Neurological

The Texas-based firm is giving up on the investigational Alzheimer’s therapy after a second Phase III failure left it with nowhere to go in the disease.

The FOCUS study met its primary and key secondary endpoints, but lack of statistical significance on the higher dose and a crowded market raised questions.

A decision from the FDA is due by 28 September.

A new “quasi-experimental” study using UK National Health Service data might confirm that GSK’s shingles vaccine helps cut dementia cases.

The Belgian firm banks nearly €77.7m to push its Charcot-Marie-Tooth to proof-of-concept.

Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.

Move to acquire profitable Tokyo-based venture part of Japanese major's strategy of building a broader ecosystem for dementia detection and care.

The potential first-in-class antibody oligonucleotide conjugate could treat a form of the muscle wasting disease, and could be the first of Avidity’s trio of rare disease drugs to gain approval.

A joint venture with AI specialist Hologen will bring in much-needed cash for the ambitious gene therapy firm.

The future of kappa opioid receptor antagonism as a therapeutic strategy in major depressive disorder is looking increasingly bleak after J&J dropped aticaprant from development, but rival Neumora is pressing on with navacaprant.

Alzheimer’s Drug Discovery Foundation CFO Karen Harris talked to Scrip about the non-profit group’s investment strategy and biopharma interest in Alzheimer’s disease.

While Lexicon’s non-opioid pain candidate missed the primary endpoint in a diabetic peripheral neuropathic pain study, the company insisted the data revealed a path forward with a 10mg daily dose.

Leqembi is finally on track for approval by the summer, though safety precautions mean fewer patients will be eligible for treatment than in the US, and its uptake is not expected to be strong.

The Essential3 program’s data monitoring committee recommended stopping one of the essential tremor studies for futility, but the company said it will continue the trials anyway.

Results from the Phase III EMERGE study showed that Axsome’s recently approved Symbravo is effective in patients dissatisfied with oral CGRP inhibitors.

Over 140 biopharma leaders share their views on developments to watch for in key therapeutic areas this year. Advances in multiple scientific fields are opening up new avenues for treatment.

The agency sent the neuroscience-focused company a refuse-to-file letter for the drug as a treatment for IH, a disease with only one drug approved and few in development.

Biogen will pay $165m up front for ex-North American rights to Stoke’s Phase III-ready Dravet syndrome candidate, an antisense drug offering disease-modifying potential.

Relmada has acquired sepranolone, a potential blockbuster for the complex neurological condition characterized by involuntary tics, for a bargain €3m from the Stockholm group that went into liquidation late last year.

Scrip surveys some of the more interesting Phase III clinical trial readouts expected this year.