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11 hours ago
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Actuate Therapeutics shares highlights from KOL eventon elraglusib data
Actuate Therapeutics (ACTU) announced key takeaways from its Key Opinion Leader event held on May 31, 2025 discussing the topline results from the Phase 2 trial of elraglusib in combination with gemcitabine/nab-paclitaxel in previously untreated patients with metastatic pancreatic ductal adenocarcinoma. The study met its primary endpoint, demonstrating a clinically meaningful increase in median overall survival of 10.1 months compared to 7.2 months, with a 37% reduction in the risk of death. The elraglusib/GnP arm also doubled the 12-month survival rate in 44.1% of patients compared to GnP alone, with a favorable safety profile. The KOL event followed the presentation of topline trial data during an oral session at the American Society of Clinical Oncology Annual Meeting. The event featured a fireside discussion with leading mPDAC clinicians, all with direct participation in the elraglusib clinical trial program: Colin Weekes, MD, PhD, Massachusetts General Hospital; Devalingam Mahalingam, MD, Northwestern University Feinberg School of Medicine; Rachna Shroff, MD, MS, FASCO, University of Arizona Cancer Center; Tanios Bekaii-Saab, MD, FACP, Mayo Clinic College of Medicine and Science. The panelists highlighted the implications of the Phase 2 Actuate 1801 Part 3B results in the broader context of the unmet need in mPDAC, the unique immune-modulating mechanism of elraglusib, and its potential to reshape the treatment landscape. Daniel Schmitt, CEO of Actuate said, 'The reception we received at ASCO confirms what we already believed – this data has the potential to change the trajectory of mPDAC treatment. As our KOLs highlighted in the panel discussion, there is a clear need for new drugs, like elraglusib, with new mechanistic pathways, and which can be successfully combined without overlapping toxicities for treatment of this devastating disease. As these thought leaders pointed out, due to a number of negative trials, there has been quite some time since we've had anything that improves outcomes for patients with these chemotherapy backbones. The thought leaders further highlighted the importance of the overall survival benefit and noted the magnitude of that benefit in the elraglusib trial is actually substantial. We are now laser-focused on turning this promise into patient access. With a clear survival benefit, favorable safety profile, and strong scientific rationale, we look forward to advancing elraglusib development and initiating regulatory discussions with both the FDA and EMA.' Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See Insiders' Hot Stocks on TipRanks >> Read More on ACTU: Disclaimer & DisclosureReport an Issue Actuate Therapeutics Reports Positive Phase 2 Trial Results Promising Clinical Trial Results Justify Buy Rating for Actuate Therapeutics' Elraglusib Actuate Therapeutics Elects Directors at Annual Meeting Actuate Therapeutics to host KOL event on Phase 2 elraglusib data Actuate Therapeutics Announces Positive Phase 2 Trial Results Sign in to access your portfolio
Yahoo
3 days ago
- Business
- Yahoo
Actuate Therapeutics Presents Topline Elraglusib Phase 2 Data at ASCO 2025 Annual Meeting: Trial Meets Primary Endpoint of Median Overall Survival and Doubles 1-Year Survival in First-Line Treatment of Metastatic Pancreatic Cancer
Phase 2 (Actuate-1801 Part 3B) trial meets primary endpoint and demonstrates a clinically meaningful increase in median overall survival (10.1 months vs 7.2 months; log-rank p=0.01) in previously untreated patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) receiving elraglusib/GnP Risk of death was reduced by 37% (HR=0.63) in patients treated with elraglusib/GnP Data featured as an oral presentation at the ASCO Annual Meeting Company plans to engage with FDA in the second half of 2025 to align on a path towards product registration Company to host KOL event today at 6:30 pm CDT to discuss 1801 Part 3B results CHICAGO and FORT WORTH, Texas, May 31, 2025 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) ('Actuate' or the 'Company'), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), today presented topline results from the Phase 2 (Actuate-1801 Part 3B) trial of elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) in previously untreated patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) at the American Society of Clinical Oncology (ASCO) Annual Meeting. Abstract Title: Preliminary results from