NeuroVive has gained exclusive rights to develop and commercialize Yungjin Pharm’s Phase I-ready orphan genetic mitochondrial disorders candidate KL1333 in most of the world, the companies said today, through a collaboration that could generate up to $57 million-plus for the Seoul-based drug developer.

The companies’ global licensing agreement gives NeuroVive rights to KL1333 worldwide except for South Korea and Japan, where Yungjin retains all commercial and marketing rights.

Yungjin has received IND and a clinical study approval from South Korean regulators for a clinical study of KL1333, set to be launched within a couple of months by the company alone. NeuroVive said it will begin preparing to initiate a complementary Phase I study of the candidate in Europe and/or the U.S. in early 2018.

KL1333 is in the clinical development stage for chronic oral treatment of primary genetic mitochondrial disorders that include mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS), Kearns–Sayre syndrome (KSS), chronic progressive external ophthalmoplegia (CPEO), progressive external ophthalmoplegia (PEO), Pearson syndrome, myoclonic epilepsy with ragged-red fibers (MERRF), and Alpers syndrome. KL1333 is designed to rectify those mitochondrial defects by modulating the cellular levels of nicotinamide adenine dinucleotide (NAD+), a key co-enzyme in cellular energy metabolism.

NeuroVive says KL1333’s mode of action complements that of two of its own mitochondrial disorder candidates. One is NVP025, which is intended to protect the mitochondria in skeletal muscle from dysfunctional calcium handling and consequential muscle wasting.

The other is NVP015, which is intended to alleviate acute episodes of energy crises in genetic mitochondrial disorders with dysfunction in respiratory complex I.

“The objective is to develop an orphan drug for a series of relatively unusual childhood diseases, and as an acute therapy for compromised mitochondrial function caused by pharmaceuticals,” NeuroVive states on its website about NPV015. “There is also potential usage for large patient groups, where the body could benefit from extra energy production, in extended surgery and intensive care, for example.”

According to Yungjin, preclinical studies have shown KL1333 to increase mitochondrial energy output, reduce lactate accumulation, diminish the formation of free radicals, and thus have long-term beneficial effects on energy metabolism.

“The project is a perfect fit with our existing project portfolio in mitochondrial disorders,” NeuroVive CEO Erik Kinnman, M.D., Ph.D., said in a statement. “The addition of KL1333 is perfectly in line with our business model focusing on the development of orphan indication project all the way to the market.”

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