[Translation] A multicenter, single-blind, placebo-controlled, multi-cohort phase 2a study evaluating an ABI-H2158-containing regimen for the treatment of chronic hepatitis B infection

在CHB受试者中评价ABI-H2158 + ETV联合治疗的安全性和耐受性; 在CHB受试者中评价ABI-H2158在降低HBV DNA方面的作用

[Translation]

To evaluate the safety and tolerability of ABI-H2158 + ETV combination therapy in CHB subjects; To evaluate the effect of ABI-H2158 in reducing HBV DNA in CHB subjects

Start Date07 Apr 2021

Sponsor / Collaborator

Ashan Berry Biotechnology (Shanghai) Co., Ltd.

[+2]

NCT04398134 / TerminatedPhase 2

A Phase 2a, Multicenter, Single-Blind, Placebo-Controlled, Multiple Cohort Study Evaluating ABI-H2158-Containing Regimens in Chronic Hepatitis B Infection

This Phase 2a study will assess the safety, antiviral activity, and pharmacokinetics (PK) of ABI-H2158 administered once daily for up to 72 weeks in combination with entecavir (ETV) in participants with chronic hepatitis B virus (HBV) infection.

Start Date28 Aug 2020

Sponsor / Collaborator

Assembly Biosciences, Inc.

NCT04142762 / CompletedPhase 1

A Phase 1, Open-Label, Fixed-Sequence Study to Evaluate CYP3A4-Mediated, Oral Contraceptive, and pH Modifier Drug Interactions for ABI-H2158 in Healthy Adult Subjects

This study is designed to assess 1) the effect of multiple doses of itraconazole (CYP3A4 inhibitor), rifampin (CYP3A4 inducer), and esomeprazole (pH modifier) on the pharmacokinetics of a single oral dose of ABI-H2158, and 2) the effect of steady-state oral ABI-H2158 on the pharmacokinetics of midazolam (sedative) and levonorgestrel/ethinyl estradiol (active oral contraceptive) in healthy adult participants.

Start Date18 Oct 2019

Sponsor / Collaborator

Assembly Biosciences, Inc.

100 Clinical Results associated with ABI-H2158

100 Translational Medicine associated with ABI-H2158

100 Patents (Medical) associated with ABI-H2158

Literatures (Medical) associated with ABI-H2158

01 Mar 2023·Journal of viral hepatitis

Safety, pharmacokinetics and antiviral activity of ABI‐H2158, a hepatitis B virus core inhibitor: A randomized, placebo‐controlled phase 1 study

Article

Author: Ding, Yanhua ; Stamm, Luisa M. ; Huang, Qi ; Ma, Julie ; Yuen, Man‐Fung ; Nguyen, Tuan T. ; Hassanein, Tarek I. ; Lim, Young‐Suk ; Gane, Edward J. ; Alves, Katia ; Yan, Ran ; Colonno, Richard ; Agarwal, Kosh ; Zomorodi, Katie ; Knox, Steven J. ; Xu, Jia ; Kim, Dong Joon ; Evanchik, Marc

Abstract:

Treatment for chronic hepatitis B virus infection (cHBV) is mostly indefinite, with new finite‐duration therapies needed. We report safety, pharmacokinetics and antiviral activity of the investigational HBV core inhibitor ABI‐H2158. This Phase 1a/b study (NCT03714152) had three parts: Part A, participants received a single ascending oral dose of ABI‐H2158 (5–500 mg) or placebo; Part B, participants received multiple doses of ABI‐H2158 300 mg once (QD) or twice (BID) daily or placebo, for 10 days; Part C, cHBV patients received ABI‐H2158 (100, 300, or 500 mg QD or 300 mg BID) or placebo, for 14 days. Ninety‐three participants enrolled. In Parts A/B, there were no serious adverse events (SAEs) or deaths, and all treatment‐emergent AEs (TEAEs) were Grade 1. In Part C, two patients had Grade 3 TEAEs unrelated to ABI‐H2158; there were no deaths, SAEs or Grade 4 TEAEs. In Part A, median time to maximum ABI‐H2158 plasma concentration (Tmax) and mean terminal elimination half‐life (t½) were 1–4 and 9.8–20.7 h, and area under the plasma concentration‐time curve increased dose proportionally. In Part B, Day 10 Tmax was 2 h, mean t½ was 15.5–18.4 h, and exposure accumulated 1.7‐ to 3.1‐fold. In Part C, Day 14 Tmax was 1 h, exposure accumulated 1.4‐ to 1.8‐fold, and ABI‐H2158 was associated with >2 log10 declines in HBV nucleic acids. In conclusion, ABI‐H2158 in cHBV patients following 14 days of dosing was well tolerated and demonstrated potent antiviral activity. Safety and pharmacokinetics supported future QD dosing.

