AstraZeneca has no plans to ramp up dealmaking in wake of Wainua failure

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AstraZeneca remains “very much on track” to hit its 2030 goal of reaching $80 billion in revenue despite the recent late-stage failure of Wainua in ATTR-cardiomyopathy, CEO Pascal Soriot said during the company’s Q2 earnings presentation on Monday.

The recent surprise stumble of the antisense therapy Wainua in a late-stage ATTR-cardiomyopathy study doesn’t seem to have shaken AstraZeneca, which sees the failure as part of the drug-making business—and one not big enough to have any appreciable effect on its business development strategy moving forward.

“It doesn’t really impact our M&A strategy,” CFO Aradhana Sarin told reporters during a press call early Monday morning to present the company’s second quarter earnings report. “This is one setback amongst a whole slew of several positive studies.”

CEO Pascal Soriot agreed, noting that while the failure is unfortunate, “it happens, and it’s included in our risk adjustment.” Pointing to the pharma’s expansive pipeline, Soriot added that AstraZeneca doesn’t “need M&A to deliver.”

Aside from Wainua, AstraZeneca on Monday also disclosed several other clinical disappointments, primarily concerning its cancer portfolio.

Developed and commercialized in partnership with Ionis Pharmaceuticals, Wainua is a transthyretin silencer that works by suppressing the overall expression of the protein that is misfolded in ATTR, leading to disease-causing clumps across various organs. The therapy was approved in December 2023 for ATTR-polyneuropathy, and the partners are looking to expand into cardiomyopathy with the Phase 3 CARDIO-TTRansform trial.

That study failed earlier this month, however, which cast considerable doubt on AstraZeneca’s ability to meet its goal of $80 billion in revenue by 2030—a target set in 2024. At the time, the pharma named Wainua as a crucial contributor toward meeting that revenue bar.

The failure of AstraZeneca and Ionis’ Wainua in a late-stage study of ATTR-CM casts doubt on Alnylam’s next-generation candidate but is good news for others in the space, including BridgeBio and Intellia Therapeutics.

“Of course, as you would expect, we are disappointed” by this failure, Soriot told the press in his opening remarks on Monday. He asserted, however, that AstraZeneca remains “very much on track” to meet the 2030 goal.

“Biology is not mathematics, it’s not as predictable,” Soriot said of the failure. “Biology doesn’t always behave the way you would expect.”

On Monday, this unpredictability struck again as AstraZeneca reported another late-stage stumble: In the Phase 3 ALXN1210-TMA-313 trial, the pharma’s rare disease powerhouse Ultomiris failed to significantly improve event-free survival versus placebo in patients 12 years and older with thrombotic microangiopathy who had received hematopoietic stem cell transplant.

Ultomiris is approved for four rare conditions: paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, generalized myasthenia gravis and neuromyelitis optica spectrum disorder. The drug made $1.3 billion in the second quarter.

AstraZeneca revealed several other study failures on Monday, including the Phase 3 PACIFIC-8 trial, testing the PD-L1 inhibitor Imfinzi plus an anti-TIGIT antibody from Gilead in patients with stage 3 unresectable non-small cell lung cancer. Gilead, which was running the study alongside Arcus Biosciences, decided to discontinue the program after disappointing data in separate studies of their TIGIT asset.

Meanwhile, in the Phase 3 CLARITY-Gastric01 trial, AstraZeneca’s antibody-drug conjguate sonesitatug vedotin met just one of its primary efficacy endpoints: Overall survival in patients receiving the therapy after at least two prior lines of treatment. Sonesitatug vedotin did not show significant progression-free survival in the overall study population, according to the company’s H1 results presentation.

AstraZeneca posted revenues of nearly $15.4 billion in Q2, a 5% year-on-year increase at constant currencies. Much of this growth was driven by the pharma’s oncology portfolio, which surged 15% to hit $7.3 billion in sales. The company also pointed to its rare disease unit as a key driver, which jumped 8% to bring in $2.4 billion in the quarter.

Tagrisso, an oral drug for non-small cell lung cancer, reigned as AstraZeneca’s best-selling product in Q2 with $1.9 billion in revenue, up 6% from the same period last year. Close behind was Imfinzi, approved for various cancers, which surged 27% to make $1.9 billion.

Farxiga, an SGLT2 inhibitor indicated for chronic kidney disease, heart failure and type 2 diabetes, remained one of AstraZeneca’s top-performing assets with $1.8 billion in Q2 revenue—even as sales took a 19% dive after losing key patent protections.

Merck’s Keytruda may be the most talked about drug facing loss of exclusivity but it’s far from the only one, as several of the industry’s top-performers are losing key market protections. Some companies are more prepared than others.

Tristan is BioSpace‘s senior staff writer. Based in Metro Manila, Tristan has more than eight years of experience writing about medicine, biotech and science. He can be reached at tristan.manalac@biospace.com, tristan@tristanmanalac.com or on LinkedIn.
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