FDA Awards Orphan Drug Designation to ABD-147 for Neuroendocrine Carcinoma

FDA

The FDA has granted orphan drug designation to ABD-147 for use as a potential therapeutic option in patients with neuroendocrine carcinoma, according to an announcement from the drug developer Abdera Therapeutics, Inc.¹

ABD-147 is a next-generation radiopharmaceutical that is designed to deliver the alpha-emitting radioisotope Actinium-225 to DLL3-expressing solid tumors.² DLL3 is a protein located in the Notch pathway that is upregulated and present on the surface of cancer cells but is rarely expressed on the surface of nonmalignant cells or tissues; it is a key player in the development and regulation of neuroendocrine vs epithelial cell differentiation in the lungs making it a strong target.

“Neuroendocrine carcinomas, including small cell lung cancer [SCLC] and large cell neuroendocrine carcinoma, are aggressive and challenging to treat effectively with current systemic therapies,” Philippe Bishop, MD, chief medical officer of Abdera Therapeutics, stated in a news release.¹ “By delivering a potent radioisotope to neuroendocrine tumors expressing DLL3 with custom-engineered pharmacokinetic [PK] properties, we believe ABD-147 has the potential to become a best-in-class DLL3-targeting treatment for aggressive neuroendocrine tumors.”

Targeted radiotherapeutics like ABD-147 are comprised of the following components: a cancer cell–targeting molecule, a chelating agent that binds a radioisotope, and a linker that connects the molecule with the radioisotope. The agent was developed using the company’s Radio Optimized Vector Engineering (ROVEr™) platform.³ With this approach, the radiopharmaceutical product is custom engineered to achieve a balance of tumor uptake and penetration without rapid renal excretion and high kidney uptake. The platform allows for optimized delivery and therapeutic index of radioisotopes, which can release powerful alpha or beta particles that will eliminate cancer cells and spare healthy cells.

In July 2024, the regulatory agency awarded fast track designation to ABD-147 for use in patients with extensive-stage small cell lung cancer who had progressed on or following a platinum-based chemotherapy.⁴

The biopharmaceutical company shared plans to launch a first-in-human phase 1 trial that will explore the agent in patients with SCLC or large cell neuroendocrine carcinoma who received prior platinum-based therapy.¹

“Along with FDA recently granting fast track designation to ABD-147 for the treatment of patients with ES-SCLC who have progressed on or after platinum-based chemotherapy, this orphan drug designation for neuroendocrine carcinoma further underscores the potential of ABD 147 development to offer a significant advantage beyond approved drugs,” Bishop added in the news release.

References

1. Abdera Therapeutics announces FDA orphan drug designation for ABD-147 for the treatment of neuroendocrine carcinoma. News release. September 5, 2024. Accessed September 5, 2024. https://abderatx.com/press-releases/abdera-therapeutics-announces-fda-orphan-drug-designation-for-abd-147-for-the-treatment-of-neuroendocrine-carcinoma/
2. Pipeline. Abdera Therapeutics, Inc. website. Accessed September 5, 2024. https://abderatx.com/pipeline/
3. Platform. Abdera Therapeutics, Inc. website. Accessed September 5, 2024. https://abderatx.com/platform/
4. Abdera Therapeutics announces FDA fast track designation for ABD-147, a next-generation precision radiopharmaceutical therapy, for the treatment of patients with extensive-stage small cell lung cancer. News release. Abdera Therapeutics, Inc. June 27, 2024. Accessed September 5, 2024. https://abderatx.com/press-releases/abdera-therapeutics-announces-fda-fast-track-designation-for-abd-147-a-next-generation-precision-radiopharmaceutical-therapy-for-the-treatment-of-patients-with-extensive-stage-small-cell-lung-cancer/