FDA Grants Orphan Drug Status to Ariceum's Radiopharmaceutical Therapy

Ariceum Therapeutics, a private biotechnology firm headquartered in Berlin, has announced a significant milestone in the development of its radiopharmaceutical product, 225Ac-satoreotide. This innovative treatment has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its potential use in treating Small Cell Lung Cancer (SCLC), a highly aggressive cancer with limited treatment options.

Small Cell Lung Cancer is notoriously difficult to treat, with a majority of patients being diagnosed at an advanced stage, leading to a bleak five-year survival rate of just 5-10%. The recognition by the FDA underscores the pressing need for new therapeutic approaches for this rare and aggressive form of cancer. Ariceum plans to initiate Phase I/II clinical trials of 225Ac-satoreotide, under the trial name SANTANA-225, in the first quarter of 2025. This trial aims to assess the efficacy and safety of the treatment in humans, following promising results from preclinical studies.

Manfred Rüdiger, CEO of Ariceum Therapeutics, expressed his enthusiasm about the FDA's decision, noting that the Orphan Drug Designation is a crucial step towards accelerating the development of 225Ac-satoreotide. This designation not only highlights the drug's potential but also offers Ariceum various incentives, including eligibility for seven years of market exclusivity upon approval, assistance from the FDA in designing clinical trials, and exemption from FDA user fees. These benefits are designed to encourage the development of treatments for diseases that affect fewer than 200,000 people in the U.S.

The preclinical data for 225Ac-satoreotide, published in October 2024, revealed its efficacy in targeting the somatostatin receptor 2 (SSTR2), which is overexpressed in aggressive neuroendocrine tumors such as SCLC and Merkel Cell Carcinoma (MCC). The treatment demonstrated a high frequency of complete, durable responses and a 100% survival rate in preclinical models, providing a strong foundation for its advancement into clinical trials. Additionally, 225Ac-satoreotide is being developed in conjunction with its companion diagnostic tracer, 68Ga-SSO120, as a 'theranostic pair.' This combination is intended to enable both the diagnosis and targeted radionuclide treatment of various cancers expressing SSTR2.

Founded in 2021, Ariceum Therapeutics has quickly established itself as a leader in the field of radiopharmaceuticals. The company acquired the rights to satoreotide from Ipsen, which remains a shareholder, and has expanded its operations across Germany, Switzerland, Australia, the United Kingdom, and the United States. Ariceum's commitment to innovation in cancer treatment is further exemplified by its development of a radiolabelled PARP-inhibitor, ATT001, which is currently in Phase 1 clinical trials.

Ariceum's management team is composed of highly experienced professionals and is backed by prominent investors such as EQT Life Sciences, HealthCap, Pureos Bioventures, Andera Partners, and Earlybird Venture Capital. This strong foundation supports the company's mission to advance the diagnosis and treatment of challenging neuroendocrine and other hard-to-treat cancers, offering hope to patients with few existing options.