Cell therapy weekly: support for commercialization of complex therapies
This week: Uniphar (Dublin, Ireland), a healthcare services business, introduces a new program to further support the commercialization journey of cell and gene therapy products, while positive preclinical data emerges for a lung cancer gene therapy. Plus, BioCardia, Inc. (CA, USA) completes its Phase III trial for an ischemic heart failure therapy.
The news highlights:
- Support for commercialization of complex therapies
- Positive preclinical data for lung cancer gene therapy
- Completion of Phase III trial for ischemic heart failure therapy
Support for commercialization of complex therapies
Uniphar, a healthcare services business, has announced its seventh cell and gene therapy program, which integrates cross-functional teams and infrastructure to aid the commercialization of complex therapies for patients worldwide. With 55 years of industry experience, Uniphar has been able to develop a platform that draws upon its expertise to support pharmaceutical and biotechnology companies to navigate the complexities of the commercialization process.
“The work that Uniphar is doing with cell and gene therapies is a perfect example of how important the right infrastructure is when it comes to bringing highly complex, lifesaving therapies to patients in a real-world setting,” commented Uniphar’s CCO, Brian O’Shaughnessy. “However, ensuring equitable access to these ground-breaking medicines is key to meeting patient need.”
Positive preclinical data for lung cancer gene therapy
Research partners of Genprex (TX, USA), a clinical-stage gene therapy company, presented positive data from studies of their lung cancer gene therapy, Reqorsa®, at the recent 024 EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics (23–25 October, 2024; Barcelona, Spain). Reqorsa®, is intended for the treatment of Ras inhibitor resistant lung cancer, mesothelioma and glioblastoma.
“We are pleased by this promising preclinical data, presented before a prestigious audience of oncology researchers, of REQORSA®’s potential in serious and difficult to treat cancers including KRAS G12C mutant lung cancer, mesothelioma and glioblastoma,” stated Ryan Confer, President and CEO of Genprex.
Reqorsa® aims to deliver the functioning tumor suppressor gene, TUSC2, to cancer cells with a negative charge while limiting uptake by non-cancerous tissue. The studies have demonstrated that the uptake of TUSC2 in tumor cells in vitro after treatment was 10 to 33 times the uptake in normal cells.
“The role of TUSC2 in lung cancer has been well established and TUSC2 restoration is being evaluated clinically. However, we are highly encouraged by our preclinical programs expanding the potential use of REQORSA® to treat a variety of cancer indications, where patient populations have unmet medical needs,” added Confer.
Positive preclinical data for lung cancer gene therapy
BioCardia, Inc. has concluded its Phase III randomized double-blind study of CardiAMP, a minimally invasive cell therapy using a patient’s own marrow cells to address microvascular dysfunction in heart failure with reduced ejection fraction, a condition affecting millions globally. The therapy is designed to increase capillary density and reduce tissue fibrosis, targeting microvascular dysfunction through the release of growth factors, cytokines and chemokines that counteract fibrotic, inflammatory, apoptotic and endothelial dysfunction.
The CardiAMP HF trial enrolled 115 heart failure patients across 18 hospitals in the United States and an addition 10 patients in an open roll-in cohort, with results anticipated by early 2025.
“Phase I and II trial results and the interim Phase III trial results have shown trends toward patient benefit across most outcome measures. These results support our belief that the CardiAMP autologous, minimally invasive, cell therapy has great promise to provide a meaningful benefit for patients suffering from heart failure,” said Peter Altman, CEO of BioCardia, Inc. “We will soon complete data monitoring and then unblind the study to perform analysis for safety and effectiveness.”
