Thursday, October 05, 2023
SwanBio Therapeutics, a company focused on gene therapy for the treatment of inherited neurological conditions, has announced the commencement of the second dose-escalation cohort in its initial interventional clinical trial, known as PROPEL. PROPEL is a Phase 1/2 clinical study that represents the first human trial to assess the safety and efficacy of SBT101, an investigational gene therapy designed to address the ABCD1 mutation responsible for adrenomyeloneuropathy (AMN).
An independent Data Safety Monitoring Board (DSMB) recommended moving forward with the higher-dose cohort after reviewing the initial safety data in August. In the first cohort, patients received a single intrathecal administration of the lower of two planned doses and were closely monitored for treatment safety and tolerability. Fortunately, the dosing procedures were completed successfully, and there have been no reports of serious adverse events related to the procedure or the gene therapy product.
David Weiner, Chief Medical Officer of SwanBio, expressed pride in the successful and well-tolerated administration of their lead candidate, which is the only gene therapy in clinical development for AMN. He also noted that progressing to the second cohort brings them closer to their goal of providing a safe and effective treatment for men and families affected by AMN.
Syncona Investment Management Limited, the lead investor and majority shareholder, has committed an additional $10 million to fund the second cohort of PROPEL following the positive data review from the first cohort.
The PROPEL study is currently active in the United States and is expanding into Europe after receiving approval for its Clinical Trial Application (CTA) from the Central Committee on Research Involving Human Subjects (CCMO) in the Netherlands. SwanBio is also active in Europe, with multiple clinical sites participating in CYGNET, a five-year natural history study of men with AMN, which completed enrollment in 2022.
PROPEL is leveraging insights from CYGNET and is exploring the use of wearable technologies to reduce onsite patient visits and capture disease progression more efficiently.
SBT101, the gene therapy candidate for AMN, has shown promise in preclinical studies, including improvements in disease markers and functional outcomes in mouse models. It has also demonstrated good tolerability and persistence in non-human primates. SBT101 has received Fast Track and Orphan Drug Designation from the U.S. FDA and Orphan Drug Designation from the European Medicines Agency.
Adrenomyeloneuropathy (AMN) is a progressive neurodegenerative disease caused by mutations in the ABCD1 gene, leading to spinal cord and tissue dysfunction. AMN results in loss of mobility, incontinence, pain, sexual dysfunction, and adrenal gland dysfunction, significantly impacting patients' quality of life. Currently, there are no approved treatments for AMN, and the standard of care focuses on managing symptoms. It affects approximately 8,000-10,000 men in the United States and EU5 countries (France, Germany, Italy, Spain, and the United Kingdom).