Durham, N.C., April 01, 2026 (GLOBE NEWSWIRE) -- Final participant randomized in the GenePHIT Phase 2 clinical trial assessing safety and efficacy of AB-1002 for adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptomsInitial results are expected in the first half of 2027Completion of enrollment marks significant milestone in progression of heart failure clinical program and included the largest number of participants to date to receive AB-1002 AskBio I ...

Astellas Pharma R&D Day Summary Company Overview - **Company**: Astellas Pharma - **Event**: R&D Day - **Key Speakers**: Naoki Okamura (CEO), Tadaaki Taniguchi (CRDO) Core Industry Focus - **Primary Focus Areas**: 1. Immuno-oncology 2. Targeted protein degradation 3. Blindness and regeneration 4. Genetic regulation Key Points and Arguments Vision and Strategy - Astellas aims to transform innovative science into value for patients, focusing on outcomes that matter to patients and the costs to the healthcare system [3][4] - The **Focus Area Approach** is employed to address high unmet medical needs, linking biology, modality, and technology [3][4] R&D Progress - Achieved proof of concept (POC) for three assets: ASP 2138, ASP 7317, and zitidagresib (ASP 3082) [5] - Over the past five years, Astellas accelerated its pipeline, achieving 12 phase II first subject doses and initiating one new phase III trial [5] - Terminated 21 clinical stage programs to reallocate resources to higher value assets, improving overall pipeline quality [6] Financial and Operational Strategy - Focus on maximizing sales of strategic brands to mitigate revenue decline from XTANDI's loss of exclusivity [8] - Implementing a new end-to-end operational model to enhance productivity and integrate research, development, commercialization, and lifecycle management [9] Pipeline Development - Building a robust pipeline with strategic brands, flagship programs, and follow-on programs [9][10] - Examples include the prostate cancer franchise and the acquisition of IZERVAY for geographic atrophy in age-related macular degeneration [10] Specific Program Updates - **Setidegrasib**: A targeted protein degrader for solid tumors with KRAS G12D mutations, showing promising results in pancreatic ductal adenocarcinoma (PDAC) and non-small cell lung cancer (NSCLC) [24][25] - ORR of 58.3% in PDAC and 37.5% in NSCLC with ongoing phase III trials [26][28] - **ASP2138**: A bispecific antibody targeting claudin 18.2 in gastric cancer, preparing for phase III trials [30] - **ASP7317**: Achieved POC for severe visual impairment in geographic atrophy, moving towards phase III [34] Future Outlook - Astellas plans to transform its R&D organization and governance structure to enhance productivity from 2024 to 2026, aiming for significant improvements in R&D productivity by 2030 to 2034 [15][36] - Continuous investment in cutting-edge technologies, including AI and robotics, to accelerate drug discovery and clinical trials [20][21] Additional Important Insights - Astellas emphasizes the importance of portfolio discipline, making strategic decisions to discontinue low-value projects [6][45] - The company is actively collaborating with external partners and academia to enhance its R&D capabilities [22] - Astellas aims to maintain a 30% operating margin sustainably while investing in R&D and managing expenses effectively [53][58] This summary encapsulates the key points discussed during Astellas Pharma's R&D Day, highlighting the company's strategic focus, R&D progress, and future outlook in the pharmaceutical industry.

Financial Data and Key Metrics Changes - Sangamo reported significant pipeline progress in 2025, including positive top-line results from the STAR study in Fabry disease, with a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients [5] - The company raised over $130 million in funding since the start of 2025 through non-dilutive license fees, milestone payments, and equity financing [6] Business Line Data and Key Metrics Changes - The STAR study for ST-920, an investigational gene therapy for Fabry disease, is in the rolling submission phase for a Biologics License Application (BLA) to the FDA, with both non-clinical and clinical modules submitted [8][9] - The company transitioned to a clinical-stage neurology company with six clinical sites activated in the phase I/II STAND study for chronic neuropathic pain [6] Market Data and Key Metrics Changes - The company received Fast Track designation from the FDA for ST-503, an investigational epigenetic regulator for intractable pain due to small fiber neuropathy (SFN), highlighting the high unmet patient need in this area [12] Company Strategy and Development Direction - Sangamo's primary focus is securing a commercial partner for Fabry disease, with ongoing discussions with multiple potential partners [16] - The company is also exploring additional capital-raising strategies and assessing all strategic options for its assets [16] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenges of regulatory and market uncertainty but expressed optimism about the progress made in the pipeline and the potential for ST-920 to transform Fabry disease treatment [5][15] - The company is managing cash carefully to ensure a sufficient runway for fulfilling partnership opportunities [20] Other Important Information - The company has made significant advancements in its neurology pipeline, including the completion of manufacturing and testing of process validation lots for ST-920 [9] - Sangamo's clinical data presented at the WORLDSymposium demonstrated improved kidney function and stabilization in cardiac function for Fabry disease patients [10] Q&A Session Summary Question: Can you provide more color on the revised timing for the Fabry BLA submission? - Management indicated that CMC activities are on the critical path for the filing, and they are following FDA guidance closely to ensure a successful submission [19][20] Question: What is the status of the Fabry partnership discussions? - Management confirmed ongoing discussions with multiple new partners and emphasized the complexity of the due diligence process [21][22] Question: Have there been any additional interactions with the FDA regarding the eGFR slope as a primary endpoint? - Management stated that they last spoke with the FDA in October and feel confident that the eGFR at one year could be used for accelerated approval [27][28]