the randomized phase 2 study (1801 Part 3B) of elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) versus GnP alone in patients with previously untreated metastatic pancreatic ductal adenocarcinoma (mPDAC).Abstract Number: 4006Session Title: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and HepatobiliaryPresenter: Devalingam Mahalingam, MD, PhDOral Presentation Date and Time: Saturday, May 31, 2025, 4:48 PM CDT The trial met its primary endpoint of improved median overall survival. Median overall survival (mOS) increased by almost three months (10.1 months vs 7.2 months, HR=0.63, log-rank p=0.01) with a 37% reduction in the risk of death and strong statistical significance, representing a clinically meaningful advance in the potential treatment of mPDAC. Patients receiving elraglusib with GnP achieved a 12-month survival rate in 44.1% of patients treated—double that of GnP alone (22.3%). Dr. Deva Mahalingam, MD, PhD, Northwestern University Feinberg School of Medicine, and lead principal investigator of the 1801 Part 3B trial commented, 'Pancreatic cancer continues to represent one of the highest unmet medical needs with no major treatment advances in recent years, bar some molecular targets in small subsets of patients. New mechanisms of action are urgently needed. The addition of elraglusib to the existing chemotherapy backbone of gemcitabine and nab-paclitaxel is promising and may represent a meaningful therapeutic advance for patients with pancreatic cancer.' Daniel Schmitt, President & Chief Executive Officer of Actuate added, 'We significantly improved mOS, cut the risk of death by 37%, and doubled the 12-month survival rate. Combined with a manageable safety profile and strong emerging mechanistic insights, these results reinforce the transformative potential of our GSK-3β inhibitor program. Based on the clear clinical benefit and well-tolerated safety profile, we intend to engage with the FDA and EMA in the second half of this year to align on a path to registration. We believe this enables us to move rapidly towards commercialization and delivery of this first-in-class therapy to patients with an urgent unmet need.' Efficacy Figure 1 below depicts the Kaplan-Meier estimate for mOS (10.1 months vs 7.2 months, HR=0.63, log-rank p=0.01) with a 37% reduction in the risk of death, displaying a clear clinical survival benefit for patients treated in the elraglusib/GnP combination arm vs GnP alone arm. Actuate-1801 Part 3B: Kaplan-Meier Estimate for mOS as of March 27, 2025 (Topline data cut-off).In addition to the improved mOS and one-year survival rate, a continued survival benefit was also observed at eighteen and twenty-four months (survival rates of 19.7% vs 4.4% and 13.8% vs 0% in the elraglusib/GnP combination arm vs the GnP arm, respectively). The elraglusib/GnP combination treatment also resulted in numerically improved overall response rates (29.0% in the elraglusib/GnP combination arm vs 21.8% in the GnP arm) and improvements in median progression-free survival and median duration of response of 5.6 months vs 5.1 months, and 5.5 months vs 4.0 months in the elraglusib/GnP combination arms vs GnP arms, respectively. Safety and Biomarker Findings The trial also met its primary safety endpoint. Treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs) in the elraglusib/GnP combination arm were similar to those observed in the GnP arm, indicating a favorable risk-benefit profile for the elraglusib/GnP combination. Treatment-related adverse events (TRAEs) were mostly Grade 1-2, with the most frequent TRAEs observed (in about two-thirds of patients) being transient visual impairments that were reversible and non-progressive While Grade 3 or higher neutropenia was observed, similar rates of febrile neutropenia and sepsis were observed in both treatment arms. Pre-dose cytokine analysis that suggested lower baseline levels of key immune modulators, including CCL3, IL-1α, IL-18, TGF-β, and TRAIL R3, were correlated with improved 1-year survival. Increased CD8-positive and granzyme B-positive T cells, increased NK cells, and decreased myeloid-derived suppressor cells were observed in tumor biopsies only from elraglusib-treated patients. This exploratory result confirms elraglusib proposed immune modulating mechanism of action in patients with mPDAC. KOL Event Actuate will host a KOL event for the investment community today, May 31, 2025, at 6:30 PM CDT to review the data. The webinar will feature a fireside discussion moderated by Daniel Schmitt, President & Chief Executive Officer of Actuate, and will include four distinguished KOLs: Tanios