After Historic Hemophilia B Approval, uniQure Targets Huntington’s and ALS

Courtesy of uniQure

2022 was a big year for uniQure with the U.S. and European approval of world's first gene therapy for hemophilia B. In its 2022 financial report Monday, the Lexington, Mass. and Amsterdam-based company provided updates on the progress of its Huntington’s disease and ALS programs. 

uniQure, along with partner CSL Behring, made history in November 2022 when Hemgenix was approved in the U.S.

Hemgenix, now also conditionally approved in Europe, tops the charts as the most expensive medicine in the world. Under the terms of its partnership with CSL, uniQure supplies the therapy from its Lexington, Mass. manufacturing facility.

The company became eligible for a $100 million payment from CSL upon its first U.S. sale. Another $75 million will follow if a sale in one of five countries in Europe is seen by mid-summer.

The cash infusion should be well-spent on uniQure’s next big target – a gene therapy for Huntington’s disease.

Huntington’s has been a notoriously difficult indication for biopharma. Despite its absolute fatality and lack of therapies to treat or even slow progression, it remains thus far untouchable.

Earlier this month, Novartis dropped its program after neurotoxicity signals occurred in the majority of trial participants. A failure in the space led Triplet Therapeutics shut down last fall.

uniQure’s AAV gene therapy approach is currently being tested in a Phase I/II study. Twenty-six patients are currently enrolled in either a lower or higher dose cohort arm.

An AAV5 vector carries mRNA tailored to silence the huntingtin gene, inhibiting production of mutant protein mHTT. Preclinical studies showed a single dose of AMT-130 can lead to improvement in brain cell function, disease neuropathology reversal and a partial reversal of hippocampus volume loss.

AMT-130 has been granted Orphan Drug and medicinal designations from the FDA and EMA.

The biotech is also expanding its focus areas to include a new target. In Jan. the company picked up an ALS gene therapy hopeful from Apic Bio. The one-time therapy utilizes a recombinant AAVrh10 vector to reduce the expression of superoxide dismutase 1 (SOD1) to slow or reverse progression of the disease.

AMT-162 already has a cleared IND. uniQure expects to initiate a Phase I/II study of its new asset in the second half of 2023.