A Phase 2a Randomized, Double-blinded, Placebo-controlled, Multi Center, Dose Ranging Study of the Safety and Efficacy of ATI-2173 and Vebicorvir in Combination With Tenofovir Disoproxil Fumarate in Subjects With Chronic Hepatitis B Virus Infection

This is a randomized, double-blinded, placebo-controlled, multi center, dose ranging study of safety and efficacy in volunteers with chronic hepatitis B virus infection. Volunteers will be administered multiple oral doses of ATI-2173 vebicorvir in combination with tenofovir disoproxil fumarate and assessed for safety and efficacy including blood tests to show how the body metabolizes and eliminates the investigational drug as well as how the drug effects the virus infection.

Start Date11 Apr 2022

Sponsor / Collaborator

Antios Therapeutics, Inc.

NCT04820686

/ TerminatedPhase 2

A Randomized Phase 2a, Multicenter, Open-Label, Multiple-Cohort Study Evaluating Regimens Containing Vebicorvir in Subjects With Chronic Hepatitis B Virus Infection

The purpose of this study is to determine if vebicorvir (VBR, ABI-H0731) in combination with AB-729 is safe and effective in participants with chronic hepatitis B infection (cHBV) receiving a standard of care nucleos(t)ide/reverse transcriptase inhibitor (SOC NrtI).

Start Date07 May 2021

Sponsor / Collaborator

Assembly Biosciences, Inc.

[+1]

CTR20210059

/ TerminatedPhase 2

一项在慢性乙型肝炎患者中评估含 ABI-H0731方案的随机、IIa期、多中心、开放性研究

[Translation] A randomized, phase IIa, multicenter, open-label study evaluating an ABI-H0731-containing regimen in patients with chronic hepatitis B

主要目的评估731联用ETV±Peg-IFNα治疗CHB受试者时的安全性和耐受性次要目的评估731联用ETV±Peg-IFNα降低CHB受试者HBV pgRNA水平的作用评估731联用ETV±Peg-IFNα降低CHB受试者HBV DNA水平的作用评估731联用ETV±Peg-IFNα降低CHB受试者HBV抗原（即HBeAg、HBcrAg和HBsAg）的作用评估731联用ETV±Peg-IFNα对使丙氨酸氨基转移酶（ALT）恢复正常的作用在CHB受试者中评估731的PK以及在未来评估Peg-IFNα和ETV的PK 评估731联用ETV±Peg-IFNα时耐药性的形成情况

[Translation]

Primary Objective To evaluate the safety and tolerability of 731 combined with ETV±Peg-IFNα in the treatment of CHB subjects Secondary Objectives To evaluate the effect of 731 combined with ETV±Peg-IFNα in reducing HBV pgRNA levels in CHB subjects To evaluate the effect of 731 combined with ETV±Peg-IFNα in reducing HBV DNA levels in CHB subjects To evaluate the effect of 731 combined with ETV±Peg-IFNα in reducing HBV antigens (i.e., HBeAg, HBcrAg, and HBsAg) in CHB subjects To evaluate the effect of 731 combined with ETV±Peg-IFNα in restoring alanine aminotransferase (ALT) to normal To evaluate the PK of 731 in CHB subjects and the PK of Peg-IFNα and ETV in the future To evaluate the development of drug resistance when 731 is combined with ETV±Peg-IFNα

Start Date18 Feb 2021

Sponsor / Collaborator

Ashan Berry Biotechnology (Shanghai) Co., Ltd.

[+2]

100 Clinical Results associated with Vebicorvir

100 Translational Medicine associated with Vebicorvir

100 Patents (Medical) associated with Vebicorvir

Literatures (Medical) associated with Vebicorvir

01 Apr 2024·JHEP reports : innovation in hepatology

Long-term open-label vebicorvir for chronic HBV infection: Safety and off-treatment responses

Article

Author: Weilert, Frank ; Nguyen, Tuan T ; Schiff, Eugene R ; Bonacini, Maurizio ; Han, Steven-Huy ; Agarwal, Kosh ; Gane, Edward J ; Kwo, Paul Yien ; Bennett, Michael ; Stamm, Luisa M ; Hassanein, Tarek ; Fung, Scott ; Hann, Hie-Won ; Ayoub, Walid S ; Yuen, Man-Fung ; Ma, Julie ; Zomorodi, Katie ; Ramji, Alnoor ; Ravendhran, Natarajan ; Sulkowski, Mark S ; Park, James S ; Jacobson, Ira M ; Ma, Xiaoli ; Chan, Sing ; Dieterich, Douglas T ; Nahass, Ronald G ; Bae, Ho S ; Lalezari, Jacob ; Knox, Steven J ; Elkhashab, Magdy ; Yan, Ran

Background & Aims:

The investigational first-generation core inhibitor vebicorvir (VBR) demonstrated safety and antiviral activity over 24 weeks in two phase IIa studies in patients with chronic HBV infection. In this long-term extension study, patients received open-label VBR with nucleos(t)ide reverse transcriptase inhibitors (NrtIs).

