FDA Feedback Paves Way for New IND Filing for PT00114 in Generalized Anxiety Disorder

Protagenic Therapeutics today announced that it has received written responses from the US Food and Drug Administration (FDA) to its Type B pre-Investigational New Drug (pre-IND) meeting request for PT00114 for the treatment of generalized anxiety disorder (GAD).¹

PT00114 is a first-in-class investigational neuropeptide targeting the teneurin C-terminal associated peptide pathway for the treatment of stress-related neuropsychiatric disorders. PT00114 is thought to directly modulate the brain's maladaptive responses to stress, offering a differentiated approach to treating these disorders.²

FDA Offers No Objections

In the written responses, the FDA identified no clinical hold issues and did not object to the overall design of the planned 3-part phase 1 program, including the dose-escalation approach and safety review governance. Additionally, the FDA did not identify any deficiencies that would preclude submission of an IND. The FDA also indicated that Protagenic's proposed potency and comparability strategy, along with its chemistry, manufacturing, and controls and nonclinical packages, appear to support an IND submission.

"[The] FDA's feedback gives us a clear path to submitting a new IND for PT00114 in generalized anxiety disorder, and it reflects the clinical and manufacturing groundwork our team has completed to date," said Bill Nichols Jr., the president of Protagenic Therapeutics.

Previous Studies Outside the US

This new IND builds directly on PT00114's completed phase 1 single ascending dose and multiple ascending dose studies, which were conducted outside the United States. In those studies, investigators reported that PT00114 was well tolerated across all dose levels studied and no serious adverse events were reported. These studies were conducted in a limited number of healthy volunteers outside the United States and were not conducted under an IND. The FDA's guidance will allow Protagenic Therapeutics to carry this clinical experience forward into a US clinical development program in GAD, incorporating refined CMC specifications and expanded dose cohorts into the upcoming submission.

"In preclinical studies, PT00114 has been observed to act directly on the brain's stress response system rather than simply blunting symptoms, and we believe that mechanism, if confirmed in clinical trials, could represent a meaningfully different approach for a patient population this large and this underserved. Submitting this IND is our next milestone, and we look forward to continuing to work with [the] FDA,” said Nichols.

Next Steps

Following this feedback, Protagenic plans to submit an IND before the end of 2026. The first patient is expected to be dosed in the first half of 2027, pending FDA review and clearance.

References

1. Protagenic Therapeutics receives FDA pre-IND feedback supporting new IND for PT00114 in generalized anxiety disorder. News release. August 20, 2026. Accessed August 20, 2026. https://www.morningstar.com/news/accesswire/1209533msn/protagenic-therapeutics-receives-fda-pre-ind-feedback-supporting-new-ind-for-pt00114-in-generalized-anxiety-disorder

2. Protagenic Therapeutics advances PT00114 into multiple-dose phase 1 trial; positioning for phase 2 in stress & mood disorders. News release. August 22, 2025. Accessed August 20, 2026. https://www.biospace.com/press-releases/protagenic-therapeutics-advances-pt00114-into-multiple-dose-phase-1-trial-positioning-for-phase-2-in-stress-mood-disorders