01 Jan 2023·Antiviral research

Preclinical characterization of ABI-H2158, an HBV core inhibitor with dual mechanisms of action

Article

Author: Cai, Dawei ; Guo, Lida ; Walker, Michael A ; Zhou, Yi ; Huang, Qi ; Yan, Ran ; Tang, Ariel ; Colonno, Richard ; Li, Lichun ; Zong, Yuhua

The HBV core protein plays an integral role in multiple steps of the HBV lifecycle. Consequently, HBV core inhibitors interrupt multiple steps of the replication cycle, including blocking pgRNA encapsidation and prematurely disassembling existing nucleocapsids, thereby preventing them from transporting relaxed circular (rcDNA) to the nucleus for conversion to covalently closed circular DNA (cccDNA). ABI-H2158 is an HBV core inhibitor that advanced into Phase 2 clinical trials for the treatment of chronic hepatitis B virus infection (cHBV) but was discontinued due to hepatotoxicity. Here, the potency, selectivity, and mechanisms of action of ABI-H2158 were evaluated using a variety of cell-based assays. Antiviral activity was measured by quantifying intracellular or secreted HBV DNA, RNA, and antigens. ABI-H2158 inhibited HBV replication by blocking pgRNA encapsidation in induced HepAD38 cells (EC₅₀ = 22 nM) and had similar potency in HBV-infected HepG2-NTCP cells (EC₅₀ = 27 nM) and primary human hepatocytes (PHH) (EC₅₀ = 41 nM). ABI-H2158 is a pan-genotypic HBV inhibitor, with EC₅₀s ranging from 7.1 to 22 nM across HBV genotypes A-E. ABI-H2158 also potently blocked the formation of cccDNA in de novo HBV infections with EC₅₀s of ∼200 nM in HepG2-NTCP and PHH assays. These results indicate ABI-H2158 has dual mechanisms of action, inhibiting both early and late steps of the HBV replication cycle.

15 Apr 2014·Journal of virologyQ2 · MEDICINE

Functional Implications of the Binding Mode of a Human Conformation-Dependent V2 Monoclonal Antibody against HIV

Q2 · MEDICINE

Article

Author: Gorny, Miroslaw K. ; Spurrier, Brett ; Sampson, Jared ; Zolla-Pazner, Susan ; Kong, Xiang-Peng

ABSTRACT:

Data from the RV144 HIV vaccine trial indicated that gp120 V2 antibodies were associated with a lower risk of infection; thus, the mapping of V2 epitopes can contribute to the design of an effective HIV vaccine. We solved the crystal structure of human monoclonal antibody (MAb) 2158, which targets a conformational V2 epitope overlapping the α4β7 integrin binding site, and constructed a full-length model of V1V2. Comparison of computational energy stability to experimental enzyme-linked immunosorbent assay (ELISA) results identified a hydrophobic core that stabilizes the V2 region for optimal 2158 binding, as well as residues that directly mediate side chain interactions with MAb 2158. These data define the binding surface recognized by MAb 2158 and offer a structural explanation for why a mismatched mutation at position 181 (I181X) in the V2 loop was associated with a higher vaccine efficiency in the RV144 clinical vaccine trial.IMPORTANCECorrelate analysis of the RV144 HIV-1 vaccine trial suggested that the presence of antibodies to the second variable region (V2) of HIV-1 gp120 was responsible for the modest protection observed in the trial. V2 is a highly variable and immunogenic region, and structural information on its antigenic landscape will be important for rational design of an effective HIV-1 vaccine. Using X-ray crystallography, computational design tools, and mutagenesis assays, we carried out a detailed and systematic investigation of the epitope recognition of human V2 MAb 2158 and demonstrated that its epitope region overlaps the integrin binding site within V2. In addition, we propose a structure-based mechanism for mismatching of the isoleucine at position 181 and the increased vaccine efficacy seen in the RV144 vaccine trial.

