Core Insights - Assembly Biosciences, Inc. announced positive topline results from a Phase 1b study of ABI-4334, demonstrating favorable safety, tolerability, and pharmacokinetics for once-daily oral dosing in chronic hepatitis B virus (HBV) infection [1][3][4] Efficacy and Safety - In the 400 mg cohort, mean plasma HBV DNA reductions of 3.2 logs IU/mL were observed over 28 days, consistent with the 150 mg cohort, indicating effective viral replication inhibition [1][2][10] - ABI-4334 showed a favorable safety profile with no serious adverse events reported, and only two grade three treatment-emergent lab abnormalities were noted, both of which resolved [8][9] Pharmacokinetics - The pharmacokinetic data indicated that ABI-4334 maintained exposure levels significantly above those required for potent antiviral activity and inhibition of cccDNA formation, supporting its once-daily dosing regimen [4][10] - Slightly higher than dose-proportional increases in clinical pharmacokinetic exposures were observed from the 150 mg to the 400 mg dose [10] Collaboration and Future Steps - The completion of the trial triggers an opt-in point for Gilead Sciences, Inc. to review the option data package for potential exclusive licensing of ABI-4334 for further development and commercialization [1][5] - Discussions regarding the next steps for ABI-4334 will be supported by the trial results as Gilead evaluates its options [3][5]

Assembly Biosciences (ASMB) 2025 Conference June 04, 2025 08:45 AM ET Speaker0 Good morning everyone. Welcome to the Jefferies Healthcare Conference. My name is Anthea Lee, part of the Healthcare Research Team here at Jefferies. We're very fortunate to have Assembly Biosciences with us today. We have Jason Okazaki, CEO, and CMO Anuj Ghajar. Welcome. For those that are new to the story, perhaps we just start with a quick overview of the company, the pipeline and key catalysts that you see coming up for the r ...

Company Overview - Assembly Biosciences, Inc. is a biotechnology company focused on developing innovative small-molecule therapeutics aimed at serious viral diseases, including herpesvirus, hepatitis B virus (HBV), and hepatitis delta virus (HDV) infections [2] Upcoming Event - Jason Okazaki, CEO, and Anuj Gaggar, CMO, will present in a fireside chat at the Jefferies 2025 Global Healthcare Conference on June 4, 2025, at 8:45 a.m. Eastern Time [1] - A live webcast of the event will be available on Assembly Bio's website, with a replay accessible afterward [1] Leadership and Commitment - The company is led by a team experienced in virologic drug development, demonstrating a commitment to improving patient outcomes affected by chronic viral infections [2]

Core Viewpoint - Assembly Biosciences reported a quarterly loss of $1.17 per share, significantly better than the Zacks Consensus Estimate of a loss of $2.01, indicating a positive earnings surprise of 41.79% [1] Financial Performance - The company posted revenues of $9.42 million for the quarter ended March 2025, exceeding the Zacks Consensus Estimate by 21.38%, compared to $5.79 million in the same quarter last year [2] - Over the last four quarters, Assembly Biosciences has surpassed consensus EPS estimates four times and topped consensus revenue estimates three times [2] Stock Performance - Assembly Biosciences shares have declined approximately 21.4% since the beginning of the year, contrasting with the S&P 500's decline of 4.3% [3] - The company's current Zacks Rank is 2 (Buy), suggesting that the shares are expected to outperform the market in the near future [6] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$1.59 on revenues of $8.19 million, and for the current fiscal year, it is -$6.78 on revenues of $33.7 million [7] - The trend of estimate revisions for Assembly Biosciences is currently favorable, which could influence future stock movements [5][6] Industry Context - The Medical - Generic Drugs industry, to which Assembly Biosciences belongs, is currently ranked in the top 18% of over 250 Zacks industries, indicating a strong industry performance potential [8]

