Vertex Pharmaceuticals, Inc.

Germany’s Merck isn’t the first pharma—or even the first Merck—to spot potential in Skyhawk Therapeutics’ high-flying RNA splicing tech.\n Merck KGaA has swooped into a neurological-focused pact with RNA biotech Skyhawk Therapeutics that could top out at $2 billion.Under the agreement, Skyhawk will use its RNA splicing platform, dubbed SkySTAR, to identify small molecule candidates directed at specific RNA targets selected by Germany-based Merck. While the companies didn’t drill into the exact neurological indications being considered, the collaboration is designed to “expand the potential of RNA modulation in diseases where traditional approaches have proven challenging,\" Skyhawk said in an Aug. 18 press release.Skyhawk will oversee this discovery and preclinical work, with Merck having an option to pay to take the candidates into human trials and beyond. Payments to Skyhawk’s could eventually stretch to $2 billion—although the release didn’t offer a breakdown of the terms of the deal—on top of tiered royalties on sales should any drugs make it to market.“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,” Amy Kao, M.D., senior vice president and global head of the neuroscience and immunology research unit at Merck KGaA, said in the release.“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,” Kao added.Going after so-called \"undruggable\" disease targets, Skyhawk and peers such as Arrakis Therapeutics and Remix Therapeutics have been betting they can alter levels of disease-driving proteins by hitting RNA upstream.Neuroscience is a focus of Skyhawk’s internal pipeline, which is led by a phase 2/3-stage splicing modulator designed to treat Huntington\'s disease by reducing the production of mutated HTT. Beyond neurology, Skyhawk also has preclinical programs for lymphoma and fibrosis in the works. Meanwhile, Germany’s Merck isn’t the first pharma—or even the first Merck—to spot potential in Skyhawk’s RNA splicing tech. U.S.-based Merck & Co. penned a pair of deals with the biotech over the course of 2019 and 2020 to work across autoimmune, metabolic and neurological diseases.Other Skyhawk collaborators have included Vertex, Ipsen and Takeda.Today\'s agreement “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,\" Skyhawk CEO Bill Haney said in a statement.

While its aircraft namesake is a single-seater carrier, Skyhawk Therapeutics does not ride solo. As of Monday morning, Merck KGaA became its ninth pharma company co-pilot through an RNA-targeting small molecule collaboration. Waltham, MA-based Skyhawk, which broke onto the scene in 2018 , will work with its new pharma partner to create small molecules for neurological disorders. The deal could be worth more than $2 billion, although the companies are staying mum on how much of that comes upfront. The duo will work on RNA splicing modulation for undisclosed diseases. “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,” Amy Kao, SVP and global leader of Merck KGaA’s neuroscience and immunology unit, said in a statement. The pharma partnership model has helped fuel Skyhawk’s internal R&D engine. Collaborators have dished out more than $525 million in upfront payments to Skyhawk, the biotech has said. That includes tie-ups with Vertex , Takeda , Sanofi , Merck , Genentech , Ipsen , Bristol Myers Squibb and Biogen . The biotech’s last disclosed $133 million round in September 2021 was likely going to be the final private round, CEO Bill Haney told Endpoints News at the time, but the public markets chilled in the ensuing quarters and have remained largely shuttered for the past three years. The company’s investors include Fidelity, GreatPoint, Rock Springs Capital, Yosemite and others. Even though Skyhawk has not raised additional capital since the 2021 round, Haney told Endpoints on Sunday that it is still well-funded and does not need to secure more financing. If the biotech decides to commercialize its lead internal therapy on its own, then “that may be a different story,” he said. Its lead drug is in a Phase 2/3 trial for Huntington’s disease. Skyhawk is one of the main biotech startups working on drugging RNA. Arrakis Therapeutics , Ribometrix and others are also in the arena. SKY-0515, Skyhawk’s lead investigational medicine, entered a Phase 2/3 in Australia and New Zealand earlier this summer. More countries will soon be added to the Huntington’s disease trial, Haney said. “Our enrollment has been really good. Our data has been really good. We all cross our fingers and look to the heavens, hoping that somebody can have a breakthrough for Huntington’s patients,” Haney said. The company’s once-daily treatment candidate is an oral drug, which could make it easier to access than other types of medicines. “Part of what we’re doing in Skyhawk is democratizing these therapeutics. God bless the people who pioneered it with ASOs [antisense oligonucleotides] and gene therapy, but it’s super expensive and tough on patients and tough on payers,” Haney said. “Healthcare systems are cracking around that. Those are drugs that can be available in Boston, but not so much in Bogotá or Bangladesh.” Behind SKY-0515, two more rare neuro programs will enter the clinic by the end of 2026, Haney said. The focus is on movement disorders, “where there’s no approved disease-modifying therapeutic and clear genetic markers,” the CEO said. The pipeline also hums with programs in oncology and fibrotic diseases, but those will likely be the focus of partnerships, Haney said. Haney co-founded the biotech with scientist Kathleen McCarthy, who helped develop Roche’s spinal muscular atrophy medicine Evrysdi. Haney had departed the CEO post in June 2024, handing the baton to CFO Clint Musil, so he could switch to the executive chairman role. But Haney is back in the CEO role once again. “We had a breakthrough in the last year, year and a half, where the pace of advances accelerated, and that means we’re in a place to put more drugs in the clinic faster than we anticipated, and that meant for me that being executive chairman wasn’t really enough,” Haney said. Part of the breakthrough, Haney said, is the amount of novel biology that the company is discovering. The startup has employed machine learning tools for years to help with that work. The AI systems have advanced quicker than expected, he said.

iStock, Anne Czichos Waltham, Massachusetts–based Skyhawk Therapeutics has been collecting collaborations with larger companies in spades since launching in 2018. Merck KGaA is broadening its neuro pipeline with a collaboration with Skyhawk Therapeutics aimed at developing new small molecule RNA-targeting drugs. Though financial details were not disclosed, the deal could be worth as much as $2 billion. According to Monday’s announcement, the Waltham, Massachusetts–based biotech would design small molecules against RNA targets selected by Merck. The indications were not announced, with the companies simply stating the targets would be “in select neurological indications with high unmet medical need,” according to the statement. Merck will dole out milestone payments as well as tiered royalties on any potential sales on molecules developed in the deal. Skyhawk will lead the discovery and preclinical efforts, after which Merck will pick up development and commercialization efforts for molecules it chooses. Skyhawk’s pipeline is based on small molecule drugs that influence RNA expression inside cells. The deal will see the biotech use its SkySTAR platform—Skyhawk Small molecule Therapeutics for Alternative splicing of RNA—to develop molecules to alter splicing and gene expression at the RNA level. The biotech’s pipeline is focused on neurology and oncology targets, with a Huntington’s drug in Phase I and another for multiple myeloma and non-Hodgkin’s lymphoma, both drugs being splicing modulators. The collaboration is the latest big ticket agreement Skyhawk has picked up from a larger company in recent years. In April 2024, the biotech struck a $1.8 billion pact with French pharma Ipsen, also in neurological indications. The startup has also signed a $2.2 billion deal with Vertex that includes a $40 million upfront payment as well as two deals with Merck & Co. Skyhawk launched in January 2018 with a rather modest $8 million in financing. Merck KGaA has been relatively quiet on the dealmaking front this year, with the only big splash so far being the $3.9 billion acquisition of Connecticut-based SpringWorks Therapeutics in April. The only other deal the company has made was the acquisition of HUB Organoids B.V., a next-gen drug testing company, for an undisclosed sum announced in early January.