Homology Medicines initiates Phase 1/2 trial for PKU gene therapy

The firm’s drug (HMI-102) is expected to be the first gene therapy candidate for phenylketonuria (PKU) to enter clinics.

Genetic drug maker, Homology Medicines, Inc. has recently announced the enrollment of its Phase 1/2 pheNIX trial for HMI-102, a one-time gene therapy development candidate for treating adults suffering from phenylketonuria (PKU).

Reportedly, the pheNIX study is designed to assess the safety and effectiveness of the investigational gene therapy in a concurrently-controlled, randomized, dose-escalation study. PKU is an innate error of metabolism which is caused mainly by a PAH gene mutation that leads to the buildup of toxic phenylalanine (Phe), an amino acid made primarily from dietary protein.

Speaking on the move, Arthur Tzianabos, Ph.D., President & CEO, Homology Medicines, said that their firm’s early work with the clinical sites has enabled them to move expeditiously from attaining Investigational New Drug (IND) clearance to enrolling patients.

He said that the company now has both internal and external operational GMP capabilities utilizing its proprietary process development and commercial manufacturing platform that can supply HMI-102 for the pheNIX trial to commercial scale.

Meanwhile, Albert Seymour, Ph.D., Chief Scientific Officer, Homology Medicines, said that with the enrollment in the Phase 1/2 pheNIX study, HMI-102 will become the first gene therapy candidate for PKU to enter clinics, a major advancement for phenylketonuria patients.

He claims that this achievement reflects on the expertise and dedication of its team, an extraordinary group that has brought this new AAVHSC vector from initial research to a human development candidate in just a period of three years.

Adding further, Seymour said that the company is looking forward to continuing its work with patient advocacy organizations and PKU community as it tries to bring clinically significant and potentially curative therapies to patients.

For the record, in April 2019, Homology announced that the U.S. FDA (Food and Drug Administration) had cleared the IND application for HMI-102 for the treatment of PKU, and in May 2019, it granted them Fast Track Designation.

Prior to this, the European Medicines Agency (EMA) and FDA had granted HMI-102 with orphan drug designation in the U.S. and EU for the use of human hematopoietic stem cell-derived AAVHSC15 (adeno-associated virus) to treat PAH deficiency, the leading cause of PKU, cite reports.

Source Credit:https://www.homologymedicines.com/news-story/homology-medicines-initiates-phase-1-2-study-for-hmi-102-gene-therapy-candi

https://www.globenewswire.com/news-release/2019/06/10/1866292/0/en/Homology-Medicines-Initiates-Phase-1-2-Study-for-HMI-102-Gene-Therapy-Candidate-for-Adults-with-PKU-and-Expects-Initial-Clinical-Results-by-Year-End-2019.html