PART I: FINANCIAL INFORMATION [Financial Statements](index=4&type=section&id=Item%201.%20Financial%20Statements) The company reported a reduced net loss in Q1 2025, with assets decreasing and collaboration revenue increasing Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $23,413 | $38,344 | | Marketable securities | $67,615 | $73,735 | | Total Assets | $99,017 | $119,168 | | Deferred revenue (short & long-term) | $63,581 | $73,000 | | Total Liabilities | $71,884 | $85,809 | | Total Stockholders' Equity | $27,133 | $33,359 | Condensed Consolidated Statements of Operations Highlights (in thousands, except per share data) | Account | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Collaboration revenue | $9,419 | $5,785 | | Research and development | $14,851 | $11,879 | | General and administrative | $4,509 | $4,635 | | Loss from operations | $(9,941) | $(10,729) | | Net loss | $(8,818) | $(9,077) | | Net loss per share, basic and diluted | $(1.17) | $(1.66) | Condensed Consolidated Statements of Cash Flows Highlights (in thousands) | Activity | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(23,439) | $(18,364) | | Net cash provided by investing activities | $6,586 | $17,272 | | Net cash provided by financing activities | $1,922 | $— | | Net decrease in cash and cash equivalents | $(14,931) | $(1,092) | [Notes to Financial Statements](index=8&type=section&id=Notes%20to%20the%20Condensed%20Consolidated%20Financial%20Statements%20(unaudited)) Notes detail the company's biotech focus, financial sufficiency, and key Gilead collaboration terms - The company is a biotechnology firm developing therapeutics for viral diseases including genital herpes, hepatitis B (HBV), hepatitis D (HDV), and transplant-related herpesviruses[19](index=19&type=chunk) - Management believes current funds are sufficient to meet operating requirements for **at least the next twelve months** from the report's issuance date[20](index=20&type=chunk) - The collaboration with Gilead is a significant component of operations. In Q1 2025, the company recognized **$9.4 million** in collaboration revenue, up from **$5.8 million** in Q1 2024[32](index=32&type=chunk)[58](index=58&type=chunk) - The transaction price for the Gilead agreement was amended to **$74.2 million**, consisting of the unrecognized portion of the original contract, a **$5.2 million** premium on a stock purchase, and a **$10.0 million** payment from an amendment[54](index=54&type=chunk) - During Q1 2025, the company sold **161,645 shares** of common stock under its "at-the-market" (ATM) offering, receiving net proceeds of **$1.9 million**[41](index=41&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=18&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses clinical pipeline progress, increased revenue and R&D, and future funding outlook - The company's pipeline includes clinical-stage therapies for recurrent genital herpes (HPIs), hepatitis delta virus (HDV entry inhibitor), and hepatitis B virus (HBV CAM), as well as a program for transplant-related herpesviruses[62](index=62&type=chunk) - The company expects its existing cash, cash equivalents, and marketable securities will fund operating expenses and capital expenditure requirements into the **middle of 2026**[122](index=122&type=chunk) R&D Expense Breakdown (in thousands) | Program | Q1 2025 | Q1 2024 | $ Change | | :--- | :--- | :--- | :--- | | 5366 (Herpes) | $1,680 | $377 | $1,303 | | 1179 (Herpes) | $734 | $1,040 | $(306) | | 6250 (HDV) | $2,445 | $1,027 | $1,418 | | 4334 (HBV) | $426 | $553 | $(127) | | 7423 (Transplant) | $752 | $— | $752 | | **Total R&D Expenses** | **$14,851** | **$11,879** | **$2,972** | - Net cash used in operating activities increased to **$23.4 million** in Q1 2025 from **$18.4 million** in Q1 2024, primarily due to increased operating expenses from advancing the clinical pipeline[119](index=119&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=33&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section is not applicable for the registrant, as it qualifies as a smaller reporting company - The company has indicated that this item is **not applicable**[126](index=126&type=chunk) [Controls and Procedures](index=33&type=section&id=Item%204.%20Controls%20and%20Procedures) Management evaluated disclosure controls and procedures, concluding they were effective with no material changes - The Chief Executive Officer and President concluded that the company's disclosure controls and procedures were **effective** at a reasonable assurance level as of the end of the period[127](index=127&type=chunk) - **No changes** in internal control over financial reporting occurred during the quarter ended March 31, 2025, that materially affected, or are reasonably likely to materially affect, internal controls[128](index=128&type=chunk) PART II: OTHER INFORMATION [Legal Proceedings](index=34&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings - The company is **not a party to any material legal proceedings**[130](index=130&type=chunk) [Risk Factors](index=34&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks from unapproved products, financial needs, and third-party and IP dependencies - The company has **no approved products** and its future success depends on obtaining regulatory approval for its pipeline candidates, which is uncertain[132](index=132&type=chunk) - The company has a history of **losses** and will need **additional financing** to complete development. Failure to raise capital could force it to scale back or discontinue operations[137](index=137&type=chunk)[138](index=138&type=chunk) - The collaboration with Gilead is **critical**. Risks include potential conflicts, Gilead choosing not to exercise its options, and dependence on Gilead for development and commercialization of optioned programs[140](index=140&type=chunk)[142](index=142&type=chunk) - The company relies on **third-party CROs and manufacturers**, which reduces control over clinical studies and manufacturing and introduces risks related to performance, compliance, and supply chain interruptions[146](index=146&type=chunk)[153](index=153&type=chunk) - The business depends on **protecting its intellectual property** through patents and trade secrets, but these protections may be inadequate, challenged by third parties, or difficult to enforce globally[190](index=190&type=chunk)[191](index=191&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=55&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) There were no unregistered sales of equity securities during the period covered by this report - **None**[208](index=208&type=chunk) [Exhibits](index=56&type=section&id=Item%206.%20Exhibits) The report includes exhibits such as the CEO's certifications pursuant to Sections 302 and 906 of the Sarbanes-Oxley Act of 2002, as well as Inline XBRL documents - Filed exhibits include **CEO certifications (31.1, 32.1) and Inline XBRL data files (101.INS, 101.SCH, 104)**[212](index=212&type=chunk)