Bekaii-Saab, MD, FACP, Mayo Clinic College of Medicine and Science; Devalingam Mahalingam, MD, Northwestern University Feinberg School of Medicine; Rachna Shroff, MD, MS, FASCO, University of Arizona Cancer Center; and Colin Weekes, MD, PhD, Massachusetts General Hospital. Event Details: Date and Time: Saturday, May 31, 2025, at 6:30 pm CDT Format: In-person and via live webcast Registration: Click here A replay of the event will be available on the Investor Relations section of the Actuate website. About Actuate-1801 Part 3B Study The Actuate-1801 Part 3B study (NCT03678883) is a randomized, controlled Phase 2 trial of elraglusib with GnP versus GnP alone in first-line mPDAC. The trial enrolled 286 mPDAC patients with no prior systemic treatment for metastatic disease, who were randomized 2:1 to the elraglusib treatment arm (elraglusib + GnP) or the control arm (GnP alone). Elraglusib is administered at a dose of 9.3 mg/kg by IV infusion on Day 1 of each week of a 28-day cycle. The primary endpoint for this study is median overall survival, with OS summarized throughout the study by estimates of 1-year survival. Secondary endpoints are DCR, ORR, PFS, and of GSK-3β may inhibit tumor growth and improve survival through several complimentary mechanisms that include enhancement of chemotherapy activity, activation of innate anti-tumor immunity, and regulation of gene expression, leading to alterations in tumor metabolism and Epithelial-to-Mesenchymal Transition (EMT). About Actuate Therapeutics, Inc. Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate's lead investigational drug, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and DNA Damage Response (DDR). Elraglusib may also mediate anti-tumor immunity through the regulation of multiple immune checkpoints and immune cell function. For additional information, please visit the Company's website at Forward-Looking Statements This press release contains forward-looking statements about us, including our and other parties' clinical trials and development plans, and our industry. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'might,' 'ongoing,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would,' or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements related to present facts or current conditions or of historical facts, contained in this press release are forward-looking statements. Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including but not limited to that preliminary and unpublished data may be subject to change and further interpretation following the availability of more data or following a more comprehensive review of the data and should not be relied upon as a final analysis; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities and within the medical community; clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies and early clinical trials are not necessarily predictive of future results, and elraglusib may not achieve positive clinical results or favorable preclinical results, and we may not be able to make regulatory submissions or receive regulatory approval on a timely basis, if at all; that we may not successfully enroll additional patients or establish or advance plans for further development, including through conversations with the FDA or EMA and the standards such bodies may impose for such development; that elraglusib could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude regulatory approval, cause us to suspend or discontinue clinical trials or result in other negative consequences; our reliance on third parties to conduct our non-clinical studies and our clinical trials; our reliance on third-party licensors and ability to preserve and protect our intellectual property rights; that we face significant competition from other biotechnology and pharmaceutical companies; our ability to fund development activities, including because our financial condition raises substantial doubt as to our ability to continue as a going concern and we require additional capital to finance our operations beyond the second quarter of fiscal year 2025, and a failure to obtain this necessary capital in the near term on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our development programs, commercialization efforts or other operations. In addition, any forward-looking statements are qualified in their entirety by reference to the factors discussed under the heading 'Item 1A. Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 13, 2025, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC on May 15, 2025, and other filings with the SEC. Because the risk factors referred to above could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements made by us or on our behalf, you should not place undue reliance on any forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Unless legally required, we do not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events. Investor ContactMike MoyerManaging DirectorLifeSci Advisors, LLCmmoyer@ A photo accompanying this announcement is available at in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
