SK Biopharmaceuticals Licenses Preclinical LRRK2/c-Abl Dual Inhibitor 1ST-104 for Parkinson's in Deal Worth Up to $314.8M
核心洞察
SK Biopharmaceuticals (搜索) has secured exclusive global rights to 1ST-104, a preclinical oral small-molecule dual inhibitor of LRRK2 (搜索) and c-Abl (搜索) for Parkinson's disease (搜索), from 1ST Biotherapeutics (搜索).
The licensing agreement is valued at up to $314.8 million, including a $1.8 million upfront payment, a $1.8 million candidate-selection milestone, and tiered royalties on global net sales.
SK Biopharmaceuticals (搜索) separately made a $2.2 million strategic equity investment in 1ST Biotherapeutics (搜索) to support a long-term partnership between the two South Korean companies.
SK Biopharmaceuticals (搜索) Co., Ltd. (KRX: 326030) and 1ST Biotherapeutics (搜索), Inc. (1STBIO) have entered an exclusive global licensing agreement and strategic equity investment partnership to advance 1ST-104, a preclinical oral small-molecule candidate for Parkinson's disease (搜索). The deal, announced September 17, 2026, is valued at up to $314.8 million including milestones, with SK Biopharmaceuticals taking on global clinical development and commercialization responsibility.
Under the terms of the agreement, 1STBIO will receive a non-refundable upfront payment of $1.8 million, payable within 30 business days of signing. The company is also eligible for a short-term milestone of $1.8 million upon candidate selection, plus additional development, regulatory, and commercial milestones that make up the headline total. Following commercialization, 1STBIO is eligible to receive tiered royalties based on global net sales of 1ST-104.
Separately, SK Biopharmaceuticals (搜索) made a $2.2 million strategic equity investment in 1STBIO, a move both companies describe as an effort to build a long-term partnership beyond a simple asset acquisition.
A Dual-Inhibition Strategy Targeting LRRK2 and c-Abl
1ST-104 is being developed as an oral small-molecule treatment for Parkinson's disease (搜索) using a dual-inhibition strategy that simultaneously inhibits leucine-rich repeat kinase 2 (LRRK2 (搜索)) and c-Abl (搜索). LRRK2 is considered a therapeutic target directly linked to the pathogenesis of Parkinson's disease, while c-Abl has also been investigated as a potential target in neurodegenerative disease.
The candidate is designed to act on multiple pathways implicated in Parkinson's disease (搜索), including endolysosomal dysfunction and alpha-synuclein accumulation. The companies expect the dual-target mechanism could position 1ST-104 as a first-in-class therapy.
1STBIO developed 1ST-104 as a next-generation Type 2 LRRK2 (搜索) inhibitor, meaning it binds LRRK2 in its inactive conformation rather than the active-state binding mode used by Type 1 inhibitors. The company said this approach addresses development challenges faced by some Type 1 LRRK2 inhibitors, particularly pulmonary findings reported in preclinical studies. According to 1STBIO, 1ST-104 has demonstrated target selectivity along with blood-brain barrier permeability in preclinical studies, and the Type 2 binding mode is intended to provide a differentiated approach from conventional Type 1 inhibitors.
Since 2024, 1STBIO has been a member of the LRRK2 (搜索) Investigative Therapeutics Exchange (LITE), a research consortium supported by The Michael J. Fox Foundation for Parkinson's Research, through which the company continues to engage in global scientific and technical collaboration.
Preclinical Stage, With IND Decision Pending
1ST-104 remains in the preclinical stage. 1STBIO is conducting research to select a preclinical candidate (PCC), after which SK Biopharmaceuticals (搜索) will assume responsibility for subsequent development. The company plans to evaluate follow-up development toward an Investigational New Drug (IND) application based on future research results. No IND filing date has been disclosed.
In its regulatory filing, SK Biopharmaceuticals (搜索) stated that cost recognition under the agreement may vary depending on the success of clinical trials and regulatory approvals, and that the agreement may be terminated in the event of research and development discontinuation by regulatory authorities or failure to obtain product approval, among other circumstances, in accordance with the contract terms.
