Skyhawk Therapeutics, Inc.

Today, a brief rundown of news involving PTC Therapeutics and Eli Lilly, as well as updates from Regenxbio, Skyhawk Therapeutics and Adaptive Biotechnologies that you may have missed.An experimental drug developed by PTC Therapeutics for the neuromuscular disorder Friedrichs ataxia was rejected by the Food and Drug Administration, the company disclosed Tuesday. According to PTC, the agency determined data supporting the drug, called vatiquinone, did not prove substantial evidence of efficacy, and said PTC would need another positive study to resubmit its application. In the main study of vatiquinone, treatment was not associated with a significant change on the trials main goal, although PTC claimed positive findings on a prespecified secondary measure. Analysts were not surprised by the rejection, as PTC executives had recently said they werent in labeling discussions with the agency. Ned PagliaruloEli Lilly priced nearly $7 billion in corporate bond issues, including $1 billion in a long-dated tranche that matures in 40 years. In a prospectus, Lilly said proceeds from the bond sales will be used for general corporate purposes, potentially including the repayment of existing notes. Lilly, which earned about $8.5 billion in net income over the first six months of the year, held around $3.4 billion in cash and cash equivalents at the end of June. It has nearly $40 billion in total debt. Ned PagliaruloThe FDA delayed by three months a decision whether to approve what would be the first gene therapy for Hunter syndrome. According to Regenxbio, which is developing the treatment, the agency extended its review deadline from Nov. 9 to Feb. 8 after the company added longer-term data from the key trial supporting its application. Those data are consistent with results previously submitted on the therapy, RGX-121, and the agency hasnt raised any safety concerns either, Regenxbio said Monday. The delay is reflective of both the recent turnover at the FDAs Center for Biologics Evaluation and Research as well as specific review issues for Regenxbio, wrote Leerink Partners analyst Mani Foroohar. Ben FidlerBiotechnology startup Skyhawk Therapeutics will collaborate with Merck KGaA to develop RNA-targeting small molecules for unspecified neurological diseases “with high unmet need,“ the companies announced Monday. Skyhawk will steer discovery and preclinical work for each program involved in the deal, with Merck then leading further development and commercialization for each drug it opts to license. The biotech is eligible to receive more than $2 billion overall, though it didn't specify how much of that total will be paid upfront. Skyhawk has a pipeline of in-house drugs as well as a portfolio of more than a dozen preclinical programs partnered with other drugmakers, according to its website. Ben FidlerRoches Genentech division has ended a yearslong alliance with Adaptive Biotechnologies to develop personalized cell therapies for cancer. In a regulatory filing on Monday, Adaptive revealed the deal will be terminated effective Feb. 9, 2026. Adaptive got $300 million upfront when the two firms first announced a deal in January 2019. The collaboration is the second cell therapy pact to be recently canceled by Genentech, following the termination of a partnership with Adaptimmune last year. Ben Fidler '

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.

Skyhawk Therapeutics enters into an option agreement to grant Merck exclusive global rights to drug candidates pursued under the collaboration upon option exercise Total deal value exceeds $2 billion including upfront and milestone payments, along with the potential for royalties on future sales Following option exercise, Merck will assume responsibility for further development and commercialization BOSTON, Aug. 18, 2025 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a leader in the discovery and development of small molecules that modulate RNA expression, today announces a strategic research collaboration with Merck, a leading science and technology company. The collaboration will focus on the discovery of novel RNA-targeting small molecules in select neurological indications with high unmet medical need. Under the terms of the agreement, Skyhawk will use 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. The collaboration aims to expand the potential of RNA modulation in diseases where traditional approaches have proven challenging, leveraging Skyhawk's industry-leading capabilities in RNA splicing modulation and Merck's expertise in drug development and commercialization. "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, Merck. "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." "We are proud to partner with Merck, a company with a strong heritage of innovation and a commitment to advancing transformative science," said Bill Haney, Chief Executive Officer of Skyhawk Therapeutics. "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." Skyhawk will lead discovery and preclinical development efforts, after which will assume responsibility for further development and commercialization upon option exercise. The overall deal is valued at over $2 billion, with Skyhawk being eligible for milestone payments, as well as tiered royalties on commercial sales. About Skyhawk Therapeutics Skyhawk Therapeutics is a clinical-stage biotechnology company focused on the discovery and development of novel small molecule therapies designed to modulate critical RNA targets and revolutionize patient treatment for some of the world's most intractable diseases. Skyhawk's discovery expertise is rooted in its proprietary drug discovery platform, which assesses, identifies, and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states. Skyhawk has built collaborations with multiple pharma partners that leverage Skyhawk's novel platform across disease areas including neurodegenerative disease, autoimmune disease, and oncology. For more information visit . Skyhawk Contacts Kyle Dow, VP Corporate Development [email protected] Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED