UniQure’s stocks up after AMT-130 trial win

In the pivotal Phase I/II study, high doses of uniqure‘s Huntington gene therapy AMT-130 showed a 75% slowing of disease progression as measured by cUHDRS versus placebo after 3 years in 12 evaluable patients versus external controls. UniQure NV also announced AMT-130 achieved a statistically significant 60% slowing of disease progression as measured by TFC (p=0.033), a key secondary endpoint of the study that has been proposed by the FDA as primary endpoint in other studies. Additional measures showed favourable trends, including improvements in motor and cognitive function, and mean CSF NfL levels were 8.2% below baseline at 36 months. AMT-130 is designed to silence a gene that produces a mutant form of huntingtin that damages nerve cells. So far, AMT-130 has delivered mixed results in clinical studies, so the current study results are a real surprise.

The facility includes three tranches: a first tranche of US$50m refinancing existing debt, a second tranche of US$100m available upon achieving regulatory and financial milestones and a third tranche of up to US$25m subject to Hercules’ approval.

UniQure also announced that it has commenced a US$200m underwritten public offering of its ordinary shares and, in lieu of ordinary shares to certain investors, pre-funded warrants to purchase its ordinary shares. All securities to be sold in the offering will be offered by UniQure. In addition, UniQure intends to grant the underwriters a 30-day option to purchase up to a number of additional ordinary shares equal to 15% of the total number of ordinary shares (and ordinary shares underlying pre-funded warrants) sold in the public offering, on the same terms and conditions.

Prior to a decision about an accelerated approval, the FDA wants to see the data, UniQure stated in a press conference.