FDA Grants Orphan Drug Designation to KL1333 for Mitochondrial Diseases Treatment


This morning, the FDA granted Orphan Drug Designation to KL1333 for the treatment of inherited mitochondrial respiratory chain diseases (MRCD).

This morning, NeuroVive Pharmaceutical AB announced that the US Food and Drug administration (FDA) has granted Orphan Drug Designation to KL1333 for the treatment of inherited mitochondrial respiratory chain diseases (MRCD).

KL1333 is a potent modulator of the cellular levels of NAD+, a central coenzyme in the cell's energy metabolism. In preclinical models, KL1333 displayed an increase in mitochondrial energy output, a reduction in lactate accumulation, a diminishment in the formation of free radicals, and long-term beneficial effects on energy metabolism, including the formation of new mitochondria.

Erik Kinnman, CEO of NeuroVive, shared his excitement for the project’s progress. "The ODD approval by the US FDA is a validation of the quality of the KL1333 documentation to date and yet an important milestone for NeuroVive and the KL1333 project. The ODD will be beneficial to us in our efforts to rapidly document the effects and safety of KL1333 in genetic mitochondrial diseases and bring this novel treatment opportunity to the market and patients who are in great need of it.”1

The clinical development stage of KL1333 intends to record the use for chronic oral treatment in primary genetic mitochondrial disorders like MELAS, KSS, CPEO, PEO, Pearson, and MERRF. The clinical study will include 8 study arms varying in dosages of KL1333 (25 mg, 50 mg, 100 mg, 200 mg, 400 mg, 600 mg, 800 mg) with placebos. One arm will solely be administered the placebo (comparator). The estimated enrollment will include 80 participants aged 19 to 45 years with diagnosed MRCDs.2

The primary outcome of the study is to evaluate the safety and efficacy of KL1333 after a single oral dose. Secondary outcome measures include maximum plasma concentration (Cmax) of KL1333, the area Under the Curve (AUC) of KL1333, and the half-life (T1/2) of KL1333. All secondary measures will be measured from day 1 to day 15 after the initial dose.

The first participant for the first clinical phase 1 study has recently been recruited, and results are expected by June. The next clinical phase I multiple ascending dose study plans to start in the second half of 2018.

Additionally, KL1333 has obtained Orphan Drug Designation in the EU for the treatment of mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes (MELAS), which is a genetic mitochondrial disease.

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  1. NeuroVive's KL1333 Receives FDA Orphan Drug Designation for Treatment of Mitochondrial Diseases. https://www.prnewswire.com/news-releases/neurovives-kl1333-receives-fda-orphan-drug-designation-for-treatment-of-mitochondrial-diseases-300632009.html. Accessed April 18, 2018.
  2. Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers. https://clinicaltrials.gov/ct2/show/record/NCT03056209?term=KL1333&rank=1. Accessed April 18, 2018.
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