Nirogacestat hydrobromide

– Long-term follow-up data highlight further reductions in tumor size, increase in ORR with additional CRs, sustained improvement in desmoid tumor symptoms, and consistent safety profile, now with a median duration of therapy of approximately 3 years – Nov. 07, 2024 -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, today announced that long-term efficacy and safety data from the Phase 3 DeFi trial of nirogacestat in adults with progressing desmoid tumors will be presented as a late-breaking oral presentation at the Connective Tissue Oncology Society (CTOS) 2024 Annual Meeting, being held November 13-16, 2024, in San Diego, CA. These results, utilizing an August 2024 data cutoff date, showed that longer-term treatment with nirogacestat was associated with further reductions in tumor size, increase in objective response rate (ORR) with additional partial responses (PRs) and complete responses (CRs), sustained improvements in desmoid tumor symptoms including pain, and a consistent safety profile compared to the April 2022 data cut utilized for the primary results of the trial. “Our findings demonstrate that longer-term nirogacestat treatment was associated with durable tumor size reductions, evidence of deepening responses, and sustained benefits in pain, physical functioning and other desmoid tumor symptoms,” said Ravin Ratan, M.D., M.Ed., Associate Professor, Department of Sarcoma Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center in Houston, and DeFi study investigator presenting the data at CTOS. “Given the oftentimes persistent and debilitating nature of desmoid tumors, these results are meaningful for patients and clinicians as they provide valuable insights on the longer-term use of this medicine.” “We are pleased that the growing body of evidence from the DeFi trial continues to support the significant benefits that OGSIVEO is providing for patients with desmoid tumors,” said Jim Cassidy, M.D., Ph.D., Chief Medical Officer of SpringWorks Therapeutics. “It is also gratifying that feedback from the real-world setting is consistent with our clinical trial experience, with patients reporting rapid, sustained and continued symptomatic relief, which is contributing to their overall improved quality of life.” Additional DeFi data to be presented at CTOS include an oral presentation of a post-hoc analysis assessing the effect of nirogacestat in subgroups of patients with desmoid tumors who have risk factors associated with poor prognosis and a poster on patients with CTNNB1 and APC mutations. Oral Presentations at the CTOS 2024 Annual Meeting Nirogacestat treatment in adult patients with desmoid tumors: Long-term efficacy and safety from the Phase 3 DeFi trial Date and Time: November 16, 3:30-4:30 p.m. PT (6:30-7:30 p.m. ET) As previously reported, 70 patients were randomized to nirogacestat in the double-blind (DB) portion of the Phase 3 DeFi trial (NCT03785964); these patients achieved statistically and clinically significant improvement in progression-free survival (PFS) and ORR, as well as rapid and sustained improvement in pain, physical functioning, role functioning and overall quality of life, as compared to those randomized to placebo. At the primary analysis data cut-off date (April 2022), the median (range) duration of nirogacestat treatment was 20.6 (0.3-33.6) months. Following the DB portion of the study, patients could continue to receive nirogacestat as part of the open-label extension (OLE). As of this long-term follow-up analysis (August 2024 data cut-off date), the median (range) duration of nirogacestat treatment in these patients was 33.6 (0.3 to 60) months. The data being presented at CTOS evaluated the overall efficacy and safety of continuous nirogacestat treatment in patients initially randomized to nirogacestat in DeFi and further explored safety and efficacy analyses at milestones of one, two, three, and four years on treatment. Results demonstrated that: Three new PRs and three new CRs were reported using the August 2024 versus the April 2022 data cut-off