Aptahem comments on the paused phase I study

BioStock published an article on 24 July 2023 about Aptahem, which can be read in full below.

Aptahem develops the drug candidate Apta-1 as an acute treatment of sepsis. Recently, the company announced that the ongoing phase Ia study had been temporarily paused after observing interesting data. To learn more about the significance of this data, BioStock contacted Aptahem’s CEO Mikael Lindstam.

Aptahems drug candidate Apta-1 targets several key factors in sepsis to reduce inflammation and restore balance in the immune system. Sepsis is a serious condition that starts with an overactive immune response to an infection, which can lead to multiple organ failure and death.

The ongoing clinical phase I study is divided into two parts: phase Ia and phase Ib. During the spring, the company conducted four cohorts in phase Ia. In the upcoming phase Ib study, the company intends to evaluate the effect of Apta-1 on a provoked inflammatory response in healthy volunteers. Phase Ib aims to minimise the risk ahead of the upcoming phase II study, which will investigate the effect of Apta-1 in patients.

The CEO comments on the paused study
Aptahem has previously aimed to complete the phase Ia study during the summer of 2023, followed by a phase II study next year and a pivotal phase III study in 2026.

However, on July 17, the company announced that it had made “interesting findings” in the phase Ia study, and after reviewing this data, the Data Review Committee recommended Aptahem to perform further analyses. This means that the study has now been temporarily paused.

To find out more about the observed data that has led to the study being paused, BioStock contacted Aptahem’s CEO Mikael Lindstam for a comment.

Mikael, can you elaborate on what kind of interesting finds you have made?
– In analyses performed during the course of the study with dose escalation in cohorts 1 to 4, these have shown signs that Apta-1 affects more markers than we have seen in previously performed animal experimental studies. Since safety comes first in clinical studies, the Committee has recommended that we investigate these further to gain as thorough an understanding as possible of how Apta-1 acts in humans. We see this as an opportunity to understand and explain more aspects of how our drug candidate’s mechanism of action works.

Do you believe that Apta-1 has no potentially negative effects that could hinder its future administration to patients?
– After each completed study cohort, the authorities and the ethics committee reviewed the results to ensure that it was safe to proceed to the next cohort. Since this is groundbreaking research, and given the new and complex mechanism of Apta-1, unexpected findings can turn up. The regulatory authorities also do not have much experience with the type of substance that Apta-1 constitutes. Therefore, more in-depth knowledge of Apta-1 in humans is needed and so far we do not see anything that indicates that it would jeopardise any further studies on patients in the future.

How long do you believe the further analyses will take and will this affect the timelines for future development?
– We work with our CRO to carry out the analyses and they in turn have sub-consultants for the various specific parts of this work. Now it is also summer and holiday time, which means that it is difficult for us to give any time estimate. The only thing I can say is that this is a high priority and everyone is working as fast as they can.

Finally, can you describe your overall strategy to ensure the successful commercialisation of Apta-1?
– Our strategy is to conduct the early clinical studies with Apta-1 in a strategic, safe and cost-effective manner to ensure and offer as fully packaged a project as possible for a future licensing partner. With this future partner and its capabilities, our goal is to reach the market together to improve and save the lives of many patients. An example of how we work strategically is the planned phase Ib study where we may see an effect at an early stage and also identify the optimal dose level for the phase II study.

Read the article on biostock.se

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