CSL has a new ray of hope in its research pipeline, with an experimental haemophilia drug it acquired last year cleared of any links to cancer following a scare in its clinical trial.
CSL entered a $US450 million ($592 million) deal in June 2020 for the global distribution rights of an experimental gene therapy being made by Nasdaq-listed biotech UniQure. The treatment, called AMT-061, is hoped to be a revolutionary therapy for haemophilia B patients. It’s designed to boost Factor IX, the blood clotting protein that those with haemophilia B lack, after just one single use.
CSL chief Paul Perreault had championed the product as a strong addition to the company’s global treatments portfolio, saying it would be a game changer for patients. “Our vision with haemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,” he said last June.
CSL’s chief scientific officer Andrew Nash told investors at the company’s research and development day in October that UniQure’s research was “a very nice program” and showed early promise helping patients.
UniQure was in phase 3 trials for the product and had released strong data at the end of last year showing participants were responding positively. But just weeks later, the US Food and Drug Administration put a hold on the drug’s clinical trial after a participant was diagnosed with hepatocellular carcinoma, a type of liver cancer.
The news sent shockwaves across the gene therapy research sector in the US, where several other companies are working in a similar field. UniQure’s share price slid 25 per cent in the days following the news.
However, UniQure this week released details of a multi-faceted investigation into the patient, concluding it was “highly unlikely” there was a link between the cancer and the company’s product.
UniQure said an independent lab had reviewed the patient’s tissue sample and had not found any evidence of a link. Instead, genomic testing found the individual had genetic risk factors as well as lifestyle factors such as smoking that could have contributed to the cancer.
UniQure chief executive Matt Kapusta said the trial was yet to restart, but the findings had been passed on to the US regulator.