The U.S. FDA has lifted the clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating adeno-associated virus (AAV) gene replacement therapy AT-845 in adults with late-onset Pompe disease.

A year and a half gone, two HIV vaccine studies shut down. That’s the case with the Janssen Pharmaceutical Cos. of Johnson & Johnson as the phase III Mosaico study of its HIV vaccine regimen was not effective in preventing infection compared to placebo. Based on a data and safety monitoring board’s report saying the study was not expected to hit its primary endpoint, Janssen discontinued the clinical trial more than a year ahead of its estimated March 2024 completion date.

Irlab Therapeutics AB’s phase IIb exploration of the dopamine D3-receptor with the Ipsen SA-partnered oral antagonist mesdopetam in Parkinson’s disease (PD) levodopa-induced dyskinesia (LID) fell short in terms of the primary endpoint, but the company is holding out hope. Mesdopetam missed the primary endpoint of change in daily hours of “on” time – periods of adequate PD control – without troublesome dyskinesia as shown in patient diaries, the company said.

In what is claimed as the largest study of how digital technologies were applied to support population level research during the pandemic, scientists at the U.K. Medical Research Council’s epidemiology unit at Cambridge University have reported high, sustained levels of engagement in a fully remote COVID-19 study that ran at a time when visits to a study center were not possible.

The accelerated approval by the U.S. FDA of Immunogen Inc.’s landmark antibody-drug conjugate Elahere (mirvetuximab soravtansine-gynx) injected further oomph into the NaPi2b space, where a handful of developers are due to yield important data shortly. In play are such names as Mersana Therapeutics Inc. and Zymeworks Inc., encouraged by historical – if not entirely successful – efforts by giant Roche Holding AG.

A study in a mouse model of tau protein build-up in the brain, similar to that seen in later stage Alzheimer’s disease (AD), shows that changes to the microbiome in these animals can impact the degree and progression of neurodegeneration observed. As reported in the Jan. 12, 2023, study published in Science, the researchers found that mice that were germ free and those given antibiotics to change their gut microbiome early in life had significant reductions in brain atrophy compared with those with a standard microbiome.

Although strides have been made in the space, biopharma’s hunt for a wider net to cast in vaccines for pneumococcal disease has recruited the likes of Vaxcyte Inc., GSK plc and Merck & Co. Inc., each racing for a candidate with improved valence.

Oramed Pharmaceuticals Inc. has had a lot of success with testing its oral insulin, until now. The company will discontinue it oral insulin clinical work in treating type 2 diabetes after ORMD-0801 failed to hit its phase III study’s primary and secondary endpoints. The late-stage stumble clobbered the company stock (NASDAQ:ORMP), which lost 76% of its value on Dec. 12 to close at $2.54 each.