Methods:

Patients in this study (NCT03780543) previously received VBR + NrtI or placebo + NrtI in parent studies 201 (NCT03576066) or 202 (NCT03577171). After receiving VBR + NrtI for ≥52 weeks, stopping criteria (based on the treatment history and hepatitis B e antigen status in the parent studies) were applied, and patients either discontinued both VBR + NrtI, discontinued VBR only, or continued both VBR + NrtI. The primary efficacy endpoint was the proportion of patients with HBV DNA <20 IU/ml at 24 weeks off treatment.

Results:

Ninety-two patients entered the extension study and received VBR + NrtI. Long-term VBR + NrtI treatment led to continued suppression of HBV nucleic acids and, to a lesser extent, HBV antigens. Forty-three patients met criteria to discontinue VBR + NrtI, with no patients achieving the primary endpoint; the majority of virologic rebound occurred ≥4 weeks off treatment. Treatment was generally well tolerated, with few discontinuations due to adverse events (AEs). There were no deaths. Most AEs and laboratory abnormalities were related to elevations in alanine aminotransferase and occurred during the off-treatment or NrtI-restart phases. No drug-drug interactions between VBR + NrtI and no cases of treatment-emergent resistance among patients who adhered to treatment were observed.

Conclusions:

Long-term VBR + NrtI was safe and resulted in continued reductions in HBV nucleic acids following completion of the 24-week parent studies. Following treatment discontinuation, virologic relapse was observed in all patients. This first-generation core inhibitor administered with NrtI for at least 52 weeks was not sufficient for HBV cure.

Clinical trial number:

NCT03780543.

Impact and implications:

Approved treatments for chronic hepatitis B virus infection (cHBV) suppress viral replication, but viral rebound is almost always observed after treatment discontinuation, highlighting an unmet need for improved therapies with finite treatment duration producing greater therapeutic responses that can be sustained off treatment. First-generation core inhibitors, such as vebicorvir, have mechanisms of action orthogonal to standard-of-care therapies that deeply suppress HBV viral replication during treatment; however, to date, durable virologic responses have not been observed after treatment discontinuation. The results reported here will help researchers with the design and interpretation of future studies investigating core inhibitors as possible components of finite treatment regimens for patients with cHBV. It is possible that next-generation core inhibitors with enhanced potency may produce deeper and more durable antiviral activity than first-generation agents, including vebicorvir.

06 Apr 2023·Antibiotics (Basel, Switzerland)

Fast and Sensitive Method for Simultaneous Quantification of Meropenem and Vaborbactam in Human Plasma Microsamples by Liquid Chromatography-Tandem Mass Spectrometry for Therapeutic Drug Monitoring.

Article

Author: Conti, Matteo ; Giorgi, Beatrice ; Barone, Rossella ; Cojutti, Pier Giorgio ; Viale, Pierluigi ; Pea, Federico ; Gatti, Milo

Meropenem (MRP)-Vaborbactam (VBR) is a novel beta-lactam/beta-lactamase inhibitor used for the management of difficult-to-treat Gram-negative infections. Among critically ill patients, MRP-VBR shows remarkable inter-individual variability in pharmacokinetic behavior, thus justifying the implementation of therapeutic drug monitoring (TDM) for improving real-time management in different challenging scenarios. In this study, we developed and validated a fast and sensitive Liquid Chromatography-Tandem Mass Spectrometry (LC-MS/MS) method for the simultaneous quantification of MRP and VBR in human plasma microsamples of 3 microliters. The analysis required only a single-step sample preparation and was performed by means of a fast chromatographic run of 4 min, followed by positive electrospray ionization and detection on a high-sensitivity triple quadrupole tandem mass spectrometer operated in multiple reaction monitoring modes. The straightforward analytical procedure was successfully validated, based on the EMA guidelines, in terms of specificity, sensitivity, linearity, precision, accuracy, matrix effect, extraction recovery, the limit of quantification, and stability. The novel method was successfully applied for simultaneously measuring MRP and VBR concentrations in more than 42 plasma samples collected from critically ill patients affected by carbapenem-resistant Gram-negative bacteria infections.

