• UC San Diego Health is among the first on the West Coast to offer Hemgenix, an FDA-approved gene therapy for hemophilia B, providing patients with a one-time infusion that reduces bleeding and increases clotting factor levels.
• A new nasal swab test offers new hope for asthma patients by enabling personalized and more effective treatments through the identification of asthma types.
• Bayer’s experimental drug elinzanetant significantly reduced hot flashes and improved sleep in women undergoing hormone therapy for breast cancer or at high risk of developing it, according to a late-stage trial.

Recent News

• Johnson & Johnson’s Spravato gains FDA nod to be used as a monotherapy
  Fierce Pharma – January 21, 2025
  Already on its way to becoming a blockbuster drug, Johnson & Johnson’s Spravato has received another potential boost as the FDA has blessed the nasal spray to be used as a monotherapy for major depressive disorder (MDD). Spravato was originally approved in 2019 to be used along with an oral antidepressant for patients who have not seen results with other antidepressant medications. In 2020, the U.S. regulator tacked on another nod for Spravato to be used by patients with MDD who experience acute suicidal thoughts or behavior. The standalone endorsement allows patients to use Spravato without taking oral antidepressants. Spravato can work as quickly as 24 hours, Bill Martin, Ph.D., who heads up J&J neuroscience, said in a release.
• Large study of Novo, Lilly weight loss drugs shows decrease in risk of Alzheimer’s
  Fierce Pharma – January 21, 2025
  As evidence has mounted that new diabetes and weight loss drugs can provide a variety of health benefits, the FDA has approved Novo Nordisk’s semaglutide for heart disease and Eli Lilly’s tirzepatide for sleep apnea. Now, as the companies run trials to flesh out the potential of the drugs to treat other conditions, an observational study from Washington University in St. Louis has shed more light on the benefits—as well as the risks—of using the metabolism-altering treatments. According to the study, published in Nature Medicine, researchers found that use of the new medicines may reduce the risk of Alzheimer’s disease and substance abuse disorders while increasing the risk of digestive issues and arthritis.
• Inflammatix collects FDA clearance for ER sepsis diagnostic test
  Fierce Biotech – January 21, 2025
  Inflammatix has secured the FDA’s blessing for its in vitro diagnostic system, aimed at rapidly profiling the body’s response to an infectious disease at the point of care. With results in about 30 minutes, the benchtop TriVerity test is designed to help emergency department clinicians determine whether a patient is fighting off a bacterial or viral infection and to see if they may be at risk for developing sepsis. Known as a host-response diagnostic, the cartridge-based blood test and Myrna instrument platform scans for about 30 genetic biomarkers that are activated by the body’s immune system as it fights off an invader.
• Brain-cell ‘periodic table’ for psychiatric disorders reveals new schizophrenia clues
  Stanford Medicine – January 20, 2025
  Stanford Medicine scientists are generating a periodic table of sorts for psychiatric disorders, providing better understanding of these conditions and paving the way toward targeted treatment. By combining two massive, publicly available databases — one flagging genes associated with psychiatric disorders, the other showing which cells in which parts of the human brain are making the most use of which of our genes — they’ve implicated certain cell types, located in particular brain regions, in schizophrenia. Like the periodic table of elements, which has enabled generations of scientists to predict the existence of yet-undiscovered elements and the behavior of those already known, the brain-cell classification system is the product of two sets of observations.
• The new immunotherapies changing the war on malignant melanoma
  UCLA Health – January 16, 2025
  While melanoma is relatively rare, accounting for only 1% of all skin cancer cases, rates of melanoma have been rising rapidly over the past few decades. It is responsible for the majority of skin cancer deaths, according to the American Cancer Society. But treatments have undergone a dramatic shift, thanks to groundbreaking advancements in immunotherapy. Today, many patients who would have faced an uncertain future are living longer, healthier lives, a transformation largely driven by pioneering research led by Antoni Ribas, MD, PhD. “When I started treating cases of melanoma that had metastasized to other organs, maybe 1 in 20 responded to treatment,” said Dr. Ribas, who directs the Tumor Immunology & Immunotherapy Program at the UCLA Health Jonsson Comprehensive Cancer Center.
