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AstraZeneca Discontinues Two Alexion Rare Disease Programs Amid Strong Q4 Performance

• AstraZeneca has terminated the development of two Alexion-acquired assets - vemircopan for rare liver and kidney conditions and ALXN1910 for bone metabolism disorders - due to insufficient efficacy data.

• The company's rare disease portfolio showed robust growth with Q4 2024 revenue reaching $2.4 billion, driven by Ultomiris which generated nearly $1.1 billion, representing a 33% increase.

• Despite the setbacks, AstraZeneca reported strong Q4 earnings of $14.90 billion, exceeding analyst expectations, with significant contributions from its oncology portfolio and rare disease treatments.

AstraZeneca announced the discontinuation of two rare disease drug candidates acquired through its Alexion acquisition, while simultaneously reporting stronger-than-expected fourth-quarter financial results for 2024.
The pharmaceutical giant has halted the development of vemircopan, an oral complement factor D inhibitor being evaluated for multiple conditions including impaired hepatic function, lupus nephritis, and immunoglobulin A nephropathy. The decision was based on safety and efficacy data from Phase II studies. Additionally, the company terminated the development of ALXN1910, an early-stage enzyme replacement therapy targeting TNSALP protein for bone metabolism disorders.

Impact on Alexion Portfolio Performance

Despite these setbacks, AstraZeneca's CEO Pascal Soriot maintained his confidence in the $39 billion Alexion acquisition from December 2020, describing it as a "fantastic acquisition." The rare disease portfolio has demonstrated significant growth, with Q4 2024 revenue reaching nearly $2.4 billion, marking a 22% increase.
Ultomiris, a flagship product from the Alexion portfolio, emerged as a particular bright spot, generating almost $1.1 billion in the fourth quarter alone – a 33% surge compared to the previous year. The drug, approved for conditions including generalized myasthenia gravis, paroxysmal nocturnal hemoglobinuria, and atypical hemolytic uremic syndrome, continues to drive growth with planned expansions into additional indications.

Strong Financial Performance

AstraZeneca's overall financial performance exceeded market expectations, with Q4 revenues reaching $14.90 billion, surpassing the consensus estimate of $14.23 billion. Net income stood at $3.24 billion, also beating analyst projections of $3.21 billion. The company's full-year 2024 revenue totaled $54 billion, representing a 21% year-on-year increase at constant exchange rates.
The oncology division played a crucial role in the quarter's success, generating $5.34 billion in revenue, with strong performances from key products including the antibody-drug conjugate Enhertu and PARP inhibitor Lynparza. Other significant growth drivers included the respiratory medication Symbicort, cardiovascular drug Brilinta, and the rare disease treatment Strensiq.

Future Outlook

Looking toward 2025, AstraZeneca has projected high single-digit percentage growth in total revenue and low double-digit percentage increase in core earnings-per-share. These projections align with market consensus and have been deemed "achievable" by BMO Capital Markets analysts.
The company continues to advance Ultomiris's development program, with ongoing efforts to expand its use in conditions such as hematopoietic stem cell transplantation-associated thrombotic microangiopathy, cardiac surgery-associated acute kidney injury, and IgA nephropathy.
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