ABL Bio will likely be able to resume its U.S. phase 1 clinical trial of ABL301, a candidate for treating Parkinson's disease licensed out to Sanofi, the company said.
ABL Bio said Wednesday it had confirmed the safety of ABL 301 in a nonclinical toxicity study to determine the feasibility of administering ABL301 at high doses. The company explained that ABL301 was administered at a dose four times higher than previously tested in monkeys and rodents and did not show any toxicity or adverse effects.
In 2022, ABL Bio drew the industry’s attention by signing a technology transfer agreement with global pharmaceutical giant Sanofi for ABL301, a candidate for treating degenerative brain diseases worth $1.06 billion (1.41 trillion won). The two companies then launched a phase 1 trial of ABL301 in the U.S.
However, in December of the same year, the U.S. FDA expressed concerns about the high dose of ABL301 in the phase 1 protocol, requesting a high-dose GLP toxicity nonclinical study and additional data submission. ABL Bio and Sanofi stopped the high dose and proceeded with a lower dose.
Unveiling the results of the nonclinical toxicity study, ABL Bio stressed that the excellent safety profile of ABL301 and its blood-brain barrier (BBB) penetration platform, GrabBody-B, were reaffirmed. Based on these results, the company expects to continue discussions with the FDA to advance to higher doses in the U.S. phase 1 study.
ABL Bio said that the U.S. phase 1 clinical trial is on track and confident of completing the study on schedule. However, the company added that it is difficult to provide an end date for the trial because it is co-conducting the study with Sanofi.
"We are not concerned about issues, such as high dosing, because we have confirmed it through many experiments,” a company official said. “Rather, we are looking forward to concluding phase 1 quickly so that our BBB shuttle platform technology, Grabody-B, can be validated with human data."
The official added that the Korean company believes Sanofi would take the lead from the phase 2 clinical trial of ABL301 as agreed in their contract.