contact FP2COMFlorence Portejoiefportejoie@ 6 07 768 283 U.S. Media ContactRooney Partners LLCKate Barrettekbarrette@ 212 223 0561 Forward-looking statementsThis press release contains express or implied information and statements that might be deemed forward-looking information and statements in respect of OSE Immunotherapeutics. They do not constitute historical facts. These information and statements include financial projections that are based upon certain assumptions and assessments made by OSE Immunotherapeutics' management considering its experience and its perception of historical trends, current economic and industry conditions, expected future developments and other factors they believe to be appropriate. These forward-looking statements include statements typically using conditional and containing verbs such as 'expect', 'anticipate', 'believe', 'target', 'plan', or 'estimate', their declensions and conjugations and words of similar import. Although the OSE Immunotherapeutics management believes that the forward-looking statements and information are reasonable, the OSE Immunotherapeutics' shareholders and other investors are cautioned that the completion of such expectations is by nature subject to various risks, known or not, and uncertainties which are difficult to predict and generally beyond the control of OSE Immunotherapeutics. These risks could cause actual results and developments to differ materially from those expressed in or implied or projected by the forward-looking statements. These risks include those discussed or identified in the public filings made by OSE Immunotherapeutics with the AMF. Such forward-looking statements are not guarantees of future performance. This press release includes only summary information and should be read with the OSE Immunotherapeutics Universal Registration Document filed with the AMF on April 30, 2024, including the annual financial report for the fiscal year 2023, available on the OSE Immunotherapeutics' website. Other than as required by applicable law, OSE Immunotherapeutics issues this press release at the date hereof and does not undertake any obligation to update or revise the forward-looking information or statements. Attachment EN_250430_AACR_Oral presentationSign in to access your portfolio
Associated Press
26-04-2025
- Business
- Associated Press
UroGen Pharma Announces Encouraging Results from a Phase 1 Dose-Escalation Study Evaluating UGN-301 in Non-Muscle Invasive Bladder Cancer
PRINCETON, N.J.--(BUSINESS WIRE)--Apr 26, 2025-- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced encouraging safety data from the Phase 1 dose-escalation study for UGN-301 (zalifrelimab) intravesical solution, an investigational drug in development for the treatment of recurrent non-muscle invasive bladder cancer (NMIBC). 'The early safety profile and clinical activity results from this study are encouraging,' said Jay Raman, M.D., Professor and Chair of Urology, and Professor of Surgery, Penn State Cancer Institute, PA. 'This innovative approach of localized drug delivery combined with immune modulation merits additional investigation in the treatment of non-muscle invasive bladder cancer.' The multi-part clinical study included up to 30 patients per arm, aimed to assess safety and determine the recommended Phase 2 dose of UGN-301 as monotherapy and in combination with other agents. In the monotherapy arm, dose escalation continued to the maximum feasible dose. No dose-limiting toxicities and no treatment-emergent adverse events leading to treatment discontinuation were observed. This study also demonstrated that local delivery of UGN-301 formulated in our proprietary reverse thermal gel ( RTGel® ) allowed sustained exposure of zalifrelimab in the bladder with limited systemic exposure, which mitigated the risk of systemic immune-related toxicities associated with CTLA-4 inhibition. With respect to clinical activity observed in the trial, among evaluable patients who received UGN-301, 46% (6 of 13) of those with Ta/T1 disease and 33% (2 of 6) of those with carcinoma in situ (CIS) ± Ta/T1 disease were recurrence-free or had achieved a complete response at week 12. Notably, 60% (3 of 5) of patients with Ta/T1 disease treated with 300 mg continued to remain recurrence-free at the 15-month disease assessment, including one patient with high-grade T1 disease. In the 500 mg cohort, 25% (1 of 4) of patients with CIS disease and 33% (1 of 3) of patients with Ta/T1 disease remained disease-free at six months, both of whom are still active participants in the study. These findings highlight the potential of UGN-301 as a targeted treatment for NMIBC with an acceptable safety profile. Presentation of data from the combination arms is planned for later this year. 'Our hypothesis is that UGN-301's unique formulation could potentially offer the dual benefits of maximizing therapeutic activity while minimizing systemic side effects, a key challenge in cancer immunotherapy,' said Mark Schoenberg, Chief Medical Officer, UroGen. 'Although this requires additional clinical investigation, we are encouraged by the potential of UGN-301 as an investigational treatment for patients with recurrent NMIBC.' About Non-Muscle Invasive Bladder Cancer and High-Grade Disease In the U.S., bladder cancer is the second most common urologic cancer in men. