Ianalumab

Novartis has shared positive top-line results from two late-stage trials of its investigational monoclonal antibody ianalumab in Sjögren’s syndrome. The phase 3 NEPTUNUS-1 and NEPTUNUS-2 trials have been evaluating the safety and efficacy of the drug in a total of 779 patients with the systemic autoimmune disease. Approximately 0.6% of the UK’s adult population is affected by Sjögren’s syndrome, a B cell-driven disease that causes inflammation and tissue damage. The condition primarily affects the exocrine glands, with more than 90% of patients experiencing dry eyes and mouth. Symptoms also include aching muscles and joints, fatigue and widespread pain. Both NEPTUNUS met the primary endpoint of improving disease activity, as assessed by a multi-dimensional disease activity measurement, compared to placebo. The candidate was also found to be well tolerated and demonstrated a favourable safety profile, Novartis said, adding that data from the trials will be presented at an upcoming medical congress. Ianalumab has a dual mechanism of action, B-cell depletion and BAFF-R inhibition, and is being investigated across various B cell-driven autoimmune diseases, including immune thrombocytopenia and systemic lupus erythematosus. The drug has the potential to become the first targeted treatment for patients with Sjögren’s syndrome, according to Novartis. Shreeram Aradhye, president of development and chief medical officer at Novartis, said: “Sjögren’s disease is a serious, progressive, systemic autoimmune disease, often unrecognised or misdiagnosed with a significant detrimental impact to quality of life, with very limited treatment options and an established unmet need. “Both phase 3 trials demonstrate that ianalumab improves disease activity in patients with Sjogren’s disease.” Aradhye added that the company is “[looking] forward to engaging with health authorities to discuss these findings in the near future”. Inflammatory diseases are a key focus area for Novartis, which recently entered into a partnership worth $1bn with Matchpoint Therapeutics to develop oral inhibitors for inflammatory diseases that have been historically difficult to treat. The company also entered into an agreement with Kyorin Pharmaceutical in March to gain exclusive worldwide rights to a pre-clinical candidate for chronic spontaneous urticaria and other allergic and inflammatory diseases involving mast cells.

Welcome back to Endpoints Weekly! We’ve got a lot to recap this week, so let’s get right to it. Bayer reached a deal worth up to $1.3 billion for the rights to a KRAS G12D inhibitor from Kumquat Biosciences. Meanwhile, Novartis read out positive results from multiple late-stage trials, and Abata Therapeutics became the latest biotech to shutter because of funding challenges. Endpoints’ Kyle LaHucik has the details on Abata, and a separate feature story on a financing tool that biotechs are increasingly using to stay afloat. Finally, Zachary Brennan covered Vinay Prasad’s return to the FDA. Wishing you a great weekend. We’ll be back in your inbox on Monday morning! — Nicole DeFeudis 💰Bayer struck a deal worth up to $1.3 billion to secure the global rights to the early-stage oncology drug from Kumquat Biosciences. The unnamed KRAS G12D inhibitor has yet to enter the clinic, but Kumquat has permission from regulators to start a Phase 1a study, Endpoints’ Elizabeth Cairns reported this week . The companies are keeping the financial terms of the deal under wraps, including the amount of Bayer’s upfront payment. Kumquat will take the drug through Phase 1a, then Bayer will take over. The companies said KRAS G12D mutations are found in 37% of pancreatic ductal adenocarcinoma (PDAC) cases, 13% of colorectal tumors, and 4% of non-small cell lung cancers. This isn’t Kumquat’s first big pharma deal. The San Diego biotech inked a KRAS inhibitor discovery deal with Eli Lilly in 2021, giving Lilly the rights to develop the resulting compounds. The company also licensed a candidate to Takeda last year . Abata Therapeutics closed due to funding challenges, Kyle LaHucik reported on Tuesday. The Watertown, MA-based biotech unveiled in June 2021 with a $95 million Series A round to develop a suite of engineered, autologous Treg cell therapies. A spokesperson for Third Rock Ventures, which helped seed Abata, said the biotech wound down “given the current fundraising environment.” Other biotechs have cited similar challenges as part of their decisions to shutter this year, including Lyndra , Kojin and NextRNA. Startups and big pharma have shown interest in Treg therapies. GentiBio closed a $157 million Series A in 2021 and announced a partnership with Bristol Myers the following year. The biotech got an IND cleared for its type 1 diabetes candidate last month. Another startup, TRex Bio, dosed the first patient in June in a Phase 1 study for its experimental atopic dermatitis treatment. And AstraZeneca also opted into a Treg cell therapy program from partner Quell Therapeutics that same month. 📢Novartis announced positive late-stage results this week for its monoclonal antibody ianalumab in a chronic autoimmune condition and a rare blood disease. On Monday, the pharma company said ianalumab cleared a pair of registrational studies in Sjögren’s disease , a disorder that can cause dry eyes, dry mouth, aching muscles and tiredness. The drug met the primary endpoint of improvement in disease activity at 48 weeks in two placebo-controlled Phase 3 trials. The following day, Novartis unveiled Phase 3 results for ianalumab in primary immune thrombocytopenia (ITP), a blood disorder that can cause bleeding, bruising and chronic fatigue. When combined with Novartis’ thrombopoietin receptor agonist Promacta, ianalumab “significantly prolonged” time to treatment failure versus placebo, the company said. The study tested ianalumab in the second-line setting after patients received corticosteroids. Ayisha Sharma has the details here. Novartis said it plans to share the Sjögren’s data with regulators. In ITP, ianalumab is in a separate ongoing Phase 3 in first-line patients, and the company said data from the two late-stage studies will be filed with regulators in 2027. 🤝Royalty deals have been the go-to financing tool this summer, Kyle LaHucik reported this week. The deals have become an increasingly appealing choice for drugmakers looking to buoy their business in exchange for payouts on future drug sales. Raymond James tracked two royalty deals in 2018. In 2022 there were 20, in 2024 there were two dozen, and in the first half of the year there were 10. “The model has finally shifted where it’s become a very attractive and desirable opportunity for companies as a way to raise capital,” XOMA Royalty chief investment officer Brad Sitko said. Read more about the model here. Prasad is back less than two weeks after his departure, a spokesperson for the agency told Endpoints News last weekend. The spokesperson said Prasad came back at FDA’s request. FDA Commissioner Marty Makary said Prasad wasn’t pushed out of his role, but rather chose to resign. His return was met with confusion and surprise from some agency staffers, Endpoints’ Zachary Brennan reported this week.