3 days ago
- Business
- Yahoo
Actuate Therapeutics Presents Topline Elraglusib Phase 2 Data at ASCO 2025 Annual Meeting: Trial Meets Primary Endpoint of Median Overall Survival and Doubles 1-Year Survival in First-Line Treatment of Metastatic Pancreatic Cancer
Phase 2 (Actuate-1801 Part 3B) trial meets primary endpoint and demonstrates a clinically meaningful increase in median overall survival (10.1 months vs 7.2 months; log-rank p=0.01) in previously untreated patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) receiving elraglusib/GnP Risk of death was reduced by 37% (HR=0.63) in patients treated with elraglusib/GnP Data featured as an oral presentation at the ASCO Annual Meeting Company plans to engage with FDA in the second half of 2025 to align on a path towards product registration Company to host KOL event today at 6:30 pm CDT to discuss 1801 Part 3B results CHICAGO and FORT WORTH, Texas, May 31, 2025 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) ('Actuate' or the 'Company'), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), today presented topline results from the Phase 2 (Actuate-1801 Part 3B) trial of elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) in previously untreated patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) at the American Society of Clinical Oncology (ASCO) Annual Meeting. Abstract Title: Preliminary results from the randomized phase 2 study (1801 Part 3B) of elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) versus GnP alone in patients with previously untreated metastatic pancreatic ductal adenocarcinoma (mPDAC).Abstract Number: 4006Session Title: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and HepatobiliaryPresenter: Devalingam Mahalingam, MD, PhDOral Presentation Date and Time: Saturday, May 31, 2025, 4:48 PM CDT The trial met its primary endpoint of improved median overall survival. Median overall survival (mOS) increased by almost three months (10.1 months vs 7.2 months, HR=0.63, log-rank p=0.01) with a 37% reduction in the risk of death and strong statistical significance, representing a clinically meaningful advance in the potential treatment of mPDAC. Patients receiving elraglusib with GnP achieved a 12-month survival rate in 44.1% of patients treated—double that of GnP alone (22.3%). Dr. Deva Mahalingam, MD, PhD, Northwestern University Feinberg School of Medicine, and lead principal investigator of the 1801 Part 3B trial commented, 'Pancreatic cancer continues to represent one of the highest unmet medical needs with no major treatment advances in recent years, bar some molecular targets in small subsets of patients. New mechanisms of action are urgently needed. The addition of elraglusib to the existing chemotherapy backbone of gemcitabine and nab-paclitaxel is promising and may represent a meaningful therapeutic advance for patients with pancreatic cancer.' Daniel Schmitt, President & Chief Executive Officer of Actuate added, 'We significantly improved mOS, cut the risk of death by 37%, and doubled the 12-month survival rate. Combined with a manageable safety profile and strong emerging mechanistic insights, these results reinforce the transformative potential of our GSK-3β inhibitor program. Based on the clear clinical benefit and well-tolerated safety profile, we intend to engage with the FDA and EMA in the second half of this year to align on a path to registration. We believe this enables us to move rapidly towards commercialization and delivery of this first-in-class therapy to patients with an urgent unmet need.' Efficacy Figure 1 below depicts the Kaplan-Meier estimate for mOS (10.1 months vs 7.2 months, HR=0.63, log-rank p=0.01) with a 37% reduction in the risk of death, displaying a clear clinical survival benefit for patients treated in the elraglusib/GnP combination arm vs GnP alone arm. Actuate-1801 Part 3B: Kaplan-Meier Estimate for mOS as of March 27, 2025 (Topline data cut-off).In addition to the improved mOS and one-year survival rate, a continued survival benefit was also observed at eighteen and twenty-four months (survival rates of 19.7% vs 4.4% and 13.8% vs 0% in the elraglusib/GnP