iPSirius case study: Supporting an early-stage company to navigate the health economic, non-clinical and regulatory pathway towards clinical trial authorisation

The Challenge

iPSirius SAS, an early-stage European biotechnology company developing cancer immunotherapy products using induced pluripotent stem cell (iPSC) technology, required guidance on how to progress its novel experimental cell therapy to clinical trials.

iPSirius were cognisant that many early-stage therapy developers face a number of complex and unexpected challenges along the Advanced Therapy Medicinal Product (ATMP) development pathway, and wanted to gain a comprehensive understanding of the key considerations to ensure their novel therapy would be clinically relevant, commercially viable, and have the best chance at gaining regulatory approval for clinical trials.

The Collaboration

Understanding the Cell and Gene Therapy Catapult’s unique collaborative approach and position in the UK ATMP sector, iPSirius reached out to the CGT Catapult in 2022, and the organisations worked together on a series of projects aimed to accelerate iPSirius’ iPSC-based immunotherapy towards the clinic.

A key early step for iPSirius was to determine a suitable indication to pursue that would be both clinically relevant and commercially viable. To help inform indication selection, the CGT Catapult’s Health Economics and Market Access (HEMA) experts provided iPSirius with comprehensive clinical and commercial insights, analysing factors such as production costs, potentials savings for healthcare systems and anticipated patient outcomes. This evidence, coupled with support in developing a robust target product profile and clinical strategy for their novel therapy, helped iPSirius to identify and focus on the prioritised target indication of non-small cell lung cancer.

With the target indication identified, CGT Catapult’s non-clinical support team designed a non-clinical strategy to guide iPSirius in generating the evidence needed to progress its experimental therapy through to first in human (FIH) clinical trials. This strategy covered essential considerations including the requisite pre-clinical studies to undertake and which of these would need to be performed under Good Laboratory Practice (GLP) conditions. Insights such as these will help iPSirius to optimise their time and use of resources, minimising unnecessary costs whilst ensuring regulatory compliance.

Engaging effectively with regulators was a key challenge for iPSirius, as it is with many early therapy developers, who often lack familiarity with the regulatory pathways. As the next step in the journey from therapy development to clinical trial authorisation, CGT Catapult provided regulatory advice to complement the non-clinical strategy. With the provision of robust regulatory strategies, and roadmaps demonstrating the route to patients across numerous geographies including the UK, US and Europe, iPSirius was able to communicate more effectively with regulators to maximise actionable feedback and mitigate potential risks associated with gaining approval for their clinical trial.

Where are they now?

Following the collaboration with CGT Catapult, iPSirius would like to further expand the collaboration and are anticipating a scientific advice meeting with the Medicines and Healthcare Products Regulatory Agency (MHRA) later in 2024 where they can fully push their strategy and gain endorsement for their forthcoming clinical trial authorisation (CTA) in the UK. iPSirius plans to continue to leverage the UK’s strong clinical infrastructure, extensive expertise in ATMPs and the government’s commitment to innovative healthcare solutions.

In addition, working with CGT Catapult has enabled iPSirius to increase its visibility in the UK advanced therapy market and tap into the broad network of industry leaders and potential investors.