AstraZeneca on Thursday revealed it has discontinued the development of two Alexion-developed rare disease assets due to underwhelming efficacy findings.
Vemircopan, an investigational small molecule drug, was culled from the pharma’s development pipeline “based on safety and efficacy data” from mid-stage studies, according to a press release issued on Thursday. The drug, an oral blocker of the alternative complement protein factor D, was being tested for impaired hepatic function, lupus nephritis or immunoglobulin A nephropathy, according to AstraZeneca’s clinical trials appendix.
AstraZeneca also dropped its early-stage enzyme replacement therapy ALXN1910, which it was trialing for bone metabolism indications. ALXN1910 was designed to target the activity of the TNSALP protein, which is crucial for bone mineralization.
Both vemircopan and ALXN1910 came from rare disease leader Alexion, which AstraZeneca acquired in December 2020 for $39 billion. The deal has paid off well for the pharma—giving it the high-performing assets Ultomiris and Soliris—but has also returned its fair share of disappointments.
In April 2023, for instance, AstraZeneca terminated its Wilson disease program, citing “feedback” from regulators that had reviewed its data. Meanwhile, in August 2021, the company reported that Alexion’s Ultomiris failed its Phase III study in amyotrophic lateral sclerosis. The company is no longer studying Ultomiris in this indication.
When asked in a media call if Thursday’s discontinuations shook the company’s confidence in the Alexion buy, CEO Pascal Soriot said AstraZeneca continues to believe that Alexion was a “fantastic acquisition.”
The discontinuations are part of “the life in our industry,” Soriot said. In biopharma, he added, “some [products] die, some others are accelerated, some new ones come in.”
Overall, AstraZeneca’s rare disease portfolio has seen a lot of growth.
In the fourth quarter of 2024, AstraZeneca recorded a 22% increase in revenue for its rare disease portfolio, hitting nearly $2.4 billion in revenue. Ultomiris, indicated for generalized myasthenia gravis, paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, accounted for much of this, surging 33% in the quarter to bring in nearly $1.1 billion.
Ultomiris will continue to be a key driver of growth for AstraZeneca in 2025, with the pharma working toward expansion into hematopoietic stem cell transplantation-associated thrombotic microangiopathy, cardiac surgery-associated acute kidney injury and IgA nephropathy, according to a company presentation.
A Strong Quarter, An Achievable Year
AstraZeneca made almost $14.90 billion in the fourth quarter, coming in slightly ahead of the consensus figure of $14.23 billion. Net income was at $3.24 billion, also exceeding analysts’ expectations of $3.21 billion. For the full year 2024, AstraZeneca recorded $54 billion in total revenue, a 21% year-on-year increase at constant exchange rates.
According to an investor note from Leerink Partners, much of AstraZeneca’s quarterly beat was driven by the better-than-expected performance of its oncology unit, which brought in $5.34 billion in the quarter to come 4% ahead of the consensus. Key cancer assets, including antibody-drug conjugate Enhertu and PARP inhibitor Lynparza, likewise outperformed expectations.
Outside of cancer—and aside from Ultomiris—other notable growth drivers include the inhaler Symbicort, the stroke therapy Brilinta and Strensiq, another rare disease asset indicated for perinatal/infantile- and juvenile-onset hypophosphatasia.
Looking ahead to 2025, AstraZeneca expects its total revenue to grow by a high single-digit percentage, while core earnings-per-share are projected to increase by a low double-digit percentage.
In an investor note Thursday, analysts at BMO Capital Markets called this goal “achievable” and in-line with the consensus expectations.
https://www.biospace.com/business/astrazeneca-axes-two-alexion-assets-as-q4-earnings-exceed-expectations