01 Nov 2022·Journal of hepatology

Safety and efficacy of vebicorvir administered with entecavir in treatment-naïve patients with chronic hepatitis B virus infection

Article

Author: Ayoub, Walid S ; Hann, Hie-Won L ; Nguyen, Tuan T ; Hassanein, Tarek ; Evanchik, Marc ; Weilert, Frank ; Alves, Katia ; Ma, Julie ; Chan, Sing ; Lalezari, Jacob P ; Ramji, Alnoor ; Knox, Steven J ; Gane, Edward J ; Yuen, Man-Fung ; Schiff, Eugene R ; Agarwal, Kosh ; Bae, Ho S ; Bonacini, Maurizio ; Lopatin, Uri ; Han, Steven-Huy B ; Park, James S ; Elkhashab, Magdy ; Kwo, Paul Yien ; Ma, Xiaoli ; Huang, Qi ; Ravendhran, Natarajan ; Stamm, Luisa M ; Jacobson, Ira M ; Nahass, Ronald G ; Fung, Scott K ; Colonno, Richard ; Bennett, Michael ; Yan, Ran ; Sulkowski, Mark S ; Dieterich, Douglas T

BACKGROUND & AIMS:

Nucleos(t)ide reverse transcriptase inhibitors do not completely suppress HBV DNA in chronic HBV infection (cHBV). Vebicorvir (VBR) is an investigational core inhibitor that interferes with multiple aspects of HBV replication. This phase II trial evaluated the safety and efficacy of VBR in combination with entecavir (ETV) in treatment-naïve patients with cHBV.

METHODS:

HBeAg-positive, treatment-naïve patients without cirrhosis were randomised 1:1 in a double-blind manner to once-daily VBR 300 mg+ETV 0.5 mg or placebo (PBO)+ETV 0.5 mg for 24 weeks. The primary endpoint was change in mean log₁₀ HBV DNA from Baseline to Week 12 and 24.

RESULTS:

All patients in both treatment groups (PBO+ETV: 12/12; VBR+ETV: 13/13) completed the study. At Week 12, VBR+ETV led to a greater mean (SD) reduction from Baseline in log₁₀ IU/ml HBV DNA (-4.45 [1.03]) vs. PBO+ETV (-3.30 [1.18]; p = 0.0077). At Week 24, VBR+ETV led to a greater reduction from Baseline in log₁₀ IU/ml HBV DNA (-5.33 [1.59]) vs. PBO+ETV (-4.20 [0.98]; p = 0.0084). Greater mean reductions in pregenomic RNA were observed at Week 12 and 24 in patients receiving VBR+ETV vs. PBO+ETV (p <0.0001 and p <0.0001). Changes in viral antigens were similar in both groups. No drug interaction between VBR and ETV was observed. Two patients experienced HBV DNA rebound during treatment, with no resistance breakthrough detected. The safety of VBR+ETV was similar to PBO+ETV. All treatment-emergent adverse events and laboratory abnormalities were Grade 1/2. There were no deaths, serious adverse events, or evidence of drug-induced liver injury.

CONCLUSIONS:

In this 24-week study, VBR+ETV provided additive antiviral activity over PBO+ETV in treatment-naïve patients with cHBV, with a favourable safety and tolerability profile.

CLINICAL TRIAL NUMBER:

NCT03577171 LAY SUMMARY: Hepatitis B is a long-lasting viral infection of the liver. Current treatments can suppress hepatitis B virus but do not offer the opportunity of cure, hence, new treatment approaches are required. Herein, we show that the combination of the novel core inhibitor vebicorvir with an existing antiviral (entecavir) in treatment-naïve patients chronically infected with hepatitis B virus demonstrated greater antiviral activity than entecavir alone. Additionally, vebicorvir was safe and well tolerated. Thus, further studies evaluating its potential role in the treatment of chronic hepatitis B are warranted.

