uniQure NASDAQ: QURE executives outlined progress and upcoming regulatory milestones for lead Huntington’s disease gene therapy candidate AMT-130 during the company’s first-quarter 2026 earnings call, while also providing updates across its epilepsy and Fabry programs and reviewing quarterly financial results.
AMT-130 regulatory strategy centers on FDA engagement and a planned U.K. filing
Chief Executive Officer Matt Kapusta said uniQure remains focused on advancing AMT-130 “globally with urgency,” while continuing discussions with U.S. regulators following a Type A meeting with the FDA in January. Kapusta said the company has been granted a Type B meeting with the FDA later in the second quarter, where uniQure plans to discuss a proposed statistical analysis plan for data expected in the third quarter, as well as “key elements of a new clinical study.”
Chief Medical Officer Dr. Walid Abi-Saab characterized the upcoming Type B meeting as “a technical meeting,” aimed at clarifying “key design elements of an additional new study to evaluate the efficacy of AMT-130” and obtaining FDA feedback on the statistical analysis plan for the phase I/II study data expected in the third quarter. When asked whether uniQure would discuss an alternative regulatory path that might avoid initiating a new study, Abi-Saab said, “We do not intend to have a specific discussion about a regulatory path to filing at this point.”
In parallel, the company is advancing an ex-U.S. strategy. Abi-Saab said uniQure held a “successful pre-submission meeting” with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and plans to submit a marketing authorization application (MAA) for AMT-130 in the third quarter, supported by its three-year clinical data analysis. He added the company has begun engaging with European regulators and is “evaluating additional opportunities internationally” to pursue access efficiently.
Kapusta discussed the company’s view of the strength of its AMT-130 dataset when responding to a question about differences between the FDA and MHRA interactions. He said uniQure believes it has “achieved 75% slowing of disease with high statistical significance out to three years on the composite UHDRS,” along with “statistical significance in a slowing of disease on Total Functional Capacity,” favorable trends across other measures, and neurofilament light “below baseline.” Abi-Saab added that rare disease policy focus in the U.K. “allows for more flexibility to be afforded in rare diseases like this,” while noting that engagement with the FDA has become “a bit more difficult” more recently.
Four-year AMT-130 dataset expected in Q3; company anticipates effects become more evident over time
Abi-Saab said the four-year analysis, expected in the third quarter, will generally mirror the three-year analysis with an additional year of follow-up. He said the dataset is expected to include 12 high-dose and 12 low-dose patients with four years of follow-up, plus three additional high-dose patients reaching three years (15 high-dose patients at the three-year timepoint). Abi-Saab said uniQure is discussing with the FDA whether additional analyses could “increase the level of confidence,” adding that the company expects treatment effects to become more apparent over time, particularly for high-dose patients compared with “well-matched external controls.”
Executives repeatedly emphasized the role of real-world evidence for trial design. Abi-Saab highlighted the Enroll-HD resource, which he said includes more than 30,000 participants and provides longitudinal clinical data that could inform efficient and statistically rigorous designs for a one-time therapy in a slow-progressing disease.
Commercial and access planning emphasizes treatment centers, patient engagement, and payer readiness
Chief Customer and Strategy Officer Kylie O’Keefe said the company is advancing commercial preparations in several geographies based on progress with the MHRA. She described three near-term priorities:
- Ensuring treatment center capacity and readiness in collaboration with multidisciplinary care teams at centers of excellence.
- Maintaining continuity in the patient journey through engagement efforts that support genetic testing and referral pathways.
- Advancing market access readiness through proactive payer engagement and an evidence-based value proposition supported by health economics and outcomes research.
O’Keefe said there are “between 7,000-8,000 patients living with HD in the U.K., with approximately 30,000 at risk,” and told analysts that “the vast majority” of diagnosed patients are treated at specialized centers. She said that following potential MHRA approval, uniQure expects to work with NICE and other access bodies, including NHS England, to pursue managed access agreements.
When asked about U.K. capacity and procedural readiness, O’Keefe said uniQure does not anticipate changes to the AMT-130 surgical procedure when moving from clinical to commercial use. She also said the cannula used in the procedure has already been shipped to multiple countries and that the U.K. “is no issue,” adding that other companies already use the cannula commercially in the U.K.
On broader ex-U.S. access, O’Keefe said uniQure aims to begin with named patient and early access programs and then pursue formal pricing and reimbursement. She cited the U.K.’s “Highly Specialized Technology” route and evolving cost-effectiveness considerations as signs of a shifting approach to advanced therapies. She also said the company is evaluating countries on a market-by-market basis, referencing Germany’s initial free-pricing period, France’s early access pathways, and named patient approaches in countries such as Italy. O’Keefe also said an MHRA approval could enable early access or named patient programs in other regions, including Gulf countries in the Middle East, Latin America, the Commonwealth of Independent States, and parts of Central and Eastern Europe.
