BGA-003

Throughout 2025, Biogenera S.p.A. has continued to make significant progress in advancing its lead drug candidate, BGA002, and expanding its pipeline based on Peptide Nucleic Acid (PNA) technology, while strengthening both scientific and industrial collaborations on a global scale. BGA002: From Research to Clinical Development The GMP (Good Manufacturing Practice) production process of the Active Pharmaceutical Ingredient (API) of BGA002 has been successfully completed — a historic milestone marking the first large-scale GMP-compliant production of a PNA-based molecule. This achievement represents a crucial step toward the clinical phase of the drug, developed for the treatment of aggressive pediatric cancers associated with the MYCN gene. The next step will be the fill & finish phase, consisting of sterile vial filling, expected to be completed by the end of 2025. In parallel, Biogenera’s regulatory team is finalizing the necessary procedures for the initiation of the Phase I clinical trial, scheduled to begin in early 2026, which will involve a population of pediatric patients. Expanding Orphan Drug Designations BGA002 has already received Orphan Drug Designation from both the EMA and the FDA for Neuroblastoma and Rhabdomyosarcoma. Further efforts are underway to extend the designation to additional oncological indications, with the goal of broadening the therapeutic potential of the drug and consolidating the company’s leadership in the field of rare pediatric and other severe diseases. Scientific Collaborations and International Development Biogenera continues to strengthen collaborations with leading research institutions worldwide. Among these, the University of Chicago, where joint preclinical studies are underway to evaluate the efficacy and mechanism of action of BGA002. Research activities also extend to new therapeutic areas in adult oncology, including Small Cell Lung Cancer (SCLC), for which additional preclinical developments are planned. Pipeline and New Areas of Innovation Beyond BGA002, Biogenera is advancing a diversified pipeline of drug candidates, including BGA003 in oncology and another research program in dermatology. At the same time, the company is developing the new “Longevity Initiative,” which aims to apply PNA technology to the field of longevity and aging medicine, with potential therapeutic and diagnostic applications. Industrial and Financial Outlook On the corporate side, Biogenera continues to engage in active discussions with potential international partners interested in the commercial development of BGA002 and in enhancing the value of PNA technology. A new fundraising round is also being prepared to support clinical development and to internalize the GMP manufacturing of PNA for clinical use — a strategic capability that represents one of the company’s most valuable assets and could serve as a future source of additional revenue streams. Looking Ahead Biogenera remains firmly committed to its mission: bringing new therapeutic options to children and adults affected by aggressive cancers, where treatment alternatives remain limited. Each step forward brings the company closer to its goal — transforming scientific research into tangible solutions for patients. In the coming months, Biogenera will explore each of these topics in greater detail through dedicated articles, offering deeper insight into the scientific, industrial, and human journey that drives our vision. Address: Via dell’Artigianato 2, 40064 – Ozzano dell’Emilia (BO) – ITALY Phone: +39 051 0218311 E-Mail: info@biogenera.com