• The AEMPS authorizes the initiation of the Phase I clinical trial of AP-2, which will begin in April at Hospital Universitario de La Princesa with 70 healthy volunteers
• AP-2, developed by Molefy Pharma based on CSIC research, targets the TDP-43 protein and has been granted orphan drug designation by the European Medicines Agency (EMA)
Molefy Pharma, a CSIC spin-off majority-owned by the ARQUIMEA group, takes a decisive step in its mission to develop innovative therapies for amyotrophic lateral sclerosis (ALS) with the authorization from the Spanish Agency of Medicines and Medical Devices (AEMPS) to initiate Phase I of its candidate AP-2 in healthy volunteers.
The trial, which will begin next April at the Clinical Trials Unit of Hospital Universitario de La Princesa (Madrid), will evaluate the safety of the compound in approximately 70 healthy volunteers. This study represents the first step in the clinical development of AP-2 and will generate the necessary data to advance to subsequent phases in patients.
AP-2 acts on TDP-43, a key protein in the pathophysiology of ALS. The alteration of this protein is directly related to motor neuron degeneration. In preclinical studies, AP-2 has demonstrated its ability to reverse this alteration and restore its function, both in cellular models and animal models, reinforcing its potential as a disease-modifying treatment.
The development of AP-2 has received international recognition. In October 2025, the European Medicines Agency (EMA) granted the compound orphan drug designation, validating its therapeutic relevance and facilitating its clinical development.
Molefy Pharma leads this development with the aim of addressing an unmet medical need. Currently, ALS remains a disease with no cure, with limited therapeutic options and a devastating impact on patients’ quality of life.
If the results of this first phase are positive, it is expected that a Phase Ib trial in patients will begin from 2027, thus advancing the development of a potential therapy capable of modifying the course of the disease.