Sanofi takes over Parkinson’s drug from ABL Bio as biotech expands BBB platform into RNA delivery

Sanofi has officially taken over the clinical development of ABL301, a Parkinson’s disease candidate originally co-developed with Korea’s ABL Bio, signaling the start of a new phase for the drug and a strategic turning point for the biotech.

ABL Bio confirmed Monday that the French drugmaker has assumed full sponsorship of ABL301’s future trials following a completed phase 1 study. 

In a formal notice shared during a corporate briefing on Monday, Sanofi wrote, “We are currently in the process of transferring the sponsorship of ABL301 from ABL Bio to Sanofi." It added, “Sanofi will solely conduct the following clinical trials of ABL301.” 

The move triggers a new milestone in ABL Bio’s 2022 licensing deal, which could reach $1.06 billion in total value. The Kosdaq-listed company has received $125 million to date, with additional payments expected as the candidate enters phase 2.

ABL301 targets alpha-synuclein, a pathogenic driver in Parkinson’s disease, and is powered by ABL Bio’s proprietary blood-brain barrier (BBB) shuttle, Grabody-B.

The platform, originally designed to transport antibodies into the brain via IGF1R targeting, is now being repositioned as a delivery system for RNA-based drugs.

“We are expanding the modality of Grabody-B,” ABL Bio CEO Lee Sang-hoon said during the briefing. “Global pharma isn’t just looking at antibodies anymore. There’s growing demand for shuttles that can carry oligonucleotides like small interfering RNA and antisense oligonucleotides across the BBB.”

Lee cited preclinical research conducted in collaboration with Ionis Pharmaceuticals, showing that Grabody-B enabled oligonucleotide delivery into skeletal muscle while avoiding the liver and heart.

“We’re seeing delivery to skeletal muscle, but no knockdown in cardiac muscle,” he said, noting the distinction is critical for avoiding toxicity in RNA-based drugs.

Separately, ABL Bio presented new data on its bispecific antibody ABL111, co-developed with I-Mab for gastric and pancreatic cancers.

The antibody targets Claudin 18.2 and 4-1BB and is being evaluated both as monotherapy and in combination with chemotherapy and Bristol Myers Squibb’s PD-1 inhibitor Opdivo (nivolumab). In a phase 1b trial, the overall response rate in patients receiving higher doses reached 83 percent, while the disease control rate hit 100 percent.

Lee noted that ABL111 showed activity in gastric cancer patients with low Claudin18.2 expression -- a group not covered by AstraZeneca’s zolbetuximab. He estimated that “35 percent is completely an unmet medical market,” and added that ABL111 could offer efficacy even in patients with expression levels as low as 1 percent.

In terms of durability, he pointed to several responders in the 8 and 12 mg/kg cohorts who remained progression-free for nearly a year. “One patient has maintained a response for more than 11 months,” he said. “This is data we’re watching very closely.”

Meanwhile, ABL’s U.S.-based subsidiary Neok Bio, named to reflect a “new era of knowledge,” is preparing to begin human trials by the end of the year for two dual-payload bispecific ADC candidates, ABL206 and ABL209.

The company has staffed Neok with former Genentech and Roche executives and plans to unveil early data at AACR next year. Lee said the goal is to either list Neok on Nasdaq within the next three to four years or pursue a strategic acquisition by a major pharma company.