Positive Phase 3 Results: CTx-1301 for the Treatment of Pediatric ADHD

Cingulate Inc today announced the release of positive phase 3 results from its pivotal trial of CTx-1301 (dexmethylphenidate HCl) in pediatric attention-deficit/hyperactivity disorder (ADHD), recently presented by Ann Childress, MD, at the American Academy of Child and Adolescent Psychiatry Annual Meeting in Chicago. CTx-1301 met its primary endpoint, demonstrating dose-dependent improvements on the ADHD ratings scale 5, and Clinical Global Impression-Severity scales, and demonstrated the ability to deliver symptom relief with the convenience of once-daily dosing.¹

CTx-1301 is a once-daily, multi-core tablet that utilizes Cingulate’s proprietary Precision Timed Release platform to deliver 3 precisely timed releases of active medication across the day, a design that aims to provide rapid onset of effect and entire active-day duration.

In the randomized, double-blind, pediatric and adolescent, placebo-controlled study, CTx-1301 delivered rapid onset of effect and sustained efficacy through the evening hours. Safety and tolerability were consistent with the stimulant class, with no unexpected adverse events reported.

“In summary, CTx1301 demonstrated dose dependent efficacy in improving ADHD symptoms in children and adolescents. The 37.5 mg dose demonstrated the largest effect size in symptom reduction and effect sizes were pretty substantial, considering this was a forced dose and not a dose optimization study,” Childress shared during her presentation. Childress is the lead investigator of Cingulate’s CTx-1301 onset and duration study and president of the Center for Psychiatry and Behavior Medicine Inc. “The safety profile did not show anything that would be of concern, as it looks like other methylphenidates.”

The US Food and Drug Administration (FDA) recently accepted for review the New Drug Application (NDA) for CTx-1301, Cingulate’s lead candidate for the treatment of ADHD in children and adults.² The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of May 31, 2026. This NDA acceptance marks a major regulatory achievement for Cingulate and the CTx-1301 program. CTx-1301 is currently being reviewed via the FDA 505(b)(2) regulatory pathway, a path that allows sponsors to reference existing data on previously approved active ingredients while demonstrating novel clinical benefit through a differentiated delivery mechanism. Additionally, Cingulate received a fiscal year 2025 PDUFA fee waiver from the FDA—granted through the small business waiver provision of the Federal Food, Drug, and Cosmetic Act—for this NDA, saving the company approximately $4.3 million.³

“If approved, I believe CTx-1301 could potentially benefit the more than 60% of patients currently using immediate-release booster doses to extend the efficacy of their medications,” said Childress.⁴

“These results demonstrate that CTx-1301 can deliver reliable symptom control for the entire active-day in a single dose—something families and clinicians have wanted for years. With this phase 3 data in hand, we believe CTx-1301 is well positioned to become a next-generation treatment for ADHD as we move toward potential approval and launch in 2026,” said Raul Silva, MD, the chief science officer of Cingulate.

References

1. Cingulate presents positive phase 3 results for CTx-1301 demonstrating statistically significant efficacy and entire active-day symptom control. News release. October 28, 2025. Accessed October 28, 2025. https://www.globenewswire.com/news-release/2025/10/28/3175360/0/en/Cingulate-Presents-Positive-Phase-3-Results-for-CTx-1301-Demonstrating-Statistically-Significant-Efficacy-and-Entire-Active-Day-Symptom-Control.html

2. Kuntz L. FDA accepts NDA for CTx-1301 for treatment of ADHD. Psychiatric Times. October 14, 2025. https://www.psychiatrictimes.com/view/fda-accepts-nda-for-ctx-1301-for-treatment-of-adhd

3. Kuntz L. Cingulate receives $4.3 million PDUFA fee waiver for ADHD treatment, CTx-1301. Psychiatric Times. July 29, 2025. https://www.psychiatrictimes.com/view/cingulate-receives-4-3-million-pdufa-fee-waiver-for-adhd-treatment-ctx-1301

4. Childress A, Brams M, Koehn K, et al. A phase 3, dose-optimized, double-blind, randomized, placebo controlled, single-center, parallel efficacy and safety laboratory classroom study in adults with attention-deficit/hyperactivity disorder (ADHD) using CTx-1301 (dexmethylphenidate): effect size assessment. American Professional Society of ADHD and Related Disorders (APSARD) Annual Meeting. Poster presentation. Accessed October 14, 2025. https://www.cingulate.com/static-files/b3c901ad-8d47-4ebc-b0a8-41c7330bc1b6