MOLEFY

Although the exact etiology and pathophysiology of ALS remain unknown, TDP-43 has been identified as a key hallmark of the disease. To tackle the urgent need for an effective treatment, MOLEFY is developing a small molecule called AP-2. This promising drug candidate aims to prevent motor neuron death by reversing the pathogenic changes of TDP-43.

AP-2 can cross all brain barriers and has demonstrated promising results in recent preclinical trials involving different animal models. These studies showed that blocking the abnormal behavior of TDP-43 not only relieves ALS symptoms but also reverses disease progression.

MOLEFY’s approach is supported by encouraging data from both animal and patient-based cellular models. AP-2 has demonstrated effectiveness and favorable pharmacological safety profile in rodent and non-rodent animal models. Our goal is to gather all necessary information to start human clinical trials by 2025.

One of MOLEFY‘s main goals for 2024 is to secure a funding round to accelerate the clinical trial development of its most promising molecule (AP-2), aiming to start these trials in 2025. Currently, €1.5 million is needed to develop phase I of the trials, and additional investment will be required for phases II and III.

We have assembled a talented team of scientists and clinicians committed to advancing ALS research and developing effective treatments. With robust intellectual property protection, we are well-positioned to scale up our operations and bring our molecule to market.