Financial Data and Key Metrics Changes - Sangamo Therapeutics reported significant pipeline progress in 2025, including positive top-line results from the STAAR study in Fabry disease, with a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients [5] - The company raised over $130 million in funding since the start of 2025 through non-dilutive license fees, milestone payments, and equity financing [6] Business Line Data and Key Metrics Changes - The registrational Phase 1/2 STAAR study for ST-920, a gene therapy for Fabry disease, has shown improved kidney function and stabilization in cardiac function, marking a departure from the historical renal decline characteristic of the disease [10][11] - The company transitioned to a clinical-stage neurology company with six clinical sites activated in the Phase 1/2 STAND study for chronic neuropathic pain [6] Market Data and Key Metrics Changes - The company received Fast Track designation from the FDA for ST-503, an investigational epigenetic regulator for patients with intractable pain due to small fiber neuropathy (SFN), highlighting the high unmet patient need in this area [12] Company Strategy and Development Direction - Securing a commercial partner for Fabry disease remains the company's number one focus, with ongoing discussions with multiple potential partners [16] - The company is assessing all strategic options for its assets while seeking additional capital to ensure a long-term cash runway [16] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenges posed by regulatory and market uncertainty but expressed optimism about the progress made in the pipeline and the potential for ST-920 to fundamentally change Fabry disease treatment [15] - The company is managing its cash carefully to ensure the best possible runway for fulfilling a Fabry partnership [20] Other Important Information - The rolling submission of a Biologics License Application (BLA) for ST-920 is in progress, with both non-clinical and clinical modules submitted to the FDA [8] - The company has completed manufacturing and testing of process validation lots for ST-920, with plans to complete the BLA submission as early as summer 2026, subject to securing adequate funding [9] Q&A Session Summary Question: Can you provide more color on the revised timing for the Fabry BLA submission? - Management indicated that the CMC activities have been on the critical path for the filing, and they are following FDA guidance closely to ensure a successful submission [19] Question: What is the status of the Fabry partnership discussions? - Management confirmed ongoing discussions with multiple new partners and emphasized the complexity of the due diligence process [21][22] Question: Have there been any additional interactions with the FDA regarding the acceptance of the eGFR slope as a primary endpoint? - Management stated that the last interaction with the FDA was in October, where the agency reiterated that the eGFR could be used for accelerated approval [27]

Financial Data and Key Metrics Changes - Sangamo reported significant pipeline progress in 2025, including a positive top-line result from the STAR study in Fabry disease, with a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients [5] - The company raised over $130 million in funding since the start of 2025 through non-dilutive license fees, milestone payments, and equity financing [6] Business Line Data and Key Metrics Changes - The STAR study for ST-920, an investigational gene therapy for Fabry disease, is in the rolling submission phase for a Biologics License Application (BLA) to the FDA, with both non-clinical and clinical modules submitted [9] - The company transitioned to a clinical-stage neurology company with six clinical sites activated in the phase I/II STAND study for chronic neuropathic pain [5] Market Data and Key Metrics Changes - The company received Fast Track designation from the FDA for ST-503, an investigational epigenetic regulator for patients with intractable pain due to small fiber neuropathy (SFN) [12] - The STAR study data showed improved kidney function and stabilization in cardiac function, which is critical given the cardiovascular risks associated with Fabry disease [11] Company Strategy and Development Direction - Sangamo's primary focus is securing a commercial partner for Fabry disease, with ongoing discussions with multiple potential partners [17] - The company aims to manage its cash runway carefully while advancing its pipeline and fulfilling regulatory requirements for its therapies [16] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenges posed by regulatory and market uncertainties but expressed optimism about the progress made in the pipeline and the potential for ST-920 to transform Fabry disease treatment [5][16] - The company is committed to prudent financial management while seeking additional capital and strategic options for its assets [17] Other Important Information - The company has made significant advancements in its neurology pipeline, including the activation of additional clinical sites for ST-503 and the completion of preclinical studies for ST-506 [14] - Sangamo's collaborations with prominent companies in the neurology space highlight its position as a collaborator of choice for neurotropic capsids [16] Q&A Session Summary Question: Can you provide more color on the revised timing for the Fabry BLA submission? - Management indicated that CMC activities are critical for the filing and that they are following FDA guidance closely to ensure a successful submission [20][21] Question: What is the status of the Fabry partnership discussions? - Management confirmed ongoing discussions with multiple new partners and emphasized the complexity of the due diligence process [22] Question: Have there been any additional interactions with the FDA regarding the eGFR slope as a primary endpoint? - Management stated that the last interaction with the FDA was in October, where they reiterated that the eGFR could be used for accelerated approval [27][28] Question: Regarding prion disease and the STAC-BBB capsid, what is the timeline for clinical proof of concept? - Management clarified that the choice of AAV9 for neuropathic pain was based on prior knowledge and that they are pleased with the enthusiasm from partners regarding the STAC-BBB capsid [33][34]

Core Insights - Rocket Pharmaceuticals (RCKT) received FDA accelerated approval for its gene therapy Kresladi (marnetegragene autotemcel) to treat severe leukocyte adhesion deficiency-I (LAD-I), an ultra-rare genetic disorder [1][7] - Kresladi is the first gene therapy approved for children with severe LAD-I due to biallelic variants in ITGB2 without a matched sibling donor for allogeneic hematopoietic stem cell transplant [2][7] - Despite the approval, RCKT shares fell by 20% following the announcement [2][6] FDA Approval Details - The FDA approved Kresladi ahead of the expected decision date, which was initially set for March 28, 2026 [5][6] - The approval was based on the treatment's ability to increase specific immune cell markers (CD18 and CD11a), with long-term clinical benefits to be confirmed through further studies [6][8] - The company received a Rare Pediatric Disease Priority Review Voucher (PRV) as an incentive for developing treatments for rare diseases, which it plans to monetize for financial flexibility [7][8] Market Performance - RCKT's stock has increased by 7.4% year-to-date, contrasting with a 0.6% decline in the industry [4] - The stock's decline post-approval may reflect investor sentiment regarding the need for long-term data to validate the treatment's efficacy [6] Historical Context - Prior to the recent approval, the FDA had issued a complete response letter (CRL) in June 2024, requesting additional information on the Chemistry Manufacturing and Controls (CMC) related to Kresladi [9][10] - This was the second request for additional information from the FDA, indicating a challenging regulatory pathway for the product [10]

Company Overview - Proactive is a financial news publisher that provides fast, accessible, informative, and actionable business and finance news content to a global investment audience [2] - The company operates with a team of experienced and qualified news journalists across key finance and investing hubs including London, New York, Toronto, Vancouver, Sydney, and Perth [2] Market Focus - Proactive specializes in medium and small-cap markets while also covering blue-chip companies, commodities, and broader investment stories [3] - The content delivered by the team includes insights across various sectors such as biotech and pharma, mining and natural resources, battery metals, oil and gas, crypto, and emerging digital and EV technologies [3] Technology Adoption - Proactive is recognized for its forward-looking approach and enthusiastic adoption of technology to enhance workflows [4] - The company utilizes automation and software tools, including generative AI, while ensuring that all content is edited and authored by humans to maintain quality and best practices in content production [5]

Core Viewpoint - Rocket Pharmaceuticals' shares increased by 10% following the FDA's approval of Kresladi, the first gene therapy for a rare and often fatal childhood immune disorder [1][2]. Group 1: FDA Approval and Treatment Details - The FDA approved Kresladi for treating severe Leukocyte Adhesion Deficiency-I, a condition that severely impairs white blood cells' ability to reach infection sites, leading to high mortality rates among untreated patients [2]. - Kresladi utilizes a one-time treatment approach by collecting a patient's blood stem cells, correcting the faulty gene, and reinfusing the modified cells [3]. - The FDA granted accelerated approval based on a surrogate endpoint that measured improved immune cell function, highlighting the agency's regulatory flexibility for rare disease therapies [3][4]. Group 2: Market Implications and Analyst Insights - Jefferies analyst Andrew Tsai noted that the approval of Kresladi reduces risks associated with Rocket Pharmaceuticals' overall gene therapy platform, although the commercial potential of Kresladi may be limited [4]. - The approval was supported by early-to-mid-stage trial results showing a 100% survival rate at 12 months post-infusion, with no serious treatment-related side effects and a significant reduction in serious infections [4].

