Core Insights - The European Commission has approved VYVGART® (efgartigimod alfa) for subcutaneous injection as a monotherapy for adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins [1][4] - VYVGART is the first targeted IgG Fc-antibody fragment for CIDP and introduces a novel mechanism of action for CIDP treatment in over 30 years [2] - The approval is based on positive results from the ADHERE clinical trial, the largest study of CIDP patients to date, and is valid across all 27 EU Member States, as well as Iceland, Norway, and Liechtenstein [4] Company Overview - Halozyme Therapeutics, Inc. specializes in biopharmaceutical solutions aimed at improving patient experiences and outcomes, particularly through its ENHANZE® drug delivery technology [5] - The company has impacted over one million patients through its commercialized products and has licensed its ENHANZE® technology to major pharmaceutical companies including Roche, Takeda, and Pfizer [5] - Halozyme also develops drug-device combination products using advanced auto-injector technologies to enhance patient comfort and adherence [6] Strategic Partnerships - The approval of VYVGART represents a significant milestone in Halozyme's partnership with argenx, expanding access to CIDP treatment in Europe [3] - The company continues to collaborate with various pharmaceutical and biotechnology firms to leverage its ENHANZE® technology for improved drug delivery [5][6]

Core Insights - GH Research PLC has submitted a complete response to the FDA regarding the clinical hold of its Investigational New Drug Application (IND) for GH001, ahead of schedule [1][2] - The company is focused on developing GH001, a treatment for treatment-resistant depression (TRD), utilizing a proprietary inhalation method [3] Company Developments - The response to the FDA includes comprehensive data and completed toxicology studies, demonstrating the company's commitment to regulatory compliance and patient care [2] - GH001 has shown promising results in the Phase 2b GH001-TRD-201 trial, achieving a significant reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) score by -15.5 points compared to placebo on Day 8 (p<0.0001) [3]

VANCOUVER, British Columbia, June 20, 2025 (GLOBE NEWSWIRE) -- Rakovina Therapeutics Inc. (TSX-V: RKV) (FSE:7JO) (“Rakovina” or the “Company”) is pleased to announce that the effective date for the previously announced share consolidation (the “Consolidation”) will be June 24, 2025. As outlined in the Company’s news release dated May 15, 2025, the Consolidation will be conducted on the basis of one (1) post-consolidation common share (“Post-Consolidation Share”) for every ten (10) pre-consolidation common s ...

NEW YORK, June 20, 2025 (GLOBE NEWSWIRE) -- Leading securities law firm Bleichmar Fonti & Auld LLP announces that a lawsuit has been filed against Organon & Co. (NYSE: OGN) and certain of the Company’s senior executives for potential violations of the federal securities laws. If you invested in Organon you are encouraged to obtain additional information by visiting https://www.bfalaw.com/cases-investigations/organon-co-class-action. Investors have until July 22, 2025, to ask the Court to be appointed to lea ...

Company Overview - Thiogenesis Therapeutics, Corp. is a clinical-stage biopharmaceutical company focused on developing sulfur-based therapeutics for rare pediatric and inherited mitochondrial disorders [2][10] - The company is publicly traded on the TSX Venture Exchange and OTCQX [10] Clinical Programs - The company is advancing two lead Phase 2 clinical programs for its novel thiol drug, TTI-0102 [3] - A European multicenter trial for Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS), initiated on May 14, 2025 [3] - A U.S.-based trial for the Leigh Syndrome Spectrum (LSS), following FDA clearance of the Investigational New Drug (IND) application on June 11, 2025 [3] Drug Candidate - TTI-0102 is a next-generation thiol-based prodrug designed to enhance intracellular levels of glutathione and taurine, which are critical for mitigating oxidative stress in mitochondrial disorders [3][8] - The drug is engineered to address challenges faced by first-generation thiol-based drugs, including short half-life and adverse side effects [8] Mitochondrial Disorders - MELAS is characterized by symptoms such as seizures, muscle weakness, and can lead to loss of motor skills and intellectual disability, affecting approximately 4.1 in 100,000 of the population [7] - LSS is diagnosed in about 1 in 40,000 births and presents symptoms like impaired feeding, loss of motor skills, and seizures, with no current cure available [6] Conference Participation - The company’s CEO, Dr. Patrice Rioux, will participate in a clinical panel discussion at the United Mitochondrial Disease Foundation's 2025 Mitochondrial Medicine Conference, which gathers over 700 scientists and industry stakeholders [2][3]

