GSK is embarking on a journey with CAMP4 Therapeutics, paying $17.5 million upfront to collaborate on regulatory RNA-targeting therapeutics in neurodegenerative and kidney disease indications.
CAMP4 is developing antisense oligonucleotides (ASOs) that hit regulatory RNAs (regRNAs). By engaging the nucleic acids, the biotech aims to boost expression of genes implicated in a range of diseases. CAMP4 sees its approach as a good fit for diseases driven by genetic mutations that either cause one gene allele to stop functioning or cause both alleles to function poorly or not at all.
GSK has secured the chance to work with CAMP4 on multiple gene targets in two therapeutic areas. The Big Pharma is paying an upfront fee and committing to development and commercial milestones, plus tiered royalties on future product sales, although it did not provide a breakdown of those costs.
In return, CAMP4 will use its platform to identify regRNAs that control the expression of multiple gene targets. After finding targets, the biotech will create regRNA-targeting ASO candidates that amplify target gene expression for potential development. GSK will develop and commercialize the candidates.
For GSK, the deal offers a chance to find a new use for one of its existing preferred modalities. The drugmaker partnered with Ionis, then called Isis Pharmaceuticals, to access antisense technology in 2010 and has continued to invest in the modality. GSK’s work with Ionis has yielded a phase 3 hepatitis B asset. The Big Pharma has penned deals with Elsie Biotechnologies, Empirico and Wave Life Sciences.
Chris Austin, M.D., senior vice president of research technologies at GSK, said in a statement that the CAMP4 deal will leverage his team’s expertise in therapeutic oligonucleotides, genetics and advanced laboratory and data technologies. GSK is pairing its capabilities with CAMP4’s knowledge of how to increase specific gene activity.
CAMP4 tried to provide early clinical validation of its platform by testing CMP-001 in healthy volunteers. However, the attempt to show the candidate had promise in urea cycle disorders generated unclear data. CAMP4 was unable to determine whether there was pharmacodynamic activity. The uncertainty led CAMP4 to switch its attention to a preclinical program and seek a partner for CMP-001.
The biotech is advancing its new lead candidate, CMP-002, while working with BioMarin on assets that engage regRNA sequences for two genetic targets. BioMarin partnered with CAMP4 last year. The deal is worth $1 million upfront plus up to $185 million per program in milestones.