A nearly two-year-old partial clinical hold has been lifted by the U.S. FDA on PTC Therapeutics Inc.’s pivotal phase II study in Huntington’s disease. The agency had paused enrollment in October 2022, saying it wanted more data on PTC-518, an orally bioavailable small-molecule splicing modifier, before enrollment could continue.

The recent licensing agreement that Alzpath Inc. signed with Roche AG for use of its pTau217 antibody to develop and commercialize an Alzheimer's disease diagnostic blood test will not only transform the company but also allow patients access to a more cost-effective test, Venkat Shastri, CEO of Alzpath told BioWorld.

Shanghai Henlius Biotech Inc. has announced preclinical data showing that its small-molecule drug HLX-99 exhibited antineurodegenerative activities in multiple models, warranting further development.

The CMT Research Foundation has formed a strategic partnership with Revir Therapeutics Inc. to advance small-molecule therapeutics that modulate the expression of the gene that causes Charcot-Marie-Tooth disease type 1A (CMT1A).

Wall Street is mulling Intra-Cellular Therapeutics Inc.’s chances of replicating with Caplyta (lumateperone) the path followed by Abbvie Inc. with Vraylar (cariprazine) in major depressive disorder (MDD), and possibly overtaking the latter in the difficult indication.

As new treatment options are being continually investigated and trialed against Parkinson’s disease, the possibilities offered by deep brain stimulation (DBS) risk being overlooked. Though not a cure, the therapy could vastly improve the quality of life for patients with the disease.

The adverse effects of PD-1 blockers on the CNS observed in cancer patients could occur through their effects on an enzyme that activates microglia. Pharmacological inhibition of the enzyme in mice reduced microglial activation and cognitive deficit without altering the antitumor capacity of the immunotherapy.

Oblique Therapeutics AB has entered into an agreement with the KTH Royal Institute of Technology to utilize KTH’s supercomputer resources for analyzing protein structures.

Italfarmaco SpA and Iktos SA have entered into a collaboration to develop next-generation histone deacetylase (HDAC) inhibitors for a variety of non-oncological diseases, including diseases affecting the central nervous system.

Disappointing top-line data from two failed phase III studies of soticlestat for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) have crumpled the stock of co-developer Ovid Therapeutics Inc., which lost three-quarters of its value June 17.