PASG - Passage Bio posts early Phase 1/2 data for lysosomal storage disease drug

Passage Bio ( NASDAQ: PASG ) announced Wednesday new interim results from the first six patients in its Phase 1/2 study for gene therapy PBGM01 targeted at GM1 Gangliosidosis (GM1), a rare, genetically driven form of lysosomal storage disease. Passage Bio ( PASG ) shares rose ~17% in reaction.

Imagin-1 dose-escalation involved four groups of two pediatric patients with early and late infantile GM1 Gangliosidosis who received PBGM01 injections.

The data from the first three cohorts indicated no treatment-related serious adverse events ((SAEs)) over a follow-up period of 3 – 20 months, the company said, adding that all treatment-related adverse events ((AEs)) were mild to moderate.

According to Passage Bio ( PASG ), PBGM01 led to dose-dependent increases in biomarkers, including beta-galactosidase (?-Gal) in cerebrospinal fluid, particularly in patients in the high-dose cohort. GM1 is characterized by a very low activity of the ?-Gal.

As for the clinical response, citing evaluations of investigators and caretakers, the company said patients with milder development delay at dosing demonstrated a higher response.

Passage Bio ( PASG ) has completed dosing in the entire dose-ascending portion of the study, and early data from Cohort 4 is expected by mid-2023.

In October, Passage Bio ( PASG ) named William Chou as its new Chief Executive replacing interim CEO Edgar Cale.

For further details see: