US-based biotech company Skyhawk Therapeutics has teamed up with Merck KGaA, Darmstadt, Germany, to discover RNA-targeting small molecules for neurological disorders.
The partnership, valued at over $2bn, aims to advance RNA modulation in diseases where traditional approaches have proven challenging.
It will combine Skyhawk’s RNA splicing expertise with Merck’s drug development capabilities.
Under the collaboration, Skyhawk will leverage its SkySTAR platform to identify small molecule candidates aimed at RNA targets specified by Merck.
The biotechnology company will lead discovery and preclinical development, while Merck will handle further development and commercialisation.
Skyhawk is eligible for milestone payments and tiered royalties from Merck.
Merck senior vice president and neuroscience and immunology research unit global head Amy Kao said: “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.
“It is also in line with our mission in bringing more medicines to more patients, faster.”
Skyhawk Therapeutics CEO Bill Haney said: “We are proud to partner with Merck, a company with a strong heritage of innovation and a commitment to advancing transformative science.
“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 to bring potential first-in-class medicines forward.”
In a separate development, Merck announced that the European Commission (EC) has authorised Ogsiveo as monotherapy to treat desmoid tumours in adults.
Developed by SpringWorks Therapeutics, a healthcare company of Merck, Ogsiveo is an oral, selective, small-molecule gamma secretase inhibitor.
It is the first approved therapy to treat desmoid tumours in the European Union (EU).
The EC approval is based on the Phase 3 DeFi trial, which showed significant improvements in progression-free survival and response rates.
In the Phase 3 DeFi trial, Ogsiveo has significantly reduced the risk of disease progression by 71% compared to placebo.
The therapy also improved objective response rates and patient-reported outcomes, including pain and quality of life.
Ogsiveo showed a manageable safety profile, with common adverse reactions including diarrhoea and rash.
DeFi trial principal investigator Bernd Kasper said: “Desmoid tumours can have a profound impact on people’s lives and are difficult to manage due to their invasive nature and high rates of recurrence.
“Until now, there have been no approved medicines in Europe. Ogsiveo is a highly innovative therapy with efficacy data demonstrating both meaningful antitumor activity and a significant improvement in desmoid tumour symptoms, including a significant reduction in pain.”
