Medical Innovation Exchange

Passage looks to offload lead program, 2 other clinical-stage assets

After sending 26% of staff and two C-suite execs packing this summer, Passage Bio once again wants to lighten its load. The gene therapy biotech is looking for a partner to pick up three of its clinical-stage pediatric candidates, including a lead program.

The gene therapies up for grabs are PBGM01, PBKR03 and PBML04. The candidate are being assessed in a phase 1/2 clinical trial for GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy (MLD), respectively.

PBGM01, the GM1 gangliosidosis candidate, was one of Passage’s lead programs, according to its website. In August, the biotech shared interim safety data evaluating two doses of PBGM01 among children and infants with the rare lysosomal storage disease in an open-label trial. At the time, Passage said the treatment was well tolerated and had a favorable safety and immunological profile. Further data was expected in mid-2024.

Now, Passage is prioritizing a preclinical Huntington’s disease program that already has secured a partnership with the Penn Gene Therapy Program.

The company has also shared early phase 1/2 data evaluating PBFT02, an adeno-associated virus-delivery gene therapy for frontotemporal dementia (FTD). The company believes PBFT02 has the potential to address progranulin deficiency, a key driver of disease progression in individuals with FTD with granulin mutations.  

An initial dose of PBFT02 resulted in a 3.6 to 6.6-fold increase in CSF progranulin at day 30 compared to baseline among the three patients included in the assessment, according to Passage.  

No serious adverse events were observed among two of the three patients. The third patient received a low level of immunosuppression and experienced two SAEs that were consistent with an immune response. Passage didn’t divulge details about treatment emergent adverse events, but said all occurrences were mild to moderate in severity.

The new strategic shift is meant to optimize resources and provide each gene therapy candidate with the best chance to reach patients in need, Passage Bio CEO and President William Chou, M.D., said in a Dec. 20 release.

In July, the company laid off 26% of its workforce, primarily among chemistry, manufacturing and controls (CMC) employees who ensure drug quality throughout the product development life cycle. Passage’s former Chief Financial Officer Simona King and former Chief Technical Officer Alex Fotopoulos both left the biotech as well.

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