Aural Analytics Inc. received a breakthrough device designation from the U.S. FDA for its Speech Vitals-ALS technology, a software application that collects and analyzes speech recordings to help monitor amyotrophic lateral sclerosis (ALS) in adults in clinic and home settings. The software could improve management of the devastating disease.
Newco Teitur Trophics ApS has raised €28 million (US$30.1 million) in a series A with which it will lay out a new route to targeting sortilin in the treatment of neurodegenerative diseases. The company is targeting the sortilin-related Vps10p domain containing receptor, which plays a role in regulating a number of pathways involved in the control of neuronal viability and function.
Teitur Trophics ApS, a spinout from Aarhus University seeded by the Bioinnovation Institute in 2020, has completed a €28 million (US$30.1 million) series A financing. Teitur has developed a platform of first-in-class cyclic peptides with a novel mechanism that preserves neuronal function, and these peptides have the potential to treat a broad range of neurodegenerative diseases.
PTC Therapeutics Inc. has synthesized heteroaryl compounds acting as huntingtin (HTT) (mutant) expression inhibitors reported to be useful for the treatment of Huntington's disease.
Upcoming catalysts from Annexon Biosciences Inc. put some joy into shares as the firm talked up its prospects during the recent J.P. Morgan Healthcare Conference, where attendees heard Jan. 8 about the news ahead with C1q protein complex inhibitor ANX-005 in Huntington’s disease (HD) as well as progress in Guillain-Barre syndrome (GBS), and more. The stock (NASDAQ:ANNX) enjoyed a 43% stock boost in the days after JPM, rising from $4.79 on Jan 8 to $6.84 on Jan. 13.
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.