Cingulate Inc. (NASDAQ: CING) announced that the U.S. Food and Drug Administration (FDA) has provided clearance for the company to file a New Drug Application (NDA) for CTx-1301, its lead Phase 3 candidate for the treatment of Attention Deficit Hyperactivity Disorder (ADHD) in patients six years and older.
According to the FDA's communication, Cingulate can proceed with the NDA submission via the 505(b)(2) pathway without continuing its fixed dose study and onset and duration study. The data collected to date from these Phase 3 studies will be included in the filing, with only a Phase 1 fed/fast study required to be conducted concurrently with NDA preparation. This study is expected to last approximately two to three weeks, with results included in the submission.
"We are pleased with the FDA's guidance and perspective regarding our pathway to a timely NDA filing, and we will proceed to complete all remaining requirements accordingly," said Shane J. Schaffer, Chairman and CEO of Cingulate. "We also believe this communication provides additional go-to-market momentum as we continue to meet with potential partners for the outlicensing of CTx-1301 in the United States and abroad."
The company has begun NDA preparation and expects to submit its filing in the first half of 2025.
Innovative Technology Addressing Unmet Needs in ADHD Treatment
CTx-1301 utilizes Cingulate's proprietary Precision Timed Release™ (PTR™) drug delivery platform to create a multi-core formulation of dexmethylphenidate, an FDA-approved compound for ADHD treatment. The technology is designed to precisely deliver three releases of medication at predefined times, ratios, and styles of release to optimize patient care in a single tablet.
The formulation incorporates a proprietary Erosion Barrier Layer (EBL) that provides control of drug release at precise, pre-defined times with no release prior to the intended time. This tablet-in-tablet dose form is designed to erode at a controlled rate until the drug is released from the core tablet.
Dr. Ann Childress, President of the Center for Psychiatry and Behavior Medicine, Inc., and lead investigator of Cingulate's CTx-1301 onset and duration study, highlighted the potential impact of the treatment: "The treatment effect size of CTx-1301 was remarkable given the current effect sizes observed in the marketplace. Many patients require longer duration of efficacy and must add a second, or booster, dose to ensure treatment effect over their entire active day."
She added, "If approved, I believe CTx-1301 could potentially benefit the more than 60 percent of patients currently using immediate-release booster doses to extend the efficacy of their medications."
Addressing a Significant Market Need
ADHD affects approximately 6.4 million children and adolescents (11 percent) under the age of 18 in the United States. Among this group, approximately 80 percent receive treatment, with 65-90 percent demonstrating clinical ADHD symptoms that persist into adulthood.
Adult ADHD prevalence is estimated at approximately 11 million patients (4.4 percent), almost double the size of the child and adolescent segment combined. However, only an estimated 20 percent of adults with ADHD receive treatment.
While stimulants are considered the gold standard for ADHD treatment due to their efficacy and safety, a significant challenge has been providing patients with medication that maintains effectiveness throughout the entire active day. CTx-1301 aims to address this gap by providing 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.
Commercial Strategy
In March 2023, Cingulate announced a joint commercialization agreement with Indegene, a digital-first life sciences commercialization company, to provide commercial support for CTx-1301 upon FDA approval. The agreement spans cross-functional services through an omnichannel marketing approach designed to manage pre-commercial support during clinical trials and effectively commercialize the product nationwide following potential approval.
The partnership covers most facets of the commercialization process, including sales, pricing, reimbursement and market access, medical affairs, medical communications, pharmacovigilance, regulatory, marketing, and commercial operations support.
"Now that we have a clear path to submission, we look forward to engaging with key payer stakeholders to achieve maximum access and market penetration to benefit ADHD patients and their families," Schaffer stated.
Clinical Evidence
Earlier this year, Cingulate presented results from the Phase 3 adult efficacy and safety study of CTx-1301 at the 2024 American Professional Society of ADHD and Related Disorders (APSARD) conference. The results were previously presented at the 2023 Psych Congress, where a poster describing the data was selected as a finalist for the Psych Congress's First Annual Poster Awards.
The poster provided additional insight regarding CTx-1301's effect size, which conveys clinical significance rather than statistical significance and allows for comparison across trials. Effect size is not reliant on sample size and is considered an important metric for evaluating treatment efficacy.
With the FDA's recent guidance, Cingulate is positioned to advance CTx-1301 toward potential approval as the first medication to provide patients with entire active-day efficacy from a single oral dose, potentially transforming the treatment landscape for ADHD patients who currently rely on multiple daily doses to manage their symptoms.
