Passage Bio will be moving into ALS with its gene therapy, which is currently being investigated in frontal temporal dementia. Image credit: Getty Images / Andrew Brookes.

Passage Bio has plans to talk with the US Food and Drug Administration (FDA) to initiate trials of its investigative gene therapy in amyotrophic lateral sclerosis (ALS).

The company will be seeking approval from the agency to initiate a trial evaluating PBFT02, an adeno-associated virus vector 1 (AAV1) capsid-based gene therapy, with hopes that conversations will take place by the end of 2024, Passage Bio CEO Will Chou told the Clinical Trials Arena.

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The candidate is currently being investigated in the Phase I/II upliFT-D trial (NCT04747431) in frontal temporal dementia (FTD) with granulin (GRN) mutations, with the company recently gaining approval to include an additional cohort of patients with FTD-C9orf72.

The GRN gene causes a deficiency of progranulin (PGRN) and is also present in ALS and Alzheimer’s disease, which is another indication Passage Bio plans to move into with this candidate.

Data previously announced from the upliFT-D trial originally included low-dose and high-dose mandatory cohorts, as well as an optional third cohort. Each cohort has a minimum of three patients up to a maximum of five patients.

After the first cohort of patients was dosed, Chou said that the company saw encouraging PGRN data and maintained the same dose for the second cohort.

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Patients will be monitored for two years during the active trial, with longer-term follow-up after the active trial period. Chou said that more data will be announced from the upliFT-D trial in September 2024.

In the FTD pathway, Passage Bio plans to move into a pivotal study after the upliFT-D trial and will be looking at other FTD investigations to monitor which biomarker-based endpoints are being looked at by the FDA as endpoints in pivotal studies.

PBFT02 acts by targeting PGRN, which rescues dopamine-producing cells in the brain and protects from the progressive cell death characteristic of the disease. In addition, PGRN preserves the normal levels of motor activity and normal control of motor function. It is administered by intra-cisterna magna injection to the cerebrospinal fluid (CSF)-filled subarachnoid space.

The FDA has granted fast track and orphan drug designation to PBFT02, which has also received orphan drug designation from the European Commission.