SynaptixBio & Evotec Extend Partnership For Rare Disease Therapies

SynaptixBio, the exclusive license holder for commercializing a treatment for a rare and fatal disease, announced today the extension of its collaboration with Evotec, a prominent life science firm, to discover more drug candidates for H-ABC, the most severe type of TUBB4A leukodystrophy. Initial drug research was conducted by the Children’s Hospital of Philadelphia (CHOP), a renowned leukodystrophy research center, through a sponsored research agreement. CHOP pinpointed a candidate antisense oligonucleotide (ASO) as a promising therapy for H-ABC. SynaptixBio and Evotec began their partnership in April 2022, focusing on developing CHOP’s candidate ASO into a treatment for H-ABC. This extension of their collaboration will allow Evotec to significantly expand the pipeline with additional ASOs as potential therapies.

Dan Williams, CEO at SynaptixBio, commented “The contract with Evotec is absolutely vital for us to ensure we have a range of viable candidates to take through further testing. Not only do they have the capabilities to identify and develop candidate drugs, they can then help us take them right through clinical trials and beyond.They are instrumental in supporting us as we prepare for those clinical trials.”

SynaptixBio operates as a virtual company with a minimal staff of two full-time and three part-time employees. All aspects of drug research and business operations are outsourced. Antisense oligonucleotides (ASOs) function by inhibiting the production of proteins from the mutated TUBB4A gene, effectively silencing the gene. Mutations in the TUBB4A gene lead to reduced myelin in the brain's nerve fibers, causing neuronal signal disruptions. Recently, the FDA granted SynaptixBio a second Orphan Drug Designation (ODD) to support the development of a treatment for another form of TUBB4A leukodystrophy, Isolated Hypomyelination.

In early 2023, SynaptixBio received its first ODD for the CHOP therapeutic targeting H-ABC. In October of the same year, the company secured a £490,000 BioMedical Catalyst grant from Innovate UK to address rarer variants of the disease. Antisense oligonucleotide (ASO) technology, which has shown success in treating conditions such as Duchenne muscular dystrophy, offers a fast and economical development process. A 2020 report from the European Commission reveals that rare diseases will impact 1 in 17 individuals during their lifetime, equating to 3.5 million people in the UK.