Novartis gets breakthrough designation for Xolair successor, setting up spotlight for PhIII readout later this year

14 Jan 2021
endpts.com
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With its Roche-partnered Xolair no longer under patent protection, Novartis and CEO Vas Narasimhan are looking for the next drug up to step into the sales void. Now, Narasimhan’s targeted successor is getting some love from the FDA, and it could spell a filing down the road. The FDA granted breakthrough therapy designation to the company’s ligelizumab compound for the treatment of chronic spontaneous urticaria in patients who respond poorly to antihistamines, Novartis said Thursday. Expected to be the successor for Xolair after the Roche-partnered drug lost patent protection in 2017, ligelizumab was highlighted by Narasimhan in an R&D presentation late last year as one of six mid-to late-stage programs that Novartis hopes will be slam dunk blockbusters. CSU, sometimes also called chronic idiopathic urticaria, is a disease that affects the skin, causing unpredictable breakouts of swelling and hives for periods lasting at least six weeks. The disease can generally persist for anywhere between one and five years, Novartis said, but sometimes lasts even longer. Ligelizumab aims to attack the disease by blocking the IgE/FcεRI pathway, thought to be an important factor in causing skin inflammation. The compound itself is a monoclonal anti-IgE antibody currently being studied in two Phase III studies with more than 2,000 patients. Data are expected in the second half of this year, and should they come back positive, a regulatory filing could follow in 2022. But it’s Phase II data from late 2018 that has Novartis confident in the candidate’s potential success. The pharma said ligelizumab surpassed Xolair in the mid-stage study, with three different dosage groups all notching higher response rates compared to the Xolair control. In the 382-patient trial, patients were either given ligelizumab at a dose of 24 mg, 72 mg, or 240 mg, Xolair at a dose of 300 mg, or placebo. Injections were given every four weeks over a 20-week period, and all patients were already on antihistamines. There was also a patient group that received a single 120-mg dose of ligelizumab. After 12 weeks, the rate of patients on ligelizumab who had complete control of their hives reached 30%, 51%, and 42% in the respective ascending-dose groups, with only 26% of those on Xolair attaining the same result. None of the patients in the placebo group reached this mark, which Novartis said at the time suggested a dose-response relationship. Additionally, about 30%, 44%, and 40% of the patients treated in the ligelizumab arms had complete control of their symptoms, versus 26% of the patients on Xolair. Again, no patients in the placebo group managed this. Though Narasimhan is expecting positive news here, his forecasts remain far from a sure thing. Among the programs included in his Nov. 2020 presentation was inclisiran, the PCSK9 candidate that the FDA surprisingly handed a CRL last month due to “unresolved facility inspection-related conditions,” Novartis said. The other candidates Narasimhan featured were iptacopan, iscalimab, pelacarsen and branaplam.
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