Aptahem on the application to initiate clinical studies
Aptahem has submitted applications to the regulatory authorities and to the Central Ethics Committee in the Netherlands for permission to start a phase I clinical trial with Apta-1. According to the company the study will be initiated on schedule this year. BioStock reached out to the company’s Clinical Research Director, Suzanne Kilany, to learn more about this milestone.
Aptahem’s drug candidate Apta-1 is being developed to improve sepsis survival chances, as well as to counteract sepsis-related damage. In preclinical studies, the candidate has shown clear results on sepsis-related mechanisms, such as reducing the risk of blood clots, slowing down the body’s uncontrolled inflammatory response, the ability to repair or inhibit tissue breakdown and modulating the body’s own immune response. Therefore, the company has worked purposefully in 2022 to reach this looming milestone – initiating clinical studies.
Application to start clinical study
Recently, Aptahem submitted an application to the Dutch regulatory authority, Centrale Commissie Mensgebonden Onderzoek (CCMO), and to the responsible ethics committee in the Netherlands for permission to start a so-called first-in-human (FIH) study in healthy volunteers with Apta-1.
The agency and the ethics committee now have about 60 days to respond to the application, provided that there are no questions that need to be answered during the evaluation period. The company can now hope for a speedy approval.
The study consists of two parts. The purpose of the first part is to evaluate the safety and tolerability of Apta-1. In a follow-up second part, the effect on the symptoms (systemic inflammation) of Apta-1 will be evaluated in participants injected with LPS (the bacterial toxin lipopolysaccharide). LPS is a well-established provocation model used to induce measurable symptoms to characterise the early stages of septic inflammation
Aptahem’s Clinical Research Director comments
Leaving the preclinical stage and entering the clinical phase is an important milestone for all biotech companies, and Aptahem is no exception. BioStock therefore sought the company’s Clinical Research Director, Suzanne Kilany, for a comment on what lies ahead for the company.
Suzanne, who leads and coordinates Aptahem’s planning and preparation for the upcoming clinical phase I study, has been a board member of Apotekarsocieteten´s (the Swedish Pharmaceutical Society) section for clinical studies and has three decades of experience in clinical studies and clinical development, including various positions within pharmaceutical companies, biotech and CRO.
Suzanne, can you talk about the work that went into the applications that have now been submitted?
– It has been an intense period where different disciplines have been involved for just over one year to systematically compile several years of work and research of all preclinical data and documentation required. This is now the basis for being able to start the clinical programme.
– The submission of the application is an important milestone for Aptahem as it marks the start of the clinical programme for Apta-1, meaning that Aptahem thus becomes a clinical stage company.
What do you believe to be the main reasons that the treatment options to counteract the high mortality and injuries related to sepsis are so limited?
– First of all, it is important to identify the condition (which is difficult to diagnose) and quickly initiate the correct treatment. The treatment, which is primarily antibiotics, should be initiated within one hour of symptom onset, as the course of the disease is rapid, which may lead to organ failure. This is due, among other things, to leaking vessels with subsequent low blood pressure leading to poor oxygenation of the tissue, i.e. septic shock. Today, there is no direct treatment when the condition worsens and the organs begin to fail; the focus is rather supportive treatment to maintain a healthy blood pressure and improve oxygenation.
– In preclinical studies Apta-1 has shown a positive effect on the inflammatory condition, the so-called cytokine storm, but also to counteract tissue degradation.
On May 17, you signed an agreement with The Centre for Human Drug Research (CHDR) in the Netherlands for the implementation of the phase I studies with Apta-1. What can you say about this collaboration?
– CHDR is not only a CRO but also a research centre with state-of-the-art facilities and knowledgeable experienced researchers who have been excellent discussion partners and that has made sure to get what is required in place to achieve success, i.e. get the study approved. In addition to this, CHDR has extensive experience in LPS provocation studies.
– We also place great value on the fact that their top priority is the safety and comfort of the participants in the study.
Given that the application has now been submitted, what are the prospects for being able to start the phase I study in 2022?
– The authorities have about 60 days to evaluate the submitted application and associated documentation. We expect the study to start in accordance with the original schedule, this year.
Looking ahead, what clinical milestones and activities do you see ahead of you in 2023?
– The most important milestone is that the FIH study will be conducted according to plan. In parallel with the ongoing study, we will begin planning of the next study (phase II) in patients with the intended indication. This means, among other things, preparation of GMP manufacturing/preparation of Apta-1 and discussions with Key Opinion Leaders (KOLs).The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.