FDA grants RMAT designation to EG110A for neurogenic detrusor overactivity

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to the investigational gene therapy EG110A for neurogenic detrusor overactivity (NDO) with urinary incontinence, Cyllene Therapeutics announced in a news release.¹

RMAT designation is granted to regenerative medicine therapies intended to treat, modify, reverse, or cure serious diseases or conditions and shows preliminary clinical evidence that indicates potential to address those unmet medical needs. The designation can facilitate more frequent interactions with FDA during development and may provide opportunities to discuss clinical trial design, surrogate or intermediate end points, and approaches to post-approval requirements.

EG110A is a nonreplicating herpes simplex virus type 1 (HSV-1) vector and non-systemic, site-specific inhibitor of CGRP+ neurons. The agent also received FDA fast track designation in October 2025.²

Early clinical findings

The designation follows interim findings from an ongoing first-in-human phase 1b/2a study (NCT06596291) in adults with spinal cord injury (SCI)-associated NDO. The trial is a first-in-human, open-label, dose-escalation study designed to evaluate EG110A in adults aged 18 to 75 years with NDO-related urinary incontinence following SCI who have persistent symptoms despite standard-of-care therapy and who regularly perform clean intermittent catheterization.³

The study plans to enroll 16 participants and will primarily evaluate treatment-emergent adverse events, with bladder diary and urodynamic assessments providing exploratory efficacy data. Patients in the study will receive a single treatment course of multiple intradetrusor injections of EG110A. The agent is being assessed across 3 dose levels (low, middle, and high).

Interim data from the study showed that at the lowest dose, EG110A was associated with a more than 88% reduction in the incidence of urinary incontinence episodes by week 12.² Treatment effect was established as early as week 4.

The company said interim results from the study will be presented at the International Continence Society annual meeting in Maastricht.

“The clinical data seen to date, demonstrating a marked reduction in urinary episodes, suggests treatment with EG110A could have a profound impact on the daily lives of patients,” said Cornelia Haag-Molkenteller, MD, Chief Medical Officer at Cyllene Therapeutics, in the news release.¹ “We look forward to working closely with the FDA as we move further into clinical development, including a planned phase 2b/3 study with EG110A in neurogenic detrusor overactivity in 2027.”

REFERENCES

1. Cyllene Therapeutics receives U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EG110A in neurogenic detrusor overactivity (NDO). News release. Cyllene Therapeutics. September 14, 2026. Accessed September 17, 2026. https://cyllene-tx.com/Cyllene-Therapeutics-Receives-U-S--FDA-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-for-EG110A-in-Neurogenic-Detrusor-Overactivity-NDO-.html

2. EG 427 receives U.S. FDA Fast Track Designation for EG110A DNA Medicine in neurogenic bladder patients. News release. Cyllene Therapeutics. October 6, 2025. Accessed September 17, 2026. https://cyllene-tx.com/EG-427-Receives-U-S--FDA-Fast-Track-Designation-for-EG110A-DNA-Medicine-in-Neurogenic-Bladder-Patients.html

3. Dose escalation study of EG110A, administered by intradetrusor injections to adults with neurogenic detrusor overactivity-related incontinence following spinal cord injury who regularly perform clean intermittent catheterization. ClinicalTrials.gov. Last updated September 3, 2026. Accessed September 17, 2026. https://clinicaltrials.gov/study/NCT06596291