Assembly Biosciences Announces Expansion of ABI-6250 Clinical Development Into Cholestatic Liver Diseases
Rhea-AI Summary
Assembly Biosciences (Nasdaq: ASMB) plans to expand development of ABI-6250, an oral NTCP inhibitor for chronic HDV, into cholestatic liver diseases PBC and PSC. A Phase 2 HDV trial is planned for Q4 2026 and a Phase 2 basket study in PBC/PSC for Q1 2027, pending regulatory input.
According to Assembly Bio, Phase 1a data showed target engagement with dose‑dependent increases in plasma bile acids, and chronic toxicology studies support longer-term dosing. ABI-6250 remains investigational, with safety and efficacy not yet established.
Positive
- Phase 2 HDV study for ABI-6250 planned to start in Q4 2026
- Phase 2 basket study in PBC and PSC expected to begin in Q1 2027
- Completed Phase 1a trial showed target engagement via bile acid elevations
- Chronic toxicology studies support potential for longer-term dosing
- Pre-IND FDA meeting on cholestatic liver diseases provided guidance for advancement
Negative
- ABI-6250 is investigational with safety and efficacy not yet established
- Planned Phase 2 trials will not start until late 2026 and early 2027
- PSC currently has no approved therapies, highlighting high development risk and unmet need
News Market Reaction – ASMB
In the May 22 session, ASMB gained 6.94%, reflecting a notable positive market reaction. Argus tracked a peak move of +12.6% during that session. Our momentum scanner triggered 13 alerts that day, indicating notable trading interest and price volatility.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Previous Clinical trial Reports
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Dec 08 | HSV Phase 1b update | Positive | +0.8% | Positive interim Phase 1b HSV data with large reductions in shedding and lesions. |
| Aug 08 | ABI-5366 interim data | Positive | +13.0% | Phase 1b ABI-5366 showed strong efficacy and supported move directly to Phase 2. |
| Aug 06 | ABI-6250 Phase 1a data | Positive | +9.2% | Interim Phase 1a ABI-6250 data showed target engagement and tolerable safety. |
| Jun 30 | ABI-1179 Phase 1b start | Positive | +4.7% | Initiation of Phase 1b for ABI-1179 with supportive PK and IND clearance. |
| Jun 25 | ABI-4334 Phase 1b topline | Positive | +0.6% | Positive Phase 1b HBV data with meaningful HBV DNA reductions and good safety. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Clinical trial updates have generally been followed by positive price reactions, with same-tag events showing an average move of about 5.66% and no clear instances of negative next-day responses.
Over the past year, Assembly Bio has steadily advanced multiple antiviral programs, including HSV helicase‑primase inhibitors ABI‑1179 and ABI‑5366, HDV entry inhibitor ABI‑6250, and HBV capsid modulator ABI‑4334. Clinical updates have typically featured strong antiviral effects and favorable safety, with all five tagged clinical events producing positive next‑day stock moves. Today’s expansion of ABI‑6250 development into cholestatic liver diseases builds on this pattern of early‑stage efficacy and mechanism‑driven trial progression.
Key Terms
ntcp medical
sodium taurocholate co-transporting polypeptide medical
cholestatic liver diseases medical
primary biliary cholangitis medical
primary sclerosing cholangitis medical
pre-ind regulatory
phase 1a medical
phase 2 medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
– Phase 2 study in cholestatic liver diseases, focused on PBC and PSC, anticipated to initiate in Q1 2027 –
– Builds on completed Phase 1a study and ongoing development in HDV –
SOUTH SAN FRANCISCO, Calif., May 22, 2026 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative therapeutics targeting serious viral diseases, today announced plans to expand the clinical development of ABI-6250, its oral entry inhibitor candidate for chronic hepatitis delta virus (HDV) infection, into primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC), broadening the program into cholestatic liver diseases.
“We are excited to expand the ABI-6250 program into PBC and PSC, where there remains significant unmet medical need despite existing PBC therapies, and in the case of PSC, no approved treatments,” said Anuj Gaggar, MD, PhD, chief medical officer of Assembly Bio. “The NTCP receptor plays an integral role in both HDV entry and bile acid transport into liver cells, making it a compelling target across multiple liver diseases. We believe ABI-6250 has the potential to address key drivers of disease pathology in these conditions and look forward to advancing clinical evaluation in these populations.”
ABI-6250 is currently being evaluated for chronic HDV infection and has completed a Phase 1a clinical trial in healthy participants. It is an investigational oral small-molecule inhibitor of the sodium taurocholate co-transporting polypeptide (NTCP), a membrane protein selectively expressed on hepatocytes that facilitates bile acid transport into cells and also serves as the entry receptor for HDV infection. By inhibiting NTCP, ABI-6250 blocks the uptake of bile acids into liver cells, a mechanism directly relevant to cholestatic liver diseases, where bile acid accumulation drives liver inflammation and liver injury.