[Company Overview and Pipeline Update](index=1&type=section&id=Assembly%20Biosciences%20Reports%20First%20Quarter%202025%20Financial%20Results%20and%20Recent%20Updates) This section outlines the company's strategic focus and clinical pipeline updates, including key milestones and data readouts [Pipeline Progress and Anticipated Milestones](index=1&type=section&id=First%20Quarter%202025%20and%20Recent%20Highlights) Assembly Biosciences reports strong progress in its antiviral portfolio, with four clinical studies on track for key data readouts in 2025 - The company is on track to deliver key clinical data sets for four candidates in 2025, reinforcing progress in its antiviral portfolio[1](index=1&type=chunk)[2](index=2&type=chunk) - Key upcoming data readouts include: - **Recurrent Genital Herpes (ABI-5366 & ABI-1179):** Interim efficacy, safety, and PK data from Phase 1b studies are expected in fall 2025[5](index=5&type=chunk) - **Hepatitis D Virus (HDV - ABI-6250):** Data from a Phase 1a study in healthy participants is anticipated in Q3 2025[9](index=9&type=chunk) - **Hepatitis B Virus (HBV - ABI-4334):** Efficacy, safety, and PK data from the 400 mg cohort in a Phase 1b study are expected in the first half of 2025[7](index=7&type=chunk) [Financial Results](index=2&type=section&id=First%20Quarter%202025%20Financial%20Results) This section details Q1 2025 financial performance, covering revenue, expenses, net loss, and the company's cash and funding outlook [Q1 2025 Financial Performance](index=2&type=section&id=First%20Quarter%202025%20Financial%20Performance) Q1 2025 saw collaboration revenue increase by 62% to $9.4 million, with a net loss of $8.8 million, an improvement from the prior year Q1 2025 vs Q1 2024 Statement of Operations Highlights (in millions, except per share data) | Financial Metric | Q1 2025 | Q1 2024 | Change | | :--- | :--- | :--- | :--- | | Collaboration Revenue | $9.4 | $5.8 | +62.1% | | R&D Expenses | $14.9 | $11.9 | +25.2% | | G&A Expenses | $4.5 | $4.6 | -2.2% | | Net Loss | $(8.8) | $(9.1) | Improvement | | Net Loss Per Share | $(1.17) | $(1.66) | Improvement | - The increase in collaboration revenue reflects higher R&D activity and increased funding from the amended Gilead agreement[10](index=10&type=chunk) - The rise in R&D expenses was largely driven by increased spending on the ABI-6250 (HDV) and ABI-5366 (herpes) programs[10](index=10&type=chunk) [Financial Position and Outlook](index=2&type=section&id=Financial%20Position) As of March 31, 2025, the company held $91.0 million in cash and marketable securities, projected to fund operations into mid-2026 - The company's cash, cash equivalents, and marketable securities totaled **$91.0 million** as of March 31, 2025[10](index=10&type=chunk) - Management projects the current cash position will fund operations into mid-2026[10](index=10&type=chunk) Balance Sheet Highlights (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $23,413 | $38,344 | | Marketable securities | $67,615 | $73,735 | | **Total Cash & Marketable Securities** | **$91,028** | **$112,079** | | Total Assets | $99,017 | $119,168 | | Total Liabilities | $71,884 | $85,809 | | Total Stockholders' Equity | $27,133 | $33,359 | [Financial Statements](index=5&type=section&id=Financial%20Statements) This section presents the unaudited condensed consolidated financial statements, including balance sheets and statements of operations [Condensed Consolidated Balance Sheets](index=5&type=section&id=ASSEMBLY%20BIOSCIENCES,%20INC.%20CONDENSED%20CONSOLIDATED%20BALANCE%20SHEETS) This section presents the unaudited condensed consolidated balance sheets as of March 31, 2025, and December 31, 2024 Condensed Consolidated Balance Sheets (In thousands) | | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **ASSETS** | | | | Total current assets | $95,527 | $115,503 | | Total assets | $99,017 | $119,168 | | **LIABILITIES AND STOCKHOLDERS' EQUITY** | | | | Total current liabilities | $46,631 | $47,803 | | Total liabilities | $71,884 | $85,809 | | Total stockholders' equity | $27,133 | $33,359 | | Total liabilities and stockholders' equity | $99,017 | $119,168 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=ASSEMBLY%20BIOSCIENCES,%20INC.%20CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20OPERATIONS%20AND%20COMPREHENSIVE%20LOSS) This section provides the unaudited condensed consolidated statements of operations and comprehensive loss for Q1 2025 and Q1 2024 Condensed Consolidated Statements of Operations and Comprehensive Loss (In thousands, except per share data) | | Three Months Ended March 31, | | :--- | :--- | :--- | | | **2025** | **2024** | | Collaboration revenue from a related party | $9,419 | $5,785 | | Total operating expenses | $19,360 | $16,514 | | Loss from operations | $(9,941) | $(10,729) | | Net loss | $(8,818) | $(9,077) | | Comprehensive loss | $(8,860) | $(9,235) | | Net loss per share, basic and diluted | $(1.17) | $(1.66) | | Weighted average common shares outstanding | 7,506,321 | 5,483,313 |

