BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) has announced the dosing of the first subjects in its global PHEARLESS Phase 1/2 trial with its investigational gene therapy, BMN 307, in treating phenylketonuria (PKU) patients.
BioMarin evaluating BMN 307
BMN 307 is the company’s AAV5-phenylalanine hydroxylase (PAH) gene therapy that has been designed to normalize concentration levels of blood phenylalanine in PKU patients. The therapy works through the insertion of the right PAH gene into the liver cells. The company is evaluating BMN 307 to establish its safety and determine if a single treatment dose can restore natural Phe metabolism, enable normal diet, and normalize plasma Phe levels in PKU patients.
The company will carry the study using material produced with a commercial ready process to facilitate quick clinical development and potential support for its approval. The therapy will be the third possible PKU treatment alternative in the company’s PKU franchise.
Tarekegn G. Hiwot, the principal investigator of the study, stated that the first child was treated for PKU over 70 years ago in the UK at Birmingham Women’s and Children’s Hospital. He said that huge strides have been made in PKU treatment via clinical trials of gene therapy. Hiwot added that there is a significant unmet need for patients with PKU, and there is a need for more research in evaluating innovative therapies that have the potential of changing the treatment paradigm from PKU.
BioMarin building on previous therapies with BMN 307
Biomarin’s President, Worldwide Research and Development, Hank Fuchs, said that the company is committed to the PKU community, and it has been committed in R&D for innovative therapies aimed at advancing the standard of care for PKU patients. Fuchs said that the development of the BMN 307 gene therapy is building on its experience of previously approved therapies for PKU.
Christine Brown, the National PKU Alliance Executive Director, stated that PKU is a serious disorder that people struggle to manage. She added that they laud the effort of Biomarin in delivering a third treatment alternative for PKU in addition to its two first drug deliveries.