Merck KGaA, Darmstadt, Germany has announced a strategic research collaboration with Skyhawk Therapeutics valued at over $2 billion to discover novel RNA-targeting small molecules for neurological disorders. The partnership represents another major validation of RNA splicing modulation as an emerging therapeutic approach for challenging disease targets.
Under the agreement, Skyhawk will utilize its proprietary SkySTAR (Skyhawk Small molecule Therapeutics for Alternative splicing of RNA) platform to identify small molecule candidates directed at specific RNA targets designated by Merck KGaA. The collaboration aims to expand the potential of RNA modulation in diseases where traditional approaches have proven challenging.
Platform Technology and Approach
Skyhawk's SkySTAR platform generates drug candidates that can be dosed orally and cross the blood-brain barrier into the central nervous system. The platform 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.
According to Skyhawk, this approach provides 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. The technology represents another way to go after hard-to-target disease mechanisms in neurological conditions.
Deal Structure and Development Responsibilities
The overall deal structure includes an option agreement that grants Merck KGaA exclusive global rights to drug candidates pursued under the collaboration upon option exercise. Skyhawk will lead discovery and preclinical development efforts, after which Merck KGaA will assume responsibility for further development and commercialization upon option exercise.
The total deal value exceeds $2 billion, including upfront and milestone payments, along with the potential for tiered royalties on future commercial sales.
Strategic Rationale and Industry Context
"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, Senior Vice President and Global Head of Neuroscience & Immunology Research Unit at Merck KGaA. "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."
Bill Haney, Chief Executive Officer of Skyhawk Therapeutics, emphasized the partnership's potential: "This collaboration underscores the power of our SkySTAR platform to address challenging disease biology through precise RNA targeting, and we look forward to working closely with Merck KGaA to bring potential first-in-class medicines forward."
Expanding Partnership Portfolio
This collaboration adds to Skyhawk's growing portfolio of pharma partnerships. The biotech has an alliance with France's Ipsen worth up to $1.8 billion signed last year that is also concentrating on neurological diseases. Additionally, Skyhawk is working with Sanofi on oncology and immunology programmes, among other partnerships.
Clinical Pipeline Progress
Skyhawk recently started dosing patients in its phase 2/3 FALCON-HD trial of lead candidate SKY-0515 for 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 demonstrated the ability to achieve reductions in HTT mRNA of up to 72%, providing clinical validation for the RNA splicing modulation approach.
