Shares of Vertex Pharmaceuticals fell in trading after the company announced it will not advance a novel small molecule corrector of the Z-AAT protein into a Phase III study for a rare disease that can lead to complications of the liver and lungs.
Skyhawk Therapeutics and Vertex Pharmaceuticals inked a strategic research collaboration and licensing deal focused on developing novel small molecules that modulate RNA splicing.
Mereo BioPharma agreed a licensing deal and acquisition option for a rare disease drug from AstraZeneca as the big drugmaker continues to divest non-core assets.
Kamada Ltd. withdrew the Marketing Authorization Application (MAA) for its proprietary inhaled Alpha-1 Antitrypsin therapy for the treatment of Alpha-1 Antitrypsin Deficiency with the EMA.
Shares of Arrowhead Pharmaceuticals were down after the company’s experimental hepatitis B drug, ARC-520, was placed on clinical hold by the U.S. FDA.