Vertex's Next-Generation Cystic Fibrosis Program Shows Efficacy With Reduced Burden, Provides Growth Opportunity: Goldman Sachs

Monday, Vertex Pharmaceuticals Incorporated VRTX released results from its once-daily vanzacaftor/tezacaftor/deutivacaftor (vanza triple) program for cystic fibrosis (CF), a progressive, multi-organ disease caused by dysfunction of the CFTR protein. 

In SKYLINE 102 and SKYLINE 103, the primary endpoint of absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1, measurement of lung function) through week 24 was met and showed that treatment with vanza triple was non-inferior to treatment with Trikafta.

Concurrently, the company released its fourth quarter 2023 financial results.

Goldman Sachs analysts acknowledge the debate on the clinical meaningfulness of sweat chloride, as physicians do not routinely measure it; it is the direct marker of CFTR protein function (which underlies disease pathophysiology). 

The analyst reiterates the Buy rating with a price target of $578 (versus $559 prior).

The analysts also note the recent results for the pain program (regulatory filing by mid-2024) and write that Vertex emerges with two large commercial verticals to support the long-term growth outlook as JP Morgan monitors the pipeline.

Treatment with VX-548 following abdominoplasty or bunionectomy surgery showed a statistically significant improvement in pain intensity difference from 0 to 48 hours (SPID48) compared to placebo and a clinically meaningful reduction in pain from baseline at 48 hours on the Numeric Pain Rating Scale (NPRS) in both studies.

JP Morgan highlights the advantages of the vanzacaftor triple therapy, emphasizing its potential for improved clinical outcomes compared to Trikafta. 

The key merits include its once-daily administration, in contrast to the twice-daily requirement of Trikafta, and a significantly lower royalty burden for vanzacaftor compared to the high-single-digit royalties associated with Trikafta.

The analyst notes that patients can seamlessly transition from Trikafta to vanzacaftor triple therapy without a washout period. 

This triple combination therapy presents a commercial opportunity, allowing patients to shift from Trikafta and potentially attract around 6,000 individuals who had previously stopped using one of Vertex’s other cystic fibrosis (CF) medications. 

This transition is anticipated to contribute to the overall growth of Vertex’s CF franchise revenue.

Price Action: VRTX shares are down 2.89% at $416.51 on the last check Tuesday.