Ionis Pharmaceuticals is looking ahead to a Phase 3 study for an antisense medicine it is developing to treat Angelman syndrome, a rare neurodevelopmental disorder with no FDA-approved therapies. Its main competition is an Ultragenyx Pharmaceutical drug set to begin pivotal testing later this year. By Frank VinluanAn Ionis Pharmaceuticals drug in development for Angelman syndrome has mid-stage clinical trial results showing improvement across a range of measures of this rare neurodevelopmental disorder, and the company now plans to advance therapy to Phase 3 testing. Angelman is an inherited disorder that presents in infancy and leads to learning disability, muscle impairment, balance problems, and seizures. Patients typically develop little to no verbal ability. While Angelman patients can be treated with drugs that manage some of these symptoms, there are no FDA-approved therapies for the disease itself. The results announced Monday for the drug, ION582, come from the multiple-ascending dose portion of ...
On July 22, the first batch of SINOPHARM’s first antibody drug Sanglijian® (rituximab injection) was officially shipped, marking the company’s antibody drug formally entered a new journey of commercialization. Patient’s Gospel, First-line Targeted Drugs for Hematologic Tumors Lymphoma is a malignant tumor originated from lymphohematopoietic system, which belongs to immune system diseases. Malignant lymphoma is mostly non-Hodgkin’s lymphoma, accounting for about 80%-90% of all lymphoma cases. According to the data released by the National Cancer Center, the current incidence rate of lymphoma in China is about 6/100,000, with nearly 100,000 new cases every year, causing serious harm to patients’ lives, health and quality of life. Sanglijian® (rituximab injection) is mainly used for the treatment of non-Hodgkin’s lymphoma (follicular non-Hodgkin’s lymphoma, CD20-positive diffuse large B-cell non-Hodgkin’s lymphoma), chronic lymphoma cell leukemia (CLL) indications, and it is the first-line targeted drug of hematological tumors. According to the research, SUNLIFE® combined with CHOP ...
Recently, Hengrui Pharmaceuticals and its subsidiary Shandong Shengdi Pharmaceutical Co., Ltd. received the Notice of Approval for Clinical Trial of Drugs approved by the State Drug Administration, approving the company’s novel and highly selective CDK4/6 inhibitor, dalsiril hydroxyethanesulfonate tablets, combined with HRS-1358 tablets to carry out a clinical trial for breast cancer. Breast cancer is the number one malignant tumor affecting women worldwide. According to the Global Cancer Epidemiology Report 2024, the incidence and mortality rates of breast cancer rank first among female malignant tumors, with approximately 2.31 million new cases of breast cancer and 670,000 deaths globally each year, ranking first in terms of incidence and mortality rates of female malignant tumors [1]. In recent years, the incidence and mortality rates of breast cancer have shown a continuous upward trend in China, and it ranks fourth among the causes of cancer death. HRS-1358 is a novel, highly efficient and ...
Scientists from the University of Glasgow have developed the first bioengineered bone marrow model that supports human stem cells, which are crucial for bone marrow transplants and in vitro study work. Published in Nature Communications, the new model replicates the key aspects of the human bone marrow microenvironment to support rare long-term haematopoietic stem cells (LT-HSCs). Estimated to cause more than 300,000 deaths worldwide every year, leukaemia is a type of blood cancer that affects the white blood cells in the bone marrow, the spongy tissue found inside some bones, where the body makes blood cells. LT-HSCs are a type of cell that can replenish the blood cells after treatment for blood cancers, such as leukaemia. However, due to culturing challenges, researchers often rely on non-human animal models to test drugs that can have effects on blood cell production and target blood diseases. “Currently used animal models are poor predictors ...
Lupus is the lead autoimmune indication for Artiva Biotherapeutics, which has an early-stage clinical trial underway. Artiva’s allogeneic cell therapies are based on natural killer cells. By Frank VinluanCell therapy first reached patients as treatments for cancer. Artiva Biotherapeutics is part of a growing group of companies working to bring cell therapy to autoimmune disease, and its IPO has raised $167 million for clinical trial plans. Artiva priced the IPO at $12 per share, which was below the $14 to $16 per share price range the biotech set in preliminary terms last week. However, it boosted the deal size by increasing the number of shares in the offering. The 8.7 million shares it initially planned to offer would have raised $130.5 million at the proposed pricing midpoint. The company was able to raise more by selling 13.92 million shares. The shares of San Diego-based Artiva shares debuted on the Nasdaq ...
