Belimumab

With litifilimab, Biogen is going after the type 1 interferon signature by inhibiting BDCA2.\n Biogen has bagged $250 million to advance a key pipeline prospect. Royalty Pharma is providing the cash to bankroll phase 3 development of a lupus candidate that Biogen has named in a bunch of assets with potential peak sales of $14 billion.The candidate, the anti-BDCA2 antibody litifilimab, is in phase 3 development in systemic and cutaneous lupus erythematosus (SLE/CLE). There is unmet need in both diseases. SLE patients have limited choices, with GSK’s Benlysta and AstraZeneca’s Saphnelo the only options, but that is rich pickings compared to CLE. Physicians have been waiting decades for an advance in the treatment of the cutaneous disease.Biogen believes litifilimab can address the unmet needs. But, with a pipeline of other programs—including two other lupus assets—to develop, the biotech has opted to seek external money to fund phase 3 trials in SLE and CLE.Royalty is providing the money, agreeing to drip-feed Biogen $250 million over six quarters in exchange for regulatory milestones and mid-single-digit royalties on annual worldwide sales. Royalty, a buyer of biopharmaceutical royalties, already receives a cut of the sales of Biogen’s Tysabri and Spinraza. The firm secured those royalties through deals with Ionis and Perrigo rather than directly from Biogen. Biogen has prioritized its pipeline under CEO Christopher Viehbacher, helping it cut R&D spending by 17% last year. The Royalty deal supports ongoing cost management while still allowing Biogen to attempt to bring litifilimab to market.Viehbacher talked up litifilimab’s prospects on an earnings call in October, including the asset among a clutch of candidates that the CEO said could collectively generate peak sales of $9 billion to $14 billion. CLE is an untapped opportunity. Litifilimab will face competition if it comes to market in SLE, potentially from Biogen’s own dapirolizumab, but Viehbacher sees room for multiple molecules.“This is a complex disease that affects different organs at a time,” Viehbacher said at the J.P. Morgan Healthcare Conference last month. “I think it\'s going to be a little like MS. You\'re really going to decide which product is best at which point in time for every patient. And so I don\'t think this is one product for the whole disease.”Dapirolizumab inhibits the CD40 ligand to reduce activation of B and T cells and downregulate interferon. With litifilimab, Biogen is going after the type 1 interferon signature by inhibiting BDCA2 in the plasma delta. The biotech is aiming to file for approval of litifilimab in SLE and CLE from 2027 to 2029.

Lupus nephritis is an inflammation of the kidneys caused by systemic lupus erythematosus (SLE). Credit: MirageC via Getty Images. Roche’s subsidiary Genentech has shared positive Phase III data with the US Food and Drug Administration (FDA) as the company targets the billion-dollar lupus market for its CD20-targeting drug Gazyva (obinutuzumab). While the market for systemic lupus erythematosus (SLE) and lupus nephritis therapies remains dominated by generic drugs, the FDA has approved only a few targeted biologics, including GSK ’s Benlysta (belimumab) and AstraZeneca ’s Saphnelo (anifrolumab), specifically for SLE in over 50 years . The latest Phase III data for Gazyva is important because, if approved, the drug would represent the first CD20-directed therapy for lupus nephritis with regulatory backing. The Phase III REGENCY trial (NCT04221477) – which enrolled 271 patients – demonstrated that 46.4% of patients with lupus nephritis who received Gazyva plus standard therapy achieved complete renal response at 76 weeks, compared to 33.1% in the placebo group. The findings were presented at the World Congress of Nephrology in New Delhi, India, with Genentech confirming that the data has been shared with both the FDA and European Medicines Agency (EMA) for regulatory review. Lupus nephritis is a severe manifestation of SLE that involves kidney inflammation and significantly increases the risk of organ damage and mortality. Current treatment options rely on immunosuppressive therapies, including steroids and mycophenolate mofetil (MMF), which have well-documented safety concerns such as infection risk, organ toxicity, and long-term complications. The positive REGENCY data comes over 15 years after the failure of Roche’s other B-cell depleting therapy, Rituxan (rituximab), in lupus nephritis. Co-developed by Genentech and Biogen , Rituxan remains widely used off-label for lupus nephritis despite the lack of FDA approval. The Phase III LUNAR trial (NCT00282347), which evaluated the therapy in lupus nephritis, did not meet its primary endpoint back in 2012 – failing to show a significant improvement in renal outcomes when added to standard therapy. Despite these results, real-world evidence has supported the use of Rituxan for refractory lupus cases, which GlobalData estimates generated approximately $2.5bn in sales in 2023 despite being an off-label therapy for lupus. Like Rituxan, Gazyva is a monoclonal antibody that targets CD20, a protein expressed in B cells. However, Gazyva is glycoengineered to induce greater B-cell depletion than Rituxan. The drug is already approved in the US for several haematological malignancies, including chronic lymphocytic leukaemia and follicular lymphoma, and its potential expansion into autoimmune diseases could significantly increase its market presence. GlobalData predicts that Gazyva could generate up to $1.7bn in sales by 2030 if approved for lupus nephritis. Genentech is also studying Gazyva in other autoimmune indications, including SLE, childhood-onset idiopathic nephrotic syndrome, membranous nephropathy, and paediatric lupus nephritis. The company has not disclosed a timeline for regulatory decisions but expects to work closely with agencies to secure approval for Gazyva in lupus nephritis. The global market for SLE and lupus nephritis is projected to reach $4.8bn by 2031, as per a report on GlobalData’s Pharma Intelligence Center. GlobalData is the parent company of Pharmaceutical Technology.