- AbbVie's first attempt at challenging the world's top-selling cystic fibrosis medicine has fallen short, though the company plans to forge ahead with another treatment expected to begin mid-stage testing early next year.
- Like Vertex's Trikafta, AbbVie's treatment joins together three drugs meant to correct and regulate the dysfunctional proteins that cause cystic fibrosis. AbbVie had been evaluating this triplet, considered by some the biggest threat to Vertex's franchise, in a mid-stage study. But according to the company, a recent interim analysis of the data found the addition of the third drug — known as ABBV-119 — did not offer a significant improvement on two key health measures for cystic fibrosis patients.
- As such, AbbVie has decided not to advance this specific combination further. It does, however, intend to substitute a different experimental drug in place for ABBV-119, for another shot at a triplet therapy. AbbVie said this newer drug is structurally distinct from its predecessor and provides higher drug exposure.
For the past decade, Vertex has held a commanding position in treating cystic fibrosis. The Boston-based company has brought four products to market, providing treatment options to an estimated 90% of patients. Trikafta, its newest offering, has been especially successful, accounting for $5.7 billion of the nearly $7.6 billion in net product revenue Vertex recorded last year.
Several rival drug developers have tried to unseat Vertex, but so far none have been able to. AbbVie remains one of the largest threats in the eyes of Wall Street analysts, who've kept close watch on the company's research and, in particular, its three-drug therapy.
AbbVie's current work in cystic fibrosis has its roots in a 2013 collaboration with Galapagos, a Belgian biotech. Though Galapagos hit setbacks developing two- and three-drug treatments for cystic fibrosis, AbbVie saw promise in the company's research and, in 2018, decided to take full control over it through an amended deal.
AbbVie has since been testing two experimental drugs, called ABBV-3067 and ABBV-2222, as both a doublet and as a triplet with ABBV-119. ABBV-3067 is designed to be a "potentiator," meaning it regulates the central protein responsible for cystic fibrosis, whereas the other two drugs are "correctors," meaning they help the protein hold the proper shape.
Trikafta has a similar composition, made with two correctors and one potentiator.
For AbbVie's triplet to move forward, it needed positive data from a mid-stage, so-called proof-of-concept study. However, recent interim results showed the combination didn't meet criteria set by AbbVie to develop it further, according to Chief Scientific Officer Thomas Hudson.
AbbVie designed its study so that some patients would start on the doublet for about a month, after which they would get the triplet for another four weeks. Others would receive just the triplet or placebos. Hudson said that, per the interim analysis, the addition ABBV-119 to ABBV-3067 and ABBV-2222 didn't provide a meaningful improvement on two measures often used to evaluate the effectiveness of cystic fibrosis drugs.
Hudson did say that the doublet "performed well," though, with effectiveness comparable to existing two-drug regimens. AbbVie has another corrector in early-stage studies that it now intends to advance as a new triplet with ABBV-3067 and ABBV-2222. The company expects to start a Phase 2 study of this combination in early 2023.
Despite those plans, AbbVie's share price fell more than 8% by late morning Friday as, along with the cystic fibrosis update, a few of the company's top drugs also missed their sales targets.
Vertex shares, meanwhile, rose about 2.5%, as competition has yet again been delayed.
To Michael Yee, an analyst at the investment firm Jefferies who covers Vertex, AbbVie's results reflect the "high efficacy bar" the company has established against would-be competitors — which, even if met, would be difficult to challenge given the "already nearly widespread uptake" of its drugs.
It's "frankly amazing" that one company has been able to keep an $11 billion market to itself with "virtually no competition in sight," wrote Evercore ISI analyst Liisa Bayko, who covers Vertex, in a note to clients.