Novel Cancer Drug PT217 Wins FDA Orphan Drug Status for Neuroendocrine Carcinoma

The novel cancer drug, PT217, has received FDA Orphan Drug Designation for treating neuroendocrine carcinoma (NEC). PT217, which targets DLL3 and CD47, offers new hope for NEC patients, typically found in the lungs and other organs.

Phanes Therapeutics has announced that its investigational drug, PT217, has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of aggressive neuroendocrine carcinoma (NEC). PT217 was previously granted ODD for small cell lung cancer (SCLC) and Fast Track designation for extensive-stage SCLC. This designation marks a crucial step forward in addressing a particularly aggressive subgroup of neuroendocrine neoplasms.

A Closer Look at PT217

PT217, a groundbreaking first-in-class bispecific antibody, targets delta-like ligand 3 (DLL3) and cluster of differentiation 47 (CD47). Neuroendocrine carcinoma, the most aggressive form of neuroendocrine neoplasms, is characterized by poorly differentiated cells and a high proliferation rate. While most NECs originate in the lungs, particularly as small cell lung cancer (SCLC), they can also develop in the gastrointestinal tract, prostate, and pancreas

This pioneering treatment has been primarily developed for patients suffering from NEC, which is often characterized by rapid proliferation and poor prognosis. NEC tumors are commonly found in the lungs as small cell lung cancer (SCLC), but can also manifest in the gastrointestinal tract, prostate, and pancreas.

Expanding Horizons

The FDA previously granted ODD to PT217 for SCLC in 2022, followed by a Fast Track designation in 2024 for treating extensive-stage SCLC that has progressed despite platinum-based chemotherapy, with or without an immunotherapy called a checkpoint inhibitor. Building on this progress, Phanes Therapeutics recently partnered with Roche to investigate PT217 in combination with Roche’s anti-PD-L1 therapy, atezolizumab, potentially enhancing its efficacy.

Phanes Therapeutics at the Forefront

Phanes Therapeutics continues to advance its innovative oncology pipeline, with PT217 at the forefront of its efforts to develop targeted therapies for patients with rare and aggressive cancers. The company is conducting multiple clinical trials, including the MORNINGSTAR, TWINPEAK, and SKYBRIDGE studies, with PT217 actively involved in the latter. 

About Author: Tracy Mokwe

With over 3 years of experience as a medical laboratory scientist and writer, I am passionate about translating complex scientific information into clear and accessible content. My expertise lies in medical sciences, laboratory science and research, digital health, AI, and cybersecurity in healthcare.