Genetic Mitochondrial Disease Therapy KL1333 Enters Phase 1 Studies Following NeuroVive-Yungjin Accord

KL1333, a new potential treatment for orphan genetic mitochondrial disorders, is entering Phase 1 studies, thanks to an accord between NeuroVive and Yungjin Pharm.

Under terms of the contract, Sweden’s NeuroVive gains exclusive worldwide rights to develop and commercialize KL1333 except in South Korea and Japan. The deal could generate up to $57 million for the South Korean drug developer.

KL1333 acts by modulating the levels of the enzyme nicotinamide adenine dinucleotide (NAD+), thereby rectifying mitochondrial defects.

“The NeuroVive team is extremely pleased to acquire the rights to the clinical stage candidate drug KL1333 for genetic mitochondrial disorders, an area of high unmet medical need. The project is a perfect fit with our existing project portfolio in mitochondrial disorders,” NeuroVive CEO Erik Kinnman said in a press release. “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. We look forward to working with Yungjin Pharm on this opportunity.”

Mitochondrial diseases can arise from mutations either in mitochondrial DNA or in nuclear DNA encoding proteins aimed at the mitochondria. Currently, these diseases have no cure, though researchers are developing treatments to ameliorate a subset of the symptoms. NAD+ is a key player in metabolism, playing an essential role in the mitochondrial respiratory chain.

NeuroVive says that in preclinical studies, KL1333 increased mitochondrial energy output, reduced lactate accumulation, diminished the formation of free radicals and showed long-term beneficial effects on energy metabolism.

Yungjin has received an investigational new drug license and approval from South Korean regulatory agencies to launch a clinical study of KL1333 in the next few months. The company also expects to start a new clinical trial in Europe or the United States in early 2018.

“With NeuroVive’s specialist expertise in mitochondrial medicine and clinical development experience, we consider them an ideal partner for Yungjin Pharm, and with this agreement global development of KL1333 has been secured,” said Yungjin CEO and President Sujun Park.

KL1333 is now being evaluated in a first in human double-blind, placebo-controlled, Phase I clinical study (NCT03056209). The study, being conducted in South Korea in healthy volunteers, is investigating the drug’s safety, tolerability and pharmokinetics after a single oral dose.