- UniQure has strengthened patent protections around its experimental gene therapy for Huntington's disease, as it prepares to start human testing later this year in a Phase 1/2 study.
- The Dutch biotech said Wednesday it secured two patents, one in the U.S. and one in Europe, that cover its gene therapy AMT-130. The therapy aims to silence the mutant huntingtin protein and the exon 1 protein fragment, the company's chief scientific officer Sander van Deventer said in a statement.
- Gbola Amusa, a sell-side analyst for Chardan, wrote in a May 22 research note that the newly-issued patents bolster UniQure's intellectual property on AMT-130, which he is optimistic could be a breakthrough success in Huntington's.
Most of the attention on UniQure has centered around its hemophilia B gene therapy, with an ongoing Phase 2b study producing incremental updates. But it could have another compelling treatment in Huntington's, expected to start a Phase 1/2 trial in the second half of 2019.
Amusa is especially keen on AMT-130's potential to be a best-in-class option for Huntington's patients, who currently have no approved treatment options.
Earlier this month, Amusa boosted Chardan's price target to $175 per share based on the AAV5-based gene therapy, nearly triple the biotech's current share price. He drew a parallel between Sarepta Therapeutics' progress last year in Duchenne muscular dystrophy with UniQure's potential in Huntington's.
Other analysts at banks including SVB Leerink, Cantor Fitzgerald and Cowen have voiced optimism in AMT-130, although not as strongly as Amusa, who also highlighted UniQure's IP strength as a key reason for his belief in the biotech.
While the patents may prove valuable down the line, the immediate critical test for AMT-130 will be succeeding in its first testing in humans, a huge hurdle for many drug candidates.
The early-stage trial will aim to randomize 25 patients among two doses, testing a range of efficacy endpoints including clinical parameters, quantitative motor function, biomarkers and patient-reported outcomes, according to a company presentation from this month.
UniQure isn't alone, however, in progressing experimental therapies for Huntington's patients. Ionis Pharmaceuticals and Roche, BioMarin Pharmaceutical and Wave Life Sciences are testing antisense oligonucleotides to silence the mutant huntingtin protein. Voyager Therapeutics and Spark Therapeutics also have gene therapies with the same goal.
Ionis and Roche published data earlier this month in the New England Journal of Medicine for a Phase 1/2a study of early Huntington's patients that showed dose-dependent reductions in the concentration of the mutant huntingtin protein. Those results were first disclosed in a topline readout last spring. The companies have started testing the higher dose of their drug, RG6042, in a Phase 3 study.
Even with competition, if AMT-130 is successful, UniQure could stand apart with a one-time gene therapy instead of an antisense therapeutic that would be taken chronically for life.
UniQure's stock has more than doubled in value this year, opening Wednesday at $63.