Vertex Pharmaceuticals Inc (VRTX)

[Monksdream]

From Cathie Wood
Gene-Editing Therapies Could Rearrange Healthcare Expenditures In The US
ARK Invest_Illustration_Ali Urman_Final_Circle 400 px By Ali Urman | @aurmanARK
Analyst
Last week, Vertex Pharmaceuticals presented3 promising results from a Phase I/II trial focused on Type 1 Diabetes (T1D): VX-880, an allogeneic stem-cell derived islet cell therapy infused into the hepatic portal vein for T1D patients with severe hypoglycemia. Importantly, the six patients produced endogenous insulin, reducing the need for exogenous insulin and showing blood glucose control with a reduction of HbA1c.

In the US today, healthcare spending approximates $4.3 trillion,4 of which therapeutics accounts for ~15%, or $600 billion,5 as shown below.



In our view, novel gene-editing therapies could command premium pricing by shifting healthcare expenditures from chronic disease management to one-dose therapies like VX-880. Addressing diabetes, for example, therapeutics account for 30% of total direct and indirect costs, as shown below. If emerging drugs like VX-880 were to shift costs from chronic disease management to one-dose therapies, curative therapeutics could take share from chronic treatments.