News (Medical) associated with Vebicorvir

08 May 2024

·www.globenewswire.com

Assembly Biosciences Reports First Quarter 2024 Financial Results and Recent Highlights

Clinical trial application clearances received for a Phase 1a/1b trial for ABI-5366 targeting recurrent genital herpes, and a Phase 1b trial for ABI-4334 in chronic HBV infection; both studies on track to initiate by mid-2024 ABI-5366 interim Phase 1a first-in-human data expected in Q3 2024 and ABI-4334 interim Phase 1b data expected by end of year SOUTH SAN FRANCISCO, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative therapeutics targeting serious viral diseases, today reported financial results and recent highlights for the first quarter ended March 31, 2024. “We are pleased that we now have regulatory clearance to initiate trials evaluating our promising investigational therapies ABI-5366 and ABI-4334 and look forward to sharing interim data from these studies later this year,” said Jason Okazaki, chief executive officer and president of Assembly Bio. “These clearances are a significant step toward our goal of reaching key clinical inflection points for multiple programs in our antiviral pipeline by year end. Our continued clinical progress reflects the strength and agility of our highly experienced team and our singular focus of improving treatment options for those living with serious viral diseases.” First Quarter 2024 and Recent Highlights Two clinical trial applications received clearance to proceed: ABI-5366, a long-acting herpes simplex virus (HSV) helicase-primase inhibitor candidate, for initiation of Phase 1a/1b clinical studies in both healthy volunteers and in individuals with recurrent genital herpesABI-4334, a next-generation, highly potent capsid assembly modulator candidate, for initiation of a Phase 1b study in individuals with chronic hepatitis B virus (HBV) infection An abstract highlighting preclinical data for ABI-6250, an oral, small molecule HBV/hepatitis delta virus (HDV) entry inhibitor candidate, has been accepted for poster presentation at the European Association for the Study of the Liver (EASL) CongressTM 2024, June 5-8, 2024 Anticipated 2024 Milestones ABI-5366 and ABI-4334 studies expected to initiate by mid-2024: ABI-5366 Phase 1a interim clinical data expected in Q3 2024 and interim Phase 1b data expected in the first half of 2025ABI-4334 Phase 1b interim clinical data expected by the end of 2024 Two additional candidates are anticipated to enter the clinic by the end of 2024: ABI-1179, a long-acting HSV helicase-primase inhibitor contributed by Gilead Sciences, Inc. (Gilead) under the collaboration between Assembly Bio and GileadABI-6250, a small molecule orally-bioavailable HDV entry inhibitor First Quarter 2024 Financial Results Cash, cash equivalents and marketable securities were $113.0 million as of March 31, 2024, compared to $130.2 million as of December 31, 2023. Assembly Bio’s cash position is projected to fund operations into the second half of 2025.Revenues from collaborative research were $5.8 million for the three months ended March 31, 2024. There was no revenue recognized for the same period in 2023. Revenue for the three months ended March 31, 2024 consists of amounts recognized under the collaboration with Gilead.Research and development expenses were $11.9 million for the three months ended March 31, 2024, compared to $14.5 million for the same period in 2023. Increased expenses related to the development of ABI-1179 and ABI-6250 were more than offset by savings from the discontinuation of ABI-H3733 and vebicorvir as well as reduced employee and contractor-related expenses.General and administrative expenses were $4.6 million for the three months ended March 31, 2024, compared to $5.0 million for the same period in 2023. The decrease is primarily due to a decrease in non-cash stock-based compensation expense.Net loss attributable to common stockholders was $9.1 million, or $1.66 per basic and diluted share, for the three months ended March 31, 2024, compared to $19.0 million, or $4.46 per basic and diluted share, for the same period in 2023. The investigational products and investigational product candidates referenced here have not been approved anywhere globally, and their safety and efficacy have not been established. About Assembly BiosciencesAssembly Biosciences is a biotechnology company dedicated to the development of innovative small-molecule therapeutics designed to change the path of serious viral diseases and improve the lives of patients worldwide. Led by an accomplished team of leaders in virologic drug development, Assembly Bio is committed to improving outcomes for patients struggling with the serious, chronic impacts of herpesvirus, hepatitis B virus (HBV) and hepatitis delta virus (HDV) infections. For more information, visit assemblybio.com. Forward-Looking StatementsThe 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., 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 presented at conferences may not differentiate Assembly Bio’s product candidates from other companies’ candidates; 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 Investor and Corporate: Shannon Ryan SVP, Investor Relations, Corporate Affairs and Alliance Management (415) 738-2992 investor_relations@assemblybio.com Media: Sam Brown Inc.  Hannah Hurdle  (805) 338-4752  ASMBMedia@sambrown.com ASSEMBLY BIOSCIENCES, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands except for share amounts and par value) March 31, December 31, 2024 2023 (Unaudited) ASSETS Current assets Cash and cash equivalents $18,749 $19,841 Marketable securities 94,227 110,406 Accounts receivable from collaboration 43 43 Prepaid expenses and other current assets 4,149 3,497 Total current assets 117,168 133,787 Property and equipment, net 367 385 Operating lease right-of-use assets 2,036 2,339 Other assets 312 312 Total assets $ 119,883 $ 136,823 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $727 $461 Accrued research and development expenses 2,059 885 Other accrued expenses 2,051 5,744 Deferred revenue from a related party - short-term 32,771 30,915 Operating lease liabilities - short-term 1,145 1,220 Total current liabilities 38,753 39,225 Deferred revenue from a related party - long-term 47,738 55,379 Operating lease liabilities - long-term 791 1,122 Total liabilities 87,282 95,726 Commitments and contingencies Stockholders' equity Preferred stock, $0.001 par value; 5,000,000 shares authorized; no shares issued or outstanding — — Common stock, $0.001 par value; 150,000,000 shares authorized as of March 31, 2024 and December 31, 2023; 5,482,752 shares issued and outstanding as of March 31, 2024 and December 31, 2023 5 5 Additional paid-in capital 827,660 826,921 Accumulated other comprehensive loss (239) (81) Accumulated deficit (794,825) (785,748) Total stockholders' equity 32,601 41,097 Total liabilities and stockholders' equity $ 119,883 $ 136,823 ASSEMBLY BIOSCIENCES, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands except for share and per share amounts) (Unaudited) Three Months Ended March 31, 2024 2023 Collaboration revenue from a related party $5,785 $— Operating expenses Research and development 11,879 14,547 General and administrative 4,635 5,012 Total operating expenses 16,514 19,559 Loss from operations (10,729) (19,559) Other income Interest and other income, net 1,652 609 Total other income 1,652 609 Net loss $ (9,077) $ (18,950) Other comprehensive loss Unrealized (loss) gain on marketable securities (158) 290 Comprehensive loss $ (9,235) $ (18,660) Net loss per share, basic and diluted $(1.66) $(4.46) Weighted average common shares outstanding, basic and diluted 5,483,313 4,251,037

