Mahzi Therapeutics' MZ-1866 Gene Therapy Receives FDA Rare Pediatric Disease Designation for Pitt Hopkins Syndrome
核心洞察
The FDA granted Rare Pediatric Disease Designation to MZ-1866 (搜索), Mahzi Therapeutics (搜索)' investigational AAV9-TCF4 (搜索) gene replacement therapy for Pitt Hopkins syndrome (搜索).
The designation makes MZ-1866 (搜索) potentially eligible for a Priority Review Voucher upon approval, with recent PRV sales ranging from $150–$205 million.
The Phase 1/2 UNITE study has enrolled 7 of 12 planned participants, surpassing 50% enrollment ahead of schedule, with completion projected by year-end.
Mahzi Therapeutics (搜索) Inc., a clinical-stage biotechnology company developing precision therapies for neurogenetic disorders, announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to MZ-1866 (搜索), its investigational gene therapy for the treatment of Pitt Hopkins syndrome (搜索). The designation was granted by the FDA's Office of Orphan Products Development and Office of Pediatric Therapeutics.
The FDA grants RPDD to therapies intended to treat serious or life-threatening diseases that primarily affect children from birth to 18 years of age. Upon approval of a qualifying marketing application, drugs with RPDD may be eligible for a Priority Review Voucher (PRV), which can be used to obtain priority review of a subsequent marketing application or sold to another sponsor. Recently disclosed PRV sales have ranged from $150–$205 million.
"Receiving Rare Pediatric Disease Designation is a major milestone for the MZ-1866 (搜索) program and an important recognition of the critical unmet need for Pitt Hopkins patients," said Yael Weiss, M.D., Ph.D., Chief Executive Officer of Mahzi. "We are grateful for the continuing support of the Pitt Hopkins community and the Pitt Hopkins Research Foundation (搜索) in advancing the MZ-1866 program and the Phase 1/2 UNITE study."
Unmet Need and Therapeutic Rationale
Pitt Hopkins syndrome (搜索) is a neurogenetic disorder for which no approved treatment currently exists. "For families living with Pitt Hopkins syndrome, the absence of any approved treatment has meant managing symptoms with no way to address the underlying cause or to change the course of this disease," said Alex Fay, M.D., Ph.D., Principal Investigator for the MZ-1866 (搜索) Phase 1/2 study at UCSF Benioff Children's Hospitals.
MZ-1866 (搜索) is a novel AAV9-TCF4 (搜索) gene replacement therapy that aims to address the underlying disease biology of Pitt Hopkins syndrome (搜索) by providing functional copies of the TCF4 gene. In addition to the newly granted Rare Pediatric Disease Designation, MZ-1866 has previously been granted Orphan Drug Designation and Fast Track Designation by the FDA.
Phase 1/2 UNITE Study Progress
The Phase 1/2 UNITE clinical trial is an open-label study evaluating a single administration of MZ-1866 (搜索) in participants with genetically confirmed Pitt Hopkins syndrome (搜索). The study is designed to enroll 12 participants across 4 sites in the United States and Israel and to evaluate a single dose of MZ-1866 delivered via intracerebroventricular administration. In addition to its primary safety objectives, the trial will evaluate developmental, communication, cognitive, and motor function exploratory endpoints.
The study has now enrolled 7 of 12 planned participants, surpassing 50% enrollment. "The MZ-1866 (搜索) Phase 1/2 trial has now enrolled more than 50% of the planned participants. Meeting this important milestone ahead of schedule reflects both the urgency families feel and the strength of the scientific rationale behind gene replacement for TCF4 (搜索) deficiency," added Dr. Fay. Enrollment is projected to be completed by the end of this year.
Development and Funding
MZ-1866 (搜索) was developed in collaboration with the Muotri Lab and licensed from the University of California San Diego. The Phase 1/2 study was made possible by funding from the California Institute for Regenerative Medicine (搜索) (CIRM), a state of California agency that funds regenerative medicine, stem cell, and gene therapy research (Grant Numbers TRAN1-13997 and CLIN2-19119).