Vertex Hopes to Channel CF Drug Successes into Other Therapies in Pipeline

 Boston, Mass.-based Vertex Pharmaceuticals said in a second-quarter earnings call on Thursday that its revenue has increased 18% compared with Q2 2020, a rise solely driven by the company's six cystic fibrosis (CF) drugs. 

Vertex's Chief Executive Officer and President, Reshma Kewalramani, M.D., said during the call that the company is on track to reach 90% of all patients with CF and is working toward addressing unmet needs in the remaining 10%. 

In the second quarter of 2021, net product revenues for Vertex rose by 4% to $1.26 billion in the U.S. and by 71% to $536 million outside the U.S., compared with the second quarter of last year.

Vertex also recently announced plans to start a Phase III trial studying a combination regimen comprising three of its therapies for CF (VX-121, tezacaftor and VX-561) after positive Phase II data on the combination was reported. The triplet drug combination is being compared with Vertex's Trikafta, the company's current lead drug for CF. 

If findings from the late-stage trial show the triple-combination therapy is superior, it may replace Trikafta. The Phase III program will launch sometime in the second half of this year and will include two 48-week trials in 800 patients.

"We have now secured reimbursement agreements for the triple combination in more than 15 countries outside the U.S. and started expansion into younger age groups with the U.S. approval in patients 6 to 11 years of age last month," said Dr. Kewalramani, in a statement. "Looking forward, we continue to see significant growth ahead in CF, with more than 30,000 CF patients who may benefit from the triple combination but who are not yet treated."

While successful in CF, the company is eager to diversify, putting Phase II clinical trials in sickle-cell disease, beta-thalassemia, kidney diseases, and pain management. Data readouts for these trials are expected over the next six to nine months.

Dr. Kewalramani noted that the company is currently seeing "impressive clinical results" with its most advanced non-CF program, CTX001, having dosed more than 45 patients with sickle cell disease and beta-thalassemia with a CRISPR/Cas9 gene-editing therapy co-developed with CRISPR Therapeutics.

In June, Vertex and CRISPR Therapeutics announced data from two ongoing Phase I/II clinical trials of CTX001 in 22 patients with beta-thalassemia and sickle cell disease. Following infusion, all 15 patients with beta-thalassemia were transfusion independent and all seven patients with sickle cell disease were free of vaso-occlusive crises. 

"These results add to the growing body of evidence that CTX001 may hold the promise for a one-time functional cure for sickle cell disease and beta-thalassemia," said Dr. Kewalramani in a statement. "We are working with urgency to complete enrollment and look forward to finalizing regulatory discussions and moving towards filing."

Charlie Wagner, Vertex's executive vice president and chief financial officer, said during the earnings call that each of these therapeutic areas represents a significant opportunity for the company. For instance, the non-opioid pain management programs could help the company latch on to an over $4 billion market.

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