Frexalimab

Novartis hailed the win as the “first ever global phase 3 trials to demonstrate statistically significant reduction in disease activity for Sjögren\'s disease.”\n Novartis may have abandoned one Sjögren\'s syndrome asset this year, but it looks like the drugmaker has a winner on its hands with another candidate.The Swiss pharma had been evaluating ianalumab, a dual-mechanism, B-cell-depleting antibody that targets the protein BAFF-R, in a pair of phase 3 studies dubbed Neptunus 1 and 2, which in total enrolled 779 patients with the systemic autoimmune disease.Both studies hit their primary endpoint of demonstrating an improvement in disease activity as measured on the EULAR Sjögren\'s syndrome disease activity index at 52 weeks compared to placebo, Novartis announced in an Aug. 11 release. The drug was also well tolerated and demonstrated a favorable safety profile, according to the pharma.The company hailed the win as the “first ever global phase 3 trials to demonstrate statistically significant reduction in disease activity for Sjögren\'s disease.” For now, Novartis is holding back the data for a future medical conference, while referencing plans to take the results to global regulators with the aim of bringing the first targeted treatment for the autoimmune disease to market.“Sjögren\'s disease is a serious, progressive, systemic autoimmune disease, often unrecognized or misdiagnosed with a significant detrimental impact to quality of life, with very limited treatment options and an established unmet need,” Novartis Chief Medical Officer Shreeram Aradhye, M.D., said in the release.“Both phase 3 trials demonstrate that ianalumab improves disease activity in patients with Sjögren\'s disease,” Aradhye added. “These phase 3 studies mark a significant milestone. We look forward to engaging with health authorities to discuss these findings in the near future.”It’s a big win for Novartis, which started the year dropping its anti-CD40 antibody iscalimab after becoming doubtful of its competitive profile. That left ianalumab as Novartis’ last and best hope for the disease.Ianalumab had already shown \"meaningful differences in patients with this very difficult-to-treat disease” in phase 2, Novartis executives pointed out at the time.Iscalimab was only the latest Big Pharma prospect to fail to make the grade in Sjögren\'s. Last year, Sanofi crossed off Sjögren’s from the list of targeted indications for its CD40L monoclonal antibody frexalimab after seeing phase 2 efficacy data, while, more recently, Kiniksa Pharmaceuticals ended work on its own Sjögren\'s prospect abiprubart. While Novartis had always sounded positive about the potential of ianalumab in the automimmune disease, the therapy has faced its own clinical setbacks. Only last month, the company reported that the B-cell-depleting antibody had failed to hit the efficacy threshold in a phase 2 study for a painful skin condition called hidradenitis suppurativa.Ianalumab\'s story begins with Novartis’ collaboration with MorphoSys, before the Big Pharma went on to buy the biotech last year. The therapy is also being investigated for other B cell-driven autoimmune diseases like immune thrombocytopenia, systemic lupus erythematosus, lupus nephritis, warm autoimmune hemolytic anemia and diffuse cutaneous systemic sclerosis.

Sanofi has agreed to spend $470 million to take control of a young biotechnology company and its experimental drug for Alzheimers disease.The newly announced acquisition has Sanofi paying $8 for each share of Vigil Neuroscience it doesnt already own, reflecting a premium of nearly 250% to the price Vigil shares traded at Wednesday afternoon. The French pharmaceutical giant invested $40 million into Vigil last June, and in exchange got an exclusive right to be first in line for deal talks if Vigil chose to license out its platform of small molecule drugs that amplify a protein called TREM2.The most advanced of those drugs, code-named VG-3927, several months ago completed an early-stage study that enrolled both healthy volunteers and patients with Alzheimers disease. Vigils president and CEO, Ivana Magovevi-Liebisch, said in a statement that the acquisition should help fully unlock and accelerate the development of VG-3927.A second molecule from the platform, VGL101, is not being acquired by Sanofi.Instead, rights to that molecule will be returned to the original licensor, Amgen.Neuroscience is one of Sanofis core strategic research focuses, alongside cancer, rare diseases and the immune system. Yet, in terms of product sales, it pales in comparison to those other three areas. Sanofis portfolio lists just one neurology drug, the multiple sclerosis therapy Aubagio, which last year accounted for less than 1% of the 41 billion euros in net sales recorded by the companys biopharma unit.Sanofi is trying to grow this pool with a few experimental medicines that have progressed to the later stages of development. One, frexalimab, is what Sanofi touts as a pipeline-in-a-product asset, since the company believes it has the potential to treat a range of illnesses, from MS to lupus to diabetes. Another, riliprubart, is being evaluated against CIPD, a rare disorder that harms the peripheral nerves, leading to arm and leg weakness.A third, tolebrutinib, has been tested across different types of MS and is currently being considered for approval by Food and Drug Administration, with a decision deadline set for late September. Sanofi got ahold of tolebrutinib through its $3.7 billion purchase of Principia Biopharma in 2020.Notably, all of these medicines work by regulating the immune system, which is characteristic of Sanofis approach to neurology.The Vigil deal follows this approach, too, as TREM2 alerts the brains resident immune cells to threats like the toxic abeta plaques that are a hallmark of Alzheimers. When this protein is impaired or loses its function, it can increase the risk of nerve cell injury, inflammation and death.TREM2 represents a compelling target at the intersection of immune dysregulation and neurodegeneration, said Houman Ashrafian, Sanofis head of R&D, in that Wednesday statement.The acquisition also supports Sanofis strategy of leveraging our expertise in immunology to solve health problems with critical unmet need, Ashrafian added.The companies expect their deal to close sometime between July and the end of September, provided investors holding a majority of the outstanding shares of Vigil common stock approve it. Sanofi said Magovevi-Liebisch as well as Vigil backers Atlas Venture and Bruce Booth, a partner at the firm have signed voting and support agreements in favor of the deal.Together, they represent roughly 16.2% of Vigils total common shares outstanding.In addition to the upfront payment, Vigils shareholders will receive a so-called contingent value right that would entitle them to receive a deferred cash payment of $2 for each share they own once VG-3927 has its first commercial sale.“We believe this is a good outcome for shareholders in the current funding environment,“ wrote Myles Minter, an analyst at William Blair who covers Vigil, in a note to clients.Vigil debuted at $14 per share when it went public in January 2022, but has lost most of its market value since.In his own note, Stifel analyst Paul Matteis argued the timing of the deal is “interesting,“ given a TREM2-targeting drug from Alector and AbbVie recently failed in a mid-stage study. That setback “significantly diminished enthusiasm for the target,“ according to Matteis, so while there were reasons to think Vigil's drug could fare better, “it was going to be hard to have conviction in this until we saw [Phase 2] data.“ '