SynaptixBio Secures Second FDA Orphan Drug Designation for TUBB4A Leukodystrophy Variant
核心洞察
SynaptixBio (搜索) received its second FDA Orphan Drug Designation for developing therapies targeting Isolated Hypomyelination (搜索), a variant of TUBB4A leukodystrophy (搜索) with reportedly milder symptoms than H-ABC (搜索).
The company is using antisense oligonucleotide technology to target mutated TUBB4A (搜索) genes that form toxic proteins, preventing normal myelin formation in brain nerve fibers.
SynaptixBio (搜索) has extended its collaboration with Evotec to broaden the pipeline with additional ASO (搜索) candidates for treating H-ABC (搜索), the most severe form of TUBB4A leukodystrophy (搜索).
SynaptixBio (搜索) has secured a second Orphan Drug Designation (ODD) from the US Food and Drug Administration for developing treatments targeting TUBB4A leukodystrophy (搜索) variants, marking a significant milestone in the company's efforts to address rare, incurable neurological diseases.
The biotech firm received its latest ODD for Isolated Hypomyelination (搜索), a recently identified variant of TUBB4A leukodystrophy (搜索) that appears similar to H-ABC (搜索) but without atrophy of the basal ganglia and cerebellum. According to the company, symptoms of Isolated Hypomyelination are reported to be milder than H-ABC, the most severe form of the disease for which SynaptixBio (搜索) received its first ODD in early 2023.
"This ODD is a huge boost to our efforts in tackling these devastating, life-limiting rare diseases," said Dan Williams, CEO at SynaptixBio (搜索). "Our whole strategy is centred around achieving these prestigious designations, which make development of therapeutics easier and more cost-effective."
Antisense Oligonucleotide Technology Platform
SynaptixBio (搜索) is employing antisense oligonucleotide (ASO (搜索)) technology to target TUBB4A (搜索)-related leukodystrophies. The ASO molecules work by targeting the mutated TUBB4A gene to prevent it from forming toxic proteins that interfere with myelin formation. A mutated TUBB4A gene ultimately results in less myelin for insulating nerve fibers in the brain, leading to disruption of signals between neurons.
With the toxic protein suppressed, other proteins can step in to help form normal myelin sheaths surrounding nerve fibers in the brain. The technology has been proven effective in treating other dystrophies, including Duchenne muscular dystrophy (搜索), and offers advantages in terms of development speed and cost-effectiveness.
Expanded Collaboration and Pipeline Development
The company has extended its collaboration agreement with Evotec, a leading life science company, to broaden its therapeutic pipeline with additional ASO (搜索) candidates for treating H-ABC (搜索). The original collaboration began in April 2022, building on research conducted by the Children's Hospital of Philadelphia (CHOP (搜索)), which identified a candidate ASO as a potential therapy for H-ABC under a sponsored research agreement.
"The contract with Evotec is absolutely vital for us to ensure we have a range of viable candidates to take through further testing," Williams explained. "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."
Regulatory Advantages and Market Positioning
The ODD designation provides SynaptixBio (搜索) with several regulatory and commercial advantages, including tax credits to reduce research costs, grants to offset development expenses, and exemption from some pre-marketing authorization requirements and regulatory fees. Upon approval, the designation grants a potential seven years of market exclusivity and data exclusivity.
The company also received a Rare Paediatric Disease Designation (RPDD) late last year, which can lead to the award of a Priority Review Voucher (PRV) once a product is approved. A PRV can expedite the FDA's product review time and can be sold or transferred to larger pharmaceutical companies, potentially offsetting the high costs associated with rare disease therapy development.
Funding and Clinical Timeline
SynaptixBio (搜索) successfully completed a second round of investment, raising total funding to £13.2 million, which will support the company through the start of in-human clinical trials scheduled for later this year. The company also secured a £490,000 BioMedical Catalyst grant from Innovate UK (搜索) specifically to tackle less common variants of TUBB4A leukodystrophy (搜索).
According to a 2020 European Commission report, "1 in 17 people will be affected by a rare disease at some point in their lives. This amounts to 3.5 million people in the UK." With over 7,000 known rare diseases and more emerging continuously, SynaptixBio (搜索)'s work addresses a significant unmet medical need in the rare disease space.
The company operates as a virtual organization with only two full-time and three part-time employees, contracting out all drug research and business functions to specialized partners like Evotec and CHOP (搜索).