Prilenia Enters into a Collaboration and License Agreement with Ferrer for the Commercialization and Co-Development of Pridopidine in Europe and Other Select Markets

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 HD development program, pridopidine has demonstrated benefits across key features of the disease impacting quality of life for patients and families, including function, cognition and motor skills, measured by validated assessments and sustained for up to two years, with a favorable safety profile.

Prilenia has submitted a Marketing Authorization 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 Human Products (CHMP) in the second half of 2025. This is the first submission seeking approval for a potential treatment that can impact disease progression in HD.

We also are in ongoing discussions with the U.S. Food and Drug Administration (FDA) to determine the next steps for pridopidine in HD in the U.S.

For ALS, Prilenia and Ferrer plan to initiate a single, pivotal Phase 3 trial to evaluate pridopidine, seeking to confirm findings from the Phase 2 HEALEY ALS Platform Trial.

Prilenia holds Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the FDA for the treatment of HD.

About Huntington’s Disease

Huntington’s disease (HD) is a rare, inherited, autosomal dominant, neurodegenerative disease that results in 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 percent chance of developing the disease. HD affects approximately 100,000 people around the world with an additional 300,000 people 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 onset HD or JHD). The disease progresses slowly over 15 to 20 years, with patients slowly losing their ability to work, communicate, manage day-to-day life and take care of themselves. This increasing disability leads to full reliance on a caregiver and, ultimately, death.

The only currently available treatments for HD focus on symptomatic relief and palliative care, with nothing impacting measures of 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 affecting approximately 350,000 people worldwide.

In people with ALS, motor neurons in the brain and spinal cord that convey messages to the muscles degenerate, affecting the brain’s ability to communicate with muscles. This leads to muscle wasting and progressive paralysis. Patients rapidly lose their ability to walk, speak, eat, and breathe, and become fully dependent on their caretakers. The average life span from diagnosis is 2 to 5 years.

The majority of ALS cases (~90%) are without a family history of the disease. About 10% of ALS cases are caused by inherited genetic mutations (often called 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 function of the S1R are associated with severe, juvenile ALS, while mutations resulting in partial, incomplete function of the S1R are associated with adult-onset ALS.

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).

Prilenia Forward Looking Statements

Prilenia cautions readers that statements contained 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. Such forward-looking statements include, but are not limited to, statements regarding: advancing development of and commercializing pridopidine, the potential benefits and value of pridopidine; and the potential benefits and outcome from 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 by 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 in clinical development, regulatory approval and commercialization processes. Prilenia cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof.

©2025 Prilenia Therapeutics B.V.