Core Viewpoint - XORTX Therapeutics Inc. has received notification of a patent grant for "Xanthine Oxidase Inhibitor Formulations" by the European Patent Office, which enhances its intellectual property portfolio and supports its programs for gout and autosomal dominant polycystic kidney disease (ADPKD) [1][2]. Company Overview - XORTX is a late-stage clinical pharmaceutical company focused on developing therapies for gout and progressive kidney disease, with three clinically advanced products: XRx-026 for gout, XRx-008 for ADPKD, and XRx-101 for acute kidney injury [1][6]. Patent Details - The newly granted patent covers formulations using proprietary xanthine oxidase inhibitors (XOIs) aimed at treating health issues related to high uric acid levels, including gout, kidney stones, and cardiovascular diseases [2]. - With this patent, XORTX now holds five granted patents in the US and EU related to uric acid-lowering agents for gout and progressive kidney disease [2]. Market Opportunity - In North America, approximately 3.5 million people suffer from gout due to elevated uric acid levels, with XOIs being the preferred first-line treatment [5]. - Allopurinol is the most commonly prescribed XOI, with around 3 million prescriptions annually, but 3-5% of patients cannot tolerate it, creating a market opportunity for alternative XOIs like XRx-026 [5]. Health Implications of Hyperuricemia - An estimated 14% of the adult population has hyperuricemia, leading to various health issues, including gout, kidney stones, and cardiovascular diseases [3]. - Lowering uric acid levels in gout patients is strongly correlated with improved health outcomes, highlighting the need for effective treatments [3]. Genetic Factors and Precision Medicine - Recent studies suggest a link between genetic factors and the overexpression of xanthine oxidase, advocating for a precision medicine approach in treating conditions associated with hyperuricemia [4].

WATERTOWN, Mass., April 28, 2025 (GLOBE NEWSWIRE) -- Neumora Therapeutics, Inc. (Nasdaq: NMRA), a clinical-stage biopharmaceutical company with a therapeutics pipeline consisting of seven brain disease programs including two clinical-stage programs, today announced that it will host a conference call and live webcast at 4:30 p.m. ET on Monday, May 12, 2025 to report its first quarter 2025 financial results and provide a business update. A live webcast of the event will be available on the events and present ...

MARLBOROUGH, Mass., April 23, 2025 (GLOBE NEWSWIRE) -- Akoya Biosciences, Inc. (NASDAQ:AKYA) ("Akoya"), The Spatial Biology Company®, today announced a collaboration with the Singapore Translational Cancer Consortium (STCC) on the STCC Unified PD1/PDL1 Evaluation of Response (SUPER) study. This initiative aims to identify and validate key biomarkers predicting response or refractoriness to immune checkpoint inhibitors using Akoya's IO60 panel for spatial proteomic analysis. Advancing Precision Immunotherapy ...

Core Insights - Oculis Holding AG will host an R&D Day on April 15, 2025, to provide updates on its clinical candidates and future development strategies [1][2][3] Company Overview - Oculis is a global biopharmaceutical company focused on innovations for ophthalmic and neuro-ophthalmic diseases, with a pipeline that includes OCS-01 for diabetic macular edema (DME), Licaminlimab for dry eye disease (DED), and Privosegtor for acute optic neuritis [16] Clinical Development Updates - The R&D Day will feature updates on the Phase 3 DIAMOND trials for OCS-01 eye drops in DME, the development plan for Licaminlimab in DED, and expanded data from the ACUITY Phase 2 trial for Privosegtor [2][3] - OCS-01 is a high concentration dexamethasone eye drop aimed at providing a non-invasive treatment for DME, addressing limitations of current invasive treatments [4][5] - Licaminlimab is an anti-TNFα eye drop candidate showing positive effects in Phase 2 trials for DED, with a genetic biomarker identified for improved response [9][10] - Privosegtor has shown neuroprotective benefits in acute optic neuritis, with orphan drug designation from both the FDA and EMA [13][14] Market Need and Impact - DME affects approximately 37 million people globally, with projections to rise to 53 million by 2040, highlighting the urgent need for effective treatments [6][8] - DED impacts over 110 million people in G7 countries, with a significant unmet need as 87% of chronic patients remain unsatisfied with current treatments [11][12] - Acute optic neuritis affects up to 8 in 100,000 people and currently lacks specific neuroprotective therapies, indicating a substantial market opportunity for Privosegtor [15]

