ProteinQure Receives Regulatory Clearance to Initiate Phase I Trial for PQ203 in the U.S. and Canada; Granted FDA Fast Track Designation

August 07, 2025 at 09:53 AM EDT

ProteinQure, a leader in computational protein drug discovery, today announced it has received regulatory clearance from both the U.S. Food and Drug Administration (FDA) and Health Canada to initiate a Phase I clinical trial evaluating the safety, pharmacokinetics, pharmacodynamics and anti-cancer activity of its lead candidate, PQ203. The FDA has also granted PQ203 Fast Track designation for patients with triple negative breast cancer, recognizing the therapy’s potential to address a serious unmet medical need.

The upcoming Phase I trial, which will be conducted in both the United States and Canada, will use an accelerated titration design to more efficiently identify the optimal dosing regimen while quickly arriving at therapeutic dose levels.

“This is a major step forward for ProteinQure and for the field of rationally designed peptide therapeutics,” said Dave Garman, VP Translation and Development at ProteinQure. “The Fast Track designation reflects the FDA’s recognition of the promise PQ203 holds for cancer patients, and we are thrilled to move swiftly into the clinic across North America.”

PQ203 is a first-in-class therapeutic developed using ProteinQure’s proprietary platform that integrates physics-based modeling with generative machine learning. The company believes PQ203 could unlock new frontiers in oncology by combining high specificity with novel mechanisms of action.

The Phase I study is expected to begin at Princess Margaret Cancer Centre in Toronto, with expansion to U.S. clinical sites later this year.

About PQ203

PQ203 is the company’s first internally owned AI designed peptide therapeutic entering the clinic. PQ203 is a novel Peptide Drug Conjugate composed of a peptide targeting the Sortilin receptor conjugated to the cytotoxic agent monomethyl auristatin E. The Sortilin receptor is expressed in a high percentage of diseased tissue from Triple Negative Breast Cancer (TNBC) patients and as such represents a novel target for this challenging sub-type of breast cancer. ProteinQure has generated data that PQ203 exhibits potent efficacy in multiple patient-derived xenograft (PDX) models including those resistant to Sacituzumab Govitecan (Trodelvy™), an antibody drug conjugate that is the emerging standard of care for metastatic TNBC.