Aptahem Newsletter March 2018

CEO comments
2018 is very much underway, and we’re really building momentum – what a start to the year we’ve had! So much has happened in the barely three months since the beginning of the year, and I look forward to the future with high expectations and great ambition. I would like to stress that a common theme in everything that we do is to create value for our shareholders, whom we hope will enjoy long-term benefit as we continue to achieve, and sometimes over-deliver on, the goals set out in our time-plan.

Before continuing, I would like to take this opportunity to welcome Dr Anders Bylock as our new Chief Medical Officer (CMO). It is a great honour to have him, with his skills and experience, in this crucial role. In the past year, Dr Bylock has done an outstanding job on our scientific advisory board, and going forward will be able to contribute his unique perspectives as we enter the clinical stage of the programme. Furthermore, I would also like to thank the outgoing Dr Kjell G Stenberg for his efforts in this post. Aptahem now has a strong team in place, and I’m proud to continue developing together.

Rapidly growing interest in sepsis
Last week, industry news outlet BioStock published an article about the US Food and Drug Administration’s push to simplify clinical data on sepsis (only available in Swedish) with the aim of enabling the development of new, effective treatments. For us, this is clearly a favourable development because we are in a highly competitive position following the positive results of our pre-clinical trials with Apta-1.

This positive trend was also evident in the considerable interest shown during recent trade shows we have attended: at Swiss Nordic Bio (February, Switzerland) we met investors and made several new contacts in Big Pharma, which we then followed-up with at BioEurope (March, the Netherlands). Luiza Jedlina, CSO, and Anders Bylock, CMO, were with me at BioEurope, and our schedule was packed with meetings. We had arranged just a fifth of these meetings ourselves, the remainder were the result of others contacting us, which suggests that more and more people know about Aptahem. The results were encouraging, and we came home with, for example, four new pharma contacts interested in our technological platform and candidate drug, Apta-1. We have also had meetings with hospital departments specialising in sepsis and acute conditions to discuss potential research work, something that in the long-term could pave the way to clinical trials. This high degree of interest is fantastic, and evidence that we are beginning to be considered as a leader in aptamers with our anti-inflammatory platform where the aptamer technology can also replace several drug types, including antibodies.

It is currently an extremely exciting time in the sepsis field, with understanding of the condition’s mechanism starting to improve among the general public. The vast, recorded number, of 31 million people who are affected by sepsis every year, means that those responsible for healthcare budgets are starting to realise that the related societal costs are high – not least due to the less effective treatments currently available. For us, Apta-1 has an obvious advantage over existing alternatives with its multi-effect mechanism that has demonstrated an efficacy – known to us – no previous drug has come close to achieving in previous sepsis studies.

According to the World Health Organization, some six million people die of sepsis every year, and many of those who survive it suffer permanent organ damage, cognitive harm, and reduced motor function. While it’s not possible to eradicate sepsis, it is entirely feasible to develop an effective treatment that could give many of these people a second chance.

Development of Apta-1
As we announced at the end of February, we have now identified a process for the production of Apta-1 in large-scale, commercial volumes, together with our partner BioSpring. Results surpassed our expectations, and the process has since been refined to further improve its effectiveness. This means that we can now move forward with GMP production, followed by several stability tests once the six-month test period has been completed. In parallel to this, we’re doing the necessary administrative work to apply for GMP certification, which is a regulatory requirement to conduct a Phase I trial.

On-going pre-clinical studies are looking in greater depth at how Apta-1 works, partly to build our basis of argumentation for Apta-1’s unique therapeutic mechanisms in discussions with potential partners, as well as strengthen our patent application, and partly to support the regulatory application to start our clinical programme in humans. For example, we’re expecting results from in vivo studies to provide meaningful insights into how Apta-1 can be expected to behave in humans. In addition, pre-clinical toxicology and safety activities are being conducted in parallel with our partner Accelera.

In short, I’m delighted to report that everything is proceeding to plan.

Business opportunities related to our Aptamer platform
I would also like to give you a snapshot of what we can expect from Aptahem, in addition to our primary Apta-1development programme. Our aptamer platform and entirely unique skillsets provide a strong basis to construct something considerably more comprehensive than our current drug candidate. Those of you who have participated in our more recent presentations have no doubt heard me say that I want to expand our pipeline. Several promising options have emerged recently, and together with the Board, I will evaluate which of these are in the best interests of the company and our shareholders. In our many business development meetings, opportunities have emerged for in-licensing or cross-licensing of complementary technologies for Apta-1. Interest also exists for Apta-2 due to it having the same effect on original symptoms and counteracting the build-up of blood clot clusters in the brain; as well as it potentially having the same or similar anti-inflammatory effects as Apta-1. Apta-2, which has a different chemical structure to Apta-1, is currently ready for testing in a variety of models to obtain a detailed understanding of its therapeutic profile, irrespective of whether these studies are financed by us or a third party. Unexpectedly, other biotech companies interested in our aptamer platform have started to contact us. The Board is therefore currently evaluating whether this can expected to be positive for Aptahem, and whether this is best achieved by a co-development project or a licensing agreement.

The opportunities are many and the future is exciting! Our goal is to derive maximum value from our development portfolio, which is why we’re carefully evaluating every co-operation opportunity prior to taking any decisions. Our meetings with potential partners will of course continue, and the co-development of ideas will expand over time, and I look forward to being able to tell you more about these initiatives in due course.

Planned activities
So, what next? We’re in the process of identifying the key industry events that we believe will be of most interest to us. Apta-1’s development continues with preparations ahead of GMP certification, as well as results from toxicology and safety studies. We’re also looking forward to several planned presentations, and opportunities to meet existing and potential shareholders as a continuous important point on our agenda. I will get back to you with specific dates and booked meetings as soon as possible!

We have a keen interest in spreading knowledge about the sepsis segment. We promote awareness among the general public about the illness by giving our support to key patient organisations Sepsisfonden and Global Sepsis Alliance. To also reach out to the scientific community, we’re currently talking to several leading periodicals in which we’re also evaluating opportunities to publish our pre-clinical results.

Furthermore, we’re continuing our on-going dialogue to engage potential co-operation partners, major drug companies and investor networks. I feel that we’re in a strong position, especially with Apta-1 that continues to deliver new positive results, and we look forward to what the next quarter can bring!

As you already know, I’m happy to answer your questions, so do get in touch via info@aptahem.com if you have any questions. I will try to answer general questions about the company in the next newsletter, otherwise I will attempt to field these by email or phone. If you haven’t done so already, you can follow us on Facebook at: https://www.facebook.com/aptahem/.

We’ll be in touch again when the next newsletter comes out at the end of May!

Malmö 20 March 2018

Mikael Lindstam
CEO, Aptahem AB

Forward-looking statements
This communication contains forward-looking statements, consisting of subjective assumptions and forecasts for future scenarios. Predictions for the future only apply as of the date they are made and are, by their nature, as is research and development work in the biotechnology segment, associated with risk and uncertainty. With this in mind, the actual outcome may deviate significantly from the scenarios as described in this newsletter.