Core Insights - Oculis Holding AG will host an R&D Day on April 15, 2025, to provide updates on its clinical candidates and business strategy [1][2][3] Company Overview - Oculis is a global biopharmaceutical company focused on ophthalmic and neuro-ophthalmic diseases, with a pipeline that includes OCS-01 for diabetic macular edema (DME), Licaminlimab for dry eye disease (DED), and Privosegtor for acute optic neuritis [16] Clinical Development Updates - The R&D Day will feature updates on the Phase 3 DIAMOND trials for OCS-01, the development plan for Licaminlimab in DED, and the expanded data analysis from the ACUITY Phase 2 trial for Privosegtor [2][3] - OCS-01 is a high concentration dexamethasone eye drop aimed at providing a non-invasive treatment for DME, which currently affects approximately 37 million people globally and is expected to rise to 53 million by 2040 [4][6][8] - Licaminlimab is an anti-TNFα eye drop candidate that has shown positive effects in Phase 2 trials for DED, which impacts over 110 million people in G7 countries [9][11][12] - Privosegtor has demonstrated neuroprotective benefits in acute optic neuritis, a condition affecting up to 8 in 100,000 people worldwide, and has received orphan drug designation from both the FDA and EMA [13][15] Market Need - There is a significant unmet need for effective treatments for DME and DED, as current therapies often require invasive methods or do not adequately address patient needs [8][12]

Financial Data and Key Metrics Changes - INmune Bio raised $29.9 million from the sale of common stock and warrants in 2024, issuing a total of 4,145,978 shares and warrants for 3,898,852 shares [37][38] - The net loss attributable to common stockholders for the year ended December 31, 2024, was approximately $42.1 million, compared to approximately $30 million for 2023 [40] - Research and development expenses totaled approximately $33.2 million for 2024, up from approximately $20.3 million in 2023 [40] - Cash and cash equivalents at December 31, 2024, were approximately $20.9 million, with an additional $5.4 million raised since year-end [41] Business Line Data and Key Metrics Changes - The ADO2 trial for Alzheimer's disease is expected to announce top-line data in less than 100 days, focusing on neuroinflammation as a primary driver of the disease [8][14] - The INmune platform has pivoted to target solid tumors, with ongoing trials for castrate-resistant metastatic prostate cancer [17][18] - CORDStrom, a new therapeutic platform, is positioned to address systemic disease modification for RDEB, differentiating itself from local wound management therapies [22][24] Market Data and Key Metrics Changes - The ADO2 trial enrolled 208 patients across eight countries, with a focus on those with neuroinflammation driving their Alzheimer's disease [15] - The CaRe PC trial for prostate cancer is progressing, with completion of dosing in the Phase 1 part and ongoing Phase 2 dosing expected to complete by 2025 [18][43] Company Strategy and Development Direction - INmune Bio aims to challenge the amyloid-centric paradigm of Alzheimer's treatment by focusing on neuroinflammation [14][33] - The company is committed to advancing its three therapeutic platforms, with a focus on achieving regulatory milestones and potential commercialization [36][45] - The management emphasizes a precision medicine approach in clinical trials, particularly in the ADO2 trial [10][29] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming results from the ADO2 trial, viewing it as a potential catalyst for a paradigm shift in Alzheimer's treatment [34][35] - The company believes that addressing neuroinflammation could have broader implications for various CNS diseases [35] - Management highlighted the importance of safety in treating elderly patients with Alzheimer's, noting no significant adverse events reported thus far [31] Other Important Information - The company plans to file a BLA for CORDStrom in the first quarter of 2026, which would mark its first market therapeutic [44][112] - Management is focused on ensuring the quality of regulatory submissions to facilitate successful approvals [113] Q&A Session Summary Question: Is the 12-month open label trial for RDEB required for filing? - Management indicated that they believe the current data is adequate for a BLA submission, but the FDA will ultimately decide [50][51] Question: Will EMACC and CDR results be released at the same time? - Management confirmed that both EMACC and CDR results will be available at the time of data release [56][60] Question: Are there any dropouts in the EXPAREL Phase 2 trial? - Management reported that dropouts are within expected ranges, primarily due to typical issues associated with elderly patients [86] Question: How does the company plan to commercialize CORDStrom? - Management aims to move towards commercialization independently but may seek a partner closer to the launch [93] Question: Will the BLA for CORDStrom be filed in the UK and US? - Management confirmed that they expect to have all necessary data ready for filing in both regions by early 2026 [101][102]

