JPM 2022: Vertex's Trikafta holds the line as company lays groundwork for gene editing launch

Reshma Kewalramani, chief medical officer at Vertex
Vertex sees potential for “significant” cystic fibrosis growth “well into the middle of the decade,” CEO Reshma Kewalramani said Monday at the J.P. Morgan Healthcare Conference. (Reshma Kewalramani)

Vertex Pharmaceuticals is well known for its slew of cystic fibrosis medications, and newcomer Trikafta is pulling its weight. Over the course of 2021, the med clinched reimbursement in 16 countries and launched for kids ages 6 to 11 in the U.S.

Together, those advances teed up “thousands” of new patients starting on the nascent blockbuster. Meanwhile, Vertex sees potential for “significant” cystic fibrosis growth “well into the middle of the decade,” CEO Reshma Kewalramani said during the company’s presentation at the 2022 virtual J.P. Morgan Healthcare Conference.

There are an estimated 83,000 patients in the U.S., Europe, Australia and Canada, the CEO said. Vertex “reached thousands of new patients in the past year,” but there are “still more than 25,000” who could benefit from Trikafta who aren’t yet on therapy, Kewalramani said.

She broke those patients down into three groups: Those who’ve yet to start on Trikafta in countries where the drug was recently reimbursed, patients in geographies where the drug’s yet to be reimbursed and younger patients, whom Vertex aims to address through future label expansions.

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Vertex used its JPM presentation to pad Trikafta's case with real-world data garnered from more than 16,000 U.S. patients. The results showed the drug led to an 87% reduction in risk of lung transplant, 77% fewer pulmonary exacerbations and a 74% reduction in risk of death.

The data “set a very high bar for any future therapy to meet,” Kewalramani said.

Vertex also has an mRNA therapy for cystic fibrosis that is in clinical trials. The company has “line of sight” to some 5,000 or more patients who could benefit from the investigational candidate, Kewalramani said.

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Also in development is a gene editing program for sickle cell disease and beta thalassemia, CTX001, which Vertex is banking on as its next commercial launch, Kewalramani said. The company plans to share more data on CTX001 in sickle cell disease and beta thalassemia this year and aims to submit global filings asking for approval toward the “tail end of 2022,” Kewalramani said.

Patient and doctor interest in the gene editing program is “strong,” the CEO added. Vertex is now wrapping up talks with regulators to finalize the data package for submission. The company foresees “tremendous” market opportunity for the med, predicting there are more than 150,000 patients in the U.S. and Europe with beta thalassemia or sickle cell disease. Some 32,000 of those patients have severe disease. Some 25,000 are severe sickle cell disease patients, the vast majority of which are in the U.S., Kewalramani said.

Meanwhile, Vertex’s precommercial efforts on the gene editing program are “well underway,” Kewalramani added. The company is beefing up manufacturing, it’s hired many of the people who will support the drug’s launch and it’s now meeting with doctors and patients.

As for business development, the company’s strategy seeks to reel in “complementary assets” and cystic fibrosis technologies for Vertex’s “toolbox,” plus programs that fit its R&D strategy, Kewalramani said.