Abliva AB (Nasdaq Stockholm: ABLI) today announced that the first primary mitochondrial disease patient in the company’s ongoing KL1333 Phase Ia/b study has been dosed. In this third part of the study, the pharmaceutical properties of KL1333 will, for the first time, be evaluated in patients.
Eight patients with primary mitochondrial disease will receive a daily dose of KL1333 for ten days, primarily for assessment of safety and pharmacokinetics of the candidate drug. In addition, biomarkers and clinical outcome measures will be evaluated ahead of the planned clinical pivotal Phase II/III study. Abliva’s Phase Ia/b clinical study is conducted in the UK (London and Newcastle).
“The dosing in the first patient in our KL1333 program is a significant step in the development of a disease modifying oral treatment for patients with primary mitochondrial diseases. This important event follows the recent announcement of our intention to start a pivotal trial with KL1333 in 2021. Primary mitochondrial diseases are devastating diseases for which there are no existing pharmaceutical treatments and we are excited by the opportunity of bringing a novel treatment to the market and to patients with unmet medical needs”, said Erik Kinnman, CEO at Abliva.
In parallel with running the patient portion of the Phase Ia/b study, the company will continue to prepare for the upcoming pivotal Phase II/III efficacy study with KL1333 planned to start H2 2021. Abliva will during H2 2020 initiate a qualitative study validating the specific patient-reported outcome measures, as well as a drug-drug interaction study in healthy volunteers. Moreover, long-term toxicology studies will be initiated that will run in parallel with the pivotal study in agreement with the FDA.