Amicus licenses exclusive U.S. rights to commercialize DMX-200 for the treatmentof Focal Segmental Glomerulosclerosis (FSGS) DMX-200 is in a pivotal Phase 3 trial for FSGS, a rare and fatal kidney disease with no FDA-approved therapies Dimerix successfully completed Type C meeting with the FDA in March 2025, aligning on proteinuria as the primary endpoint for approval Dimerix to receive US$30 million (~AU$48 million) upfront payment, up to US$560 million (~AU$892 million) for success-based milestone paymen ...

Phase 2b/3 Alzheimer’s trial enrolment expected to reach 100 this quarter, triggering an interim analysis 6 months laterSYDNEY, April 30, 2025 (GLOBE NEWSWIRE) -- Actinogen Medical Limited (ASX: ACW) announces that its Alzheimer’s disease phase 2b/3 trial has now enrolled 60 participants and expects to reach 100 by the end of this quarter. That milestone will trigger an interim analysis of available trial data by an independent Data Monitoring Committee approximately 6 months later. Final results for the fu ...

Highlights Reported positive topline data for ATH434-201 randomized, double-blind Phase 2 clinical trial in Multiple System Atrophy (MSA) led by robust clinical efficacyPhase 2 data featured in an oral presentation at the American Academy of Neurology Annual MeetingATH434-202 open-label trial in advanced MSA completed in March 2025Cash balance on 31 March 2025 of A$17.96M with an additional A$27.1M raised subsequent to the end of the quarter MELBOURNE, Australia and SAN FRANCISCO, April 30, 2025 (GLOBE NEWS ...

NEW YORK, April 30, 2025 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders, today announced that it will present at The Citizens Life Sciences Conference, taking place in New York, NY, from M ...

KIRKLAND, Wash., April 29, 2025 (GLOBE NEWSWIRE) -- Kestra Medical Technologies, Ltd. (Nasdaq: KMTS) ("Kestra"), a wearable medical device and digital healthcare company, today announced the presentation of new real-world data on the ASSURE Wearable Cardioverter Defibrillator (WCD) at Heart Rhythm 2025, the annual meeting of the Heart Rhythm Society (HRS), held April 24–27 in San Diego. The abstract, titled "Real-world Experience with a Novel Wearable Cardioverter Defibrillator in a Community Setting: Advan ...

Core Insights - The year 2024 was a transformational period for Santhera Pharmaceuticals, marked by the successful launch of AGAMREE® for Duchenne muscular dystrophy (DMD) in Germany, Austria, and the U.S. [1][4][12] - The company secured financing of up to CHF 69 million, which is expected to support growth initiatives and provide liquidity until cash flow break-even in 2026 [1][10][67] - Santhera aims to expand its product portfolio and establish itself as a leading specialty pharmaceutical company in rare diseases [1][4] Operational Highlights - AGAMREE was launched in Germany and Austria, with over 300 patients on treatment by year-end, representing nearly 30% of those on steroid treatment [4][16] - Catalyst Pharmaceuticals launched AGAMREE in the U.S. in March 2024, achieving USD 46 million in revenue for the year, exceeding guidance [4][19] - The partnership with Sperogenix in China initiated an early access program, with commercial rollout expected in mid-2025 [4][20] Financial Highlights - Total revenue for 2024 was CHF 39.1 million, a decrease from CHF 103.4 million in 2023, primarily due to significant licensing milestones recognized in the previous year [4][49] - Product sales amounted to CHF 14.8 million, driven by the successful launch of AGAMREE in Germany and Austria [4][49] - Operating loss for 2024 was CHF 33.2 million, compared to a profit of CHF 68.8 million in 2023, influenced by the absence of prior out-licensing milestones [10][56] Market Access and Reimbursement - AGAMREE received full reimbursement in Germany and Austria, facilitating patient access [22][23][24] - In the UK, AGAMREE was recommended as a cost-effective treatment by NICE, with availability for prescription starting in April 2025 [25][18] - Ongoing negotiations for pricing and reimbursement in other EU countries are expected to progress through 2025 and 2026 [17][26] Partnerships and Licensing - Santhera's partnership with Catalyst Pharmaceuticals in the U.S. includes tiered royalties and potential milestone payments, enhancing market access [34][66] - The collaboration with Sperogenix in China is set to provide tiered royalties and milestone payments, supporting commercialization efforts [35][66] - Santhera is pursuing a hybrid model of direct commercialization in key markets while establishing distribution agreements in secondary territories [36][38] Manufacturing and Supply Chain - Santhera is validating additional manufacturing sites to meet global demand and improve supply chain efficiencies [39][40] - A secondary manufacturing site in the U.S. is being qualified to support growing demand and mitigate supply risks [41] - The company is also exploring local production options in China to enhance pricing and reimbursement discussions [42] R&D Strategy and Pipeline Development - Santhera will focus on maximizing AGAMREE's potential in DMD and generating long-term safety data, with results from the GUARDIAN study expected in Q4 2025 [46] - The company is actively seeking to expand its late-stage pipeline through licensing and distribution agreements, particularly in the rare disease field [47] Financial Guidance and Outlook - For 2025, Santhera expects total revenues in the range of CHF 65-70 million, driven by continued global rollout of AGAMREE [71] - The company anticipates operating expenses to be between CHF 50-55 million, excluding non-cash share-based compensation [71] - Long-term revenue guidance includes expectations of EUR 150 million by 2028, excluding milestones [72]

