AstraZeneca released comprehensive results from two late-stage clinical trials on Tuesday for tozorakimab, an experimental biologic treatment for chronic obstructive pulmonary disease (COPD). The positive data has intensified expectations that the drug could become a blockbuster, potentially generating multibillion-dollar annual revenues for the pharmaceutical giant.
The Food and Drug Administration is currently reviewing the drug under priority status, with U.S. approval anticipated in the first quarter of 2027. Administered as a once-every-four-weeks injection, tozorakimab demonstrated a meaningful ability to reduce acute exacerbations, or flare-ups, across a broad spectrum of patients suffering from the progressive lung condition.
“We truly believe that a large population of COPD patients can benefit from this new medicine,” said Ruud Dobber, president of AstraZeneca’s biopharmaceuticals business unit. The trial data indicated that the treatment lowered moderate and severe flare-ups by 29% and 34%, respectively, among former smokers compared to a placebo. In the overall population of current and former smokers, the drug reduced these events by 30% and 29%.
The findings have prompted AstraZeneca to raise its peak annual sales forecast to more than $5 billion, a figure that supports the company’s broader objective of reaching $80 billion in total revenue by 2030. CEO Pascal Soriot highlighted the significant unmet need in the market, noting that many patients remain undiagnosed or are not candidates for existing therapies.
“We believe it can be more than 5 billion, simply because the unmet need is huge,” Soriot said. “Patients are underdiagnosed; they need to be better diagnosed, and biologics in this class are totally underused.” He pointed out that while asthma patients in some countries receive biologic treatments at rates of 30% to 40%, only about 10% of COPD patients currently benefit from such therapies.
Unlike current COPD biologics such as Sanofi and Regeneron’s Dupixent and GlaxoSmithKline’s Nucala, which are restricted to patients with high levels of eosinophils—a type of white blood cell—tozorakimab targets the IL-33 protein, a key driver of inflammation and mucus production. This mechanism allows it to potentially treat a wider demographic, including those with lower eosinophil counts.
Dr. Meilan Han, chief of the division of pulmonary and critical care at the University of Michigan Health and a study investigator, noted that there is currently no biologic option for COPD patients with blood eosinophil counts below 150. The new drug reduced flare-ups by 23% in this specific subgroup, compared to 34% in patients with counts of 150 or higher, and 43% in those with counts of 300 or above.
Soriot admitted that the drug’s success was unexpected given the historical struggles of other IL-33 inhibitors. “Tozorakimab is a product nobody thought would work. We ourselves had a very low probability of success,” he said. “Everybody thought it would fail. If you look at the consensus before we announced the results, the consensus had almost zero in its forecast, and it actually worked.”
AstraZeneca attributes this success to its antibody’s ability to inhibit two distinct IL-33 pathways, addressing inflammation, mucus production, and airway damage simultaneously, whereas earlier attempts focused on only one pathway.
Despite the enthusiasm, clinical questions remain regarding how tozorakimab fits into the current treatment hierarchy. Dr. Han stated she plans to prescribe the drug for patients with low eosinophil levels but emphasized the need for more detailed subgroup analyses to determine its optimal use relative to existing therapies for patients with high eosinophil counts. She also noted that the Global Initiative for Chronic Obstructive Lung Disease (GOLD) Science Committee will play a crucial role in establishing future clinical guidelines.
Regulatory review is ongoing in major markets including the European Union and China. Additionally, the drug is being investigated in Phase 2 trials for severe asthma and Phase 3 studies for severe viral lower respiratory tract disease.
Dobber expressed confidence that the drug could become one of the largest respiratory products in history. He also drew attention to environmental risk factors, such as air pollution and wildfire smoke, as increasingly important triggers for COPD. “Not everyone has a smoking history,” Dobber said, highlighting the need for greater awareness of the disease and efforts to mitigate environmental lung damage.
Interesting that AstraZeneca admitted they expected it to fail. Double pathways must make all the difference here.
Ive been waiting years for this. My dad is in the low-eosinophil group and has nowhere to turn right now.
Five billion in sales? That seems wildly optimistic given previous IL-33 failures. Let’s wait for real-world data before celebrating.
Finally, a biologic option for the vast majority of COPD patients with lower eosinophil counts. This could change everything.