FundaMental Pharma GmbH Announces Groundbreaking Proof-of-Concept Study Using a TwinF Interface Inhibitor for the Treatment of Amyotrophic Lateral Sclerosis

FundaMental Pharma GmbH recently announced the publication of a groundbreaking study by Neurobiologists in Heidelberg using FundaMental Pharma’s small molecule TwinF interface inhibitor, FP802. The study provides seminal preclinical proof-of-concept data for the treatment of amyotrophic lateral sclerosis (ALS).

The research team led by Heidelberg University Professor Hilmar Bading, who is co-founder of FundaMental Pharma, showed that FP802 treatment resulted in reduced neurological scores and mortality in a gold standard mouse model of ALS. Most importantly, the validated ALS clinical biomarker neurofilament light chain (Nf-L) was reduced in line with the positive treatment effects. The team went further to demonstrate that FP802 also protected human ALS patient-derived brain organoids from glutamate neurotoxicity, a key driver of ALS pathogenesis, highlighting the likely translatability of the preclinical findings to patients.

FundaMental Pharma is engaged in pioneering research around a breakthrough molecular mechanism that allows it to safely counteract glutamate neurotoxicity (also known as excitotoxicity) by specifically targeting the interface between NMDA receptors and TRPM4 with small molecule TwinF interface inhibitors (Yan et al., Science 2020). TwinF interface inhibitors constitute an entirely new class of drugs that safely ameliorate glutamate neurotoxicity, a common cause of neurodegeneration.

Thomas Schulze, Chief Executive Officer, Co-Founder of FundaMental Pharma, said “As we are currently in the process of developing orally bioavailable TwinF interface inhibitors, this latest publication by our academic co-founder provides tremendous validation of our work and guides us towards ALS as prime indication for our new medicines.”

Professor Hilmar Bading, Co-Founder of FundaMental Pharma, added “The success of FP802 in protecting brain organoids derived from human ALS patients underscores the translatability of our preclinical findings, offering a glimpse into the potential impact of TwinF interface inhibitors on patients.”

The full paper can be read online here: TwinF interface inhibitor FP802 stops loss of motor neurons and mitigates disease progression in a mouse model of ALS: Cell Reports Medicine.

FundaMental Pharma (FundaMental) is a neuroscience company, spun out of Heidelberg University, developing first-in-class small molecule inhibitors for treatment of a range of neurodegenerative diseases. FundaMental scientists are developing an entirely new class of drugs that safely counteract glutamate neurotoxicity, a common cause of neurodegeneration. While FundaMental’s current focus is on Amyotrophic Lateral Sclerosis (ALS) and Huntington’s Disease, the applicability of these molecules could extend to a range of neurogenerative disorders such as dementia and aging-related memory loss. For more information, visit www.fundamentalpharma.com.