ALS Association awards $1 million to Molefy Pharma to advance its clinical trial against ALS

ARQUIMEA, Spanish technology group and co-founder of the spin-off Molefy Pharma together with researchers from the CSIC, celebrates the award of a $1 million grant from the ALS Association (ALS.org) to accelerate the development of the AP-2 compound, aimed at treating amyotrophic lateral sclerosis (ALS). This international backing recognizes Molefy Pharma’s scientific approach and ARQUIMEA’s strategy of supporting technologies with transformative potential in healthcare.

Molefy Pharma is working on the development of AP-2, a molecule that has shown great potential in preclinical studies as a possible treatment for ALS, a neurodegenerative disease that currently has no cure. ALS affects around 270,000 people worldwide, with approximately 40,000 new cases diagnosed each year. Its socioeconomic impact is enormous, with an estimated global cost of more than $2 billion annually, and it represents one of the greatest unmet medical needs in the field of rare and neurodegenerative diseases.

The funding will enable Molefy Pharma to move forward to phase I of its clinical trial with healthy volunteers, which will serve to verify the safety of the drug as a potential cure for ALS, as it is a “First in Human” trial. This will be carried out at the Clinical Trials Unit of La Princesa Hospital. Subsequently, a phase 1B trial is planned with a group of 24 patients diagnosed with ALS.

This non-refundable funding is part of the ALS Association’s commitment to accelerating research into transformative treatments for patients with ALS, a disease for which there is still no cure.

“This support represents much more than just funding. It is international recognition of the therapeutic potential of our approach and the impact we want to achieve for patients and their families,” explains Ana Martinez, co-founder of Molefy Pharma.

The ALS Association, based in the US, is one of the world’s leading organizations in the field of ALS research. Its innovation support program seeks to identify and promote projects with high potential for clinical impact and international scalability.

“We are proud to support the work of Molefy Pharma, a company with a solid, scientific approach that seeks to change the prognosis of ALS at its root,” says Diego Fernández, president of ARQUIMEA.

With this support, Molefy Pharma and ARQUIMEA consolidate their position in the international biotechnology ecosystem focused on rare and neurodegenerative diseases, advancing therapies that aim not only to slow down but also to halt the progression of ALS.