News (Medical) associated with ABI-H2158

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

08 Nov 2023

·www.globenewswire.com

Assembly Biosciences Reports Third Quarter 2023 Financial Results and Recent Updates

Established partnership with Gilead Sciences to advance differentiated treatments for herpesviruses, HBV, HDV and beyondNow anticipate four development candidates from expanded pipeline in clinical development by the end of 2024Named Anuj Gaggar, MD, PhD, as chief medical officer $100 million upfront payment and investment received from Gilead in October extends projected cash runway into 2H 2025 SOUTH SAN FRANCISCO, Calif., Nov. 08, 2023 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative antiviral therapeutics targeting serious viral diseases, today reported financial results for the third quarter ended September 30, 2023, and recent corporate updates. “The Assembly Bio team has made exceptional progress in building a portfolio of promising antiviral candidates for herpesviruses, hepatitis B and hepatitis D,” said Jason Okazaki, chief executive officer and president of Assembly Bio. “With our new long-term partnership with Gilead, a pioneer in virology, we are excited by the opportunity to accelerate progress on our antiviral pipeline to bring next-generation therapeutics to individuals living with serious viral infections. We believe that the financial and scientific resources provided by the collaboration position us well to reach critical short- and long-term milestones and deliver value for patients and our shareholders. We are also pleased to announce the addition of Dr. Anuj Gaggar to our leadership team as chief medical officer as we look ahead to having multiple antiviral candidates in the clinic next year.” Third Quarter 2023 and Recent Updates Entered a long-term partnership with Gilead Sciences to advance discovery and development of novel antiviral therapies. Collaboration combines Assembly Bio’s advanced virology research capabilities with Gilead’s established development and commercialization expertise in antiviral medicines to create a synergistic partnership focused on propelling scientific innovation and delivering new treatments to patients with significant unmet need.Partnership includes Gilead opt-in rights on Assembly Bio’s current and future pipeline candidates, including two contributed Gilead herpesvirus programs.$100 million upfront investment by Gilead, including equity investment, plus potential for future regulatory, commercial, opt-in and collaboration extension payments, provides runway to drive portfolio growth and support advancement of company’s current and future clinical and preclinical programs. Strengthened the leadership team with the naming of Anuj Gaggar, MD, PhD, as chief medical officer, as announced separately today. Dr. Gaggar is an infectious disease specialist who has focused on the development of new therapies in viral diseases including chronic hepatitis B virus (HBV), hepatitis C virus and hepatitis D virus (HDV) infections.Nominated first development candidate for the treatment of chronic HDV infection. ABI-6250, an orally bioavailable small molecule entry inhibitor, is moving toward IND-enabling studies with the goal of beginning clinical studies by the end of 2024.Presented preclinical data in two oral and one poster presentation highlighting the progress of multiple HBV and HDV pipeline programs at the 2023 International HBV Meeting held September 19-23, 2023. Anticipated Milestones Assembly Bio anticipates having four candidates in clinical studies by the end of 2024: ABI-5366, a long-acting helicase-primase inhibitor targeting high-recurrence genital herpes, is expected to enter the clinic by mid-2024.The herpes simplex virus (HSV) helicase-primase inhibitor candidate contributed by Gilead is expected to enter the clinic by the end of 2024.ABI-4334, a potent next-generation capsid assembly