• Lilly’s Omvoh approved by FDA for Crohn’s
  BioPharma Dive – January 16, 2025
  Omvoh is part of a slate of new medicines designed to build on Lilly’s success with diabetes and obesity treatments that have boosted earnings and sent its shares soaring. The company won approval in colitis in October 2023, after an initial rejection in April 2023 based on manufacturing issues. Drugmakers have been pouring money into the gastrointestinal field in recent years and expanding the use of blockbusters such as AbbVie’s Skyrizi to include colitis and Crohn’s disease. Like Skyrizi, Omvoh is designed to target a specific subunit of the IL-23 protein to prevent inflammation. J&J’s Tremfaya also blocks IL-23 while binding to the CD64 receptor on cells that produce IL-23 as well.
• Drug in clinical trials for breast cancer could also treat some blood cancers
  Medical Xpress – January 16, 2025
  Two new studies led by researchers at Washington University School of Medicine in St. Louis have identified a possible way to block the progression of several forms of blood cancer using a drug already in clinical trials against breast cancer. The studies found that inhibiting a protein called RSK1 reduces inflammation and stops the progression of blood cancers called myeloproliferative neoplasms (MPNs) as well as an aggressive form of acute myeloid leukemia (AML). With the RSK1 inhibitor already in clinical testing, the path to expanded use as a treatment for blood cancers could be accelerated. In humans, MPNs can be slow-growing blood cancers that simmer for years. Doctors can monitor the disease and treat symptoms, but there is no reliable way to cure it or slow progression.
• Bayer says menopause drug succeeds in breast cancer study
  BioPharma Dive – January 9, 2025
  So-called vasomotor symptoms are typically associated with menopause and, in over one third of menopausal women, can be severe, according to Bayer. But they’re also a side effect of endocrine therapies used to treat or prevent the most common form of breast cancer, which can impact adherence to those medicines. Bayer believes elinzanetant may be helpful in treating both groups of women and, through four Phase 3 trials, has now accumulated enough evidence to suggest it can. In the latest study, Bayer said elinzanetant met all of its main and secondary objectives. Though it didn’t provide specifics, the company said elinzanetant reduced the frequency and severity of hot flashes versus placebo at four and 12 weeks.
• UC San Diego Health First to Offer Novel Gene Therapy for Hemophilia B
  UCSD Health – January 8, 2025
  UC San Diego Health is one of a few health care systems on the West Coast to offer a new gene therapy for hemophilia B, a bleeding disorder that prevents blood from clotting properly. The FDA-approved therapy is a one-time infusion that leads to reduced bleeding and elevated levels of clotting factors in the blood. The treatment replaces a lifetime of at-home infusion, providing patients with hemophilia the ability to live an improved quality of life without the pain and burden of self-infusions and side effects. “Recent advancements in gene therapy have revolutionized how we think about hemophilia treatment,” said Annette von Drygalski, MD, PharmD, director of the Center for Bleeding and Clotting Disorders at UC San Diego Health.
• Novel test can detect different types of asthma via nasal swab
  NBC News – January 2, 2025
  For many children diagnosed with asthma, pinpointing the particular nature of their case and finding appropriate treatment is far from simple. The lung disease, which affects more than 4.6 million children in the U.S., is often broken down into two categories. The first, known as “T2-high” asthma, is caused by inflammation from a particular type of immune system cell called T helper 2. Until recent years, it was assumed to be the most common form. The second category, “T2-low” asthma, is essentially a catchall for other forms. It encompasses two subtypes — one involving less inflammation, and another characterized by inflammation from a different kind of T cell. In some moderate or severe cases, figuring out which type of asthma a child has can help doctors match patients with proper treatment.
• Blood test can predict how long vaccine immunity will last, Stanford Medicine-led study shows
  Stanford Medicine – January 2, 2025
  When children receive their second measles-mumps-rubella vaccine, around the time they start kindergarten, they gain protection against all three viruses for all or most of their lives. Yet the effectiveness of an influenza vaccine given in October starts to wane by the following spring. Scientists have long been stymied by why some vaccines can coax the body to produce antibodies for decades, while others last mere months. Now, a study led by researchers at Stanford Medicine has shown that variation in vaccine durability can, in part, be pinned on a surprising type of blood cell called megakaryocytes, typically implicated in blood clotting. “The question of why some vaccines induce durable immunity while others do not has been one of the great mysteries in vaccine science,” said Bali Pulendran, PhD, a professor of microbiology and immunology.