Vanishing white matter disease (VWM) is a rare and progressive leukoencephalopathy caused by loss-of-function mutations, in a recessive pattern of inheritance, in any of the genes encoding eIF2B, a guanine nucleotide exchange factor for eIF2 and an effector of the integrated stress response (ISR). At last week’s American Academy of Neurology meeting, Calico Life Sciences LLC and Abbvie Inc. presented preclinical results for their brain-penetrant compound ABBV-CLS-7262 (fosigotifator sodium tromethamine) in VWM.
Researchers from the U.K. reported seeking protection for a device and method that combines electromyography (EMG) and Raman spectroscopy to improve the diagnostic pathway for patients with neuromuscular disorders, and that may be used as a minimally invasive bedside test of muscle health.
South Korea’s SK Biopharmaceuticals Co. Ltd. and Shanghai-based Ignis Therapeutic Co. Ltd. signed a ₩804 billion (US$58 million) licensing deal on April 18, granting the latter global rights to a non-narcotic pain treatment candidate dubbed SKL-22544. A sodium channel blocker, SKL-22544 is in late discovery.
Cerevel Therapeutics Inc.’s positive results from the long-shot pivotal phase III Tempo-3 trial with tavapadon – the first D1/D5 receptor partial agonist being studied as a once-daily treatment for Parkinson’s disease – added gravy to the $8.7 billion takeover by Abbvie Inc., disclosed late last year. The buyout’s centerpiece was the late-stage asset emraclidine, a positive allosteric modulator of the muscarinic M4 receptor, touted as a potential best-in-class, next-generation antipsychotic for schizophrenia, which strikes more than 5 million people in the G7 (U.S., France, Germany, Italy, Spain, U.K. and Japan).
Wista Laboratories Ltd. has disclosed thiazole-containing compounds acting as microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of corticobasal syndrome, Alzheimer’s disease, Down syndrome and more.
In what represents its first patenting, Alva Health Inc. has been granted protection for a method for detecting stroke from falls using a combination of wearable motion sensors and artificial intelligence.
“The development of psychedelic medicines should adhere to the current European regulatory framework, at both the pan-European and member state level. It is imperative that developers understand and thoroughly apply the regulatory guidance and requirements that are in place.” That was the somewhat discouraging opening statement at the stakeholder workshop convened by the EMA to discuss the development and therapeutic use of psychedelic substances to address unmet medical needs in the area of mental health.
Eli Lilly and Co. is planning to file for U.S. FDA approval later this year after reporting that tirzepatide met all primary and key secondary endpoints in two phase III trials in obstructive sleep apnea (OSA). Assuming approval, tirzepatide could become the first drug approved specifically for OSA, while providing potential entry access for Medicare Part D coverage, which is denied for the GLP-1 class of drugs approved as obesity medications.
After Sage Therapeutics Inc. reported a phase II failure with oral dalzanemdor, also known as SAGE-718, in mild cognitive impairment related to Parkinson’s disease (PD), Wall Street’s eyes turned to ongoing mid-stage efforts with the same N-methyl-D-aspartate receptor-positive allosteric modulator in Huntington’s disease and Alzheimer’s disease.
Neurotech International Ltd.’s cannabinoid NTI-164 met the primary and secondary endpoints in a phase II/III trial in children with autism spectrum disorder. The company also reported positive top-line data for Rett syndrome in a phase I/II trial.
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