AbbVie tosses its triplet therapy for cystic fibrosis, leaving Vertex to reign supreme
AbbVie went all in on cystic fibrosis a few years ago, shelling out $245 million for a portfolio of drugs from Galapagos in the hopes of dethroning Vertex’s blockbuster Trikafta. On Friday, it became clear that Vertex still reigns supreme — at least for now.
AbbVie’s sending its experimental triplet therapy to the scrap heap after it whiffed on an interim analysis in a Phase II proof-of-concept study, execs revealed during the Q1 call.
“The efficacy results from this interim analysis did not meet our pre-specified criteria for advancing this triple therapy and development,” Tom Hudson, CSO and senior VP of R&D, said.
The study tested a dual combination therapy — including AbbVie’s C1 corrector ABBV-2222 and potentiator ABBV-3067 — alongside C2 corrector ABBV-119. Researchers concluded that the addition of ABBV-119 “did not provide a meaningful improvement in FEV1,” or forced expiratory volume in 1 second, an established marker of CF. The addition of the C2 corrector also didn’t lead to a meaningful reduction in sweat chloride concentration over the dual therapy.
“We were able to again assess the efficacy of our dual therapy, which performed well,” Hudson said. “So based on the performance of our dual therapy, we plan to continue our CF program.”
The company is lining up a new C2 corrector, ABBV-567, which it plans to test as a triplet therapy with the existing C1 corrector and potentiator in a Phase II study launching before early next year.
“‘576 is structurally distinct from our previous C2 corrector ‘119 and has a better PK profile, and provides higher drug exposure which has the potential to deliver better efficacy,” Hudson said.
AbbVie’s stock $ABBV was down more than 8.5% on Friday, trading at around $149.92 per share.
R&D chief Michael Severino, who’s set to leave the company at the end of next month, made clear earlier this year that AbbVie intends to “exceed the bar that is being set by Vertex,” either by superior efficacy marks or a better tolerability profile.
“We want to have a regimen that is very competitive that delivers efficacy to patients in the manner that they need to improve their long-term functioning and ultimately, long-term survival,” he said back in January.
Now, AbbVie’s Phase II flop has cleared the way for Vertex to maintain its tight grip on the CF market. Trikafta — Vertex’s triplet therapy darling first approved back in 2019 — raked in more than $7.5 billion last year. And the company has its own next-gen therapy in the works, as CEO Reshma Kewalramani aims to stay one step ahead of the competition.
“It’s frankly amazing (and I struggle to find another example of this) that 1 company (VRTX) has the entire $11B CF modulator market to themselves but virtually no competition in sight!” Evercore ISI’s Liisa Bayko and Jingming Chen wrote in a note to investors on Friday.
That wasn’t the only blow AbbVie suffered in its Q1 results. Humira — AbbVie’s star rheumatoid arthritis drug that’s expected to face a slew of generic rivals in the US next year — missed sales estimates in Q1, offset by biosimilar competition in other parts of the world.
The drug brought in $4.7 billion, down 2.7% on a reported basis. As several biosimilar versions prepare for US launches next year, AbbVie is depending on Skyrizi and Rinvoq to make up for any losses.
“We expect combined peak sales for Skyrizi and Rinvoq to exceed the peak revenues achieved by Humira,” CEO Rick Gonzalez said earlier this year, upon reading out the company’s Q4 results.
A number of variables will impact the effect of biosimilars on Humira sales, Gonzalez said on the call, including pricing and availability.
“There’s no market like the United States for Humira anywhere around the world,” he said. “The United States is significantly larger than any other market around the world. There are certainly biosimilar players that are like an AbbVie, and I would expect them to have manufacturing capacity. There are generic players that could have sufficient manufacturing capacity. And then there are very small companies.”
Skyrizi, meanwhile, brought in $940 million last quarter, up 63.7%. Execs touted a recent launch in psoriatic arthritis, as well as an upcoming decision in Crohn’s disease. Rinvoq sales soared 53.6% to $465 million last quarter, but still fell short of sales estimates.
“We expect growth in the second-line-plus RA setting going forward where our field force is now focused on leveraging compelling data from two important Phase III trials,” said Jeff Stewart, executive VP and chief commercial officer.
In 2019, AbbVie sealed a $63 billion deal to buy out Allergan as another way to reposition itself ahead of the loss of US patent protection for Humira. And according to the company, it’s now paying off. The aesthetics unit was a key growth driver in Q1, AbbVie said, with the Botox franchise raking in $641 million.
“Aesthetics is once again exceeding expectations,” Gonzalez said. “The category continues to grow robust double digits, especially in toxins and fillers, where there is substantial opportunity for further market penetration.”
Correction: AbbVie’s new C2 corrector is called ABBV-576