915 days left
REGN analysis is going smoothly. Should have the final outcome for you next week.
Can you really have a drug pricing hearing without me? That’s fine. I’ll be out in 2 years and we can do it then. Sanctimonious ignorance won’t be eliminated in politics anytime soon.
In a bombshell paper in The Lancet, researchers found that Novartis Access, which donates key drugs to poor countries such as Kenya for $1 per month, did NOT help access to medicine. Systems are complex, they say. They are in the U.S., too.
Papers I’ve Read
MeCP2 isoform e1 mutant mice recapitulate motor and metabolic phenotypes of Rett syndrome. Ciernia et al. Hum Mol Genet 2019.
Rett Syndrome is very promising to drug developers. It’s a somewhat large monogenic illness. Knowing exactly what is ‘going wrong’ in a disease is the first step to designing a treatment. Unfortunately all that is really known about Rett is there is a MeCP2 dosage issue. Nobody knows what MeCP2 does, and the fact that most patients are female creates an interesting gene dosage/X-inactivation question (MeCP2 is on the X chromosome). At four exons, two isoforms of MeCP2 are spliced. MeCP2-exon2 proteins don’t seem to cause disease while MeCP2-exon1 proteins do. This group created the former and spent a lot of time on metabolic profiling, which is interesting but they don’t quite recapitulate the phenotype of the human illness in their mice. Progress will probably be slow in this illness but keep in mind AveXis (now a part of Novartis) has started a phase 1 for their gene therapy.
Clinical Trials I’ve Noticed
Study With Lu AF20513 in Patients with Mild Alzheimer’s Disease (AD) or Mild Cognitive Impairment (MCI) Due to AD. Lundbeck A/S.
Lundbeck has had this weird Abeta vaccine in Phase 1 for 5 years or so. I don’t think it will work, but one never knows. It’s certainly an interesting approach–not one that hasn’t been tried though. I believe Elan had some other active immunization program that blew up (this might be why the trial has taken so long).