modulator for HBV, has completed Phase 1a development and is expected to enter Phase 1b by mid-2024.ABI-6250, a HDV entry inhibitor, is expected to enter the clinic by the end of 2024. Upcoming Conferences Preclinical and clinical data from the company’s viral hepatitis portfolio will be highlighted 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. Third Quarter 2023 Financial Results Cash, cash equivalents and marketable securities were $46.2 million as of September 30, 2023, compared to $59.8 million as of June 30, 2023. With the $100 million received from Gilead in October, Assembly Bio’s cash position is projected to fund operations into the second half of 2025.Research and development expenses were $10.8 million for the three months ended September 30, 2023, compared to $18.1 million for the same period in 2022. The decrease is due to completion of the clinical trials for ABI-3733 and ABI-4334, discontinued development of vebicorvir and ABI-2158, and decreases in employee and contractor-related expenses.General and administrative expenses were $4.2 million for the three months ended September 30, 2023, compared to $5.3 million for the same period in 2022. The decrease is due to overall cost-saving initiatives.Net loss attributable to common stockholders was $14.4 million, or $0.27 per basic and diluted share, for the three months ended September 30, 2023, compared to $23.1 million, or $0.48 per basic and diluted share, for the same period in 2022. 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 ASSEMBLY BIOSCIENCES, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(In thousands except for share amounts and par value) September 30, December 31, 2023 2022 (Unaudited) ASSETS Current assets Cash and cash equivalents $26,656 $52,418 Marketable securities 19,554 39,192 Accounts receivable from collaboration — 944 Prepaid expenses and other current assets 3,462 4,413 Total current assets 49,672 96,967 Property and equipment, net 561 743 Operating lease right-of-use assets 844 3,195 Other assets 552 889 Total assets $ 51,629 $ 101,794 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $758 $2,493 Accrued research and development expenses 1,619 3,122 Other accrued expenses 3,886 7,317 Operating lease liabilities - short-term 869 3,364 Total current liabilities 7,132 16,296 Deferred revenue 2,733 2,733 Operating lease liabilities - long-term 50 101 Total liabilities 9,915 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 September 30, 2023 and December 31, 2022; 52,614,194 and 48,894,973 shares issued and outstanding as of September 30, 2023 and December 31, 2022, respectively 53 49 Additional paid-in capital 816,722 807,938 Accumulated other comprehensive loss (275) (803)Accumulated deficit (774,786) (724,520)Total stockholders' equity 41,714 82,664 Total liabilities and stockholders' equity $ 51,629 $ 101,794 ASSEMBLY BIOSCIENCES, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(In thousands except for share and per share amounts)(Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Operating expenses Research and development $10,824 $18,130 $37,894 $53,127 General and administrative 4,224 5,271 14,201 18,009 Total operating expenses 15,048 23,401 52,095 71,136 Loss from operations (15,048) (23,401) (52,095) (71,136) Other income Interest and other income, net 628 256 1,829 439 Total other income 628 256 1,829 439 Net loss $ (14,420) $ (23,145) $ (50,266) $ (70,697) Other comprehensive loss Unrealized gain (loss) on marketable securities 50 (1) 528 (580)Comprehensive loss $ (14,370) $ (23,146) $ (49,738) $ (71,277) Net loss per share, basic and diluted $(0.27) $(0.48) $(0.97) $(1.46)Weighted average common shares outstanding, basic and diluted 52,565,333 48,448,399 51,951,123 48,289,501

Financial StatementExecutive ChangePhase 2

09 Aug 2023

·www.biospace.com

Assembly Biosciences Reports Second Quarter 2023 Financial Results and Recent Updates

