MedCity News eNewsletter

Fat-based Parkinson’s drug headed to clinical trial with $6.5M in angel money

There’s a generalized consensus that “good” fats – like Omega-3 fatty acids – could help improve mental acuity, even combat neurodegenerative disease. Bay Area startup Retrotope is riffing off this idea, engineering what could be a super-good fat that treats aging-related conditions like Alzheimer’s, Parkinson’s and diabetic retinopathy. It just received $6.5 million in angel funding to bring its fatty drug to the […]

There’s a generalized consensus that “good” fats – like Omega-3 fatty acids – could help improve mental acuity, even combat neurodegenerative disease. Bay Area startup Retrotope is riffing off this idea, engineering what could be a super-good fat that treats aging-related conditions like Alzheimer’s, Parkinson’s and diabetic retinopathy. It just received $6.5 million in angel funding to bring its fatty drug to the clinic, CEO Robert Molinari said.

Here’s Retrotope’s rationale:

Good fats could help mitigate oxidative stress – a process that has  long been thought to be a precursor to neurodegenerative disease, cancers and autoimmune illness. Essentially, oxidative stress occurs when free radicals “steal” electrons from the fatty acids found on cell membranes, degrading their quality. This, in turn, causes mitochondrial dysfunction – which could trigger disease like Parkinson’s and Alzheimer’s.

Researchers thought as far as two decades back they could treat oxidative stress by giving patients antioxidants and even good fats, but have since found that such a heavy-handed approach to neurodegenerative disease just doesn’t quite work.

Retrotope has a more fine-tuned bent that’s shaping up to be quite promising preclinically. Its lab-evolved lipid basically stabilizes the essential fatty acids that are oxidating on the cellular membrane. The engineered fat shuts down the oxidation process at its very start – thus preventing a cascade effect that leads to symptom manifestation and progression.

“This is very new, in that pretty much no drugs on the planet are lipids that have a lipid as a target,” Molinari said. “Basically, we have a drug that isn’t a natural lipid – it’s a modified lipid, or lipid mimetic, that’s really safe.”

The company has found more than a dozen preclinical models in which it can “rescue cells that have been damaged by various stresses of oxidation,” Molinari said.

presented by

And after five years of preclinical development in vitro and in animal models, this lipid-on-lipid approach is about ready for the next step. Retrotope plans to file its IND in the next month or two, Molinari said, with plans to enter the clinic for a 30-40 patient Phase 1B/2A trial by year’s end. The FDA’s granted Retrotope a 505B2 route into human clinical trial – a kind of expedited NDA that allows companies to bypass performing certain safety studies by relying on published literature.

Molinari describes the company as the “classic, ultimate virtual pharma,” conducting work through CROs and more than 60 collaborators around the world, including researchers at University of Pennsylvania, University of California, San Diego and University City College London.

Since its 2008 start that operated just on friends-and-family funding, Retrotope has brought in $11 million in angel investments and foundation capital. Though it has raised $6.5 million in this current go-around, it hopes to reach $10 million for its clinical trials. The company has also received funding from organizations like the Michael J. Fox Foundation, the Research Alliance and the National Institutes of Aging, Molinari said.

“We’ve been pretty stealthy,” Molinari said. “But we’re about to come out of stealth mode.”