Core Insights - Assembly Biosciences, Inc. is progressing well with its antiviral portfolio, expecting key clinical data sets in 2025, including interim Phase 1b data for ABI-5366 and ABI-1179 in fall 2025 [2][3] Financial Results - As of March 31, 2025, the company reported cash, cash equivalents, and marketable securities totaling $91.0 million, down from $112.1 million as of December 31, 2024, with projections to fund operations into mid-2026 [12] - Revenue from collaborative research with Gilead was $9.4 million for Q1 2025, an increase from $5.8 million in Q1 2024, attributed to increased research and development spending and collaboration funding [12] - Research and development expenses rose to $14.9 million in Q1 2025 from $11.9 million in Q1 2024, primarily due to increased spending on ABI-6250 and ABI-5366 [12] - General and administrative expenses slightly decreased to $4.5 million in Q1 2025 from $4.6 million in Q1 2024 [12] - The net loss attributable to common stockholders was $8.8 million, or $1.17 per share, for Q1 2025, compared to a net loss of $9.1 million, or $1.66 per share, for the same period in 2024 [12] Clinical Development Updates - Ongoing clinical studies for four candidates are on track, with data expected in 2025, including interim Phase 1b data for ABI-5366 and ABI-1179 anticipated in fall 2025 [1][4] - The company plans to run concurrent studies for ABI-5366 and ABI-1179, evaluating weekly and monthly oral dosing in participants with recurrent genital herpes over a 28-day period [8] - In Q3 2025, data from a Phase 1a study in healthy participants for ABI-6250 will be assessed for safety and pharmacokinetic measures [8] - Efficacy, safety, and pharmacokinetic data from a Phase 1b study for ABI-4334 in chronic HBV infection is expected in the first half of 2025 [8] Upcoming Conferences - Assembly Biosciences presented multiple abstracts at various conferences, including ICAR, ESCMID, and EASL in 2025, showcasing data on its antiviral candidates [8]

