The U.S. Food and Drug Administration (FDA), during a Type B meeting, accepted the 3-year Phase I/II data as the basis for an application for accelerated approval of uniQure’s AMT-130 therapy. According to representatives of the multiQure project, this is an important signpost for all gene therapies – not only those related to Huntington’s disease, but also other genetically driven neurodegenerative diseases, including multiQure.
The FDA’s Change in Approach and Its Impact on Gene Therapies
Gene therapies represent a major opportunity for hundreds of thousands of patients suffering from genetic diseases, including Huntington’s disease. Commonly used treatments only alleviate symptoms without halting the disease. Gene therapies based on RNAi technology make it possible to address the root cause of the condition, which is why their introduction into clinical practice has long been awaited by patients, their families, and the medical community.
Currently, the most advanced RNAi therapy in development for Huntington’s disease is AMT-130, the program of the Dutch, NASDAQ-listed company uniQure. It is currently in Phase I/II clinical trials and, according to recent reports, is preparing to submit a Biologics License Application (BLA) to the FDA under a conditional approval pathway. On 17 June this year, following a formal meeting with the FDA, uniQure announced that the agency had accepted the 3-year Phase I/II data as an acceptable basis for filing under the accelerated approval pathway, planned for the third quarter of this year. The market reacted to the news with great enthusiasm. On the day the news was announced, uniQure’s share price rose by nearly 80%, and its market capitalization exceeded USD 3 billion.
An Opportunity for the multiQure Project
According to the head of the multiQure project, whose goal is to develop an RNAi-based therapy that would compete with AMT-130, this is an important development for organizations working on gene therapies.
– The FDA’s position on uniQure’s AMT-130 therapy is a landmark moment not only for organizations conducting R&D in gene therapies for Huntington’s disease, but also for the broader field of research into drugs for genetic neurodegenerative diseases. The Food and Drug Administration has confirmed that three years of data from early clinical phases can constitute a sufficient basis for seeking accelerated approval of a therapy – and this fundamentally changes the risk calculus for our project, and therefore also for investors and potential partners – says Tomasz Nocuń, head of the multiQure project.
According to Tomasz Nocuń, it is particularly important for the multiQure project that the U.S. regulator has accepted the possibility of using comparative data sets from observational clinical studies of Huntington’s disease.
– This is a concrete facilitation that will allow us to design our own clinical trial more effectively. Importantly, the multiQure project starts from a potentially stronger scientific position: our therapy targets selectively – only the production of the mutant version of huntingtin – which represents a significant advantage over the non-selective approach represented by uniQure. Their path through the clinical trial, however, shows that the concept of gene therapy in this condition brings patients real benefits. Our task now is to follow a path that has already been partly cleared, with a therapy distinguished by a better safety profile. – says Tomasz Nocuń.
The impact of the FDA’s position toward uniQure on other gene therapies, primarily multiQure, has been assessed and commented on by Dom Maklerski INC (INC Brokerage House). The authors of the commentary point above all to the significant similarities between the two projects, the possibility of following the path already cleared by uniQure, and the advantages of the Polish project over AMT-130 stemming from the selectivity of its therapy.
– uniQure’s positive clinical data and the FDA’s constructive approach provide external validation of the logic underlying multiQure’s strategy. At the same time, multiQure’s technology may stand out for its preferential effect on the mutant transcript containing the expanded CAG repeat sequence, with a potential reduction in impact on the normal copy of the HTT gene, which may be significant from a biological safety standpoint – comments Dr. Maciej Kietliński, analyst at Dom Maklerski INC, author of the analytical report on the multiQure project.
multiQure’s Stock Market Plans
uniQure’s success and the market’s reaction show how significant an impact the achievement of milestones – and the related regulatory positions and decisions – can have on the valuation of biotechnology companies. This is why the management and investors of the multiQure project intend to take the project public, enabling investors to participate as the company reaches its next milestones.
– We are pursuing efforts to take multiQure public, so that Polish investors, too, can gain exposure to a venture focused on developing a gene therapy for Huntington’s disease – one that is potentially more effective and safer than uniQure’s. Earlier this week we signed an agreement to contribute the project to Pure Biologics and rename it multiQure. We are in the final stretch of this process – comments Dr. Kris Siemionow, investor in the multiQure project, co-founder and shareholder of Biofund.
The process of listing the project on the Warsaw Stock Exchange (GPW) is being consistently carried out. In addition to signing the agreement to contribute the project to Pure Biologics (to be renamed multiQure S.A.), the company’s shareholders have also decided on a share issue, through which funds are planned to be raised to allow the multiQure team to administer the therapy to the first patients – expected within the next 12–18 months – and to obtain initial results by mid-2028.
Summary
The FDA’s position conveyed to uniQure sets a new standard for the entire gene therapy sector, confirming that a regulatory pathway is crystallizing for this class of drugs. For the multiQure project, this means not only a reduction in regulatory risk, but also external confirmation of the soundness of its chosen scientific strategy – alongside a safety advantage stemming from the therapy’s selectivity. Given that work is underway to bring the project to the stock exchange, Polish investors will soon gain exposure to a best-in-class gene therapy for Huntington’s disease.