Positioning as a Next-Generation Disease-Modifying Therapy
Parkinson's disease (搜索) is a degenerative brain disorder in which dopamine-producing neurons progressively die, leading to deterioration of motor function and other symptoms. Most currently available treatments focus on symptom relief, leaving what the companies describe as a significant unmet need for disease-modifying therapies (DMTs) that can slow or alter disease progression. SK Biopharmaceuticals (搜索) plans to develop 1ST-104 as a next-generation DMT that intervenes in the disease process itself.
"This agreement serves as a strong validation of 1ST Biotherapeutics (搜索)' proprietary discovery capabilities in next-generation target identification and lead optimization," said Jamie Jae Eun Kim, CEO of 1ST Biotherapeutics. "It marks a major milestone by uniting our early-stage R&D strength with SK Biopharmaceuticals (搜索)' proven global development and commercial expertise."
Donghoon Lee, CEO of SK Biopharmaceuticals (搜索), said the collaboration combines 1STBIO's early-stage discovery capabilities with SK Biopharmaceuticals' clinical development and commercialization expertise. "We will work to translate this candidate into successful clinical development, opening up new possibilities for the treatment of Parkinson's disease (搜索)," he added.
First In-Licensing Deal Through SKBP's Open Innovation Center
The transaction is the first candidate in-licensing led by SK Biopharmaceuticals (搜索)' Open Innovation Center (OIC). The company has been pursuing an "East-West Bridge" strategy, connecting drug candidates discovered in Asia with its in-house clinical development and commercialization capabilities. This year, SK Biopharmaceuticals has expanded external candidate sourcing through an open innovation program with Seoul Biohub and a joint AI-based drug discovery research collaboration with Insilico Medicine (搜索). With this deal, the company has extended its scope beyond joint research to include in-licensing of preclinical-stage candidates.
The agreement follows two other significant SK Biopharmaceuticals (搜索) in-licensing announcements in 2026: a collaboration with Insilico Medicine (搜索) valued at up to $2.5 billion for AI-enabled CNS drug discovery, and a license agreement with Biohaven Ltd (搜索) for the Kv7 ion channel platform and opakalim (搜索) for epilepsy, valued at up to $795 million. The combined value of the Parkinson's and epilepsy deals reaches approximately ₩1.5 trillion (approximately $1.1 billion), reflecting a strategic effort to diversify revenue away from the company's heavy reliance on cenobamate. An industry source noted that with Xenon Pharmaceuticals' azetukalner, a potential competitor, also nearing commercialization, the need to secure follow-on pipeline assets is significant.
According to market research firm Grand View Research, the global CNS therapeutics market is projected to grow from $138.6 billion in 2025 to $273.3 billion by 2033. SK Biopharmaceuticals (搜索) is leveraging its commercialization experience in epilepsy, built through cenobamate, and its global network to expand into adjacent disease areas such as Parkinson's disease (搜索).
Company Profiles and Broader Pipelines
Founded in 2016, 1ST Biotherapeutics (搜索) is a science-driven biopharmaceutical company developing therapies for neurodegenerative diseases, immuno-oncology, and rare diseases. Its immuno-oncology candidate FB849 is being evaluated in a Phase 1/2 clinical trial following dosing of the first patient in the U.S. in 2023, with clinical development also underway in Korea; MSD (搜索) has agreed to provide KEYTRUDA (pembrolizumab) at no cost for combination clinical trials. The company's Parkinson's disease (搜索) portfolio also includes FB-101, a c-Abl (搜索) inhibitor that has completed a Phase 1 single ascending dose (SAD) study in the U.S.
SK Biopharmaceuticals (搜索), together with its U.S. subsidiary SK Life Science (搜索), Inc., focuses on the discovery, development, and commercialization of treatments for central nervous system disorders and oncology. The company independently developed and commercialized XCOPRI (cenobamate tablets) for the treatment of partial-onset seizures in adults, and continues to build a global pipeline spanning CNS disorders and oncology.