NEW YORK – SwanBio on Wednesday said it has received $10 million from its lead investor and majority shareholder Syncona Investment Management, which it will use to advance a second dose-escalation cohort within the PROPEL trial of its gene therapy SBT101 in adrenomyeloneuropathy (AMN).
The Phase I/II PROPEL trial is comparing the safety and efficacy of SBT101 against an imitation procedure over two years. In the Phase I portion, researchers are assessing two doses of SBT101 across two cohorts.
An independent data safety monitoring board recently found that patients in the study had no serious adverse events when given the therapy at the initial lower dose. As such, the DSMB told SwanBio it can administer the higher dose of the gene therapy in the trial.
"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," Syncona CEO Chris Hollowood said in a statement. "Our further investment of $10 million will enable the company to deliver additional data to deliver its long-term potential."
SBT101 uses a recombinant adeno-associated virus serotype 9 vector to deliver a functional copy of the ABCD1 gene. AMN, a neurodegenerative disease that affects between 8,000 and 10,000 men in the US and certain European countries, is caused by ABCD1 mutations, which hinder spinal cord cells and other tissues from functioning normally. Patients with AMN lose mobility and experience incontinence, pain, and sexual dysfunction. There are no approved treatments for AMN.
"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," SwanBio Chief Medical Officer David Weiner said in a statement. "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."
The PROPEL trial is currently enrolling patients in the US but is expanding into Europe after having garnered permission from health authorities in the Netherlands. SwanBio is already conducting a natural history study of AMN in Europe, which completed enrollment last year and is exploring the use of wearable technologies to track disease progression.