1ST-104

SK Biopharmaceuticals has added a second preclinical Parkinson’s disease therapy to its pipeline courtesy of a $314.8 million biobucks deal with a fellow South Korean drug developer. The deal centers on 1ST-104, an oral small molecule dual inhibitor of both LRRK2 and c-Abl being developed by 1ST Biotherapeutics. The biotech is set to receive $1.8 million upfront and a near-term payment of the same amount. Combining these payments with the potential from milestones brings the deal total to $314.8 million, with 1STBIO also in line for a slice of the royalties if 1ST-104 makes it to market. Separately, SK is also making a $2.2 million equity investment in 1STBIO. Too much LRRK2 disrupts the formation of little projections called cilia on brain cells. These cilia operate like antennae to receive signals from the surrounding environment. Research over recent years has continued to suggest that inhibiting the LRRK2 enzyme causes cilia to regrow. Neuron23 has a LRRK2 inhibitor in phase 2 development, while Denali also has a candidate in mid-stage development, despite Biogen walking away from the program following a phase 2 trial miss in May. Separate preclinical research has raised concerns about the safety of LRRK2 inhibitors due to histopathological changes in the lungs, although follow-up investigations have suggested that these changes are temporary and not dangerous. Still, 1ST Bio said the company had developed 1ST-104 as a Type 2 LRRK2 inhibitor to “address these limitations.” “This agreement serves as a strong validation of 1ST Biotherapeutics' proprietary discovery capabilities in next-generation target identification and lead optimization,” 1STBIO CEO Jamie Jae Eun Kim said in a Sept. 17 release. “It marks a major milestone by uniting our early-stage R&D strength with SK Biopharmaceuticals' proven global development and commercial expertise,” the CEO added. “Through close collaboration with SK Biopharmaceuticals, we will leverage the strengths of both companies to advance the subsequent development of 1ST-104.” SK currently markets the epilepsy drug Xcopri, and developed the sleepiness treatment Sunosi. The company’s pipeline encompasses a range of central nervous system disorders, including a GCase activator in preclinical development for Parkinson’s. SK CEO Donghoon Lee said that licensing 1ST-104 “combines 1ST Biotherapeutics' 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,” Lee added. SK’s most recent licensing deal ran into trouble last week when the FDA put trials of the epilepsy drug BHV-7000 on hold mere days after the South Korean company had paid $350 million upfront for the worldwide license to the selective activator of Kv7.2/7.3 potassium channels.