uniQure (NASDAQ: QURE ) reported first-quarter financial results on Tuesday. The transcript from the company's first-quarter earnings call has been provided below. This content is powered by Benzinga APIs. For comprehensive financial data and transcripts, visit https://www.benzinga.com/apis/ . The full earnings call is available at https://edge.media-server.com/mmc/p/f2xsw8zj/ Summary uniQure reported an increase in revenue to $3.6 million for Q1 2026, driven by license revenue, while research and development expenses decreased to $29.2 million due to reduced program spending and staff costs. The company is advancing its AMT130 program for Huntington's disease, with a Type B meeting with the FDA planned to discuss study design and statistical analysis, and a marketing authorization application in the UK expected in Q3. uniQure discontinued development of AMT162 for SOD1 ALS due to safety concerns, but is progressing with AMT260 for epilepsy and AMT191 for Fabry disease, with promising early data and plans for further FDA engagement in the second half of 2026. The company maintains a strong balance sheet with $586.6 million in cash and investments, projecting sufficient funds to support operations into the second half of 2029. Management emphasized commitment to regulatory engagements, advancing the pipeline, and exploring international opportunities for AMT130, highlighting ongoing efforts in the UK and potential early access programs in other regions. Full Transcript Liz (Conference Operator) Thank you for standing by. My name is Liz and I'll be your conference operator today. At this time I would like to welcome everyone to the UNICURE first quarter 2026 earnings call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press STAR followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one again. Thank you. I would now like to turn the call over to Kiara Russo, Senior Director of Investor Relations. Please go ahead. Kiara Russo (Senior Director of Investor Relations) Good morning and thank you for joining us for uniQure's first quarter of 2026 earnings call. Earlier this morning, Unicure released its financial results for the first quarter of 2026 and our press release is available on the Investors and Media section of our websiteat unicure.com Our 10Q was also filed with the SEC earlier today. Joining me on the call this morning are Matt Capista, Chief Executive Officer, Dr. Waleed Abi, SAB Chief Medical Officer, Kylie O'Keefe, Chief Customer and Strategy Officer and Christian Klempt, Chief Financial Officer. After our formal remarks, we'll open the call up for Q and A. Before we begin, please know that we will be making forward looking statements during this investor call. All statements other than statements of historical fact are forward looking statements. They are based on management's beliefs and assumptions and on information available to management only as of the date of this conference call. Our actual results could differ materially from those anticipated in these forward looking statements for many reasons, including without limitation, the factors described in Unicure's most recent SEC filings. Given these risks, you should not place undue reliance on these forward looking statements and we assume no obligation to update these statements even if new information becomes available in the future. Now let me introduce Matt Capista, Unicure's CEO. Matt Capista (Chief Executive Officer) Thanks Kiara. Good morning and thank you for joining us today. During the first quarter of 2026, Unicure remained focused on advancing AMT130 to patients while continuing to execute across our broader pipeline. Following our Type A meeting with the FDA in January, we acknowledged the agency's feedback and remain focused on engaging constructively to find a feasible path forward. We have since been granted a Type B meeting with the FDA later this quarter where we plan to discuss our proposed statistical analysis plan for the four year data expected in the third quarter and key elements of a new clinical study. In parallel, we are progressing toward a potential regulatory Submission in the United Kingdom. Following a successful pre submission meeting with the UK mhra, we are preparing to submit a Marketing Authorization application in the third quarter based on the three year data. Taken together, these efforts reflect our commitment to advancing AMT130 globally with urgency beyond Huntington's disease. We continue to make progress across our pipeline for AMT260 and refractory mesial temporal lobe epilepsy. Enrollment in our phase 1.2A study is on track and we expect to report data from the first cohort in the second quarter in Fabry disease. Updated data from our AMT191 program showed sustained and dose dependent increases in alpha Gal A activity, stable LysoGB3 levels and the discontinuation of enzyme replacement therapy in 11 patients, supporting the potential of AMT191 as a meaningful treatment option. Regarding AMT162 and SOD1 ALS, we announced our decision to discontinue development following a comprehensive review of the available data reflecting our disciplined, data driven approach to capital allocation. Looking ahead, Key milestones include our Type B FDA meeting later in the second quarter, clinical update from AMT 260 in the second quarter, the four year AMT 130 data analysis in the third quarter and the planned MAA submission for AMT 130 in the UK in the third quarter. We believe these milestones represent important opportunities to advance our programs and demonstrate the potential of our platform. In summary, we are executing with focus advancing our lead program through important regulatory interactions and and managing our strong balance sheet to support our long term strategy. We remain committed to delivering on the promise of gene therapy for patients and creating durable value for shareholders. With that, I'll turn the call over to Waleed to provide more information on the pipeline. Waleed Abi Thank you Matt. Good morning and good afternoon everyone. I'll start with AMT130 in Huntington's disease. As Matt noted, we continue to engage with the FDA and have a Type B meeting scheduled for later in the second quarter. Our goal is to work through the key design considerations for a potential new clinical study addressing the Agency's concern for an adequate and well controlled trial while also ensuring the approach is practical, feasible and appropriate in a rare, slow progressing neurodegenerative disease. Huntington's Disease is supported by one of the most robust natural history resources in rare diseases. Enroll HD includes more than 30,000 participants and provides high quality longitudinal clinical data collected over many years. Through the extraordinary efforts of the Huntington's disease community, we believe this body of real world evidence can inform efficient and statistically