Medicxi founds Parkinson’s startup, commits €9M

A Lewy body in a brain cell of the substantia nigra in Parkinson's disease.(By Marvin 101 (Own work) [CC BY-SA 3.0 (], via Wikimedia Commons)

Medicxi has unveiled its second new co in as many weeks. The VC shop returned to France to source the underlying science, teaming with former Merck Serono partner Domain Therapeutics to access a drug intended to induce production of glial cell line-derived neurotrophic factor (GDNF).

Mavalon Therapeutics, the asset-centric company founded to advance the candidate, will receive up to €9 million ($10 million) from Medicxi. As with other Medicxi-founded startups that have received similar sums of money, the funding is intended to support Mavalon through to the delivery of clinical efficacy data, at which point the range of business development options open to the company could expand significantly.

Medicxi’s interest in the asset is underpinned by its potential to overcome a persistent problem that has held back a promising field. Mavalon is working on an orally available small molecule designed to induce production of GDNF in the brain. The potential of GDNF to slow or reverse cell death in the brain has attracted interest for years--Amgen paid $240 million for the biotech that discovered the protein in 1994--but delivery challenges have stymied progress.

Companies have tried an array of tricks to get GDNF across the blood-brain barrier. Amgen delivered the protein directly into the fluids separating the lobes of the brain, but data from the 38 patients who received this regimen fell well short of the potential pointed to by animal testing. Since then, researchers have investigated whether intranasal delivery or gene therapy can bypass the barrier, but the long wait for success in GDNF continues.

Domain Therapeutics stepped up its interest in the protein in 2011 when it landed a grant from the Michael J. Fox Foundation (MJFF). That grant enabled the biotech to advance a positive allosteric modulator of metabotropic glutamate receptor type 3 (mGluR3 PAM) through preclinical testing.

By targeting mGluR3, Domain thought it could make striatal neurons produce GDNF. A summary of data presented by MJFF backs up the idea, but the progress of lead candidate DT010709 and related compounds have stuttered in recent years.

Strasbourg, France-based Domain has been looking for a collaborator to work on lead optimization for a couple of years without success, despite having a track record in metabotropic glutamate receptors. Domain is perhaps best known for its Parkinson’s deal with Merck Serono. That collaboration fell foul of a rejig at Serono but was given a second chance when Prexton Therapeutics took on the asset. The drug, a mGluR4 PAM, is set to enter Phase II next year.

While the Prexton program is looking to treat the symptoms of Parkinson’s, Mavalon is aiming to stop the progression of the disease.