MultiQure, a project developing an RNAi therapy targeting Huntington’s disease and other polyglutamine disorders, has been valued by Baker Tilly at USD 111.3 million. At the current exchange rate, this corresponds to a value exceeding PLN 400 million. The project’s management assumes that one of the next stages of development will be to bring MultiQure to the stock exchange.
The most important objective for the coming months is to begin studies involving the first patients. According to investors, administration of the therapy to the first patient could take place within the next 12–18 months. Study results are expected to be available no later than 2028.
RNAi Therapy Aims to Target the Mechanism of the Disease
MultiQure is developing a therapeutic platform based on RNAi technology, or RNA interference. This mechanism makes it possible to silence the expression of selected genes, which may, in principle, reduce the production of proteins responsible for the development of specific genetic diseases.
In the case of Huntington’s disease, the aim of the therapy is to address the underlying cause of the condition, rather than merely relieve its symptoms. At present, treatment for patients with Huntington’s disease is primarily symptomatic and focuses, among other things, on controlling chorea, psychiatric disorders and other neurological problems.
According to representatives of the project, the technology being developed by MultiQure has the potential to slow the progression of the disease. Investors also emphasise that the intended therapeutic model is designed as a one-time procedure. At the current stage, however, the efficacy and safety of the solution still need to be confirmed in clinical trials.
Huntington’s Disease Remains Incurable
Huntington’s disease is a rare, inherited and progressive neurodegenerative disorder. It leads to the gradual damage of neurons in the brain and is associated with motor, cognitive and psychiatric symptoms. As the disease progresses, patients lose independence, while currently available treatments do not stop its underlying causal mechanism.
The condition is sometimes described as combining symptoms characteristic of several neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral sclerosis. This comparison is illustrative, as Huntington’s disease is a separate medical condition with its own genetic basis.
Baker Tilly Valued the Project at USD 111.3 Million
The economic potential of MultiQure has been estimated by Baker Tilly at USD 111.3 million. The valuation relates to the current stage of the project, which is still ahead of key clinical validation. The authors of the report point to the strong potential of the gene therapy market and the fact that competing therapies for Huntington’s disease and other polyglutamine disorders remain at a relatively early stage of development.
MultiQure representatives estimate that the addressable market for the project may be worth tens of billions of dollars. These estimates result not only from the potential application of the therapy in Huntington’s disease, but also from the possibility of developing the platform for other polyglutamine disorders.
“The RNAi technology we are working on is a real breakthrough in the treatment of Huntington’s disease and other polyglutamine disorders. The current standard of care addresses only the symptoms, while our platform effectively inhibits disease progression, creating an opportunity for hundreds of thousands of patients around the world. The mechanism of our therapy itself is already ready; we are now working on its clinical implementation so that it can be administered to the first patient. This is realistic within the next 12–18 months,” said Dr Kris Siemionow, an investor in the MultiQure project and one of the shareholders of the Biofund fund.
The Project Could Enter the Regulated Market
MultiQure’s management is working on introducing the project to the regulated market in Poland. One of the scenarios under consideration is contributing the project as an in-kind asset to a company already listed on the stock exchange. Such a model could accelerate access to the capital market and facilitate the raising of funds for further research and development.
“The addressable market for the MultiQure project is worth tens of billions of dollars, and within the next dozen or so months we will reach the moment of truth. The innovative therapy will then be administered to the first patient, and no later than 2028 we will see its effects. The RNAi platform has enormous potential to change the standard of treatment for Huntington’s disease and other PolyQ disorders, which is why we are working on bringing it to the stock exchange,” said Paul Lewicki, an investor in MultiQure and a data mining expert.
A Polish Project Following the US Biotech Model
The creators of MultiQure say the project is being developed according to a model known from the US biotechnology market. This approach is based on concentrating capital, the team and resources on one leading asset, while moving as quickly as possible to the clinical stage, where the therapy’s effect can be verified in patients.
The next two years will be crucial for the project. This is when the therapy is expected to be administered to the first patients, followed by an assessment of its effects. The results of clinical trials will determine not only the further development of the technology, but also the project’s real value from the perspective of investors and potential industry partners.
