In a battered field, AstraZeneca's lupus drug clears pivotal study — but can they get it approved now?
A year after AstraZeneca admitted defeat in a late-stage study testing its lupus drug anifrolumab, the British drugmaker has cleared their second pivotal trial, hitting the primary endpoint and leaving their research team hopeful that they have enough data in hand to get an approval.
Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP 1 study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — and the study met the main goal of reducing disease severity.
Both trials measured disease activity using the British Isles Lupus Assessment Group based Composite Lupus Assessment (BICLA) — which requires improvement in all organs with disease activity at baseline with no new flares — at week 52. In typical fashion, the company is holding the data back for a scientific conference.
Regulators, though, won’t have to wait.
Usually, a drug developer needs positive data from two pivotal trials to secure approval, but regulators have proved increasingly likely to overlook that standard — particularly if they’re up against a big challenge like lupus. AstraZeneca’s Mene Pangalos is looking to capitalize on that, in a statement on Thursday he said the company is exploring pathways to get anifrolumab on the market.
The drug is a monoclonal antibody engineered to thwart the activity of all type I interferons — cytokines involved in inflammatory pathways. Roughly 60% to 80% of adults with systemic lupus erythematosus (SLE) carry increased type I interferon gene signature, according to AstraZeneca $AZN.
Lupus is a drug developer’s nightmare. In the last six decades, there has been one FDA approval. In recent years, the field has resembled a graveyard. Last October, UCB and Biogen‘s $BIIB anti-CD40L drug failed in a late-stage study, months after Xencor $XNCR and Sanofi’s $SNY Ablynx also conceded defeat in their programs.
Meanwhile, there is cause for some cautious optimism. Some biologics that are approved for other autoimmune disease are being tested for use in lupus — including Eli Lilly’s $LLY Olumiant and J&J’s $JNJ Stelara. French biotech Neovacs is also in mid-stage development with a lupus vaccine.
The only biologic so far to win approval for lupus is GSK’s $GSK Benlysta — which was cleared for adult use in 2011 and for rare cases of childhood lupus this year. (GSK is in the midst of testing Benlysta in combination with Roche’s rituximab in the hope the combination will have a more potent effect on the disease versus Benlysta monotherapy.)
Apart from that, patients are usually given NSAIDS, antimalarial drugs, corticosteroids and immunosuppressants to control the symptoms of the systemic autoimmune disease, in which the body’s immune system launches an attack on its own tissues and organs. About 1.5 million Americans and at least five million people globally suffer from a form of lupus, estimates The Lupus Foundation of America.