INmune Bio Inc. (INMB) Q4 2024 Earnings Conference Call March 27, 2025 04:30 PM ET Company Participants Margo - Conference OperatorDavid Moss - Chief Financial Officer, ImmuneBioDr. RJ Tessie - CEO and Co-founder, ImmuneBioDr. CJ Barnum - Clinical Executive, ImmuneBioDr. Mark Liddell - Clinical Executive, ImmuneBioModerator - Call ModeratorCall Facilitator - Investor Relations Facilitator Conference Call Participants George Farmer - Analyst, ScotiabankTom Schrader - Analyst, BTIGDennis Resnick - Analyst (su ...

Core Insights - AbbVie is set to present new data from its early oncology research at the upcoming AACR Annual Meeting, focusing on investigational molecules ABBV-969 and ABBV-514 for hard-to-treat tumors [1][4][12] - The company aims to advance innovative therapies for patients with difficult-to-treat cancers, emphasizing the importance of early-stage research in addressing unmet medical needs [1][9] AbbVie’s Investigational Therapies - ABBV-969 is a dual-targeted antibody-drug conjugate (ADC) designed to target tumor cells expressing STEAP1 and/or PSMA antigens, currently in Phase 1 clinical trials for metastatic castration-resistant prostate cancer (mCRPC) [1][7][12] - ABBV-514 is a novel CCR8-targeting antibody that shows promise in enhancing anti-tumor immunity and is being evaluated in Phase 1 trials for non-small cell lung cancer (NSCLC) and head and neck cancer [1][7][12] Research Focus Areas - Presentations will include analyses of treatment resistance and biomarker discovery based on real-world data, which are critical for developing precision medicine [1][5][12] - A study will explore the overlap of folate receptor alpha expression in ovarian cancers, potentially aiding in treatment matching and sequencing [5][12] - Another study will utilize multi-omics approaches to understand long-term responses and resistance to immunotherapy in NSCLC [5][12] Clinical Trials and Presentations - ABBV-969 data will be presented in an oral session on April 27, 2025, highlighting its potential as a first-in-class treatment for advanced prostate cancer [3][4] - ABBV-514 data will be showcased in a poster presentation on April 29, 2025, focusing on its ability to deplete immunosuppressive Tregs in tumors [3][4] - Additional presentations will cover various aspects of cancer research, including germline variants and their association with patient prognosis [5][12]