Core Insights - GenSight Biologics reported estimated full-year consolidated financial results for 2025, highlighting significant regulatory milestones and a strengthened financial foundation for future development [1][2][3] Financial Performance - Cash and cash equivalents as of December 31, 2025, were €2.4 million, a slight decrease from €2.5 million in 2024 [2][18] - Total assets decreased to €8.3 million in 2025 from €10.8 million in 2024, while total liabilities also decreased from €37.7 million to €33.2 million [4][32] - Operating income fell to €0.7 million in 2025 from €2.6 million in 2024, primarily due to a reduction in the research tax credit [5][8] - Research and development expenses decreased by 47.9% to €6.4 million in 2025, reflecting a strategic focus on prioritizing activities [9] - General and administrative expenses decreased by 12.5% to €4.7 million, demonstrating ongoing cost discipline [11] - The net loss for 2025 was €12.0 million, an improvement from a loss of €14.0 million in 2024, with loss per share decreasing from €0.15 to €0.08 [14][37] Cash Flow and Financing - Net cash flows from operating activities improved to €(9.2) million in 2025 from €(12.9) million in 2024, mainly due to reduced operating expenses [15] - Net cash flows from financing activities amounted to €8.9 million in 2025, down from €13.5 million in 2024, reflecting capital increases aligned with the company's financing strategy [17] - The company successfully completed multiple financing rounds in 2025, extending its cash runway into 2026 [2][3] Regulatory Milestones - 2025 marked a turning point for GenSight, achieving key regulatory milestones, including authorizations for early access programs in France, Israel, and the USA [2][3] - The ANSM granted Compassionate Use Authorization for the GS010/LUMEVOQ® gene therapy in France, with the first patient treatments commencing in March 2026 [19][20] Future Outlook - Management anticipates that revenues from early access programs in 2026 will cover operating expenses, excluding costs associated with the upcoming Phase III clinical trial [20] - Additional financing will be necessary to fully fund the RECOVER Phase III clinical trial, expected to start in the second quarter of 2027 [21]

Core Insights - MeiraGTx Holdings plc announced financial and operational results for Q4 and full-year 2025, highlighting significant advancements in their clinical programs and strategic collaborations [1][2]. Financial Performance - As of December 31, 2025, cash and cash equivalents were approximately $65.9 million, down from $105.7 million in 2024 [15]. - Total revenue for 2025 was $81.4 million, a significant increase from $33.3 million in 2024, primarily due to a $75 million upfront license fee from the collaboration with Eli Lilly [16][17]. - The net loss attributable to ordinary shareholders for 2025 was $114.2 million, or $1.42 per share, compared to a net loss of $147.8 million, or $2.12 per share in 2024 [25]. Clinical Developments - The FDA granted Breakthrough Therapy Designation for AAV2-hAQP1, aimed at treating Grade 2 and Grade 3 late xerostomia caused by radiotherapy for upper aerodigestive tract cancers [4][5]. - A program update for AAV2-hAQP1 is scheduled for April 16, 2026, where long-term data will be presented [6]. - The company is preparing to initiate a Phase 3 study for AAV-GAD in Parkinson's disease, supported by a strategic collaboration with Hologen AI, which includes a $200 million upfront payment [11][8]. Strategic Collaborations - MeiraGTx entered a collaboration with Eli Lilly for the AAV-AIPL1 program, receiving an upfront payment of $75 million and potential milestone payments exceeding $400 million [11][9]. - The partnership with Hologen AI aims to enhance clinical data analysis and support the development of the AAV-GAD program, with additional funding commitments of up to $230 million [11][8]. Research and Development - Research and development expenses for 2025 were $129.6 million, an increase from $119.5 million in 2024, driven by higher manufacturing costs and clinical program investments [21]. - The company is advancing its Riboswitch technology platform, with plans to submit an IND for Ribo-leptin, targeting metabolic diseases [19][20]. Market Position and Future Outlook - MeiraGTx is positioned as a leader in genetic medicines with a broad pipeline, including four late-stage clinical programs targeting inherited and common diseases [25]. - The company has developed comprehensive manufacturing capabilities, including five facilities globally, to support its clinical and commercial needs [25].