An Amgen drug improved signs and symptoms of Sjögren’s disease in a pivotal clinical trial, building the case the case for the biologic medicine as a potential treatment for this common autoimmune disorder that currently has no FDA-approved therapies.
The preliminary Phase 3 results for the drug, dazodalibep, were statistically significant and clinically meaningful, Amgen said Tuesday. The company is holding back more specific details about those results, reserving them for presentation at a future medical meeting. Those details could help show how dazodalibep matches up against a Novartis therapy currently under FDA review in Sjögren’s with a regulatory decision expected in coming months.
While Sjögren’s affects the entire body, its effects are most noticeable in the eyes and mouth. The disease’s attack on tear ducts and salivary glands leaves patients with dry eyes and dry mouth. Up to 4 million Americans have Sjögren’s, making it the second most common rheumatic autoimmune condition in the country, according to the Sjögren’s Foundation.
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Amgen’s approach to Sjögren’s is to target and block CD40 ligand, or CD40L, a protein that plays a role in the immune system. Dazodalibep is a fusion protein that disrupts the interaction of CD40L with immune cells. The results reported Tuesday are from a placebo-controlled Phase 3 test that enrolled patients with Sjögren’s classified as moderate to severe according to a rating scale used to assess disease activity across organ systems. The main trial goal was assessing the change in score on this scale at week 48. Key secondary goals included evaluating dryness, joints, and fatigue.
While specific figures from the trial are still under wraps, Amgen did say improvement was observed as early as week 4 and sustained through week 48. The most common adverse events reported from the study include nasopharyngitis, urinary tract infection, hypertension, and infusion-related reactions. These problems were characterized as generally mild to moderate. In Amgen’s announcement, lead investigator Dr. Ghaith Noaiseh, who is associate professor of medicine, division of allergy, clinical immunology and rheumatology at the University of Kansas Medical Center, said the preliminary results “provide further support for dazodalibep as an emerging treatment for improving systemic disease activity and represent an important advance for the field.”
Meanwhile, Novartis’s approach to Sjögren’s addresses B cells, a type of immune cell associated with many immunological disorders. The company’s antibody drug ianalumab offers a dual mechanism of depleting B cells and blocking their activation by binding to the BAFF receptor. During the American College of Rheumatology Convergence congress last fall, results from two Phase 3 studies were presented showing ianalumab led to statistically significant and clinically meaningful improvement measured at 48 weeks.
Amgen is conducting a second Phase 3 test evaluating dazodalibep in patients with high symptom burden and low systemic Sjögren’s disease activity. The pharma company said this study could be completed in the fourth quarter of this year.
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In a note sent to investors, William Blair analyst Matt Phipps said he expects two Phase 3 studies will be required to support FDA approval of the drug in Sjögren’s. While the preliminary results for dazodalibep are encouraging, Phipps said this drug is relatively high risk. That’s because Sjögren’s has historically proved challenging for drug development due to high placebo response rates and the many differences in disease presentation and severity among patients who have this chronic condition. That said, the first Phase 3 study could have positive readthrough to the ongoing Phase 3 test, though Phipps noted that each study enrolled distinct patient populations.
“More broadly for [Sjögren’s] drug development, the positive readout also potentially adds support for Amgen’s novel patient segmentation strategy into separate systemic disease and symptomatic disease buckets,” Phipps said.
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