AstraZeneca dumps $855M, near-approval rare disease drug after talks with regulators
BIOMARKER
1. AstraZeneca has discarded a rare disease drug candidate once valued at $855 million following two failed phase 2 trials.
2. This drug candidate had previously shown promise in a larger phase 3 trial.
3. Alexion had originally acquired this phase 3 asset, known as WTX101 or ALXN1840, from Wilson Therapeutics in 2018 for $855 million.
4. AstraZeneca, after acquiring Alexion for $39 billion, announced last year that a phase 3 trial of ALXN1840 had met its primary endpoint.
5. AstraZeneca had planned to file for approval in the second half of 2023, but two phase 2 trials missed their primary endpoints.
6. The decision to drop ALXN1840 was based on phase 2 results and discussions with regulatory authorities, according to AstraZeneca's CFO, Aradhana Sarin.
7. CEO Pascal Soriot elaborated that the phase 2 studies failed to demonstrate that copper was effectively eliminated from the body.
8. AstraZeneca also announced the removal of cotadutide, a GLP-1/glucagon dual agonist, from its phase 2 pipeline.
9. The company halted enrollment in a phase 2/3 NASH trial of cotadutide after recruiting only 45 of the targeted 1,860 patients.
10. Lastly, AstraZeneca discontinued a phase 1 trial of its anti-GDF15 candidate AZD8853 due to "strategic portfolio prioritization".