Financial Data and Key Metrics Changes - The company ended the year with a cash balance of $4.6 million, with cash used in operations for Q4 2024 at $1.7 million and for the full year at $9.6 million, compared to $3 million and $15.8 million for the same periods in 2023, reflecting efforts to contain spending while advancing programs [27][28] - The net loss for the year ended 2024 was $10.8 million or $2.86 per share, compared to a net loss of $22.5 million or $6.54 per share in the prior year [28] Business Line Data and Key Metrics Changes - The company made significant progress in its clinical programs, particularly with the iNKT cell therapy, which has shown effectiveness in treating solid tumors and immune-driven diseases [11][12] - The collaboration with Autonomous Therapeutics aims to enhance the efficacy of iNKT cell therapy by integrating their encrypted RNA technology, targeting metastatic cancer with greater precision [9][10] Market Data and Key Metrics Changes - The company presented data at major conferences, demonstrating that its iNKT cell therapy enhances immune activation and overcomes resistance in challenging cancers, including gastric cancer [12][13] - The Phase 1 study of AgenT-797 in patients with severe acute respiratory distress showed an 80% survival rate, significantly higher than the 10% survival rate of in-hospital controls [20][21] Company Strategy and Development Direction - The company is focused on delivering scalable, durable, and effective allogeneic iNKT cell therapy to patients, with plans to advance multiple clinical programs in 2025, including gastric cancer and GvHD [5][31] - The addition of Dr. Robert Kadlec to the Board of Directors is expected to enhance the company's strategic depth in biodefense and pandemic preparedness [6][7] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the company's momentum entering 2025, highlighting a differentiated technology and a clear plan to reach the next value inflection point [26][32] - The company remains committed to operational efficiency and fiscal responsibility while advancing its innovative therapies [32] Other Important Information - The company has received probable funding from the National Institute of Allergy and Infectious Diseases (NIAID) to explore the activity of 797 in acute GvHD, with plans for a Phase 1 trial [23][24] - The company is advancing its PRAME-TCR program, which targets intracellular tumor antigens, demonstrating high specificity and potent tumor killing [15][18] Q&A Session Summary Question: Status of the Phase 2 study and focus for 2025 - Management confirmed that the majority of patients are enrolled in the Phase 2 study for gastric cancer, with data expected in the second half of the year, and emphasized the importance of advancing both gastric cancer and GvHD programs in 2025 [36][41] Question: KOL investigator feedback and potential for approval - Management noted positive feedback from key opinion leaders and emphasized the goal of accumulating data to demonstrate clinical benefits, with plans for regulatory discussions [47][53] Question: Timing of funding for GvHD study and cash runway - Management indicated that funding for the GvHD study is fluid but expressed optimism about securing it, while confirming that the company has cash runway through the end of 2025 [66][78]

Core Viewpoint - Alumis Inc. announced positive 52-week data from the open-label extension of its Phase 2 STRIDE clinical trial for ESK-001, indicating its potential as an effective oral therapy for moderate-to-severe plaque psoriasis [1][2][3] Group 1: Clinical Trial Results - Patients receiving 40 mg of ESK-001 twice daily showed sustained clinical responses at Week 52, with PASI 90 at 61.3% compared to 52.4% at Week 12, and PASI 100 at 38.8% compared to 26.8% [2] - At Week 52, 81.3% of patients reported improved control of itch (NRS≤4) and 61.3% reported enhanced quality of life (DLQI0/1) [2] - ESK-001 was well tolerated at Week 52, with safety profiles consistent with earlier data and no new safety findings reported [2][6] Group 2: Drug Profile and Mechanism - ESK-001 is a next-generation oral TYK2 inhibitor designed to correct immune dysregulation related to proinflammatory mediators such as IL-23, IL-17, and type 1 interferon [3][4] - The drug's selective targeting aims to maximize inhibition while minimizing off-target effects, positioning it as a potential best-in-class treatment for plaque psoriasis [4][6] Group 3: Ongoing and Future Studies - The Phase 3 ONWARD clinical program is currently underway, consisting of two parallel trials (ONWARD1 and ONWARD2) with approximately 840 patients each, comparing ESK-001 to placebo and apremilast [5][7] - Topline data from the ONWARD studies is expected in the first quarter of 2026, with ongoing patient enrollment for moderate-to-severe psoriasis [4][5] Group 4: Broader Development Strategy - Alumis is also developing a once-daily modified release formulation of ESK-001 and evaluating its application in systemic lupus erythematosus through the LUMUS Phase 2b trial [8] - The company leverages a precision data analytics platform to explore ESK-001's potential in other immune-mediated conditions, aiming to build a diverse pipeline of therapeutic options [9]