Latest news with #EXACT
Agriland
3 days ago
- Climate
- Agriland
Met Éireann awards €2.8m funding to climate forecasting projects
Met Éireann has announced that it is awarding €2.8 million in funding to six new university research projects to develop new climate services and flood forecasting models. The awards are being made via the Met Éireann Research Call 2024 to projects aimed at addressing climate change, extreme weather events and environmental sustainability, the national forecaster said. Met Éireann said its Weather and Climate Research Programme, which has been running since 2018, aims to strengthen national research capacity and scientific expertise in weather and climate, through high-impact projects. Five of the six projects are funded under Met Éireann's 'TRANSLATE' climate services programme. It is hoped that those projects will expand knowledge and understanding of climate change impacts. According to Met Éireann, those projects will explore the nature of how extreme events are changing, and will develop services that support adaptation and decision-making for greater climate resilience in Ireland. Outside of those five projects, the remaining project is focused on flood forecasting, and is aimed at improving understanding of the interaction between river discharge, tide, and storm surge, providing insights on flood risk. The six projects that are receiving the funding are as follows: Project Institution Project lead Amount T3UD: TRANSLATE-3: Underpinning Data University of Galway Dr. Paul Nolan €599,992 BRACE: Building Resilience and Adaptation Capacity for future extreme Events University College Cork Dr. Paraic Ryan €599,981 EXACT: Extremes Across Climates and Timeframes Maynooth University Prof. Conor Murphy €599,704 CLIMB: Climate services for Multi-sector Benefits University College Cork Dr. Paul Holloway €299,392 ClimEnergise: Climate-Informed Energy Systems Planning to Support Ireland's Net Zero Ambitions University College Cork Dr. Vahid Aryanpur €299,546 IMUFF: Integrated Multi-model Multi-hazard Flood Forecasting University of Galway Dr. Indiana Olbert €399,976 Commenting on the funding award, director of Met Éireann Eoin Moran said: "On behalf of Met Éireann, I extend my sincere congratulations to University College Cork, Maynooth University and University of Galway. We look forward to the significant advancements each institution will contribute to our collective understanding of weather, climate, and environmental systems," he added. "These research projects represent critical contributions to both national and international efforts to address climate change, extreme weather, and environmental sustainability." Moran added: "As a proud supporter of innovative research, Met Éireann is committed to ongoing exploration that supports adaptive responses and strategic planning for improved climate resilience." The Met Éireann Weather and Climate Research Programme was launched in 2018, initially through co-funding partnerships with other research funding organisations. The Research Call 2024 was the start of the fourth round of funding, with €10.3 million already dispersed across Irish academic institutions. According to Met Éireann, nearly 80% of researchers who applied to Research Call 2024 had not previously applied. Met Éireann said it welcomed the growing reach of the programme and the increased number of active researchers in weather and climate science in Ireland.
Yahoo
20-05-2025
- Business
- Yahoo
EXACT Therapeutics announces positive final results in Phase 1 ACTIVATE trial in liver metastases from colorectal cancer
These results indicate that EXACT Therapeutics' Acoustic Cluster Therapy (ACT®) significantly enhances the local effect of chemotherapy In the patients who had a response to chemotherapy, ACT-treated tumours showed a significantly greater reduction in diameter compared to control tumours (-29% vs. -7%, p<0.05) Final analysis mirrors the excellent safety profile of PS101 seen in the interim read-out and suggests a dose-response relationship The data supports the company's plans for its Phase 2 trial (ENACT) in patients with locally advanced or borderline resectable pancreatic cancer OSLO, Norway, May 20, 2025 (GLOBE NEWSWIRE) -- EXACT Therapeutics (Euronext Growth: EXTX), a clinical-stage precision medicine company, is pleased to announce the final and positive data set from its Phase 1 ACTIVATE trial. The trial investigated the use of EXACT's proprietary Acoustic Cluster Therapy with chemotherapy in hard-to-treat patients with liver metastases of colorectal origin. These encouraging clinical data provide proof of principle for the clinical application of ACT technology and offer strong support for initiating the ENACT Phase 2 study in pancreatic cancer, a disease with a high unmet medical need. "I am excited about the data from the ACTIVATE trial, which yet again underscore the attractive therapeutic proposition from our proprietary ACT technology. The strength of the response to treatment, the safety profile and its non-invasive nature points to a unique and highly differentiated therapeutic regimen. We continue our efforts to bring ACT to cancer patients as fast as possible. Building on the momentum of the