PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 Phase 1/2 clinical trial in Spinal Muscular Atrophy (SMA) Type I at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore, Maryland on May 8, 2024 (poster #627).
EXG001-307 is a next generation recombinant adeno-associated virus (rAAV) gene therapy in development for Spinal Muscular Atrophy (SMA) Type 1. Data from nine patients demonstrate improved head control and sitting without external assistance as early as three months following dosing.
EXG001-307 has a unique AAV design, including a novel pro-NS promoter, leading to high expression in target spinal cord tissue and reduced off target expression in liver and heart tissue versus currently available gene therapy.
Data Highlights:
- EXG001-307 has a unique engineered AAV design that includes a novel pro-NS promoter, which is believed to contribute to improved efficacy and safety in SMA Type 1.
- EXG001-307 has demonstrated high expression in target spinal cord tissue and reduced off target expression in liver and heart tissue than AAV9.CBA.SMN1.
- Patients in the EXG001-307 low-dose group (1.1 E 14 vg/kg) achieved head control 3 to 6 months after dosing and sitting without external assistance 11 months following dosing.
- The first patient in the mid-dose group (1.5 E 14 vg/kg) achieved sitting with assistance 3 months following dosing.
- EXG001-307 demonstrated high tolerability and no dose-limiting toxicity, no >Grade 2 test article related serious adverse events, and no >Grade 1 elevation of transaminases or cardiac enzymes.
“Spinal Muscular Atrophy is a debilitating and fatal genetic disease that impacts the lives of thousands of babies and young children and their families. I am excited that our team is making rapid progress in our mission to bring this much-needed treatment option to SMA patients worldwide. We are excited about the clinical efficacy and safety data emerging from our Phase 1/2 SMA clinical trial in China. This is leading us to accelerate development in the US. We expect to file our US IND for SMA Type 1 in 4Q 2024. We are also exploring an accelerated development path in the slightly older SMA Type 2/3 patient population. We look forward to sharing further details during our poster session at ASGCT in Baltimore on May 8, 2024. I would like to congratulate our team for embracing our mission to bring innovative and life-changing genetic medicines to patients globally and for driving our ambitious clinical development timelines,” stated Dr. Zhenhua Wu, CEO of Exegenesis Bio.
About EXG001-307
EXG001-307 is a recombinant adeno-associated virus (rAAV)-based gene therapy in clinical development for Spinal Muscular Atrophy (SMA) Type 1. EXG001-307 has demonstrated significantly improved efficacy in SMA Type 1 patients (CHOP Intend score) and lower off target effects in liver and cardiac tissue. Patients in both low and mid-dose cohorts demonstrated improved head control and sitting ability within the 3 to 11 months of dosing. Exegenesis Bio will share additional clinical and non-clinical efficacy and safety data during a poster session at ASGCT on Wednesday May 8, 2024. Poster # 627.
About Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is an autosomal recessive inherited neuromuscular disease, characterized by motor neuron degeneration, skeletal muscle atrophy, muscle weakness, and ultimately death. SMA is the most common fatal disease in infants, with incidence in newborns ranging from one in six thousand to one in ten thousand.
SMA is classified into five subtypes, based on the age of onset, severity of symptoms, and motor milestones. SMA Type 0 manifests prenatally, Type 1 appears during the first six months of age, Type 2 appears between 6 and 18 months of age, Type 3 begins as early as 18 months of age, while Type 4 has late onset and symptoms develop in adulthood1. Data suggest that infants with SMA type 1, the most common type, have a median survival of 10.5 months, and 8% survival rate to 20 months without permanent respiratory support2.
SMA is caused by a homozygous loss of SMN1 genes on chromosome 5q13 due to mutations or deletions, leading to a decreased level of survival motor neuron (SMN) protein, which triggers motor neuron overexcitation and degeneration, synaptic dysfunction, and reduced muscle fiber volume. Current SMA therapies work by increasing levels of SMN protein in the body3.
About Exegenesis Bio
Exegenesis Bio is a clinical stage global gene therapy company with operations in USA and China. The company’s innovative gene therapy pipeline is based on proprietary capsids, promoters and unique protein engineering designs. The company has received IND clearances from US FDA and China CDE and is currently conducting Phase 1/2 clinical trials in USA and China.
EXG001-307 is a recombinant AAV (rAAV) gene therapy in Phase 1/2 clinical development for Spinal Muscular Atrophy Type 1 in China. The company plans to file a US IND for EXG001-307 in 2024.
EXG102-031 is a recombinant AAV (rAAV) based gene therapy in clinical development for neovascular Age Related Macular Degeneration (nAMD) in USA and China. The EXG102-031 clinical study is being conducted at two clinical sites in the USA and eight additional sites in China.
Exegenesis Bio has built full end-to-end capabilities that include discovery, translational, non-clinical and clinical development, quality and manufacturing. The company operates state-of-the-art cGMP manufacturing facilities that include 500 Liter and 2,000 Liter disposable bioreactors for viral vectors and 30 Liter disposable fermenters for plasmids. The company has raised over $200 Million since inception in 2019 and currently employs over 200 scientific and operations staff worldwide.
Please contact us to discuss US and Global licensing and collaboration opportunities.
References:
- Annoussamy et al., Ann Clin Transl Neurol. 2021 8(2):359-373
- Finkel et al., Neurology 2014;83;810-817
- Kirwin et al., Mol Genet Genomic Med. 2013; 1(2): 113–117