Herpesvirus candidate ABI-5366 anticipated to enter clinic in first half of 2024 New data for several pipeline programs presented at IHW and EASL 2023 Additional development candidate nomination expected in 2023 SOUTH SAN FRANCISCO, Calif., Aug. 09, 2023 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative antiviral therapeutics targeting serious viral diseases, today reported financial results and recent updates for the second quarter ended June 30, 2023. “We are excited about the advancement of 5366, our long-acting helicase-primase inhibitor targeting high-recurrence genital herpes, into GLP toxicology studies and are working quickly to begin clinical trials for this candidate in the first half of next year,” said Jason Okazaki, chief executive officer and president of Assembly Bio. “In parallel, our research organization continues to progress towards a second development candidate nomination, which we expect later this year. We are pleased with the progress of our expanded virology pipeline, for which strong preclinical data from multiple programs were highlighted at recent scientific meetings.” Second Quarter 2023 and Recent Updates Continued IND-enabling studies, including initiation of GLP toxicology studies, for development candidate ABI-5366 (5366), a long-acting helicase-primase inhibitor targeting high-recurrence genital herpes Scientific conferences: Presented data highlighting the preclinical characterization of 5366 in one oral presentation and one poster at the 47th Annual International Herpesvirus Workshop (IHW) held July 15-19, 2023 Presented five posters at the International Liver CongressTM, the Annual Meeting of the European Association for the Study of the Liver (EASL), held June 21-24, 2023, including preclinical data for the company’s oral, small-molecule hepatitis B virus (HBV)/hepatitis delta virus (HDV) entry inhibitor program and results from in vitro and clinical studies of the next-generation core inhibitors ABI-4334 (4334) and ABI-H3733 (3733) Presented during the Science of HBV Cure meeting held June 2-3, 2023, on 4334 Anticipated Milestones and Events Nominate an additional development candidate from the expanded research portfolio in 2023 Complete IND/CTA submission for 5366 and initiate clinical studies in the first half of 2024 Advance an additional development candidate into clinical studies by year-end 2024 Upcoming Conferences Three abstracts accepted for presentation at the 2023 International HBV Meeting taking place September 19-23, 2023, in Kobe, Japan, highlighting preclinical and in vitro data for the company’s oral, small-molecule HBV/HDV entry inhibitor program, small-molecule, liver-focused interferon alpha receptor agonist program and next-generation core inhibitor candidate 4334 Second Quarter 2023 Financial Results Cash, cash equivalents and marketable securities were $59.8 million as of June 30, 2023, compared to $73.0 million as of March 31, 2023. Assembly Bio’s cash position is projected to fund operations through the third quarter of 2024. Research and development expenses were $12.5 million for the three months ended June 30, 2023, compared to $17.8 million for the same period in 2022. The decrease is primarily due to prior year preclinical work and start-up costs preparing for the trials for 3733 and 4334 as well as the discontinued development of vebicorvir and ABI-H2158 and decreases in employee and contractor-related expenses. General and administrative expenses were $5.0 million for the three months ended June 30, 2023, compared to $6.8 million for the same period in 2022. The decrease is primarily due to overall cost-saving initiatives. Net loss attributable to common stockholders was $16.9 million, or $0.32 per basic and diluted share, for the three months ended June 30, 2023, compared to $24.5 million, or $0.51 per basic and diluted share, for the same period in 2022. About Assembly Biosciences Assembly 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 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 maintain financial resources necessary to continue its clinical studies and fund business operations; Assembly Bio’s ability to initiate and complete clinical studies involving its therapeutic product candidates, including studies contemplated by Assembly Bio’s collaboration agreements, in the currently anticipated timeframes; 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 sryan@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) June 30, December 31, 2023 2022 (Unaudited) ASSETS Current assets Cash and cash equivalents $ 21,623 $ 52,418 Marketable securities 38,208 39,192 Accounts receivable from collaboration 342 944 Prepaid expenses and other current assets 3,908 4,413 Total current assets 64,081 96,967 Property and equipment, net 541 743 Operating lease right-of-use (ROU) assets 1,623 3,195 Other assets 332 889 Total assets $ 66,577 $ 101,794 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $ 904 $ 2,493 Accrued research and development expenses 2,037 3,122 Other accrued expenses 4,188 7,317 Operating lease liabilities - short-term 1,702 3,364 Total current liabilities 8,831 16,296 Deferred revenue 2,733 2,733 Operating lease liabilities - long-term 64 101 Total liabilities 11,628 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 June 30, 2023 and December 31, 2022; 52,450,731 and 48,894,973 shares issued and outstanding as of March 31, 2023 and December 31, 2022, respectively 52 49 Additional paid-in capital 815,588 807,938 Accumulated other comprehensive loss (325 ) (803 ) Accumulated deficit (760,366 ) (724,560 ) Total stockholders' equity 54,949 82,624 Total liabilities and stockholders' equity $ 66,577 $ 101,794 ASSEMBLY BIOSCIENCES, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands except for share and per share amounts) (Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Operating expenses Research and development 12,523 17,792 27,070 34,997 General and administrative 4,965 6,781 9,977 12,738 Total operating expenses 17,488 24,573 37,047 47,735 Loss from operations (17,488 ) (24,573 ) (37,047 ) (47,735 ) Other income Interest and other income, net 592 112 1,201 183 Total other income 592 112 1,201 183 Net loss $ (16,896 ) $ (24,461 ) $ (35,846 ) $ (47,552 ) Other comprehensive loss Unrealized gain (loss) on marketable securities 188 (90 ) 478 (579 ) Comprehensive loss $ (16,708 ) $ (24,551 ) $ (35,368 ) $ (48,131 ) Net loss per share, basic and diluted $ (0.32 ) $ (0.51 ) $ (0.69 ) $ (0.99 ) Weighted average common shares outstanding, basic and diluted 52,260,088 48,293,495 51,638,928 48,208,735

