Pasireotide long-acting repeatable (LAR) (SOM230, Signifor) is a novel multireceptor ligand somatostatin (sst) analogue with a high affinity binding profile for sst1, sst2, sst3 and sst5 receptor subtypes, mimicing the action of natural sst. Receptor subtypes sst2 and sst5 are expressed in 90% of GH-secreting pituitary tumours and once activated signal the pituitary gland to suppress GH secretion1. Pasireotide LAR also exhibits antiangiogenic activity (inhibiting vascular endothelial growth factor [VEGF] secretion) and works to reduce both the incidence and volume of pituitary tumours 2. Pasireotide LAR is intended to treat acromegaly first or second line and is administered as an intramuscular (IM) depot injection. In trials it has been administered at a dose of between 20 and 60mg every 28 days.
Leriglitazone is expected to work in patients with ALD by activating receptors called ‘PPAR gamma’, found in the mitochondria, which are components within cells that generate energy. Leriglitazone will improve the ALD pathogenic cascade by improving mitochondrial dysfunction, reducing oxidative stress and neuroinflammation, promoting demyelination and halting axonal degeneration. Hence, leriglitazone is expected to protect cells from damage and slow the progression of the disease. If licensed, leriglitazone would offer the first drug treatment option for adult ALD patients with AMN who currently have no effective therapies available.