“Treatment options for cholestatic liver diseases have expanded in recent years, but important gaps remain, particularly for patients with PSC for whom no treatments currently are approved and for those with PBC who do not adequately respond to the current therapies,” said Christopher Bowlus, MD, Lena Valente Professor and Chief, Division of Gastroenterology and Hepatology, University of California, Davis. “Targeting NTCP represents a mechanistically distinct approach compared to currently approved therapies, given its central role in bile acid transport. An oral agent like ABI-6250 that modulates this pathway could offer a differentiated strategy to impact disease biology and patient symptoms.”
The company believes the expansion of ABI-6250 into PBC and PSC is supported by preclinical data, the compound’s pharmacologic profile, and clinical findings from the Phase 1a study. These data demonstrated target engagement, including dose-dependent elevations in plasma total bile acids, consistent with NTCP inhibition. In addition, chronic toxicology studies have been completed and support the potential for longer-term dosing in planned Phase 2 trials. The company plans to initiate a Phase 2 clinical study of ABI-6250 in HDV in the fourth quarter of 2026. In addition, a Phase 2 basket study in cholestatic liver diseases, focused on PBC and PSC, is expected to begin in the first quarter of 2027, subject to regulatory feedback.
Assembly Bio recently conducted a pre-IND meeting with the U.S. Food and Drug Administration to discuss the planned development of ABI-6250 in cholestatic liver diseases. While official meeting minutes are pending, the company believes the discussion was constructive and provided helpful guidance to support advancement of the program.
PBC and PSC are chronic, autoimmune cholestatic liver diseases characterized by impaired bile flow, accumulation of bile acids in the liver and progressive liver damage. Clinical symptoms for both PBC and PSC include itch and fatigue, with development of fibrosis and cirrhosis as the diseases progress. There are multiple therapies available for the treatment of PBC; however, a significant percentage of individuals have an inadequate response to these treatments, and there remains an unmet medical need for improved treatment of PBC. There is a significant unmet medical need for the management of PSC as there are currently no approved therapies.
ABI-6250 is an investigational product candidate that has not been approved anywhere globally, and its safety and efficacy have not been established.
About Assembly Biosciences
Assembly Biosciences is a biotechnology company dedicated to the development of innovative small-molecule therapeutics designed to change the path of serious viral and liver diseases and improve the lives of patients worldwide. Led by an accomplished team of leaders in antiviral and liver disease drug development, Assembly Bio is committed to improving outcomes for patients struggling with the serious, chronic impacts of herpesvirus, hepatitis delta virus (HDV) infections, cholestatic liver diseases and hepatitis B virus (HBV). For more information, visit assemblybio.com.
Forward-Looking Statements
The information in this press release contains forward-looking statements that are subject to certain risks and uncertainties that could cause actual results to materially differ. These risks and uncertainties include: Assembly Bio’s ability to realize the potential benefits of its collaboration with Gilead Sciences, Inc. (Gilead), including all financial aspects of the collaboration and equity investments; Assembly Bio’s ability to initiate and complete clinical studies involving its therapeutic product candidates, including studies contemplated by Assembly Bio’s collaboration with Gilead, in the currently anticipated timeframes or at all; safety and efficacy data from clinical or nonclinical studies may not warrant further development of Assembly Bio’s product candidates; clinical and nonclinical data may not differentiate Assembly Bio’s product candidates from other companies’ candidates; Assembly Bio’s ability to maintain financial resources necessary to continue its research activities, clinical studies and other business operations; potential effects of changes in government regulation; results of nonclinical studies may not be representative of disease behavior in a clinical setting and may not be predictive of the outcomes of clinical studies; and other risks identified from time to time in Assembly Bio’s reports filed with the U.S. Securities and Exchange Commission (the SEC). You are urged to consider statements that include the words may, will, would, could, should, might, believes, hopes, estimates, projects, potential, expects, plans, anticipates, intends, continues, forecast, designed, goal or the negative of those words or other comparable words to be uncertain and forward-looking. Assembly Bio intends such forward-looking statements to be covered by the safe harbor provisions contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. More information about Assembly Bio’s risks and uncertainties are more fully detailed under the heading “Risk Factors” in Assembly Bio’s filings with the SEC, including its most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Except as required by law, Assembly Bio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Contacts:
Investors:
Patrick Till
Meru Advisors
(484) 788-8560
investor_relations@assemblybio.com
Media:
Jamie Strachota
Sam Brown Healthcare Communications, Inc.
(703) 819-7647
ASMBMedia@sambrown.com
