- BioMarin Pharmaceutical Inc BMRN faces another setback in its gene therapy development program after the FDA placed the phenylketonuria (PKU) gene therapy trial on hold.
- The Phase 1/2 Phearless study is evaluating BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy for PKU.
- PKU is a rare genetic disease that manifests at birth and is characterized by an inability to break down phenylalanine, an amino acid commonly found in many foods.
- The agency ordered the pause on the study after liver tumors were seen in mice given the therapy in preclinical testing.
- BioMarin’s preclinical study was carried out to explore the durability of BMN 307 activity in mice with two germline mutations that may predispose them to cancer development.
- One mutation eliminated the PAH gene that’s missing in PKU, while the second rendered the animals immunodeficient, which could also raise the risk of malignancy.
- Of 63 animals treated, six of seven animals administered BMN 307 at the highest dose group – 2e14 Vg/kg – developed liver tumors.
- Five of the mice had adenomas, and one had hepatocellular carcinoma.
- So far, participants have received doses of either 2e13 vg/kg or 6e13 vg/kg of BMN 307, although the protocol does include a third, higher-dose group.
- Read Next: Drug For Short-Limbed Dwarfism From Biomarin Wins European Approval.
- The clinical hold for the PKU program comes after BioMarin’s hemophilia A gene therapy was rejected by the FDA last summer.
- The rejection would delay the approval by around two years, as the FDA asked for two-year data on all study participants.
- The last patient will complete two years of follow-up in November 2021.
- Related content: Benzinga's Full FDA Calendar.
- Price Action: BMRN shares closed at $84.95 on Friday.
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