Watchers of the Bruton’s kinase (BTK) inhibitor space may be casting renewed skepticism in that direction after Merck KGaA disclosed April 12 that the U.S. FDA placed a partial clinical hold on the sign-up of more patients in work testing evobrutinib in relapsing multiple sclerosis (MS) – but BTK efforts in MS continue in various quarters.
It’s the season for reevaluation as companies weed out programs that don’t offer much promise. At the head of the line is Sanofi SA’s once-potential myasthenia gravis blockbuster tolebrutinib. A partial clinical hold on the phase III study is part of the reasoning for stopping its development. But so is competition, the company said. Other companies eliminating development programs include Roche Holding AG, Gilead Sciences Inc., AB Science SA and Merck & Co. Inc.
The FDA has awarded orphan drug designation to Canbridge Pharmaceuticals Inc.'s CAN-106, a clinical-stage long-acting recombinant humanized monoclonal antibody targeting C5, for the treatment of myasthenia gravis.
NMD Pharma A/S has announced top-line results showing its orally available ion channel inhibitor, NMD-670, has a positive clinical impact in myasthenia gravis (MG), with patients having stronger hand grip and improvements in quantitative MG scores.
CSPC NBP Pharmaceuticals Co. Ltd. entered an agreement that will grant it exclusive rights to Harbour Biomed Co. Ltd.’s anti-FcRn antibody, batoclimab (HBM-9161), in greater China, including Hong Kong, Macau and Taiwan.
The U.S. FDA has put five phase III studies of Sanofi SA’s potential multiple sclerosis and myasthenia gravis blockbuster tolebrutinib on partial clinical hold after several cases of liver injury were identified after exposure to the drug. Sanofi said new recruitment in the U.S. is paused and participants who have been part of the trial for fewer than 60 days should stop taking tolebrutinib, although those taking the drug for longer can continue.
The first gene therapy to treat severe hemophilia A was among the drugs recommended for European approval by regulators from the EMA’s CHMP at its monthly meeting. Manufactured by Biomarin Pharmaceutical Inc., Roctavian (valoctocogene roxaparvovec) was recommended for conditional marketing authorization in the EU for severe hemophilia A in adults who do not have factor VIII inhibitors and no antibodies to adeno-associated virus serotype 5.
Brightinsight Inc. and UCB SA struck a deal to jointly develop a digital disease management solution for patients with rare diseases. The companies will start with a mobile app for myasthenia gravis (MG) built on Brightinsight’s platform. UCB joins CSL Behring, Novo Nordisk A/S, Sanofi SA and Roche AG in partnering with Brightinsight on digital disease management.
LONDON – Neuromuscular disease specialist NMD Pharma A/S has raised €35 million (US$39.7 million) in a new financing, as it awaits initial data from its first clinical trial, in the treatment of myasthenia gravis. The new money enables NMD to complete that phase IIa study and to launch another trial of the same compound, NMD-670, in spinal muscular atrophy. The Aarhus, Denmark-based company is preparing the IND and aims to treat the first patient before the end of 2022.