By Don Tracy, Associate Editor JAMA study aims to determine whether financial barriers impact access to mental health services.In the United States, a number of low-income adults struggle to gain access to appropriate mental health care. Despite efforts such as new legislations aimed at extending both private and public insurance through the Affordable Care Act (ACA), financial barriers are still an issue. In a study published in JAMA Psychiatry, researchers aimed to discover whether there was an association between medical debt and delayed or forgone mental health care among adults in the United States. The study included data from the 2022 National Health Interview Survey, which measured responses from 27,651 adults with current or lifetime diagnoses of depression or anxiety. Additionally, Asian, Black, and Hispanic households were oversampled to improve precision of estimates for these groups. To provide accurate results, mental health status was assessed for depression and anxiety, measured ...
Mushroom gummies being sold to promote brain function may contain the illegal hallucinogen psylocibin and other harmful ingredients not listed on the label, UVA Health experts are warning after five people – including a 3-year-old child – were sickened. A spate of cases seen in UVA Health Medical Center’s emergency department between September and June prompted poison experts with UVA’s Blue Ridge Poison Center to test five different brands of the products sold in Central Virginia gas stations and smoke shops. Of those, three contained psilocybin or psilocin, substances classified as “Schedule I” drugs by the U.S. Food and Drug Administration, meaning they have no medical use and high potential for abuse. Psilocybin and psilocin are illegal at both the federal and state level. All the products tested claimed to contain the Amanita muscaria mushroom, which is legal, or a proprietary mushroom blend. But none disclosed the inclusion of psilocybin ...
In a Perspective, Daniel Drucker highlights the growing body of evidence that hints at the potential of glucagon-like peptide-1 (GLP-1)-based medications in treating conditions other than diabetes and obesity, including cardiovascular disease and neurodegenerative disorders. GLP-1 is a hormone released from the gut after eating that enhances glucose-dependent insulin secretion. Pharmacological GLP-1 receptor (GLP-1R) activation reduces glucagon secretion and slows gastric emptying, making it an effective treatment for type 2 diabetes. Later studies found that GLP-1 administration also inhibited food intake through GLP-1R activation in the brain, leading to their use in treating obesity. Recently, research has demonstrated that GLP-1 drugs produce additional health benefits beyond glucose and weight control, including reduced heart and kidney diseases. Here, Drucker discusses the potential mechanisms underlying these benefits, such as reducing systemic inflammation, and their implications for future clinical applications and drug development. According to Drucker et al., GLP-1 drugs have shown promise ...
By Mike Hollan The network includes organizations that represent over 45,000 agents and brokers. Industry leaders are taking steps to combat unauthorized plan switching in the Affordable Care Act (ACA) Marketplace. Unauthorized switching occurs when a user’s plan or agent of record is changed without their knowledge or consent. This is a form of fraud and can cause users to face unexpected costs, along with other consequences. Insurers across multiple states have joined together to form a network to prevent users from facing unauthorized switching.1 These insurers include BluCross BlueShield of North Carolina, Group Health Cooperative of South Central Wisconsin, Highmark BlueCross Blue Shield, and others. The network is named the Member Defense Network and it will launch in Florida in July, 2024. According to a press release, the network’s partners represent over 45,000 insurance agents and brokers. The cause of unauthorized switching has been a topic of debate. In ...
By Mike Hollan The tech company’s data platform will be implemented into the genomic research department’s workflow. Genomics research is growing in importance for the pharma industry with each passing year. One issue that researchers face, however, is the significant amount of data that genomics research generates and requires. Not surprisingly, recent advancements in data technology have caught the eye of many in the life sciences industry. Texas A&M Health and BurstIQ announced that they have formed a partnership which will leverage BurstIQ’s LifeGraph management platform to improve genomic data governance. The Texas A&M Health PROVENANCE clinicogenomics registry programs will be the primary benefactors of this partnership. In a press release, Texas A&M’s executive director of clinical, translational, and industry collaborations at the Institute of Biosciences and Technology, Center for Precision and Genomic Medicine Rick Silva, PhD, said, “LifeGraph provides a unique foundation for a modern, data-driven, and scalable approach ...
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