We enter into a collaboration and license agreement with Prilenia for the commercialization and co-development of Pridopidine
Barcelona (Spain), April 28th, 2025 – Together with Prilenia Therapeutics B.V., a clinical-stage biotech company, we have announced the signing of a strategic co-development and license agreement in which Ferrer obtains the rights to develop, manufacture and commercialize Pridopidine in the European Region, the Middle East and North African Region, the Southern African Region, the Central and South American Region, and the Commonwealth of Independent States Region.
Pridopidine, a potent and highly selective, orally administered sigma-1 receptor agonist, designed to regulate key neuroprotective mechanisms that are often impaired in neurodegenerative diseases, is a promising candidate for the treatment of Huntington’s Disease (HD), a rare inherited neurodegenerative disease, with a high unmet medical need1. It has been studied in more than 1,700 people and long-term safety data of up to 7 years duration are available from previous clinical studies2. These investigational studies demonstrate that at the therapeutic dose, Pridopidine has an observed safety and tolerability profile comparable to placebo2.
Pridopidine is currently being reviewed by the European Medicines Agency, which has accepted the Marketing Authorisation Application submission for the treatment of HD. An opinion from the Committee for Medicinal Products for Human Use is expected in the second half of 2025. The terms of the agreement include an upfront payment and multiple regulatory, development, commercial and sales milestone payments. Prilenia will also receive tiered double-digit royalties on net sales of Pridopidine.
“This agreement with Prilenia means we can continue making our purpose of using business to fight for social justice a reality, while focusing our pipeline development on diseases with high unmet medical need,” stated Mario Rovirosa, CEO of Ferrer. “The combination of strengths and capabilities of our two companies makes the future brighter for the patients suffering from such underserved conditions.”
“We are proud to partner with Ferrer as we advance our shared mission to bring transformative therapies to people living with neurodegenerative diseases around the world,” said Dr. Michael R. Hayden, CEO of Prilenia. “Ferrer continues to grow their already significant presence throughout Europe and key international markets with particular focus on innovative products for rare diseases. By combining our unique strengths and shared commitment to these patient communities, we believe that this partnership has the potential to accelerate the delivery of Pridopidine to the thousands of people who are waiting for a new treatment option as well as to broaden its impact through additional indications in the future.”
“Securing rights to this molecule represents a pivotal step in our research strategy in the neurodegeneration arena,” said Oscar Pérez, Chief Scientific and Business Development Officer at Ferrer. “Given the mechanism of action of Pridopidine, we are fully committed to exploring its potential use across a range of indications.”
About Huntington’s disease
Huntington's disease (HD) is a rare, inherited neurodegenerative disorder that follows an autosomal dominant pattern of inheritance. It is characterized by a range of functional, motor, cognitive, and behavioural symptoms caused by a mutation in the huntingtin gene. This mutation leads to a toxic form of the multifunctional huntingtin protein, which disrupts neuron function and ultimately results in neuronal death3-5.
HD affects approximately 5 to 10 people per 100,000 inhabitants globally, with an estimated incidence of one new case per year for every 100,000 individuals6. The disease is typically diagnosed between the ages of 30 and 50, though it can develop at any age, including in children and young adults (known as juvenile onset Huntington's disease or JHD)7. The progression of HD is generally slow, spanning 15 to 20 years, during which patients gradually lose their ability to work, communicate, manage daily activities, and care for themselves1-5. This increasing disability eventually leads to complete reliance on caregivers and, ultimately, death.
Currently, available treatments for Huntington's disease focus on alleviating symptoms and providing palliative care, but they do not alter the overall progression of the disease1-3.
About Prilenia
Prilenia is a private biopharmaceutical company driven by an unwavering commitment to scientific excellence and accelerating progress for people affected by Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS). Our mission is simple but urgent: to develop and provide sustainable access to transformative medicines for people affected by devastating neurodegenerative diseases.
Prilenia operates across the United States, Canada, Europe and Israel. The company is incorporated in the Netherlands and backed by leading life sciences investors.
For more information, please visit www.prilenia.com and connect with us on LinkedIn or X (Twitter).
References:
1. Naia L, Ly P, Mota SI, Lopes C, Maranga C, Coelho P, Gershoni-Emek N, Ankarcrona M, Geva M, Hayden MR, Rego AC. The Sigma-1 Receptor Mediates Pridopidine Rescue of Mitochondrial Function in Huntington Disease Models. Neurotherapeutics. 2021 Apr;18(2):1017-1038. doi: 10.1007/s13311-021-01022-9. Epub 2021 Apr 1. PMID: 33797036; PMCID: PMC8423985.
2. Goldberg YP, Navon-Perry L, Cruz-Herranz A, Chen K, Hecker-Barth G, Spiegel K, Cohen Y, Niethammer M, Tan AM, Schuring H, Geva M, Hayden MR. The Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington's Disease. CNS Drugs. 2025 May;39(5):485-498. doi: 10.1007/s40263-025-01171-x. Epub 2025 Mar 7. PMID: 40055280; PMCID: PMC11982116.
3. Bates, G., Dorsey, R., Gusella, J. et al. Huntington disease. Nat Rev Dis Primers 1, 15005 (2015). https://doi.org/10.1038/nrdp.2015.5
4. Bachoud-Lévi AC, Ferreira J, Massart R, Youssov K, Rosser A, Busse M, Craufurd D, Reilmann R, De Michele G, Rae D, Squitieri F, Seppi K, Perrine C, Scherer-Gagou C, Audrey O, Verny C, Burgunder JM. International Guidelines for the Treatment of Huntington's Disease. Front Neurol. 2019 Jul 3;10:710. doi:10.3389/fneur.2019.00710.PMID: 31333565; PMCID: PMC6618900.
5. Tabrizi, S.J., Flower, M.D., Ross, C.A. et al. Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities. Nat Rev Neurol 16, 529–546 (2020). https://doi.org/10.1038/s41582-020-0389-4
6. Medina, A., Mahjoub, Y., Shaver, L. and Pringsheim, T. (2022), Prevalence and Incidence of Huntington's Disease: An Updated Systematic Review and Meta-Analysis. Mov Disord, 37: 2327-2335. https://doi.org/10.1002/mds.29228
7. Anil M, Mason SL, Barker RA. The clinical features and progression of late-onset versus younger-onset in an adult cohort of Huntington’s disease patients. J Huntingtons Dis. 2020;9(3):275-282. doi:10.3233/JHD-200404. PMID: 32675419; PMCID: PMC7683085.