combination arm vs the GnP arm, respectively). The elraglusib/GnP combination treatment also resulted in numerically improved overall response rates (29.0% in the elraglusib/GnP combination arm vs 21.8% in the GnP arm) and improvements in median progression-free survival and median duration of response of 5.6 months vs 5.1 months, and 5.5 months vs 4.0 months in the elraglusib/GnP combination arms vs GnP arms, respectively. Safety and Biomarker Findings The trial also met its primary safety endpoint. Treatment-emergent adverse events (TEAEs) and Serious Adverse Events (SAEs) in the elraglusib/GnP combination arm were similar to those observed in the GnP arm, indicating a favorable risk-benefit profile for the elraglusib/GnP combination. Treatment-related adverse events (TRAEs) were mostly Grade 1-2, with the most frequent TRAEs observed (in about two-thirds of patients) being transient visual impairments that were reversible and non-progressive While Grade 3 or higher neutropenia was observed, similar rates of febrile neutropenia and sepsis were observed in both treatment arms. Pre-dose cytokine analysis that suggested lower baseline levels of key immune modulators, including CCL3, IL-1α, IL-18, TGF-β, and TRAIL R3, were correlated with improved 1-year survival. Increased CD8-positive and granzyme B-positive T cells, increased NK cells, and decreased myeloid-derived suppressor cells were observed in tumor biopsies only from elraglusib-treated patients. This exploratory result confirms elraglusib proposed immune modulating mechanism of action in patients with mPDAC. KOL Event Actuate will host a KOL event for the investment community today, May 31, 2025, at 6:30 PM CDT to review the data. The webinar will feature a fireside discussion moderated by Daniel Schmitt, President & Chief Executive Officer of Actuate, and will include four distinguished KOLs: Tanios Bekaii-Saab, MD, FACP, Mayo Clinic College of Medicine and Science; Devalingam Mahalingam, MD, Northwestern University Feinberg School of Medicine; Rachna Shroff, MD, MS, FASCO, University of Arizona Cancer Center; and Colin Weekes, MD, PhD, Massachusetts General Hospital. Event Details: Date and Time: Saturday, May 31, 2025, at 6:30 pm CDT Format: In-person and via live webcast Registration: Click here A replay of the event will be available on the Investor Relations section of the Actuate website. About Actuate-1801 Part 3B Study The Actuate-1801 Part 3B study (NCT03678883) is a randomized, controlled Phase 2 trial of elraglusib with GnP versus GnP alone in first-line mPDAC. The trial enrolled 286 mPDAC patients with no prior systemic treatment for metastatic disease, who were randomized 2:1 to the elraglusib treatment arm (elraglusib + GnP) or the control arm (GnP alone). Elraglusib is administered at a dose of 9.3 mg/kg by IV infusion on Day 1 of each week of a 28-day cycle. The primary endpoint for this study is median overall survival, with OS summarized throughout the study by estimates of 1-year survival. Secondary endpoints are DCR, ORR, PFS, and of GSK-3β may inhibit tumor growth and improve survival through several complimentary mechanisms that include enhancement of chemotherapy activity, activation of innate anti-tumor immunity, and regulation of gene expression, leading to alterations in tumor metabolism and Epithelial-to-Mesenchymal Transition (EMT). About Actuate Therapeutics, Inc. Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate's lead investigational drug, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and DNA Damage Response (DDR). Elraglusib may also mediate anti-tumor immunity through the regulation of multiple immune checkpoints and immune cell function. For additional information, please visit the Company's website at Forward-Looking Statements This press release contains forward-looking statements about us, including our and other parties' clinical trials and development plans, and our industry. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'might,' 'ongoing,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would,' or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements related to present facts or current conditions or of historical facts, contained in this press release are forward-looking statements. Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including but not limited to that preliminary and unpublished data may be subject to change and further interpretation following the availability of more data or following a more comprehensive review of the data and should not be relied upon as a final analysis; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities and within the medical community; clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies and early clinical trials are not necessarily predictive of future results, and elraglusib may not achieve positive clinical results or favorable preclinical results, and we may not be able to make regulatory submissions or receive regulatory approval on a timely basis, if at all; that we may not successfully enroll additional patients or establish or advance plans for further development, including through conversations with the FDA or EMA and the standards such bodies may impose for such development; that elraglusib could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude regulatory approval, cause us to suspend or discontinue clinical trials or result in other negative consequences; our reliance on third parties to conduct our non-clinical studies and our clinical trials; our reliance on third-party licensors and ability to preserve and protect our intellectual property rights; that we face significant competition from other biotechnology and pharmaceutical companies; our ability to fund development activities, including because our financial condition raises substantial doubt as to our ability to continue as a going concern and we require additional capital to finance our operations beyond the second quarter of fiscal year 2025, and a failure to obtain this necessary capital in the near term on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our development programs, commercialization efforts or other operations. In addition, any forward-looking statements are qualified in their entirety by reference to the factors discussed under the heading 'Item 1A. Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 13, 2025, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC on May 15, 2025, and other filings with the SEC. Because the risk factors referred to above could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements made by us or on our behalf, you should not place undue reliance on any forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Unless legally required, we do not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events. Investor ContactMike MoyerManaging DirectorLifeSci Advisors, LLCmmoyer@ A photo accompanying this announcement is available at
Yahoo
4 days ago
- Business
- Yahoo
Late-breaking analysis demonstrates characteristics associated with long-term overall survival with Onivyde® regimen in metastatic pancreatic adenocarcinoma
PARIS, France, 31 May 2025 Late-breaking (LBA4175) post-hoc analysis data from the Phase III NAPOLI 3 study were presented today at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. These results found a median overall survival (mOS) of 19.5 months among long-term survivors (n=15) with metastatic pancreatic adenocarcinoma (mPDAC) treated with the Onivyde (irinotecan liposome injection) plus oxaliplatin, fluorouracil and leucovorin (NALIRIFOX) regimen as a first-line treatment (n=120), with younger age at diagnosis, and certain tumor and metastasis locations associated with long-term survivorship. Pancreatic adenocarcinoma (PDAC) is the most common type of cancer that forms in the pancreas, with more than 60,000 people diagnosed annually in the U.S. and nearly 500,000 people globally.34 It is often detected after the disease has spread to other parts of the body (metastatic or stage IV)5 and fewer than 20% of people diagnosed with metastatic pancreatic adenocarcinoma (mPDAC) survive longer than one year. Overall, pancreatic cancer has the lowest five-year survival rate of all cancer types globally and in the U.S. 'When people are diagnosed with metastatic pancreatic adenocarcinoma, the most important question remains: how long will they have with their loved ones,' said Dr. Vincent Chung, Medical Oncologist, City of Hope. 'Findings from the NAPOLI 3 post-hoc analysis provide important context on long-term overall survival with the Onivyde (NALIRIFOX) treatment regimen.' The analysis included patients who survived for 18 months or longer (N=15), with findings showing long-term survivors living with mPDAC had a mOS of 19.5 months (interquartile range [IQR]: 18.8–22.6). Clinical and pathological factors of long-term survivors included younger than average age at time of diagnosis (median age 61.0 (IQR: 49.0–70.5) as well as tumor location. Fewer patients had tumors in the head or tail of the pancreas (53.3% had the main pancreatic tumor located in the body of the pancreas), a substantial proportion had liver metastasis (66.7%) and ≥3 metastatic sites (53.3%). Additionally, findings indicate dose reduction and treatment delays resulted in prolonged exposure and higher cumulative doses of the Onivyde (NALIRIFOX) regimen. Liver metastasis and ≥3 metastatic sites, dose modifications and an otherwise good clinical profile enabled people to achieve a long mOS. Consideration should be taken when interpreting these results as a post-hoc analysis with a small sample size. "Data from the Phase III NAPOLI 3 trial were the first positive data of its kind in a decade and continue to reinforce the potential for long-term outcomes with the Onviyde (NALIRIFOX) regimen,' said Sandra Silvestri, MD, PhD, Executive Vice President, Chief Medical Officer, Ipsen. 