rigorous study designs and it should be considered as we evaluate with the agency the appropriate design of an adequate and well controlled study for a one time administered therapy. Additionally, we plan to solicit feedback on our statistical analysis plan for the four year Phase one. Two study data expected in the third quarter. Turning now to our ex US regulatory efforts, we held a successful pre submission meeting with the UK MHRA earlier this quarter. Based on this interaction, we plan to submit a marketing authorization application for AMT130 in the third quarter of this year supported by our three year clinical data analysis. This is an exciting potential milestone for UNICURE and the Huntington's disease community as we look to bring AMT130 to patients around the world. We have also started engaging with regulatory authorities in Europe and are evaluating additional opportunities internationally potentially bring AMT130 to patients as quickly and efficiently as possible. Finally, we expect a manuscript for our complete three year analysis to be submitted in a peer reviewed medical journal this year. Moving on to the rest of our clinical stage pipeline, starting with AMT260 for temporal lobe epilepsy, we continue to collect data on the fully enrolled first dose cohort which included those with both non dominant and dominant hemisphere mtle and we plan to provide an update later in the second quarter on all six treated patients with at least six months of safety, tolerability and seizure frequency outcomes. These are expected to be presented at the Epilepsy Foundation Pipeline conference in Leesburg, Virginia in June of this year. Turning to AMT 191 for the treatment of Fabry disease in February, we reported preliminary safety and exploratory efficacy data from 11 patients in the ongoing Phase1.2 trial of AMT191. As of January 8th of this year, the study cutoff date, all 11 patients across three dose cohorts demonstrated elevated alpha gal A enzyme activity that was dose dependent and durable over the observed follow up period ranging from more than one year and the longest follow up patient at the high dose to four months in a patient treated at the mid dose. Stable Plasma LysoGV3 levels were maintained post dose across all dose cohorts regardless of enzyme replacement therapy status. As of February 18th of this year, all 11 dose patients have discontinued ERT on safety as previously reported, two patients in the mid dose cohort experienced asymptomatic grade 3 liver enzyme elevations. These events met protocol defined criteria for protocol for potential dose emitting toxicity and were reviewed and confirmed as such by the independent data Monitoring Committee. Accordingly, DOSING at the mid dose and high doses were paused per protocol. To date, no new AMT 191 related serious adverse events have been observed and the program continues to demonstrate a manageable safety profile. Lastly, there's AMT162 for sod1als. As previously disclosed, the phase one. 2 Episode 1 trial of AMT162 for sod1als has been on voluntary recruitment pause based on an independent data monitoring committee recommendation after a serious adverse event of dorsal root ganglia toxicity in one patient in the second cohort. This event was determined to be related to AMT162. Following review of the preliminary efficacy and safety Data generated from episode one, the decision was made to discontinue development of AMT162. We will continue to collect follow up safety from the five patients dosed consistent with applicable safety and regulatory requirements. Now I will turn the call over to Kylie to discuss our ongoing work with the HD community and our XUS commercial efforts. Kylie O'Keefe (Chief Customer and Strategy Officer) Thank you Waleed Before I turn to AMT 130, I want to take a moment to acknowledge the Huntington's disease community, the patients, the families and the caregivers who live with this disease every day, and the researchers, clinicians and advocates who have spent decades refusing to accept the status quo. Their resilience and their trust in us is not something we take lightly. It is what holds us accountable to the progress we are here to discuss today. We remain committed to continuing the efforts in the US and globally to advance AMT130 as responsibly and efficiently as possible. We are encouraged by the path ahead in the UK following our recent engagement with the MHRA and our advancing commercial preparations across several key geographies. Based on this progress, our market preparation efforts have centered on three near term priorities. First, ensuring treatment center capacity and readiness and working closely with the multidisciplinary care teams at the Centers of Excellence that will be critical to success. Secondly, in parallel, ongoing patient engagement is critical to maintain continuity across the care journey, supporting genetic testing and referral pathways. Thirdly, market access readiness is advancing including proactive payer engagement and development of a clear evidence based value proposition. This is underpinned by robust health economics and outcomes research, generating data to demonstrate long term clinical benefit and broader societal impact to support pricing, access and adoption. We believe the UK unlocks a meaningful opportunity for unicure and to deliver a potentially transformational therapy to patients with hd. A community with no approved disease modifying therapies. Today There are between 7,000 to 8,000 patients living with HD in the UK with approximately 30,000 at risk. The UK has world renowned neurosurgical capabilities and several leading HD Centers of Excellence which we believe will be instrumental partners in making this potential therapy available to patients. Importantly, an MHRA approval would not only enable access in the uk, we believe it could also enable early access or name patient programs in other geographies including the Gulf countries in the Middle East, Latin America, Commonwealth of Independent States and Central and Eastern Europe, enabling access to therapies ahead of formal reimbursement decisions offering hope to patients and families. While local regulatory and broader market access processes continue. We believe this disciplined approach helps drive building a scalable global strategy to maximize the long term value of our program for all stakeholders. Moving to AMT260 in mesial temporal lobe epilepsy where many patients remain completely refractory to anti seizure medications cycling through treatment after treatment with no meaningful seizure control. For those who do progress to surgical intervention, the options currently available are at its core a tissue destructive procedure. We believe being able to deliver a precisely targeted gene therapy in MTLE ... Full story available on Benzinga.com
Transcript: uniQure Q1 2026 Earnings Conference Call
