BlackfinBio Gets FDA Nod for Phase 1/2 Trial of Gene Therapy BFB-101 for SPG47

BlackfinBio Ltd, based in Cheshire, UK, has announced a significant advancement in their efforts to develop treatments for rare neurological diseases. The U.S. Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application. This approval allows BlackfinBio to initiate a Phase 1/2 clinical trial for their innovative adeno-associated virus (AAV) gene therapy, BFB-101, targeting children diagnosed with Hereditary Spastic Paraplegia, Type 47 (SPG47).

The clinical trial is set to be conducted at Boston Children’s Hospital in the U.S., with recruitment expected to start by the end of 2025. This groundbreaking therapy, BFB-101, has received both orphan drug designation (ODD) and rare pediatric disease designation (RPDD) from the FDA, underscoring its potential significance in treating SPG47.

SPG47 is a rare genetic disorder that affects children, characterized by progressive spasticity in the lower limbs, developmental delays, and intellectual disabilities. The condition stems from harmful mutations in the AP4B1 gene. At present, there are no effective treatments available for SPG47. BFB-101 aims to address the genetic root of SPG47 by delivering a functional copy of the AP4B1 gene, potentially halting or reversing the progression of the disease. Preliminary studies have shown promising safety and efficacy results for BFB-101.

The planned clinical trial will adopt an open-label, single-center design. The primary goal is to evaluate the safety and effectiveness of a single injection of BFB-101 into the cerebrospinal fluid at the base of the brainstem. This method, known as 'ICM' administration, facilitates rapid delivery of the therapy to the central nervous system. The trial will involve up to five children with the AP4B1-associated condition. Secondary objectives include assessing the impact of the therapy on motor skills, development, and overall quality of life.

Peter Nolan, Founder and CEO of BlackfinBio Ltd, emphasized the importance of the FDA's approval, marking a pivotal moment for the company's program aimed at treating rare neurological diseases. Nolan expressed enthusiasm about starting patient enrollment in the U.S. and collaborating with the Boston Children’s Hospital team to explore BFB-101’s therapeutic potential for children with SPG47.

Dr. Darius Ebrahimi-Fakhari, serving as the Principal Investigator at Boston Children’s Hospital, highlighted the significance of this trial for children impacted by SPG47, emphasizing the urgent need for effective treatments. Dr. Ebrahimi-Fakhari views BFB-101 as a promising candidate that could potentially transform the lives of patients and families affected by this disorder.

Professor Mimoun Azzouz, Founder and Chief Scientific Officer of BlackfinBio Ltd, shared his excitement about reaching this critical IND milestone. Azzouz affirmed the company's commitment to transforming treatment options for children with SPG47 and significantly improving the lives of affected families.

BFB-101 represents a potential breakthrough in addressing the genetic causes of SPG47 by restoring AP-4 function. It is administered as a single lifetime dose directly to the brain. Initially developed by Professor Azzouz at the University of Sheffield, the project has received support from the Cure AP-4 Foundation and LifeArc, as well as funding from the European Union’s Horizon 2020 research and innovation program.

BlackfinBio Ltd continues to advance its pipeline of gene therapies targeting rare neurological conditions, with BFB-101 at the forefront as a potential treatment for the ultra-rare genetic disorder, spastic paraplegia 47.