Potential Therapy FB-101 Well-tolerated in Healthy Volunteers

1ST Biotherapeutics shares early data from Phase 1 clinical trial

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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FB-101, an experimental oral therapy that 1ST Biotherapeutics is developing as a treatment for Parkinson’s disease, was well-tolerated in the first part of a Phase 1 clinical trial in healthy volunteers, the company announced.

In the trial of (NCT04165837), which is being conducted in Baltimore,  24 adults (ages 19 to 55) were given a single dose of FB-101 and monitored for safety and pharmacological outcomes.

Results showed that “oral administration of FB-101 was well-tolerated across all cohorts with favorable safety results,” according to a press release from 1ST. The company stated there were “no clinically significant findings” on safety measures, including assessments of vital signs, laboratory tests, and measures of the heart’s electrical activity (electrocardiogram, or ECG).

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Pharmacological data from the first portion of the trial were generally as expected, showing a dose-dependent relationship between the amount of FB-101 administered and the amount of the medication that then was detectable in participants’ body fluids. There were no differences in pharmacological data based on participants’ age or sex.

According to 1ST, analyses of the participants’ cerebrospinal fluid — the liquid that surrounds the brain and spinal cord — suggested that FB-101 is able to cross the the blood brain barrier (BBB).

The BBB acts to regulate which substances from the blood are able to enter into the brain. This can help protect the brain from toxins, but also often acts as an obstacle when it comes to designing therapies intended to treat brain conditions. According to 1ST, other trials of therapies with a similar mechanism of action to FB-101 have failed to show effectiveness, and this is largely attributable to a failure to cross the BBB and enter the brain in substantial quantities.

The company is waiting on authorization from the U.S. Food and Drug Administration to start the second portion of the trial, in which participants will receive multiple dosages of FB-101 and be evaluated further for safety and pharmacological outcomes. The second part of the Phase 1 trial is expected to start before the end of the year, and results are expected to inform the design of subsequent trials in Parkinson’s patients, 1ST stated.

Parkinson’s disease is characterized by the buildup of aggregates (clumps) of alpha-synuclein in the brain, which are toxic to brain cells and are thought to drive the disease. FB-101 is designed to block the activity of c-Abl (Abelson tyrosine kinase), a protein that has been implicated in the toxic buildup of alpha-synuclein. 1ST is developing the therapy as a potential treatment for Parkinson’s and other diseases characterized by toxic alpha-synuclein clumps.

The company also is developing another investigational medication for Parkinson’s called FB418, which works by simultaneously blocking the activity of c-Abl and another Parkinson’s-related protein called LRRK2.

FB418 is expected to enter initial clinical studies next year, according to 1ST.