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Shivendu Jauhari
As uniQure prepares its closely watched Huntington’s disease gene therapy for FDA review, the biotech is also making waves in chronic epilepsy with another gene therapy that Stifel says is off to a “promising start.”
As uniQure’s closely watched Huntington’s disease asset nears its FDA submission, uniQure is lining up another investigational asset—this time for a form of chronic epilepsy, demonstrating robust drops in seizure frequency and an overall encouraging safety profile.
The company tested its investigational therapy AMT-260 in the Phase 1/2a GenTLE trial, which enrolled patients with refractory mesial temporal lobe epilepsy (MTLE). Results,
presented Friday
, come from six patients in the low-dose cohort, with three patients demonstrating a 79% to 100% decline in disabling seizures from baseline during four to six months of follow-up. The other three patients experienced “variable changes” in disabling seizures, ranging from a 33% decrease to a 36% increase.
While cautioning that these data come from “a small open-label dataset,” Stifel on Friday told investors that the findings represent “a promising start” for uniQure and AMT-260 “as we may see better efficacy in the high-dose cohort.”
GenTLE
is currently enrolling patients for this group—with an expected total recruitment of 12 patients—and has a primary completion date of Nov. 30.
“We note that these refractory patients are unlikely to show a response by chance,” Stifel continued in its note, while conceding that such an early and small program is still risky. Still, the analysts concluded, “this program should be on investors’ radar after UCB’s acquisition of Neurona.”
The analysts were referring to an April deal in which UCB
put down $650 million upfront
to absorb Neurona Therapeutics and its star asset NRTX-1001, a cell therapy being tested for drug-resistant unilateral and bilateral MTLE. NRTX-1001 in December showed an
89% median decrease in debilitating seizures
seven to 12 months after dosing.
But even as AMT-260 shows potential in epilepsy, Stifel on Friday noted that for investors, “focus remains on upcoming [biologics license application] submission for AMT-130,” uniQure’s gene therapy for Huntington’s disease. Last week, the biotech announced that it was
planning to submit
an application in the third quarter.
Huntington’s disease
UniQure plans Q3 submission for Huntington’s gene therapy after FDA reverses course—again
After a regulatory odyssey
that delayed a filing for what would be the first genetic medicine for Huntington’s disease, the FDA has agreed that three-year data from uniQure’s Phase 1/2 trial are sufficient to support an accelerated biologics license application.
June 17, 2026
·
4 min read
·
Heather McKenzie
Read more
It’s been a long road for AMT-130. In November last year, the FDA
said
that it “no longer agrees” that a Phase 1/2 study would be “adequate to provide the primary evidence in support of a BLA submission.” According to uniQure, the biotech had previously aligned with the regulator on the design of its development program for AMT-130.
In February, then–FDA Commissioner Marty Makary
spoke to
CNBC
to defend Vinay Prasad, who was at the time director of the Center for Biological Evaluation and Research. In the interview, Makary singled out a drug candidate that matched AMT-130’s description: “There was a product where the researchers drilled a burr hole, literally a hole, in people’s skulls” to deliver the therapy directly into the brain, he said. “At the end of the randomization period, it was found no benefit, and yet this is one of the drugs that we were pressured to approve.”
A few weeks later, an unnamed FDA official
blasted uniQure
—which by that point had been raising ethical concerns about the need to run a sham-controlled study—arguing that “we did not ask them to compare against a partial sham burr hole.” Instead, the official said, the company would just need to “anesthetize the patient and put one to three nicks in the scalp.”
Now, though, the agency has appeared to reverse course yet again. Following the departure of Makary and Prasad, the FDA has
indicated
that it will no longer require uniQure to use a sham surgery for its confirmatory study of AMT-130, instead mulling the use of standard-of-care control. The agency wants to first align on the design of the confirmatory trial before accepting uniQure’s biologics application.
