Twilight of the stem cell transplant adjuncts?
The ripples of Molmed’s pivotal study failure could stretch beyond the company’s Zalmoxis product.
EHA 2019 – Sunesis chases Arqule, but remissions prove elusive
Sunesis’s vecabrutinib has yet to put a patient into remission, so hope rests on higher dosing of this next-generation BTK inhibitor.
EHA 2019 – Global Blood faces sickle cell questions
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.
EHA 2019 – turnaround puts Arqule in the takeover frame
A key update confirms ARQ 531 as the leading contender to treat patients who relapse on Imbruvica or Calquence.
Upcoming events – Crucial data approach on zanubrutinib and filgotinib
Beigene seeks very good data in Waldenström's macroglobulinaemia, while Galapagos aims to extend filgotinib's reach.
Emmaus tries to move forwards by reversing
The company insists that its sickle cell disease therapy Endari still has a future, despite questionable efficacy and the approach of new treatments.
Upcoming events – Protagonist and Pfizer target rare diseases
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
Takeda casts doubt on the remaining approach to amyloidosis
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.
Biomarin suffers minimal bleeding on valrox readout
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.
Kickstarting ideas
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