Developer acquires global rights to Parkinson’s drug in deal worth $300M
Experimental therapy targets 2 proteins driving disease's development
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- In a deal worth more than $300 million, a Korean biotech has acquired exclusive global rights to a next-gen Parkinson's treatment.
- The oral therapy, known as 1st-104, targets two proteins thought to help drive the development of the neurodegenerative disease.
- New partners 1st Biotherapeutics and SK Pharmaceuticals say the collaboration combines each company's area of expertise.
In a deal that could be worth more than $300 million, a Korean biotech has acquired exclusive global rights to develop and commercialize 1ST Biotherapeutics‘ 1ST-104, an oral treatment candidate targeting the causes of Parkinson’s disease.
SK Biopharmaceuticals will bring its “clinical development and commercialization expertise” to the deal, combining its experience with “1ST Biotherapeutics’ early-stage discovery capabilities,” Donghoon Lee, SK’s CEO, said in a press release announcing the licensing agreement.
The collaboration aims to advance the clinical development of 1ST-104, a small molecule designed to simultaneously target two proteins, LRRK2 and c-Abl, implicated in Parkinson’s. The experimental treatment aims to block these proteins — which together are thought to play a key role in the disease’s development.
“This agreement serves as a strong validation of 1ST Biotherapeutics’ proprietary discovery capabilities in next-generation target identification and lead optimization,” said Jamie Jae Eun Kim, CEO of 1ST Bio. “Through close collaboration with SK Biopharmaceuticals, we will leverage the strengths of both companies to advance the subsequent development of 1ST-104.”
Parkinson’s is caused by the loss of nerve cells that produce dopamine, a chemical messenger involved in controlling movement. Reduced dopamine signaling can lead to the disease’s motor symptoms, such as tremor, stiffness, and slowed movement.
Several biological processes are thought to contribute to Parkinson’s, including the toxic accumulation of the protein alpha-synuclein. Mutations in the LRRK2 gene, which provides instructions for producing an enzyme of the same name involved in cell signaling and waste disposal, are among the most common genetic causes of Parkinson’s. When the gene is mutated, the enzyme can become overactive, contributing to nerve cell dysfunction and death.
1st-104 is a next-generation therapy targeting the LRRK2 protein
1ST-104 is a dual-target inhibitor designed to block both LRRK2 and c-Abl, a protein kinase linked to the toxic accumulation of alpha-synuclein. As a next-generation Type 2 inhibitor, 1ST-104 binds to and locks both enzymes in their inactive conformations, preventing their activation — a different approach from conventional Type 1 inhibitors that target LRRK2 in its active state.
In preclinical studies, 1ST-104 demonstrated target selectivity and successfully crossed the blood-brain barrier, a protective membrane that controls which substances can enter the brain. This is important because reaching the brain is a major challenge for many treatments designed to target neurological diseases.
We will work to translate this candidate into successful clinical development, opening up new possibilities for the treatment of Parkinson’s.
Lee called the new collaboration “significant,” noting that it combines each company’s area of expertise.
“We will work to translate this candidate into successful clinical development, opening up new possibilities for the treatment of Parkinson’s,” Lee said.
Under the agreement, 1ST Bio will receive an upfront payment of $1.8 million and is eligible for additional milestone payments that could make the deal’s total value reach $314.8 million. If the therapy ultimately reaches the market, 1ST Bio also would receive royalties based on global sales. Separately, SK will make a $2.2 million strategic equity investment in 1ST, the companies noted.
1ST Bio is a member of the LRRK2 Investigative Exchange, known as LITE, which is a research consortium supported by The Michael J. Fox Foundation for Parkinson’s Research. The goal of LITE is to develop LRRK2-targeting treatments for Parkinson’s.
The company is also developing FB-101, a c-Abl inhibitor, as a potential treatment for Parkinson’s. The developer completed a Phase 1 trial (NCT04165837) testing the treatment’s safety when administered as single ascending doses to healthy volunteers. Another Phase 1 trial (NCT07126704), expected to launch early next year, will test single- and multiple-ascending doses of FB-101 in healthy men at a single site in South Korea.
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