'With people on average living just 4-6 months following diagnosis with pancreatic adenocarcinoma, these data help us to understand the characteristics associated with long-term survival seen in the NAPOLI trial, an important advancement for this difficult-to-treat cancer where data of this kind are scarce.' ENDS About Onivyde (irinotecan liposome injection)Onivyde is a long-circulating liposomal topoisomerase inhibitor. In Onivyde, irinotecan is enclosed in tiny fat particles called liposomes which accumulate in the tumor and release slowly over time. Onivyde is administered via intravenous infusion over 90 minutes every two weeks with recommendations on dosing modifications. Onivyde, as part of the NALIRIFOX regimen (combined with oxaliplatin, fluorouracil (FU) and leucovorin (LV)), is for people living with mPDAC who are treatment naïve or used in combination with FU and LV following gemcitabine-based therapy. Onivyde is not indicated as a single agent for the treatment of adult patients with metastatic pancreatic adenocarcinoma. Ipsen has exclusive commercialization rights for the current and potential future indications for Onivyde in the U.S. Servier, an independent international pharmaceutical company governed by a foundation and with an international presence in 140 countries, is responsible for the commercialization of Onivyde outside of the U.S., Taiwan and Canada. PharmaEngine is a commercial stage oncology company headquartered in Taipei and is responsible for the commercialization of Onivyde in Taiwan. About NAPOLI 3 Study NAPOLI 3 is a randomized, open-label Phase III trial of an Onivyde treatment regimen (NALIRIFOX) in treatment-naïve mPDAC. NAPOLI 3 enrolled 770 patients across 187 trial site locations in 18 countries across Europe, North America, South America, Asia, and Australia. Patients were randomized to receive Onivyde plus oxaliplatin, fluorouracil and leucovorin (NALIRIFOX regimen; n=383) twice in a month (days 1 and 15 of 28-day cycle) compared to an injection of nab-paclitaxel and gemcitabine (n=387) administered three times a month (days 1, 8, 15 of a 28-day cycle). About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit Ipsen Media contacts Investors Khalid Deojee | +33 666019526 | Media Sally Bain | +1 8573200517 | Anne Liontas | +33 0767347296 | Disclaimers and/or Forward-Looking StatementsThe forward-looking statements, objectives and targets contained herein are based on Ipsen's management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen's future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words 'believes', 'anticipates' and 'expects' and similar expressions are intended to identify forward-looking statements, including Ipsen's expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen's patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen's activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen's partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen's business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen's business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen's latest Universal Registration Document, available on References 1 Wainberg et al. NALIRIFOX versus nab-paclitaxel and gemcitabine in treatment-naive patients with metastatic pancreatic ductal adenocarcinoma (NAPOLI 3): a randomised, open-label, phase 3 trial. Lancet. 2023 Oct 7;402(10409):1272-1281.2 Chung et al. NAPOLI 3 phase 3 study of NALIRIFOX in patients with metastatic pancreatic ductal adenocarcinoma (mPDAC): final overall survival (OS) analysis and characteristics of the long-term survivors. As presented at ASCO Congress 2025 Chicago, USA3 American Cancer Society – Cancer Facts and Figures 2024. Available : Orth, M., Metzger, P., Gerum, S. et al. Pancreatic ductal adenocarcinoma: biological hallmarks, current status, and future perspectives of combined modality treatment approaches. Radiat Oncol 14, 141 (2019). Attachment Ipsen PR_ASCO NAPOLI_31052025
