NellOne's lead candidate for skeletal muscle wound healing and myocardial infarction demonstrates potential efficacy

Two key, independent proof-of-concept studies conducted for NellOne Therapeutics, Inc. (NellOne) demonstrate the potential efficacy of the company’s lead therapeutic candidate for both skeletal muscle wound healing and myocardial infarction.

“Following further proof-of-concept studies this year, we hope to be ready to commence preclinical testing and toxicology studies for both protein treatments derived from the Nell1 platform.”

The regenerative medicine company, which was spun out of the U.S. Department of Energy’s Oak Ridge National Laboratory (ORNL) in 2008, is developing novel therapeutic treatments that restore both mass and function to damaged human tissues, such as heart and skeletal muscle. These treatments are based on the discoveries of company founder Dr. Cymbeline Culiat, who, as an ORNL systems genetics researcher, identified the role that the Nell1 pathway plays in tissue growth and maturation.

The two efficacy studies, in vivo for skeletal muscle and in vitro for heart muscle, sought to establish the Nell1 protein therapeutic’s promise to effectively improve wound healing and to restore cardiac tissue while also preventing further damage to heart cells, explained Dr. Culiat, who also is chair of the NellOne Scientific Advisory Board.

“Evaluation of the protein for skeletal muscle wound healing in a murine diabetic model showed that Nell1 significantly increased new blood-vessel formation and muscle regrowth,” she said, adding that the results were consistent with earlier genetic and genomic studies she conducted at ORNL—that Nell1 triggers the production of several extracellular matrix (ECM) proteins that support cell proliferation and maturation in muscle and the blood-vessel network.

The cardiac study evaluated the protein as a treatment for myocardial infarction by testing if Nell1 can protect cardiomyocytes (heart muscle cells) in a low-oxygen (hypoxic) environment. “These conditions mimic what heart-muscle cells are subjected to during a heart attack,” Dr. Culiat continued.

The data showed that the Nell1 protein significantly protects cells exposed to low-oxygen conditions and prevents them from dying, she said.

NellOne CEO Tracy Warren said that the results “are important milestones on NellOne’s path to create and commercialize protein therapeutics that can be delivered to damaged tissue with the unique potential to restore both tissue mass AND function in patients recovering from heart attacks.”

“The studies are early positive indications that the Nell1 protein may be an effective therapy for restoration of functioning tissue, both in healing injured skeletal muscle after trauma or immobility, or following a heart attack. Annually, more than 1.2 million Americans suffer new or recurrent coronary attacks, with a greater-than-30-percent mortality rate,” she noted.

Warren, who also is a general partner of Battelle Ventures, which financially made possible the spinout from ORNL to commercialize the technology, added, “Following further proof-of-concept studies this year, we hope to be ready to commence preclinical testing and toxicology studies for both protein treatments derived from the Nell1 platform.”