- AstraZeneca on Thursday said its lupus drug anifrolumab succeeded in a Phase 3 trial, scoring "statistically significant and clinically meaningful" reductions in disease activity among 373 patients.
- Success in this trial, called TULIP 2, comes almost exactly a year after the British pharma announced the failure of anifrolumab in TULIP 1, which used a different primary goal to define success. To approve a new drug, regulators often ask for two successful trials.
- The last lupus drug to reach the market was GlaxoSmithKline's Benlysta, which had mixed results in the clinic yet achieved Food and Drug Administration approval in 2011. It was the first lupus drug to achieve approval in decades.
Mixed clinical results for anifrolumab puts AstraZeneca in a tough position.
Had TULIP 2 failed, it could have written off anifrolumab as a failure, but now that it has positive results it will need to talk with the FDA and European Medicines Agency about a pathway for approval. That could very well include a third confirmatory trial, which could take several more years.
Anifrolumab blocks the production of inflammatory proteins called interferons, which the body typically manufactures to block viral replication. Interferons are elevated in lupus patients.
In TULIP 2, patients were treated every four weeks with 300 milligrams of anifrolumab. After a year of treatment, patients were measured for response using a lupus assessment score called BICLA.
AstraZeneca didn't release any specific numbers from TULIP 2, stating that results from both TULIP 1 and 2 would be presented at an upcoming medical meeting.
Last year, TULIP 1 failed to meet its primary endpoint, defined as improvement on a scale called the systemic lupus erythematosus (SLE) responder index.
The BICLA composite in TULIP 2 included the responder index, but a positive BICLA score could be achieved even without improvement on that measure. Physicians who treat lupus patients will likely be keen to see how the individual components affected the overall BICLA score.
Even without two successful trials in hand, AstraZeneca may have a shot at approval. Benlysta (belimumab) won FDA support on mixed clinical outcomes, with one of its three pivotal trials showing no benefit over placebo.
GlaxoSmithKline reported Benlysta sales of 473 million pounds ($577 million) in 2018.