Proteros Biostructures GmbH has established a new collaboration with Orion Corp. The two companies are engaging in a joint multitarget collaboration for early-stage drug discovery projects in oncology and pain using Proteros’ discovery platform, and now have decided to combine their expertise to assemble a chemically diverse small-molecule library collection. The nonexclusive agreement enables Proteros to exclusively offer access to this high throughput screening (HTS) library to all its clients.

In multiple sclerosis (MS), macrophages and microglia play a dual role that could be used to treat this neurodegenerative disease. These cells promote inflammation that demyelinates neurons but also sweep away the debris of damaged myelin and produce neurotrophic factors that would allow its restoration. According to a group of scientists from the University of Hasselt in Belgium, damage or repair depends on a double switch that combines the action of two enzymes, one that desaturates and another that elongates fatty acids. By reducing the levels of these enzymes, phagocytic cells would replenish the myelin instead of engulfing it.

Pepgen Inc. has received a no objection letter from Health Canada for its clinical trial application (CTA) to initiate a phase I study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). Initial data from the study are expected next year.

The FDA has awarded orphan drug designation to Healx Ltd.’s HLX-1502 for the treatment of neurofibromatosis type 1 (NF1). HLX-1502, discovered through the company’s proprietary artificial intelligence (AI) drug discovery platform, has a first-in-class mechanism and supporting data that indicate the potential for a good safety profile.

Bloomsbury Genetic Therapies Limited has disclosed a new pipeline program – BGT-PD...

While the U.S. FDA didn’t ask for more study data or have safety or efficacy concerns, it does want modifications to Alexion, Astrazeneca Rare Disease’s sBLA for Ultomiris (ravulizumab-cwvz) to treat adults with the rare central nervous system disease neuromyelitis optica spectrum disorder. The agency has issued a complete response letter (CRL) requesting changes to Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) to better validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics before being treated.

People who can’t verbalize their pain often end up being under medicated, and Painchek Ltd. uses artificial intelligence (AI) to identify the presence of pain even when it may not be obvious. This gives a voice to those who cannot verbalize pain, while also driving objectivity and consistency in pain assessments.

At the same time it reported U.S. FDA clearance for bedside software to diagnose electrographic status epilepticus (ESE) seizures, Ceribell Inc. declared Claritypro the first ICU monitoring device to receive an NTAP reimbursement from the Centers for Medicare and Medicaid Services. The clearance of Claritypro to diagnose ESE follows receipt of an FDA breakthrough device designation in two different categories in the past year, setting the stage, Ceribell CEO Jane Chao told BioWorld, for expanded access to critical seizure diagnosis technology.

Tetra Pharm Technologies Aps has completed preclinical testing and announced promising in vivo study results with its candidate compound, TPT-0301, which targets the endocannabinoid system.

A large-scale genetic study found 26 risk loci for epilepsy, a chronic brain disease with multiple forms, not all of them heritable. The work, by more than 300 authors from the International League Against Epilepsy (ILAE), investigated seven different subtypes of this neurological condition. “There are over 100 genes that we know can harbor mutations that cause epilepsy,” the co-corresponding author Gianpiero Cavalleri told BioWorld. These genes have rare forms that cause that epilepsy. However, “this particular GWAS is focused more on common forms of epilepsy,” he said.