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. High-grade non-muscle invasive bladder cancer (HG-NMIBC) is a serious and potentially life-threatening form of bladder cancer that remains confined to the inner layers of the bladder wall but exhibits aggressive behavior and a higher risk of progression. In the U.S., HG-NMIBC accounts for approximately 30–40% of all newly diagnosed NMIBC cases. Patients with HG-NMIBC face a significantly elevated risk of recurrence and progression to muscle-invasive disease, necessitating close surveillance and aggressive treatment. The standard of care includes complete transurethral resection of bladder tumor, often followed by intravesical therapy such as Bacillus Calmette-Guérin (BCG). However, BCG has a treatment failure rate of approximately 40-50%, leaving patients with limited treatment options short of radical cystectomy. Given the high recurrence and progression rates, HG-NMIBC presents a substantial clinical and quality-of-life burden. Upon recurrence, which occurs in approximately 70% of patients, the patients undergo another round of BCG therapy with a response rate of approximately 30%. About UGN-301 UGN-301 is an anti-CTLA-4 monoclonal antibody (zalifrelimab), originally licensed from Agenus Inc. in 2019. It is formulated with RTGel, our proprietary 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 in a multi-arm Phase 1 study of UGN-301 as monotherapy and in combination with other agents. The safety of UGN-301 is being evaluated in the monotherapy arm of the study as combination therapy for HG-NMIBC. About UroGen Pharma Ltd. UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen's sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with low-grade intermediate risk NMIBC are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit to learn more or follow us on X (Twitter), @UroGenPharma. Forward-Looking Statements This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: the potential benefits of UGN-301, including as a targeted treatment for NMIBC; implications that may follow from the early clinical activity and safety profile observations; UGN-301's innovative approach of localized drug delivery combined with immune modulation meriting additional investigation in the treatment of NMIBC; the potential for UGN-301 to maximize therapeutic activity and minimize systemic side effects for patients with recurrent NMIBC; the potential benefits of the intravesical delivery of UGN-301 using UroGen's RTGel proprietary technology; statements regarding our ongoing clinical studies of UGN-301; the estimated patient population and demographics for bladder cancer and HG-NMIBC; the potential of UroGen's proprietary RTGel technology to improve therapeutic profiles of existing drugs; and UroGen's sustained release technology making local delivery potentially more effective as compared to other treatment options. Words such as 'could,' 'potential' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: UroGen's development plans for UGN-301, including in combination with other investigational agents; preliminary results may not be indicative of results that may be observed in the future; the timing and success of clinical trials and potential safety and other complications thereof; unforeseen delays that may impact the timing of progressing clinical trials and reporting data; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to obtain and maintain adequate intellectual property rights and adequately protect and enforce such rights; complications associated with commercialization activities; the labeling for any approved product; competition in UroGen's industry; the scope, progress and expansion of developing and commercializing UroGen's product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen's ability to attract or retain key management, members of the board of directors and other personnel; UroGen's RTGel technology may not perform as expected; and UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 10, 2025. The events and circumstances discussed in such forward-looking statements may not occur, and UroGen's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release. View source version on CONTACT: INVESTOR CONTACT: Vincent Perrone Senior Director, Investor Relations [email protected] 609-460-3588 ext. 1093MEDIA CONTACT: Cindy Romano Director, Communications [email protected] 609-460-3583 ext. 1083 KEYWORD: UNITED STATES NORTH AMERICA NEVADA NEW JERSEY INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH PHARMACEUTICAL CLINICAL TRIALS ONCOLOGY SOURCE: UroGen Pharma Ltd. Copyright Business Wire 2025. PUB: 04/26/2025 10:00 AM/DISC: 04/26/2025 09:59 AM
Business Wire
26-04-2025
- Business
- Business Wire
UroGen Pharma Announces Encouraging Results from a Phase 1 Dose-Escalation Study Evaluating UGN-301 in Non-Muscle Invasive Bladder Cancer
PRINCETON, N.J.--(BUSINESS WIRE)--UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced encouraging safety data from the Phase 1 dose-escalation study for UGN-301 (zalifrelimab) intravesical solution, an investigational drug in development for the treatment of recurrent non-muscle invasive bladder cancer (NMIBC). "The early safety profile and clinical activity results from this study are encouraging,' said Jay Raman, M.D., Professor and Chair of Urology, and Professor of Surgery, Penn State Cancer Institute, PA. 