Discovery

Capabilities Spanning Discovery to Clinical Development

From early discovery to clinical development, we integrate advanced computation, peptide engineering, and translational expertise to accelerate the path from concept to therapy.

Leveraging ProteinStudio™, we are advancing a pipeline of both internal and partnered drug discovery projects. Our goal is clear: to discover and develop novel medicines that will significantly improve patient outcomes.

Overview

A Robust Pipeline of Novel Therapeutics Addressing High Unmet Medical Need

Discovery
Hit-To-Lead
Lead Optimization
Preclinical
IND
Clinical
Advanced Solid Tumors (incl. breast cancer) Peptide-Drug Conjugate (PQ203)
Glioblastoma Multiforme Peptide-siRNA Conjugate (Intrathecal)
BBB Shuttle Peptide Conjugate (SubQ)

SORT1

Sortilin [SORT1] Delivery System

We have computationally designed and optimized high-affinity peptides to target the cell surface receptor SORT1. The Sortilin receptor is expressed in a high percentage of tumor tissue from triple-negative breast cancer patients and, as such, represents a novel target for this challenging sub-type of breast cancer.

Peptide engagement of SORT1 efficiently internalizes the peptide-receptor complex which is subsequently targeted to the endosomal compartment for cargo release. These SORT1 engaging peptides serve as a platform for cargo delivery via conjugation of the peptides to therapeutic modalities including cytotoxic agents, oligonucleotides, and radioisotopes.

PQ203

PQ203 for Advanced Solid Tumors including Breast Cancer

PQ203, our most advanced AI-designed peptide-drug conjugate, is currently in a Phase 1 clinical trial in patients with advanced solid tumors, including those with triple-negative breast cancer. PQ203 is composed of a Sortilin-targeting peptide that is conjugated to the cytotoxic agent MMAE. This lead program combines a potent payload with unique pharmacokinetics and an ideal clearance profile suited to that payload.

Our lead asset has exhibited robust efficacy in 17+ in vivo models and much of the data was shared at AACR 2024 and SABCS 2024 (click to view our posters). PQ203 is currently being assessed for safety, tolerability and initial signals of tumor inhibition in a Phase 1A/B clinical trial in Canada and the United States.

Patients who choose to participate in clinical trials play a vital role in advancing the development of new medicines by helping demonstrate their safety, tolerability, and effectiveness. Information on clinical trials for PQ203—including trial locations and eligibility criteria—can be found at www.clinicaltrials.gov. Patients interested in whether enrolling in a PQ203 trial is an option should discuss participation with their treating physician.
vehicle
Aggressive growth of triple-negative breast cancer (TNBC) tumors in the absence of drug treatment in a mouse model of human disease.
trodelvy
Marginal inhibition of TNBC tumors using current standard of care (antibody-drug conjugate) in a mouse model of human disease.
pq203
Potent inhibition of TNBC tumor growth by ProteinQure's PQ203 (peptide-drug conjugate) in a mouse model of human disease.

ProteinQure Expanded Use Statement

ProteinQure is committed to establishing the safety, tolerability, and efficacy of PQ203 through well-designed clinical studies conducted under the regulations and oversight of health authorities in regions such as Canada, the United States, and Europe. Data generated from these studies are expected to serve as the foundation for future requests for market approval, with the goal of making PQ203 available to patients who need it or may benefit from it.
At this time, ProteinQure is not providing Expanded Access to PQ203. Expanded Access will only be considered once sufficient safety and efficacy information has been obtained through clinical trials. Until then, participation in clinical studies remains the only pathway to access PQ203.

Collaborating

Partner With Us

We are also exploring a breadth of additional opportunities to design peptide-based delivery therapeutics to target cell surface receptors with tissue and cell type specificity. We are open to working with collaborators interested in delivery to tissues where we have already identified receptors amenable to our discovery process.

Our current focus includes the central nervous system, the kidney (proximal tubules and podocytes) and blood brain barrier transport.

Testimonials
It’s exciting to be part of a groundbreaking program that was discovered using the latest in computational science. Peptides open up a new and promising path for targeted cancer treatments, and we’re looking forward to seeing how PQ203 might help patients who currently have limited treatment options.
Dr. Philippe Bedard
Medical Oncologist at the Princess Margaret Cancer Centre
The success of this unique peptide-drug conjugate in diverse patient-derived mouse models is a testament to the potential of combining advanced computational techniques, precision oncology, and our labs' robust preclinical platform.
Dr. David Cescon
Medical Oncologist and Clinician Scientist at the UHN Princess Margaret Cancer Centre
Picture CreditPic: CCTG