Targeting toxic protein aggregation, German biotech banks €12M to develop drug for rare neurodegenerative disorder
The accumulation of misfolded proteins is considered the hallmark of various neurodegenerative diseases. Now, a German biotech is going after toxic protein aggregation in patients with multiple system atrophy (MSA), a rare, progressive disorder caused by shrinking nerve cells in the brain.
The company, MODAG, was founded in 2013 and based on research conducted by scientists at Ludwig Maximilian University of Munich and the Max-Planck-Institute for Biophysical Chemistry. On Thursday the biotech unveiled a €12 million (about $13.64 million) round of financing — led by Massa Investment AG — which will help shepherd its lead experimental drug, anle138b, into human trials.
The drug, anle138b, is a small molecule engineered to bind to toxic oligomeric structures of alpha-synuclein — a protein in the brain that tends to be concentrated near the tips of neurons in association with synaptic vesicles responsible for neurotransmission. The assault is designed to dissolve the toxic oligomers and prevent new oligomer formation to address the etiology of the MSA, versus existing treatments designed to manage symptoms that progress alongside the disease.
MSA, considered an atypical form of Parkinson’s disease, is characterized by impaired speech, movement, balance and blood pressure control. It is estimated to affect 50,000 individuals across the United States, Europe and Japan. The company’s drug — an oral compound that is formulated to penetrate the blood-brain barrier — in preclinical studies has shown promising potential in halting disease progression.
“Although MSA is considered a rare disease, being able to halt its progression would have significant implications for many neurodegenerative diseases that currently have no treatment…,” said newly crowned MODAG chief Torsten Matthias, in a statement.
In connection with the financing, Armin Giese has been appointed chief scientific officer and Massa Investment’s Jeff Putman is set to join the MODAG’s board.