A Landmark Collaboration in RNA Therapeutics
Merck KGaA has entered into a collaboration with Skyhawk Therapeutics focused on the development of small molecule RNA-targeting drugs for neurological conditions, in a deal that could reach a value of $2 billion.
Under the agreement, Skyhawk will apply its proprietary RNA splicing platform, known as SkySTAR, to discover small molecule candidates against RNA targets selected by Merck. The biotech will conduct discovery and preclinical studies, after which Merck will have the option to advance chosen candidates into human trials and through later stages of development.
Targeting Neurological Diseases
The companies did not disclose the specific neurological indications being addressed. However, the collaboration is intended to broaden the use of RNA modulation in diseases where traditional methods have faced difficulties.
According to the announcement, the total value of the partnership could reach $2 billion through milestone payments, in addition to tiered royalties for Skyhawk on any commercialized products. No detailed breakdown of the payment structure was provided.
Skyhawk’s Pioneering Platform
Skyhawk specializes in small molecules that regulate RNA expression inside cells. Its SkySTAR platform—Skyhawk Small molecule Therapeutics for Alternative splicing of RNA—is designed to precisely target and modulate RNA splicing, thereby influencing gene expression. Skyhawk’s pipeline is concentrated on neurology and oncology, with programs that include a splicing modulator for Huntington’s disease now in phase 2/3 trials, as well as additional candidates for lymphoma and fibrosis in preclinical stages.
The biotech, headquartered in Waltham, Massachusetts, has been steadily building partnerships since its founding in 2018. Previous collaborations have been announced with Vertex, Ipsen, Takeda, and Merck & Co. Notably, in April 2024 Skyhawk signed a $1.8 billion agreement with Ipsen in neurology, while an earlier deal with Vertex reached a potential value of $2.2 billion, including $40 million in upfront funding. Merck & Co. also entered into separate deals with Skyhawk in 2019 and 2020 covering autoimmune, metabolic, and neurological conditions.
Merck KGaA’s Strategic Move
For Merck KGaA, the deal represents a continuation of its interest in expanding its neuroscience portfolio. The company has been relatively restrained in its dealmaking this year, with only two other significant transactions announced: the $3.9 billion acquisition of SpringWorks Therapeutics in April, and the purchase of HUB Organoids B.V., a Netherlands-based drug testing company, earlier in January.
The newly announced Skyhawk collaboration adds to Merck’s efforts to advance treatments in areas of neurological disease with high unmet need through novel RNA-based approaches.
