Ovid Therapeutics Inc. Common Stock 12th Annual Cantor Fitzgerald Global Healthcare Conference

It's a GABA aminotransferase inhibitor, and we also are prosecuting a program for a target in the brain called KCC2. Sounds like a Soviet satellite, but it is potentially the great white whale of neurology and neuropsychiatry. It's a target that strategics have long wanted to drug because activating this ion transporter in the brain, KCC2, may have very broad therapeutic utility. And now we have a molecule that's suggesting that is indeed what could be the case. So, in the span of the next 6, 12, 18, and 24 months, we have 4 to 6 proof of concept and proof of signal readouts across that epilepsy program and our KCC2 direct activator program. Starting a couple of months from now and reading out both in terms of translational biomarker studies, but importantly, in terms of also RCT proof of concept studies as well.

I have a lot of opinions on this, but where I think our stock is valued today is primarily in our epilepsy program. I would argue it's undervalued. I think we've got some pretty exciting card turnovers ahead, both in focal-onset seizures, which is a well-characterized space, but an area where there's still a lot of unmet need, as well as in developmental epileptic encephalopathies. But what's not valued in our stock today, but sitting in the room and one-to-ones today and with other funds and investors, is really getting value for our KCC2 program. There's probably reasons why that hasn't translated in our stock, but I think that is set up to change in the very near future.

We're in field right now with a phase I study, the first company that has ever drugged this target, KCC2, and we're within a reasonably short period of time of results from a translational study called a ketamine challenge. And I think that will be very meaningful because if we see the results from that that we want, we will be the only public company to have ever drugged KCC2.

What we will be able to establish in that is that we're getting into the brain, we're neuroactive, hopefully that we're modulating electrophysiology that's consistent with the mechanism of drugging KCC2, which would be more GABAergic, so more inhibition or the braking system in the brain working the right way. Maybe we have some really exciting biomarkers that we're applying, where we may actually even be able to see predictive data relative to clinical effect and efficacy, which, if KCC2 is as broad of a therapeutic target as what our pharmacodynamic data is suggesting and what the literature has long suggested, this would be early validation about the right first indications for us to walk into. Because there actually may be many that we could go into. We have extremely positive data for KCC2 acting as an antipsychotic.

Yeah. It also has applications in areas like epilepsies.

Yeah Rett syndrome, anxiety, chronic neuropathic pain.

Yeah. Clearly, we're not going to do all those things at Ovid.

Yeah but making the best indications about the most direct shot on goal for proof of concept is the aspiration.

I've said broad therapeutic utility a few different times. Why I say that is KCC2 is very unique as a target. It's essentially a more precise way of tuning GABA, the primary braking system in our brain. Why it's a target that strategics and clinicians have long wanted to drug is it's an ion transporter. We hear that. There's other companies that have programs for ion transporters, but KCC2 is only in the brain, in the CNS, so it's neuron-specific. Essentially, when this particular transporter, which balances potassium and chloride, is dysfunctional, GABA can't do its job. It can't be a braking system. So, this transporter essentially pumps out enough chloride from the neuron that enables GABA to be the brakes, to be inhibitory.

When you start to take a look across the literature, and you look at where KCC2 is implicated, you start to reveal essentially a panoply of studies showing that it's related to either the underlying disease biology or symptoms of many diseases. Specifically, in some of the indications that we're looking at, we know KCC2 expression and function is reduced in key regions of the brain that are driving symptoms of diseases. For example, we're very interested in it for psychoses because we now have more than 40 pharmacodynamic studies that are showcasing really profound rescue effect of positive, negative, and cognitive symptoms, which is the holy grail for an antipsychotic, particularly if you don't come with all the safety and tolerability baggage that, unfortunately, prior classes of medicines have done. But KCC2, in the case of schizophrenia, we know is reduced in the prefrontal cortex.