Prilenia and Ferrer Enter €500M Collaboration for Phase III CNS Asset Pridopidine

On April 28 local time, Prilenia announced that it had entered into a collaboration and license agreement with Ferrer regarding the pipeline of Pridopidine, an oral sigma-1 receptor (S1R) agonist. Under the terms of the agreement, Prilenia will receive an upfront payment of approximately €80 million, plus up to €45 million in near-term development, regulatory, and commercial milestones.The total transaction value reached approximately €500 million (approximately RMB 4.15 billion).In addition, Prilenia will receive tiered double-digit royalties on net sales.

Under the agreement, Ferrer and Prilenia will co-develop and potentially commercialize pridopidine in Europe and other undisclosed specific markets. This co-development agreement supports the further expansion of pridopidine for Huntington’s disease, amyotrophic lateral sclerosis (ALS), and future additional indications. Prilenia will retain full rights to pridopidine in other major markets, including North America, Japan, and the Asia-Pacific region.

Pridopidine is a potent and highly selective oral sigma-1 receptor (S1R) agonist designed to modulate key neuroprotective mechanisms that are often impaired in neurodegenerative diseases such as Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS).

Huntington’s disease (HD) is a rare, hereditary, autosomal dominant neurodegenerative disorder. Patients present with brief, uncontrollable facial grimacing, head nodding, and finger twitching; as the disease progresses, involuntary movements worsen progressively. Due to the characteristic choreiform involuntary movements, dysphagia, and dysarthria observed in patients, this condition is also known as Huntington’s chorea. Currently, treatment for HD is primarily symptomatic and palliative, aiming to improve patients’ quality of life by alleviating tremor (using VMAT2 inhibitors and benzodiazepines) and psychiatric symptoms (using SSRIs and SNRIs). There are currently no curative treatments or therapies that can effectively slow disease progression.

In April 2023, Prilenia reported that the Phase III PROOF-HD study for this disease failed to meet its primary and key secondary endpoints. However, further analysis of the data by Prilenia indicated thatThe candidate drug demonstrated clinically meaningful efficacy in patients not taking antidopaminergic or anti-chorea medications.Furthermore, pridopidine has been shown to benefit key disease features that impact the quality of life for patients and their families, including functional, cognitive, and motor skills, as measured by validated assessments over a period of up to two years. Pridopidine demonstrates a favorable safety profile.

Based on previous positive clinical data, Prilenia has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for pridopidine in the treatment of Huntington’s disease (HD). The EMA has accepted this MAA. The Committee for Medicinal Products for Human Use (CHMP) is expected to issue its opinion in the second half of 2025. If the EMA’s review ultimately results in approval, this would be the first drug in the HD treatment landscape potentially capable of affecting markers of disease progression.

ALS is also a key focus of the Pridopidine development pipeline. For ALS, Prilenia and Ferrer plan to initiate a single pivotal Phase III clinical trial to evaluate Pridopidine, aiming to confirm the results from its Phase II HEALEY ALS Platform Trial. Previously, Pridopidine failed to improve disease severity in ALS during Phase II testing; however, the company stated at the time that further investigation of the candidate drug was warranted. A post hoc analysis by Prilenia revealed that among patients experiencing rapid decline in speech, writing, and other daily functions, those treated with Pridopidine showed a “substantially reduced rate of decline” compared to patients receiving placebo.

Currently, Prilenia holds orphan drug designation in the United States and the European Union for pridopidine in the treatment of Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS). Furthermore, pridopidine has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the treatment of HD. In addition to HD and ALS, Ferrer retains priority rights to develop pridopidine for other potential indications.

In fact, this marks the second time Ferrer has entered into a collaboration on an ALS-related pipeline.

In March 2024, Ferrer and Verge Genomics announced a collaboration to jointly develop Verge’s lead candidate, VRG50635, for the treatment of sporadic and familial ALS in Europe, Central and South America, Southeast Asia, and Japan. VRG50635 is a potent, orally available PIKfyve inhibitor that enhances neuronal survival in patients with ALS and has demonstrated efficacy in multiple preclinical studies using models of ALS-related motor neuron degeneration. PIKfyve was identified as a therapeutic target for ALS through analysis of patient-derived tissues using Verge’s AI platform, CONVERGE.

Currently, VRG50635 is undergoing a Phase 1b proof-of-concept (PoC) study in Canada and several European countries. Verge’s PoC study employs innovative technology capable of collecting substantial amounts of unbiased, objective, disease-related data to accurately assess safety, tolerability, pharmacological dose-response, and potential efficacy during the early stages of clinical development. Under the agreement, Ferrer has committed to upfront payments and potential development and commercial milestone payments exceeding €112.5 million.

Compared with other well-known multinational corporations (MNCs), Ferrer has acquired two ALS-related pipeline assets within approximately one year, with the total known deal value amounting to €612.5 million (approximately RMB 5.086 billion). Given its frequent investments in the CNS field and the substantial transaction amounts, what exactly is the background of this Spanish pharmaceutical company?

Ferrer, founded in 1959, is a highly esteemed company in Spain with a diverse portfolio spanning biopharmaceuticals, diagnostic therapies, vaccines, fine chemical raw materials, and animal feed and nutrition. The group operates direct subsidiaries in 26 countries worldwide, with its products distributed in over 90 countries. As a leading enterprise in the Spanish pharmaceutical industry, Ferrer conducts research in cardiology, central nervous system disorders, dermatology, metabolism, oncology, pediatrics, pain management, and other fields. Its scientific research capabilities have received the highest rating from the Spanish Pharmaceutical Evaluation System (Profarma).

In addition to Ferrer, the long-established Spanish pharmaceutical company that is actively expanding its presence in CNS disorders such as ALS and HD, numerous multinational corporations (MNCs) and global pharmaceutical companies—including GSK, AbbVie, Takeda, Novartis, Roche, Sumitomo, and Merck & Co.—have also made high-profile entries into the CNS field in recent years.Massive investments correspond to the vast, unmet patient needs. According to Frost & Sullivan, the global market for CNS drugs will reach $172.1 billion by 2034, with China’s market reaching $57.1 billion. The WHO predicts that within two decades, CNS diseases will become the second leading cause of death.

Given the complexity of the central nervous system (CNS) and the challenges inherent in drug development, there remains a substantial unmet clinical need for pharmacological treatments for many CNS disorders. With the continuous advancement of cutting-edge instrumentation, basic research in the CNS field has reached a critical tipping point for breakthroughs. It is anticipated that over the next 5 to 10 years, effective pharmacotherapies will become available for a broader range of CNS diseases. Within the industry, any entity that first identifies a key breakthrough will achieve a milestone advancement and rapidly capture the blue-ocean market for specific niche indications.

Reference: [Express] AI Platform Discovers Novel Target; Potential “Best-in-Class” ALS Therapy Secures Over €100 Million in Funding