Vertex Pharmaceuticals' Triple Combo Treatment For Lung Disorder Meets Primary Goal In Adult And Pediatric Patients

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. 

The Phase 3 program included two trials: SKYLINE 102 and SKYLINE 103, evaluating vanzacaftor (20 mg)/tezacaftor (100 mg)/deutivacaftor (250 mg) in patients aged 12 and above, compared to Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor). 

A third Phase 3 single-arm, 24‑week, open-label study, RIDGELINE 105, evaluated vanza triple in children ages 6 to 11.

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.

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Head-to-head against Trikafta, on the first key secondary endpoint, the vanza triple was superior in reducing sweat chloride (SwCl) levels in SKYLINE 102 and SKYLINE 103. 

In the second and third key secondary endpoints, which were pooled across SKYLINE 102 and SKYLINE 103, the vanza triple achieved superiority in the proportion of patients below 60 mmol/L (the diagnostic threshold for cystic fibrosis) and below 30 mmol/L (carrier level) compared to Trikafta.

Safety was the primary endpoint in the RIDGELINE 105 study in children 6 to 11. On the secondary endpoint measuring the absolute change in mean SwCl levels through week 24, the vanza triple reduced SwCl by -8.6 mmol/L compared to a baseline on TRIKAFTA. 

95% of children achieved SwCl levels below 60 mmol/L and most children treated with the vanza triple achieved normal levels of CFTR function with SwCl levels below 30 mmol/L.

Treatment with the vanza triple was well tolerated in all three studies.

Vertex is on track to make global regulatory submissions by mid-2024.

Monday, Vertex Pharmaceuticals reported fourth quarter 2023 adjusted EPS of $4.20, up from $3.76 a year ago, beating the consensus of $4.10.

The company reported sales of $2.52 billion, up from $2.3 billion a year ago, almost in line with the consensus of $2.51 billion.

Guidance: Vertex Pharmaceuticals expects fiscal year 2024 sales of $10.55 billion-$10.75 billion versus the consensus of $10.61 billion.

Vertex is on track to submit new drug applications to the FDA by mid-2024 for VX-548 in acute pain and vanzacaftor triple in cystic fibrosis.

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Price Action: VRTX shares are down 0.92% at $424.93 on the last check Tuesday.