Phase 1a study will evaluate single and multiple ascending doses of ABI-6250 in healthy participants with data expected in Q3 2025
Biomarker of ABI-6250 target engagement, serum bile acids, will be assessed in addition to safety and pharmacokinetic measures
Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative therapeutics targeting serious viral diseases, today announced that the first participant has been dosed in the Phase 1a trial of ABI-6250, the company’s orally bioavailable, small molecule hepatitis delta virus (HDV) entry inhibitor candidate.
This Phase 1a study will evaluate the safety, tolerability, and pharmacokinetics (PK) of ABI-6250 across single and multiple ascending dose cohorts in healthy participants. In addition, the study will look at serum bile acids as a biomarker of ABI-6250’s engagement of its target, the transporter used by HDV to infect hepatocytes (sodium taurocholate cotransporting polypeptide or NTCP). Assembly Bio expects to share data from the Phase 1a study in Q3 2025.
Currently, one therapy is approved for chronic HDV infection (cHDV) in the European Union with no therapies approved in the United States. This approved therapy is a peptide inhibitor of NTCP requiring daily injections. Well-tolerated serum bile acid elevations have been observed clinically with the approved NTCP inhibitor.
“ABI-6250 has the potential to be the first oral therapy for cHDV, a life-threatening chronic viral infection with limited treatment options available,” said Anuj Gaggar, MD, PhD, chief medical officer of Assembly Bio. “Chronic HDV is the most severe form of viral hepatitis with 70% of individuals with cHDV progressing to cirrhosis within 10 years, and we are excited to have clinical studies underway for this candidate with the potential to simplify cHDV treatment.”
In preclinical studies, ABI-6250 has demonstrated low nanomolar potency across multiple HDV genotypes in vitro, selectivity for NTCP versus other bile acid transporters, and a PK profile supportive of once-daily oral dosing.
ABI-6250 is an investigational product candidate that has not been approved anywhere globally, and its safety and efficacy have not been established.
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