The Malmö-based biotechnology company Aptahem began its first clinical trial with the drug candidate Apta-2022, an emergency treatment for sepsis, in 1. Sepsis is a potentially life-threatening inflammatory condition that affects nearly 50 million people annually, of which 11 million die.
Despite this, there is still no specific, especially acute, treatment. Rather, care focuses on treating the underlying symptoms, with antibiotics, anti-inflammatory treatments, fluids, and oxygen. However, not all patients respond to these treatments, and the risk of tissue and organ damage remains.
Aptahem's goal is therefore to fill this medical vacuum and position Apta-1 as the world's first specific emergency treatment for sepsis.
First-in-human study initiated in December 2022
After several years of preclinical studies, Aptahem began its first phase I study on December 5, 2022. It is divided into a phase Ia and a phase Ib part, led by The Center for Human Drug Research (CHDR) in the Netherlands.
The study evaluates the safety, tolerability and pharmacodynamic effects of Apta-1 in healthy volunteers. Aptahem is conducting the study in several study cohorts and on March 6, 2023, cohort three was completed. When the Phase Ia part is completed, Aptahem intends to initiate a Phase Ib study, hopefully already this year. The goal is to complete the upcoming Phase Ib study during the fourth quarter of 2023 and to communicate preliminary results during the beginning of 2024.
Comments from the CEO
In parallel with the Phase I study being ongoing and following the planned timeframes, Aptahem expanded its focus on making the business and Apta-1 visible to potential partners. BioStock contacted the CEO Mikael Lindstam to find out more about his impressions from the conferences he has attended in recent months.
Mikael, how is your participation in conferences different today compared to two years ago?
– To some extent, it differs in that the people you are trying to meet to present Apta-1 and that the company is expanding as we reach milestones and we are now in the clinic. Some we have had long-term contact with and others are completely new. Some potential takers are changing their strategies, which either end up being more focused on Apta-1's qualities or the fact that we are in the clinic, while others are starting to look at the early clinical phase. Interests and trends, as well as their strategies, change quite regularly.
– This has opened up greater interest in Apta-1, but not only that, but also interest in the company itself, where meetings with various financial stakeholders have increased. Today, we are looking more outside the usual ranks to identify potential partners or licensees who are narrower in their search area than the very largest pharmaceutical elephants, even if we meet with them. All to increase the possibility of getting a stable deal and also with the right partner.
You have attended a number of high-profile events over the past six months. Can you tell us about some of the highlights and the interest you have seen in Apta-1?
– Participation at TIDES was important given the world we find ourselves in with our RNA therapeutic candidate Apta-1, where the synthetic oligonucleotide (oligo) world is constantly growing as well as the increased interest in this type of chemical compounds and its several advantages compared to, for example, antibodies. The area has received a real boost since Covid and its RNA-based vaccines, which are, however, biologically produced. There is also a lot happening on the manufacturing side due to the increased need for RNA-based drug candidates.
– Aptahem can also benefit from this by creating even more efficient manufacturing methods and reducing costs while improving quality. Interest in synthetic oligos/aptamers is gaining more and more interest in countries such as China, Japan, South Korea, etc. as they are very curious about disruptive solutions in drug development. But, as I said, there is generally an intensified interest not only there but also in the USA and Europe.
– We take home new contacts, impressions and also how we should rhetorically work with aptamers, and one of them is to use the word RNA therapeutics to reach out to more people. I just got back from BioEurope and I also participated in the digital event that took place this week. This time the physical event exceeded expectations with both new and old acquaintances. Many of whom have now changed their focus to being more interested in projects in the early clinical phase, where we are. There was also a large proportion of participating financial players and overall the meetings we held had a good focus with informed stakeholders.
How is the ongoing Phase Ia study progressing?
– It is proceeding according to plan! We are approaching the end of the study and look forward to compiling the preliminary results.
What are your expectations for the upcoming development stages?
– It will be exciting as the next steps will focus partly on mapping Apta-1's potential therapeutic effect through phase Ib studies in toxin-stimulated healthy volunteers. This is to get an indication of how we want to position ourselves for phase II in patients. These are important steps in minimizing risk for phase II and at the same time showing the therapeutic potential as early as possible and increasing interest in a license/partner deal.