Ionis cholesterol project fails to meet Astra’s high hopes

The anti-PCSK9 MAbs might have failed to meet commercial dreams, but these agents are extremely potent cholesterol lowerers, an attribute that has kept biopharma interested in this mechanism. Longer-acting projects are the name of the development game now, but Astrazeneca bowed out on one of the most advanced of these next-gen agents today: the Ionis antisense project ION449. Results from the phase 2b Solano trial “did not achieve pre-specified efficacy criteria”, and the project will not be advanced into phase 3 for hypercholesterolemia, the pharma giant said in a statement. That sounds like the end of the road for Astra, which must have set an extremely high bar. The LDL reductions reported in Solano and the previous Etesian trial certainly look competitive. Perhaps Astra is taking a longer view here. ION449 was looking like a monthly dose, but gene editing players Verve and Precision are promising even longer term treatments. Human data on their respective agents are some way off  – Verve has achieved a 68% reduction in LDL in chimp studies at one year after one dose. Considering the likely cost of ION449’s pivotal programme, perhaps Astra has decided its R&D bets are best placed elsewhere.