Neurology-Focused Passage Bio Reveals Initial Data From Dementia Study, Outlines Strategic Priorities

Zinger Key Points
  • Pipeline to focus on continued advancement of PBFT02 in FTD-GRN and explore PBFT02 in additional neurodegenerative diseases.
  • Passage Bio is pursuing potential partnership opportunities for pediatric lysosomal storage disease programs.

Passage Bio Inc PASG announced initial safety and biomarker data from three Cohort 1 patients in the ongoing global Phase 1/2 upliFT-D clinical trial evaluating PBFT02, an adeno-associated virus (AAV)-delivery gene therapy for frontotemporal dementia (FTD) with granulin (GRN) mutations

FTD is a form of early-onset dementia (memory loss). 

  • Dose 1 of PBFT02 was generally well-tolerated in patients 2 and 3, who received an enhanced steroid regimen following protocol amendment.
  • Patient 1 received a low level of immunosuppression and experienced two SAEs that were both asymptomatic and consistent with an immune response.
  • Following patient 1, the protocol was amended to increase the steroid regimen for patients 2 and 3.
  • No evidence of dorsal root ganglion (DRG) toxicity, as measured by nerve conduction studies, and no complications related to ICM administration were observed across any of the three patients.
  • Dose 1 of PBFT02 treatment resulted in a 3.6 to 6.6-fold increase in cerebrospinal fluid (CSF) progranulin (PGRN) at day 30 (n=3) relative to baseline. 
  • CSF PGRN increased to supraphysiologic levels of 10.7 to 17.3 ng/mL at day 30, exceeding the range found in healthy adult controls of 3.3 to 8.2 ng/mL (n=61).
  • CSF PGRN remained at supraphysiologic levels at six months with a concentration of 27.3 ng/mL (n=1).
  • Plasma PGRN levels remained below levels found in healthy adult controls through the available follow-up period across all patients.

The company also shared updated strategic priorities to optimize its portfolio for treating neurodegenerative conditions further.

  • Continuing clinical development of PBFT02 to treat FTD-GRN.
  • Pursuing PBFT02 in additional adult neurodegenerative diseases, including FTD-C9orf72, ALS, and Alzheimer's disease.
  • Prioritizing Huntington's disease preclinical program through existing Penn Gene Therapy Program partnership.
  • Pursuing potential partnership opportunities for clinical-stage pediatric programs in GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy.

Price Action: PASG shares are up 7.77% at $0.82 on the last check Wednesday.

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