Investigational Therapy
FDA panel supports imetelstat for anemia in lower-risk myelodysplastic syndrome
An FDA advisory committee voted 12-2 that the benefits of the investigational therapy imetelstat outweigh the risks for the treatment of transfusion-dependent anemia in certain adults with myelodysplastic syndromes.
Dosing begins in phase 2 study of Duchenne muscular dystrophy therapy for boys
A clinical-stage RNA medicines firm has announced the initiation of dosing in a phase 2 clinical trial of its investigational therapy for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping.
Investigational therapy may be effective for amygdala-driven mental health conditions
MIAMI BEACH, Fla. — An emerging investigational therapeutic may be effective while being safe and generally well-tolerated for the treatment of mental health conditions driven by activity in the amygdala, according to research presented here.
FDA grants fast-track designation to potential CD19-CAR T cell therapy for lupus
The FDA has granted fast-track designation to CABA-201, a CD19-CAR T cell investigational therapy, for the treatment of systemic lupus erythematous and lupus nephritis, according to a press release from the manufacturer.
Biogen’s investigational therapy reduced tau protein levels in patients with Alzheimer’s
Biogen Inc. has reported promising data from a phase 1b trial of BIIB080, an investigational antisense oligonucleotide therapy that reduced soluble tau protein in cerebrospinal fluid in patients with early Alzheimer’s disease.
Q&A: Investigational therapy shows potential for treating patients with IPF
Saracatinib appeared to be “equal or superior” in stopping fibrotic responses compared with two FDA-approved antifibrotic drugs, according to a preclinical study published in American Journal of Respiratory and Critical Care Medicine.
Vera Therapeutics shares updates on investigational therapy atacicept
FDA grants breakthrough designation to IPF investigational therapy
The FDA has granted breakthrough designation to a novel investigational therapy, BI 1015550, for treatment of patients with idiopathic pulmonary fibrosis, according to a press release from Boehringer Ingelheim.
Interim data show preliminary safety, efficacy of AU-011 for choroidal melanoma
The majority of patients with choroidal melanoma treated with AU-011 have maintained vision and experienced tumor control and reduced growth rate, according to interim results of a phase 1b/2 open-label clinical trial.