SAN DIEGO – At Neurosciences 2018, researchers highlighted mouse experiments and human biomarker studies further straightening the connection between dementia and the cardiovascular system.
SAN DIEGO – As gene therapy advances in the eye and hematopoietic stem cells, researchers are going after the harder-to-treat diseases of the central nervous system (CNS). At the Neurosciences 2018 conference, preclinical data showed the right vector and delivery technique can improve symptoms in mouse models of amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD) and Batten disease as the treatments advance to the clinic.
Medical cannabis companies developing pharmaceuticals have largely focused on using purified cannabinoids as monotherapies to treat neurological diseases. This year, for example, Cambridge, U.K.-based GW Pharma plc gained FDA approval for Epidiolex (cannabidiol) to treat two rare pediatric epilepsies, Dravet syndrome and Lennox-Gastaut syndrome, and others are on GW's heels, developing drugs for epilepsy and pain indications.
Medical cannabis companies developing pharmaceuticals have largely focused on using purified cannabinoids as monotherapies to treat neurological diseases. This year, for example, Cambridge, U.K.-based GW Pharma plc gained FDA approval for Epidiolex (cannabidiol) to treat two rare pediatric epilepsies, Dravet syndrome and Lennox-Gastaut syndrome, and others are on GW's heels, developing drugs for epilepsy and pain indications. (See BioWorld Insight, Oct. 17, 2016.)
Like most medical conferences, the 19th Annual World Conference on Lung Cancer (WCLC) in Toronto was the typical mix of high-profile phase III studies with a large number of earlier-stage programs looking to disrupt the current treatment paradigm.
If there was an award for the most accurate nomenclature in clinical development, window of opportunity (WOO) studies would certainly be in the running since they provide just that: an opportunity to test a drug during the window between diagnosis and excising of the primary tumor.
It was a mixed bag for Sangamo Therapeutics Inc. at the 2018 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Athens, Greece, on Wednesday where the company presented data from its phase I/II Champions trial testing SB-913 in patients with mucopolysaccharidosis (MPS) type II.
With multiple recent approvals of biologics to treat psoriasis Cosentyx (secukinumab, Novartis AG), Ilumya (tildrakizumab-asmn, Sun Pharmaceutical Industries Ltd.), Taltz (ixekizumab, Eli Lilly and Co.) and others companies are turning to topical treatments to capture the estimated 80 percent to 90 percent of psoriasis patients with mild to moderate disease where cheaper topical medications are more appropriate.
Takhzyro's (lanadelumab-flyo) breakthrough designation was only good for a one-business-day advantage in gaining FDA approval ahead of its Prescription Drug User Fee Act action date on Sunday.
