Aptahem presents a new development plan for Apta-1

Aptahem AB (publ) can announce today that the company’s board has finalized a revised development plan for the drug candidate Apta-1. The new development plan was established together with the company’s scientific advisory board who assessed Apta-1’s pharmacological properties. The largest commercial potential and synergies of Apta-1’s properties are expected to be realized by addressing emergency care sepsis patients. Sepsis is a commonly occurring and serious condition in which an overreaction in the immune system which, if not halted, can cause life-threatening organ damage and can also negatively affect blood coagulation. The primary goal of Aptahem’s new development plan is to develop Apta-1 into an acute drug to stop the damage to organs and tissues that can lead to high mortality rates in sepsis patients.

Aptahem AB (publ) can announce today that the company’s board has decided on a new development plan for its drug candidate Apta-1. With this decision, Apta-1 will refocus its primary clinical direction and thus obtain an advantageous commercial positioning in a vulnerable patient group that has few clinical alternatives.

CEO of Aptahem Mikael Lindstam comments, “Our strategic goal has always been to identify a development plan that therapeutically utilizes the unique combined activity profile of Apta-1, addresses a large clinical need, which would entail a willingness to pay among prescribing doctors, in turn attracting future collaborators. It is with great pleasure that we are finally able to announce to our shareholders, who have waited patiently, that we have now realized this opportunity.

The new development plan is the result of thorough background research by the company’s board, management, scientific advisory, and external strategic advisors. In order to determine the ideal positioning, several factors have been taken into consideration including, Apta-1’s commercial potential, its medical properties, intellectual property rights opportunities, as well as the positive outcomes of the company’s preclinical studies. The collective results have been carefully assessed by the company’s various experts and constituted the grounds for the decision to execute the development plan.

About sepsis
Sepsis is a serious and acute condition that commonly occurs and develops when the immune system reacts strongly to an infection. As a form of defense, the immune system excretes several inflammatory and anti-inflammatory substances that disturb the circulatory control which can lead to low blood pressure and reduced oxygen supply to the vital organs. The condition can be life-threatening if the infection is not controlled. Left untreated, the situation can worsen with the formation of blood clots in arteries and veins. Currently, health care lacks an effective treatment for minimizing the risk of organ failure until the infection is controlled. The interaction between inflammation and coagulation causes the usually quick development and high mortality rate in sepsis, which, as indicated by the results of preclinical studies, Apta-1 counteracts.

Despite having been identified by Hippocrates in 400 B.C., there are still no effective drugs for the treatment of sepsis on the market besides antibiotics and other supportive treatments. Acute sepsis has a mortality rate of 20-50% and is one of the leading causes of death in the West. The economic consequences of sepsis are large, amounting to USD 14.6B in the US alone, according to the Centers for Disease Control and Prevention (US). The condition and its related complications are judged to be the third leading cause of death after cardiovascular diseases and cancer.

Bengt-Åke Henriksson, PhD, who has previously worked with sepsis patients as a doctor in emergency care units, and a member of Aptahem’s scientific advisory board, comments, “If treatment with Apta-1 in humans is able to reduce the risks in a similar way to the reduction seen in animal studies, it can become a valuable treatment for sepsis. The data indicates not only increased survival rates, but also the maintenance of blood circulation and other vital functions, which are crucial clinical aspects for the treatment of these vulnerable patients both during and after the illness.”

Due to the combination of anticoagulation and anti-inflammation effects in its profile, Aptahem considers Apta-1 to be suitable for preventing the development of organ damage that leads to the high morality in this vulnerable group of patients. Results from studies in clinically relevant models for sepsis have shown that in non-toxic doses, Apta-1 significantly improves survival and increases and restores blood circulations in test animals when Apta-1 is administered intravenously. Intravenous administration of Apta-1 also resulted in a significantly positive effect on biomarkers related to coagulation and inflammation.

The market for acute treatment of sepsis with Apta-1
The Centers for Disease Control and Prevention (USA) estimates that more than 1.1 million patients with sepsis as a primary or secondary illness arrive to the emergency care unit yearly. The number of new sepsis cases is slightly lower in Europe and Japan, with approximately 817,000 patients and 100,000 patients per year, respectively. In total, using conservative estimates and pricing within the indication area, the market is estimated to value approximately USD 10B annually. Aptahem considers Apta-1 to have the potential to achieve a strong position on the market and obtain a large market share, since there is currently no equivalent drug on the market.

Development plan and timeline
Aptahem has recently announced its new agreement with the German company, BioSpring, one of the world’s leading producers of oligonucleotides. According to the new development plan, BioSpring is carrying out the substance development of Apta-1 which is estimated to be completed by Q2 2018. The company will perform a toxicological evaluation up until Q3 2018, which will provide the basis for the application for clinical testing to the Medical Products Agency (Sw. Läkemedelsverket). In parallel with the toxicological evaluation, the company will develop additional analytical measurement methods and Apta-1 will be evaluated in two separate stability studies in accordance with the clinical framework. The goal of Aptahem is to submit the initial application to begin clinical phase 1 tests during Q4 2018 and to initiate a clinical study in Q1 2019.

For further information:
Mikael Lindstam, CEO, Aptahem AB
Tel: + 46 (0)766-33 36 99
E-mail: ml@aptahem.com

About Aptahem
Aptahem AB (APTA) is a biotechnology company that develops aptamer-based pharmaceuticals for the treatment of life-threatening conditions in which a combination of coagulation and inflammation are involved. The company’s primary pharmaceutical candidate, Apta-1, is being developed with the aim of preventing the high mortality rate caused by organ and tissue damage in sepsis patients, among others. The company possesses patent protection in strategic target markets and actively seeks business development opportunities with potential collaborators.

This information is information which Aptahem AB (publ) is obliged to publish under the EU Market Abuse Regulation. The information was provided by the auspices of the above contact person, for publication on 11 August 2017.