RALEIGH – Aruna Bio, a Georgia pharmaceutical company with a growing Raleigh presence, has been awarded a $4.5 million grant from the National Institutes of Health to support its development of a potential stroke treatment.

The Phase 2 Small Business Innovation Research funding reflects NIH interest in Aruna’s developing platform using microbiologics called neural exosomes to treat neurodegenerative disorders.

Exosomes are micro-sized naturally occurring biologics that are produced by stem cells. They play a central role in the body’s important cell-to-cell communication.


The company says the grant will support research and development leading up to an Investigational New Drug application for the use of neural stem cell-derived extracellular neural exosomes to treat acute ischemic stroke.

Acute ischemic stroke occurs in nearly 800,000 people annually and is a leading cause of long-term disability in the U.S. There is currently a high unmet need for new therapies, as 90 percent of all ischemic stroke patients do not meet the inclusion criteria for use of current treatment interventions and suffer poor functional outcomes resulting in death or severe long-term impairment.

Attempts to develop effective therapeutics for stroke have led to many failures. But, in multiple pre-clinical models, the Aruna-developed neural exosome AB126 demonstrated improved outcomes and represents a potential novel approach to stroke treatment.

Grant funds progress to IND

The NIH provided a $2.5 million Phase 1 grant to Aruna several years ago. “This second grant allows us to continue to progress our neural exosome, AB126, to an IND filing which will position us to begin our first human studies,” said Mark Sirgo, Pharm.D., Aruna CEO.

In an interview with the Biotech Center, Sirgo said, “This grant allows us the significant advantage of advancing this product with non-dilutive dollars. It will allow us to do the preclinical toxicology animal studies” necessary for the IND. “An NIH grant in some respects validates what we’re doing,” Sirgo said. “You have to go through a rigorous process to get a grant of this size.”

Mark Sirgo (Aruna photo)

Aruna’s proprietary neural exosomes are particularly promising for neurodegenerative diseases and stroke treatment because they can cross the blood-brain barrier. Sirgo said many see them primarily as a delivery vehicle, “Which they are, but they also have therapeutic properties. They are anti-inflammatory, neuro-protective and neuro-regenerative. They could offer lots of possibilities as a therapeutic.”

He added, “We could also load other therapeutics onto these and deliver them across the blood-brain barrier.”

Sirgo pointed out that Aruna is not primarily focused on stroke and is continuing its work on treatments for neurodegenerative diseases. “We continue to position our internal resources on development programs using our neural exosome platform for the treatment of neurodegenerative diseases including amyotrophic lateral sclerosis, or ALS,” he said.

The company was founded by Chief Scientific Officer and Founder Steven Stice, Ph.D., who Sirgo said spent 15 to 20 years working in neural stem cell research and about five years ago realized the potential therapeutic value of neural exosomes. The company is a spin-out from the University of Georgia in Athens. Stice remains actively involved with the Aruna’s research.

Sirgo said the solid science and deep expertise of Stice is one reason Sirgo has returned to the startup scene. Sirgo retired in late 2017 as president and CEO of Raleigh-based BioDelivery Sciences, which he led through three U.S. Food and Drug Administration approvals and subsequent commercialization. That was after 16 years of leadership positions with Glaxo, Glaxo Wellcome, and GlaxoSmithKline.

“If I was going to do this again, I wanted to be someplace where I could make a difference,” Sirgo said. “This platform has that ability.”

Aruna raised $5.3 million from angel investors in 2018 and $13 million in July, 2019 from existing and several institutional investors in an oversubscribed round. The company has a total of 25 employees, with three in the Triangle, where it is looking to recruit a few others for the management team, Sirgo said.

Aruna Bio is also leveraging its proprietary exosomes and manufacturing platform to create synergistic therapies by enhancing exosomes with RNA, oligonucleotides, antibodies and small molecules.

 (C) N.C. Biotech Center