Prilenia enters into a collaboration and licensing agreement with Ferrer for the commercialization and co-development of pridopidine in Europe and certain other markets
Tuesday, April 29, 2025
Prilenia Therapeutics BV , a 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), today announced that it has entered into a collaboration and license agreement with Ferrer to commercialize and develop pridopidine in Europe and certain other markets. Pridopidine is a potent and highly selective, orally administered sigma-1 receptor (S1R) agonist designed to regulate key neuroprotective mechanisms often impaired in neurodegenerative diseases such as HD and ALS.
Under the terms of the agreement, Prilenia will receive an upfront payment of approximately EUR 80 million, plus up to EUR 45 million in near-term development, regulatory, and commercial milestone payments. The total transaction is valued at approximately EUR 500 million in upfront and milestone payments. In addition, Prilenia will receive double-digit tiered royalties on net sales. Prilenia and Ferrer have agreed to jointly develop and finance the expansion of pridopidine in the territory for additional indications beyond HD. Prilenia will retain all rights to pridopidine in other major markets, including North America, Japan, and Asia-Pacific.
“We are proud to partner with Ferrer as we pursue our shared mission of bringing transformative therapies to people with neurodegenerative diseases worldwide,” said Dr. Michael R. Hayden, CEO of Prilenia. “Ferrer continues to build on its already significant presence throughout Europe and key international markets, with a particular focus on innovative products for rare diseases. By combining our unique strengths and shared commitment to these patient communities, we believe this partnership has the potential to accelerate the availability of pridopidine to the thousands of people waiting for a new treatment option and to broaden its impact through additional indications in the future.”
“This agreement with Prilenia means we can continue to make our ambition to fight for social justice a reality, while focusing our pipeline development on diseases with significant unmet medical need,” said Mario Rovirosa, CEO of Ferrer. “Combining the strengths and capabilities of our two companies makes the future brighter for patients suffering from these diseases with insufficient treatment options.”
“Securing the rights to this molecule represents a crucial step in our research strategy in the field of neurodegeneration,” said Oscar Pérez, Director of Scientific and Business Development at Ferrer. “Given pridopidine’s mechanism of action, we are fully committed to exploring its potential use in a range of indications.”
About Pridopidine
Pridopidine (45 mg twice daily) is a potent and highly selective orally administered sigma-1 receptor (S1R) agonist designed to regulate key neuroprotective mechanisms often impaired in neurodegenerative diseases such as HD and ALS. i
In its extensive development program for HD, pridopidine has demonstrated benefits across key disease features impacting quality of life for patients and families, including function, cognition, and motor skills, as measured by validated assessments and maintained for up to two years, with a favorable safety profile.
Prilenia has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA), seeking regulatory approval of pridopidine for the treatment of HD. Our MAA has been accepted for review, and we expect an opinion from the Commission for Medicinal Products for Human Use (CHMP) in the second half of 2025. This is the first application seeking approval for a potential treatment that may impact disease progression in HD.
We are also in discussions with the U.S. Food and Drug Administration (FDA) to determine the next steps for pridopidine in the treatment of HD in the United States. If approved, Prilenia will continue to work as diligently as possible to make pridopidine available to patients with HD.
For ALS, Prilenia and Ferrer plan to initiate a single pivotal Phase 3 trial to evaluate pridopidine, seeking to confirm the results of the Phase 2 HEALEY ALS Platform trial.
Prilenia holds Orphan Drug Designation for pridopidine in the treatment of HD and ALS in the US and EU. In addition, pridopidine has received Fast Track designation from the FDA for the treatment of HD.
About Huntington's Disease
Huntington's disease (HD) is a rare, inherited, autosomal dominant neurodegenerative disorder that causes functional, motor, cognitive, and behavioral symptoms. HD is caused by a mutation in the huntingtin gene , and each child of a parent with HD has a 50% chance of developing the disease.
HD affects approximately 100,000 people worldwide, with another 300,000 at risk of developing HD. i,ii It is usually diagnosed between the ages of 30 and 50, although HD can occur at any age, including in children and young adults (known as juvenile HD or JHD). The disease progresses slowly over 15 to 20 years, with patients slowly losing their ability to work, communicate, manage their daily lives, and care for themselves. This increasing disability leads to total dependence on a caregiver and death.
The only treatments currently available for HD focus on symptomatic relief and palliation, with nothing impacting overall progression.
About Amyotrophic Lateral Sclerosis (ALS)
ALS, also known as Lou Gehrig's disease or motor neuron disease, is a chronic, progressive neurodegenerative disease that affects approximately 350,000 people worldwide.
In people with ALS, the motor neurons in the brain and spinal cord that transmit messages to muscles degenerate, affecting the brain's ability to communicate with muscles. This leads to muscle loss and progressive paralysis. Patients rapidly lose their ability to walk, speak, eat, and breathe, and become completely dependent on their caregivers. The average lifespan from diagnosis is 2 to 5 years.
The majority of ALS cases (~90%) do not have a family history of the disease. Approximately 10% of ALS cases are caused by inherited genetic mutations (often referred to as familial ALS). One of the genes discovered to cause ALS encodes the sigma-1 receptor (S1R) protein. Mutations in this gene that result in complete loss of S1R function are associated with severe juvenile-onset ALS, while mutations resulting in partial and incomplete S1R function are associated with adult-onset ALS.
About Prilenia
Prilenia is a privately held 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 game-changing medicines for people affected by devastating neurodegenerative diseases.
Prilenia operates in 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 join us on LinkedIn and X (Twitter) .
Prilenia Forward-Looking Statements
Prilenia cautions the reader that statements in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. These forward-looking statements include, but are not limited to, statements regarding: the continued development and commercialization of pridopidine, the potential benefits and value of pridopidine; and the potential benefits and results of this collaboration. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated in the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include uncertainties related to clinical development, regulatory approval, and marketing authorization processes. Prilenia cautions the reader not to place undue reliance on forward-looking statements, which speak only as of the date hereof, and the Company undertakes no obligation to update such statements to reflect events or circumstances that occur after the date hereof.
2025 Prilenia Therapeutics BV
To obtain a copy of this press release, visit the Prilenia website www.prilenia.com .
- Medina et al., Prevalence and Incidence of Huntington's Disease: An Updated Systematic Review and Meta-Analysis. Mov Disord. 2022 Dec;37(12):2327-2335.
- Jiang, A., Handley, R.R., Lehnert, K., & Snell, RG (2023). From Pathogenesis to Therapeutics: A Review of 150 Years of Huntington's Disease Research. International Journal of Molecular Sciences, 24(16), 13021. https://doi.org/10.3390/ijms241613021
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Contacts
Prilenia Contact
Communications Team
info@prilenia.com
Source: businesswire.com
