Adrenomyeloneuropathy Gene Therapy Granted Fast Track Review

The FDA cleared SBT-101's investigational new drug application earlier in February 2022.

The FDA has granted fast track designation to SwanBio Therapeutics’ SBT-101, an investigational adeno-associated virus gene therapy candidate for the treatment of adrenomyeloneuropathy (AMN).

The FDA cleared the therapy’s investigational new drug application earlier in February; the company plans to initiatea phase 1/2 clinical trial in the second half of 2022 to evaluate the safety and efficacy of SBT-101 in patients with AMN.2

“People living with AMN currently rely on a combination of symptom control, physical therapy and mobility aids, with no approved treatment to slow or alter the progression of this debilitating disease,” Tom Anderson, chief executive officer and director, SwanBio Therapeutics, said in a statement.1 “The FDA’s decision to grant Fast Track designation for SBT101, following the recent clearance of our investigational new drug application for the program, further underscores the serious and unmet need for an effective treatment for AMN. We look forward to continuing to advance SBT101 as we work toward our goal of bringing life-changing treatments to patients.”

SBT101 is the first investigational adeno-associated virus (AAV) gene therapy for AMN. It contains a corrected ABCD1 gene designed to increase ABCD1 expression levels and to reduce very long chain fatty acid (VLCFA) levels in AMN.

READ MORE: Around the Helix: Cell and Gene Therapy Company Updates - February 16, 2022

The therapy previously showed efficacy in preclinical studies. SwanBio presented positive preclinical data at the European Society for Gene and Cell Therapy (ESGCT) Virtual Congress in October 2021 that showed dose-dependent expression of hABCD1 and reduction of VLCFA levels in mouse models.3 The therapy was well-tolerated in non-human primates at 6 months posttreatment.

SwanBio is also conducting the natural history CYGNET study (NCT05008874) in adult men with AMN to learn more about the natural progression of the disease, changes in quality-of-life, and AMN-related disabilities that may arise. Findings from CYGNET may also inform future research and clinical development of therapies targeting the disease. The study continues to recruit with an expected enrolment of 80 participants.

"SBT101 has the potential to become the first disease-modifying treatment for patients with AMN, a devastating and progressive disease with no approved treatments. We look forward to initiating clinical development of SBT101 later this year, bringing us closer to our ultimate goal of delivering life-changing treatments to patients,” Anderson said in a previous statement.2

In addition to SBT101, Swan Bio is also exploring several other candidates that address various neurological conditions, including undisclosed programs in monogenic neuropathies and polygenic neuropathies.

1. FDA grants fast track designation to SBT101, the first investigational AAV-based gene therapy for patients with adrenomyeloneuropathy (AMN). News release. SwanBio. February 16, 2022.
2. SwanBio Therapeutics Announces FDA investigational new drug clearance for first AAV-based gene therapy for the treatment of adrenomyeloneuropathy. News release. SwanBio Therapeutics. January 25, 2022.
3. SwanBio Therapeutics announces new preclinical data demonstrating proof of principle for SBT101 gene therapy as a potential treatment of adrenomyeloneuropathy. News release. SwanBio Therapeutics. October 19, 2021.
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