Amgen is awaiting another Phase 3 readout for the investigational Sjögren’s disease therapy, set for later this year. William Blair anticipates that at least two trials will be needed to support an FDA application.
Amgen’s investigational fusion protein significantly reduced disease activity in a Phase 3 study in Sjögren’s disease, setting the asset up to become what analysts say could be a “multi-billion-dollar franchise.”
The pharma didn’t provide specific data from the Phase 3 OASIZ 301 study, but said in a Tuesday release that the drug candidate dazodalibep met its primary endpoint of significantly improving systemic disease activity across various organs at 48 weeks. Improvements emerged as early as week four, the company said.
OASIZ 301 enrolled approximately 621 patients with moderate-to-severe systemic disease activity. Aside from disease activity, the trial also assessed fatigue and tender and swollen joints, among other secondary endpoints.
As for safety, Amgen reported that discontinuations due to adverse events occurred at a low rate and were similar across dazodalibep and placebo groups. The most common side effects were mild colds, urinary tract infection, hypertension and infusion-related reactions.
With these data, dazodalibep “clears the first hurdle” in Sjögren’s disease, shifting the focus from Phase 3 efficacy risk “toward the magnitude & commercial relevance,” analysts at Truist Securities said in a Tuesday note. The firm also said that the anti-CD40L fusion protein has the potential to become a “multi-billion-dollar franchise,” citing meaningful market opportunity and competition concentrated in systemic disease.
The firm nevertheless cautioned that Tuesday’s readout was limited “with no effect size, dose breakdown or secondary endpoints,” and the full Phase 3 data is needed “to establish the quality of the win.” Amgen plans to present detailed findings from OASIZ 301 at an upcoming medical congress.
Sjögren’s disease is an autoimmune condition characterized by dryness, fatigue and pain, as well as several systemic and potentially progressive symptoms. There are currently no approved therapies designed specifically to treat Sjögren’s. Dazodalibep—which Amgen obtained from its $27.8 billion takeover of Horizon Therapeutics largely centered on thyroid eye disease medicine Tepezza—targets the CD40 ligand, a protein crucial for activating the immune system. Horizon picked up dazodalibep in 2021 from its $3 billion acquisition of AstraZeneca spinout Viela Bio.
Amgen is also studying dazodalibep in the Phase 3 OASIZ 303 study, which is focused on patients with Sjögren’s with moderate-to-severe symptom burden but low systemic disease activity. Data from this trial are expected later this year. OASIZ 301 “could have positive read-through to the ongoing OASIZ 303 study, although we note that the two studies did enroll distinct patient populations,” William Blair told investors on Tuesday.
“We continue to have a base case that two positive Phase III studies will be required to support FDA approval,” the firm added, expecting that the pharma is likely to reveal regulatory plans after OASIZ 303 reads out.
