Phanes Therapeutics’ PT217 receives FDA orphan drug designation for neuroendocrine carcinoma

Phanes Therapeutics, Inc., a clinical-stage biotechnology company dedicated to advancing oncology treatments, has achieved a significant milestone with the granting of Orphan Drug Designation (ODD) for its novel therapy, PT217. The U.S. Food and Drug Administration (FDA) awarded this status to PT217 for its potential in treating neuroendocrine carcinoma (NEC), a particularly aggressive form of cancer. This designation underscores Phanes Therapeutics’ commitment to addressing rare and complex cancers with innovative solutions.

PT217: A Game-Changer in Cancer Treatment

PT217 is a cutting-edge bispecific antibody that targets delta-like ligand 3 (DLL3) and cluster of differentiation 47 (CD47). This dual-target approach is designed to combat NEC, a highly malignant cancer known for its rapid proliferation and poorly differentiated cells. NEC often originates in the lungs, gastrointestinal tract, prostate, or pancreas, making it challenging to treat with conventional therapies.

The mechanism of action of PT217 involves its unique ability to simultaneously bind to DLL3, which is overexpressed in various neuroendocrine tumors, and CD47, a marker that helps tumors evade immune surveillance. By targeting both, PT217 aims to enhance the immune system’s ability to recognize and destroy cancer cells.

In 2022, PT217 was also granted Orphan Drug Designation for small cell lung cancer (SCLC), and earlier this year, it received Fast Track designation for treating extensive-stage small cell lung cancer (ES-SCLC) with disease progression following platinum-based chemotherapy. This Fast Track designation is intended to facilitate more rapid development and review of PT217, reflecting its potential to offer significant benefits to patients with limited treatment options.

Phanes Therapeutics has strengthened its development efforts through a clinical supply agreement with Roche, focusing on evaluating PT217 in combination with Roche’s anti-PD-L1 therapy, atezolizumab. This collaboration aims to explore the synergistic potential of combining these therapies to enhance treatment outcomes for cancer patients.

The Significance of Orphan Drug Designation

The Orphan Drug Designation by the FDA is awarded to drugs intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. This status provides several key benefits, including financial incentives for clinical development, tax credits for research expenses, and the possibility of up to seven years of market exclusivity in the U.S. following regulatory approval. These advantages are crucial for fostering the development of treatments for rare and challenging diseases.

Phanes Therapeutics’ Expanding Clinical Trials

Phanes Therapeutics continues to make strides with its robust pipeline of clinical trials. In addition to the ongoing studies of PT217, the company is advancing other promising therapies, including PT199 and PT886. PT199 is being evaluated in the MORNINGSTAR study, focusing on its potential as a best-in-class monoclonal antibody (mAb) therapy. PT886 is under investigation in the TWINPEAK study.

The company’s proprietary technology platforms—PACbody, SPECpair, and ATACCbody—are instrumental in developing novel biologics that address significant unmet medical needs in oncology. These platforms enable Phanes Therapeutics to innovate and advance its pipeline of therapies, further solidifying its position in the oncology field.

Phanes Therapeutics’ PT217 has achieved Orphan Drug Designation from the FDA for neuroendocrine carcinoma, marking a crucial advancement in the treatment of rare and aggressive cancers. With its innovative approach and ongoing clinical trials, Phanes Therapeutics is poised to make a significant impact in oncology and offer new hope to patients with high unmet medical needs.