Why this biotech startup is going after rare neurological diseases

Ovid Therapeutics CEO Jeremy Levin. Courtesy Ovid Therapeutics

A decade ago, there were just two cancer drug programs exploring a way to harness the body’s immune system so it could better attack tumour cells. One of them was on the verge of shutting down.

Researchers had noticed that, right off the bat, tumours were growing instead of shrinking, Dr. Jeremy Levin, previously an SVP at competing company Bristol-Meyers Squibb, told Business Insider.

But, Levin recounted, that was actually just the white cells going in to attack the tumour — and a sign that Bristol-Meyers, which had not yet slashed its immunotherapy program, should stick with it.

In the first half of 2016, BMS’ drug, called Opdivo, made roughly $1.5 billion in sales, and is now approved to treat a number of different cancer types. Some 500 companies are currently developing drugs to treat cancer, according to a June IMS Institute for Healthcare Informatics report.

Now, Levin is the CEO of Ovid Therapeutics, a pharma company focusing on rare neurological diseases. And he’s betting that researchers working on neurology drugs, in particular those designed to treat rare diseases, are in 2016 where people developing cancer drugs were in 2006: on the cusp of coming up with treatments that are laser-focused, with specific targets in mind — not just shots in the dark.

“What’s occurred in the last decade has been a complete change,” he said. “As science and oncology increased in its understanding of pathways, so that teaching fell into neurology.”

Levin pointed to a couple key things that put neurology in a key spot that other fields aren’t in just yet:

  • Many genetic neurological diseases are very specific, so scientists are beginning to learn exactly how they work, what the symptoms are, and what it will take to treat them.
  • That precision is combined with enhanced imaging technology that can better pick up on changes in the brain, in part because the same imaging technology has improved to get a clearer look at tumours.

Levin’s company is starting with a genetic condition called Angelman Syndrome that affects the nervous system and leads to developmental delays, trouble moving, and difficulty sleeping. The company picked up a drug called gaboxadol, which was originally studied as a sleep drug. It failed by the time it was in late-stage trials, but that was after it was already shown to be safe in humans.

“As you enter into these fields which are so difficult to get into, genetic disorders of the brain offer you a window,” he said. “You can get to them quickly, effectively, and now you can start trying out different ways of treating them.”

And Ovid isn’t the only company with this start small mentality: GenSight Biologics, a company that’s developing gene therapies for certain causes of blindness, told Business Insider that they started with one condition, Leber’s hereditary optic neuropathy (LHON), because it’s a genetic disease where the mutation’s in the mitochondira. If the therapy works in the eye, then GenSight can begin to see if there are other mitochondrial DNA-related diseases that they can take on. Ideally, the company would have started with the central nervous system, but that’s a bit harder to access.

“The retina offers and almost perfect setting, since it is the visible part of our brain,” GenSight CEO Bernard Gilly told Business Insider. And since the eye is a closed system, the gene therapy has a better chance of going where it’s supposed to.

Ovid’s phase 2 trial to treat Angelman’s Syndrome is getting underway, while GenSight is in the midst of two phase 3 trials for its gene therapy.

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