The new combination, dubbed Alyftrek, is designed to improve on the Trikafta, a product that generated sales of $8.9 billion in 2023. It’s welcome good news for Vertex following last week’s subpar clinical results for its non-opioid analgesic.
Vertex Pharmaceuticals said Friday the FDA has approved its next cystic fibrosis product Alyftrek, clearing the biotech to market a product designed to improve on Trikafta.
William Blair analysts see the early approval of Alyftrek, which had a PDUFA of Jan 2., 2025, as a positive for the company’s stock, though it has yet to rebound from last week’s crash following disappointing results from a Phase II trial of its non-opioid painkiller. That data drop sent the share price tumbling more than 10%, and the stock continues to hover at that depressed level.
Like Trikafta, the newly approved product is a triple-combination therapy intended for use in people with at least one F508del mutation or another mutation in the CFTR gene that is responsive to the treatment. The products only share one active ingredient, though, and Vertex said the combination of drugs used in Alyftrek has benefits over Trikafta, which generated sales of $8.9 billion in 2023.
Stuart Arbuckle, chief operating officer at Vertex, said at a Stifel event in November that the profile of Alyftrek “overall is incredibly appealing to both physicians and patients” but admitted “I’m not sure it’s necessarily as clear on its face as why.” The appeal reflects the “sum of the parts,” the Vertex executive said, rather than any single standout feature.
Non-inferiority to Trikafta on lung function was “basically the ticket to entry into this marketplace” and Alyftrek met that mark, the executive said. Beyond that, Alyftrek “showed greater levels of CFTR function control as measured by improved reductions in sweat chloride,” Arbuckle said, and has demonstrated efficacy in 31 additional mutations. The final benefit is Alyftrek is given once a day, as opposed to twice a day for Trikafta.
Arbuckle predicted “there’s going to be a lot of enthusiasm from patients who . . . have tried a CFTR modulator before and not been able to stay on one.” There are more than 6,000 people globally who have tried one of Vertex’s CFTR modulators only to stop treatment.
The biotech sees those people, who came off CFTR modulators for a range of reasons, as one group of potential Alyftrek users. Arbuckle also sees opportunities to market Alyftrek to patients who are already taking one of Vertex’s other treatments.
Leerink Partners analysts said in a note to investors that “converting patients from Trikafta to Alyftrek should be fairly straightforward because it is a once daily vs. Trikafta twice daily, and in Ph3, Alyftrek demonstrated non-inferiority vs. Trikafta on the primary endpoint of change in ppFEV1 (a measure of lung function) and superiority on the secondary outcome of reduction in sweat chloride.”
William Blair analysts offered a contrasting view in their note, warning that “the positive clinical experience with Trikafta could be an initial headwind to switching patients without an FEV1 superiority claim.” The William Blair team said it expects “penetration into the estimated 20,000 eligible patients not currently on therapy and a slower switch rate from Trikafta” than seen in other launches.
Vertex has established a list price of around $370,000 for Alyftrek. Leerink analysts said the price is a 7% premium over Trikafta.
