MASSACHUSETTS GENERAL HOSPITAL

Pfizer's sudden withdrawal of Oxbryta, a sickle cell disease drug, raises concerns over patient trust and treatment safety. The abrupt removal, following European regulatory flags, poses risks like severe anemia if patients stop abruptly. Pfizer's lack of transparency and the FDA's limited notice add to the uncertainty, potentially impacting future sickle cell treatments and clinical trials.

The FDA approved Cobenfy, a new antipsychotic medication for schizophrenia, developed by Karuna Therapeutics. Despite its efficacy in clinical trials, concerns about insurance coverage and high list price ($22,500/year) may limit patient access. Bristol Myers Squibb, which acquired Karuna, plans to expand Cobenfy's use to bipolar disorder and Alzheimer's agitation, with ongoing studies.

OSE Immunotherapeutics reports H1 2024 results with €82.5 million income, strategic partnerships with AbbVie and Boehringer Ingelheim, and €80.7 million cash position. Clinical highlights include positive Phase 1/2 results for OSE-279 and Lusvertikimab, and the launch of the Artemia Phase 3 study for Tedopi®.

FDA approves AstraZeneca's Tagrisso for stage 3 EGFR-mutated NSCLC previously treated with chemoradiotherapy, based on LAURA trial showing 84% reduction in disease progression risk.

Weekend warrior workouts offer similar health benefits to daily exercise, reducing risk of over 200 diseases, according to a study in 'Circulation'. Both patterns lower risks for conditions like high blood pressure and diabetes, suggesting total activity volume is key.

Inebilizumab-cdon shows efficacy in treating IgG4-related disease and generalized myasthenia gravis in phase 3 trials, reducing flare risk by 87% and improving daily activities, with no new safety signals identified.

Harvard's Joel F. Habener wins 2024 Lasker-DeBakey Award for discovering GLP-1 hormone, key to diabetes drugs like Ozempic. Habener, Svetlana Mojsov, and Lotte Bjerre Knudsen's work led to treatments revolutionizing obesity and type 2 diabetes. GLP-1 receptor antagonists offer benefits beyond blood sugar control and weight loss, potentially treating heart disease, inflammatory disorders, addictions, and depression.

Steritas LLC's pGTI will be used in the STAR-JIA trial to compare efficacy, safety, and cost-effectiveness of intravenous vs. oral glucocorticoids for JIA in children. The trial aims to assess steroid toxicity and is funded by the National Institute for Health and Care Research.

ARS Pharmaceuticals announces the U.S. availability of neffy® (epinephrine nasal spray) for treating Type I Allergic Reactions, including anaphylaxis, in adults and children weighing ≥30 kg. neffy is accessible through various programs, including a $25 co-pay for insured patients and a $199 cash price for uninsured patients. ARS also plans to expand access to EURneffy® in the EU by Q4 2024 and has submitted an FDA application for pediatric use.

Jason Efstathiou discusses the PARTIQoL trial, comparing proton beam therapy to IMRT for localized prostate cancer, focusing on patient-reported outcomes and bowel function at 24 months. The trial, involving 450 patients from 29 centers, employs innovative strategies to overcome accrual challenges, emphasizing rigorous data generation to assess proton therapy's benefits. Despite proton therapy's potential advantages, its limited availability and high costs in the U.S. highlight the need for comparative studies like PARTIQoL.