UniQure stock doubled Tuesday after the company reached a testing agreement with the Food and Drug Administration for its Huntington's disease treatment.
The biotech company is testing a gene therapy called AMT-130 in patients with Huntington's, a genetic brain disorder that causes nerve cells to break down. This leads to a decline in thinking and ability to move.
After meeting with the FDA, UniQure said it could snag an accelerated approval if AMT-130 shows a benefit on natural history of Huntington's as well as improvement on a scale called the composite Unified Huntington's Disease Rating Scale, or cUHDRS. An accelerated approval allows a drug to launch early on promising test results. The company must continue testing the drug.
The news is also a boon to PTC Therapeutics, which just inked a deal with Novartis for its Huntington's disease drug.
"We think it does signal some openness overall to what might be a 'middle ground,' modestly faster registrational path for HD drugs," RBC Capital Markets analyst Brian Abrahams said in a report.
UniQure stock skyrocketed 109.7%, closing at 15.30. PTC stock jumped 4% to 49.99.
UniQure Stock: Preparing To File
The accelerated approval pathway significantly fast forwards UniQure's plans. The company won't need to run another study before asking for approval of AMT-130.
"We have initiated BLA (biological license applications) readiness activities and look forward to further engaging with the FDA in the first half of 2025 to discuss our statistical analysis plan and the technical CMC (chemistry, manufacturing and controls) requirements," UniQure said in its news release.
The news potentially benefits PTC as well. Its drug, PTC518, works by lowering levels of HTT, a protein that becomes mutated in Huntington's disease. Novartis recently agreed to pay $1 billion up front and up to $1.9 billion in milestones to license PTC's drug.
It could be a "stretch" for PTC to gain an accelerated approval solely based on whether PTC518 can knock down mutant HTT protein, RBC's Abrahams said.
But "we believe some combination of the ongoing ph. I/II (PTC518) clinical data with longer follow-up and more (patients), plus perhaps initial/interim ph.III data, could potentially be compared to natural history to support an accelerated approval path without requiring full ph.III results," he said.
He has an outperform rating on PTC stock, but didn't list a rating for UniQure stock.
Follow Allison Gatlin on X, the platform formerly known as Twitter, at @IBD_AGatlin.
