Ultragenyx’s Setrusumab falls short in Phase 3 osteogenesis imperfecta trials
The trials did not show a statistically significant reduction in annualized clinical fracture rates compared to placebo or bisphosphonates
The trials did not show a statistically significant reduction in annualized clinical fracture rates compared to placebo or bisphosphonates
The study will run across 18 sites and enroll roughly 200 patients over 24 months
Applications include detailed scientific rationale and supporting clinical evidence
The applications aim to launch Phase Ib/II trials for relapsed/refractory multiple myeloma and primary plasma cell leukemia, respectively
The interim efficacy readout, involving around 170 participants with 15-month data, remains on track for the end of March 2026
The study met its primary endpoint with flying colors
Despite some promising subgroup signals, the company indicated it will not pursue further psychiatric trials
CIRRUS-HCM, an open-label, multi-part study, evaluates EDG-7500 in patients with obstructive and nonobstructive HCM
CX-5461 is “the world's first and most advanced G-quadruplex (G4) stabilizer in development
Lucid-MS represents a potential breakthrough in the treatment of multiple sclerosis
The drug, currently in a phase 1b/2 clinical trial for small cell lung cancer, is a polymer nanoparticle formulation of SN-38
CLE, a common form of lupus erythematosus, primarily causes diverse skin lesions
The multicenter study will enroll 75 patients across 16 sites in Germany, Bulgaria, Poland, and Spain
Study published in laboratory investigation, the official journal of the United States and Canadian Academy of Pathology
Under the 2023 collaboration agreement, Assembly Bio will receive $35 million for Gilead’s exercise of the HSV program option
Adverse events were consistent with valbenazine’s established safety profile
The study focused on patients without actionable genomic alterations whose disease had progressed after prior immunotherapy and platinum-based chemotherapy
The trial will evaluate the efficacy and safety of IPX203 versus immediate-release levodopa/carbidopa
The therapy also drove additional weight loss at the higher 1.8 mg dose without plateauing and maintained a favorable tolerability profile
FOP is a rare genetic disorder caused by mutations in the ALK2 kinase
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