FDA Grants Orphan Drug Designation to PYX-201 in Pancreatic Cancer

Development of PYX-201 in advanced solid tumors is underway, and the drug has grabbed the FDA's attend with a phase 1 study in pancreatic cancer.

The FDA has granted an orphan drug designation (ODD) for PYX-201 to continue its development as a treatment for patients with pancreatic cancer.A phase 1 study of PYX-201 in pancreatic cancer is currently dosing patients (PYX-201-101; NCT05720117).1

“The first quarter of 2023 was marked by the transition of Pyxis Oncology to a clinical-stage company as we initiated 2 phase 1 trials for PYX-201 and PYX-106,” said Lara S. Sullivan, MD, president, and chief executive officer of Pyxis Oncology. “Receipt of ODD for PYX-201 in pancreatic cancer is an important achievement highlighting the need for new treatment options, and we remain focused on execution as our 2 clinical programs advance.”

About the Phase 1 Study

Trial Name: A First-in-Human, Open-label, Multicenter, Phase 1 Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of PYX-201 in Participants With Advanced Solid Tumors

ClinicalTrials.gov Identifier: NCT05720117

Sponsor: Pyxis Oncology, Inc

Recruitment Contact: Loan Vuong, (339) 545 8252, clinicaltrials@pyxisoncology.com

Completion Date: July 2026

PYX-201 is an antibody-drug conjugate that is composed of a fully human IGG1 antibody, as well as a cleavable mcValCitPABC linker, and 4 Auristatin 0101 (Aur0101, PF-06380101) payload molecules.2 The agent was previously studied preclinically in animal models.

In the first-in-human, open-label, multicenter PYX-201-101 study, the safety/tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of PYX-201 will be investigated in approximately 45 patients with advanced solid tumors.

The primary end pints of the study are the number of patients who have a dose-limiting toxicity, and the number of patients who experience adverse events (AEs). The secondary end points of the study include pharmacokinetic and pharmacodynamic measures, objective response rate, duration of response, progression-free survival, disease control rate, time to response, overall survival, and the number of patients with anti-drug antibodies to PYX-201.3

Patients are eligible for study enrollment given they are aged 18 years or older, have histologically or cytologically confirmed disease, an ECOG performance status, measurable disease per RECIST v1.1, a life expectancy of at least 3 months, and tumor samples available for biopsy. The study excludes patients who have a history of another cancer, known asymptomatic brain metastases requiring 0 mg/day of prednisolone, and evidence of active systemic bacterial, fungal, or viral infection that require treatment. Patients are also ineligible for enrollment if they underwent major surgery within 4 weeks before the start of PYX-201 in the study or had prior solid organ or bone marrow progenitor cell transplantation.

Investigators are actively recruiting patients who meet the enrollment criteria at study sites in Texas and Virginia.

“We continue to anticipate preliminary data, including biomarker results and early signs of potential clinical activity, from both trials in the late-2023 to early-2024 timeframe,” said Sullivan.


1. Pyxis Oncology reports financial results for the first quarter 2023 and provides corporate update. New release. May 12, 2023. Accessed May 11, 2023. https://bit.ly/3NXjaX3

2. Yin F, DECiantis C, Pinkas J, et al Quantification of antibody-drug conjugate PYX-201 in rat and monkey plasma via ELISA and its application in preclinical studies. Bioanalysis. 2023 Jan;15(1):43-52. doi:10.4155/bio-2022-0233.

3. Study of PYX-201 in solid tumors. ClinicalTrials.gov. Updated March 9, 2023. Accessed May 11, 2023. https://clinicaltrials.gov/ct2/show/NCT05720117

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