Financial StatementPhase 1INDPhase 2

28 Mar 2024

·www.biospace.com

Assembly Biosciences Reports Fourth Quarter and Year End 2023 Financial Results and Recent Highlights

Four candidates planned to be in clinical development in 2024 with interim data from two studies anticipated by year end Clinical development timelines accelerated for ABI-5366; now anticipating interim data from Phase 1a available in 3Q2024, initiation of Phase 1b in individuals with recurrent genital herpes by end of 2024 and interim Phase 1b data available in 1H2025 Gilead Sciences, Inc. partnership supports advancement and strengthening of antiviral pipeline targeting serious viral diseases SOUTH SAN FRANCISCO, Calif., March 28, 2024 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative therapeutics targeting serious viral diseases, today reported financial results and recent highlights for the fourth quarter and year ended December 31, 2023. “Our progress in 2023 demonstrated our capacity to drive our expanded antiviral pipeline forward and our commitment to individuals suffering from life-impacting viral diseases,” said Jason Okazaki, chief executive officer and president of Assembly Bio. “With our long-term partnership in place with Gilead and the addition of Dr. Anuj Gaggar to our management team as CMO, we are well-positioned to make critical progress clinically across multiple programs in 2024 and look forward to sharing preliminary data on 5366 and 4334 later this year.” Fourth Quarter 2023 and Recent Highlights Entered a long-term partnership with Gilead to advance discovery and development of novel antiviral therapies Strengthened the leadership team with the naming of Anuj Gaggar, MD, PhD, as chief medical officer Filed a clinical trial application for ABI-5366 to support initiation of Phase 1a/1b clinical studies Anticipated Milestones and Events By mid-year 2024: ABI-5366, a long-acting herpes simplex virus (HSV) helicase-primase inhibitor targeting recurrent genital herpes, is expected to enter a Phase 1a/1b study ABI-4334, a next-generation, highly potent capsid assembly modulator, is anticipated to enter a Phase 1b study in individuals with chronic hepatitis B virus infection Q3 2024: Interim clinical data from the ABI-5366 Phase 1a study portion expected to be available By the end of 2024: Interim clinical data from the ABI-4334 Phase 1b study expected to be available The Phase 1b portion of the ABI-5366 study, in individuals with recurrent genital herpes, is anticipated to be underway Two additional candidates are anticipated to enter the clinic: ABI-1179, a long-acting HSV helicase-primase inhibitor contributed by Gilead under the collaboration between Assembly Bio and Gilead ABI-6250, a small molecule orally-bioavailable hepatitis delta virus entry inhibitor First half 2025: Interim clinical data from the Phase 1b portion of the ABI-5366 study expected to be available Fourth Quarter 2023 and Year End Financial Results Cash, cash equivalents and marketable securities were $130.2 million as of December 31, 2023, compared to $46.2 million as of September 30, 2023, and $91.6 million as of the year ended December 31, 2022. The company’s cash position is projected to fund operations into the second half of 2025. Revenues from collaborative research were $7.2 million for the year ended December 31, 2023. There was no revenue recognized for the same period in 2022. Revenue for the year ended December 31, 2023, consists of $4.4 million recognized under the collaboration with Gilead and the remaining deferred revenue balance of $2.7 million under the collaboration agreement with BeiGene following Assembly Bio’s decision to discontinue further development of ABI-H3733 in conjunction with entering into the Gilead collaboration, following the previous prioritization of ABI-4334 over ABI-H3733. Research and development expenses were $48.9 million for the year ended December 31, 2023, compared to $70.0 million in 2022. The decrease is due to completion of the