Abliva AB (Nasdaq Stockholm: ABLI) today announced that it has received positive feedback from the US Food and Drug Administration (“FDA”) on its KL1333 clinical development plan for the treatment of primary mitochondrial disease (PMD) at a pre-Investigational New Drug (“pre-IND”) meeting. Feedback was received on the existing KL1333 documentation to date and the remaining development plan, including the design of the clinical efficacy program in primary mitochondrial disease patients.
The FDA Formal Advice feedback supports the existing documentation and the main features of Abliva’s plan to develop KL1333 towards approval for primary mitochondrial disease. The clinical KL1333 development plan will target patients with genetically confirmed MIDD-MELAS or KSS-CPEO spectrum disorders with multi-organ systemic symptoms. Moreover, the endpoints in Abliva’s proposed clinical efficacy study will include evaluation of efficacy with regard to patient reported outcomes, as well as biomarkers of pharmacodynamic response.
“This is important feedback, for our KL1333 clinical program. It is also reassuring that it is aligned with the external expert input we have received and our internal plans. The next steps in the regulatory process are the submission and approval of an IND that will enable initiation and conduct of a clinical efficacy study with KL1333 in the US, and starting a similar dialogue with European regulators, said Magnus Hansson, Chief Medical Officer at Abliva
“The positive feedback from the FDA validates the preclinical, product development, and clinical documentation to date. Also, it is an additional step forwards in our efforts to deliver a needed new treatment to PMD patients”, said Abliva´s CEO Erik Kinnman.