Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage biopharmaceutical company developing medicines for the treatment of rare and severe primary mitochondrial diseases, today announced it has received positive feedback from the UK Medicines and Healthcare products Regulatory Agency (MHRA) on the accelerated clinical development plan of KL1333 in primary mitochondrial disease (PMD). The feedback positions Abliva for a clinical trial approval also in the UK, of its pivotal clinical Phase II/III study, planned to start in the second half of 2021.
The company recently received positive recommendations from the US Food and Drug Administration (FDA) leading to the current plan to conduct a pivotal Phase II/III study.
“The positive feedback from MHRA further validates Abliva’s decision to advance the development of KL1333, our primary mitochondrial disease asset with blockbuster potential, directly into a pivotal clinical study, a decision that will potentially cut down time to market approval by up to two years. The support from both the US and UK regulatory authorities is very beneficial in our efforts to bring a much-needed novel treatment opportunity to PMD patients”, said Abliva´s CEO Erik Kinnman.
The pivotal Phase II/III study will target patients with genetically confirmed MELAS-MIDD or KSS-CPEO spectrum disorder with multi-organ systemic symptoms. Preparatory activities are ongoing, including the patient portion of the Phase Ia/b study, a drug-drug interaction study in healthy volunteers, and collection of data from the UK patient registry study MitoCohort. In addition, the Company will perform a study validating patient-reported outcome measures, a dosing study in healthy volunteers, and long-term in vivo toxicology studies.