Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.
The final minutes from uniQure’s pre–biologics license application meeting with the FDA are in, and they confirm the company’s and analysts’ suspicions—it’s going to be a longer regulatory road to market than anticipated for the company’s Huntington’s disease gene therapy.
The final minutes are “consistent” with a previous update on Nov. 3. At that time, five weeks after reporting highly positive three-year data from a pivotal trial of AMT-130, uniQure shared initial feedback from this meeting, revealing that the FDA “no longer agrees” that data from its Phase I/II trials would be “adequate to provide the primary evidence in support of a BLA submission.”
In uniQure’s press release on Thursday, the company added that the agency “conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission.” UniQure is “carefully” evaluating this feedback and intends to “urgently” seek a follow-up meeting with the FDA, to take place during the first quarter of 2026.
UniQure’s stock, which fell to just over $26 on Nov. 3 from the previous closing price of $67.49, was down to just under $23 as of this writing.
Investors had not been expecting a speedy reversal of the November guidance, according to Stifel analysts.
“As time has passed since the 11/3 announcement, we think investors’ optimism has waned around a quick reversal at FDA,” they wrote in a note to investors Thursday morning.
“As we see it, there are still many scenarios on the table here,” the analysts continued, ranging from, “(1) an unfortunately challenging path forward for AMT-130--i.e. need an outright new study, to (2) some sort of compromise, i.e. more patients followed for longer, to (3) even a FDA reversal, perhaps helped by political/external pressure.”
The past four months have been a rollercoaster ride for uniQure—and for patients with Huntington’s, a genetic, neurodegenerative disease.
On Sept. 24, uniQure reported three-year data from a pivotal Phase I/II trial AMT-130, showing that the gene therapy slowed disease progression by 75%, beating analyst expectations. Plans were underway to file a biologics license application (BLA) in the first quarter of 2026, and uniQure’s shares soared 248%.
But thatwas followed by the FDA’s feedback in November—a seeming reversal of position as uniQure had previously aligned with the agency on the protocols and statistical analyses used in its trials—specifically, comparing AMT-130 to a natural history external control, according to uniQure’s announcement.
“We still view the agency’s change in guidance as very surprising given the numerous interactions uniQure has had with the FDA since its November 2024 meeting,” William Blair analysts wrote in a note to investors on Thursday. They added that uniQure’s trial design aligns with the FDA’s recent publication of a plausible mechanism pathway for personalized therapies, as well as draft guidance on gene therapies for small populations, “which specifically stipulates that external controls may be an appropriate option in certain contexts.”
If approved, AMT-130 would be the first genetic treatment for Huntington’s disease.
