Pipeline Therapeutics Inc. has received FDA clearance to initiate a phase I trial of PIPE-791 in healthy volunteers, with dosing expected to begin in the second half of this year. PIPE-791 is advancing toward clinical development for remyelination and neuroinflammation, with a leading indication of multiple sclerosis (MS).
Researchers have ameliorated both monogenic and complex inflammation-driven diseases through transplantation of hematopoietic stem cells with an inserted IL-1 receptor antagonist (IL-1RA) gene. The team showed that in animal models the transplanted cells worked better than monoclonal antibodies to reduce symptoms in systemic autoinflammatory diseases (SAIDs), a group of childhood-onset, lifelong diseases that vary in severity depending on the underlying mutation, but can be life-threatening.
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.
Therini Bio Inc. has closed a US$36 million series A financing round that will support its work on developing fibrin-targeted therapies for diseases driven by chronic inflammation, including Alzheimer’s disease, multiple sclerosis and retinal diseases such as diabetic macular edema.
The loss of myelin in the cerebral cortex of multiple sclerosis (MS) patients could be recovered if oligodendrocytes, the cells that myelinate neuronal axons, work at a higher rate than they are destroyed. However, a group of scientists from the University of Munich have shown, in cortical MS mice, that this does not occur. The oligodendrocytes do not contribute to remyelination efficiently.
Pipeline Therapeutics Inc., which received U.S. FDA approval to move into a phase Ib/IIa study of PIPE-307 in relapsing-remitting multiple sclerosis (RRMS) last year, will be advancing the oral, small-molecule muscarinic M1 receptor antagonist in collaboration with Janssen Pharmaceutica NV in an agreement that could be worth more than $1 billion.
Aural Analytics Inc. received a breakthrough device designation from the U.S. FDA for its Speech Vitals-ALS technology, a software application that collects and analyzes speech recordings to help monitor amyotrophic lateral sclerosis (ALS) in adults in clinic and home settings. The software could improve management of the devastating disease.
Research at Bristol Myers Squibb Co. has led to the development of [11]-carbon-labeled compounds targeting Bruton tyrosine kinase (BTK) as positron emission tomography (PET) imaging agents for the diagnosis of multiple sclerosis.
