uniQure (NASDAQ:QURE) is on track to read out data from its pivotal trial of AMT-061 for patients with hemophilia B. Given the gene therapy appears safe, the company can complete the pivotal trial and eventually file for a regulatory approval. The closing of the deal with CSL Behring may provide significant cash flow to fund the pipeline. uniQure remains a high-risk, high-reward investment due to the Huntington's disease program.
AMT-061 appears to have a favorable safety profile
uniQure released findings regarding their investigation into the cause of hepatocellular cancer that was diagnosed in a patient in the HOPE-B gene therapy trial for patients with hemophilia B. The company concluded that its gene therapy for hemophilia B is not likely to be related to the patient developing hepatocellular cancer. The patient who developed the cancer had multiple risk factors including hepatitis B and C infections which are associated with 80 percent of all hepatocellular cancers.
According to a statement released by uniQure, "The external lab analyzed more than 220,000 cells from the tissue sample and identified 60 cells with random integration events that have no known association with the development of HCC. Moreover, whole genome sequencing of the tumor showed that this patient had large abnormalities on chromosomes 1 and 8 that are commonly associated with HCC, as well as mutation of TP53 and several other potentially oncogenic genes." Discussions with the FDA about these findings should occur in Q2 and based on this data, uniQure seeks to have the clinical hold lifted.
The clinical hold placed on the HOPE-B trial due to the hepatocellular cancer case resulted in QURE's stock falling significantly in December. QURE shares had been trading in the high $40 range and fell as low as $28 as investors waited for a resolution to the issue. bluebird bio (