'This innovative approach of localized drug delivery combined with immune modulation merits additional investigation in the treatment of non-muscle invasive bladder cancer.' The multi-part clinical study included up to 30 patients per arm, aimed to assess safety and determine the recommended Phase 2 dose of UGN-301 as monotherapy and in combination with other agents. In the monotherapy arm, dose escalation continued to the maximum feasible dose. No dose-limiting toxicities and no treatment-emergent adverse events leading to treatment discontinuation were observed. This study also demonstrated that local delivery of UGN-301 formulated in our proprietary reverse thermal gel (RTGel ®) allowed sustained exposure of zalifrelimab in the bladder with limited systemic exposure, which mitigated the risk of systemic immune-related toxicities associated with CTLA-4 inhibition. With respect to clinical activity observed in the trial, among evaluable patients who received UGN-301, 46% (6 of 13) of those with Ta/T1 disease and 33% (2 of 6) of those with carcinoma in situ (CIS) ± Ta/T1 disease were recurrence-free or had achieved a complete response at week 12. Notably, 60% (3 of 5) of patients with Ta/T1 disease treated with 300 mg continued to remain recurrence-free at the 15-month disease assessment, including one patient with high-grade T1 disease. In the 500 mg cohort, 25% (1 of 4) of patients with CIS disease and 33% (1 of 3) of patients with Ta/T1 disease remained disease-free at six months, both of whom are still active participants in the study. These findings highlight the potential of UGN-301 as a targeted treatment for NMIBC with an acceptable safety profile. Presentation of data from the combination arms is planned for later this year. "Our hypothesis is that UGN-301's unique formulation could potentially offer the dual benefits of maximizing therapeutic activity while minimizing systemic side effects, a key challenge in cancer immunotherapy,' said Mark Schoenberg, Chief Medical Officer, UroGen. 'Although this requires additional clinical investigation, we are encouraged by the potential of UGN-301 as an investigational treatment for patients with recurrent NMIBC.' About Non-Muscle Invasive Bladder Cancer and High-Grade Disease In the U.S., bladder cancer is the second most common urologic cancer in men. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. High-grade non-muscle invasive bladder cancer (HG-NMIBC) is a serious and potentially life-threatening form of bladder cancer that remains confined to the inner layers of the bladder wall but exhibits aggressive behavior and a higher risk of progression. In the U.S., HG-NMIBC accounts for approximately 30–40% of all newly diagnosed NMIBC cases. Patients with HG-NMIBC face a significantly elevated risk of recurrence and progression to muscle-invasive disease, necessitating close surveillance and aggressive treatment. The standard of care includes complete transurethral resection of bladder tumor, often followed by intravesical therapy such as Bacillus Calmette-Guérin (BCG). However, BCG has a treatment failure rate of approximately 40-50%, leaving patients with limited treatment options short of radical cystectomy. Given the high recurrence and progression rates, HG-NMIBC presents a substantial clinical and quality-of-life burden. Upon recurrence, which occurs in approximately 70% of patients, the patients undergo another round of BCG therapy with a response rate of approximately 30%. About UGN-301 UGN-301 is an anti-CTLA-4 monoclonal antibody (zalifrelimab), originally licensed from Agenus Inc. in 2019. It is formulated with RTGel, our proprietary 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 in a multi-arm Phase 1 study of UGN-301 as monotherapy and in combination with other agents. The safety of UGN-301 is being evaluated in the monotherapy arm of the study as combination therapy for HG-NMIBC. About UroGen Pharma Ltd. UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen's sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with low-grade intermediate risk NMIBC are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit to learn more or follow us on X (Twitter), @UroGenPharma. Forward-Looking Statements This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: the potential benefits of UGN-301, including as a targeted treatment for NMIBC; implications that may follow from the early clinical activity and safety profile observations; UGN-301's innovative approach of localized drug delivery combined with immune modulation meriting additional investigation in the treatment of NMIBC; the potential for UGN-301 to maximize therapeutic activity and minimize systemic side effects for patients with recurrent NMIBC; the potential benefits of the intravesical delivery of UGN-301 using UroGen's RTGel proprietary technology; statements regarding our ongoing clinical studies of UGN-301; the estimated patient population and demographics for bladder cancer and HG-NMIBC; the potential of UroGen's proprietary RTGel technology to improve therapeutic profiles of existing drugs; and UroGen's sustained release technology making local delivery potentially more effective as compared to other treatment options. Words such as 'could,' 'potential' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: UroGen's development plans for UGN-301, including in combination with other investigational agents; preliminary results may not be indicative of results that may be observed in the future; the timing and success of clinical trials and potential safety and other complications thereof; unforeseen delays that may impact the timing of progressing clinical trials and reporting data; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to obtain and maintain adequate intellectual property rights and adequately protect and enforce such rights; complications associated with commercialization activities; the labeling for any approved product; competition in UroGen's industry; the scope, progress and expansion of developing and commercializing UroGen's product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen's ability to attract or retain key management, members of the board of directors and other personnel; UroGen's RTGel technology may not perform as expected; and UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 10, 2025. The events and circumstances discussed in such forward-looking statements may not occur, and UroGen's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.
