MaxCyte: A win in gene silencing with APEIRON Biologics
MaxCyte’s new clinical and commercial licence agreement with Apeiron Biologics allows the use of MaxCyte’s proprietary Flow Electroporation technology for manufacturing Apeiron’s gene-silencing siRNA therapy, APN401, for clinical studies. The deal is the latest in a series of clinical and commercial collaborations in cell and gene therapy and is also one of the first in the siRNA field for MXCT. It highlights MXCT’s expertise and the value of its Flow Electroporation technology, which is rapidly being adopted as the gold standard in non-viral cell and gene engineering and enhances the likelihood of milestone payment releases. MXCT has over 100 cell therapy licences with partners, including 11 at the clinical stage, with commercial rights with aggregate milestone payments worth over US$800mln just starting to be released.
Austria-based Apeiron, a private company, has gained non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation technology and ExPERT platform to progress clinical studies of APN401, a next-generation siRNA-based checkpoint inhibitor, currently in phase II clinical development. The drug candidate has already shown preliminary clinical activity and favourable tolerability in phase I studies for solid tumours. The mechanism is based on blocking the Cbl-b master checkpoint gene which inhibits the body’s immune response to tumour growth.
The mechanism of APN401 differs from first-generation checkpoint inhibitors, which include blockbuster immunotherapies such as OPDIVO and Keytruda, by recruiting the potent immune response of T cells and natural killer (NK) cells. The approach with APN401 is to silence or lift the brakes caused by Cbl-b. This is done by engineering the patient’s own blood cells ex vivo with siRNA by means of electroporation for subsequent reinfusion. As such it has potential for treating broad patient groups and is designed to induce a transient and therefore less toxic benefit for cancer patients.
The terms of the deal include undisclosed development and approval milestones and sales-based payments in addition to other licensing fees in common with MXCT’s other 10 clinical and commercial deals. In addition, manufacturing scale-up for clinical trials allows for the generation of recurring revenues from the consumable elements and technology access required for cell engineering using the ExPERT platform.
Flow Electroporation has applications for cell engineering for drug discovery and development – for any type of cell and at any scale, providing MXCT enormous scope for deal-making. Indeed, there has been rapid development and investment into the field of biological and cellular therapies. It is interesting to note the Apeiron deal is one of the first European cell therapy deals the company has struck. This reflects sustained levels of investment in the space, driving growth and innovation. Approximately US$3bn was raised in 2019, up from $2.2bn in 2018, and there was a jump in the number of clinical trials from end-2018 from 216 to 260 by the end of 2019 across the European regenerative medicine landscape, according to the Association of Regenerative Medicine.
There are other events on the horizon, including spinout of the CARMA Cell Therapy arm via a NASDAQ-listing; the acceleration of milestone release; and the strong underlying revenues, supported by MXCT’s recent £25mln gross equity raise. We also look forward to hearing more news on the use of proceeds and its expansion strategy, all factors that can enhance the investment case for MXCT. Our SOTP valuation is £244mln.