date, resulting in an ORR of 45.7% (34.3% PR, 11.4% CR) (N=70). The median best percent reduction from baseline in target tumor size by RECIST 1.1 with continuous nirogacestat treatment was −32.3% at year one (n=46) and −75.8% for patients completing at least four years (n=15) of treatment. Improvement in patient reported outcomes (PROs) of pain, desmoid tumor-specific symptom severity, and desmoid tumor-specific physical functioning, which occurred early (at Cycle 2, the first post-treatment timepoint evaluated as disclosed at the primary analysis) and were sustained with up to 45 months of treatment with nirogacestat. The most frequently reported treatment emergent adverse events (TEAEs) that occurred in >20% of patients receiving nirogacestat over the entire treatment period were diarrhea, nausea, fatigue, hypophosphatemia, and headache, and were generally consistent between the April 2022 and August 2024 data cutoffs. Most events were Grade 1 or 2, with first onset occurring in the first year of treatment for most patients. Overall, the incidence and severity of frequently reported TEAEs decreased through years two, three and four of treatment. Efficacy of nirogacestat in patients with poor prognostic factors for desmoid tumors: patient-reported outcomes, progression-free survival, and objective response in the phase 3 DeFi trial Date and Time: November 16, 3:30-4:30 p.m. PT (6:30-7:30 p.m. ET) A post hoc analysis of the DeFi trial was conducted to assess the effect of nirogacestat on PFS, ORR and PROs in subgroups of patients with desmoid tumors who have risk factors associated with a poor prognosis: larger tumor size (>10 cm), younger age (≤30 years), specific types of CTNNB1 gene mutations, and presence of pain at baseline. Results include: Treatment with nirogacestat led to consistent improvements in PFS, ORR and PROs versus placebo regardless of the patient subgroups. The ORR risk difference between nirogacestat and placebo ranged from 18.1% to 56.0%, favoring nirogacestat. Compared with placebo, patients treated with nirogacestat generally reported greater improvement from baseline at cycle 10 in PROs (pain, desmoid tumor-specific symptom burden, physical and role functioning, and overall quality of life) across subgroups of patient-related and tumor-related poor prognostic factors. The authors concluded that nirogacestat demonstrates uniform efficacy and consistent improvement in PROs across the desmoid tumor population. Poster Presentations at the CTOS 2024 Annual Meeting Descriptive evaluation of patients with desmoid tumor and co-occurring somatic mutations of CTNNB1 and APC in the Phase 3 DeFi trial Date and Time: November 14, 5:45-6:45 p.m. PT (8:45-9:45 p.m. ET) This analysis identified three patients with desmoid tumors enrolled in the DeFi trial who had co-occurring somatic mutations of CTNNB1 and APC, including two treated with nirogacestat. Both patients achieved a best overall response of partial response (with time to response of 6.0 and 13.8 months). Although the small number of patients limited a formal analysis, descriptive results suggest that patients with this mutational profile benefit from nirogacestat treatment in a manner that is generally consistent with the overall DeFi study population but may take longer to experience a treatment response. DeFi (NCT03785964) is a global, randomized (1:1), multicenter, double-blind, placebo-controlled pivotal Phase 3 trial evaluating the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The double-blind phase of the study randomized 142 patients (nirogacestat, n=70; placebo n=72) to receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria included tumor progression by ≥20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to screening. The primary endpoint was progression-free survival (PFS), as assessed by blinded independent central review, or death by any cause. Secondary and exploratory endpoints include safety and tolerability measures, objective response rate (ORR), duration of response, changes in tumor volume assessed by magnetic resonance imaging (MRI), and changes in patient-reported outcomes (PROs). DeFi includes an open-label extension phase, which is ongoing. Desmoid tumors (sometimes referred to as aggressive fibromatosis, or desmoid fibromatosis) are rare, aggressive, locally invasive tumors of the soft tissues that can be serious, debilitating, and, in rare cases when vital structures are impacted, life-threatening.1,2 Desmoid tumors are most commonly diagnosed in patients between the ages of 20 and 44 years, with a two-to-three times higher prevalence in females.3,4 It is estimated that there are 1,000-1,650 new cases diagnosed per year in the United States.4,5,6 Although they do not metastasize, desmoid tumors are associated with recurrence rates of up to 77% after surgical resection.3,7.8 Desmoid tumor experts and treatment guidelines now recommend systemic therapies as first-line intervention instead of surgery for most tumor locations requiring treatment.8 OGSIVEO (nirogacestat) is an oral, selective, small molecule gamma secretase inhibitor approved in the United States for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. OGSIVEO is not approved for the treatment of any other indication in the United States, or for any indication in any other jurisdiction by any other health authority. SpringWorks is also evaluating nirogacestat as a potential treatment for patients with ovarian granulosa cell tumors and for patients with multiple myeloma as part of several B-cell maturation agent (BCMA) combination therapy regimens in collaboration with leaders in industry and academia. SpringWorks is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and delivering life-changing medicines for people with severe rare diseases and cancer. OGSIVEO® (nirogacestat), approved in the United States for the treatment of adult patients with progressing desmoid tumors who require systemic treatment, is the Company’s first FDA-approved therapy. SpringWorks also has a diversified targeted therapy pipeline spanning solid tumors and hematological cancers, with programs ranging from preclinical development through advanced clinical trials. In addition to its wholly owned programs, SpringWorks has also entered into multiple collaborations with innovators in industry and academia to unlock the full potential for its portfolio and create more solutions for patients in need. References Sbaraglia M, Bellan E, Dei Tos AP. The 2020 WHO Classification of Soft Tissue Tumours: news and perspectives. Pathologica. 2021;113(2):70-84. doi:10.32074/1591-951X-213. Penel N, Chibon F, Salas S. Adult desmoid tumors: biology, management and ongoing trials. Curr Opin Oncol. 2017;29(4):268-274. doi:10.1097/CCO.0000000000000374. Skubitz KM. Biology and treatment of aggressive fibromatosis or desmoid tumor. Mayo Clin Proc. 2017;92(6):947-964. doi:10.1016/j.mayocp.2017.02.012. van Broekhoven DLM, Grünhagen DJ, den Bakker MA, van Dalen T, Verhoef C. Time trends in the incidence and treatment of extra-abdominal and abdominal aggressive fibromatosis: a population-based study. Ann Surg Oncol. 2015;22(9):2817-2823. doi:10.1245/s10434-015-4632-y. Orphanet Report Series: Rare Diseases collection. Prevalence and incidence of rare diseases: bibliographic data. Number 1, January 2022. Accessed November 5, 2024. https://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf. U.S. Department of Commerce. News Blog. U.S. population estimated at 332,403,650 on Jan. 1, 2022. Accessed November 5, 2024. https://www.commerce.gov/news/blog/2022/01/us-population-estimated-332403650-jan-1-2022. Easter DW, Halasz NA. Recent trends in the management of desmoid tumors. Summary of 19 cases and review of the literature. Ann Surg. 1989;210(6):765-769. doi:10.1097/00000658-198912000-00012. Gronchi A, Kasper B, et al. Desmoid Tumor Working Group. The management of desmoid tumours: a joint global consensus-based guideline approach for adult and paediatric patients. Eur J Cancer. 2020;127:96-107. doi:10.1016/j.ejca.2019.11.013. The content above comes from the network. if any infringement, please contact us to modify.

