SK Biopharmaceuticals has licensed global rights to Parkinson’s drug candidate 1ST-104 in a deal worth up to US$314.8m.
The agreement gives the Korean biotechnology company exclusive rights to develop and commercialise the experimental oral therapy worldwide.
1ST-104 is a small molecule designed to target LRRK2 and c-Abl simultaneously, two proteins implicated in biological processes associated with Parkinson’s disease.
The drug was developed by 1ST Biotherapeutics, which will work with SK Biopharmaceuticals to advance its clinical development.
Donghoon Lee, chief executive of SK Biopharmaceuticals, said the agreement combines its “clinical development and commercialisation expertise” with “1ST Biotherapeutics’ early-stage discovery capabilities”.
He added: “We will work to translate this candidate into successful clinical development, opening up new possibilities for the treatment of Parkinson’s.”
Parkinson’s is caused by the loss of nerve cells that produce dopamine, a chemical messenger involved in controlling movement. Reduced dopamine signalling can contribute to symptoms including tremor, stiffness and slowed movement.
Several biological processes are thought to contribute to the condition, including the toxic accumulation of the protein alpha-synuclein.
Mutations in the LRRK2 gene are among the most common genetic causes of Parkinson’s. The gene provides instructions for producing an enzyme involved in cell signalling and waste disposal.
When mutated, the enzyme can become overactive, contributing to nerve cell dysfunction and death.
1ST-104 is designed to inhibit both LRRK2 and c-Abl, a protein kinase linked to the toxic accumulation of alpha-synuclein.
The therapy is a Type 2 inhibitor, meaning it binds to both enzymes in their inactive forms and prevents their activation. This differs from conventional Type 1 inhibitors, which target LRRK2 while it is active.
Preclinical studies found that 1ST-104 demonstrated target selectivity and crossed the blood-brain barrier, the protective membrane that controls which substances can enter the brain.
Under the agreement, 1ST Biotherapeutics will receive an upfront payment of US$1.8m and could receive further milestone payments taking the total value of the deal to US$314.8m.
The company would also receive royalties based on global sales if the therapy reaches the market.
Separately, SK Biopharmaceuticals will make a US$2.2m strategic equity investment in 1ST Biotherapeutics.
Jamie Jae Eun Kim, chief executive of 1ST Biotherapeutics, said: “This agreement serves as a strong validation of 1ST Biotherapeutics’ proprietary discovery capabilities in next-generation target identification and lead optimisation.
“Through close collaboration with SK Biopharmaceuticals, we will leverage the strengths of both companies to advance the subsequent development of 1ST-104.”
1ST Biotherapeutics is also part of the LRRK2 Investigative Exchange, known as LITE, a research consortium supported by The Michael J. Fox Foundation for Parkinson’s Research that aims to develop treatments targeting LRRK2.
The company is separately developing FB-101, a c-Abl inhibitor, as a potential Parkinson’s treatment.
A Phase 1 trial assessing single ascending doses of FB-101 in healthy volunteers has been completed, while another Phase 1 study is expected to begin early next year to assess single and multiple ascending doses in healthy men at a single site in South Korea.
