Exegenesis Bio to Present 9-Patient Data from a Phase 1/2 Clinical Trial of EXG001-307, a Novel rAAV Gene Therapy for Spinal Muscular Atrophy (SMA) Type 1: Improved Head Control and Sitting Without External Assistance
25 Abril 2024 - 11:33AM
Business Wire
27th Annual Meeting of the American Society
of Gene and Cell Therapy in Baltimore, MD on May 8, 2024
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
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version on businesswire.com: https://www.businesswire.com/news/home/20240425546173/en/
Company Contact: Mahen Gundecha Chief Business Officer
MahenGundecha@ExegenesisBio.com
Company Website: www.ExegenesisBio.com