Core Insights - Actuate Therapeutics, Inc. announced promising biomarker data from the Phase 2 trial of elraglusib in combination with gemcitabine/nab-paclitaxel for first-line metastatic pancreatic adenocarcinoma, highlighting the potential for improved patient stratification based on pre-dose plasma biomarkers [1][2][3] Group 1: Study Findings - The analysis of 40 cytokines, chemokines, soluble cell receptors, and growth factors identified 7 significant biomarkers predicting favorable survival in the elraglusib-treated cohort, particularly highlighting CXCL2's unique survival trend [2][3] - Univariate analysis showed that elevated levels of CXCL2 and TRAIL were associated with improved overall survival, while lower levels of CCL3, IL-1α, IL-18, TGF-β, and TRAIL R3 were linked to better survival outcomes [3] - Multivariate machine learning models were developed to accurately predict patients likely to survive over one year when treated with elraglusib and GnP [3] Group 2: Company Strategy and Future Plans - The results support the use of non-invasive blood-based markers to identify patients who may benefit from elraglusib, indicating a significant advancement in the company's biomarker strategy [4] - The company plans to prospectively test the identified biomarkers in future trials and focus on optimizing patient stratification and predictive accuracy through refined machine learning models [4] - Actuate aims to apply this biomarker identification approach to guide the development of elraglusib in metastatic pancreatic cancer and other advanced cancer indications [4] Group 3: Company Overview - Actuate Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for difficult-to-treat cancers, with elraglusib as its lead investigational drug targeting pathways involved in tumor growth and resistance to conventional therapies [5] - Elraglusib may also enhance anti-tumor immunity by regulating immune checkpoints and immune cell functions, indicating its potential in cancer treatment [5]

Core Viewpoint - Soleno Therapeutics, Inc. is actively participating in the 2025 United In Hope: International Prader-Willi Syndrome Conference, showcasing its commitment to advancing research and improving the lives of individuals affected by Prader-Willi syndrome (PWS) through multiple presentations [1][3]. Company Overview - Soleno Therapeutics focuses on developing novel therapeutics for rare diseases, with its first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, recently approved by the FDA on March 26, 2025 [11]. Conference Participation - The conference, anticipated to be the largest focused on PWS, is a collaboration between the Prader-Willi Syndrome Association | USA, the Foundation for Prader-Willi Research, and the International Prader-Willi Syndrome Organisation [2]. - Soleno will present one oral and six poster presentations, highlighting the significance of data and advocacy in addressing the needs of the PWS community [1][3]. Presentation Details - Key presentations include: - "Swallowability and Dosing Compliance of Diazoxide Choline Extended-Release (DCCR) Tablets in Patients with Prader-Willi Syndrome" [4]. - "Diazoxide Choline Extended-Release (DCCR) Tablets Significantly Reduce Hyperphagia in Patients with PWS Who Are Managed with Strict Food Controls" [4]. - "Long-term Efficacy Results of Diazoxide Choline Extended-Release (DCCR) Tablets in Participants with Prader-Willi Syndrome" [7]. - Presenters include experts from Queen Mary University of London and the University of Florida, indicating a strong academic collaboration [4][7][8]. About Prader-Willi Syndrome - PWS is a rare genetic neurodevelopmental disorder affecting approximately one in every 15,000 live births, characterized by hyperphagia, which can lead to severe health complications [9][10].

