After recent landmark approvals in other neurodegenerative diseases such as Alzheimer’s and amyotrophic lateral sclerosis, hopes are rising that Huntington’s disease could be next to reach a breakthrough.
UniQure is awaiting updated data from its potential first-in-class gene therapy, AMT-130, with the most important being a Phase I/II trial in the US monitoring a small group of patients at 12 and 24 months after treatment.
The gene therapy uses an AAV5 vector to transports a micro-RNA into the central nervous system with the goal of...
The company will move ahead in testing RDX-002 among overweight and obese patients coming off GLP-1s, but the CEO said antipsychotic-induced weight gain could represent a multibillion-dollar market.
Leading a field of rival potassium channel modulators, azetukalner will have a pivotal epilepsy readout early next year, with Phase III trials in depression and biopolar disorder also underway.
Sumitomo Pharma's iPSC therapy for Parkinson's has been filed for approval in Japan after showing safety and efficacy in a Phase I/II trial and may become one of the first such therapies to reach the market in the country, which has invested heavily in the modality.
A Phase II trial to develop a second NaV1.8 pain signal inhibitor for acute pain has failed and the US FDA has given a thumbs down on Journavx’s path to broader use in peripheral neuropathic pain.
The drug, combined with Merck’s Keytruda, was successful among certain chemotherapy-ineligible MIBC patients, with another Phase III readout expected by March 2026.
The US FDA approved Hernexeos for previously treated HER2-mutant NSCLC, making it the first oral option in a setting where drugs like AstraZeneca/Daiichi Sankyo’s Enhertu dominate.
Novartis has strengthened its argument that ianalumab, its BAFF-R inhibitor and ADCC-mediated B-cell depletor candidate, has PIP potential with successful topline results in two Phase III Sjögren’s syndrome trials, after recently dropping the product in hidradenitis suppurativa.
