Merck has a $2bn flutter on Skyhawk's RNA platform

Germany's Merck KGaA has become the latest drugmaker to partner with Skyhawk Therapeutics, hoping to tap into the US-Swiss biotech's RNA drug discovery engine.

Only the financial bare bones of the deal have been disclosed – a total value of up to $2 billion, including upfront and milestone payments – but the focus will be on the discovery of novel RNA-targeting small molecules for neurological disorders, an area that is one of Merck's R&D priorities.

Skyhawk's SkySTAR drug discovery and development platform generates drug candidates that can be dosed orally and cross the blood-brain barrier into the central nervous system. It draws on computational biology data from public and proprietary databases, along with artificial intelligence and machine learning, to design candidate drugs that can correct RNA mis-splicing.

It is another way to go after hard-to-target disease mechanisms and – according to the biotech – is an effective way to target diseases caused by the loss of function of key proteins that are hard to address with antisense drugs or gene therapies.

Skyhawk will lead discovery and preclinical development efforts in the new collaboration, and Merck will assume responsibility for further development and commercialisation if it chooses to exercise an option on the projects.

The biotech already has a number of collaborations in place with other pharma groups, notably including France's Ipsen signed up for an alliance worth up to $1.8 billion last year that is also concentrating on neurological diseases. It is also working with Sanofi on oncology and immunology programmes, amongst other partnerships.

"Our collaboration with Skyhawk aligns with our strategic focus on innovative science and next-generation technologies that have the potential to deliver impactful medicines to patients with neurological conditions," said Amy Kao, Merck's global head of neuroscience & immunology research.

"We believe RNA splicing modulation represents an exciting frontier in drug discovery, and Skyhawk's expertise positions them as an ideal partner in this space," she added. "It is also in line with our mission in bringing more medicines to more patients, faster."

Skyhawk recently started dosing patients in its phase 2/3 FALCON-HD trial of lead candidate SKY-0515, involving patients with Huntington's disease. The drug is an investigational oral RNA splicing modulator designed to reduce the production of huntingtin (HTT) and PMS1 proteins, both thought to be drivers of the Huntington's disease process.

In a phase 1 trial involving healthy volunteers, SKY-0515 was shown to achieve reductions in HTT mRNA of up to 72%.