clinical trials for ABI-H3733 and ABI-4334, discontinued development of vebicorvir and ABI-H2158, and decreases in employee and contractor-related expenses. General and administrative expenses were $22.9 million for the year ended December 31, 2023, compared to $24.1 million in 2022. The decrease is due to overall cost-saving initiatives. Net loss attributable to common stockholders was $61.2 million, or $13.38 per basic and diluted share, for the year ended December 31, 2023, compared to $93.1 million, or $23.08 per basic and diluted share in 2022. The investigational products and investigational product candidates referenced here have not been approved anywhere globally, and their 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 diseases and improve the lives of patients worldwide. Led by an accomplished team of leaders in virologic drug development, Assembly Bio is committed to improving outcomes for patients struggling with the serious, chronic impacts of herpesvirus, hepatitis B virus (HBV) and hepatitis delta virus (HDV) infections. 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., 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 presented at conferences may not differentiate Assembly Bio’s product candidates from other companies’ candidates; 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 Investor and Corporate: Shannon Ryan SVP, Investor Relations, Corporate Affairs and Alliance Management (415) 738-2992 investor_relations@assemblybio.com Media: Sam Brown Inc.  Hannah Hurdle  (805) 338-4752  ASMBMedia@sambrown.com ASSEMBLY BIOSCIENCES, INC. CONSOLIDATED BALANCE SHEETS (In thousands except for share amounts and par value) As of December 31, 2023 2022 ASSETS Current assets Cash and cash equivalents $ 19,841 $ 52,418 Marketable securities 110,406 39,192 Accounts receivable from collaboration 43 944 Prepaid expenses and other current assets 3,497 4,413 Total current assets 133,787 96,967 Property and equipment, net 385 743 Operating lease right-of-use assets 2,339 3,195 Other assets 312 889 Total assets $ 136,823 $ 101,794 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $ 461 $ 2,493 Accrued research and development expenses 885 3,122 Other accrued expenses 5,744 7,317 Deferred revenue - short-term ($30,915 and $− to a related party) 30,915 — Operating lease liabilities - short-term 1,220 3,364 Total current liabilities 39,225 16,296 Deferred revenue - long-term ($55,379 and $− to a related party) 55,379 2,733 Operating lease liabilities - long-term 1,122 101 Total liabilities 95,726 19,130 Commitments and contingencies Stockholders' equity Preferred stock, $0.001 par value; 5,000,000 shares authorized; no shares issued or outstanding — — Common stock, $0.001 par value; 150,000,000 shares authorized as of December 31, 2023 and December 31, 2022; 5,482,752 and 4,074,552 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively 5 4 Additional paid-in capital 826,921 807,983 Accumulated other comprehensive loss (81 ) (803 ) Accumulated deficit (785,748 ) (724,520 ) Total stockholders' equity 41,097 82,664 Total liabilities and stockholders' equity $ 136,823 $ 101,794 ASSEMBLY BIOSCIENCES, INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands except for share and per share amounts) Year Ended December 31, 2023 2022 Collaboration revenue ($4,430 and $− from a related party) $ 7,163 $ — Operating expenses Research and development 48,900 69,980 General and administrative 22,909 24,134 Total operating expenses 71,809 94,114 Loss from operations (64,646 ) (94,114 ) Other income Interest and other income, net 3,451 1,022 Total other income 3,451 1,022 Loss before income taxes (61,195 ) (93,092 ) Income tax expense (33 ) — Net loss $ (61,228 ) $ (93,092 ) Other comprehensive loss Unrealized gain (loss) on marketable securities 722 (384 ) Comprehensive loss $ (60,506 ) $ (93,476 ) Net loss per share, basic and diluted $ (13.38 ) $ (23.08 ) Weighted average common shares outstanding, basic and diluted 4,577,371 4,034,105

