Efficacy Results Announced from Cingulate’s Phase 3 Pediatric Study of CTx-1301 (dexmethylphenidate) for ADHD
- Statistically significant improvements in ADHD symptoms across all tested doses
- Strong effect sizes ranging from 0.737 to 1.185 achieved within 5 weeks
- Positive food effect study results showing flexibility in administration
- Consistent safety profile demonstrated across eight clinical trials
- Plans to launch with eight dosage strengths, providing multiple treatment options
- FDA approval still pending and not guaranteed
- Commercial success yet to be demonstrated
- Potential competition in the ADHD treatment market
Insights
Cingulate's CTx-1301 demonstrated statistically significant ADHD symptom improvements with impressive effect sizes in pediatric Phase 3 trial, advancing toward summer NDA submission.
Cingulate has reported compelling Phase 3 efficacy data for CTx-1301 (dexmethylphenidate HCl) in pediatric ADHD patients that significantly strengthens its regulatory position. The trial design was robust—randomized, double-blind, and placebo-controlled—using the industry-standard ADHD-RS-5 rating scale as the primary endpoint. The most striking finding is the effect size range of 0.737 to 1.185 across all fixed doses (18.75mg, 25mg, and 37.5mg). In clinical psychiatry, effect sizes above 0.8 are considered large, making these results particularly impressive for a CNS therapeutic.
What sets CTx-1301 apart is Cingulate's Precision Timed Release™ platform technology, which appears to optimize the delivery profile of dexmethylphenidate—a well-established ADHD medication. The statistically significant efficacy across all tested doses suggests a reliable dose-response relationship, critical for regulatory approval. The comprehensive clinical program includes eight clinical trials in total, with recently reported positive food effect study results (meaning patients can take it with or without food) and a consistent safety profile.
The planned NDA submission this summer represents the culmination of a complete data package including both adult and pediatric populations. With eight planned dosage strengths (six for patients under 18), Cingulate is positioning CTx-1301 to address various patient needs within the substantial ADHD market. The company appears to have systematically addressed the key regulatory requirements for approval, though the FDA review process will ultimately determine if the efficacy and safety profile warrant approval for this next-generation formulation in the competitive ADHD treatment landscape.
Subjects Demonstrated Marked Improvement of ADHD Symptoms at Week 5
New Drug Application on Schedule for this Summer
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KANSAS CITY, Kan., May 20, 2025 (GLOBE NEWSWIRE) -- Cingulate Inc. (NASDAQ: CING), a biopharmaceutical company utilizing its proprietary Precision Timed Release™ (PTR™) drug delivery platform technology to build and advance a pipeline of next-generation pharmaceutical products, announced the efficacy results from an FDA required Phase 3, randomized, fixed dose, double blind, placebo controlled clinical study in pediatrics (aged 6-17), for its lead asset CTx-1301 (dexmethylphenidate HCl) for the treatment of Attention Deficit/Hyperactivity Disorder (ADHD).
The primary efficacy endpoint of the study was change in total score on the ADHD-RS-5 rating scale (a scale which measures aspects of ADHD in pediatrics). The fixed doses used in the study were 18.75-mg; 25-mg and 37.5-mg. Pending approval by the FDA, Cingulate expects to launch CTx-1301 with a total of eight dosage strengths, six of which are expected to treat patients under the age of 18.
"In a randomized, double-blind, placebo-controlled Phase 3 trial in pediatric ADHD patients, CTx-1301 demonstrated statistically significant improvements in ADHD symptoms across all fixed doses, achieving robust effect sizes (0.737 to 1.185) within 5 weeks,” said Cingulate Chief Science Officer Raul R. Silva, MD. “These results illustrate that CTx-1301, if approved by the FDA, is positioned to become a potential first-line treatment for providers to overcome the longstanding unmet needs facing the millions of ADHD patients worldwide.”
Cingulate Chairman and CEO Shane J. Schaffer stated, “These compelling results once again validate the therapeutic benefits and commercial potential of CTx-1301. Achieving statistically significant outcomes and remarkable effect sizes across all tested dose levels illustrates the potential benefits of our product and the value of our Precision Timed Release ™ Platform. These findings represent a significant value inflection point for Cingulate as we advance toward regulatory submission and commercialization.”
Last month Cingulate announced positive top-line results of a high dose (50-mg) food effect study for CTx-1301, with the primary endpoints establishing that it can be taken with or without food.
In March, Cingulate released Phase 3 safety data for CTx-1301, demonstrating that the safety profile of CTx-1301 has remained consistent over the course of eight clinical trials. A final analysis that combines both adult and pediatric safety and efficacy data will be prepared and included in the NDA submission planned for this summer.
About the Phase 3 Children/Adolescent Fixed Dose Study (CTx-1301-005)
- Efficacy results from an FDA required Phase 3, randomized, fixed dose, double blind, placebo controlled clinical study in pediatrics (aged 6-17).
