A drug that Novartis AG discontinued in fragile X syndrome in 2014 after it failed in two phase II/III trials has been in-licensed by neuroscience specialist Stalicla SA, which plans to revive the prospects of the glutamate receptor antagonist by applying its precision medicine technology to identify likely responders.
Fundamental Pharma GmbH has raised €10 million (US$10.3 million) in a seed round to develop a new class of glutamate inhibitors, after uncovering a route to maintaining the protective effects of the neurotransmitter in the synapses while preventing neurotoxicity when it is released elsewhere.
The U.S. FDA wants more data on PTC-518 before PTC Therapeutics Inc.'s phase II study of Huntington’s disease can continue enrollment. While stopped in the U.S., the study of the oral, small-molecule splicing modifier still is enrolling participants at sites in several European countries and in Australia.
The mutant gene causing Huntington’s disease (HD) is active from the earliest stages of brain development, even though the pathology is not evident until between 30 and 50 years of age. That delay is ascribed to plasticity enabling the brain to compensate to such an extent that overt signs of disease take time to develop. As a result, it is difficult to plot a route from early molecular defects to development of HD several decades later.
As Novartis AG works to streamline the company and finalized a long-considered plan to separate its Sandoz Inc. business by creating a standalone company, it temporarily stopped dosing in a study of branaplam for treating Huntington’s disease. Several findings from the phase IIb study suggested the presence of peripheral neuropathy in some participants. An independent data monitoring committee recommended the dosing halt but fell short of recommending the study be terminated. The VIBRANT-HD steering committee agreed with the committee’s recommendation.
Uniqure NV rolled out stock-pleasing safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase I/II trial with AMT-130 for the treatment of Huntington’s disease (HD), but investors must wait for details on MRI scans. “We have communicated that we will discuss the MRI findings in the middle of next year,” CEO Matt Kapusta told investors during a conference call. “That’s all I can say.”
Mitorx Therapeutics Ltd. is poised to develop small molecules that reverse impaired sulphide signaling underlying degenerative diseases ranging from Duchenne muscular dystrophy to Alzheimer’s disease. The company was formed some time ago as a spinout from Exeter University, where the founding scientist Matt Whiteman is professor of experimental therapeutics. It is showing its colors for the first time after closing a seed funding round.
A Chinese study led by researchers at Fudan University in Shanghai has described a novel strategy to target the "undruggable" mutant Huntingtin protein by screening for compounds that directly bind to the 'undruggable' target and rescue disease-relevant phenotypes.
Annexon Inc. deemed promising – as did analysts – the interim phase II data with C1q protein complex inhibitor ANX-005 in Huntington’s disease (HD), but the safety profile took Wall Street aback, and shares (NASDAQ:ANNX) sank $3.75, or 34%, to close at $7.26.
Researchers at PTC Biotherapeutics Inc. have identified orally available small-molecule compounds that broadly lowered the levels of mutant huntingtin protein in both the brain and the periphery by affecting its splicing. One of those compounds, PTC-518, is currently in phase I trials as a therapy for Huntington's disease.
