Prilenia partners with Ferrer to advance pridopidine for ALS
License agreement reached to codevelop therapy in Europe, other regions

Prilenia Therapeutics has entered a collaboration and license agreement with Ferrer to codevelop and commercialize pridopidine, its therapy candidate for neurodegenerative diseases like amyotrophic lateral sclerosis (ALS), in Europe and other select markets.
The partnership comes as pridopidine nears an expected regulatory decision in the European Union, where it’s being reviewed by the European Medicines Agency for use in Huntington’s disease. Under the deal, Ferrer will support the therapy’s potential launch in the EU for Huntington’s while helping to advance its development in ALS and other indications.
In ALS, the companies are planning the launch of a pivotal Phase 3 trial involving people with the motor neuron disease. That comes after encouraging results from the HEALEY ALS Platform Trial (NCT04297683), which showed that pridopidine may slow disease progression and extend survival in individuals with early ALS.
Under the terms of the agreement, Prilenia will receive an upfront payment of about €80 million (more than $90 million), plus additional revenue of as much as €45 million (about $51 million) for near-term development, regulatory, and commercial milestones, per a company press release. Prilenia also will receive tiered double-digit royalties on net sales.
Meanwhile, the company will retain all rights to pridopidine in major markets, such as North America, Japan, and the Asia-Pacific region.
“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 broaden its impact through additional indications in the future,” said Michael R. Hayden, PhD, CEO of Prilenia.
Regulatory decision expected in EU on pridopine as Huntington’s treatment
Pridopidine is an oral small molecule designed to activate a protein involved in crucial processes needed for the functioning and survival of neurons, or nerve cells. It regulates a number of mechanisms that protect from ALS progression, including the clearance of toxic molecules and energy production, while reducing cellular stress and inflammation.
A Phase 2/3 arm (NCT04615923) of HEALEY — a perpetual multicenter, multiregimen clinical trial — enrolled 163 adults with ALS who were randomly assigned to receive pridopidine or a placebo for about six months. This was followed by an open-label extension in which all received the active treatment.
While the trial failed to meet its primary and secondary goals of slowing disease progression and showing changes in muscle strength, lung function, and survival, a subgroup of patients with early ALS seemed to benefit from the therapy.
Those encouraging findings will now be explored in the Phase 3 trial.
Meanwhile, people with ALS who are not eligible for clinical trials of pridopridine may access the drug via an expanded access program (NCT06069934), or EAP. These programs, also called compassionate-use programs, allow people with serious or life-threatening conditions to access investigational therapies outside of clinical trials. About 200 participants are being enrolled, and all will receive treatment for as long as two years.
The combination of strengths and capabilities of our two companies makes the future brighter for the patients suffering from such underserved conditions.
Pridopidine has been granted orphan drug designation for ALS in both the U.S. and Europe. That status aims to boost the development of treatments for rare and serious diseases by providing certain benefits, such as a period of market exclusivity upon regulatory approval.
In addition to Huntington’s, for which an application has already been filed, Prilenia and Ferrer will collaborate to develop and fund the expansion of pridopridine for ALS and other indications.
“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.”
Added 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.”