Piston Bio Secures FDA Alignment for First-in-Class Cancer-Related Apathy Treatment PST-101
Key Insights
Piston Bio completed a Type B Pre-IND meeting with the FDA, achieving alignment on key elements of the Phase 2 study design for PST-101 (search) in treating cancer-related apathy.
PST-101 (search) is an adenosine A2A receptor antagonist that modulates dopaminergic signaling and represents a potential first-in-class therapy for an underrecognized neuropsychiatric syndrome.
Cancer-related apathy affects patients with diminished motivation and reduced goal-directed behavior, with no approved therapies currently available for this significant unmet medical need.
Piston Bio LLC (search) announced the successful completion of a Type B Pre-IND meeting with the U.S. Food and Drug Administration (FDA) to discuss the clinical development program for PST-101 (search), a potential first-in-class therapy for cancer-related apathy. The meeting involved both the Division of Psychiatry and the Division of Oncology, reflecting the interdisciplinary nature of this emerging indication.
FDA Alignment on Phase 2 Study Design
The biotechnology company achieved alignment with the FDA on key elements of the proposed Phase 2 study design, including endpoints, patient population, treatment duration, and dose selection. The agreed Phase 2 outcome measures include the Apathy Evaluation Scale – Clinician Version, clinician and patient global ratings of disease severity and improvement, and the EORTC QLQ-C30 Role Functioning subscale.
"We are encouraged by the open and constructive tone of our meeting with FDA," said Walter Hong, MD, Founder and Chief Executive Officer of Piston Bio. "This clarity from FDA de-risks our development strategy, confirms alignment with regulatory expectations, and strengthens our position as we advance PST-101 (search) toward IND submission."
Addressing an Unmet Medical Need
Cancer-related apathy represents an underrecognized neuropsychiatric syndrome in patients with cancer characterized by diminished motivation, reduced goal-directed behavior, and impaired engagement in daily activities. The condition has significant clinical implications, as reduced motivation among patients with advanced cancer has been associated with poorer survival outcomes.
Emerging evidence suggests inflammatory signaling may disrupt dopaminergic motivation circuits in the brain, contributing to apathy. Currently, no therapies are approved for cancer-related apathy, representing a significant unmet medical need in oncology.
PST-101 Mechanism of Action
PST-101 (search) is an adenosine A2A receptor antagonist that modulates dopaminergic signaling in the central nervous system and may restore motivational function in patients with cancer-related apathy. The drug candidate is designed as a brain-penetrant small molecule targeting neuroimmune mechanisms in cancer.
Development Timeline
Based on FDA feedback from the meeting, Piston Bio plans to submit an Investigational New Drug (IND) application for PST-101 (search) in 2026. The clinical-stage biotechnology company is developing therapeutics targeting neuroimmune mechanisms in cancer, with PST-101 as its lead candidate in a pipeline aimed at improving survival and functional outcomes in patients with advanced cancer.