Tag Archive for: biomarkers

Yesterday the agency published a revised draft guidance document, aimed at helping sponsors and pharmaceutical companies to develop therapies for Alzheimer’s disease, particularly in its early stages before the onset of overt dementia.

The 2023 Alzheimer’s Association International Conference will feature further analyses of the recent traditionally approved Leqembi and a highly anticipated update on Eli Lilly’s donanemab.

The proposed guidelines, unveiled by experts on Sunday in a report issued at an Alzheimer’s Association conference in Amsterdam, embrace a numerical staging system assessing disease progression similar to the one used in cancer diagnoses. They also eliminate the use of terms like mild, moderate and severe.

Shares of Sarepta Therapeutics soared 29% premarket on Monday as a backing by the U.S. health regulator’s advisers increased the certainty of an accelerated approval for the company’s gene therapy for a muscle-wasting genetic disorder.

“The FDA views gene therapy as an excellent opportunity to expedite the delivery of potentially life-saving therapies to patients with rare diseases,” said Marks, who heads the FDA’s Center for Biologics Evaluation and Research, in a statement.

Biomarkers as a surrogate endpoint in ALS will go on trial on March 22 as Biogen and Ionis’s tofersen faces the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee.

Med Ad News talked to Ittai Dayan, M.D., co-founder and CEO of Rhino Health, about federated learning in clinical trials.

Alzheimer’s research is having a moment, and the discovery of novel biomarkers is enabling progress far beyond diagnostics.  

Topline data from a Phase IIa study showed ChemomAb’s (Chemomab) investigational monoclonal antibody, CM-101, met its primary endpoints of safety and tolerability in nonalcoholic steatohepatitis (NASH), the Israel-based biotech reported Tuesday.

Athira Pharma’s experimental Alzheimer’s therapy, fosgonimeton, continues to show signs of being able to improve cognition and function in patients.