FB-101 / 1ST Bio, D&D Pharmatech - LARVOL DELTA

Safety, Tolerability, and Pharmacokinetics of Oral FB-101 in Healthy Subjects (clinicaltrials.gov) - P1 | N=24 | Completed | Sponsor: 1ST Biotherapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Movement Disorders • Parkinson's Disease

"wow this is amazing @fb0101 @zane_blanton @CFFILMZ @benskigen @XXXmetaybXXX @skyyfettiNFT @7012io @_tnk @BrumbackLab @monish00 @gonzoffs @thom_6light @prachyaklui" (@Deborahkang1997)

1ST Biotherapeutics, Inc., Announces Positive Phase I Data of c-Abl Inhibitor in Neurodegeneration (GlobeNewswire) - P1 | N=24 | NCT04165837 | Sponsor: 1ST Biotherapeutics, Inc. | "1ST Biotherapeutics, Inc...announced a successful completion of its Phase I single ascending dose (SAD) study of FB-101 in healthy adult subjects. 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. 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....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."

New P1 trial • P1 data • Trial status • CNS Disorders • Parkinson's Disease

Intravenous Administration of FBX-101 (AAVrh10. hGALC) Following Hematopoietic Stem Cell Transplantation, a Potential Treatment for Infantile Krabbe Disease: RESKUE Phase 1/2 Clinical Trial (ANA 2022) - "FBX101 was well tolerated with no treatment-related serious adverse events at Day 90... The first patient’s data of the RESKUE clinical trial shows that IV FBX-101following HSCT has been safe and well tolerated through Day 90 with DTI results suggesting adequate GALC to support normal brain development."

Clinical • P1/2 data • CNS Disorders • Pain

Safety, Tolerability, and Pharmacokinetics of Oral FB-101 in Healthy Subjects (clinicaltrials.gov) - P1 | N=24 | Active, not recruiting | Sponsor: 1ST Biotherapeutics, Inc. | Unknown status ➔ Active, not recruiting | N=48 ➔ 24 | Trial completion date: Jun 2020 ➔ Aug 2022 | Trial primary completion date: Mar 2020 ➔ Mar 2022

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease

First Biotherapeutics, Parkinson's Disease Phase 1 Phase 1 Completed in the US [Google translation] (Hankyung) - "First Biotherapeutics announced...that it has completed Phase 1 Phase 1 (Part A) of US clinical trial for 'FB-101', an oral (oral) Parkinson's disease treatment candidate....Phase 1 Part B is a multiple-dose escalation study (MAD) in which the dose of the drug is increased with repeated administration. It aims to launch within the year."

Trial status • CNS Disorders • Parkinson's Disease

DYSCOVER-1: FB101 Intervention in Women Screened to Have Vaginal Dysbiosis (clinicaltrials.gov) - P=N/A; N=40; Recruiting; Sponsor: Freya Biosciences ApS

Clinical • New trial

A Novel, Selective c-Abl Inhibitor, Compound 5, Prevents Neurodegeneration in Parkinson’s Disease. (PubMed, J Med Chem) - "Additionally, compound 5 markedly ameliorated the loss of dopaminergic neurons, c-Abl activation, Lewy body pathology, neuroinflammatory responses, and behavioral deficits induced by α-syn PFF injection in vivo. Taken together, these results suggest that compound 5 could be a pharmaceutical agent to prevent the progression of PD and α-synucleinopathies."

Journal • CNS Disorders • Inflammation • Movement Disorders • Parkinson's Disease • ABL1

Journal of Medicinal Chemistry 'c-Abl inhibitor' as a Parkinson's disease treatment mechanism and preclinical efficacy results paper presentation.."Cover paper decoration" [Google translation] (Biospectator) - "A study showing the mechanism of action and efficacy of a c-Abl inhibitor developed by 1ST Biotherapeutics to slow stage progression in α-synucleinopathies including Parkinson's disease (PD) has been published....First Bio announced on the 29th that research on c-Abl inhibitors was published in the Journal of Medicinal Chemistry, an international scientific journal in the field of pharmaceutical chemistry. The research results adorned the cover of the offline version of the latest issue, October 28. This research was conducted in collaboration with Professor Hanseok Koh's team at Johns Hopkins University and Neuraly, a US established by Professor Seulgi Kim."

Preclinical • CNS Disorders • Parkinson's Disease

[VIRTUAL] Disease Correction by delivery of FBX101, an Intravenous AAV Gene Replacement Therapy, Administered with Unrelated Umbilical Cord Blood Transplantation for the Treatment of Infantile Krabbe Disease (ESGCT 2021) - "In conclusion, the administration of FBX101 with UCBT is a safe and highly effective approach to address the CNS and PNS symptoms of Krabbe disease. FBX101 is entering the clinical phase in 2021."

Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Peripheral Neuropathic Pain • Transplantation

First Bio, US patent registration for ’FB418, a candidate substance for degenerative brain disease’ [Google translation] (Yakup Shinmoon) - "First Biotherapeutics...announced that it has obtained material approval for 'FB418', a candidate substance for the treatment of degenerative brain diseases such as Lou Gehrig's disease, Parkinson's disease, and Alzheimer's....The patents acquired this time have been registered with the U.S. Patent and Trademark Office as of August 24, 2021....'The company also holds 100% of the patent rights for FB418, and it is developing simultaneously with FB-101, which is currently undergoing clinical trials...'"

Patent • Alzheimer's Disease • CNS Disorders • Parkinson's Disease

Newly added product (clinicaltrials.gov) - P1, Parkinson's Disease

Pipeline update