Financial StatementPhase 1Phase 2Executive Change

10 Nov 2023

·www.globenewswire.com

Assembly Biosciences Presents New Data Highlighting Viral Hepatitis Portfolio at AASLD The Liver Meeting®

--Poster highlights potential of oral small molecule IFNAR agonist program to engage the IFNα pathway through a liver-focused approach to improve tolerability-- SOUTH SAN FRANCISCO, Calif., Nov. 10, 2023 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative antiviral therapeutics targeting serious viral diseases, today announced new data from its virology portfolio featured in two presentations at the American Association for the Study of Liver Diseases (AASLD), The Liver Meeting®, taking place November 10-14, 2023, in Boston. “These data underscore our continued commitment to advancing innovative programs with the potential to change the treatment paradigm for patients impacted by serious viral diseases and highlight the promise of our preclinical IFNAR agonist program, which is in lead optimization,” said William Delaney, PhD, chief scientific officer of Assembly Bio. “Additionally, the Phase 2 data reported for vebicorvir in combination with Nrtl and Arbutus Biopharma’s AB-729 provide further insights that may inform future studies for the HBV and liver disease scientific community." A poster presentation entitled “In Vitro and in Vivo Profiling of Orally Bioavailable Small Molecules Inhibiting Hepatitis B Virus by Mimicking Interferon Alpha” highlights preclinical data describing the inhibition of hepatitis B virus (HBV) and hepatitis C virus (HCV) in vitro through the activation of interferon (IFN) signaling by novel small molecule interferon-α (IFNα) receptor (IFNAR) agonists. The IFNAR agonists described in this poster closely mimic the biological activity of IFNα in vitro by stimulating cytokine secretion and inducing IFN-stimulated genes (ISG) following treatment of human cells. In addition, oral administration to preclinical species resulted in ISG induction in the liver and peripheral blood mononuclear cells (PBMCs). Additionally, an oral presentation is scheduled entitled “Preliminary Off-Treatment Responses Following 48 Weeks of Vebicorvir, Nucleos(t)ide Reverse Transcriptase Inhibitor, and AB-729 Combination in Virologically Suppressed Patients With Hepatitis B e Antigen Negative Chronic Hepatitis B: Analysis From an Open-Label Phase 2 Study.” Results described in this presentation indicate that first-generation core inhibitor vebicorvir (VBR) in combination with nucleos(t)ide reverse transcriptase inhibitor (Nrtl) and Arbutus Biopharma’s AB-729 in virologically suppressed patients with hepatitis B e antigen negative chronic HBV did not result in significantly greater on- or post-treatment improvements in markers of active HBV infection versus the dual combination without VBR. Assembly Bio discontinued development of VBR in 2022. Assembly Bio intends to make the presentations available on the “Events & Presentations” page in the “Investors” section of its website at www.assemblybio.com. About Assembly BiosciencesAssembly Biosciences is a biotechnology company dedicated to the development of innovative small-molecule antiviral therapeutics designed to change the path of serious viral diseases and improve the lives of patients worldwide. Led by an accomplished team of leaders in virologic drug development, Assembly Bio is committed to improving outcomes for patients struggling with the serious, chronic impacts of herpesvirus, hepatitis B virus (HBV) and hepatitis delta virus (HDV) infections. For more information, visit assemblybio.com. Forward-Looking StatementsThe 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, 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; the occurrence of any event, change or other circumstance that could give rise to the termination of Assembly Bio’s collaboration with Gilead; safety and efficacy data from clinical or nonclinical studies may not warrant further development of Assembly Bio’s product candidates; clinical and nonclinical data presented at conferences may not differentiate Assembly Bio’s product candidates from other companies’ candidates; 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. ContactsInvestor and Corporate: Shannon Ryan SVP, Investor Relations, Corporate Affairs and Alliance Management (415) 738-2992 sryan@assemblybio.com Media: Sam Brown Inc.Hannah Hurdle(805) 338-4752ASMBMedia@sambrown.com

