Eli Lilly is doubling down on RNA research as it pens another major biobucks pact with a small but promising biotech in the gene-silencing space.
In May, Lilly teamed up with MiNA Therapeutics to tap its small activating RNA (saRNA) technology platform for up to five targets, which remained under wraps and would be chosen by Lilly.
The Big Pharma paid MiNA just $25 million upfront, but throws in development and sales-based biobucks of up to $245 million per target, bringing the total to a cool $1.25 billion should all go to plan.
It’s using the same playbook today for ProQR, as Lilly has penned a $50 million upfront pact with $1.25 billion in biobucks for five targets using its RNA platform, dubbed Axiomer. Though staying mum on details, the biotech’s CEO Daniel de Boer told Fierce Biotech that the deal is focused on “genetic disorders in the liver and nervous system.”
RNA is finding its way into lots of R&D these days, whether it’s the messenger RNA the whole world now knows because of Moderna and BioNTech’s COVID vaccines or the saRNA from the likes of MiNA.
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But even in the normal realms of RNA tech, there are differences. Compared to other approaches focused on upregulating certain proteins (such as MiNA does) by activating the transcription of messenger RNA, ProQR’s Axiomer technology is based on RNA base editing, tapping editing oligonucleotides (EONs) to attract the ADAR editing machinery already present in human cells, which enables the body to potentially repair mutations in its own RNA and treat genetic diseases.
“This approach can potentially reverse more than 20,000 G to A mutations that are known to cause human disease,” de Boer explained. “A patient’s DNA remains unchanged, and the Axiomer technology avoids the use of complex delivery vectors, has a reversable mode of action and acts only where the target RNA is expressed.” This is the tech Lilly is paying to get involved with.
ProQR has been flying a little under the radar news-wise for the past year or so, but internally is working on its RNA therapies for genetic eye diseases, including two programs in pivotal stage for Leber congenital amaurosis (LCA), the most common genetic cause of childhood blindness, and Usher syndrome, the leading cause of combined deafness and blindness.
Back in March, the Netherlands-based biotech posted results from a phase 1/2 trial in Usher syndrome and retinitis pigmentosa that showed some benefit on multiple measures of vision.
Despite the pandemic, de Boer said his company was still able to enroll the phase 2/3 pivotal trial for lead program sepofarsen for LCA in early 2021 “and is on track for data during the first half of 2022.”