- The primary efficacy endpoint in this study was change of the ADHD-RS-5 total score from Study Day 0 to Week 5. The ADHD-RS-5 is a scale which measures various aspects of ADHD in pediatrics.
- The fixed doses used in the study were 18.75-mg; 25.0-mg and 37.5-mg.
- The protocol originally required enrollment of 385 subjects, however with FDA guidance the study was terminated early resulting in the intent to treat the (ITT) population of 103 subjects in the completed study.
- Within 5 weeks of subjects receiving once daily dosing of CTx-1301 the p-values for ADHD-RS-5 were 0.018, 0.011 and 0.001 for the 18.75-mg, 25-mg, and 37.5-mg doses, respectively.
- This level of significance was achieved with less than one third of the planned subjects.
- In this study, the pre-defined statistical parameter that determined success using the ADHD-RS-5 score at Week 5 compared to Study Day 0 was a p-value of less than 0.017.
- Effect size represents the magnitude of a change in an outcome, or the strength of a relationship which was calculated for the ADHD-RS-5 primary endpoint versus placebo for each dose at week 5. The mean effect size for long-acting stimulants was 0.73. (J of Clin Psychiatry; 71:6, June 2010).
- In this study the statistically significant mean effect size compared to placebo at week 5 was: 0.737 at 18.75-mg; 0.782 at 25-mg; 1.185 at 37.5-mg; an overall effect size of 0.901.
- The Clinical Global Impression scale (CGI-I) is an objective measure of a patient’s change (improvement or worsening) in ADHD symptoms compared to baseline and was monitored during the study. The results of the CGI-I demonstrated that at all tested doses subjects demonstrated an improvement versus the placebo group at week 5.
About Attention Deficit/Hyperactivity Disorder (ADHD)
ADHD is a chronic neurobiological and developmental disorder that affects millions of children and often continues into adulthood. The condition is marked by an ongoing pattern of inattention and/or hyperactivity-impulsivity that interferes with functioning or development. In the U.S., over 20 million patients have been diagnosed with ADHD. Among this group, 12 million are adults and over 8 million are under the age of 17. According to the CDC, just 53.6 percent of all children and teens with ADHD reported they were actively treating their symptoms with medication in 2022, with 65–90 percent demonstrating clinical ADHD symptoms that persist into adulthood. Current market trends demonstrate that adult ADHD prevalence is larger and growing faster than the child and adolescent segments combined.
About CTx-1301
Cingulate’s lead candidate, CTx-1301, utilizes Cingulate’s proprietary PTR drug delivery platform to create a breakthrough, multi-core formulation of the active pharmaceutical ingredient dexmethylphenidate, a compound approved by the FDA for the treatment of ADHD. Dexmethylphenidate is part of the stimulant class of medicines and increases norepinephrine and dopamine activity in the brain to affect attention and behavior. While stimulants are the gold standard of ADHD treatment due to their efficacy and safety, the long-standing challenge continues to be providing patients with an entire active-day duration of action. CTx-1301 is designed to precisely deliver three releases of medication at the predefined time, ratio, and style of release to optimize patient care in one tablet. The result is a rapid onset and entire active-day efficacy, with the third dose being released around the time when other extended-release stimulant products begin to wear off.
About Precision Timed Release™ (PTR™) Platform Technology
Cingulate is developing ADHD and anxiety disorder product candidates capable of achieving true once-daily dosing using Cingulate’s innovative PTR drug delivery platform technology. It incorporates a proprietary Erosion Barrier Layer (EBL) providing control of drug release at precise, pre-defined times with no release of drug prior to the intended release. The EBL technology is enrobed around a drug-containing core to give a tablet-in-tablet dose form. It is designed to erode at a controlled rate until eventually the drug is released from the core tablet. The EBL formulation, OralogiK™, is licensed from BDD Pharma. Cingulate intends to utilize its PTR technology to expand and augment its clinical-stage pipeline by identifying and developing additional product candidates in other therapeutic areas in addition to Anxiety and ADHD where one or more active pharmaceutical ingredients need to be delivered several times a day at specific, predefined time intervals and released in a manner that would offer significant improvement over existing therapies. To see Cingulate’s PTR Platform, click here.
About Cingulate Inc.
Cingulate Inc. (NASDAQ: CING), is a biopharmaceutical company utilizing its proprietary PTR drug delivery platform technology to build and advance a pipeline of next-generation pharmaceutical products, designed to improve the lives of patients suffering from frequently diagnosed conditions characterized by burdensome daily dosing regimens and suboptimal treatment outcomes. With an initial focus on the treatment of ADHD, Cingulate is identifying and evaluating additional therapeutic areas where PTR technology may be employed to develop future product candidates, including to treat anxiety disorders. Cingulate is headquartered in Kansas City. For more information, visit Cingulate.com.
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Investor & Public Relations:
Thomas Dalton
Vice President, Investor & Public Relations, Cingulate
tdalton@cingulate.com
(913) 942-2301
Matt Kreps
Darrow Associates
mkreps@darrowir.com
(214) 597-8200
FAQ
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