Clinical UncertaintyThe potential for a larger sample size in Phase II to lead to an inferior effect compared to Phase I results suggests uncertainty in replicating earlier success.
Market OpportunityThe HAE market and available treatments limit Lonvo-z opportunity size, suggesting limited uptake.
Valuation ConcernsThe valuation disconnect between the HAE opportunity and NTLA’s market cap is one of the widest, which may persist until NTLA releases pivotal HAE data or addresses investor concerns on the balance sheet.