Innovation / 06.02.2025
U.S. FDA Grants Orphan Drug Designation to Ariceum Therapeutics’ Proprietary Radiopharmaceutical Cancer Therapy

• ²²⁵Ac-satoreotide is a first-in-class Actinium-labelled somatostatin receptor 2 antagonist targeting extensive-stage Small Cell Lung Cancer or Merkel Cell Carcinoma
• Orphan Drug Designation follows outstanding preclinical data and FDA IND clearance
• Phase I/II human trials set to commence in Q1 2025 as a ‘theranostic’ targeted radionuclide treatment

Berlin, Germany, 6 February 2025 – Ariceum Therapeutics (Ariceum), a private biotech company developing radiopharmaceutical products for the diagnosis and treatment of certain hard-to-treat cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ²²⁵Ac-SSO110 (satoreotide) for the treatment of patients with Small Cell Lung Cancer (SCLC).

SCLC is a deadly condition that represents a significant unmet medical need due to the limited number of treatment options available to patients with this aggressive form of cancer. Two-thirds of SCLC patients are diagnosed at an advanced stage where the disease has already spread significantly, resulting in a poor prognosis and a 5-10% overall five-year survival rate. Ariceum will commence Phase I/II human clinical development of ²²⁵Ac-satoreotide under the trial name, SANTANA-225 in Q1 2025.

Manfred Rüdiger, Chief Executive Officer at Ariceum Therapeutics, said: “Receiving ODD for ²²⁵Ac-satoreotide is a recognition of its potential as a treatment option for patients with SCLC and an important regulatory milestone for Ariceum. The FDA’s ODD will support our objective to accelerate the development of ²²⁵Ac-satoreotide through human trials to provide a potentially life-saving therapy to patients with limited alternatives.”

The FDA provides ODD to drugs and biologics that demonstrate potential for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S. The designation provides development and commercial incentives for designated compounds and medicines, including eligibility for seven years of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design, and an exemption from FDA user fees.

In October 2024, Ariceum published outstanding preclinical data demonstrating the significant potential of ²²⁵Ac-satoreotide to outperform SSTR2 targeting agonists. ²²⁵Ac-satoreotide showed a high frequency of complete durable responses and 100% survival supporting advanced clinical development in SCLC, MCC, and other aggressive cancers. ²²⁵Ac-satoreotide in combination with its companion patient selection tracer 68Ga-SSO120 is being developed as a ‘theranostic pair’ for the combined diagnosis and targeted radionuclide treatment of multiple indications expressing SSTR2, such as SCLC, MCC, and other aggressive, hard-to-treat cancers.
 

About Ariceum Therapeutics
Ariceum Therapeutics is a private, clinical stage radiopharmaceutical company focused on the diagnosis and precision treatment of certain neuroendocrine and other aggressive, hard-to-treat cancers. The name Ariceum is an anagram of ‘Marie Curie’ whose discovery of radium and polonium have been huge contributions to finding treatments for cancer.

Ariceum’s lead targeted systemic radiopharmaceutical candidate, SSO110 (“satoreotide”) labelled with Lutetium-177 (¹⁷⁷Lu) and Actinium-255 (²²⁵Ac) is an antagonist of the somatostatin type 2 (SSTR2) receptor which is overexpressed in aggressive neuroendocrine tumours (NETs) such as small cell lung cancer (SCLC) or Merkel Cell Carcinoma (MCC), all of which have limited treatment options and poor prognosis. Satoreotide is being developed as a ‘theranostic pair’ for the combined diagnosis and targeted radionuclide treatment of these tumours. Ariceum is also developing a radiolabelled PARP-inhibitor (ATT001), currently in Phase 1 clinical development under the trial name CITADEL-123. ATT001 was part of the acquisition of Theragnostics Ltd which was closed in June 2023.

Ariceum Therapeutics, launched in 2021, acquired all rights to satoreotide from Ipsen, which has remained a shareholder of the Company. Ariceum is headquartered in Berlin, with operations in Germany, Switzerland, Australia, the United Kingdom, and the United States.

Ariceum is led by a highly experienced management team and supported by specialist investors including EQT Life Sciences (formerly LSP), HealthCap, Pureos Bioventures, Andera Partners, and Earlybird Venture Capital. For further information, please visit www.ariceum-therapeutics.com.