- Preliminary results from the low-dose cohort in a Phase 1/2 clinical trial assessing uniQure's (NASDAQ:QURE) AAV5/FIX gene therapy, AMT-060, in patients with moderately severe to severe hemophilia B showed a significant clinical benefit in two participants who had completed at least 12 weeks of follow up and an initial treatment benefit in two more who had not completed the full follow up period (out of three).
- All five subjects had documented Factor IX (FIX) levels less than 1 - 2% and required chronic prophylactic treatment with recombinant FIX (rFIX) at the time of enrollment (results from an earlier study performed at St. Jude Children's Hospital showed that a durable FIX expression of ~5% of normal produced long-term clinical benefits). AMT-060 was administered through a peripheral vein in one treatment session lasting ~30 minutes. There are two treatment arms: low dose and high dose. To date, there have been no patient screening failures due to pre-existing neutralizing antibodies against AAV5 and no patients have developed inhibitory FIX antibodies.
- The two participants who completed at least 12 weeks of follow up showed FIX expression levels of 5.5% and 4.5%, respectively. Three additional subjects had been dosed but had not achieved the full 12 weeks of follow up. Two of the three, though, met a secondary endpoint of discontinuing prophylactic rFIX based on their FIX expression levels.
- The company intends to present a more complete analysis of the data at a scientific conference in Q2. Assuming Data Monitoring Committee approval, enrollment in the high dose cohort will commence this quarter.
- Management will host a conference call this morning at 8:30 am ET to discuss the data.