Core Insights - Neurocrine Biosciences announced significant reductions in cognitive and motor-related burdens in adults with Huntington's disease chorea treated with INGREZZA (valbenazine) capsules, based on new patient-reported post-hoc data from the KINECT-HD study [1][4] Group 1: Study Findings - The KINECT-HD study is the first clinical trial to show measurable changes in cognitive and motor disease burden with a vesicular monoamine transporter 2 inhibitor in Huntington's disease patients [1][2] - In the 12-week Phase 3 KINECT-HD clinical trial, INGREZZA showed a significant reduction in chorea severity, with a placebo-adjusted mean reduction in the Total Maximal Chorea (TMC) score of 3.2 units (p<0.0001) [11] - Patient-reported analyses indicated greater reductions in cognitive and motor-related disease burden for INGREZZA compared to placebo across various domains, including memory loss and mobility limitations [3][4] Group 2: Cognitive and Motor-Related Improvements - Statistically significant improvements were observed in cognition-related items, such as memory loss (-0.9 for INGREZZA vs -0.2 for placebo) and decision-making abilities (-1.0 for INGREZZA vs -0.3 for placebo) [3] - For motor-related items, INGREZZA demonstrated larger reductions in scores compared to placebo, including abnormal movements (-1.5 for INGREZZA vs -0.8 for placebo) and impaired coordination (-1.0 for INGREZZA vs -0.4 for placebo) [3][5] Group 3: Background on Huntington's Disease - Huntington's disease is a hereditary progressive neurodegenerative disorder affecting approximately 41,000 adults in the U.S., characterized by motor, cognitive, and psychiatric symptoms [7] - Chorea, a common symptom of Huntington's disease, involves irregular and unpredictable movements that can interfere with motor coordination and daily activities [7] Group 4: About INGREZZA - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating chorea associated with Huntington's disease and tardive dyskinesia [14][15] - The drug is designed for once-daily administration without the need for titration, making it convenient for patients [16]

TARPON SPRINGS, Fla., June 27, 2025 -- Allarity Therapeutics, Inc. (“Allarity” or the “Company”) (NASDAQ: ALLR), a Phase 2 clinical-stage pharmaceutical company dedicated to developing stenoparib—a differentiated, dual PARP and WNT pathway inhibitor today announced that the second patient has been dosed in its new Phase 2 clinical trial protocol evaluating stenoparib in patients with advanced, recurrent, platinum-resistant or platinum-ineligible ovarian cancer. Commenting on the development, Thomas Jensen ...

BERKELEY HEIGHTS, N.J., June 27, 2025 (GLOBE NEWSWIRE) -- CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for life-threatening diseases and conditions, today announced the pricing of its previously announced underwritten public offering of 6,604,507 shares of its common stock. The total gross proceeds from the offering to the Company are expected to be approximately $85 million. In addition, CorMedix granted the underwriters a 30-day o ...

Core Insights - Ultragenyx Pharmaceutical Inc. has received Breakthrough Therapy Designation from the FDA for GTX-102 as a treatment for Angelman syndrome [1][2] - The designation highlights the urgent need for effective treatments and the promising clinical results observed with GTX-102 [2] - The Phase 3 Aspire study is currently enrolling participants, with plans for an additional Aurora study to evaluate GTX-102 in other genotypes and ages expected to start later in 2025 [3] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing therapies for serious rare and ultra-rare genetic diseases [7] - The company has a diverse portfolio of approved medicines and treatment candidates aimed at addressing high unmet medical needs [7] - Ultragenyx's management team has extensive experience in the development and commercialization of rare disease therapeutics [8] Product Information - GTX-102 (apazunersen) is an investigational antisense oligonucleotide therapy designed to reactivate expression of the UBE3A gene [4] - The therapy has received multiple designations from the FDA, including Orphan Drug Designation and Fast Track Designation [4] - The Phase 1/2 study demonstrated consistent developmental gains in 74 patients aged 4-17 with a full maternal UBE3A gene deletion [2] Angelman Syndrome Insights - Angelman syndrome is a rare neurogenetic disorder affecting approximately 60,000 people in commercially accessible geographies [5] - The disorder is characterized by cognitive impairment, motor impairment, and seizures, requiring continuous care for affected individuals [6] - There are currently no approved therapies for Angelman syndrome, but animal models suggest potential for symptom improvement at any age [6]

Core Insights - HCW Biologics has developed second-generation, multi-specific T-cell engagers targeting pancreatic cancer, utilizing its proprietary TRBC product discovery platform, which activates T cells and reduces immunosuppression in the tumor microenvironment [1][2][3] Company Overview - HCW Biologics Inc. is a clinical-stage biopharmaceutical company focused on developing novel immunotherapies aimed at chronic inflammation and age-related diseases, with a belief that their products can significantly change cancer treatment and improve patients' quality of life [4][5] - The company has created over 50 molecules using the TRBC platform, which allows for the construction of various classes of immunotherapeutic compounds, including multi-functional immune cell stimulators and multi-specific targeting fusions [5] Product Development - The lead product candidate, HCW9302, was developed using the legacy TOBI platform, while the TRBC platform is designed to create immunotherapeutics that activate immune responses and specifically target cancerous cells [4][5] - The two lead T-cell engagers target tissue factor and mesothelin, demonstrating potent anti-pancreatic cancer activities in preclinical studies, with 100% survival in treated tumor-bearing mice [2][3]

SAN FRANCISCO and SUZHOU, China, June 27, 2025 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncologic, autoimmune, cardiovascular and metabolic, ophthalmologic and other major diseases, announces that China's National Medical Products Administration (NMPA) has approved mazdutide, a first-in-class dual glucagon (GCG)/glucagon-like peptide-1 (GLP-1) recepto ...

