Yungjin Pharm's new drug candidate gets Fast Track from FDA

South Korea's Yungjin Pharm has secured Fast Track designation from the US Food and Drug Administration (FDA) for its new drug candidate KL1333, designed to treat rare and severe primary mitochondrial diseases.

The FDA's nod coincides with encouraging phase 2/3 clinical trials, bolstering Swedish partner Abliva AB's efforts to fast-track the drug's commercialization. 

The FDA granted Fast Track designation to KL1333 on Sept. 4. The drug aims to address mitochondrial diseases, rare genetic metabolic disorders that disrupt cellular energy production, affecting an estimated 12 out of 100,000 individuals, primarily in childhood.

KL1333 works by regulating the levels of the enzyme NAD+ within cells, a critical component in cellular energy metabolism. In preclinical trials, the drug not only ramped up mitochondrial energy production but also cut down the accumulation of lactic acid, affirming its potential as a long-term oral treatment for mitochondrial diseases.

In May 2017, Yungjin Pharm transferred the global rights for the development and sales of KL1333 to Abliva, with the exception of South Korea and Japan.

As per the terms of this deal, Yungjin received an initial payment of around 1.3 billion won ($1 million), followed by another installment of the same amount in May 2018, and a milestone payment of 2.6 billion won in Dec. 2021. The maximum payment under this contract could reach up to 62.7 billion won.

KL1333 previously secured Orphan Drug designation from the FDA in 2018, which promises a seven-year market exclusivity period in the US once the drug is launched. Furthermore, the FDA approved the Phase 2/3 trial plan for the drug in November last year.

Abliva initiated patient dosing in its Phase 2 trial, dubbed FALCON, in June. The trial is set to compare the effectiveness of KL1333 against a placebo in a cohort of 180 patients. Abliva anticipates an interim analysis by mid-2024 and aims for study completion by Dec. 2025.

Write to Ji-Hyun Lee at bluesky@hankyung.com