FDA Grants Orphan Drug Status to Phanes Therapeutics' PT217 for Neuroendocrine Carcinoma

Phanes Therapeutics, Inc. (Phanes), a biotech company at the clinical stage specializing in oncology drug discovery and development, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to their product PT217 for the treatment of neuroendocrine carcinoma (NEC).

PT217 is a pioneering bispecific antibody that targets two specific proteins: delta-like ligand 3 (DLL3) and cluster of differentiation 47 (CD47). This drug is being developed for patients suffering from NEC, a very aggressive form of neuroendocrine neoplasm characterized by poorly differentiated cells and high rates of proliferation. NEC most frequently originates in the lung, specifically small cell lung cancer (SCLC), but can also develop in the gastrointestinal tract, prostate, and pancreas.

Previously, PT217 received ODD from the FDA for treating SCLC in 2022. Furthermore, in 2024, it was awarded Fast Track designation for treating patients with extensive-stage small cell lung cancer (ES-SCLC) who experience disease progression after undergoing platinum chemotherapy, with or without a checkpoint inhibitor. Additionally, Phanes entered into a clinical supply agreement with Roche earlier this year to explore the effects of combining PT217 with Roche's anti-PD-L1 therapy, atezolizumab.

The FDA's Office of Orphan Products Development is responsible for granting orphan designation status to drugs and biologics that aim to treat, diagnose, or prevent rare diseases affecting fewer than 200,000 individuals in the United States. This designation provides several benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

Phanes Therapeutics, Inc. is committed to the innovative discovery and development of oncological drugs. The company is currently conducting three Phase I clinical trials. These include the MORNINGSTAR study featuring PT199, a best-in-class monoclonal antibody (mAb) program; the TWINPEAK study with PT886; and the SKYBRIDGE study with PT217. Both PT886 and PT217 are first-in-class bispecific antibodies and have received orphan drug designation and Fast Track designation by the FDA.

Phanes has developed a strong pipeline by utilizing its proprietary technology platforms: PACbody®, SPECpair®, and ATACCbody®. These platforms are instrumental in creating novel biologics that address pressing unmet medical needs in cancer treatment.