Financial Statement

100 Deals associated with ABI-H2158

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Hepatitis B	Phase 2	CN	BeiGene Ltd.	21 Jul 2020
Hepatitis B, Chronic	Phase 2	US	Austarpharma, Inc.	25 Jun 2020
Hepatitis B, Chronic	Phase 2	US	Assembly Biosciences, Inc.	25 Jun 2020
Hepatitis B, Chronic	Phase 2	AU	Assembly Biosciences, Inc.	25 Jun 2020
Hepatitis B, Chronic	Phase 2	AU	Austarpharma, Inc.	25 Jun 2020
Hepatitis B, Chronic	Phase 2	CA	Austarpharma, Inc.	25 Jun 2020
Hepatitis B, Chronic	Phase 2	CA	Assembly Biosciences, Inc.	25 Jun 2020
Hepatitis B, Chronic	Phase 2	NZ	Assembly Biosciences, Inc.	25 Jun 2020
Hepatitis B, Chronic	Phase 2	NZ	Austarpharma, Inc.	25 Jun 2020
Hepatitis B, Chronic	Phase 2	KR	Austarpharma, Inc.	25 Jun 2020

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04398134 (CTgov)	Phase 2	Hepatitis B, Chronic	88	ETV+ABI-H2158 (ABI-H2158 Plus ETV (HBeAg Positive Population))	tjrndswhdl(znktzhmian) = wowhnpfgqq vublkbevnn (wvrdwtmhum, bizmiwvvlc - vujosiajpv) View more	-	26 Aug 2022
				Placebo+ETV (Placebo Plus ETV (HBeAg Positive Population))	tjrndswhdl(znktzhmian) = hkpafxejym vublkbevnn (wvrdwtmhum, oiwzcipwgm - psbakpdkvc) View more
EASL2021	Phase 1/2	Hepatitis B	-	Vebicorvir (VBR)	qvvlnzhuos(zbdjgmwwac) = zthnytpnne mmrqggpobs (yguknuswys ) View more	-	23 Jun 2021
				ABI-H2158 (2158)	qvvlnzhuos(zbdjgmwwac) = oxaqkfmqko mmrqggpobs (yguknuswys ) View more

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ABI-H2158

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