Retrogenix data in regulatory applications

Background

The Retrogenix Cell Microarray technology is a powerful tool for off-target or specificity screening for a variety of different molecules, from antibodies and proteins to whole cells and small molecules. Data from High Peak’s IND-enabling specificity studies have been included in numerous regulatory applications.

To help our team and our clients better understand how Retrogenix Cell Microarray data can support BLA and IND submissions, we carry out an ongoing feedback process to follow up on specificity screening studies and find out how those molecules are progressing. We have received responses from over 80 of the clients surveyed since 2015. Many of our sponsors were unable to disclose, and therefore these numbers represent a sample of the true dataset.

*These figures were last updated in September 2021.

Overall submissions

Around one third (35%) of respondents have submitted applications to regulators including Retrogenix Cell Microarray data, and a further 13% are waiting to file applications. This report covers 78 IND submissions made to major regulatory agencies, including the FDA, EMA (and other European regulators), PMDA and NMPA (Fig.1). The remainder mainly used Retrogenix Cell Microarray data for lead selection purposes, although, some molecules were not progressed due to unexpected cross-reactivity or for other internal project reasons.

Fig.1: Retrogenix IND-enabling specificity data have been extensively used in 78 IND submissions to major regulators around the world.

Success Rate

Across the board, for all IND submissions that used Retrogenix data, 95% of submissions were approved with 5% of the applications still in the process of submission (Fig. 2).

Fig. 4: Number of each type of biotherapeutic submitted across all regions

Cross-Reactivity Screening Methods

Retrogenix data is utilised as both an alternative to and complementary to cross tissue reactivity. Until recently, standard toxicology target-binding assessment relied heavily on tissue cross reactivity (TCR) or immunohistochemistry. More recent FDA guidance states that other methods can be used. We saw that 34% of the molecules put forward for regulatory approval relied on Retrogenix Cell Microarray data only for target specificity screening (Fig. 3), with a further 66% using both Retrogenix and tissue cross reactivity data.

Fig.3: 34% of IND submissions contained only Retrogenix data for cross-reactivity assessment

Type of Molecule Submitted

Due to the wide variety of specificity screening projects performed using the Retrogenix Cell Microarray, we have seen many different molecule types being submitted to the regulators. However, antibodies or antibody-related molecules and CAR-T cells or scFvs are the predominant candidates, with a total of 39 antibody and 37 cell therapy applications being put forward for IND approval by our respondents (Fig.4).

Fig. 4: Number of each type of biotherapeutic submitted across all regions

If would like a more detailed analysis of how Retrogenix data have been used in NDA or IND submissions, or would like to discuss a potential study, please get in touch with us using our contact form below.

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