Bristol-Myers Squibb Advances Phase 3 Autism Irritability Program With KarXT Combo

Bristol-Myers Squibb Company (BMY) announced an update on their ongoing clinical study.

Study Overview

Bristol-Myers Squibb is launching a late-stage study to test a new treatment combo, KarXT plus KarX-EC, for irritability linked to autism in children and teens aged 5 to 17. Officially titled “A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Safety, and Tolerability of KarXT + KarX-EC in Children and Adolescents (5 to 17 Years of Age) With Irritability Associated With Autism Spectrum Disorder,” the trial aims to show whether this approach can safely reduce challenging behaviors in a high-need group where treatment options are limited. The study is important because a positive outcome could open a new, protected market segment within pediatric neuropsychiatry.

Intervention/Treatment

The study tests two drugs used together: KarXT and KarX-EC. Both are drug treatments designed to help manage irritability in young people with autism. The active arm gets KarXT plus KarX-EC, while the comparison group receives matching placebo pills. The goal is to see if the combo offers clear benefits over no active treatment while staying safe and well tolerated.

Study Design

This is an interventional Phase 3 trial, meaning patients are assigned to take either the active drugs or placebo. Participants are randomized, so they are placed into groups by chance, which helps keep the results unbiased. The study uses a parallel design, where each group stays on its assigned treatment for the duration of the trial. It is quadruple-blinded: patients, caregivers, study doctors, and outcome assessors do not know who is on active drug or placebo. The main goal is treatment-focused, measuring how well the regimen reduces irritability versus placebo.

Study Timeline

The study was first submitted on December 10, 2025, signaling BMY’s formal move to advance this program in autism-related irritability. As of the latest update on January 2, 2026, the status is “not yet recruiting,” so sites are being prepared but enrollment has not started. Primary completion and final completion dates have not been posted yet, but investors should expect data to land several years after first patient dosing. The timing places this program on a medium- to long-term horizon for any revenue impact.

Market Implications

For investors, this update shows Bristol-Myers Squibb pushing into a specialty CNS niche with high unmet need and limited direct competition, which can support premium pricing if successful. While near-term earnings will not move on this trial alone, the program broadens BMY’s future pipeline beyond oncology and immunology, which may help diversify revenue and support a higher pipeline-driven valuation multiple over time. Competitors in neuropsychiatry, including mid-cap players working on pediatric behavioral symptoms, may feel pressure if KarXT-based regimens prove both effective and safe, as BMY has the scale to rapidly commercialize across major markets. Short term, sentiment impact is modest but positive; longer term, confirmation of efficacy in Phase 3 could become a meaningful upside driver and support re-rating of BMY’s growth profile.

The study remains ongoing at the planning stage with status recently updated, and further details are available on the ClinicalTrials portal.

To learn more about BMY’s potential, visit the Bristol-Myers Squibb Company drug pipeline page.