Tralesinidase alfa is in clinical development for the treatment of patients with mucopolysaccharidosis type IIIB (MPS IIIB) also known as Sanfilippo syndrome type B. MPS IIIB is a rare and progressive genetic disorder caused by the deficiency of one of the enzymes needed to break down complex sugar molecules called mucopolysaccharides. Clinically, patients have behavioural problems, intellectual deterioration, sleep disorders, hearing impairment, facial dysmorphism, organomegaly, bowel disturbances, mild skeletal changes, and shortened life span. The clinical severity ranges from mild to severe.
Tralesinidase alfa is an investigational enzyme replacement therapy (ERT) designed to replace the faulty enzyme with a healthy one in patients with MPS IIIB. Tralesinidase alfa is delivered directly to the fluid surrounding the brain (cerebrospinal fluid). If licensed, tralesinidase alfa will offer new therapy option for patients with MPS IIIB who currently have no treatment options.
Finerenone is selective inhibitor of a specific protein, which blocks the damaging effects of hormones (such as aldosterone and cortisol) which can cause damage to the heart and kidneys in diabetic patients. Finerenone is administered orally in tablet form, and evidence suggests it may offer organ protection compared to similar inhibitors which are currently used. If licensed, finerenone will offer patients with type 2 diabetes mellitus who have chronic kidney disease an add-on therapy to slow down long-term kidney damage and reduce the adverse impact on the structure and function of the heart and vessels.