The frontrunner in hemophilia A gene therapy has been hit by data doubts, but this and achondroplasia drug could deliver in 2020.
BioMarin Pharmaceutical Inc. is preparing to bring two first-in-class medicines to market in 2020 and has just provided extra reassurances that the therapies will make a real difference to patients, and help it reach a new growth phase.
Chief executive Jean-Jacques Bienaimé and his leadership team visited New York on 14 November to present their commercial vision and R&D strategy for 2020 and beyond
The therapy has been developed for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.
The presentation and publication of the Phase IIIb study showing greater efficacy for Zepbound over Wegovy come at a time when competition has been heating up between the two obesity medications.
InSilico will leapfrog Phase IIb to progress its AI-generated candidate for idiopathic pulmonary fibrosis directly into a Phase III trial in China, as it prepares for an IPO in Hong Kong.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
Seven years after acquiring it, Viatris will seek approval of a meloxicam formulation for acute pain backed by data from two Phase III studies in post-surgical pain settings.
The Phase I results are the best to date among ANGPTL3-targeting candidates in the pipeline, but analysts will wait for further updates before predicting a likely winner.
Houman Ashrafian tells delegates at Swiss Biotech Day that ‘literally only two things matter in our industry – target credentialing and differentiated pharmacology. The rest is execution.’
