First Patient With Adrenomyeloneuropathy Treated In Higher Dose Cohort of PROPEL Trial With SBT101 Gene Therapy

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The move was carried out in accordance with a recommendation for dose-escalation made by the trial’s independent Data Safety Monitoring Committee.

SwanBio Therapeutics has announced that the first patient has been dosed in the higher dose cohort of the phase 1/2 PROPEL clinical trial (NCT05394064) for SBT101, an investigational gene therapy intended to treat adrenomyeloneuropathy (AMN).1

The move was carried out in accordance with a recommendation for dose-escalation made by the trial’s independent Data Safety Monitoring Committee (DSMB). The recommendation was made after the DSMB carried out a review of safety data in August from the patients treated in the trial’s lower dose cohort, who received SBT101 via a single intrathecal administration. SwanBio noted that among these patients, there were no serious adverse events related to SBT101 or the administration procedure observed.

“We’re proud to share that our lead candidate – the only gene therapy in clinical development for the treatment of AMN – has been successfully administered to and well-tolerated by men living with this destructive, degenerative disease,” David Weiner, MD, the chief medical officer ofSwanBio, said in a statement.1 “Today’s milestone of progressing to our second cohort takes us one step closer to our goal of bringing a safe and therapeutically meaningful drug to men and families impacted by this disease.”

SwanBio stated that it is making moves to expand PROPEL, which was initiated in the United States in December 2022, to include locations in Europe, noting that it recently received clearance of a clinical trial application submitted to the Netherlands’ Central Committee on Research Involving Human Subjects.1,2 SwanBio also announced that, in light of the DSMB’s positive recommendation, its lead investor Syncona Investment Management Limited has provided the company with an additional infusion of $10 million to help cover the costs of carrying out trial activities for the higher dose cohort.

“Following a review of the patient data from the first cohort, we are encouraged that the DSMB has recommended the initiation of the higher-dose cohort and we look forward to seeing the data generated from this cohort,” Chris Hollowood, the chief executive officer of Syncona, added to the statement.1 “Our further investment of $10M will enable the company to deliver additional data to deliver its long-term potential.”

SwanBio’s complementary natural history study in patients with AMN, CYGNET (NCT05008874), is already in operation at several centers in Europe.1-3 CYGNET, which was designed to assess the disease progression of AMN in adult men, includes the observation of participants over the course of 2 years and is using wearable technology to track traditional motor tasks, novel activity, and sleep outcomes. The study is also tracking body sway and several other disease severity and functional impairment measures. SwanBio noted that CYGNET reached full enrollment in 2022; the findings of the study are being used to help inform PROPEL.

Earlier this year, SwanBio reported findings from a healthcare resource use investigation it undertook in order to get a better picture of the burden of AMN and the disease’s economic impact.4 Among the results of the inquiry, which was carried out via a retrospective review of insurance claims, was the observation that men with AMN aged 18 to 64 years who are enrolled in Medicare have a mortality rate 5.3 times higher and an age at death 9 years younger than men without AMN from a similar demographic group.

“The real-world data we have gathered illuminate the under-acknowledged burden of AMN: affected individuals are experiencing more comorbidities, higher rates of hospitalization, substantially more prescription fills, higher overall healthcare costs, and poorer clinical outcomes than their peers,” Tom Anderson, the chief executive officer and director of SwanBio, said in a May 2023 statement.4 “These findings demonstrate that the standard of care for AMN is not enough – these patients need and deserve more.”

REFERENCES
1. SwanBio advances to higher-dose cohort in first-in-human study of gene therapy for adrenomyeloneuropathy. News release. SwanBio Therapeutics. October 4, 2023. Accessed October 4, 2023. https://swanbiotx.com/investors-and-media/swanbio-advances-to-higher-dose-cohort-in-first-in-human-study-of-gene-therapy-for-adrenomyeloneuropathy/
2. Swanbio Therapeutics initiates first-in-human study of AAV gene therapy for adrenomyeloneuropathy. News release. SwanBio Therapeutics. December 15, 2022. Accessed October 4, 2023. https://swanbiotx.com/investors-and-media/swanbio-therapeutics-initiates-first-in-human-study-of-aav-gene-therapy-for-adrenomyeloneuropathy/
3. SwanBio presents design of innovative natural history study aimed to evolve understanding of adrenomyeloneuropathy and inform future treatments. News release. SwanBio Therapeutics. June 27, 2022. Accessed October 4, 2023. https://swanbiotx.com/investors-and-media/swanbio-presents-design-of-innovative-natural-history-study-aimed-to-evolve-understanding-of-adrenomyeloneuropathy-and-inform-future-treatments/
4. SwanBio Therapeutics confirms substantial burden of disease of adrenomyeloneuropathy (AMN) in both men and women. News release. SwanBio Therapeutics. May 10, 2023. Accessed October 4, 2023. https://swanbiotx.com/investors-and-media/swanbio-therapeutics-confirms-substantial-burden-of-disease-of-adrenomyeloneuropathy-amn-in-both-men-and-women/
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