Phase 2Clinical Result

100 Deals associated with Vebicorvir

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Indication	Highest Phase	Country/Location	Organization	Date
Hepatitis B, Chronic	Phase 2	Australia	Assembly Biosciences, Inc.	15 Jun 2017
Hepatitis B	Phase 2	-	Indiana University	-

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04781647 (CTgov)	Phase 2	Hepatitis B, Chronic	54	ETV+ABI-H0731 (ABI-H0731 + ETV)	blddnmueew = yodiiphwfc rbbldyrpaf (viirlleuvj, zjdjwgrilk - hzeqbcynli) View more	-	06 Oct 2023
				Peg-IFNα+ETV+ABI-H0731 (ABI-H0731 + ETV + Peg-IFNα)	blddnmueew = bvvddzkhxr rbbldyrpaf (viirlleuvj, slrfenubmw - sffwnsrusb) View more
NCT04454567 (CTgov)	Phase 2	Hepatitis B, Chronic	2	NrtI+ABI-H0731 (ABI-H0731 + SOC NrtI)	khwsvgaccz = nwqkbzhuqo jdtmibgzeo (phgftutddc, qutmceheso - qvtrtipvgx) View more	-	20 Oct 2022
				NrtI+ABI-H0731 (Placebo + SOC NrtI)	khwsvgaccz = wyrpqitxdi jdtmibgzeo (phgftutddc, tnsscymyjg - akotwkqebv) View more
NCT03577171 (Study 202, EASL2022)	Phase 2	Hepatitis B, Chronic	25	Vebicorvir 300 mg + Entecavir 0.5 mg	jdvtochyhx(snlrtqwgoi) = whnankondh qlwwkhheps (scmajhkpou )	-	25 Jun 2022
				Placebo + Entecavir 0.5 mg	qtulzdxeki(adwqjaubit) = uatkgeapxw esrfblviry (ndiwzuyuxf ) View more
NCT03577171 (Study 202, EASL2022)	Phase 2	Hepatitis B HBeAg positive \| HBcrAg \| HBV DNA ... View more	-	Vebicorvir+Entecavir	ywbvxiahig(chcdcgqumw) = yyfcioxhhm javfcaouym (xbykkyusjr )	-	23 Jun 2022
NCT03577171 (Pubmed)	Phase 2	Hepatitis B, Chronic	25	Vebicorvir 300 mg+Entecavir 0.5 mg	vafzgkzymz(gzegxaylwv) = heixyrpfeh pdasnyfuel (ksoiacpsjz ) View more	-	10 Jun 2022
				Placebo+Entecavir 0.5 mg	vafzgkzymz(gzegxaylwv) = cgprirdivb pdasnyfuel (ksoiacpsjz ) View more
Study 211 (EASL2021)	Not Applicable	Hepatitis B, Chronic eAg positive \| sAg \| crAg	41	Vebicorvir	yuwzkmjlgi(xrbnlelbdi) = ALT elevation in 4/41 (10%) pts gvxrlkegqk (wkgojekjpk ) View more	-	23 Jun 2021
				Placebo+NrtI
NCT03577171 (ABI-H0731-202 \| Study 202, CTgov)	Phase 2	Hepatitis B, Chronic	25	SOC ETV+ABI-H0731 (ABI-H0731 + SOC ETV)	udyvzcrnhx(bzvdzadisb) = jsbnyidusl joaxwbdwnn (kdasqrwykl, 0.890) View more	-	28 Jan 2021
				Placebo Oral Tablet+SOC ETV (Placebo + SOC ETV)	udyvzcrnhx(bzvdzadisb) = gzxluwebsj joaxwbdwnn (kdasqrwykl, 0.999) View more
NCT03576066 (ABI-H0731-201, CTgov)	Phase 2	Hepatitis B, Chronic	73	SOC NUC+ABI-H0731 (ABI-H0731 + SOC NUC)	xpnnrnnhzp = qcdkohbsdb matrvdjyiu (cyiacxmpsf, xboqpunlpj - gmmywpqyot) View more	-	28 Jan 2021
				Placebo Oral Tablet (Placebo + SOC NUC)	xpnnrnnhzp = sqbgwpjotf matrvdjyiu (cyiacxmpsf, omwdnlarfr - sebezqfxtn) View more
ABI-H0731-211 (EASL2020)	Phase 2	Hepatitis B, Chronic	-	ABI-H0731	jrmvfucqlq(xcijzpfyzp) = dttvxcxios tscsomqajg (wwdmspmmbz ) View more	-	27 Aug 2020

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