Curated news and analysis on clinical trials, drug approvals, and medical research.
Retrospective study analyzed longitudinal data from six immunotherapy-naive MuSK-MG patients over 2013-2024, finding significant positive correlation between MuSK antibody levels and disease severity as measured by MG-ADL scores. The correlation was specific to MuSK antibodies, with total IgG showing no independent relationship.
Clinical trials are offering new options for cancer patients, with some achieving long-term survival even with advanced disease. Precision medicine programs are expanding access to these trials by identifying patients with specific tumor mutations, reshaping oncology care.
Allarity Therapeutics will present a Trial-in-Progress poster at the ESMO Gynaecological Cancers Congress 2026 on its Phase 2 ovarian cancer trial of stenoparib. The company also dosed first patients in February 2026 in a VA-funded Phase 2 trial of stenoparib plus temozolomide for relapsed small cell lung cancer.
Recent research highlights advances in managing hemolytic disease of the fetus and newborn (HDFN). A meta-analysis found that combining phototherapy with certain adjunctive drugs like clofibrate may improve bilirubin reduction. Other studies emphasize the importance of preconception stability for women with rare diseases and the clinical impact of rare maternal antibodies.
A breakthrough ovarian cancer drug, mirvetuximab soravtansine, is now available on the NHS, offering extended survival and improved quality of life. The 'biological missile' therapy targets cancer cells directly, reducing side-effects compared to standard chemotherapy. Up to 400 patients in England could benefit each year.
The FDA has reversed its earlier rejection of UniQure's Huntington's disease therapy AMT-130, accepting existing trial data for accelerated review. The company plans to submit a marketing application in Q3 2026, ending a dispute over sham surgery requirements.
Immune checkpoint inhibitors are associated with a significantly lower risk of non-neovascular age-related macular degeneration, while sociodemographic disparities strongly influence diabetic retinopathy screening adherence, according to two separate ophthalmology studies.
The International Cardio-Oncology Society has released new guidelines for managing cancer therapy-related cardiac dysfunction, addressing educational gaps identified in a survey of 220 healthcare providers. The survey found highest educational needs for topics like immune checkpoint inhibitor-associated myocarditis and atrial fibrillation management.
Ocular Therapeutix stock surged 30% in premarket trading as the company prepared to release topline Phase 3 data for AXPAXLI in wet AMD. The SOL-1 trial results will be presented at the Macula Society meeting later in February. The company plans to file for FDA approval using one-year data if results are favorable.
The FDA issued final guidance advising drug companies to set duration limits for medically important antibiotics in livestock feed without legal obligation. Public health advocates criticize the guidance as insufficient to combat antibiotic resistance, which causes over 2.8 million infections and 35,000 deaths annually in the U.S.
The FDA agreed uniQure's existing trial data could support an accelerated approval filing for Huntington's therapy AMT-130, a reversal from earlier positions. Meanwhile, UK-based Harness Therapeutics selected HRN001 as its lead experimental candidate, targeting the FAN1 protein to address somatic expansion of Huntington's-causing genetic repeats.
The American College of Sports Medicine has published the first evidence-based exercise guidelines for children and adolescents with cancer, recommending safe, supervised activity during treatment. A new clinical trial is also evaluating structured exercise for young sarcoma patients undergoing chemotherapy to improve outcomes and quality of life.
NMD Pharma reported Phase 2a trial data showing ignaseclant improved muscle strength and motor function in adults with Charcot-Marie-Tooth disease, with benefits lasting seven days after treatment. The study met key secondary endpoints but missed its primary endpoint of six-minute walk test improvement. The drug has received FDA orphan drug designation for CMT.
Two neuroscience studies reveal brain mechanisms behind anxiety and compulsive behavior. One found restoring balance in an amygdala circuit reversed anxiety in mice, while another showed inflammation in the striatum makes behavior more deliberate.
The FDA has approved WAKIX for cataplexy in pediatric narcolepsy patients, making it the first non-scheduled treatment for all ages. Harmony Biosciences reported 2025 WAKIX revenue of $868.5 million and projects 2026 sales over $1 billion, nearing blockbuster status.
Acurx Pharmaceuticals is advancing ibezapolstat to international Phase 3 clinical trials for C. difficile infection following final regulatory guidance from FDA and EMA. The company launched a new clinical trial program for recurrent CDI and presented structural biology research demonstrating ibezapolstat's mechanism of action. Acurx reported year-end 2025 cash of $7.6 million.
Sensei Biotherapeutics has changed its name to Faeth Therapeutics following its February 2026 acquisition of the biotechnology company. The name change accompanies a $200 million private placement financing to advance PIKTOR, an investigational all-oral multi-node inhibitor targeting the PI3K/AKT/mTOR pathway in endometrial and breast cancer. Topline Phase 2 data in endometrial cancer is anticipated in the second half of 2026.
The U.S. FDA has accepted Gilead's application for an investigational once-weekly oral lenacapavir for HIV prevention, with a review date set for February 2027. Concurrently, PEPFAR and The Global Fund are expanding access to the twice-yearly injectable version to a total of 3 million people by 2028. Gilead will present new data on both lenacapavir and a novel bictegravir/lenacapavir combination at CROI 2026.
HHS and CMS issued a proposed rule to codify the Medicare Drug Price Negotiation Program, largely formalizing existing guidance. The rule includes new provisions, such as clarifications for new drug formulations. The proposal is now open for public comment.
The FDA has approved three new targeted therapies for different stages of advanced prostate cancer: Truqap for PTEN-deficient tumors, rucaparib for BRCA-mutated castration-resistant disease, and Akeega for BRCA2-mutated castration-sensitive cancer.