Core Insights - Telix Pharmaceuticals has launched a new PET radiochemistry solution called AlFluor™, based on 18F-aluminium fluoride, aimed at enhancing PSMA imaging capabilities [1][2]. Group 1: Product Development - The AlFluor™ platform allows flexible radiolabeling of PSMA with either AlF or gallium-68, and can also be used with ligands targeting neuroendocrine tumors and fibroblast activation protein [2]. - A strategic agreement has been signed with University Hospital Ghent and Ghent University for the development of a novel [18F]AlF-PSMA-11 targeting agent, which includes a comprehensive CMC package for a Drug Master File [3]. - The Phase 3 trial involving 96 prostate cancer patients showed that PSMA-11 labeled with 18F demonstrated diagnostic performance comparable to commercial 68Ga-labeled agents, achieving high specificity (~90%) for metastatic detection [4]. Group 2: Clinical and Regulatory Engagement - [18F]AlF-PSMA-11 has shown favorable biodistribution and high tumor-to-background ratios, with low off-target uptake in multiple studies, indicating its potential effectiveness [4]. - Telix is currently engaging with regulators to determine the approval pathway for [18F]AlF-PSMA-11, indicating progress towards commercialization [4]. Group 3: Company Strategy and Vision - The CEO of Telix emphasized the company's goal to expand PSMA-PET imaging through innovative and flexible product deployment, aligning with the evolving needs of physicians and patients [5]. - The introduction of AlFluor™ is part of Telix's strategy to offer a broad range of PSMA-PET imaging agents, enhancing service, flexibility, and reliability for customers [5]. Group 4: Company Overview - Telix Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing therapeutic and diagnostic radiopharmaceuticals, with operations in multiple countries including the U.S., Brazil, Canada, Europe, and Japan [6]. - The company is listed on both the Australian Securities Exchange and the Nasdaq Global Select Market, indicating its international presence and investment appeal [6].

Core Insights - Nirogacestat is poised to become the first and only approved therapy in the EU for desmoid tumors if the European Commission grants marketing authorization, with a decision expected in Q3 2025 [1][2] Company Overview - SpringWorks Therapeutics, Inc. is a biopharmaceutical company focused on severe rare diseases and cancer, currently advancing nirogacestat for desmoid tumors [2][10] - The company has previously received Orphan Drug designation for nirogacestat from the European Commission [3] Clinical Trial Results - The positive opinion from the EMA's CHMP is based on the Phase 3 DeFi trial, which demonstrated a 71% lower risk of disease progression for nirogacestat compared to placebo [3][6] - The DeFi trial involved 142 patients, with nirogacestat showing significant improvements in progression-free survival, objective response rate, and patient-reported outcomes [6] Safety Profile - Nirogacestat has a manageable safety and tolerability profile, with common adverse reactions including diarrhea, nausea, and fatigue [4] Disease Context - Desmoid tumors are rare, aggressive tumors with an estimated 1,300-2,300 new cases diagnosed annually in the EU, predominantly affecting individuals aged 20 to 44 [7][8] - Despite not metastasizing, desmoid tumors have high recurrence rates, leading to a shift in treatment guidelines recommending systemic therapies as first-line interventions [8]

Group 1: Conference Overview - The 2025 United In Hope: International Prader-Willi Syndrome Conference is the first-ever international meeting jointly hosted by the Prader-Willi Syndrome Association | USA, the Foundation for Prader-Willi Research, and the International Prader-Willi Syndrome Organisation [1][2] - The conference is expected to be the largest focused on Prader-Willi syndrome (PWS) ever held, taking place from June 24-28, 2025, in Phoenix, AZ [2] Group 2: Company Involvement - Soleno Therapeutics, Inc. announced one oral and six poster presentations at the conference, highlighting its commitment to advancing research and improving the lives of those impacted by PWS [1][3] - The company's first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, was approved by the U.S. FDA on March 26, 2025, for the treatment of hyperphagia in individuals with PWS [8] Group 3: Presentation Details - The oral presentation will cover long-term efficacy results of diazoxide choline extended-release tablets in participants with PWS from completed studies, presented by Evelien Gevers [4] - Multiple poster presentations will address various aspects of diazoxide choline extended-release tablets, including swallowability, dosing compliance, and the emotional impact of hyperphagia [4][5] Group 4: About Prader-Willi Syndrome - Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder caused by an abnormality in gene expression on chromosome 15, occurring in approximately one in every 15,000 live births [6][7] - The defining symptom of PWS is hyperphagia, which can lead to severe health issues and significantly diminish the quality of life for affected individuals and their families [7]