Buying Alexion remains a “fantastic acquisition” for AstraZeneca, the Big Pharma’s CEO has said, even as the company takes a $753 million hit for abandoning one of the drugs from that acquisition.
The drug in question, vemircopan, was once a hot prospect at AstraZeneca. In 2022, the company listed (PDF) the asset as part of a wave of new molecules with significant potential in 2025 and beyond and named it one of the candidates from its $39 billion Alexion buyout deal that could drive incremental rare disease growth beyond Soliris and Ultomiris.
AstraZeneca stopped developing vemircopan in paroxysmal nocturnal hemoglobinuria in 2023 but kept going in other indications. Work in the last of those indications terminated last month, when the company ended a phase 2 trial in patients with lupus nephritis or immunoglobulin A nephropathy and a phase 1 study in people with impaired hepatic function. It follows the abandonment of other Alexion drugs in the form of ALXN1840 for Wilson disease and ALXN1820 for sickle cell disease.
The drugmaker said (PDF) in its fourth-quarter clinical trials update that it stopped the studies because of a lack of efficacy. AstraZeneca used slightly different language when disclosing the broader termination of the program, attributing the decision to phase 2 safety and efficacy data.
Marc Dunoyer, CEO of AstraZeneca's rare disease group Alexion, told Fierce Biotech at a press conference Thursday morning in London that Alexion “is not immune to the odds of the industry, where not all of the pioneering work that you do is going to work.”
“Returning to vemircopan, these were phase 2 studies,” Dunoyer said. “And obviously, when you add the level of phase 2 studies, the risk is even higher than in phase 3, because you progress each asset, there's nothing specific about it. It's just looking at the delicate risk of each molecule, and also the competitive field that you are you want to enter,” he continued. “So if this is based on this, we made [the] decision to target an asset.”
Axing vemircopan contributed to more than $1 billion in impairment charges in the quarter, but, when asked by Fierce whether this affected whether the Alexion buyout still seemed good value for money four years on, AstraZeneca CEO Pascal Soriot had no doubts.
“I can say with no hesitation, the Alexion acquisition was a fantastic acquisition,” Soriot said.
Vemircopan was joined on the scrap heap today by the radiopharmaceutical FPI-2059, which brought with it another $165 million impairment charge. AstraZeneca acquired FPI-2059 last year through its takeover of Fusion Pharmaceuticals. The drugmaker took the hit because of “portfolio prioritization decisions” and is still developing other Fusion assets.
The company disclosed the vemircopan and FPI-2059 setbacks as part of a pipeline update that included the removal of four other phase 2 programs. AstraZeneca stopped phase 2 trials of mitiperstat in heart failure and metabolic dysfunction-associated steatohepatitis because of strategic portfolio prioritization. The company completed a 711-patient phase 2b mitiperstat heart failure trial last year.
Sabestomig, a PD-1xTIM3 bispecific antibody, was the other victim of AstraZeneca’s strategic portfolio prioritization. The company was testing the candidate in patients with lung and gastric cancers in a phase 1/2a trial but stopped enrollment well short of its target in December.
Data did for the other two programs. AstraZeneca scrapped a phase 2 trial of anti-LIF antibody AZD0171 in pancreatic cancer because of efficacy data, although it still lists the asset as part of the multiregimen NeoCOAST-2 trial. And the company ended a phase 2 trial of its opioid use disorder prospect AZD4041 over the safety of the orexin 1 receptor antagonist.