Ariceum Therapeutics receives FDA’s Orphan Drug Designation for targeted radionuclide cancer treatment

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.

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.”

Manfred Rüdiger, Chief Executive Officer, Ariceum Therapeutics

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