Malmö-based biotechnology company Aptahem has announced significant progress in the development of its lead candidate Apta-1, an RNA-based aptamer designed to treat acute inflammatory and coagulation-related diseases. The company recently received a formal expression of interest from a well-reputed European pharmaceutical company, marking a potential turning point in Aptahem's commercialization strategy.
The partnership interest follows an extended period of constructive dialogue and relates primarily to Apta-1's innovative anti-inflammatory and organ-protective properties. This development comes at a strategic time as Aptahem prepares to advance Apta-1 into Phase II clinical trials.
Phase II "Basket Trial" Design Finalized
Aptahem has completed the clinical synopsis for its upcoming Phase II study, designed as an open-label, single-arm "basket trial." This innovative approach will evaluate Apta-1's efficacy and safety across multiple related conditions, including acute urogenital diseases (such as prostatitis and cystitis), inflammatory kidney diseases, acute kidney injury (AKI), and acute lung diseases.
The basket trial concept allows Aptahem to target multiple conditions that share similar inflammatory and thrombotic pathologies, potentially positioning Apta-1 as a First-in-Class treatment. Several of the targeted indications hold orphan drug status, representing significant unmet medical needs and potential market opportunities.
"The growing market focus on oligonucleotide-based treatments, where Apta-1 stands out from traditional antibodies due to its multifaceted effects and strong safety profile, further strengthens our position," said CEO Mikael Lindstam in the company's Q1 2025 report.
Strategic Manufacturing Partnership with Hongene Biotech
In April 2025, Aptahem initiated a strategic collaboration with Hongene Biotech, a global leader in RNA and oligonucleotide manufacturing with production facilities in China and Hungary. The partnership aims to develop cost-effective and sustainable production methods for Apta-1, addressing one of the key challenges in oligonucleotide drug development.
Through innovative process systems, the collaboration is expected to significantly reduce the cost of goods for Apta-1 production, a critical factor for scaling up manufacturing ahead of clinical trials and eventual commercialization.
Scientific Validation and Patent Protection
Aptahem's scientific credibility received a significant boost in late 2024 when their manuscript titled "Antithrombotic but not anticoagulant activity of the thrombin-binding RNA aptamer Apta-1" was published in the prestigious British Journal of Pharmacology. The publication, which resulted from a collaboration with Örebro University, describes the results of preclinical and mechanistic studies performed over several years.
The company has also continued to strengthen its intellectual property portfolio, recently securing patent protection in South Korea until 2038. This adds to existing protections in other key markets and enhances Apta-1's commercial value, particularly in the strategically important Asian biotechnology sector.
Increasing Industry Interest Following BIO-Europe
Aptahem's presence at BIO-Europe in Milan and the publication of its clinical synopsis have catalyzed interest in Apta-1 from multiple pharmaceutical companies. Several discussions with international biotech and pharmaceutical companies have progressed to information exchange regarding potential partnerships.
"The ongoing partnership discussions, the growing interest and diversity of stakeholders, and the progress we've made in manufacturing, regulatory, and clinical strategies all point to a very exciting future for Aptahem," Lindstam commented.
Financial Position and Rights Issue
Despite reducing operational costs in Q1 2025 to SEK 2.6 million (down from SEK 3.6 million in Q1 2024), Aptahem faced a tight cash position with just under SEK 1.3 million at the end of the quarter. To address this, the board launched a rights issue of approximately SEK 9.7 million at a subscription price of 1 SEK per share, with the subscription period running until May 30, 2025.
The proceeds will be used to support continued business development activities and position the company for potential partnerships, which have become increasingly promising with the recent formal expression of interest from the European pharmaceutical company.
Looking Forward: Apta-1's Unique Positioning
Apta-1 represents a novel approach to treating acute inflammatory conditions, with a mechanism of action that differs significantly from traditional antibody therapies. The drug candidate has demonstrated antithrombotic properties without the anticoagulant effects that often lead to bleeding complications in current treatments.
With the Phase II trial design finalized, manufacturing partnerships in place, and increasing interest from potential pharmaceutical partners, Aptahem appears well-positioned to advance Apta-1 through clinical development as a potential First-in-Class treatment for severe inflammatory conditions with significant unmet medical needs.
