A few weeks ago, the US Food and Drug Administration approved Abliva’s Investigational New Drug application for KL1333, enabling the start of a registrational phase II/III study with first patients due to be recruited in 2022. BioStock reached out to Magnus Hansson, CMO & VP Preclinical and Clinical Development, for a comment.

Lund-based Abliva develops treatments for rare primary mitochondrial diseases. The company currently has two ongoing, clinical-phase projects within the field: NV354 and KL1333.  NV354, the earlier program, is being developed as a chronic oral treatment of Leigh syndrome, a severe mitochondrial disease that mainly affects young children.

KL1333, the company’s latest-stage asset, targets a subset of adult primary mitochondrial disease patients suffering from multiple debilitating symptoms, including chronic fatigue and myopathy. The candidate has been evaluated in both healthy volunteers and in patients and has been granted orphan drug designation in both the United States and Europe.

The CMO comments on the positive FDA ruling

During 2021 the company has achieved several milestones within the project and recently reached perhaps the most important milestone in the KL1333-project. This was when the US Food and Drug Administration (FDA) approved Abliva’s Investigational New Drug (IND) application for KL1333, enabling the start of a registrational phase II/III study with first patients due to be recruited in 2022. Read more here.

BioStock has spoken with Magnus Hansson, CMO & VP Preclinical and Clinical Development, to get his view on the FDA ruling, and what to expect in the coming months as the company initiates the phase II/III study.

Magnus, can you start by telling us why the IND approval is so important to Abliva?

– The IND is a quality stamp for the KL1333 program, validating not only the hard work that the team has done but also the intellectual scholarship and decision making that went into designing the full nonclinical and clinical program.

– We know from our previous studies that we have a strong drug candidate in KL1333, and it’s exciting to be able to now investigate its efficacy in patients over a clinically-relevant time period. The IND means we have a ‘go’ to start the study in the US and we plan to expand to other countries as soon as we can.

Your team has chosen two primary endpoints for KL1333 in the upcoming trial, which will allow you to assess the impact of KL1333 on multiple aspects of the disease including fatigue and muscle weakness. Can you elaborate on this?

– Fatigue is the most common and debilitating symptom amongst patients with mitochondrial diseases with muscle weakness, or myopathy, coming in a close second. These symptoms severely obstruct the patient’s ability to live a normal life.

– The two disease expressions are measured in different ways, and, by including both as primary endpoints, we have optimised the chance of catching potential improvements in either fatigue or myopathy following dosing with our drug candidate KL1333.

What can you tell us about the upcoming global registrational study itself, in terms of timelines, number of patients and location?

– The study will be a randomised, double-blind, and placebo-controlled trial in up to 180 patients with primary mitochondrial diseases. Patients included will have a mutation in the mitochondria’s own DNA (mtDNA) and suffer from chronic fatigue and muscle weakness.

– Sixty percent of the patients will receive tablets containing KL1333 and forty percent will receive visually indistinguishable placebo tablets, twice daily for twelve months.

–  As discussed earlier, we will evaluate two separate primary endpoints, as well as a number of complementary secondary endpoints. Now that we have the approval to start the study in the US, we will start to submit the study for approval in other geographies with the goal to dose our first patient in 2022.

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