MeiraGTx Announces It Has Been Granted FDA Regenerative Medicine Advanced Therapy Designation For AAV-GAD For Treatment Of Parkinson's Disease

-  This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD as a one-time treatment for Parkinson's disease

-  RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review

 MeiraGTx Holdings plc (NASDAQ:MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for the treatment of Parkinson's disease not adequately controlled with anti-Parkinsonian medications.

This RMAT was awarded following the presentation to the FDA of positive data from 3 clinical studies demonstrating the benefit of AAV-GAD when administered in a one-time stereotactic infusion to the subthalamic nucleus in the brain. A Phase 1 dose escalating clinical study (n=12) was conducted, followed by a double-blind, sham-controlled Phase 2 study (n=45) and a second randomized, double-blind, sham-controlled dose ranging clinical bridging study (n=14).

"We are very pleased to have been awarded this RMAT designation for our AAV-GAD product in Parkinson's disease," said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. "The data from our double-blind, sham-controlled Phase 2 studies show significant clinically meaningful benefit on the standard motor endpoint in Parkinson's disease, UPDRS Part 3, as well as other validated measures of Parkinson's symptoms. What is most exciting is the analysis of the data from these Phase 2 studies using the AI technology of our partner Hologen. Through the use of Hologen's technology applied to the data from our double-blind, sham-controlled studies, we have demonstrated disease modifying changes in the circuitry of the brain of patients treated with AAV-GAD as well as potentially protective changes in the substantia nigra and regions of the brain involved in cognition and mood. This is the first time sham-controlled gene or cell therapy Phase 2 studies have shown significant benefit in UPDRS and now, to our knowledge, the only demonstration of disease modification in a sham or placebo-controlled study in Parkinson's disease."

Dr. Forbes continued, "We are excited to have been granted RMAT designation for our AAV-GAD program in Parkinson's disease and we look forward to working closely with the FDA to bring this potential life changing therapy to this large population of Parkinson's patients in need of effective and disease modifying treatments for this serious neurodegenerative disorder."

The requirements for receiving an RMAT designation include that the drug candidate is an advanced regenerative medicine, in this case a gene therapy; that the therapy is targeting a serious condition, in this case, Parkinson's disease; and that the applicant has presented clinical evidence demonstrating that the drug candidate has the potential to address an unmet need in the serious condition. The RMAT requirement for clinical data supporting a benefit in an unmet need is a high hurdle, with less than half of all RMAT designation applications granted.

The RMAT designation is aimed to expedite the development and review of promising Regenerative Medicine therapeutic candidates, including human gene therapies, that treat, modify, reverse or cure serious or life-threatening diseases. Similar to Breakthrough Therapy designation, RMAT designation allows for increased interaction with the FDA and immediate multidisciplinary comprehensive discussions of the ongoing product development program, clinical trials and plans for expediting the manufacturing development strategy. RMAT designation includes the benefits of Fast Track and Breakthrough Therapy designations with rolling review and potential Priority Review of a product's biologics license application (BLA).