Psilera, a biotechnology company focused on developing therapies for neurological disorders, has announced a strategic agreement with organ-on-a-chip technology leader Hesperos to accelerate preclinical development of PSIL-006, a neuroplastogen candidate targeting frontotemporal dementia (FTD). The partnership aims to leverage advanced biological modeling to address the significant unmet medical need in treating this progressive neurodegenerative disorder.
Advanced Platform Technology
The collaboration centers on Hesperos' proprietary Human-on-a-Chip® platform, which integrates patient-derived induced pluripotent stem cells (iPSCs) into interconnected, multi-organ systems. This technology has been recognized for its ability to replicate human physiological responses and provide valuable data on drug interactions across multiple organ systems.
"Our collaboration with Hesperos represents a significant step forward in our mission to develop new treatments for neurodegenerative diseases," said Dr. Jackie von Salm, Co-Founder and Chief Scientific Officer of Psilera. "Utilizing their cutting-edge platform allows us to gain deeper insights into PSIL-006's mechanism of action as we design our upcoming first-in-human trials."
The Hesperos platform has demonstrated its utility in drug development, having successfully supported multiple Investigational New Drug (IND) and Orphan Drug Designation (ODD) applications with the FDA.
Targeting Frontotemporal Dementia
PSIL-006 represents a next-generation neuroplastogen designed to address frontotemporal dementia, a progressive neurological disorder with currently limited treatment options. The compound integrates advanced neuroplastogen technology aimed at developing targeted treatments for this challenging condition.
"We are excited to partner with Psilera as they advance next-generation neuroplastogens to potentially treat a range of hard-to-treat diseases," said Dr. James Hickman, Co-Founder and Chief Scientist at Hesperos. "Our platform offers a unique opportunity to assess the compound's effects on neural tissues derived from patients with neurodegeneration, potentially accelerating the development of a much-needed therapy utilizing clinically relevant functional readouts."
Regulatory Alignment and Timeline
The partnership aligns with recent FDA policy adjustments promoting human-based lab models, including organ-on-a-chip systems that mimic heart and liver functions, as part of efforts to reduce and eventually replace animal testing in drug safety evaluations. FDA Commissioner Marty Makary noted that these approaches represent "a more efficient pipeline for novel treatments" and "an added margin of safety, since human-based test systems may better predict real-world outcomes."
This advanced modeling approach enables precise evaluation of drug efficacy and safety while potentially expediting the generation of clinic-ready datasets. The technology offers benefits not only in reducing reliance on animal testing but also in providing more clinically relevant data for drug development decisions.
Results from Psilera's preclinical modeling using the Hesperos platform are expected in Q3 2025, supporting the company's preparation for first-in-human trials of PSIL-006.
