A month after sparking optimism for patients with Huntington’s disease with highly positive data for AMT-130, uniQure revealed Monday that the FDA may be changing its tune on the evidence required for an approval application, a change of direction Stifel called “very challenging” for uniQure.
Five weeks after reporting highly positive three-year data from its investigational gene therapy for Huntington’s disease, uniQure has run into a possible snag in its plans to file a biologics license application for the treatment early next year.
The Massachusetts and Amsterdam–based biotech received feedback from the FDA during a recent pre-BLA meeting, leading it to believe the agency “no longer agrees” that the Phase I/II data for the treatment, called AMT-130, will be “adequate to provide the primary evidence in support of a BLA submission,” according to a press release issued Monday.
That’s despite the company having worked with the agency to settle on the protocols and statistical analyses used, uniQure continued. This therefore represents a “key shift” from multiple Type B meetings with the FDA over the past year, the company said in its announcement. “Consequently, the timing of the BLA submission for AMT-130 is now unclear.”
In a note to investors Monday morning, Stifel analysts called the FDA’s “about face” on the filing path “very surprising; very challenging for [uniQure] and [a] possible overhang for the space.”
The company’s announcement that the FDA no longer views the Phase I/II data, which compares treated patients to an external control, as the primary basis for AMT-130’s BLA filing “is a shock, and we believe it’s going to pressure QURE shares substantially and potentially the shares of other development stage companies in the gene therapy space,” the analysts wrote.
UniQure’s stock fell to just over $26 as the markets opened on Monday, compared to Friday’s closing price of $67.49.
In a statement on Monday, uniQure CEO Matt Kapusta also expressed his company’s surprise at the turn of events. “We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024,” Kapusta said.
The announcement comes just over a month after uniQure sparked a new wave of excitement in the Huntington’s community, announcing that three-year data from a pivotal trial of AMT-130 showed 75% slowing of the intractable neurodegenerative disease. This exceeded Stifel’s hopes for a 60% slowing of disease, the analyst wrote at the time, and was “largely consistent” with the 80% slowing seen at the two-year mark in the same trial.
Shares of the company exploded, rising nearly 250% that day.
Despite the celebration, experts such as Ignacio Munoz-Sanjuan, CEO and chairman at Rumi Scientific and president of the Huntington’s-focused non-profit Factor-H, have urged caution in interpreting the results. In a September article, Munoz-Sanjuan pointed to the low number of participants who completed three years in the trial, writing that “with such a small number of individuals receiving the therapy, one or two people could be disproportionately contributing to the signals of protection demonstrated for the group average.”
In a subsequent interview last month with BioSpace, Munoz-Sanjuan cautioned scientific leaders against communicating the results in a way that generates unrealistic expectations at this stage of clinical development.
UniQure expects to receive final minutes from the pre-BLA meeting within 30 days, at which time it will “urgently interact with the FDA to find a path forward for the timely accelerated approval of AMT-130,” according to the Monday announcement.
“The next steps here are uncertain,” Stifel wrote Monday, “but given the highly mobilized patient community in Huntington’s, and other external political forces that may have influence on the agency, the story here may not be over.”
