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Clinical Trial Updates
We've been keeping EyesOn the many clincial updates that are being discussed lately. The J&J and Amgen data caught our interest this week.
With blockbuster sales of Caplyta, Johnson & Johnson’s $14.6 billion investment in Intra-Cellular Therapies continues to pay off. The atypical antipsychotic has passed a phase 3 test, moving it one step closer to a broader psychiatric designation.
In a late-stage trial in adults with manic episodes linked to bipolar I disorder, Caplyta (lumateperone), which was first approved in 2019 under Intra-Cellular’s stewardship for schizophrenia, helped deliver rapid improvement in manic symptoms and improved overall illness severity, among other benefits versus placebo, over three weeks, according to J&J.
At the recent 2026 Psych Congress annual meeting in New Orleans, the data, along with a number of other J&J readouts, demonstrated improvements in bipolar mania on Caplyta beginning as early as day three of treatment. The trial (code 451) is one of two examining Caplyta in people with manic episodes linked to bipolar disease.
The mood stabiliser lithium, antiseizure drugs like carbamazepine and valproate that serve a similar purpose, and other atypical antipsychotics are among the many decades-old treatments available to treat such episodes.
However, J&J feels that a solution like Caplyta is justified due to tolerability concerns and the different reactions patients have to current manic episode medications.
According to the firm, mania is one of the most dangerous and concerning stages of bipolar disease, and effective treatment requires more than just temporary or partial symptom alleviation. Referring back to the three-day start of benefit observed in Study 451, J&J states that controlling those occurrences as soon as feasible is also crucial.
Adults with bipolar I-associated manic episodes, either with or without mixed features - a combination of manic and depressive symptoms - were specifically evaluated for Caplyta at 42 mg.
Caplyta helped the study reach its main goal by showing a 4.8-point larger reduction on a measure of manic symptoms after three weeks compared to placebo. J&J noted a significant improvement as early as day 3 and maintained it throughout the entire 21-day trial period.
Another clinical indicator, one of the trial’s primary secondary objectives, showed that patients taking the J&J medication had considerably improved overall sickness severity at week three when compared to those receiving a placebo.
Using the same Young Mania Rating Scale that underpins the primary goal, J&J also noted that 45.8% of patients on Caplyta saw a clinical response, compared to 20.9% of patients in the control cohort.
According to J&J, Caplyta was well tolerated in the trial and did not raise any new safety concerns beyond its established profile.
The business claims that these Phase 3 results are a significant step in assessing Caplyta’s potential to treat both depressed and acute manic episodes linked to bipolar I disease, building on the medication’s proven effectiveness in treating bipolar depression.
The pharmaceutical company also stated that data analysis for the second phase 3 research of Caplyta in people with bipolar I-associated manic episodes has already been completed.
In addition to its first approval for schizophrenia, the FDA authorised Caplyta in 2021 to treat bipolar depression and, more recently, as an adjunctive treatment for major depressive disorder (MDD) in November.
Caplyta, which opened a sizable new patient front with its MDD nod last year, is expected to reach peak sales of several billions of dollars, according to J&J.
Inspired in large part by the medication’s MDD goals, J&J acquired the drug through its $14.6 billion acquisition of Intra-Cellular early last year.
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Amgen’s phase 3 victory for an investigational medication for the autoimmune disorder Sjögren’s disease is encouraging for the pipeline bought by Horizon Therapeutics, which has so far had trouble gaining traction.
651 patients with moderate-to-severe Sjögren’s disease - a condition in which the patient’s immune system targets the salivary and tear glands - were enrolled in the phase 3 OASIZ 301 research. Although dry mouth and eyes are typical signs, the illness can potentially be more serious causing severe fatigue, as well as nervous system, circulatory, and gland issues. The FDA has not approved any medications to especially treat Sjögren’s disease; instead, anti-inflammatory drugs are frequently used to manage symptoms.
Dazodalibep, a fusion protein that targets the CD40 ligand, significantly improved scores on the EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) compared to placebo, meeting the trial’s primary aim, according to a study released today by Amgen.
According to Amgen, infusion-related responses, hypertension, urinary tract infections, and mild to moderate nose irritation were the most frequent side events.
The trial’s specific findings will be presented at a future medical conference.
As efforts are made to provide a new, highly differentiated choice for individuals with this crippling autoimmune disease, the business claims that the quick and continuous improvement in systemic disease activity supports its trust in dazodalibep.
Patients with systemic illness and moderate-to-severe Sjögren’s symptoms were enrolled in OASIZ 301. Patients with comparable symptoms but mild systemic disease activity are the target of another phase 3 trial, OASIZ 303.
Additionally, patients from both OASIZ studies are eligible to participate in a long-term extension study being conducted by Amgen.
Although it has been observed that the two studies did enroll different patient populations, analysts believe that this first positive phase 3 readout with dazodalibep offers validation for the CD40L mechanism of action in Sjögren’s and may have positive read-through to the ongoing OASIZ 303 research. The analysts said that before considering clearance, the FDA will probably need two favourable phase 3 readouts.
According to the company, patients with Sjögren’s illness struggle with crippling symptoms and systemic signs for which there is currently no approved disease-modifying treatment. These topline results are a significant advancement for the field and offer more evidence in favour of dazodalibep as a new medication for enhancing systemic disease activity.
Recently, Sjögren’s has caused problems for the biopharma sector. Although Novartis is still working on an antibody for Sjögren’s disease called ianalumab, Sanofi and Novartis have discontinued such efforts in recent years. Despite having significant placebo effects, that antibody won two phase 3 trials last year.
In the meantime, Amgen dropped another Horizon-acquired medication intended to treat the illness in May, while Astellas eliminated a STING inhibitor for the illness in April after initially ending a phase 1 trial.
Adezkibart, an antibody that targets the FMS-like tyrosine kinase 3 ligand, was discontinued following an interim futility analysis of its phase 2 trial. Amgen’s $27.8 billion acquisition of Horizon in 2023, which focused on the approved drugs Uplizna, Krystexxa, and Tepezza, has left few experiential drugs.
Horizon did not develop dazodalibep; instead, it acquired it through the $3 billion purchase of Viela Bio, an Astra Zeneca spinout, in 2021.
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