In the Formal Advice feedback, FDA supported the existing documentation and the main elements of the clinical KL1333 development plan. The FDA furthermore recommended the company to extend and redesign the proposed KL1333 proof of concept (“PoC”) study, which would be followed by a pivotal study, into one pivotal placebo-controlled study, a recommendation the Board has today acted on. Abliva believes this decision today combined with the FDA feedback reinforces the strategic intent of the Company to focus on orphan drugs and primary mitochondrial diseases (“PMD”).
Abliva’s management has carefully considered the opportunities and recommended updating the KL1333 clinical development plan in accordance with the FDA feedback. Today, the Board has consequently decided to support the recommendations to simplify and accelerate the KL1333 clinical program by moving directly into one pivotal clinical study towards a possible market approval of a treatment for PMD.
“I am happy that we, with the one pivotal KL1333 study, potentially could reach PMD patients with a high unmet medical need much earlier than would have been the case if we were to conduct a PoC study first. Furthermore, I am delighted that the FDA accepted starting the pivotal trial in parallel with the long-term toxicology studies enabling a H2 2021 start. In view of this good news Abliva will actively pursue financing for this program including targeting international capital markets”, said Erik Kinnman, CEO of Abliva.
As a consequence of the updated KL1333 clinical development plan, 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, to enable the start of the pivotal study. These studies will run in parallel with the remaining patient portion of the ongoing phase Ia/b study. Moreover, long-term toxicology studies will be initiated that will run in parallel with the pivotal study in agreement with the FDA.