Sanofi Genzyme and Novartis’ venture arms have backed NeuroVia, an upstart that wants to help treat the rare CNS disorder X-linked adrenoleukodystrophy.
The $14 million series A, which was also backed by BioMed Ventures and Enso Ventures, will go toward finishing off preclinical work on the company’s lead candidate, NV1205, and taking it into a clinical proof-of-concept development program.
The candidate is seeking to treat X-linked adrenoleukodystrophy (X-ALD), a rare condition that primarily affects newborn boys, although heterozygous women also develop the disease later in life.
X-ALD is characterized by the accumulation of very long chain fatty acids (VLCFA), leading to a neurodegenerative disorder in which the most affected tissues are the spinal cord, the brain and the adrenal cortex. The CNS-related effects lead to two main phenotypes: adrenomyeloneuropathy (AMN), characterized by progressive motor dysfunction, and cerebral ALD (cALD), characterized by severe neuroinflammation leading to early death.
Current treatments are limited, namely: Lorenzo’s Oil (a dietary supplement) and stem cell transplantation. The former does not significantly improve clinical symptoms of the disease, and the latter has serious limitations, according to NeuroVia.
NV1205 works as a small molecule that has shown in preclinical models to reduce levels of VLCFAs in the brain, adrenal tissue and blood of mice lacking a functioning ABCD1 gene. The startup believes this shows it has a potential as a disease-modifying therapy. NeuroVia says its hopes to create an X-ALD treatment for patients of all phenotypes.
Henry Skinner, M.D., of Novartis Ventures and Jason Hafler, senior director of investments at Sanofi Genzyme's venture arm, have joined the board of NeuroVia alongside scientific co-founder Thomas Scanlan and co-founder Giovanni Ferrara, who is also president of the biotech.
The company is currently pursuing a virtual model with a small core team, mimicking a number of other startups in the industry. It does have an management team in place, which includes CMO Masoud Mokhtarani, M.D., formerly of Hyperion (now owned by Horizon Pharma); chief development officer and Pfizer vet John Henderson, M.D.; and a director of clinical operations, Catherine Norris, who was also at Hyperion.
Ferrara tells FierceBiotech that he hopes to be “in the clinic before the end of this year. We will update the X-ALD community as soon as sites become active. While we will limit activation to a few major centers, we are committed to opening the trial to any qualifying individual.”
Minoryx Therapeutics is also at work in the clinic on X-ALD, specifically on a PPAR gamma agonist, and recently reported data from a phase 1/2 test for MIN-102. This was predominately safety focused, which it passed, and the biotech is now planning a later stage trial this year.
I asked Ferrara how his company’s lead candidate differs from Minoryx: “I will restrict comments to NeuroVia. We believe we have a solid biochemical and mechanistic rationale as to why our drug can benefit a spectrum of X-ALD patients. Our approach addresses the metabolic defect in X-ALD and restores the ability of the body to break down toxic VLCFAs, fatty acids that build up in X-ALD patients.
“VLCFAs otherwise severely damage neuronal tissues. Ultimately, it is possible that our approach may be complementary to that of a competitor, and X-ALD may one day be treated with a polypharmacy approach.”
On potential partners, Ferrara said: “We will pursue and evaluate opportunities to enter into partnerships or corporate agreements that will be of strategic value to accelerate the delivery of our proprietary programs to patients and/or help us to advance our technology.”