Pipeline updates: AMT-260 data coming in Q2; AMT-191 dosing continues at low dose; AMT-162 discontinued
Beyond Huntington’s disease, Kapusta and Abi-Saab highlighted several pipeline updates. For AMT-260 in refractory mesial temporal lobe epilepsy, Kapusta said enrollment in the phase I/IIa study is on track, with data from the first cohort expected in the second quarter. Abi-Saab said uniQure plans to provide an update later in the second quarter on six treated patients with at least six months of safety, tolerability, and seizure frequency outcomes, with results expected to be presented at the Epilepsy Foundation Pipeline Conference in June. He said the trial’s primary objective is safety, while seizure frequency is a key exploratory endpoint; he added that the company would view “perhaps a 50% reduction in seizure frequency” as a meaningful signal to pursue in subsequent controlled studies.
For AMT-191 in Fabry disease, executives reiterated previously disclosed data from 11 patients across three dose cohorts. Abi-Saab said patients demonstrated dose-dependent and durable increases in alpha-galactosidase A activity, with stable plasma lyso-Gb3 levels maintained post-dose regardless of enzyme replacement therapy (ERT) status. He also said that as of Feb. 18, all 11 dosed patients had discontinued ERT.
On safety, Abi-Saab said two mid-dose patients experienced asymptomatic grade 3 liver enzyme elevations that met protocol-defined criteria for potential dose-limiting toxicity, which the independent data monitoring committee reviewed and confirmed. As a result, dosing at the mid and high doses was paused per protocol. He said no new AMT-191-related serious adverse events have been observed to date and described the program as demonstrating “a manageable safety profile.” Abi-Saab told analysts uniQure is continuing to dose at the low dose while following affected patients closely and plans to submit the liver enzyme data to the FDA and discuss resuming dosing at the mid or high dose.
For AMT-162 in SOD1-ALS, Abi-Saab said uniQure discontinued development following a review of EPISOD1 trial data after a serious adverse event of dorsal root ganglia toxicity in one patient that was determined to be related to AMT-162. He said the company will continue safety follow-up for the five dosed patients consistent with regulatory requirements.
First-quarter financial results show higher revenue, lower R&D spending, and increased SG&A
Chief Financial Officer Christian Klemt reported first-quarter 2026 revenue of $3.6 million, up from $1.6 million in the same period of 2025, attributing the increase to higher license revenue. Research and development expenses were $29.2 million, down from $36.1 million a year earlier, driven by decreases including a $2.6 million reduction in fair value of contingent consideration, lower external program spend, reduced employee and contractor-related expenses (including share-based compensation), and lower facilities and other costs.
Selling, general and administrative expenses rose to $20.1 million from $10.9 million, which Klemt said was primarily driven by higher employee and contractor-related expenses, including share-based compensation, “mainly as a result of employees recruited in 2025 to support commercial planning for AMT-130,” along with increases in professional fees, intellectual property fees, and information technology and other expenses.
Cash, cash equivalents, and investment securities totaled $586.6 million as of March 31, 2026, compared with $622.5 million at the end of 2025. Klemt said uniQure expects its cash resources to be sufficient to fund operations into the second half of 2029. Abi-Saab later added that runway assumptions include completing ongoing clinical trials in temporal lobe epilepsy, Huntington’s disease, and Fabry disease, but that the plan does not support advancing “all the candidates in kind of the most expedited manner” simultaneously without prioritization decisions.
Looking ahead, Kapusta highlighted 2026 milestones including the Type B FDA meeting later in the second quarter, an AMT-260 clinical update in the second quarter, the four-year AMT-130 data analysis in the third quarter, and the planned U.K. MAA submission for AMT-130 in the third quarter.
About uniQure NASDAQ: QURE
uniQure N.V. is a biotechnology company focused on the development and commercialization of gene therapies for patients with severe medical needs. Using its proprietary adeno‐associated viral (AAV) vector platform, the company designs single‐dose treatments aimed at addressing the underlying genetic causes of disease rather than solely managing symptoms. Its most advanced program, Hemgenix® (etranacogene dezaparvovec), received regulatory approval in the United States and Europe for adult patients with hemophilia B, marking one of the first gene therapies for a bleeding disorder to reach the market.
Beyond hemophilia B, uniQure's pipeline includes preclinical and clinical-stage candidates targeting rare and debilitating conditions such as aromatic l-amino acid decarboxylase (AADC) deficiency, Huntington's disease, and Parkinson's disease.
Read More
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.
Before you consider uniQure, you'll want to hear this.
MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and uniQure wasn't on the list.
While uniQure currently has a Moderate Buy rating among analysts, top-rated analysts believe these five stocks are better buys.
View The Five Stocks Here
MarketBeat's analysts have just released their top five short plays for May 2026. Learn which stocks have the most short interest and how to trade them. Click the link to see which companies made the list.
Get This Free Report
