PIPELINE

Rozanolixizumab is an investigational humanized monoclonal antibody that specifically binds to human neonatal Fc receptor (FcRn). It has been designed to block the interaction of FcRn and IgG, inhibiting IgG recycling and inducing the removal of pathogenic IgG autoantibodies.

Rozanolixizumab is an investigational humanized monoclonal antibody that specifically binds to human neonatal Fc receptor (FcRn). It has been designed to block the interaction of FcRn and IgG, inhibiting IgG recycling and inducing the removal of pathogenic IgG autoantibodies.

Fenfluramine is an investigational serotonin releasing agent, that has shown to stimulate multiple 5-HT receptor sub-types through the release of serotonin. Fenfluramine may reduce seizures by acting as an agonist at specific serotonin receptors in the brain, including the 5-HT1D, 5-HT2A, and 5-HT2C receptors, and also by acting on the sigma-1 receptor

MT1621 is an investigational therapy that combines two small molecules, deoxycytidine (dC) and deoxythymidine (dT). It targets the underlying pathophysiology of Thymidine kinase 2 deficiency (TK2d) by restoring mitochondrial DNA (mtDNA) replication fidelity.

Dapirolizumab pegol is an investigational humanised monovalent pegylated Fab antibody fragment against the CD40 ligand (CD40L). Through interactions with its receptor, CD40, CD40L plays an important role in regulating interactions between T cells and other immune cells and thus affects several important functional events thought to be involved in autoimmune disease.

Dapirolizumab pegol is being co-developed with Biogen. 1st phase 3 study.

STACCATO® alprazolam is an investigational drug-device combination using STACCATO® delivery technology with alprazolam, a benzodiazepine, that has the potential to be the first rescue treatment to be administered by a patient or caregiver in an out-patient setting to rapidly terminate (within 90 seconds) an ongoing seizure.

Bepranemab is an investigational recombinant, humanised, full length IgG4 monoclonal anti-tau antibody with specificity for human tau protein.

Bepranemab is being co-developed with Roche/Genentech

minzasolmin is an investigational small molecule that prevents the pathological misfolding and accumulation of alpha-synuclein, a protein which plays a role in Parkinson’s disease (PD) pathology. Inhibition of alpha-synuclein misfolding has the potential to slow down the progression of PD.

minzasolmin belongs to a series of molecules discovered by Neuropore, which were in-licensed by UCB in 2014.

minzasolmin is being co-developed with Novartis.

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