Pricing Projections And Market Potential For Ionis' Newly Approved Nerve Damage Drug - Analyst Sees Over $700M Potential

Friday, the FDA approved AstraZeneca Plc AZN and Ionis Pharmaceuticals Inc's IONS Wainua (eplontersen) for polyneuropathy (nerve damage) of hereditary transthyretin-mediated amyloidosis in adults (hATTR-PN or ATTRv-PN). 

Wainua is the only approved medicine for ATTRv-PN that can be self-administered via an auto-injector.

William Blair sees potential for greater than $700 million in peak Wainua sales in ATTR-PN alone, which will be a significant value driver. 

Ionis is also positioned to benefit from Alnylam Pharmaceuticals Inc's ALNY HELIOS B cardiovascular outcomes trial readout in 1H of 2024 ahead of its CARDIO-TTRansform study readout expected in 2025. Ionis remains William Blair's top pick for 2024, and the analyst reiterates the Outperform rating.

While the official list price for Wainua has not yet been disclosed, the analyst anticipates that it will likely match Alnylam's Amvuttra (Vutrisiran) at approximately $463,500 per year. 

William Blair's current model suggests a considerably reduced net price of $291,000 for Wainua. This projection is conservative and factors in consideration for the more prevalent cardiomyopathy indication.

Looking ahead, the analyst perceives the ATTR-PN market as substantial enough to accommodate two competitors, especially with the inclusion of mixed phenotype PN/CM patients. 

Nevertheless, they acknowledge that the primary commercial opportunity and investor attention will likely revolve around the potential expansion of indications.

Price Action: IONS shares are up 5.30% at $51.72 on the last check Friday.