As of the end of August 2024, the BioWorld Neurological Diseases Index (BNDI) reported a decline of 8.89%, lagging behind the Nasdaq Biotechnology Index, which gained 11.7%, and the Dow Jones Industrial Average, which rose by 7.05%. While a slight recovery from BNDI’s lowest point this year, in late April when it was down 12.2%, the index had shown modest gains over recent months before slipping again.
One of Australia’s newest biotech investment funds is set to triple investments on the back of its success and strong investor demand. The three-year old Merchant Biotech Fund (MBF) invested in several high growth ASX-listed and private life sciences companies and finished the past financial year up more than 70%. It is up 10% for the current financial year, Portfolio Manager Reece O'Connell told BioWorld.
With two drugs cleared by the U.S. FDA for Niemann-Pick disease type C (NPC) in less than seven days, Wall Street was pondering the differences between the compounds, given what’s known so far about each. Most recently, the FDA approved Intrabio Inc.’s Aqneursa (levacetylleucine) on its PDUFA date for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing at least 15 kg, making Aqneursa the only approved stand-alone therapy indicated for NPC. On Sept. 20, Zevra Therapeutics Inc. won FDA clearance for Miplyffa (arimoclomol) as the first treatment for NPC.
Chong Kun Dang Pharmaceutical Corp. has disclosed 1,3,4-oxadiazole-containing compounds acting as histone deacetylase 6 (HDAC6) inhibitors reported to be useful for the treatment of prion diseases, asthma, psoriasis, inflammatory bowel disease, autosomal dominant polycystic kidney, stroke, Charcot-Marie-Tooth disease and uveitis, among others.
Merck Sharp & Dohme LLC has identified compounds targeting α-synuclein (ASYN; NACP) acting as positron emission tomography agents reported to be useful for the diagnosis of Parkinson’s disease.
Mabylon AG has been awarded three grants totaling more than CHF1.3 million (US$1.5 million) from Innosuisse Swiss Innovation Agency, Target ALS and the ALS Association.
The homeostasis of cytosolic calcium (Ca2+) is crucial for several physiological functions such as cell motility and neuronal transmission, among others. In this process, the inositol 1,4,5-trisphosphate receptor type 3 (ITPR3) plays a key role in the release of intracellular Ca2+.
The assessment of glycosylated autoantigens as immunotolerance therapies is emerging as a potential strategy for the treatment of several autoimmune diseases, such as type 1 diabetes, Crohn’s disease or multiple sclerosis.
Breakthrough – or even better, revolutionary breakthrough – is perhaps the most overused term in drug development. But the discovery and development of GLP-1 receptor agonists (GLP-1RAs), which was honored with the 2024 Lasker-DeBakey Clinical Medical Research Award just last week, is one of the rare innovations that is deserving of the title.
At the 2024 European College of Neuropsychopharmacology (ECNP) Congress, researchers have presented work that could lead to ways to boost brain development and prevent neurodegeneration in individuals with Down syndrome.
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