Shares of Uniqure NV (NASDAQ:QURE) were trading at $43.52, up $29.86, or 218%, after the Amsterdam-based firm disclosed positive top-line data from the pivotal phase I/II study of AMT-130 for the treatment of Huntington’s disease. The study met its prespecified primary endpoint, with high-dose AMT-130 turning up a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control. The study also met a key secondary endpoint by reaching statistical significance in the slowing of disease progression as measured by Total Functional Capacity at the same time point vs. the control.

Acadia, with a failed phase III, drops Prader-Willi candidate

After a phase III stumble, Acadia Pharmaceuticals Inc. will drop development of ACP-101, intranasal carbetocin, to treat hyperphagia in patients with the rare genetic disorder Prader-Willi syndrome (PWS). Top-line data from the 12-week, double-blind, randomized phase III study missed its primary endpoint by not producing a statistically significant improvement over placebo. The company’s stock (NASDAQ:ACAD) was down 9% at midday, with shares going for $21.44 each. It was the opposite for Soleno Therapeutics Inc., which has Vykat XR, the first approved drug for treating hyperphagia in PWS. Shares (NASDAQ:SLNO) were up 12.4% at midday at $63.92 each.

REMS under scrutiny as Agios, Cytokinetics approvals pending

Regulatory holdups due to risk evaluation and mitigation strategies (REMS) have drawn the attention of Wall Street lately. Agios Pharmaceuticals Inc. submitted a REMS related to the sNDA for Pyrukynd (mitapivat), and Cytokinetics Inc. composed one for aficamten in obstructive hypertrophic cardiomyopathy. Among the questions is whether and to what degree the REMS might foretell a complete response letter.

Human Cell Atlas project ties fibroblast subtypes to multiple diseases

The Human Cell Atlas project has delivered a fresh tranche of data, mapping fibroblasts in healthy and diseased skin and pointing to drug targets with potential in multiple diseases, across a range of tissues. “The clinical relevance of our work is huge. It is paradigm-shifting in terms of how we move from understanding the role of individual cell types to uncovering how disease specific cell types form ‘tissue neighborhoods’ that mediate inflammation,” said Muzz Haniffa, head of cellular genomics at the Sanger Center, Cambridge University, U.K., and lead author of a paper describing the research in Nature Immunology, Sept. 24, 2025. “We find the same disease-related fibroblasts involved in several diseases across the body [in] atopic eczema, inflammatory bowel disease, rheumatoid arthritis, meaning we could find universal therapeutic targets in these cells to treat many diseases,” Haniffa said.

Deep dive nets sex differences in HIV reservoir

Globally, over half of people living with HIV are women. But in clinical cure trials, they make up only about 20% of participants. And that gender imbalance is causing researchers to miss out on ways to improve cure strategies. Because women’s immune systems appear to be better at controlling HIV infection in a way that silences the reservoir – the provirus integrated into host cells in infected persons. “I think we’re missing a whole promising participant group with a very powerful immune system that actually can make a difference [in] our cure studies,” Xu Yu told BioWorld. In the Sept. 17, 2025, issue of Science Translational Medicine, Yu and her colleagues reported that in women, intact proviruses were more likely to be in positions where the virus is blocked from replicating. Yu is a professor of medicine at Harvard Medical School, an investigator at the Ragon Institute of Mass General Brigham, MIT and Harvard, and the senior author of the paper.

Also in the news

Angelini, Aphaia, Arrivent, Atai, Beckley Psytech, BMS, Boehringer Ingelheim, Eisai, Eli Lilly, Eupraxia, General Oncology, H. Lundbeck, Kelun-Biotech, Kinea, Lexicon, Merck, Mlab, Moderna, Neuraclick, Neurocrine, Otsuka, Sanofi, Santhera, Scholar Rock, Seed, Sensory Cloud, Sernova, Solid, Soligenix, Sovargen, Tegmine, Variational AI, Vor