Phanes Therapeutics, Inc., a clinical-stage biotech company focused on innovative drug discovery and development in oncology, has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to PT217 for the treatment of neuroendocrine carcinoma (NEC).

PT217 is a first-in-class bispecific antibody targeting delta-like ligand 3 (DLL3) and cluster of differentiation 47 (CD47) being developed for patients with NEC. NEC is known to be the most aggressive subgroup of neuroendocrine neoplasms and defined as cancers with poorly differentiated morphology and a high proliferation rate. Most of them have their origin in the lung such as SCLC. Others can develop within the gastrointestinal tract, prostate, and pancreas.

PT217 was granted ODD for the treatment of SCLC by the FDA in 2022 and granted Fast Track designation by the agency in 2024 for the treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression following platinum chemotherapy with or without a checkpoint inhibitor.

Earlier this year, Phanes entered into a clinical supply agreement with Roche to study PT217 in combination with Roche's anti-PD-L1 therapy, atezolizumab.

The FDA's Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended to treat, diagnose, or prevent rare diseases that affect fewer than 200,000 people in the United States.

Orphan drug designation provides certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the US upon regulatory approval.