Apnimed Inc.’s lead oral therapy for obstructive sleep apnea has delivered a second round of positive phase III data, bringing a potential U.S. FDA filing closer. Top-line results taken from the second pivotal late-stage study show AD-109 (aroxybutynin 2.5mg/atomoxetine 75 mg) hit its primary endpoint with clinically meaningful and statistically significant drops in airway obstruction at 26 weeks.
Brain-derived neurotrophic factor (BDNF) is a neurotrophin that supports neuronal growth and survival and plays a critical role in regulating synaptic plasticity and cognitive function. Reduced BDNF levels have been observed in Alzheimer’s disease, Parkinson’s disease and mood disorders, suggesting that agents capable of enhancing BDNF expression may represent promising therapeutic strategies for both neurodegenerative diseases and depression.
The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene in order to restore near full-length dystrophin protein within the body to improve functional outcomes.
Alterations in the GABAergic pathway in the brain contribute to the pathophysiology of neurodevelopmental disorders, including autism spectrum disorder and Angelman syndrome. Targeting the GABA-A receptor α5 subunit (GABRA5) with positive allosteric modulators represents a new therapeutic strategy for the treatment of these conditions.
In a 10-1 vote, the U.S. FDA’s Psychopharmacologic Drugs Advisory Committee said Otsuka Pharmaceutical Co. Ltd.’s studies of its combination post-traumatic stress disorder (PTSD) treatment did not show enough efficacy. Had the adcom felt otherwise, the path may have been smoother for a new treatment in an indication that hasn’t seen regulatory advancement in decades. The committee’s conclusion, however, is not binding on the FDA, which will make its own decision whether to approve.
CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co. Ltd. has reported new monoamine oxidase-B (MAO-B) inhibitors potentially useful for the treatment of depression, stroke, hypertension, obesity, Alzheimer’s and Parkinson’s disease.
Researchers at the University of Minnesota and collaborating institutions have developed a promising stem cell-based therapy for the treatment of muscular dystrophies. The team has successfully created a novel myogenic progenitor cell product called Myopaxon, derived from human-induced pluripotent stem cells (iPSCs).
After two decades of research elucidating the basic science, Tafalgie Therapeutics SA has delivered the first clinical data for its non-opioid analgesic.
Work at Genecode AS has led to the identification of sulfonamide or sulfone compounds acting as GFRα1-RET receptor complex activators potentially useful for the treatment of neurological disorders.
Parkinson’s disease (PD), the second most prevalent neurodegenerative disorder worldwide, is characterized by the progressive degeneration of dopaminergic neurons in the substantia nigra, leading to a decline in both motor and cognitive functions. Incretin-based therapies, initially designed for the treatment of type 2 diabetes, have demonstrated relevant neuroprotective effects in preclinical models of PD.
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