Alzheon Inc.’s oral treatment for people in the early stages of Alzheimer’s disease missed its phase III primary endpoint, adding yet another therapy to a long list by many developers that can’t beat dementia. The study also received grant money, which is in increasingly short supply.
As developers continue to search for better amyotrophic lateral sclerosis (ALS) therapies, Neurosense Therapeutics Ltd. turned up some hopeful findings from its phase IIb Paradigm trial with PrimeC. The drug, a combination therapy (ciprofloxacin and celecoxib) designed to target multiple ALS pathways, is having salutary effects on microRNA modulation (miRNA), Neurosense said, with the study showing a “profound and consistent” downregulation of 161 mature miRNAs across all time points in the double-blind period of the experiment.
The U.S. FDA’s accelerated approval of Vanrafia (atrasentan) from Novartis AG for primary immunoglobulin A nephropathy (IgAN) is the company’s second approval for the indication in the past year and a half. The nod also came without a required safety program through a black box warning.
Aldeyra Therapeutics Inc. is hoping that research already underway will satisfy the U.S. FDA, which delivered to the company another complete response letter (CRL) related to the NDA for reproxalap in dry eye disease (DED).
Top-line data from the second and third parts of Edgewise Therapeutics Inc.’s four-part phase II Cirrus-HCM study in hypertrophic cardiomyopathy (HCM) has produced more positive data. There was however less enthusiasm for the results than there had been in September for the first batch of data.
Pannexin 1 (PANX1) forms channels that may release signaling metabolites that are involved in a variety of pathophysiological processes, such as asthma, diabetes, hypertension or inflammatory bowel disease (IBD), among others. Inhibition of PANX1 when dysregulated has been proposed as a therapeutic approach in the treatment of IBD.
In type 2 diabetes patients, inadequate hyperglycemic control can lead to insulin-secreting β-cell exhaustion, dedifferentiation and eventual loss. Since adult β cells have limited proliferative capacity, this final stage cannot be reversed.
Cannabinoid CB1 receptors have been a potential target for nonopioid-based pain treatment, but actually targeting the pathway has been hindered by issues with tolerance and unwanted CNS side effects. Peripherally selective CB1 agonists developed to overcome these problems have not fully resolved these issues, meaning the peripheral selectivity has to be substantially enhanced.
It was a mixed day for Daewoong Pharmaceutical Co. Ltd., which disclosed separately on March 28 both the termination of a $336 million licensing deal with CS Pharmaceuticals Ltd. and upbeat plans to unveil three new oncology assets at the upcoming American Association for Cancer Research in April.
Despite missing the primary endpoint in a phase III study of solriamfetol in major depressive disorder, Axsome Therapeutics Inc. still sees a path to another phase III study. It’s a path some analysts and investors are concerned about, though its precision-medicine approach got plenty of nods of approval.
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