Usman “Oz” Azam
Usman “Oz” Azam, MD, CEO at Empyrean Neuroscience.

Empyrean Neuroscience, Inc., a genetic engineering company dedicated to developing neuroactive compounds to treat neuropsychiatric and neurologic disorders, has launched with a $22 million Series A financing.

Based in New York City and Cambridge, U.K., the company’s genetic engineering platform is advancing a pipeline of neuroactive compounds targeting disorders of the central nervous system (CNS). Veteran biotech executives Usman “Oz” Azam, MD, leads the company as CEO, along with Fred Grossman, DO, FAPA, who is Chief Medical Officer.

Spring awakening

In this new venture, Azam, a genetic engineering expert best known for his work with Carl June, MD, of the University of Pennsylvania on CAR T-cells, has teamed up with CNS-targeted small molecule expert Grossman to tackle what they think is a clear unmet medical need in psychiatry, starting with Major Depressive Disorder (MDD).

“The best treatment available in psychiatry over the past 50 years is less than 50% effective,” said Grossman.

Fred Grossman
Fred Grossman, DO, FAPA, Chief Medical Officer at Empyrean Neuroscience.

Empyrean Neuroscience is developing a new class of therapeutics that the company is calling mycogenomic and phytogenomic molecules. Home to both plant biologists and genetic engineers, the newly launched company is developing homegrown technology without licensing out academic IP for plants and fungi to create novel therapeutics.

“The whole premise is applying genetic engineering technology that we use in oncology and T-cell engineering to species of fungi and plants for creating a whole new class of therapies,” Azam told GEN Edge. “We don’t know anybody else doing this with the focus on neuropsychiatry and neurology.”

And while there are certainly companies who are extracting neuroactive compounds like psilocybin from fungi, Empyrean is planning on giving the entire mushroom because this approach takes advantage of other native compounds, such as alkaloids, for an entourage effect. “There’s a big difference in giving the entire mushroom versus just the synthetic psilocybin,” said Grossman. “The entourage effect is an effect that has been seen in animal studies and implied in humans that may allow for these alkaloids to enhance the clinical effect that we would expect from psilocybin. We’ve never seen this in psychiatry, and that’s why everyone is leaning in.”

Good manufacturing practices for genetically modified fungi and plants

Empyrean Neuroscience’s platform has a foundation of genomics/bioinformatics and proprietary genetic engineering IP for plants and fungi. The newly launched company is applying its genetic engineering technology via vector- and CRISPR-based approaches to create plants with small molecules that either upregulate, downregulate, or silence genes like oncology.

“This platform is so exciting because you can apply genetic engineering and create concentrations of effective neuroactive molecules in plants and fungi that have never been seen before,” Grossman told GEN Edge.

Empyrean is then taking the genetically modifying species and producing a stable lineage of plants and fungi that produce consistent therapeutic molecules of interest. “We’re technically transferring that benchtop process into a GMP (good manufacturing practices) facility,” said Azam. “We’re using bioreactors with trays of genetically modified species growing to very specific regimes and times to harvest, grind, cryopreserve, analyze, and encapsulate the contents after doing the appropriate GMP analytics to provide the patient with an encapsulated product just like you will an antibiotic or any other encapsulated product.”

“I think the magical pieces that fusion of genetic engineering and cutting edge science to application neuroscience field, but once you’ve created the small molecule, it’s then straightforward drug development,” said Azam. “Once you’ve produced and understood how to GMP-scale it, the paradigm is a small molecule development program. That’s the exciting piece because that allows us to be very fast toward a clinic. We’re scaling that lead program into the clinic by the end of next year with our first IND, for which you need a GMP-validated process.”

Flying high

Azam and Grossman said that Empyrean has successfully met with the FDA regarding their lead medicine—the world’s first genetically whole-engineered mushroom product for MDD. “They’ve agreed to our plans moving forward for our clinical study, and we’ll be in the clinic in a little over a year or so,” said Grossman.

“We are taking the same approach we would for any small molecule and applying it to this pipeline, hence the preclinical studies, the pre-IND studies, and the discussions with the FDA,” said Grossman. “There’s nothing magical about what we’re doing, but we’re doing it in a clear scientific way. What’s very important is that we legitimize true drug development in this field for people.”

Grossman insists that this work is not merely theoretical as the company already has pharmacokinetic, toxicology, and cell biology studies to see if and how these compounds work to create or enhance the connections in various brain areas safely. And Empyrean Neuroscience has its eyes set on getting to clinical studies on efficacy as soon as possible.

“One of the agreements we have with the FDA is bypassing Phase I,” Grossman said. “Our study will move right into Phase II, and it’ll be a controlled study with multiple arms that can be a pivotal study as we move into Phase III.”