Antibody-Drug Conjugate Receives FDA Orphan Drug Designation in Pancreatic Cancer

The FDA has granted orphan drug designation to PYX-201 for the treatment of pancreatic cancer, according to a press release from Pyxis Oncology.

Patients with histologically or cytologically confirmed solid malignancies and an ECOG performance status of 0 or 1 will be eligible for enrollment in a phase 1 trial assessing PYX-201.

PYX-201 is currently being investigated as part of a phase 1 trial (NCT05720117) in patients with advanced solid malignancies. In addition to PYX-201, PYX-106 is also being evaluated in tumor-specific expansion cohorts once the dose escalation portion of the trial is complete.

“Receipt of [orphan drug designation] 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. 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,” Lara S. Sullivan, MD, president, and chief executive officer at Pyxis Oncology, said in the press release.

The intent of the phase 1 study is to identify the recommended dose of PYX-201. The first-in-human, open-label, multicenter trial has an estimated enrollment of 45 patients. PYX-201 will be administered intravenously at escalating doses to determine the agent’s safety and anti-tumor activity.

The primary end points are dose-limiting toxicities and adverse effects (AEs). Secondary end points include objective response rate (ORR), duration of response (DOR), progression-free survival (PFS), disease control rate (DCR), time to response (TTR), overall survival (OS), and number of patients with anti-drug antibodies up to 2 years.

Disease sites include non–small cell lung cancer, hormone receptor–positive breast cancer, ovarian cancer, thyroid cancer, pancreatic ductal adenocarcinoma, soft tissue sarcoma, hepatocellular carcinoma, and kidney cancer.

To enroll, patients are required to have histologically or cytologically confirmed solid malignancies with an ECOG performance status of 0 or 1. Moreover, patients needed to have at least 1 measurable lesion by RECIST 1.1 criteria except for patients with bone-only metastatic breast cancer without measurable disease. A life expectancy of over 3 months was also required.

Those with a history of another malignancy except adequately treated basal cell or squamous cell carcinoma of the skin, noninvasive bladder cancer, other adequately treated stage I/II disease currently in remission, or another cancer in remission for more than 2 years or a low risk of recurrence. Known symptomatic brain metastases in need of over 10 mg of prednisolone at the time of signing informed consent; evidence of bacterial, fungal, or viral infection; major surgery within 4 weeks of starting treatment; and prior solid organ or bone marrow progenitor cell transplantation were also grounds for exclusion.

Additionally, the phase 1 study (NCT05718557) assessing PYX-106 in patients with relapsed/refractory solid tumors has an estimated enrollment of 45 patients. The agent is also administered intravenously. The study’s primary end points are dose-limiting toxicities and AEs. Key secondary end points include ORR, DOR, PFS, DCR, TTR, and OS.

Patients with continuing AEs from previous radiotherapy or chemotherapy or the presence of grade 2 or higher peripheral neuropathy are not able to participate in the trial. Receipt of palliative care within 14 days of starting study treatment and receipt of a live vaccine within 28 days of the first study dose are additional grounds for exclusion.

Reference

Pyxis Oncology reports financial results for the first quarter 2023 and provides corporate update. News release. Pyxis Oncology. May 11, 2023. Accessed May 11, 2023. https://bit.ly/3I3WIrw