Core Viewpoint - Biocon Biologics Ltd. has received a Notice of Compliance from Health Canada for its biosimilar YESAFILI (aflibercept), marking it as the first biosimilar to EYLEA approved in Canada, with a launch scheduled for July 4, 2025 [1][3]. Company Overview - Biocon Biologics Ltd. is a fully integrated global biosimilars company and a subsidiary of Biocon Ltd., focused on providing affordable access to high-quality biologics [1][10]. - The company has commercialized nine biosimilars and has a pipeline of 20 biosimilar assets across various therapeutic areas, including diabetology, oncology, and ophthalmology [11]. Product Details - YESAFILI is a vascular endothelial growth factor (VEGF) inhibitor indicated for the treatment of several serious retinal diseases, including neovascular age-related macular degeneration and diabetic macular edema [6][4]. - The approval of YESAFILI was based on comprehensive data demonstrating its similarity to EYLEA in terms of quality, safety, and efficacy, with no clinically meaningful differences observed in a Phase 3 study [2][4]. Market Impact - The approval of YESAFILI is seen as a significant achievement for Biocon Biologics in Canada, enhancing access to advanced biologic therapies for Canadian patients and ophthalmologists [3][10]. - The launch of YESAFILI will be the company's 10th biosimilar to be commercialized globally, reflecting its commitment to expanding access to affordable biologics [3][11].

Core Viewpoint - The article discusses a class action lawsuit against Sarepta Therapeutics, alleging violations of the Securities Exchange Act of 1934 due to misleading statements regarding the safety and efficacy of its gene therapy product, ELEVIDYS, for Duchenne muscular dystrophy [1][3]. Company Overview - Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on developing therapies for Duchenne muscular dystrophy, including the gene therapy ELEVIDYS [2]. Allegations of the Lawsuit - The lawsuit claims that Sarepta and its executives made false or misleading statements about ELEVIDYS, including significant safety risks, failure to detect severe side effects in trials, and the potential for halting recruitment and dosing due to adverse events [3]. - Specific incidents cited include: - On March 18, 2025, a patient treated with ELEVIDYS suffered acute liver failure leading to death, causing Sarepta's stock to drop over 27% [4]. - On April 4, 2025, Sarepta disclosed that EU authorities requested a review of the death, leading to a further stock decline of over 7% [5]. - On June 15, 2025, a second patient died from acute liver failure, prompting Sarepta to suspend shipments and pause dosing, resulting in a stock price drop of over 42% [6]. - On June 24, 2025, the FDA announced an investigation into the risk of acute liver failure associated with ELEVIDYS, causing an additional stock decline of over 8% [7]. Legal Process - Investors who purchased Sarepta securities during the class period can seek appointment as lead plaintiff in the lawsuit, which allows them to act on behalf of other class members [8].

Core Viewpoint - CorMedix Inc. plans to offer and sell $85.0 million of its common stock in an underwritten public offering, with a potential additional 15% option for underwriters [1][2] Group 1: Offering Details - The offering is subject to market conditions, and there is no assurance regarding its completion or terms [1] - RBC Capital Markets is acting as the sole bookrunner for the offering [2] - The net proceeds will be used for general corporate purposes, including working capital and research expenses [2] Group 2: Regulatory Information - The securities are being offered under a shelf registration statement filed with the SEC on May 6, 2024, and declared effective on May 22, 2024 [3] - A preliminary prospectus supplement and accompanying prospectus will be filed with the SEC and available on their website [4] Group 3: Company Overview - CorMedix is focused on developing therapeutic products for life-threatening conditions, with its lead product DefenCath® approved by the FDA on November 15, 2023 [6] - DefenCath was commercially launched in inpatient settings in April 2024 and in outpatient settings in July 2024 [6] - The company plans to commence clinical studies in Total Parenteral Nutrition and Pediatric populations in 2025 [6]

Shares in biopharmaceutical company Viking Therapeutics (VKTX 5.35%) were up by 6.3% at 12:30 a.m. ET today. The move likely stems from the market digesting the previous day's news that Viking had initiated a phase 3 trial of its most promising program in its pipeline, namely VK2375, a treatment for metabolic disorders, including obesity.Why VK2375 is exciting investorsThere are probably two key reasons why the market likes the initiation of a phase 3 trial:VK2375 is a dual GLP-1/GIP receptor agonist simila ...