USA News Group News Commentary Issued on behalf of Oncolytics Biotech Inc. VANCOUVER, BC, Nov. 14, 2024 /PRNewswire/ -- USA News Group News Commentary – As many biotech companies within the oncology space begin to roll out their end-of-quarter results, the American Medical Association is pointing out the alarming trend of rising cancer rates, especially with younger people. According to data released from the National Cancer Institute (NCI), a division of the National Institutes of Health (NIH), a map shared by Newsweek showed that the prevalence of cancer varies significantly from state to state. Thankfully, there are plenty of new breakthroughs in the fight against cancer that the World Economic Forum recently highlighted, which are giving hope. Behind the scenes, biotech companies are making strides, with several touting their recent wins in their Q3 2024 financial results, including developments from O ncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), ImmunityBio, Inc. (NASDAQ: IBRX), Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), Syndax Pharmaceuticals, Inc. (NASDAQ: SNDX), and SpringWorks Therapeutics, Inc. (NASDAQ: SWTX). The article continued: As reported by The Guardian, a new study is raising hopes of treating aggressive cancers by zapping rogue DNA that helps tumors thrive and become resistant to chemotherapy. Showing the shifting market, researchers at Statista recently released a report showing the Top 5 Oncology Products Worldwide by Market Share in 2017 and 2024. Oncolytics Biotech® Reports Third Quarter 2024 Financial Results and Operational Highlights Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), a leading clinical-stage company specializing in immunotherapy for oncology, recently reported their Q3 2024 financial results and operational highlights. Among these highlights included impressive overall survival benefit results from BRACELET-1 data in HR+/HER2- metastatic breast cancer affirming its pelareorep + paclitaxel combination should be evaluated in a registrational study. "With positive BRACELET-1 results, we have two randomized phase two studies confirming pelareorep's potential in HR+/HER2- metastatic breast cancer," said Wayne Pisano, Chair of Oncolytics' Board of Directors and Interim CEO of Oncolytics. "After discussions with regulators and key opinion leaders and based on an estimated overall survival benefit of more than a year provided by pelareorep-based therapy, a registration-enabling study that is designed to support an accelerated approval is the next logical step for the development of pelareorep." In the BRACELET-1 study, pelareorep combined with paclitaxel significantly improved outcomes for patients with advanced HR+/HER2- breast cancer. Patients receiving this treatment lived without cancer progression for an average of 12.1 months, compared to just 6.4 months with paclitaxel alone—showing a benefit of nearly six months. Additionally, 64% of patients treated with pelareorep and paclitaxel lived for at least two years, compared to only 33% for paclitaxel alone. Median overall survival could not be calculated because over half of the patients on pelareorep were still alive at the study's end, but if they only lived to their next follow-up, their survival would average 32.1 months, notably higher than the 18.2 months for paclitaxel alone. Oncolytics also highlighted upcoming milestones in its GOBLET study to include efficacy data from the anal cancer cohort and safety run-in data from the modified FOLFIRINOX pancreatic cancer cohort. "We continue to develop our gastrointestinal cancer program and look forward to presenting updated efficacy data from our anal cancer cohort and safety data from our new modified FOLFIRNOX pancreatic cancer cohort," continued Pisano. "Both of these indications represent a significant unmet medical need, and we have shown pelareorep provides the potential to meaningfully improve patient outcomes. This coming year will be critical for pelareorep, as well as for Oncolytics, and I've never been more confident in the potential pelareorep can deliver to cancer patients in need." Also, as of September 30, 2024, Oncolytics had a cash position of $19.6 million, providing a cash runway through key milestones and into 2025. Looking ahead, in H1 2025, Oncolytics is set to finalize the master protocol for the adaptive registration-enabling trial for pelareorep, gemcitabine, nab-paclitaxel, and atezolizumab in first-line pancreatic ductal adenocarcinoma (PDAC) with the Global Coalition for Adaptive Research (GCAR) and submit it to the FDA. Oncolytics also expects in H1 2025: safety run-in data from cohort 5 of the GOBLET study, investigating pelareorep and modified FOLFIRNOX (mFOLFIRINOX) with or without atezolizumab in newly diagnosed pancreatic cancer; and updated efficacy data from cohort 4 of the GOBLET study, investigating pelareorep and atezolizumab in second-line or later anal cancer. As well, mid 2025 should see the first patient enrolled in registration-enabling study evaluating pelareorep and paclitaxel in metastatic HR+/HER2- breast cancer, and H2 2025 should see initial efficacy results from cohort 5 of the GOBLET study, investigating pelareorep and mFOLFIRINOX with or without atezolizumab in newly diagnosed pancreatic cancer. CONTINUED… Read this and more news for Oncolytics Biotech at: In other industry developments and happenings in the market this week include: ImmunityBio, Inc. (NASDAQ: IBRX), an immunotherapy innovator, recently reported its Q3 2024 financial results, which included highlights such as its ANKTIVA® receiving a J-code (HCPCS Level II Code) effective January 1, 2025, and becoming widely accessible to patients through commercial and government insurance programs (VA, DoD, Medicare), leading ImmunityBio to secure coverage for over 200 million medical lives through medical reimbursement policies. "The U.S. launch of ANKTIVA for [non-muscle invasive bladder cancer with carcinoma in situ] (NMIBC CIS) continues to gain momentum, and we are pleased to see the clinical impact for patients," said Richard Adcock, President and CEO of ImmunityBio. "Our permanent J-code has been issued by Centers for Medicare and Medicaid Services and will be effective January 1, 2025. Our submission of ANKTIVA for NMIBC CIS to the MHRA in the UK for potential approval demonstrates our plans for global expansion. Further, we anticipate an EU submission this quarter." The financial results came just weeks after ImmunityBio announced that the first patients had been dosed in an initial trial studying the potential of the company's CAR-NK cell therapy targeting CD-19 in the treatment of non-Hodgkin's lymphoma (NHL). Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, recently reported its Q3 2024 financial results and corporate updates. Among the highlights were the reporting that significant demand for Amtagvi™ (Lifileucel) continues with $58.6M in Total 3Q24 Product Revenue and Iovance reaffirming guidance of $160-$165 million for FY 2024 and $450-$475 million for FY 2025 of total product revenue. " Iovance is executing a successful U.S. commercial launch of Amtagvi™ for patients with previously treated advanced melanoma," said Frederick Vogt, Ph.D., J.D., Interim President and CEO of Iovance. "Robust demand for Amtagvi and Proleukin® continues to grow as our expanding network of authorized treatment centers (ATCs) and outreach to community oncologists broaden the utilization of Amtagvi, driving a higher volume of patient referrals. Demand trends are expected to accelerate growth throughout the remainder of the year and over the following years. As a fully integrated company, Iovance is well positioned to remain the global leader in innovating, developing, and delivering current and future generations of TIL cell therapy for patients with cancer." Syndax Pharmaceuticals, Inc. (NASDAQ: SNDX), a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, recently reported its Q3 2024 financial results and business update. Within the highlights were the mention that new revumenib and Niktimvo™ clinical data will be highlighted at the 66th ASH Annual Meeting and that mNPM1 AML topline data from AUGMENT-101 is expected in Q4 2024, with a potential sNDA filing in H1 2025. "This has been a historic period for Syndax as we transitioned to a commercial-stage company with the approval of Niktimvo," said Michael A. Metzger, CEO of Syndax. "We have a very exciting quarter ahead with the anticipated FDA approval and U.S. launch of revumenib for adults and pediatrics with R/R KMT2Ar acute leukemia, as well as the expected readout of topline pivotal data from patients with R/R mNPM1 AML. Our commercial organization is well-prepared to launch revumenib and leverage our first-to-market position to drive long-term value creation." SpringWorks Therapeutics, Inc. (NASDAQ: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, recently reported their Q3 2024 financial results and provided an update on recent company developments that included achieving $49.3 million in OGSIVEO® (nirogacestat) net product revenue in the quarter, and being granted FDA Priority Review on NDA and received validation of EU Marketing Authorization Application for mirdametinib for the treatment of adults and children with NF1-PN. "As we approach nearly one full year on market, we are very encouraged by the metrics we are seeing regarding the breadth of physician prescribing, the number of patients on OGSIVEO, and the number of patients identified who may stand to benefit in the future from our medicine," said Saqib Islam, CEO of SpringWorks. "Our focus for the remainder of the year is to continue building on OGSIVEO's momentum in the U.S. while working to also bring it to patients in Europe, to advance our commercial preparations for mirdametinib in anticipation of serving patients with NF1-PN in the U.S. and Europe, and to progress our emerging portfolio." Source: CONTACT: USA NEWS GROUP [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. USA News Group is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Oncolytics Biotech Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Oncolytics Biotech Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Oncolytics Biotech Inc. which were purchased in the open market, and reserve the right to buy and sell, and will buy and sell shares of Oncolytics Biotech Inc. at any time without any further notice commencing immediately and ongoing. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material, including this article, which is disseminated by MIQ has been approved by Oncolytics Biotech Inc.; this is a paid advertisement, we currently own shares of Oncolytics Biotech Inc. and will buy and sell shares of the company in the open market, or through private placements, and/or other investment vehicles. 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