For a long time, it was often said that we know less about our brains than we do about the depths of the ocean or the dark side of the moon. But in the field of drug development, that has changed.

Pharmaceutical companies have reignited their interest in the long-neglected field of psychiatry, and recent innovations in neurodegenerative diseases such as Alzheimer's disease have received FDA approval. As regulators show a willingness to consider biomarker data rather than harder-to-measure clinical outcomes, investors have also joined in, with a string of deals in the fourth quarter of last year ultimately making neuroscience the second-largest area of biopharma M&A, behind only cancer.

As CEO and Chairman of Ovid Therapeutics, a company focused on neuroscience, Dr. Jeremy Levin is at the forefront of this shift. He has been named to the PharmaVoice 100 list multiple times, serves as honorary chairman of BIO, the biotech industry's largest trade organization, and previously led Israeli generics giant Teva Pharmaceuticals. Now, in the field of neuroscience, he says, "The future has arrived."

"I have never seen a more exciting environment for drug development than this."

— Dr. Jeremy Levin, CEO of Ovid Therapeutics

Ovid's lead candidate, soticlestat, is in Phase III clinical trials for two rare epilepsy disorders—Dravet syndrome and Lennox-Gastaut syndrome. The company licensed the development and commercialization rights to soticlestat to Takeda in 2021 but remains eligible for up to $660 million in regulatory milestone payments as well as royalties on potential sales. Takeda expects to announce trial results later this year. Levin says that, if approved, the drug has a competitive advantage over similar rivals because it has been shown to be effective and "demonstrates an excellent safety profile," potentially making it a "blockbuster product."

Ovid plans to reinvest the proceeds from this deal into the company's small molecule development programs, including an early-stage selective ROCK2 inhibitor for cerebral cavernous malformations, a condition that can cause a range of neurological problems and, in rare cases, can be fatal. The company is also developing preclinical candidates for other types of epilepsy.

Levin explains that the company is positioned to pursue sustainability rather than any one-off neurological innovation. "This is not a flash in the pan," he says.

Looking ahead, Levin is focused not only on Ovid's own milestones. To understand where the science is heading, Ovid maintains an internal list of upcoming trial results, which currently includes the Phase I/II results of uniQure's Huntington's disease candidate AMT-130, as well as the full dataset from Harmony Biosciences' mid-stage study of pitolisant in myotonic dystrophy type 1.

"It would be foolish not to pay attention to where the entire field is going," Levin says. "This list gives me insight into how some of the top thinkers are designing clinical trials, what we can learn from them... and how to leverage that."

Why is neuroscience experiencing such a boom? Here, Levin describes the scientific shifts that paved the way for this pivotal moment, and why his candidate drug could have a broader impact beyond epilepsy.

This interview has been edited for length and style.

PHARMAVOICE: In a previous interview, you mentioned that the industry has the potential to achieve a "moonshot for the central nervous system." What is the moonshot you envision?

Dr. Jeremy Levin:The moonshot is a series of fundamentally new therapies. It changes the paradigm of how we think about addressing brain diseases. In epilepsy, it's not just suppressing the disease and symptoms, but curing it at the same time. In neurodegenerative diseases, it's not just managing symptoms, but addressing the basic underlying mechanisms of the disease. So, we are entering a phase where these two concepts—which were never part of everyday conversation before—are now becoming more possible.

What changes in the industry have contributed to this moment?

In the CNS field, there are new concepts about how small molecules target diseases. AI... has also completely transformed how you can target multiple targets with a single compound.

With high-resolution MRI, you can see the actual anatomy of the brain... so you can see target engagement, and then correlate that with functional EEG tests, building a complete picture of how the brain reacts. Then, you can look at the energy metabolism of glial cells, which are actually the brain's infrastructure. These are things that were not possible before. Behind this concept is a shift in science from single broad-spectrum drugs to specific treatments and cures.

"We are opening the door to how we address the root causes of inflammation in the brain."

— Dr. Jeremy Levin, CEO of Ovid Therapeutics

There are also more resources. The FDA has opened the floodgates for these drugs. Over the past eight years, neuroscience has been one of the top three areas for FDA approvals, and it was part of many approvals in 2023. Within this change, you also see... clearer thinking about how to stratify and optimize trials.

We have many reasons to dream... and there are many areas that should be of interest to all of us. In psychiatry, a host of new therapeutic modalities are being introduced, [such as] psychedelics. Then there's the idea of regenerative therapies using cell therapies... like what a company called Neurona Therapeutics is doing. We are finally seeing implantable devices... that can merge mind and machine, [and] help paralyzed patients move.

I have never seen a more exciting environment for drug development than this. It has developed over the past 10 years, bringing with it a great deal of optimism.

Do recent failures in some neurodegenerative diseases like ALS and Alzheimer's disease dampen this optimism?

The concepts behind many of these trials originated 15 years ago. So, many of these trials... have been based on a single argument for years. The whole idea that 'a disease is driven by only one biological mechanism' has been overturned.

We learn not only from these failures—often the FDA will allow the industry to advance a program even if the results are not optimal, but it is the first result that allows the industry to think about the next possibility. The best example is the approval of the first prostate cancer cell therapy in 2011. That was a complete failure; it didn't work. Like Aduhelm, it had minimal impact. But by opening the door, the FDA showed it is prepared... to approve a new therapy... as long as it is harmless and will ultimately help... and now we have over 100 companies working on cell therapies.

Ovid's lead epilepsy candidate, soticlestat, targets glutamate mechanisms, which you say are known to cause epilepsy. It also inhibits glial cell activation associated with inflammation. Since many investigational neurodegenerative drugs target inflammation, do you see broad potential for soticlestat beyond epilepsy?

The underlying mechanism and open-label data would suggest possible effects in a variety of other epilepsies. We won't over-speculate before approval.

But the underlying mechanism is likely to need exploration in many other brain diseases, because we know that many [of these] problems are caused by inflammation—from Alzheimer's disease to multiple sclerosis and Parkinson's disease. We are opening the door to how we address the root causes of inflammation in the brain. High levels of the metabolite cholesterol and overactive glial cells [may] cause this, so if you can inhibit that, perhaps there is an opportunity to improve inflammation. That's the beauty of this approach—you open a door to other possibilities.