Vertex lost $6bn in market cap in June after failing in its second attempt in alpha-1 antitrypsin deficiency. Meanwhile, Arrowhead has more than doubled in value, to $6.7bn, since its RNAi approach to the rare disease first showed promise.
There is always more than one driver behind a company’s valuation, of course, but these two examples help illustrate the substantial potential that investors see in a successful AATD treatment. There are only a handful of projects in clinical development, and Arrowhead remains in pole position, though approaching readouts from the likes of Mereo and Inhibrx could firm up the pipeline.
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