The ripples of Molmed’s pivotal study failure could stretch beyond the company’s Zalmoxis product.
Sunesis’s vecabrutinib has yet to put a patient into remission, so hope rests on higher dosing of this next-generation BTK inhibitor.
More data with voxelotor shouldn’t hurt the project’s bid for accelerated approval, but there are still doubts about its effect on a harder patient outcome.
A key update confirms ARQ 531 as the leading contender to treat patients who relapse on Imbruvica or Calquence.
Beigene seeks very good data in Waldenström's macroglobulinaemia, while Galapagos aims to extend filgotinib's reach.
The company insists that its sickle cell disease therapy Endari still has a future, despite questionable efficacy and the approach of new treatments.
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
After last year’s flop of Prothena’s NEOD001, Takeda’s Ninlaro fails to treat the underlying cause of the non-genetic form of this disease.
Three-year data from Biomarin’s haemophilia A gene therapy fail to quash concerns about durability, but the company insists that the results are good enough to file on.