Core Insights - Silo Pharma, Inc. has filed a patent application for its neurology drug SPC-14, aimed at treating Alzheimer's disease, which is exclusively licensed from Columbia University [1][3] Patent Details - The patent titled "Compositions and Methods for the Treatment of Alzheimer's Disease and Other Neurogenerative Disease" focuses on SPC-14's mechanism of action, targeting glutamate receptor NDMAR and serotonin 5-HT4 to alleviate cognitive and neuropsychiatric symptoms in Alzheimer's disease [2][4] Drug Development - Early pre-clinical studies indicate that SPC-14 has the potential to improve cognitive memory function [3] - The drug has shown efficacy in preclinical studies against luteinizing hormone (LH) stress, reducing learned helplessness, perseverative behavior, and hyponeophagia, which is a measure of anxiety [4] Company Overview - Silo Pharma is a developmental stage biopharmaceutical company that focuses on addressing underserved conditions, including stress-induced psychiatric disorders, chronic pain, and central nervous system diseases [5] - The company's portfolio includes other innovative programs such as SPC-15 for PTSD and SP-26 for fibromyalgia and chronic pain, alongside preclinical assets targeting Alzheimer's disease and multiple sclerosis [5]

Initial real-world data from nearly 40,000 EYLEA HD patients will provide early insights on effectiveness of EYLEA HD in everyday clinical practice New indirect comparisons will evaluate EYLEA HD and faricimab on measures of efficacy, dosing frequency and potential economic benefits in patients with wet age-related macular degeneration and diabetic macular edema TARRYTOWN, N.Y., April 28, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the upcoming presentation of 27 ...

Core Viewpoint - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of VYVGART® (efgartigimod alfa) for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) in adult patients, marking a significant advancement in treatment options for this rare autoimmune disease [1][4]. Company Overview - argenx SE is a global immunology company focused on developing innovative treatments for severe autoimmune diseases, aiming to address significant unmet medical needs [2][9]. - The company has developed VYVGART, the first targeted IgG Fc-antibody fragment for CIDP, which, if approved, would be the first novel treatment for CIDP in Europe in over 30 years [2][5]. Clinical Trial Insights - The CHMP recommendation is based on positive results from the ADHERE clinical trial, which is the largest study of CIDP patients to date, involving 322 participants [3][5]. - In the ADHERE trial, 66.5% of patients treated with VYVGART showed clinical improvement, with a primary endpoint met demonstrating a 61% reduction in the risk of relapse compared to placebo [3][5]. - The trial also indicated significant functional improvements in various clinical assessment tools, with 99% of participants opting to continue in the open-label extension of the study [3][5]. Market Implications - The CHMP's positive opinion serves as a scientific recommendation for marketing authorization, with the European Commission expected to make a decision within approximately two months [4][5]. - If approved, VYVGART will be available for subcutaneous injection, providing a new treatment option for CIDP patients across all 27 EU member states, as well as Iceland, Norway, and Liechtenstein [4][5]. Disease Context - CIDP is a rare autoimmune disease affecting the peripheral nervous system, leading to symptoms such as fatigue, muscle weakness, and loss of sensation, which can significantly impair daily functioning [7]. - There are an estimated 31,413 individuals living with CIDP in the European Union, highlighting the need for effective treatment options [7].

- Label includes full spectrum of PKU patients -- European launch preparations underway -WARREN, N.J., April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive ...