Accumulations of these tau tangles ... disease’s defining features. In recent years, two drugs that clear amyloid plaques have been approved by the Food and Drug Administration—the first new Alzheimer’s therapies in about two decades. Meanwhile, many experimental treatments targeting tau have failed, despite research suggesting that tau tangles are a stronger predictor than amyloid of brain deterioration. Now, findings from a mid-stage clinical trial presented ... Accumulations of these tau tangles and sticky clumps of the protein amyloid beta are the neurodegenerative disease’s defining features. In recent years, two drugs that clear amyloid plaques have been approved by the Food and Drug Administration—the first new Alzheimer’s therapies in about two decades. Meanwhile, many experimental treatments targeting tau have failed, despite research suggesting that tau tangles are a stronger predictor than amyloid of brain deterioration. Now, findings from a mid-stage clinical trial presented on July 14 at the Alzheimer’s Association International Conference in London could change that. Recently FDA-approved treatments work to clear clumps of the protein amyloid beta in the brain. A drug that instead goes after tangles of the protein tau can yield similar cognitive outcomes, a new study suggests The work offers hope for the treatment of a disease that affects more than seven million Americans ages 65 and older, though a larger follow-up study will need to confirm the therapy’s benefits. “This is really quite promising if it were to hold up” in further testing, says Jessica Langbaum, senior director of Alzheimer’s prevention and research at the Banner Alzheimer’s Institute, who wasn’t involved in the trial, to Lauran Neergaard at the Associated Press. “This was a well-tolerated trial where people wanted to continue on treatment,” neurologist Cath Mummery of University College London said as she presented the study at the conference, reports Andrew Joseph at STAT. The company now plans to test diranersen in a late-phase clinical trial to see how it fares with a larger group of participants.

Original title: “Experimental Alzheimer's Drug That Fights a Tough-to-Target Protein Shows Promise at Slowing Cognitive Decline in a Clinical Trial”

Jideytro offers option for previously treated patients

Original title: “GSK wins US approval for ROS1 lung cancer therapy”

Designation supports development of novel orexin therapy

Original title: “Lundbeck secures FDA Fast Track for narcolepsy drug”

The U.S. Food and Drug Administration today announced the first manufacturer selected for the Technology-Enabled Meaningful Patient Outcomes (TEMPO) for Digital Health Devices Pilot.

Original title: “FDA Announces First Participant Selected for TEMPO for Digital Health Devices Pilot”

To analysts, the drug’s apparent safety and effects on both triglyceride levels and pancreatitis cases should make it competitive with a rival Ionis therapy.

Original title: “Arrowhead hits ‘best-case scenario’ with latest results for fat-lowering drug”

First patient randomised as study advances novel pan phosphate transporter inhibitor

Original title: “R1 Therapeutics begins global phase 2b trial of AP306 for hyperphosphatemia”

With a Phase 3 failure denting the rare disease drug’s prospects, AstraZeneca has little room for error as it plots a path toward its ambitious 2030 sales target.

Original title: “Wainua flop narrows AstraZeneca’s path to $80B goal ahead of high-risk readouts”

Agios Pharmaceuticals’ sickle cell disease candidate didn’t prove any better than Novo Nordisk’s investigational PK activator or the biopharma’s own asset that is currently under FDA review for the indication.

Original title: “Agios axes next-gen sickle cell candidate after it fails to best Novo’s asset”