Core Insights - Assembly Biosciences, Inc. presented new preclinical and in vitro data for two therapeutic candidates, ABI-6250 and ABI-4334, at the EASL Congress, highlighting their potential in treating chronic viral hepatitis [1][2] Group 1: ABI-6250 - ABI-6250 is a first-in-class oral therapeutic candidate for chronic hepatitis D virus (HDV) infection, currently in Phase 1a clinical study [3] - Preclinical results show ABI-6250 specifically inhibits both HDV and hepatitis B virus (HBV) at low nanomolar levels, with minimal effects on cell viability across multiple cell types [4] - The mechanism of ABI-6250 involves blocking the sodium taurocholate cotransporting polypeptide (NTCP), a validated target for HDV infection, which is crucial as current treatments for chronic HDV are limited [5] Group 2: ABI-4334 - ABI-4334 is a next-generation capsid assembly modulator (CAM) for HBV, with in vitro studies demonstrating sustained inhibition of HBV replication and HBsAg levels after long-term treatment in human hepatocytes [6] - The World Health Organization estimates that 254 million people are chronically infected with HBV, leading to significant health issues, highlighting the need for innovative treatments like ABI-4334 [7] Group 3: Company Overview - Assembly Biosciences is focused on developing innovative small-molecule therapeutics aimed at serious viral diseases, particularly targeting herpesvirus, HBV, and HDV infections [9]

Company Overview - Assembly Biosciences (ASMB) shares increased by 24% to close at $14.18, with notable trading volume compared to typical sessions, and a 22.9% gain over the past four weeks [1][2] Development Pipeline - The company is investigating four developmental candidates in early-stage studies targeting chronic infections such as herpesvirus, hepatitis B virus, and hepatitis delta virus, with several clinical data readouts expected in 2025 [2] Financial Expectations - Assembly Biosciences is projected to report a quarterly loss of $2.01 per share, reflecting a year-over-year decline of 21.1%, while revenues are anticipated to be $7.76 million, marking a 34% increase from the previous year [3] Earnings Estimate Trends - The consensus EPS estimate for the upcoming quarter has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Assembly Biosciences is part of the Zacks Medical - Generic Drugs industry, where another company, Taysha Gene Therapies, Inc. (TSHA), saw a 7.1% increase in its stock price, closing at $2.58, with a remarkable 91.3% return over the past month [4]

Core Insights - Assembly Biosciences, Inc. is developing ABI-5366, a long-acting helicase-primase inhibitor for recurrent genital herpes, with interim proof-of-concept data expected in fall 2025 [1][5] - The company presented data on ABI-5366 at the 2025 Congress of the European Society of Clinical Microbiology and Infectious Diseases, highlighting its potential as a once-weekly or once-monthly oral treatment option [1][2][8] Clinical Data - ABI-5366 was well tolerated in a Phase 1a study, with no serious adverse events reported at doses up to 350 mg, and a half-life of approximately 20 days supports infrequent dosing [3][4] - Preclinical studies demonstrated broad activity against HSV-1 and HSV-2, reinforcing the potential for weekly or monthly oral dosing [4] Genital Herpes Prevalence and Treatment Patterns - A retrospective analysis estimated 262,457 total genital herpes cases and 148,067 recurrent cases in 2023 among healthcare-engaged individuals in the U.S., with a projected 1.35 million individuals experiencing recurrent genital herpes nationwide [6][7] - Among recurrent cases, 97% received pharmacologic treatment, with 57% on suppressive therapy, indicating a significant opportunity for improved treatment strategies [7] Company Overview - Assembly Biosciences focuses on innovative therapeutics for serious viral diseases, including herpesvirus and hepatitis infections, aiming to enhance patient outcomes [12]