Neuromyelitis optica spectrum disorder (NMOSD) is neuroinflammatory disease characterized by optic neuritis and myelitis with presence of anti-aquaporin-4 (AQP4) antibodies. Satralizumab is an anti-IL-6 receptor (IL-6R) humanized antibody approved for preventing relapses in NMOSD (AQP4 IgG positive) in adults and children in Japan.
Kyorin Pharmaceutical Co. Ltd. is licensing Hinge Bio Inc.’s multispecific antibody-based therapy HB-2198 in Japan for multiple autoimmune indications, starting with systemic lupus erythematosus.
Anaptysbio Inc. said it intends to split into two publicly traded companies – one for developing a pipeline, the other for managing royalties – each with its own business model. The royalty company will hold and manage the rights to big-earning Jemperli (dostarlimab) royalties from collaborator GSK plc, along with imsidolimab milestones and royalties from Vanda Pharmaceuticals Inc.
Rakuten Medical Inc. is advancing a pipeline of solid tumor therapeutics built on its Alluminox platform worldwide, having gained conditional early approval of ASP-1929, an Alluminox-derived photoimmunotherapy, in Japan in 2020.
Experts agree that the earlier Alzheimer's disease is detected, the sooner action can be taken. And so, the key to preventing deterioration is identifying the most effective early biomarkers, those that can spot the disorder and help halt its progression. Recent advances in the field have pushed a new era of early detection through blood-based biomarkers and personalized medicine strategies based on each patient’s genetic, immunological and clinical profile.
Experts agree that the earlier Alzheimer's disease is detected, the sooner action can be taken. And so, the key to preventing deterioration is identifying the most effective early biomarkers, those that can spot the disorder and help halt its progression. Recent advances in the field have pushed a new era of early detection through blood-based biomarkers and personalized medicine strategies based on each patient’s genetic, immunological and clinical profile.
Although the development pipeline for obesity treatments is expanding rapidly, weight loss is often accompanied by a concurrent reduction in lean muscle mass, highlighting the need for novel therapeutic approaches that promote fat loss while preserving muscle. Activin type II receptors (ACTRIIA/B) are regulators of muscle homeostasis, while the glucose-dependent insulinotropic polypeptide receptor (GIPR) plays a role in energy balance and adiposity.
Shape Therapeutics Inc. could bring in as much as $1.2 billion in a new option and license deal with Vectory Therapeutics BV. It’s another collaboration for both companies that are known for working with large and small pharmas. Vectory is getting the exclusive option to evaluate Shape’s brain-penetrating adeno-associated virus capsid, SHP-DB1, against three targets, including mHTT, TDP-43 for Huntington’s disease and phosphorylated tau for Alzheimer’s disease.
As Avidity Biosciences Inc. brought the second-largest follow-on offering of the year to the market, the company also released positive early and midstage stage results of del-zota, an antibody-oligonucleotide conjugate, in treating Duchenne muscular dystrophy. Phase I/II results showed a reversal of disease progression in patients who have been continuously treated for a year, plus improvements in several functional measures.
Autosomal dominant polycystic kidney disease (PKD) is a chronic and debilitating condition affecting over 12 million patients worldwide. PKD arises from mutations in the polycystin-1 (PC1) or PC2 genes. PKD is characterized by the formation of fluid-filled cysts in the kidneys, leading to inflammation, fibrosis and organ damage, ultimately resulting in renal failure.