– Compound demonstrates excellent safety and tolerability in single ascending dose trial

– Efficient penetration of blood-brain barrier

YONGIN, South Korea I October 18, 2022 I 1ST Biotherapeutics, Inc., a clinical-stage biotechnology company dedicated to the discovery and development of novel small molecule therapeutics in neurodegenerative diseases and immuno-oncology, announced a successful completion of its Phase I single ascending dose (SAD) study of FB-101 in healthy adult subjects. FB-101 is a c-Abl inhibitor in development for the treatment of Parkinson’s disease and other neurodegenerative indications associated with the accumulation of alpha-synuclein. The compound was evaluated in a randomized, double-blind, placebo-controlled study to assess its safety, tolerability, and pharmacokinetics.

The SAD portion of the Phase I clinical trial of FB-101 was completed in Baltimore, MD, USA, and demonstrated that oral administration of FB-101 was well-tolerated across all cohorts with favorable safety results. No clinically significant findings were observed on safety measures including laboratory findings, vital signs, and ECGs at any dose.

FB-101’s blood concentration increased proportionally in a dose-dependent manner with oral administration of FB-101. No age or sex difference was observed. Of note, cerebrospinal fluid collected from the subjects confirmed FB-101’s efficiency in penetrating the blood brain barrier (BBB). 1ST Biotherapeutics stated that strong BBB penetration was well expected as it was observed in the preclinical studies. Other recent clinical trials targeting c-Abl and not sponsored by 1ST Biotherapeutics failed to meet their respective endpoints, largely due to the low concentrations of their drug in the brain, attributable to inefficient BBB penetration.

With FDA’s authorization, 1ST Biotherapeutics is planning on commencing the multiple ascending dose (MAD) portion of the Phase I study by the end of 2022. In this part of the study, repeated-doses of FB-101 will be administered to further assess and evaluate the safety and the pharmacokinetics of FB-101, including monitoring of pharmacodynamics biomarkers. Following the conclusion of the study, the Company plans to determine the efficacious dose to evaluate efficacy, safety, and tolerability of FB-101 in Parkinson’s Disease patients.

A subsequent Parkinson’s drug candidate called FB418, a dual inhibitor of c-Abl and LRRK2, will be entering a Phase I clinical study in the first half of 2023.


About FB-101

FB-101, 1ST Biotherapeutic’s lead c-Abl inhibitor, is being developed for the treatment for Parkinson’s disease and potentially for other neurodegenerative disease associated with alpha-synucleinopathy. FB-101’s Phase I clinical trial (identifier: NCT04165837) is currently ongoing.

About 1ST Biotherapeutics

1ST Biotherapeutics is a science-driven biopharmaceutical company focused on the development of breakthrough therapies in neurodegenerative diseases, immuno-oncology, and rare diseases. Founded in 2016, 1ST Biotherapeutics has rapidly developed a deep pipeline of first-in-class investigational therapeutics with clear mechanisms for proper disease modification and with excellent pharmacological properties. 1ST Biotherapeutics is based in Yongin, South Korea.

For more information, please visit 1stbio.com.

SOURCE: 1ST Biotherapeutics