ACTIVATE study, we are excited to be approaching the first patient dosing in our Phase 2 ENACT trial in locally advanced pancreatic cancer." said Per Walday, CEO of EXACT Therapeutics. The final results affirm the previously reported positive ACTIVATE outcomes and indicate the significant added efficacy of ACT with chemotherapy compared with chemotherapy alone. Among patients who responded to chemotherapy, tumor shrinkage was significantly greater with ACT and chemotherapy compared to chemotherapy alone in the same individuals (−29% vs. −7%, p<0.05). ACT treatment with EXACT's proprietary agent PS101 had a clear dose-response relationship. Shrinkage of tumour lesions was significantly greater in patients who received 40 µl/kg PS101 compared with 20 µl/kg PS101. In the group of patients who showed a response to chemotherapy in the control lesions, 3 out of 4 patients who received 40 µl/kg PS101 showed tumour shrinkage of more than 30% in diameter. Tumour shrinkage was seen in 6 out of 9 patients who received ACT with chemotherapy. PS101 was safe and well tolerated when given with chemotherapy. Chief Medical Officer of EXACT Therapeutics, Amir Snapir continued, "Today's update marks the successful completion of the ACTIVATE trial. The results give us great confidence in the treatment potential of the ACT technology for patients with solid tumours." EXACT Therapeutics is committed to advancing cancer therapies and sharing its research findings with the broader scientific community. The company plans to present the detailed data from the Phase 1 ACTIVATE trial in a future scientific publication and at a forthcoming medical conference. The company wishes to thank the patients, their families and the investigators for their participation and contribution to the ACTIVATE trial. About the Phase 1 ACTIVATE trial ACTIVATE is a multi-center, single arm, open-label Phase 1 trial to assess the safety and tolerability, pharmacokinetics, and preliminary anti-cancer activity of ACT treatment with the chemotherapy regimens FOLFOX or FOLFIRI in patients with liver metastases of colorectal origin. The trial is designed in the way that each patient serves as their own control, with primary assessment of anti-tumour activity consisting of within-patient comparison of radiographic responses between ACT-treated lesions (insonated plus chemotherapy) and control lesions (chemotherapy alone, non-insonated). Assessment was done at week 8 by blinded central review. The trial enrolled 11 hard-to-treat patients, hereof 9 evaluable, at sites in the United Kingdom. Additional information about the ACTIVATE trial is available at (NCT04021277). About Acoustic Cluster TherapyAcoustic Cluster Therapy (ACT®) consists of PS101 administered intravenously and insonated over a target tissue with ultrasound. PS101 is a formulation of microclusters of perfluorobutane microbubbles and perfluoromethylcyclopentane microdroplets. High frequency insonation causes PS101 to form larger ACT bubbles that are temporarily lodged in capillaries, followed by low frequency insonation, which induces oscillation of the lodged ACT bubbles, enhancing the delivery of the concurrently given chemotherapy to the target tissues. About EXACT TherapeuticsEXACT Therapeutics is a clinical-stage precision medicine company utilizing the power of ultrasound and microbubbles to enable targeted drug delivery in oncology. Acoustic Cluster Therapy (ACT®) follows a unique approach and may be applied to a wide range of therapeutic agents within oncology and across a multitude of other indications, including brain diseases. EXACT Therapeutics' shares are traded on Euronext Growth Oslo (EXTX). Further information may be found here: For further information, please contact: Per WaldayCEO EXACT TherapeuticsEmail: Forward looking statements:This announcement and any materials distributed in connection with this announcement may contain certain forward-looking statements. By their nature, forward-looking statements involve risk and uncertainty because they reflect the Company's current expectations and assumptions as to future events and circumstances that may not prove accurate. A number of material factors could cause actual results and developments to differ materially from those expressed or implied by this forward-looking 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
Business Wire
09-05-2025
- Business
- Business Wire
Neurogene Reports First Quarter 2025 Financial Results and Highlights Recent Updates
NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced first quarter 2025 financial results and highlighted recent corporate updates. 'We have made significant progress in planning for our future registrational trial with NGN-401 gene therapy for Rett syndrome and expect to provide an update on our plans during the first half of this year,' said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. 'As we work to finalize our proposed trial design to differentiate and position the product candidate as potentially best-in-class for future commercialization, we appreciate the continued collaboration with the FDA. We also advanced the Phase 1/2 trial of NGN-401, and we look forward to sharing updated interim clinical data from the trial in the second half of 2025.' First Quarter 2025 and Recent Highlights, and Anticipated Milestones Phase 1/2 Trial of NGN-401 Gene Therapy for Treatment of Rett Syndrome NGN-401 is currently being evaluated in an open-label Phase 1/2 trial. Key updates include: Remains on track to provide a regulatory update on registrational trial plans in the first half of 2025 Enrolling additional participants in the Phase 1/2 trial, and continues to expect to report additional interim clinical data from the trial in the second half of 2025; the trial includes a pediatric cohort of females aged 4-10 years old (N=8) and an older cohort of females aged 11 years and older (N=3) Announced a peer-reviewed publication in Science Translational Medicine providing proof-of-concept for Neurogene's EXACT™ transgene regulation technology and showing promising tolerability and efficacy in nonclinical studies compared to conventional gene therapy; the publication details EXACT's ability to regulate transgene expression to a tolerable and therapeutic level in non-clinical studies, which led to the development of NGN-401 Upcoming Events American Society of Gene and Cell Therapy (ASGCT) Annual Meeting: Fireside chat: Reviving Hope in Deprioritized Cell and Gene Therapy Programs at 3:45 p.m. CT on May 14 Panel presentation: FDA's START Pilot Program in Action: Insights from Year One at 3:45 p.m. CT on May 15 Oral presentation: Hemophagocytic Lymphohistiocytosis (HLH)/ Hyperinflammatory Syndrome Following High Dose AAV9 Therapy at 3:00 p.m. CT on May 16 H.C. Wainwright 3rd Annual BioConnect Investor Conference: Management will participate in a fireside chat at 9:30 a.m. ET on May 20 and participate in 1x1 meetings Goldman Sachs 46 th Annual Global Healthcare Conference: Management will participate in a corporate presentation at 8:00 a.m. ET on June 11 and participate in 1x1 meetings First Quarter 2025 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of March 31, 2025 were $292.6 million and are expected to provide runway into the second half of 2027, which would allow for the completion of enrollment of a future registrational trial for NGN-401 for Rett syndrome, chemistry, manufacturing and controls (CMC) scale-up to support NGN-401 registrational activities and further development of Neurogene's EXACT gene therapy pipeline. Research & Development (R&D) Expenses: R&D expenses were $17.8 million for the three months ended March 31, 2025 compared to $13.5 million for the three months ended March 31, 2024. The increase in R&D expenses for the three months ended March 31, 2025 was primarily driven by an increase in Rett syndrome clinical trial costs and employee-related expenses due to an increase in headcount. General & Administrative (G&A) Expenses: G&A expenses were $8.2 million for the three months ended March 31, 2025 compared to $5.2 million for the three months ended March 31, 2024. The increase in G&A expenses for the three months ended March 31, 2025 was primarily driven by an increase in employee-related expenses due to an increase in headcount and other corporate expenses. Net Loss: Net loss was $22.6 million for the three months ended March 31, 2025 compared to $16.9 million for the three months ended March 31, 2024. About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company's novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; trial designs, clinical development plans and timing for NGN-401, including anticipated timing of additional updates for a registrational trial of NGN-401 for Rett syndrome; expected timing for additional interim data from the Company's NGN-401 Phase 1/2 trial for Rett Syndrome, expected future interactions with or positions of the FDA; and the time period over which existing cash resources may be sufficient to fund the Company's operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as 'may,' 'will,' 'should,' 'would,' 'expect,' 'anticipate,' 'plan,' 'likely,' 'believe,' 'estimate,' 'project,' 'intend,' 'on track,' and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene's control. Such risks, uncertainties and assumptions include, among other things: risks related to the timing and success of enrolling patients in the Company's Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the expected timing and results of dosing of patients in the NGN-401 clinical trial; the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company's ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene's Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the Securities and Exchange Commission ('SEC') on May 9, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. Neurogene Inc. Condensed Consolidated Statements of Operations (In thousands of U.S. dollars, except share information) Three Months Ended March 31, 2025 2024 Operating expenses: Research and development expenses 17,760 13,541 General and administrative expenses 8,159 5,238 Total operating expenses 25,919 18,779 Loss from operations (25,919 ) (18,779 ) Other income, net 3,272 1,858 Net loss $ (22,647 ) $ (16,921 ) Per share information: Net loss per share, basic and diluted $ (1.08 ) $ (1.00 ) Weighted-average shares of common stock outstanding, basic and diluted 20,996,287 Expand
