April 19, 2022
PHILADELPHIA, April 19, 2022 – SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, will present safety, efficacy, and biodistribution data from preclinical studies of its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN) at the American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting in Washington, DC May 16-19. These data have formed the basis of SwanBio’s progress to advance SBT101 into clinical trials.
“At this year’s ASGCT Annual Meeting, we look forward to sharing more information about the data, decisions, and guardrails that informed the design of our upcoming Phase 1/2 study of SBT101, an AAV-based therapy we’re exploring for patients with AMN, a progressive and debilitating neurodegenerative disease with no available treatment options,” said Karen Kozarsky, Ph.D., chief scientific officer of SwanBio Therapeutics. “As we move closer to bringing SBT101 to the clinic, we are pleased to share a broader view of the preclinical data we have gathered that support our approach to target the root cause of this disease safely and effectively.”
Abstract #57: Selection of Clinical Doses for SBT101, an AAV9-hABCD1 Vector for the Treatment of Adrenomyeloneuropathy
Session: Oral Presentation – “AAV Preclinical CNS Gene Therapy” Room 204
Date & Time: May 16, 2022, 3:45-4:00 pm ET
In addition, SwanBio will be presenting new proof-of-concept data, in collaboration with the Bellvitge Biomedical Research Institute (IDIBELL) in Spain.
Abstract #529 / Poster Board #Tu-34: An AAV9-Encoding Human ABCD1 Shows Functional Improvement Following Spinal Cord Delivery in a Rodent Model of AMN
Session: Poster Presentation – “AAV Vectors – Preclinical and Proof-concept Studies II” Hall D
Date & Time: May 17, 2022 5:30-6:30 pm ET
SwanBio will also present two encore posters, showcasing positive, preclinical safety data for SBT101 as well as data demonstrating effective delivery of SBT101 to target tissue in preclinical models.
Abstract #162 / Poster Board #M-43: Preclinical Pharmacology and Toxicology of Intrathecally Infused AAV9-hABCD1 (SBT101), a Gene Therapy Candidate for AMN, in Non-Human Primates
Session: Poster Presentation – “AAV Vectors – Preclinical and Proof-concept Studies I” Hall D
Date & Time: May 16, 2022, 5:30-6:30 pm ET
Abstract #922 / Poster Board #W-48: Optimization of Intrathecal Delivery of an Infused AAV9 Vector for Delivery of a Gene Therapy Candidate for AMN in Non-Human Primates
Session: Poster Presentation – “AAV Vectors – Preclinical and Proof-concept Studies III” Hall D
Date & Time: May 18, 2022, 5:30-6:30 pm ET
SBT101 is the first AAV-based gene therapy candidate cleared by the FDA for human studies, designed to compensate for the disease-causing ABCD1 mutation in people living with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of AMN disease markers in mouse models and was shown to be well-tolerated in non-human primates at six months post-treatment. The clinical program for SBT101 builds on this positive preclinical data, plus the company’s deep understanding of the underlying pathophysiology of the disease and the patient experience of AMN, including new insights being gathered in an ongoing natural history study.
SwanBio expects to initiate a randomized, controlled Phase 1/2 clinical trial designed to assess the safety and efficacy of SBT101 in patients with AMN in the second half of 2022. In early 2022, the FDA cleared SwanBio’s Investigational New Drug application for SBT101 and granted SBT101 Fast Track and Orphan Drug Designation.
Adrenomyeloneuropathy (AMN) is a progressive and debilitating neurodegenerative disease caused by mutations in the ABCD1 gene that disrupt the function of spinal cord cells and other tissues. AMN is characterized by loss of mobility in adulthood, incontinence, pain, and sexual dysfunction which all affect quality of life. Between 8,000-10,000 men in the United States and European Union are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom control.
